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Drone-Enhanced Emergency Medical Services: Redefining Ground Zero in Medical Emergencies

Written By: Priya Bhaware, M.Pharm Pharmacology

Reviewed and Fact-Checked By: Vikas Londhe, M.Pharm Pharmacology

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Source: Freepik.com

Discover how drones are transforming emergency medical services with faster response times, real-world success stories like Rwanda’s Zipline, and the science behind drone-assisted critical care delivery

Introduction: A Race against Time

Imagine a rural village nestled deep in the hills, 30 miles from the nearest hospital. A 45-year-old farmer collapses from a sudden cardiac arrest. His family called the local emergency helpline, but due to poor roads and distance, the ambulance would take over 45 minutes. His survival window is less than 10 minutes. But something extraordinary happens within 4 minutes: a drone appears in the sky, delivering an automated external defibrillator (AED) to the family. Trained via mobile video instructions, they use the AED, and the man’s heartbeat is revived.

This is not science fiction; it is the future of emergency medical care, powered by drones.

In emergencies, rapid access to life-saving medical supplies can mean the difference between life and death. Countless lives are lost each year during the crucial minutes immediately following a medical crisis. Every minute saved in delivering help can increase the chance of survival by up to 10%.

Drones are changing the landscape of emergency medical services (EMS) by offering a faster, more efficient, and cost-effective channel of delivering assistance in medical emergencies. A drone’s ability to be set out in a flash enables it to reach medical emergency ground zero quickly, facilitating rapid assessment, locating patients, and delivering essential medical supplies and equipment, often overtaking traditional response methods. This improved response time can significantly enhance patient outcomes and sometimes save the life of the patient, especially in remote or underserved areas.

Drone Enhanced Medical Emergency Services

Drone Emergency Medical Services (DEMS) use highly independent Beyond Visual Line of Sight (BVLOS) drones to speedily deliver vital medical supplies such as Automated External Defibrillators (AEDs), critical medications, and diagnostic equipment directly to medical emergency locations such as cardiac arrest, stroke, postpartum hemorrhage, anaphylaxis, or trauma, where even a few minutes’ delay can cost lives.

Traditional emergency services are while life-saving but face several limitations, like traffic delays in urban settings or poor road infrastructure in rural regions, slow ambulance dispatch times, and limited reach in remote or disaster-prone areas.

Unmanned Aerial Vehicles (UAVs), commonly known as drones, are emerging as vital tools in healthcare logistics. Capable of bypassing traffic and terrain-related barriers, they can access both densely populated urban centers and hard-to-reach rural locations. The need for faster and more efficient emergency responses, especially in underserved or isolated areas, highlights the expanding role of drones in enhancing emergency medical services (EMS).

Drones can be deployed within seconds to deliver critical medical equipment ahead of ambulance arrival, while also transmitting real-time aerial footage to enhance situational awareness and support informed decision-making. In large-scale disasters or mass casualty incidents, they play a crucial role in coordinating resources and minimizing exposure risks for emergency personnel

Clinical Trials and Pilot Studies: Evidence behind the Buzz

Karolinska Institute, Sweden: Out-of-hospital cardiac arrest (OHCA) trial

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One of the most influential studies in this field was published in the New England Journal of Medicine (NEJM) in December 2022, titled “Drone Delivery of an Automated External Defibrillator.” Conducted in Sweden, this interventional trial evaluated the effectiveness of drones delivering AEDs (Automated External Defibrillators) in out-of-hospital cardiac arrest (OHCA) scenarios. The study involved 18 actual cardiac arrest cases in which drones were dispatched in parallel with emergency medical services. In 64% of these cases, the drones arrived before ambulances, achieving a median time saving of 2 minutes and 15 seconds—a significant reduction considering that survival from cardiac arrest drops by 7–10% for every minute without defibrillation. In three instances, bystanders successfully retrieved and used the drone-delivered AEDs before EMS arrived, marking a critical advancement in community-assisted emergency care. Importantly, no safety incidents were reported, proving the operational feasibility of drone delivery in populated areas.

U.S. FAA and WakeMed Hospital (North Carolina)

In the United States, a notable pilot project by WakeMed Hospital in North Carolina, in collaboration with UPS Flight Forward and Matternet, focused on transporting laboratory samples via drones. Over 2,000 successful drone flights were completed, demonstrating a 30–40 minute reduction in turnaround time for diagnostic tests a crucial factor in speeding up emergency treatment decisions, especially in stroke, sepsis, or trauma cases.

Zipline’s Internal Data (Rwanda & Ghana)

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Internationally, perhaps the most impactful real-world data comes from Zipline, a drone logistics company operating in Rwanda and Ghana. Though not part of a randomized clinical trial, Zipline has released comprehensive operational metrics. The drones have completed over 1 million deliveries, primarily of blood products, vaccines, antivenoms, and emergency medications, often to hard-to-reach rural clinics. Their data show a 75% reduction in delivery time compared to traditional ground transportation. In Rwanda, these drones have been especially critical in delivering blood for postpartum hemorrhage a leading cause of maternal mortality helping save thousands of lives by eliminating fatal delays.

India—Medicine from the Sky Project

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In India, the government-led initiative “Medicine from the Sky” launched in Telangana and Himalayan regions and also tested the use of drones in delivering vaccines and emergency medications to remote tribal areas. This pilot, supported by the World Economic Forum and private healthcare providers, proved that drones could reduce delivery time by 20–30 minutes per mission and operate effectively across difficult terrain. Healthcare workers reported high levels of trust in the technology, and the pilot was deemed a success in improving access to urgent care supplies.

Step By Step Process of Drone Delivery

1. Emergency call triggered: When an emergency call (911/112) is made, both the Drone-Enhanced Emergency Medical Services (DEMS) and traditional EMS are activated simultaneously, ensuring a swift and coordinated response for faster on-site assistance.

2. Drone dispatched immediately: Within seconds, a DEMS drone is launched, carrying essential life-saving medical supplies such as defibrillators, haemorrhage control kits, or Naloxan. Its dynamic payload system allows for efficient customization based on the specific emergency, ensuring the right tools reach the scene as quickly as possible.

3. Arrival within 2-3 minutes: The DEMS drone reaches the emergency scene within 2–3 minutes—far faster than conventional EMS vehicles. It delivers critical equipment directly to bystanders or citizen responders on-site, empowering them to take immediate, potentially life-saving action before professional help arrives.

4. Real-time guidance provided: Using live video feeds and two-way communication, dispatchers guide bystanders through the process of administering life-saving actions, such as CPR, defibrillation, or the application of anti-bleeding kits. This ensures intervention is effective before EMS teams arrive.

5. EMS arrival and follow-up: Traditional EMS teams usually arrive within 9–10 minutes; however, by that time, crucial life-saving measures have often already been initiated by bystanders, guided by dispatchers, and equipped by DEMS drones. This early intervention greatly increases the patient’s chances of survival

Challenges

Airspace regulations and FAA compliance

Limited payload capacity (3-5 kg)

Battery life and weather limitations

Need for real-time coordination with EMS

Conclusion: A Sky Route for Saving Lives

Drones are revolutionizing emergency medical services by enabling faster, more efficient delivery of critical aid. They significantly reduce response times, especially in remote or congested areas, and improve patient outcomes through early intervention. Drones are not replacing emergency medical services they are augmenting them, creating a hybrid model where technology bridges critical care gaps in time and location. From Rwanda’s blood drops to India’s tribal medicine delivery, drones have ascended from concept to clinical impact.

As infrastructure, regulations, and technology evolve, drone-enhanced EMS is poised to become the new norm in global emergency care delivering not just medicines, but hope and life.

References

Revolutionized Healthcare: Drone Based Medical Services for Remote Healthcare and Emergency Response, available from https://www.aerogo.live/post/revolutionized-healthcare-drone-based-medical-services-for-remote-healthcare-and-emergency-response#:~:text=Here’s%20how%20drones%20are%20transforming,and%20enhance%20emergency%20response%20capabilities.

Sanz-Martos S, Lopez-Franco MD, Álvarez-García C, Granero-Moya N, Lopez-Hens JM, Camara-Anguita S, Pancorbo-Hidalgo PL, Comino-Sanz IM. Drone applications for emergency and urgent care: a systematic review. Prehospital and disaster medicine. 2022 Aug;37(4):502-8. DOI: 10.1017/S1049023X22000887

Habibi S, Ivaki N, Barata J. A Systematic Literature Review of Unmanned Aerial Vehicles for Healthcare and Emergency Services. arXiv preprint arXiv:2504.08834. 2025 Apr 10. https://doi.org/10.48550/arXiv.2504.08834

Schaerbeek S, Svensson L, Claesson A. Use of a drone-delivered automated external defibrillator in an out-of-hospital cardiac arrest. New England Journal of Medicine. 2022 May 19; 386(20):1953-4. Doi:10.1056/NEJMc2200833

Aggarwal S, Gupta P, Mahajan N, Balaji S, Singh KJ, Bhargava B and Panda S (2023) Implementation of drone based delivery of medical supplies in North-East India: experiences, challenges and adopted strategies. Front. Public Health 11:1128886. Doi: 10.3389/fpubh.2023.1128886Sharma S, Sharma H. Drone a technological leap in health care delivery in distant and remote inaccessible areas: A narrative review. Saudi J Anaesth. 2024 Jan-Mar;18(1):95-99. Doi: 10.4103/sja.sja_506_23. Epub 2024 Jan 2. PMID: 38313723; PMCID: PMC10833029.

Amukele, Timothy, Using drones to deliver blood products in Rwanda, The Lancet Global Health, Volume 10, Issue 4, e463 – e464

Rwanda signs agreement with zipline to use drones for delivery of essential medical products https://www.minict.gov.rw/news-detail/rwanda-signs-agreement-with-zipline-to-use-drones-for-delivery-of-essential-medical-products

Hospital Uses FAA-Approved Drones for the First Time, he American Society of Mechanical Engineers.

Medicine from the Sky: Community Outcomes of Drone Deliveries in the Himalayan Region, World Economic Forum

 https://www3.weforum.org/docs/WEF_Medicine_from_the_Sky_2024.pdf

Step-by-step breakdown of DEMS, https://dems.se/

 

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Triphala: The Timeless Ayurvedic Elixir for Detox, Digestion & Rejuvenation

Medically Written and Reviewed By: Ayurvedacharya Dr. Gaurav Pathare (BAMS)

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Source: Triphala Powder Stock photos/Vecteezy.com

Triphala (त्रिफला) is a legendary polyherbal blend in Ayurveda, cherished for its detoxifying, rejuvenating, and healing properties. This ancient remedy combines the dried fruits of Haritaki (Terminalia chebula), Bibhitaka (Terminalia bellirica), and Amalaki (Emblica officinalis) each known for its own powerful benefits. Used for over 3,000 years in traditional Indian medicine, this herbal blend is classified as a Rasayana (rejuvenative tonic) known for promoting overall health and longevity. Modern research has validated its traditional uses, revealing a broad spectrum of health benefits that make it valuable in contemporary wellness practices.

What is Triphala?

One of the biggest misconceptions about Triphala is that it is a single plant. In fact, it is a traditional Ayurvedic formulation made from a blend of three dried fruits: Amalaki (Emblica officinalis), Bibhitaki (Terminalia bellirica), and Haritaki (Terminalia chebula).

Triphala means “three fruits” and is described in classical texts like Bhavprakash:

पथ्याविभीतकधात्रीणां फलैः संयत्र त्रिफला स्मृता
फलवृत्तिकां त्रिफला सा वरा प्रकीर्तिता

– Bhavprakash

Which means the combination of the fruits of Haritaki (Pathya), Bibhitaka (Vibhitaka), and Amalaki (Dhatri) is known as Triphala. This fruit-based formulation is also called Phalavritti (composed of fruits) and is revered as Vara (the best or excellent one).”

According to tradition, the ideal combination is one Haritaki, two Bibhitaka, and four Amalaki balanced by weight for maximum efficacy. This is explained in the Bhavprakash:

एका हरीतकी योज्या द्वी योज्यी च विभीतकी। चरिाच्यामलकावन च….. ।।

– Bhavprakash

Triphala’s Ayurvedic Profile

Property

 Description

Rasa (Taste)

 Five tastes except salty; mainly astringent

Vipaka

Madhura (Sweet post-digestion)

Veerya

Ushna (Hot potency)

Prabhava

Virechana (Mild laxative)

Dosha Action

Tridoshahara is best for Kapha disorders

Top Health Benefits of Triphala

त्रिफला कफवातघ्नी मेहकुष्ठहरा सरा
चक्षुष्या दीपनी रुच्या विषमज्वरनाशिनी

Bhavprakash

Which means, Triphala destroys Kapha and Vata doshas.
It is beneficial in Meha (urinary disorders, including diabetes) and Kushta (skin diseases).
Acts as a mild laxative (Sara),
Improves eyesight (Chakshushya)
Stimulates digestive fire (Dipani),
Enhances taste (Ruchya), and
Cures intermittent fevers (Vishamajvara).

Balances Kapha and Vata (कफवातघ्नी)

Triphala helps balance Kapha and Vata, two of the primary doshas in Ayurveda. Excess Kapha leads to heaviness, congestion, and sluggish digestion. Vata imbalance causes dryness, anxiety, and bloating. Triphala’s drying (rooksha) and warming (ushna) qualities counter these imbalances effectively. Its tridoshic nature makes it suitable for long-term, gentle balancing without side effects.

Useful in Meha (Urinary Disorders & Diabetes) (मेहहरा)

The term Meha encompasses a range of urinary issues, including frequent urination and diabetes. Triphala acts as a Pramehaghna, detoxifying urinary channels and improving renal function. It’s bitter and astringent taste tones tissues and helps control sugar metabolism. It reduces excess moisture (kleda) and normalizes urine flow, a key aspect in diabetes care.

Effective in Kushta (Skin Diseases) (कुष्ठहरा)

Triphala cleanses toxins (ama) from the blood and lymph, the root cause of many skin disorders. It is particularly beneficial in chronic conditions like eczema, psoriasis, and even leprosy. Its rooksha (drying) and kledaghna (anti-exudative) actions reduce skin inflammation and discharge. Regular use promotes clear, healthy, and balanced skin from the inside out.

Acts as a Natural Laxative (Saraa – सरा)

Triphala gently stimulates the bowels without causing dependency or dehydration. It supports regular elimination, especially in cases of mild constipation (malavashtambha). Its virechana property helps cleanse the digestive tract and supports detox. Unlike harsh purgatives, Triphala works harmoniously with the body’s natural rhythm.

Improves Vision (Chakshushya – चक्षुष्या)

Triphala is highly valued for maintaining eye health and clarity of vision. Its antioxidant-rich fruits nourish the optic nerves and tissues. Used internally and in formulations like Triphalaghrita, it supports relief in eye fatigue, redness, and infection. In Ayurveda, it is a key remedy for netra rogas (eye disorders), especially with Kapha involve men.

Stimulates Digestion (Dipani – दीपनी)

Triphala kindles the digestive fire (Agni), improving assimilation and metabolism.
Its bitter and pungent tastes reduce sluggishness caused by Kapha. By promoting digestion, it helps prevent the formation of toxins (ama). It is especially effective for those with mandagni (weak digestion).

Enhances Taste Perception (Ruchya – रुच्या)

Loss of taste (aruchi) is often linked to indigestion and toxin buildup. Triphala cleanses the tongue and gut, restoring natural appetite and flavor perception. Its astringent and bitter elements stimulate salivary and gastric secretions. This makes it useful during recovery from illness or post-antibiotic therapy.

Relieves Intermittent Fevers (Vishamajvara – विषमज्वरनाशिनी)

Vishamajvara refers to fevers that come and go irregularly, often due to hidden toxins. Triphala performs pachana (digestive purification) and shodhana (systemic cleansing) of the doshas. It targets the underlying imbalances causing the fever rather than just the symptom. Thus, it supports natural immune function and systemic recovery.

When to take?

To get the maximum health benefits from Triphala, timing, form, and method of intake are crucial and should align with your health goal and dosha balance. Here’s a clear, Ayurveda-based guide:

Health Goal

Best Time to Take

Effect

Constipation, Detox

At bedtime

Overnight cleansing, gentle laxative

Weight Loss, Fat Metabolism

Early morning (empty stomach)

Activates metabolism, burns fat

Eye health, Skin benefits

Consistently at night

Enhances tissue repair overnight

Diabetes, Blood Sugar

Morning + Night (low dose)

Supports sugar balance and urination

General Rejuvenation

Daily, the night before sleep

Rasayana (anti-aging) and immunity

How to Take Triphala

Triphala Churna (Traditional and most effective

Dosage: 1 to 3 grams (¼ to ½ teaspoon)

How: Mix in warm water (Koshnajala) and drink

When: 30 minutes before bed or early morning on an empty stomach

Optional: Add a few drops of honey or lemon if the taste is too bitter

 

Triphala Vati (Convenient

Dosage: 1–2 tablets (250–500 mg each)

How: Swallow with warm water

When: Same timing as powder, night preferred

Triphala Infusion (Herbal Tea)

Method: Soak 1 tsp. of Triphala powder in a glass of warm water overnight. Strain and drink the next morning.

Benefit: Mild, long-acting detox and weight-balancing effect

Triphala Ghrita (Ghee) – For Eye & Nerve Health

Use: Internally or externally under supervision

Benefit: Soothes eye disorders, nourishes the nervous system

 

Safe for Regular Use

Yes! Triphala is gentle and non-habit-forming. It doesn’t create dependency like harsh laxatives. But always consult a certified Ayurvedic practitioner for long-term or high-dose usage.

Conclusion: A Must-Have Ayurvedic Superblend

From the ancient Ayurvedic sages to modern wellness enthusiasts, Triphala continues to be a holistic powerhouse for health. Whether you seek detoxification, digestion, glowing skin, better vision, or disease prevention, Triphala has something for everybody’s type (Prakriti). Add this tridoshic marvel to your daily routine and experience the balance of Ayurveda in its purest form.

References

Textbook of Dravya Guna Vidnyan Prof Dr A.P Deshpande Second edition December2007 Mishrak gana page 183 -185

Bali chouhan, Ramesh Chandra Kumawat, Mita Kotecha, A. Ramamurthy, Sumit Nathani. Triphala: A comprehensive Ayurvedic review. Int. J. Res. Ayurveda Pharm. 2013;4(4):612-617 http://dx. doi.org/10.7897/2277-4343.04433

Peterson CT, Denniston K, Chopra D. Therapeutic Uses of Triphala in Ayurvedic Medicine. J Altern Complement Med. 2017 Aug;23(8):607-614. doi: 10.1089/acm.2017.0083. Epub 2017 Jul 11. PMID: 28696777; PMCID: PMC5567597.

Jantrapirom S, Hirunsatitpron P, Potikanond S, Nimlamool W and Hanprasertpong N (2021) Pharmacological Benefits of Triphala: A Perspective for Allergic Rhinitis. Front. Pharmacol. 12:628198. doi: 10.3389/fphar.2021.628198

Tarasiuk, A., Mosińska, P. & Fichna, J. Triphala: current applications and new perspectives on the treatment of functional gastrointestinal disorders. Chin Med 13, 39 (2018). https://doi.org/10.1186/s13020-018-0197-6

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“Tulsi (Holy Basil) for Wellness: A Natural Remedy Backed by Science and Tradition”

Written By Lavanya Chavhan B.Pharm

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Source: Freepik.com

Discover the scientifically proven health benefits of Tulsi (Holy Basil), the revered Ayurvedic herb known for its powerful antioxidant, anti-inflammatory, and adaptogenic properties.

When you enter any Indian home, one thing that immediately catches your attention is the Tulsi plant placed at the entrance whether in a humble pot or a beautifully carved pedestal. As children, we often wondered why this plant holds such a special place in our homes. The answer lies in India’s rich Ayurvedic heritage. Tulsi is not just a sacred plant it is a powerhouse of health. From easing a simple cold to helping manage chronic conditions like stress and high blood sugar, Tulsi’s benefits are vast and impressive.

Tulsi, also known as Holy Basil (Ocimum sanctum or Ocimum tenuiflorum), is more than just a sacred plant in Indian households it’s a cornerstone of traditional medicine often referred to as the “Queen of Herbs,” Tulsi has been used for centuries in India for its medicinal, spiritual, and therapeutic properties. Tulsi is now gaining global recognition for its remarkable health benefits, many of which are supported by modern science. From boosting immunity to managing stress, Tulsi offers a natural solution to many contemporary health concerns making it a powerful natural remedy for various ailments.

In this article, we’ll explore how Tulsi contributes to wellness, drawing on both traditional wisdom and modern scientific research.

Phytochemicals involved in the health benefits of Tulsi

Tulsi, a time-tested herb in traditional medicine, contains key phytochemicals like polyphenols, flavonoids, terpenoids, amino acids, unsaturated fatty acids, and essential vitamins and minerals. Polyphenols and flavonoids are primarily responsible for its pharmacological effects, including anticancer, antioxidant, antimicrobial, and anti-inflammatory activities. Its antimicrobial action is further enhanced by unsaturated fatty acids (e.g., linoleic and linolenic acids), which disrupt microbial membrane integrity by interfering with the electron transport chain and oxidative phosphorylation, leading to cell destabilization. Tulsi leaves contain volatile oils responsible for their aroma and pharmacological activity, with major components being eugenol (up to 70% in some varieties). Tulsi contains several essential vitamins, like vitamins A, C, and K, and folate (B9), and minerals like calcium, iron, magnesium, potassium, and zinc, contributing to its immune-boosting and anti-inflammatory effects.

Health benefits

Anti-Inflammatory and Antioxidant Effects

Eugenol, in particular, exhibits strong anti-inflammatory properties. A 2011 study in Evidence-Based Complementary and Alternative Medicine noted Tulsi’s effectiveness in reducing inflammation markers in patients with chronic illnesses. Rosmarinic acid, a key polyphenol found in Tulsi, exhibits strong antioxidant activity. It works by neutralizing free radicals and reducing oxidative stress, thereby protecting cells from damage that contributes to aging, cancer, and various chronic diseases. A 2022 study further supported these effects, highlighting rosmarinic acid’s ability to modulate oxidative pathways and enhance cellular antioxidant defenses.

Powerful Adaptogen: Combats Stress and Anxiety

Scientific studies have classified Tulsi as adaptogen natural substances that help the body adapt to stress and restore balance. Research published in the Journal of Ayurveda and Integrative Medicine has shown that Tulsi reduces cortisol levels and improves symptoms of anxiety and depression. Its calming effects support mental clarity and emotional well-being. A 2014 clinical trial demonstrated that subjects taking Tulsi extract experienced significantly reduced symptoms of general stress and improved sleep quality.

Boosts Immunity

Tulsi enhances the body’s immune defense by stimulating the production of antibodies and promoting the activity of immune cells such as natural killer (NK) cells and T-lymphocytes. It’s essential oils and bioactive compounds particularly eugenol, ursolic acid, and apigenin confer broad-spectrum antibacterial, antiviral, and antifungal properties. Studies, including those published in Frontiers in Microbiology, have demonstrated Tulsi’s antimicrobial efficacy against various pathogens, including Escherichia coli, Staphylococcus aureus, and certain strains of influenza virus.

Supports Respiratory Health

Tulsi is a traditional remedy for respiratory conditions such as asthma, bronchitis, and sinusitis. It acts as an expectorant, helping to clear mucus, and its anti-inflammatory nature soothes inflamed airways. A study in the Indian Journal of Clinical Biochemistry reported improved lung function and reduced breathlessness in asthma patients using Tulsi-based formulations.

Balances Blood Sugar Levels

Tulsi helps regulate blood glucose levels, making it a supportive herb for managing type 2 diabetes. It enhances insulin secretion and reduces insulin resistance.

A randomized controlled trial showed significant reductions in fasting and post-meal blood sugar levels in diabetic patients who consumed Tulsi leaf powder.

Cardioprotective Properties

Tulsi improves lipid profiles and reduces high blood pressure, protecting the heart from oxidative damage and inflammation. Its vasodilatory effect helps in maintaining healthy blood flow and reduces the risk of plaque formation in arteries. A 2020 animal study demonstrated that Tulsi extract reduced LDL (bad cholesterol) and increased HDL (good cholesterol), suggesting cardioprotective potential.

Supports Oral and Dental Health

Thanks to its antimicrobial and anti-inflammatory actions, Tulsi is beneficial in preventing dental plaque, gingivitis, and oral infections. It is often used in natural toothpaste and mouth rinses. A comparative study showed that Tulsi mouthwash had similar plaque reduction effects as chlorhexidine, the standard chemical mouthwash.

Antibacterial and Antiviral Effects

Tulsi contains terpenes, carvacrol, and β-caryophyllene, which have potent antimicrobial activity. These compounds disrupt microbial membranes and interfere with the electron transport chain, leading to the death of pathogens.Effective against bacteria, viruses, and fungi, Tulsi helps in preventing infections and treating conditions like skin infections, sore throat, and cough.

Antifertility Effects of Tulsi: A Natural Contraceptive Potential

Ursolic acid, a key phytochemical in Tulsi, has shown antifertility effects in both males and females in experimental studies. In females, it may inhibit ovum implantation, while in males; it can interfere with spermatogenesis, leading to reduced sperm count and motility. These effects suggest Tulsi’s potential role as a natural contraceptive agent, offering an alternative approach without the hormonal side effects commonly seen with conventional birth control methods.

Digestive Support

Tulsi aids in digestion, relieves bloating and cramps, and helps eliminate intestinal worms. Stimulates liver function, detoxifies blood, and supports recovery from liver disorders.

Skin and Anti-aging Benefits

Tulsi’s antimicrobial and detoxifying properties help treat: Acne, eczema, psoriasis, leprosy, and staph infections. Ursolic acid prevents premature aging and wrinkles, making it popular in cosmetic products. Tulsi powder and pastes are used for blemish control and rejuvenating skin.

How to Use Tulsi

Fresh Leaves: Chew daily or brew into herbal tea.

Dried Leaves/Powder: Add to smoothies, soups, or teas.

Tulsi Capsules or Tinctures: Available in standardized extracts.

Tulsi Essential Oil: For external use in aromatherapy and skincare.

Always consult a healthcare provider before starting any herbal supplement, especially if you’re pregnant, breastfeeding, or on medication.

Conclusion

Tulsi is a versatile and powerful herb with a broad spectrum of health benefits backed by both traditional use and modern science. Its adaptogenic, anti-inflammatory, and immune-boosting properties make it an essential part of any natural health regimen. Incorporating Tulsi into your daily routine, whether as a tea, supplement, or part of your meal,s can contribute to overall wellness and resilience against disease. Always consult a healthcare provider before starting any new herbal regimen, especially if you are pregnant, nursing, or taking medication.

References

Cohen MM. Tulsi – Ocimum sanctum: A herb for all reasons. J Ayurveda Integr Med. 2014 Oct-Dec;5(4):251-9. doi: 10.4103/0975-9476.146554. PMID: 25624701; PMCID: PMC4296439.

Jamshidi N, Cohen MM. The Clinical Efficacy and Safety of Tulsi in Humans: A Systematic Review of the Literature. Evid Based Complement Alternat Med. 2017;2017:9217567. doi: 10.1155/2017/9217567. Epub 2017 Mar 16. PMID: 28400848; PMCID: PMC5376420.

Latesh Y. Chaudhari et al. A brief review on tulsi: A holy plant with high medicinal values and therapeutic uses. Int. J. Res. Ayurveda Pharm. 2022;13(3):

Hanaa A. Yamani Edwin C. Pang Nitin Mantri et al, Antimicrobial Activity of Tulsi (Ocimum tenuiflorum) Essential Oil and Their Major Constituents against Three Species of Bacteria, Front. Microbiol., 17 May 2016 Sec. Antimicrobials, Resistance and Chemotherapy Volume 7 – 2016 | https://doi.org/10.3389/fmicb.2016.00681

Verma S. Chemical constituents and pharmacological action of Ocimum sanctum (Indian holy basil-Tulsi). J Phytopharmacol 2016;5(5):205-207.

HL Kalbharati, Anti inflammatory activity of fresh tulsi leaves (Ocimum Sanctum) in albino rats October 2011 International Journal of Pharma and Bio Sciences 2(4):45-50

Arun Kumar , Puja Senthi, Rattandeep Singh, Antioxidant potential of Ocimum Sanctum: A review, Journal of Chemical Health Risks, JCHR (2023) 13(4s), 163-166

Saravanan, R., & Ramamurthy, J. (2022). Evaluation of antioxidant activity of ocimum sanctum: An in vitro study. International Journal of Health Sciences, 6(S5), 8869–8878. https://doi.org/10.53730/ijhs.v6nS5.11140

Arun K. Srivastava and Vinay K. Singh, (2021). Tulsi (Ocimum sanctum): A Potent Adaptogen. J. Clinical Research Notes. 2(2). DOI: 10.31579/2690-8816/037

Lopresti AL, A randomized, double-blind, placebo-controlled trial investigating the effects of an Ocimum tenuiflorum (Holy Basil) extract (HolixerTM) on stress, mood, and sleep in adults experiencing stress, Front. Nutr., 02 September 2022 Sec. Nutrition, Psychology and Brain Health Volume 9 – 2022 | https://doi.org/10.3389/fnut.2022.965130

Tulsi – Immune Booster For This Monsoon Season, netmeds.com, https://www.netmeds.com/health-library/post/tulsi-immune-booster-for-this-monsoon-season?srsltid=AfmBOopgQwLt0HvH4KnKOUFbwQ-FCz6oOqP4og9xeCGCkTcXouq2QQKn

Mondal S, Varma S, Bamola VD, Naik SN. Double-blinded randomized controlled trial for immunomodulatory effects of Tulsi (Ocimum sanctum Linn.) leaf extract on healthy volunteers. J Ethnopharmacol. 2011 Jul 14;136(3):452-6. Doi: 10.1016/j.jep.2011.05.012. Epub 2011 May 17. PMID: 21619917.

Rahul Kumar Burolia, Mahesh Vyas, Broncho-protective Effect of Tulsi by Using Total Lung Capacity analysis on PranvehaSrotas – A Case Series, Annals Ayurvedic Med. 2024;13(2) 147-156, DOI. 10.5455/AAM. 92550

Breathe Easy: How Tulsi Supercharges Your Lungs, https://www.garuda.hu/en/Breathe-Easy-How-Tulsi-Supercharges-Your-Lungs

Avinash Malhotra, Role of tulsi for preventing diabetes, Glob. J. Med. Plant. Res., February, 2022

Health Benefits of Tulsi (Basil) For Diabetes Patients: Nutritional Value, and Uses, Metropolice sets, https://www.metropolisindia.com/blog/preventive-healthcare/benefits-of-tulsi-leaf

Negar Jamshidi, Cliff Da Costa, Marc Cohen, Holybasil (tulsi) lowers fasting glucose and improves lipid profile in adults with metabolic disease: A meta-analysis of randomized clinical trials, Journal of Functional Foods, Volume 45, 2018, Pages 47-57, https://doi.org/10.1016/j.jff.2018.03.030.

Vishnu Sharma, Ocimum: The Holy Basil Against Cardiac Anomalies, January 2018 DOI:10.1007/978-3-319-97430-9_3

Patil A, Gunjal S, Abdul Latif AA. Tulsi: a medicinal herb for oral health. Galore International Journal of Health Sciences & Research. 2018; 3(4): 37-39.

The article is extensively reviewed and fact-checked by the editorial team of pharmacally.com

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Yutrepia (Treprostinil) Inhalation Powder developed on Liquidia’s PRINT technology Gets FDA Approval for Pulmonary Hypertension and Pulmonary Hypertension Associated with Interstitial Lung Disease (PH-ILD)

Written By: Dewanshee Ingale (B.Pharm)

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Source: Freepik.com

Yutrepia is an FDA-approved, inhaled dry-powder dosage form of treprostinil for treating pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD). This approval provides patients with a novel, practical, and efficient therapy alternative, marking a substantial development in caring for these complicated conditions. Even though treprostinil has been in practice since 2002 in various dosage forms, recently treprostinil (Yutrepia) was approved by the FDA on May 23, 2025, especially as an inhalation powder formulation for the management and control of pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD). Earlier, other inhaled forms, most remarkably an inhalation solution of treprostinil, were sanctioned in 2009 for the same indications (Tyvaso). The fundamental difference is that Yutrepia introduced the first FDA-approved dry powder inhalation dosage form of treprostinil, which proposes an entirely novel delivery technique and is based on Liquidia’s proprietary PRINT™ technology, which yields uniform, free-flowing particles intended for enhanced deep lung delivery via an easy-to-use, low-effort device requiring less inspiratory effort as compared to the prior nebulized solution.

Present data and background

Pulmonary arterial hypertension (PAH) and pulmonary hypertension with interstitial lung disease (PH-ILD) are progressive conditions that increase pulmonary artery pressure. This can be followed by right heart failure (associated with the right ventricle) and exercise intolerance. PAH was initially treated with four main mechanisms: endothelin-1, nitric oxide, prostacyclin, and bone morphogenetic protein/activin signaling, more recently. In recent times, physicians increasingly prescribe combination therapy, which is more effective for symptoms and outcomes compared to single treatment.

People with fibrotic lung diseases often develop a serious complication called pulmonary hypertension (PH-ILD), which makes their condition worse and increases the risk of death. Until recently, there were no approved treatments for this. Now, fast and effective treatment is important to manage the disease and improve outcomes. Initially the agents were unsuccessful and not safe, which revealed a lack of therapy and the need for effective and nontoxic treatment. The advent of inhaled treprostinil was innovative. The recent trials enhanced exercise capacity and reduced the severity of disease in patients with PH-ILD, which led to approval of the drug. As the traditional nebulized form was clumsy and time taking, led to decreased patient compliance. The dry inhalational powder of treprostinil was developed to provide a suitable, handy delivery system. The novel delivery technology is an enhancement in the therapy of PAH and PH-ILD, improving patient convenience and compliance in treatment.

Yutrepia: a novel approach

Treprostinil is a prostacyclin (PGI₂) analogue primarily used to treat Pulmonary Arterial Hypertension (PAH). It mimics the action of endogenous prostacyclin, a potent vasodilator and inhibitor of platelet aggregation. Treprostinil binds to IP receptors (prostacyclin receptors) on vascular smooth muscle cells. This activates adenylate cyclase, increasing cyclic AMP (cAMP) levels, which leads to smooth muscle relaxation and vasodilation, particularly in the pulmonary and systemic circulation. Treprostinil also inhibits platelet aggregation through the same cAMP-mediated pathway, which helps reduce the risk of thrombosis, a big alarm in PAH. Elevated cAMP levels also exert anti-proliferative effects on vascular smooth muscle cells, helping to prevent vascular remodelling, a characteristic of PAH progression. By mimicking prostacyclin, treprostinil can enhance endothelial cell function and reduce oxidative stress and inflammation in pulmonary arteries.

About Yutrepia and PRINT technology

Lungs_Graphic-new
Source:liquidia.com

Yutrepia is developed meticulously using Liquidia’s PRINT technology. The new drug is administered via a tiny, compact device that is lightweight and could be placed in the palms of hands. Development of Yutrepia efficiently utilizes Liquidia’s PRINT technology to formulate drug particles that are precise as well as uniform in size, shape, and composition in such a way that they can deliver more in the lungs when inhaled. The particle’s diameter is found to be 1.3 μm, which implies that the size of the particles is well controlled. The particles have a three-leaf clover shape, which facilitates their ability to effectively deliver drug.

Clinical trials and approval

The sanction of Yutrepia (Treprostinil) dry inhalational powder for pulmonary arterial hypertension(PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD) was supported on the pivotal, open-label, multicentre Phase 3 INSPIRE trial (ClinicalTrials.gov Identifier: NCT03399604). Furthermore, the ongoing ASCENT study (ClinicalTrials.gov Identifier: NCT06129240) is estimating the long-term safety criteria and acceptability of Yutrepia in patients with PH-ILD.

NCT03399604 an open-label, multicentre INPSPIRE trial phase III study was designed to assess the safety and tolerability of Yutrepia (dry-powder inhalational formulation of treprostinil) in adults with PAH. Overall 121 patients aged 18 years or above were registered, including the ones who transformed from nebulized treprostinil and prostacyclin naive patients which receive up to two non-prostacyclin oral therapies. Transition patients were started with Yutrepia at a dose that is equivalent to the prior nebulized dosing. The prostacyclin-naive patients were treated with 26.5 mcg four times daily, with dose modification in 26.5mcg in which growth can be allowed for both the groups.

The preliminary aims were to evaluate the occurrence of adverse events (AEs) and serious adverse (SAEs) throughout the entire study. Investigative efficacy parameters including changes in the 6-minute walk distances, NYHA (New York heart association), NT-proBNP levels (N-terminal pro-B-type natriuretic peptide) exposed that most patients stayed stable or enhanced over the one-year treatment time lapse. Moreover, quality of life scores enhanced, it was being observed that most of the patients preferred the Yutrepia inhaler over earlier used nebulized devices. In general, Yutrepia was discovered to be a suitable and very well-tolerated inhaled prostacyclin treatment therapy for PAH patients, assisting its use as a novel therapy option in this population.

The primary endpoints were the occurrence of AEs and SAEs. Throughout the one-year treatment period, 80% of the transition group and 96% of the prostacyclin-naive group modified to a dose of not less than 79.5 mcg four times daily, with at least one patient reaching 212 mcg daily four times. Majority of adverse effects were found to be mild to modest and steady prostacyclin therapy, comprising cough, headache, upper respiratory infection, dyspnoea, and throat irritation. Most of the patients remained stable or improved during the study.

Clinical trial NCT06129240, known as the ASCENT study, is an ongoing Phase 3, open-label, multicenter trial. It is designed to evaluate the long-term safety and tolerability of Yutrepia, a dry powder inhaled formulation of treprostinil, in patients with pulmonary hypertension (PH) and PH associated with interstitial lung disease (PH-ILD). The trial is still ongoing and recruiting participants.

Safety profile

Yutrepia (treprostinil) inhalation powder has established an appropriate safety and tolerability outline n clinical trials, remarkably in the pivotal phase III INSPIRE study. Approximately all the patients (99.2%) observed at least one adverse event (AE), while most of these being mild (47.9%) or moderate (28.1%) in seriousness. Harmful AEs were unusual (3.3%). In this clinical trials there were no serious adverse effects or mortality reported throughout the study.

The frequently reported adverse reactions <10% included not so serious side effects like cough, headache, and upper respiratory tract infections. The side effects were consistent with the known safety profile of the inhalational powder therapies and did not prevent patients from continuing treatment.

The patients who received higher percentage of prostacyclin-naive observed dose related AEs as compared the ones who were transitioned from the nebulized treprostinil (84.8% vs 72.7%). Although in general the incidence of moderate or severe AEs were quite controllable. Approximately 12.4% of patients did not continue the treatment due to AEs, among which 9.1% of these events were related to Yutrepia.

The medical monitor significantly did not observe any of the SAEs related to the Yutrepia in the trials. The maximum number of hospitalizations was because of unrelated causes like accidents, comorbidities, or any kind of viral infection, for example, COVID-19. There were no deaths reported during the study of the drug.

Impact and future viewpoint

The FDA approval of Yutrepia (treprostinil) provides with a new delivery system of inhalation dosage form by using dry inhalation powder for the treatment of pulmonary arterial hypertension and pulmonary hypertension associated interstitial lung disease this led to enhanced exercise capability, suitability, and quality of life of patients who have limited treatment alternatives. The importance of constant research and invention in cardiopulmonary diseases were highlighted by the approval. Directing to extend therapeutic pathways and improve patient results.  

The upcoming research will discover the longstanding profits, ideal dosing approaches and the ability of Yutrepia to be utilized in wider patient populations. The present and upcoming studies, like the ASCENT trials, will moreover provide extra data on long-term safety and efficacy. With Yutrepia being more widely available, its distinctive dry powder dosage form and simple inhaler are probably to expand patient acceptance to prostacyclin therapy, leading to improved disease treatment for patients with PAH and PH-ILD.         

Refrences

YUTREPIA is an FDA-approved, inhaled dry-powder formulation of treprostinil indicated for the treatment of pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD)  https://liquidia.com/pipeline-and-products

FDA approval history for Yutrepia (treprostinil) used to treat Pulmonary Arterial Hypertension; Pulmonary Hypertension Associated with Interstitial lung disease (PH-ILD)https://www.drugs.com/history/Yutrepia.html

U.S. FDA Approves Liquidia’s YUTREPIA™ (treprostinil) Inhalation Powder for Patients with Pulmonary Arterial Hypertension (PAH) and Pulmonary Hypertension Associated with Interstitial Lung Disease (PH-ILD)https://liquidia.com/news-releases/news-release-details/us-fda-approves-liquidias-Yutrepiatm-treprostinil-inhalation

FDA approval history for Tyvaso (treprostinil) used to treat Pulmonary Arterial Hypertension. Supplied by United Therapeutics Corporation https://www.drugs.com/history/tyvaso.html

Pulmonary Hypertension in Interstitial Lung Disease: Management Options to Move beyond Supportive Care https://pmc.ncbi.nlm.nih.gov/articles/PMC10200699/

Therapeutic Potential of Treprostinil Inhalation Powder for Patients with Pulmonary Arterial Hypertension: Evidence to Date https://pmc.ncbi.nlm.nih.gov/articles/PMC11162632/

INSPIRE: Safety and tolerability of inhaled Yutrepia (treprostinil) in pulmonary arterial hypertension (PAH) https://pmc.ncbi.nlm.nih.gov/articles/PMC9400582/

Liquidia Announces the Publication of Long-Term Clinical Data from Completed INSPIRE Study in the Journal Pulmonary Circulation https://liquidia.com/news-releases/news-release-details/liquidia-announces-publication-long-term-clinical-data-completed

FDA Approves Yutrepia (treprostinil) Inhalation Powder for Pulmonary Arterial Hypertension (PAH) and Pulmonary Hypertension Associated with Interstitial Lung Disease (PH-ILD) https://www.drugs.com/newdrugs/fda-approves-Yutrepia-treprostinil-inhalation-powder-pulmonary-arterial-hypertension-pah-pulmonary-6529.html

Transitioning from Parenteral Treprostinil to LIQ861 in a Patient with PAH San Francisco, CA https://liquidia.com/publications

Hill, N.S. et al., INSPIRE: A Phase 3 Open-Label, Multicenter Study to Evaluate the Safety and Tolerability of LIQ861 in Pulmonary Arterial Hypertension (PAH) (Investigation of the Safety and Pharmacology of Dry Powder Inhalation of Treprostinil NCT03399604), The Journal of Heart and Lung Transplantation, Volume 38, Issue 4, S11

Hill NS, Feldman JP, Sahay S, INSPIRE study investigators. INSPIRE: Safety and tolerability of inhaled Y et al, utrepia (treprostinil) in pulmonary arterial hypertension (PAH). Pulm Circ. 2022 Jul 1;12(3):e12119. doi: 10.1002/pul2.12119. PMID: 36034402; PMCID: PMC9400582.

Roscigno R, Vaughn T, Anderson S, Wargin W, Hunt T, Hill NS. Pharmacokinetics and tolerability of LIQ861, a novel dry-powder formulation of treprostinil. Pulm Circ. 2020 Nov 19; 10(4):2045894020971509. Doi: 10.1177/2045894020971509. PMID: 33282202; PMCID: PMC7682229.

An Open-Label ProSpective MultiCENTer Study to Evaluate Safety and Tolerability of Dry Powder Inhaled Treprostinil in PH (ASCENT), ClinicalTrials.gov ID NCT06129240, https://clinicaltrials.gov/study/NCT06129240

The article is extensively reviewed and fact-checked by the editorial team team of pharmacally.com

pile-dried-natural-cloves-with-wooden-spoon-black-surface_11zon

Clove for Oral Health: Benefits, Uses, and Dental Remedies

Written by: Lavanya Chavhan B.Pharm

pile-dried-natural-cloves-with-wooden-spoon-black-surface_11zon
Source: Freepik.com

“Discover how clove supports oral health, fighting bacteria, soothing toothaches, and promoting fresh breath all from a natural, time-tested remedy.”

Maintaining oral hygiene goes beyond brushing and flossing it also involves supporting your mouth with natural ingredients that have been trusted for centuries. One such time-tested remedy is clove, a spice sacrosanct in both traditional and modern dental care for its powerful medicinal properties.

Used in Ayurvedic medicine and folk remedies for generations, clove (Syzygium aromaticum or Eugenia caryophyllata) contains Eugenol, a bioactive compound responsible for most of its oral health benefits. From fighting bacteria to relieving pain, here’s how clove can boost your dental routine naturally.

8 Powerful Oral Health Benefits of Clove

1. Antibacterial Properties

Clove’s key compound, eugenol, has strong antibacterial effects. It targets harmful bacteria like Streptococcus mutans, a major contributor to tooth decay. It inhibits Gram-negative and Gram-positive bacteria and also yeasts. Regular use may reduce plaque and the risk of infection.

2. Anti-Inflammatory Effects

Clove reduces gum inflammation, easing discomfort and swelling. It’s invaluable for people suffering from gingivitis or mild periodontal issues. In one study, clove extract at a concentration of 0.011% showed strong antiproliferative effects on human dermal fibroblasts. It drastically inhibited the increased production of several proinflammatory biomarkers such as vascular cell adhesion molecule-1 (VCAM-1), interferon γ-induced protein 10 (IP-10), interferon-inducible T-cell α chemoattractant (I-TAC), and monokine induced by γ interferon (MIG). 

Natural Pain Relief (Analgesic)

“When applied topically, clove oil works as a natural anesthetic by numbing nerve endings, providing temporary relief from toothaches, sore gums, and mouth ulcers. Its fast-acting, soothing properties make it a trusted go-to remedy for managing dental discomfort during emergencies until professional care is available.”

4. Antiseptic Power

Its antiseptic nature is crucial in maintaining a clean and healthy oral environment. Clove helps disinfect the mouth by eliminating harmful microorganisms that can accumulate on the teeth, gums, and tongue. By controlling the growth of these bacteria, it not only prevents the onset of infections and gum inflammation but also addresses the root causes of bad breath. Regular use of clove-based oral products can therefore contribute to the prevention of plaque buildup, gingivitis, and periodontal disease, supporting long-term gum and tooth health.

5. Freshens Breath Naturally

Clove neutralizes foul-smelling bacteria in the mouth by targeting the microbial sources responsible for unpleasant odors, such as volatile sulfur compounds. Its strong antimicrobial properties help eliminate these bacteria, while its naturally spicy, aromatic scent provides an immediate sense of freshness. This dual action not only masks odors but also actively combats their cause, making clove effective in managing halitosis (bad breath) and promoting a cleaner, more refreshed mouthfeel.

6. Toothache Relief

A traditional remedy passed down through generations involves placing a whole clove or applying a few drops of clove oil directly onto the affected tooth or gum area. This method provides quick, natural relief from dental pain and sensitivity due to clove’s potent analgesic and numbing properties, primarily attributed to eugenol. The clove acts on the nerve endings to dull the pain, while its antibacterial effects help prevent infection, making it an effective and accessible option for temporary toothache relief until professional dental care is available.

7. Supports Gum Health

Its powerful anti-inflammatory and antibacterial actions work together to reduce bleeding, swelling, and irritation in the gums. By soothing inflamed tissues and combating harmful bacteria that contribute to gum disease, clove helps maintain a healthier oral environment. Regular use can support the prevention of conditions like gingivitis and periodontitis, ultimately keeping your gums strong, resilient, and less prone to infection or recession.

8. Helps Prevent Cavities

By minimizing bacterial growth and neutralizing harmful acids produced by plaque-forming microbes, cloves play a key role in the prevention of cavities. Eugenol, the active compound in cloves, inhibits the activity of Streptococcus mutans, a primary bacterium responsible for tooth decay. When incorporated into a holistic oral care routine that includes regular brushing, flossing, and healthy dietary habits, clove can help protect the enamel from demineralization and reduce the risk of developing dental caries.

Clove’s Dental Benefits at a Glance

 Why Clove Belongs in Your Oral Care Routine”

Benefit

Key Action

Kills Bacteria

Eugenol disrupts harmful microbes

Reduces Inflammation

Soothes swollen, irritated gums

Numbs Pain

Clove oil relieves toothaches and soreness

Disinfects Mouth

Acts as a natural oral antiseptic

Freshens Breath

Fights bad-breath bacteria

Toothache Remedy

Traditional use for instant relief

Boosts Gum Health

Supports healing and gum strength

Prevents Cavities

Limits bacteria that erode enamel

How to Use Clove in Oral Care

Clove Oil: Apply a drop to a cotton swab and place on the affected area (dilute with carrier oil to avoid irritation).

Mouth Rinse: Add a drop of clove oil to warm water and rinse for 30 seconds.

Tooth Powder: Mix clove powder with a bit of baking soda for a natural tooth-cleaning paste.

Clove oil is potent. Always dilute before using, and avoid ingesting large amounts. For children or those with sensitive gums, consult a dentist before use.

Clove is more than a kitchen spice, it’s a natural dental ally. From fighting bacteria to easing pain, its versatile properties make it a valuable part of a holistic oral care routine. Combine traditional wisdom with modern hygiene habits to keep your smile healthy, naturally.

Conclusion

In the realm of natural remedies, clove stands out as a potent ally for oral health. Packed with antimicrobial, anti-inflammatory, and analgesic properties, this tiny spice offers powerful support in combating toothaches, gum disease, and bad breath. From soothing dental pain to promoting overall oral hygiene, clove proves that effective solutions don’t always come in complex packages. As modern dentistry increasingly recognizes the value of traditional remedies, incorporating clove into your oral care routine, whether through clove oil, mouth rinses, or natural toothpaste, can be a simple yet impactful step toward a healthier mouth. Nature has provided a tiny powerhouse in the form of clove one that deserves a lasting place in your daily dental regimen.

References

Cortés-Rojas DF, de Souza CR, Oliveira WP. Clove (Syzygium aromaticum): a precious spice. Asian Pac J Trop Biomed. 2014 Feb;4(2):90-6. Doi: 10.1016/S2221-1691(14)60215-X. PMID: 25182278; PMCID: PMC3819475.

Nuñez L, Aquino MD. Microbicide activity of clove essential oil (Eugenia caryophyllata). Braz J Microbiol. 2012 Oct; 43(4):1255-60. Doi: 10.1590/S1517-83822012000400003. Epub 2012 Jun 1. PMID: 24031950; PMCID: PMC3769004.

Han X, Parker TL. Anti-inflammatory activity of clove (Eugenia caryophyllata) essential oil in human dermal fibroblasts. Pharm Biol. 2017 Dec; 55(1):1619-1622. Doi: 10.1080/13880209.2017.1314513. PMID: 28407719; PMCID: PMC6130734.

Kamkar Asl M, Nazariborun A, Hosseini M. Analgesic effect of the aqueous and ethanolic extracts of clove. Avicenna J Phytomed. 2013 spring; 3(2):186-92. PMID: 25050273; PMCID: PMC4075701.

Sang-Eun Moon, Hye-Young Kim, Jeong-Dan Cha, Synergistic effect between clove oil and its major compounds and antibiotics against oral bacteria, Archives of Oral Biology, Volume 56, Issue 9, 2011, Pages 907-916, https://doi.org/10.1016/j.archoralbio.2011.02.005.

Dr. Suresh Amarnathan, BDS, Dental Health Benefits of Cloves, Dr. Amaranths Dental Clinic, https://www.dramarnathansdentalcare.com/dental-health-benefits-of-cloves/

Medico-Dental History of Cloves. Nature151, 194 (1943). https://doi.org/10.1038/151194a0

Kothiwale SV, Patwardhan V, Gandhi M, Sohoni R, Kumar A. A comparative study of antiplaque and antigingivitis effects of herbal mouthrinse containing tea tree oil, clove, and basil with commercially available essential oil mouthrinse. J Indian Soc Periodontol. 2014 May;18(3):316-20. Doi: 10.4103/0972-124X.134568. PMID: 25024544; PMCID: PMC4095623.

Dr. D.Sendhil Nathan, Benefits of clove oil for teeth and gums, https://www.sendhildental.com/clove-oil-for-teeth-and-gums/

How to Use Clove Oil for Toothache Relief and Cavity Prevention, Dentistry for You, https://dentistryforyousandsprings.com/how-to-use-clove-oil-for-toothache-relief-and-cavity-prevention/

The article is extensively reviewed and fact-checked by editorial team

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“Treating Spinal Muscular Atrophy (SMA) in the Womb: Early Evidence for Prenatal Risdiplam Therapy”

Written and Reviewed By:  Vikas Londhe (M.Pharm, Pharmacology)

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Source: Freepik.com

Spinal muscular atrophy (SMA) is a rare but serious genetic neuromuscular disorder characterized by the degeneration of motor neurons in the spinal cord. This leads to progressive muscle weakness, respiratory failure, and early mortality. The severity of the disease varies depending on the number of SMN2 gene copies an individual has, with type 0 SMA being the most severe and typically fatal in the prenatal or neonatal period.

A pioneering new study, recently published in The New England Journal of Medicine, presents the administration of risdiplam, a gene-modifying therapy, during pregnancy to a fetus who is at risk of being diagnosed with SMA. Risdiplam, an orally available small-molecule drug, works by modifying the splicing of the SMN2 gene to increase production of functional SMN protein, which is essential for motor neuron survival.

This is the first-of-its-kind prenatal intervention that has shown potential to revolutionize the treatment of SMA, particularly for the most lethal forms of the disease. By initiating therapy before birth, researchers hope to prevent the irreversible loss of motor neurons in utero, offering the potential for normal neuromuscular development and vastly improved outcomes. The implications of this study are thoughtful which indicates a proactive and early management of this devastating condition.

Background: Understanding SMA and Risdiplam

Spinal muscular atrophy (SMA) is primarily caused by a genetic mutation in the SMN1 gene, which leads to a deficiency of the survival motor neuron (SMN) protein. This protein is essential for the health and function of motor neurons, nerve cells that control voluntary muscle activity such as walking, breathing, and swallowing.

In healthy individuals, the SMN1 gene produces adequate amounts of full-length SMN protein. However, in individuals with SMA, the SMN1 gene is either missing or nonfunctional, resulting in a reduction in SMN protein levels.

The body does have a natural backup in the form of the SMN2 gene, a nearly identical copy of SMN1. But due to a single nucleotide difference, the SMN2 gene undergoes inefficient splicing, leading to the exclusion of exon 7 in most transcripts. As a result, only a small fraction of functional SMN protein is produced from SMN2 typically not enough to fully counterbalance for the loss of SMN1. This imbalance in SMN protein production leads to motor neuron degeneration and the progressive muscle weakness characteristic of SMA. The number of SMN2 copies a person has can influence disease severity: more copies generally correlate with milder forms of SMA.

Risdiplam is a small molecule SMN2 splicing modifier approved for treating SMA in patients two months and older. It works by promoting exon 7 inclusions during SMN2 pre-mRNA splicing, thus increasing the production of functional SMN protein.

Study Highlights: N-of-1: A case study of prenatal risdiplam 

This study represents the first documented case of prenatal risdiplam therapy in a foetus that was at risk of developing type 1 spinal muscular atrophy (SMA). This inference was drawn because the fetus’s older deceased sibling had a genetically confirmed diagnosis of type 1 SMA. The fetus was tested by amniocentesis and confirmed to have Type 1 SMA, which is characterized by a complete absence of the SMN1 gene and two copies of the SMN2 gene. This genetic profile leads to minimal production of functional SMN protein, resulting in early motor neuron degeneration, evident as reduced fetal movement, joint contractures, and respiratory failure shortly after birth. Without intervention, type 1 SMA usually leads to death in the neonatal period, making early diagnosis and potential treatment critical.

Key Aspects of the Study

This landmark case of prenatal risdiplam therapy began with early genetic detection and a carefully monitored treatment strategy:

Prenatal Diagnosis: The foetus was diagnosed with spinal muscular atrophy (SMA) type 1 through amniocentesis and also from the previous family history of type 1 SMA diagnosed in older sibling who is unfortunately died at 16 months of age. To add this, the parents were both known carriers of SMA genetic variants. Genetic testing revealed a complete absence of the SMN1 gene and the presence of two copies of SMN2. This pattern is diagnostic of type 1 SMA, the severe form, which often results in death shortly after birth.

Approval and availability of risdiplam: The local institutional board, followed by the USFDA, has approved the single-patient investigational therapy. F. Hoffmann-La Roche has provided advice on the safety of prenatal exposure to risdiplam, along with a supply of risdiplam at no cost due to a confidentiality agreement with sponsor St. Jude Children’s Research Hospital. Parents have provided informed consent.

Initiation of Therapy: With a confirmed diagnosis and serious prognosis, the risdiplam was administered to the mother orally. The dosing was adjusted to 5 mg once per day. The risdiplam was administered from 32 weeks 5 days of gestation up to delivery at 38 weeks 6 days of gestation. However, the fetus continued to be administered risdiplam daily post-delivery from 8 days of birth to the present time (30 months of age in February 2025).

Monitoring: The mother had weekly checkups to monitor her pregnancy health and for any side effects from the medication. The fetus was also regularly checked using ultrasound to monitor growth, movement, and overall development.

Outcomes and Findings

The infant appeared healthy at birth but was later found to have a heart murmur caused by a ventricular septal defect, which resolved on its own. The child also has slightly reduced vision and experienced brief episodes transient fixation nystagmus, linked to underdevelopment of the optic nerves in both eyes. Additionally, mild weakness on the right side of the body (right hemiparesis) was observed, associated with underdevelopment of the left midbrain. The infant has shown global developmental delays but has not experienced any waning of skills. The child has not shown any signs of spinal muscular atrophy (SMA) such as low muscle tone, muscle weakness, absence of reflexes, or muscle twitching. Motor function, muscle imaging (ultrasound), and nerve tests (electrophysiology) have been conducted every six months and consistently show normal development of nerves and muscles for the child’s age.

The blood sample results revealed increased levels of SMN protein and lower neurofilament levels, which indicates the drug successfully reached its intended target and had a positive effect on motor neuron development. The above-mentioned congenital abnormalities seen in the infant were believed to have occurred early in fetal development before risdiplam treatment began, and no specific cause was identified. Animal studies also support this claim, where risdiplam was given during prenatal and postnatal stages, and no abnormalities have occurred in them.

While this is just a single case and the results cannot be widely applied, the findings suggest that prenatal risdiplam therapy could be a promising option for treating SMA when diagnosed before birth.

Significance and Implications

This study represents the first documented case of prenatal risdiplam therapy in humans, offering proof-of-concept that in utero intervention can alter the course of spinal muscular atrophy (SMA) even in its severe form, type 1. By initiating treatment during fetal development, before irreversible motor neuron loss occurs, this case displays the potential to preserve neuromuscular function, improve survival outcomes, and redefine the clinical management of SMA.

While results are based on a single case, the findings open the door to a new era of prenatal therapies for genetic neurodegenerative disorders. Future research will determine whether prenatal risdiplam should become part of the standard of care for high-risk SMA pregnancies. For now, it offers a glimpse of hope for families facing this devastating diagnosis.

Reference

Finkel RS, Hughes SH, Parker J, Civitello M, Lavado A, Mefford HC, Mueller L, Kletzl H; Prenatal SMA Risdiplam Study Group. Risdiplam for Prenatal Therapy of Spinal Muscular Atrophy. N Engl J Med. 2025 Mar 13;392(11):1138-1140. doi: 10.1056/NEJMc2300802. Epub 2025 Feb 19. PMID: 39970420.

Promising results from first prenatal therapy for spinal muscular atrophy, 19 Feb 2025, St. Jude’s Childrens Hospital, https://www.stjude.org/media-resources/news-releases/2025-medicine-science-news/promising-results-from-first-prenatal-therapy-for-spinal-muscular-atrophy.html

Treating spinal muscular atrophy in the womb, nature medicine, https://www.nature.com/articles/d41591-025-00017-9

Kakazu J, Walker NL, Babin KC, et al, Risdiplam for the Use of Spinal Muscular Atrophy. Orthop Rev (Pavia). 2021 Jul 12;13(2):25579. Doi: 10.52965/001c.25579. PMID: 34745484; PMCID: PMC8567805.

Kolb SJ, Kissel JT. Spinal Muscular Atrophy. Neurol Clin. 2015 Nov;33(4):831-46. Doi: 10.1016/j.ncl.2015.07.004. PMID: 26515624; PMCID: PMC4628728.

laboratory-worker-examining-green-substance-petri-dish-while-conducting-coronavirus-research_11zon

“Saarvienin A: A Novel Antibiotic from Rare Earth Mine in China Targets Vancomycin-Resistant Gram-Positive Bacteria”

Written by: Utkarsha Patil (M.Pharm, Pharmacology)

laboratory-worker-examining-green-substance-petri-dish-while-conducting-coronavirus-research_11zon
Source: Freepik.com

The growing threat of antimicrobial resistance (AMR) strengthens the urgent need to search for novel therapeutic agents. Amid this growing worry, a promising new antibiotic known as Saarvienin A has surfaced. Saarvienin A was isolated from a rare environmental actinomycete Amycolatopsis species.

Saarvienin A was identified by an international research team led by scientists from the University of Vienna and the Helmholtz Institute for Pharmaceutical Research Saarland (HIPS). The antibiotic was isolated from a strain of Amycolatopsis sp. YIM10, a soil-dwelling actinobacterium collected from a rare earth mine in China. Amycolatopsis is known for producing clinically important antibiotics like vancomycin.

Saarvienin A has attracted significant scientific interest due to its potent activity against multidrug-resistant infections. It is thought that Saarvienin A operates by a new mechanism due to its unique structure, which does not resemble any other glycopeptides, including vancomycin. This uniqueness will be the factor that works against drug-resistant bacteria, particularly against Staphylococcus aureus, especially methicillin-resistant strains (MRSA), making it a convincing candidate in the fight against resistant pathogens.

A Unique Habitat

The rare earth mining environment of Southern China is harsh, mineral-rich, and has biologically unique conditions that challenge microbial life and promote the production of uncommon secondary metabolites. During a targeted bioprospecting journey, researchers collected soil samples from this ecosystem. After doing an extensive analysis of this sample, scientists isolated Amycolatopsis sp. YIM10, a previously uncultured strain of actinobacteria.

This strain belonged to the prolific genus Amycolatopsis, known for producing the clinically important rifamycin antibiotics. Still, YIM10 was distinctive because scientists discovered something unusual harbored by this species: biosynthetic gene cluster (BGCs) that encodes previously unexplored compounds.

Saarvienin A

Amycolatopsis sp. YIM10 having rich reservoir of biosynthetic gene clusters (BGCs). Scientist discovered only few secondary metabolites identified in fermentation broth or via genome mining and all of these metabolites had no or very weak antimicrobial activity.  However, some culture extracts showed a significant antimicrobial effect. Further fractionation of these extracts identified a new glycopeptide, which they named it Saarvienin A, to recognize and show appreciation for Saarland, Germany, where part of the research was conducted.

Detailed NMR and mass spectrometry analyses revealed that the compound features a pentasugar/aminosugar chain attached to a halogenated peptide core, with three of its four amino acids forming a macrocyclic structure via a urea-type carbonyl linkage. A SciFinder structural similarity search identified vancomycin derivatives as the closest known compounds, even though they are structurally distinct. This suggests that the compound referred to as saarvienin A represents a novel class of glycopeptides. It can distinguish by its ability to overcome vancomycin resistance and exhibit selective antibacterial activity against Gram-positive pathogens.

The spectrum of activity

The in vitro antibacterial activity of Saarvienin A was evaluated against a range of Gram-positive and Gram-negative pathogens. It exhibited potent activity against Gram-positive bacteria, including vancomycin-resistant enterococci (VRE), methicillin-resistant and vancomycin-intermediate Staphylococcus aureus (MRSA/VISA), and Daptomycin-resistant S. aureus (DRSA).

Saarvienin A showed minimum inhibitory concentrations (MICs) of 1 µg/mL against Enterococcus faecalis (vanB-positive) and Enterococcus faecium (vanA-positive), significantly surpassing the efficacy of vancomycin. It also demonstrated low MICs against MRSA/VISA and DRSA strains. Saarvienin A showed moderate activity against Mycobacterium smegmatis but was less effective against Mycobacterium tuberculosis.

In contrast, it lacked activity against Gram-negative bacteria, including Klebsiella pneumoniae, Escherichia coli, Salmonella enterica, and Pseudomonas aeruginosa. This lack of efficacy is likely due to its inability to penetrate the outer membrane barrier of Gram-negative organisms.

Mechanism of Action

In the initial discovery and characterization studies, researchers identified its strong antibacterial activity especially against Gram-positive, drug-resistant pathogens but did not yet determine the precise molecular target or how it exerts its antibacterial effect. However, some early evidence suggests that Saarvienin A may represent a new class of glycopeptides-like compounds, structurally distinct from existing antibiotics such as vancomycin. Because it shows activity against vancomycin-resistant and daptomycin-resistant strains, it likely acts through a novel or modified mechanism, different from known glycopeptides antibiotics.

Safety

The cytotoxicity of Saarvienin A was evaluated using the HepG2 human liver carcinoma cell line, revealing an IC₅₀ of 13 µg/mL, This suggests a potential therapeutic window. The observed cytotoxic effects highlight the need for further structural optimization to improve selectivity and minimize toxicity, despite the compound’s promising antibacterial activity.

Implications and Conclusion

The discovery of Saarvienin A represents a significant milestone in the ongoing battle against antimicrobial resistance, especially among Gram-positive pathogens. Isolated from Amycolatopsis sp. YIM10, a strain collected from a rare earth mine in China. These novel glycopeptides show potent antibacterial activity against vancomycin-resistant and daptomycin-resistant strains, including VRE and MRSA/VISA.

Saarvienin A’s novel structure and potential mechanism of action, suggesting it could form the basis of a new class of glycopeptides. Despite its promising activity, particularly against drug-resistant Gram-positive bacteria, its limited effect on Gram-negative pathogens and observed cytotoxicity underscore the need for further structural optimization and preclinical evaluation.

The findings highlight the potential of extreme and underexplored ecosystems, such as rare earth mining environments, in yielding unique microbial metabolites with therapeutic potential. As resistance to existing antibiotics continues to rise, Saarvienin A serves as a gripping lead compound for the development of next-generation antibiotics capable of overcoming established resistance mechanisms.

References

Amninder KaurJaime Felipe Guerrero-GarzónSari Rasheed, et al, Saarvienin A—A Novel Glycopeptide with Potent Activity against Drug-Resistant Bacteria, Angew. Chem. Int. Ed. 2025, e202425588, doi.org/10.1002/anie.202425588

Liu L, Liu Y, Liu S, Nikandrova, et al, (2023) Bioprospecting for the soil-derived actinobacteria and bioactive secondary metabolites on the Western Qinghai-Tibet Plateau. Front. Microbiol. 14:1247001. doi: 10.3389/fmicb.2023.1247001

Scientists discover first “Saar-drug“, Saarvienin A shows promising properties for combating resistant hospital germs, Helmholtz Centre for Infection Research, https://www.helmholtz-hzi.de/en/media-center/newsroom/news-detail/scientists-discover-first-saar-drug/

Li T, Yu X, Li M, Rong L, Xiao X, Zou X. Ecological insight into antibiotic resistome of ion-adsorption rare earth mining soils from south China by metagenomic analysis. Sci Total Environ. 2023 May 10;872:162265. doi: 10.1016/j.scitotenv.2023.162265. Epub 2023 Feb 17. PMID: 36801324.

The article is extensively reviewed and fact-checked by the editorial team of pharmacally.com

Tiny people, overweight man on scales and doctor showing obesity deseases. Obesity health problem, obesity main causes, overweight treatment concept. Pinkish coral bluevector isolated illustration

Beyond Weight Loss: First-Generation GLP-1 Drugs Like Liraglutide and Exenatide Show Promising Anti-Cancer Effects

Written By: Smaiksha Benke, M.Pharm Pharmacology

Tiny people, overweight man on scales and doctor showing obesity deseases. Obesity health problem, obesity main causes, overweight treatment concept. Pinkish coral bluevector isolated illustration
Source: Freepik.com

Obesity is a major global health concern and a known risk factor for at least 13 different types of cancer. The increasing prevalence of obesity-related cancers highlights the urgent need for effective and widely accessible prevention strategies. Conventional weight loss approaches often provide only transitory benefits, while bariatric surgery though effective is invasive and not appropriate for everyone. However, first-generation GLP-1 receptor agonists (GLP-1 RAs) such as liraglutide and exenatide, initially designed to manage type 2 diabetes and obesity, are now showing encouraging anti-cancer potential, according to a recent study published in eClinicalMedicine, part of The Lancet Discovery Science series. This discovery indicates that the benefits of these medications may go beyond weight control. They appear to exert anti-cancer effects through mechanisms such as reducing inflammation, inhibiting the growth of cancer cells, and promoting programmed cell death (apoptosis).

Study Design

The study was a retrospective, observational cohort study using electronic medical records from Clalit Health Services in Israel. Researchers compared adults aged ≥24 years with obesity and type 2 diabetes who received either bariatric metabolic surgery (BMS) or first-generation glucagon-like peptide-1 receptor agonists (GLP-1RAs), primarily liraglutide, between 2010 and 2018. A total of 3178 matched pairs (N=6356) were followed for a median of 7.5 years (up to 12.9 years). Matching was based on age, sex, BMI, treatment initiation year, and smoking status. The primary endpoint was the incidence of obesity-related cancers (ORCs), including colorectal, postmenopausal breast, pancreatic, and other specified malignancies. Secondary analysis assessed mediation effects through changes in BMI and HbA1c.

Results

The final study cohort consisted of 6356 individuals (3178 pairs), matched on sex, age, baseline BMI, year of treatment initiation, and smoking status. At baseline, the mean age was 52.3 years, and the average BMI was 41.5 kg/m². The median follow-up time was 7.5 years, with some individuals followed for up to 12.9 years.

During the follow-up period, obesity-related cancers (ORCs) were diagnosed in 298 individuals:

150 cases occurred among those who underwent bariatric metabolic surgery (BMS), with an incidence rate of 5.76 cases per 1000 person-years.

148 cases occurred among those treated with GLP-1 receptor agonists (GLP-1RAs), with an incidence rate of 5.64 per 1000 person-years.

The adjusted hazard ratio (HR) for developing ORC in the GLP-1RA group compared to the BMS group was 1.03, indicating no statistically significant difference in overall cancer risk between the two treatment modalities.

Mediation Analysis

To explore potential mechanisms beyond weight loss, a mediation analysis was conducted:

Patients in the BMS group experienced a mean BMI reduction of 31.1%, whereas those in the GLP-1RA group had a mean decrease of 12.9%.

When the change in BMI was included in the adjusted model, the hazard ratio for GLP-1RA vs. BMS shifted to 0.59, indicating a direct effect of GLP-1RA therapy beyond weight loss, equivalent to a 41% relative risk reduction.

In contrast, when the change in HbA1c levels was tested as a mediator, it did not alter the main association. The adjusted HR remained non-significant, suggesting glycemic control alone does not explain the protective effect of GLP-1RAs.

Subgroup Observations

Among all diagnosed ORC cases:

Breast cancer (postmenopausal) was the most common (26%),

Followed by colorectal (16%) and uterine cancer (15%)

These distributions are aligning with known obesity-related cancer patterns.

Broader Implications

These findings are important in view of the current rising use of newer GLP-1 RAs like semaglutide (Ozempic, Wegovy) and tirzepatide (Mounjaro). While most attention has focused on weight loss and diabetes control, these early-generation drugs may offer additional therapeutic value as cancer-preventive agents.

Conclusions

The study’s findings suggest that treatment with first-generation GLP-1 receptor agonists (GLP-1RAs) like Liraglutide and exenatide may be doing more than helping patients lose weight or manage blood sugar they could also be reducing cancer risk. Also they are not associated with an increased risk of obesity-related cancers when compared to bariatric metabolic surgery (BMS) in patients with obesity and type 2 diabetes. Despite the greater weight loss achieved through BMS, the incidence of obesity-related cancers was statistically similar between the two groups over a long-term follow-up. Importantly, mediation analysis revealed that GLP-1RAs may exert a protective effect against these cancers independent of weight loss, potentially through other mechanisms such as anti-inflammatory pathways. These results position GLP-1RA therapy as a viable, non-surgical alternative for reducing obesity-associated cancer risk in this population. However, confirmation through larger prospective studies and randomized controlled trials is warranted to strengthen causal inference and explore the underlying biological mechanisms.

Reference

Wolff Sagy, Y., Ramot, N., Battat, E., Arbel, R., Reges, O., Dicker, D., & Lavie, G. (2025). Glucagon-like peptide-1 receptor agonists compared with bariatric metabolic surgery and the risk of obesity-related cancer: An observational, retrospective cohort study. eClinicalMedicine, 83, 103213. https://doi.org/10.1016/j.eclinm.2025.103213

The article is extensively reviewed and fact-checked by the editorial team of pharmacally.com

father-holds-hand-newly-born-babe-diapers_11zon (1)

“The Curious Case of KJ: How the World’s First CRISPR-Cas9 Gene Editing Therapy Saved a Child from Fatal CPS1 Deficiency”

Written by: Shital Gaikwad M.Pharm (Pharmacology)

father-holds-hand-newly-born-babe-diapers_11zon (1)
Source: Freepik.com

In a landmark medical breakthrough, a young child identified only as KJ became the first known patient to be successfully treated for a fatal genetic disorder using CRISPR gene editing inside the body. Researchers used a customized, CRISPR-based therapy to treat carbamoyl phosphate synthetase 1 (CPS1) deficiency, a rare and life-threatening genetic condition. This represents the first clinical application of a personalized CRISPR treatment tailored to an individual patient. KJ’s remarkable recovery is not only his triumph but also a significant milestone for genomic medicine, which offers a new hope for treating ultra-rare genetic diseases and paving the way for future individualized therapies.

What Is CPS1 Deficiency?

Carbamoyl Phosphate Synthetase 1 (CPS1) deficiency is an ultra-rare genetic disorder characterized by the liver’s inability to fully break down byproducts of protein metabolism, leading to a toxic buildup of ammonia in the body. The enzyme carbamoyl phosphate synthetase 1, encoded by the CPS1 gene, is essential for the urea cycle; a process that converts ammonia, a byproduct of protein breakdown, into urea to prevent harmful accumulation. Urea is then safely excreted from the body. Mutations in the CPS1 gene results in CPS1 deficiency, a hereditary urea cycle disorder that impairs the body’s ability to eliminate excess nitrogen. In the absence of this enzyme, ammonia accumulates in the blood, a condition known as hyperammonemia, which can lead to serious brain damage, coma, or even death, particularly in infants.

This condition typically presents within the first few days after birth, with symptoms like vomiting, lethargy, seizures, and difficulty breathing. The prognosis is poor, even with aggressive treatment such as dialysis or protein-restricted diets. Medication includes ammonia-scavenging agents and citrulline supplementation. These short-term management strategies are limited in effectiveness, as even slight sickness or dehydration can trigger sudden and potentially fatal organ failure.

Enter CRISPR: A Genetic Scalpel

CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) is a cutting-edge gene-editing technology that allows scientists to cut, remove, or replace faulty DNA sequences formed during mutation with high precision. Originally discovered as a natural defense system in bacteria, CRISPR functions like an immune system. Bacteria use it to recognize and destroy invading viral DNA. Scientists discovered that this bacterial defense mechanism could be adapted to precisely edit genes in other organisms, including humans.

While CRISPR has been widely used in research laboratories and clinical trials, editing DNA directly inside a living human body represents a giant leap in medicine and has never before been attempted to treat CPS1 deficiency.

How CRISPR Works: Step-by-Step

Guide RNA Design: Scientists create a synthetic RNA molecule called a guide RNA (gRNA) that matches the specific faulty DNA sequence targeted for editing.

Cas9 Binding: The guide RNA directs the Cas9 enzyme to the exact location of the faulty DNA within the genome.

DNA Cutting: Cas9 acts like molecular scissors, cutting the DNA precisely at the target site.

Repair or Rewrite: Scientists introduce a healthy copy of the gene, a template or blueprint which the cell can use to repair the cut DNA through a process called homology-directed repair (HDR). This allows the faulty gene to be corrected accurately.

KJ’s Journey: From Diagnosis to a CRISPR-Enabled Recovery

A Shocking Diagnosis

KJ was born as a healthy baby. But within 48 hours of birth, his condition deteriorated, he began vomiting, became unusually sleepy, and had trouble breathing. The blood test results were shocking. KJ’s blood showed extremely high levels of ammonia, 1000 μmol/liter (reference range, 9 to 33 μmol/liter). The plasma amino acid report revealed a vitally elevated level of glutamine, undetectable citrulline, and a normal level of urinary orotic acid. These findings were indicative of a proximal urea-cycle defect, a clear sign that something was wrong with his metabolism. Further genetic testing confirmed that the Q335X variant is absent in the Genome Aggregation Database. The absence of the Q335X variant suggests that this mutation is extremely rare or not typically found in the general population. However, it has been previously reported in one case of neonatal-onset CPS1 deficiency. Based on this genetic finding, the rare and life-threatening diagnosis of CPS1 deficiency was made. This disorder is so rare that it affects fewer than 1 in 1 million babies worldwide.

This condition meant that KJ’s liver lacked a critical enzyme needed to remove ammonia from his blood. Every time he ate protein, even small amounts found in baby formula, his body built up toxic levels of ammonia that could damage his brain or cause death within hours.

Early Treatments: A Desperate Race against Time

KJ’s care team at Children’s Hospital of Philadelphia (CHOP) immediately began intensive treatment to manage his condition. The treatment plan included:

Dialysis, to rapidly remove excess ammonia from his bloodstream

Nitrogen-scavenger medication (glycerol phenylbutyrate), to help eliminate nitrogen through alternative pathways

Citrulline supplementation, administered at 200 mg per kilogram of body weight per day, a dose that remained consistent throughout his clinical course

Strict protein restriction, to minimize ammonia production

Frequent hospitalizations, triggered even by minor infections or dietary errors

Despite these aggressive interventions, the severity of KJ’s condition continued to deteriorate. By the age of five months, he was scheduled to undergo a liver transplant, a last-resort option for managing his life-threatening disorder.

A Radical Option: CRISPR Gene Editing

As the time running out, doctors decided to use CRISPR technology to correct mutated gene. As a result, the therapy was created by a team at the Children’s Hospital of Philadelphia (CHOP), specifically within the Raymond G. Perelman Centre for Cellular and Molecular Therapeutics, in collaboration with genetic medicine experts, including Dr. Kiran Musunuru and Dr. Rebecca C. Ahrens-Nicklas, as well as Acuitas Therapeutics, which provided the lipid nanoparticle (LNP) delivery system. This was not a commercial pharmaceutical effort but rather a personalized, hospital-based investigational therapy, an example of an N-of-1 gene editing treatment tailored for a single patient.

To develop the therapy, researchers needed to correct the patient’s Q335X nonsense mutation in the CPS1 gene. Since primary human hepatocytes with the mutation were not available, they used a HuH-7 liver cancer cell line as a surrogate. Into these cells, they inserted a synthetic DNA cassette containing the patient’s specific mutation and other relevant sequences using a lentiviral vector. They then tested a range of adenine base editors (ABEs) and guide RNAs (gRNAs) to find the most effective and precise combination for correcting the mutation. The final chosen tools were NGC-ABE8e-V106W, a highly specific base editor, and a gRNA that positioned the target adenine in the ideal editing location. Their tests confirmed that the edits were successful and that any bystander edits were synonymous, meaning they did not alter the resulting protein.

The components of this custom therapy were uniquely named to reflect their personalized design. The selected guide RNA (gRNA) was called kayjayguran and the messenger RNA (mRNA) encoding the base editor was named abengcemeran.” The complete therapy, comprising both components and delivered via lipid nanoparticles, was referred to as k-abe.” These names helped distinguish the patient-specific formulation from general-purpose gene editing tools.

The administration of the therapy was carried out intravenously. The gRNA and base editor mRNA were encapsulated in lipid nanoparticles using Acuitas Therapeutics’ proprietary LNP technology, including ionizable lipids and stabilizers designed for efficient liver targeting. The patient received three intravenous infusions of the therapeutic particles. This delivery method ensured that the gene-editing components reached the liver, the organ responsible for expressing the CPS1 gene. Post-treatment monitoring showed evidence of successful gene editing and improvement in metabolic function, marking this as a milestone in personalized medicine.

freepik__an-abstract-artistic-representation-of-crisprcas9-__28061

Source: The Children’s Hospital of Philadelphia (YouTube)

Source: Freepik.com

Successful Outcome and Impact

KJ experienced significant clinical improvement following treatment. Blood ammonia levels returned to normal, significantly lowering the risk of neurological injury. Liver function tests also began to normalize, suggesting that metabolic function was being restored. Remarkably, KJ avoided additional metabolic crises, which are often fatal in untreated CPS1 deficiency.

In terms of nutritional recovery, KJ was able to tolerate increased dietary protein, a key sign of improved urea cycle function. Additionally, there was a reduced dependence on ammonia-scavenging medications, reflecting the therapy’s effectiveness in correcting the underlying metabolic defect.

Broader Implications

This achievement not only saved the life of a KJ but also represents a potential paradigm shift in how rare genetic disorders are treated. The project received support through various federal initiatives, including the NIH’s Somatic Cell Genome Editing (SCGE) program, and benefited from in-kind contributions by biotech collaborators such as Acuitas Therapeutics, Integrated DNA Technologies, Aldevron, and the Danaher Corporation.

“This is a platform technology with the potential to lead in a new era of precision medicine for hundreds of rare diseases,” said Dr. Joni Rutter, Director of the National Centre for Advancing Translational Sciences (NCATS).

Dr. Kiran Musunuru added, “Our ambition is to apply this strategy across a wide range of rare diseases so more patients can access life-saving therapies. This represents the future of medicine.”

Conclusion

This case powerfully demonstrates the practicability  of individualized gene editing, often referred to as N of 1 therapy, “highly customized treatments designed for a single patient. It highlights the adaptability and precision of CRISPR-Cas9 technology in addressing even the rarest and life-threatening genetic disorders.

Beyond its scientific success, the therapy offers renewed hope to patients and families affected by ultra-rare conditions that were once considered untreatable due to their uniqueness. Notably, in this case, the FDA played a key role by allowing the therapy to proceed based on preclinical studies, enabling a rapid response to a life-threatening condition. This pioneering effort may influence future regulatory frameworks, promoting more compassionate and flexible pathways that support the accelerated development and approval of personalized genetic therapies.

The scientists presented their groundbreaking work at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting on May 15 and published the study in The New England Journal of Medicine.

References

Musunuru K, et al. “Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease.” New England Journal of Medicine. Published online May 15, 2025. DOI: 10.1056/NEJMoa25

Regalado, A. (2024, May 17). CRISPR gene editing used to treat baby with rare genetic disease. MIT Technology Review. https://www.technologyreview.com/2024/05/17/crispr-therapy-cps1-deficiency

Infant with rare, incurable disease is first to successfully receive personalized gene therapy treatment, News Releases, National Institute of Health, May 15, 2025, https://www.nih.gov/news-events/news-releases/infant-rare-incurable-disease-first-successfully-receive-personalized-gene-therapy-treatment

World’s First Patient Treated with Personalized CRISPR Gene Editing Therapy at Children’s Hospital of Philadelphia, May 15, 2025, Children’s Hospital of Philadelphia, https://www.chop.edu/news/worlds-first-patient-treated-personalized-crispr-gene-editing-therapy-childrens-hospital

Noori M, Jarrah O, Al Shamsi A. Carbamoly-phosphate synthetase 1 (CPS1) deficiency: A tertiary center retrospective cohort study and literature review. Mol Genet Metab Rep. 2024 Oct 18;41:101156. Doi: 10.1016/j.ymgmr.2024.101156. PMID: 39469307; PMCID: PMC11513499.

Carbamoyl phosphate synthetase I deficiency, Health Resource and Service Administration, https://newbornscreening.hrsa.gov/conditions/carbamoyl-phosphate-synthetase-i-deficiency

Li Q, Gao Y, Wang H. CRISPR-Based Tools for Fighting Rare Diseases. Life (Basel). 2022 Nov 24; 12(12):1968. Doi: 10.3390/life12121968. PMID: 36556333; PMCID: PMC9787644.

Amneet Kaur Badwal, Sushma Singh, A comprehensive review on the current status of CRISPR based clinical trials for rare diseases, International Journal of Biological Macromolecules, Volume 277, Part 2, 2024, 134097, https://doi.org/10.1016/j.ijbiomac.2024.134097.

Li, T., Yang, Y., Qi, H. et al. CRISPR/Cas9 therapeutics: progress and prospects. Sig Transduct Target Ther 8, 36 (2023). https://doi.org/10.1038/s41392-023-01309-7

American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, 15 May 2025, Conference presentation and press release.

The article is extensively reviewed and fact-checked by the editorial team of pharmacally.com

cervical cancer

“Teal Wand Becomes First FDA Approved At Home Cervical Cancer Screening Device for High-Risk HPV – A Breakthrough for Women’s Health”

Written by: Priya Bhaware M.Pharm (Pharmacology)

cervical cancer
Source: Freepik.com

On May 9, 2025, the U.S. Food and Drug Administration (FDA) approved the Teal Wand medical device. Teal Health, the first FDA-authorized at-home cervical cancer screening device in the United States, developed Teal Wand. This innovative medical tool empowers women to self-collect vaginal samples for human papillomavirus (HPV) testing, which is the primary cause of cervical cancer.

The Teal Wand Device is the direct replacement of the traditional Pap smear test. By offering a more private, comfortable, and accessible alternative to traditional in-clinic Pap smears, the Teal Wand has the potential to radically improve early detection rates. It also encourages more women to participate in routine cervical cancer screening, especially those women who face barriers to in-person care.

As per Teal Health, they are working hard to roll out the product, with initial distribution set to begin in June 2025 in California first. Nationwide expansion is planned to follow soon thereafter, as the company aims to increase accessibility and convenience for women across the country. This FDA approval is seen as a transformative step toward modernizing women’s healthcare and reducing preventable deaths from cervical cancer.

Background and Need for New Treatments

Cervical cancer is the third most common cancer among women in the United States. The physiology of disease typically develops as a result of persistent infection with high-risk types of human papillomavirus (HPV), most notably the HPV-16 and HPV-18 variants. The virus enters into the basal epithelial cells of the cervix through microscopic abrasions, where it expresses oncogenic proteins E6 and E7. These proteins inactivate crucial tumor suppressor pathways, p53 and retinoblastoma (Rb), leading to loss of cell cycle control. This disruption promotes genomic instability, setting the stage for the development of cervical intraepithelial neoplasia (CIN), which can progress to invasive cervical cancer if not identified and managed early.

In the U.S., over 20 million women are currently overdue for their cervical cancer screenings. Common obstacles include limited time, demanding work, childcare responsibilities, and financial constraints. Many individuals also avoid screenings due to fear, discomfort, or pain associated with the traditional clinic-based speculum exam. For some, especially those with a history of sexual trauma, the experience can be particularly distressing. This includes the estimated 1 in 4 women who have experienced sexual assault or childhood abuse, as well as individuals who are transgender. For these groups, the standard screening process can feel invasive and create a barrier to essential preventive care.

Traditional cervical cancer screening tools, including Pap smears and physician-collected HPV tests, are effective enough; however, these tools are alleged to involve a small surgery-like procedure, are uncomfortable, and require in-person clinical appointments, which can discourage participation, particularly among underserved or timid patients. In contrast, the Teal Wand offers an at-home alternative that is non-invasive, private, and user-friendly. Another reason was that the younger women are being diagnosed with cervical cancer more often, which requires immediate early detection and treatment. This alarming situation needs correction immediately. By making screening more accessible and less intimidating, the Teal Wand has the potential to increase early detection rates, improve screening adherence, and ultimately reduce the burden of cervical cancer across the country.

Teal Wand: A Novel Approach

Source: tealhealth.com

Teal Health is a women’s healthcare company based in San Francisco that has developed the Teal Wand, a pioneering at-home cervical cancer screening device that allows women to self-collect vaginal samples for human papillomavirus testing. Teal Wand is designed as per FDA-approved standards. The Teal Wand offers a private, convenient, and clinically validated alternative to traditional in-clinic procedures. To carry out the collection of the sample at home with the help of the teal wand, patients can follow the following steps:

Request kit: Requesting a Teal Wand collection kit at-home order. Once confirmed, the kit will be directly shipped to the address.

Collect your sample & mail it to the lab: Once the kit is received, collect the sample from the comfort of your home. Once done, seal the sample and send it to the lab for further testing.

Processed at CLIA-certified labs: The sample will be collected at CLIA-certified labs, analyzed with an FDA-approved primary HPV test, and the results on the Teal Wand portal.

Clinical trial—the SELF-CERV Study

The FDA approval of Teal Wand comes after the extensive nationwide clinical trial called the SELF-CERV nonrandomized clinical trial (ClinicalTrials.gov ID: NCT06120205) conducted by Teal Health to validate the performance of Teal Wand. This study aimed to compare the effectiveness of self-collected vaginal samples (SC) using the Teal Wand with clinician-collected (CC) cervical samples collected using a speculum and brush for detecting high-risk human papillomavirus (hrHPV).

The 609 eligible participants aged between 25 to 65 years of age were recruited from 16 different sites in the USA with an intact cervix. Exclusion criteria included pregnancy, vaginal bleeding, and a cervical alteration in the prior 5 months.

Source: tealhealth.com

Procedure for the collection of a  sample using a teal wand during trial

Participants conducted self-collection (SC) in a private space designed to simulate an at-home environment. Following the provided instructions, they inserted the device into the vagina, extended the collection sponge using the dial, rotated it ten times, and then removed the device. After collection, the sponge was detached and placed into an empty vial.

For method comparison, a clinician subsequently collected a cervical sample using a Rovers Cervex-Brush, which was deposited into a 20-mL PreservCyt vial. Both self-collected and clinician-collected (CC) samples were sent to a designated laboratory for analysis.

Self-collected samples were stored dry in their vials for up to nine days. Before processing, a lab technician eluted each sponge into 20 mL of PreservCyt solution. All samples were then analyzed using the Roche cobas high-risk HPV test on the Roche cobas 8800 System.

Results

Out of total 609 eligible participants 599 paired SC-CC samples (262 aged 30-39 years [43.7%]; 583 identified as female [97.3%]) were scrutinized. 362 individuals (59.4%) were recruited from a high-risk HPV-enriched population, while the remaining 247 participants (40.6%) were enrolled from the general cervical cancer screening population.

The SC method showed 95.2% agreement with CC method in detecting high-risk HPV and 95.8% sensitivity for detecting high-grade cervical dysplasia, matching CC performance. Most participants (over 92%) found the instructions easy and said they would prefer SC if results were comparable to CC. The study found that the SC device was a safe and accurate option for cervical screening suitable for at-home use. The intersection of increased health autonomy and highly sensitive diagnostic tools make this an optimal time to implement at-home SC cervical cancer screening in the US, thereby improving access and accelerating progress toward cervical cancer elimination.

Safety Profile

Clinicians visually examined the cervix and vagina after both self-collection (SC) and clinician collection (CC). All observed events were expected and mostly mild, typically linked to CC or later procedures like colposcopy or biopsy. Only 2 of 602 participants (0.3%) experienced mild issues directly related to the SC device: one minor cervical abrasion and one case of spotting (not confirmed on exam). Mild spotting or bleeding from either method was reported in 0.5% of participants (3 of 602).

Conclusion

 The U.S. Food and Drug Administration’s approval of the Teal Wand represents a pioneering landmark in the evolution of cervical cancer screening. As the first FDA-authorized at-home self-collection device for HPV testing, the Teal Wand introduces a new era of accessibility, sovereignty, and innovation in women’s healthcare.

The teal wand is backed by clinical evidence demonstrating safety and diagnostic accuracy comparable to traditional clinician-collected methods. The device empowers women to take charge of their reproductive health in a more comfortable and private setting, removing common barriers such as discomfort, inconvenience, and limited access to in-clinic appointments.

This advancement is particularly important in reaching populations that are underserved or under-screened, potentially boosting participation rates and enabling earlier detection of high-risk human papillomavirus (hrHPV), the leading cause of cervical cancer. By allowing users to collect their sample from the comfort of home, the teal wand supports public health efforts to increase screening adherence and reduce cervical cancer incidence.

References

FDA Approves Teal Health’s Teal Wand™—The First and Only At-Home Self-Collection Device for Cervical Cancer Screening, Introducing a Comfortable Alternative to In-Person Screening, teal health, 09 May 2025, https://www.getteal.com/news/fda-approves-teal-healths-teal-wand-tm—the-first-and-only-at-home-self-collection-device-for-cervical-cancer-screening-introducing-a-comfortable-alternative-to-in-person-screening

Teal Health Completes Clinical Trial at Record Speed and Receives FDA Breakthrough Designation for Its At-Home Cervical Cancer Screening Device, the Teal Wand https://www.prnewswire.com/news-releases/teal-health-completes-clinical-trial-at-record-speed-and-receives-fda-breakthrough-designation-for-its-at-home-cervical-cancer-screening-device-the-teal-wand-302138565.html

https://tealhealth.webflow.io/teal-wand

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Teal Health Self-Cerv Report, May 2025, https://cdn.prod.website-files.com/63d5330e6841081487be0bd6/681d6148758c5c1fe0c81f2e_Teal-Health-SELF-CERV-Report-May92025-sml.pdf

Fitzpatrick MB, Behrens CM, Hibler K, Parsons C, Kaplan C, Orso R, Parker L, Memmel L, Collins A, McNicholas C, Crane L. Clinical Validation of a Vaginal Cervical Cancer Screening Self-Collection Method for At-Home Use: A Nonrandomized Clinical Trial. JAMA Network Open. 2025 May 1;8(5):e2511081-. DOI: 10.1001/jamanetworkopen.2025.11081

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