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FDA Approves Zevaskyn (Prademagene Zamikeracel) for Recessive Dystrophic Epidermolysis Bullosa (RDEB): Gene Therapy Targets COL7A1 Gene and Boosts Type VII Collagen (C7)

May 17, 2025

by official.ajinkya11@gmail.com with No Comment New Drug Approval

Written by: Rikesh Dighore (M Pharm., Pharmacology) , and Soniya Hajare (M Pharm.., Pharmacology)

For the treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB), the FDA has approved Zevaskyn (prademagene zamikeracel), the first autologous cell-based gene therapy. The treatment of RDEB, a rare and disabling genetic skin condition marked by fragile skin, prone to blistering and recurrent wounds, has advanced significantly with this approval. Abeona Therapeutics has developed Zevaskyn, a novel cell-based gene therapy scientifically known as prademagene zamikeracel (previously referred to as pz-cel or EB-101). By directly addressing the genetic mutation responsible for the disease, it represents a significant advancement in the treatment of RDEB. By using genetically modifying skin cells to create type VII collagen, Zevaskyn provides a novel therapeutic approach that improves wound healing and quality of patient’s life.

About Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Recessive Dystrophic Epidermolysis Bullosa (RDEB) is one of the subtypes of various Epidermolysis Bullosa. Epidermolysis Bullosa is the heterogeneous group of inherited mechanobullous disorders triggered by mutations in genes that encode structural proteins in the skin. RDEB involves mutations in the COL7A1 gene, which codes for type VII collagen (C7). Type II collagen protein is essential to the skin’s structural integrity. The mutation in this gene leads to anomalous synthesis of type II collagen or faulty compilation of the protein into anchoring fibrils, which results in poor epidermal-dermal cohesion.

As a result skin is become very delicate because the layers do not attach correctly without this protein. Hence, even with little contact or impact, people with RDEB experiences open wounds and persistent blistering. A complication which arises due to this condition includes severe infections, malnourishment, scarring, joint contractures, and an elevated risk of aggressive squamous cell carcinoma (SCC). The disease affects both the skin and internal mucous membranes. Many patients have a shorter life expectancy and frequently do not live past their 30s or 40s.

However, there is currently no cure for RDEB. Symptomatic treatment which includes pain management, infection prevention and control, and careful wound care, has been the main focus of standard care. Despite being necessary, these supportive treatments are quite strenuous, necessitate intensive daily interventions, and fail to address the underlying genetic problem. Additionally, current topical treatments like Vyjuvek which was approved for Dystrophic Epidermolysis Bullosa are insufficient for large, chronic, or deep wounds that considerably lower quality of life and raise the risk of death, even though they work well for minor, superficial wounds.

Hence, a targeted, long-lasting treatment alternative was in urgent need as evident from the significant morbidity and mortality rates linked to RDEB.

Zevaskyn (prademagene zamikeracel): A Novel Approach

Zevaskyn (prademagene zamikeracel) received FDA approval in April 2025, tackled a critical gap in treatment. This autologous, ex vivo gene therapy uses a retroviral vector to modify a patient’s own keratinocytes, allowing them to produce functional type VII collagen. The corrected cells are then grafted onto the patient’s chronic wounds. Unlike topical treatments that require ongoing application, Zevaskyn is designed as a one-time therapy offering long-lasting effects by providing permanent structural repair to the skin.

By enhancing skin integrity, reducing infection rates, and maybe slowing the growth of skin malignancies, this therapeutic breakthrough not only lessens the burden of care and discomfort, but also has the potential to extend life. Zevaskyn thus fills a long standing medical gap in the treatment of RDEB and marks a significant breakthrough.

Clinical Trials and FDA Approval

The FDA approval of ZEVASKYN (prademagene zamikeracel) was primarily based on the pivotal Phase 3 VIITAL™ study (NCT04227106). This multicenter, randomized, intrapatient-controlled trial evaluated the efficacy of a single application of ZEVASKYN in treating large, chronic wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB). Patients with RDEB received skin grafts made of their own genetically modified cells as part of this randomized, controlled study. The study met its two co-primary endpoints.

Wound Healing: 81% of wounds treated with ZEVASKYN achieved 50% or more healing at six months, compared to 16% in control wounds treated with standard care (P<0.0001).

Pain Reduction: Patients reported significant pain reduction from baseline, as assessed by the Wong-Baker FACES scale.

Additionally, the FDA considered data from a Phase 1/2a study (NCT01263379), demonstrating long-term improvement in treated sites over a median follow-up of 6.9 years. These studies collectively supported the approval of ZEVASKYN for treating wounds in adult and pediatric patients with RDEB.

Rowan is a 3-year-old girl living with Recessive Dystrophic Epidermolysis Bullosa in her daily life.

Source: EB Research Partnership (YouTube) _ Rowan’s Story

Safety Profile

In clinical trials, Zevaskyn has shown a good safety profile. Localized to the treatment site, the most often reported side effects included itching and soreness during the grafting process. Significantly, the trials showed no significant side effects that were directly linked to the treatment.

The risk of immunological rejection and other issues related to allogeneic transplants is decreased when the patient’s own cells are used in the treatment.

Impact

Zevaskyn’s approval gives patients with RDEB new hope and marks a major advancement in the field of gene therapy. Zevaskyn has the potential to change the standard of care and enhance the lives of those badly affected by this debilitating illness by offering a medication that targets the underlying cause of the illness and not just symptoms.

Additionally, Zevaskyn enhances current treatments like Vyjuvek (beremagene geperpavec), a topical gene therapy that was authorized in 2023 to treat minor wounds in people with DEB. The dual-action armament in treatments like Vyjuvek, treating smaller lesions, and Zevaskyn, targeting larger, chronic wounds, enables a more thorough approach to disease management.

Conclusion

Zevaskyn’s FDA clearance represents a major advancement in the management of recessive dystrophic epidermolysis bullosa a rare and devastating genetic skin disorder that has long imposed a severe burden on patients and families. ZEVASKYN, the first approved topical gene therapy for this condition, offers new hope by directly addressing the underlying genetic defect.

Its approval was backed by compelling data from the pivotal Phase 3 VIITAL™ trial, which showed significant improvements in wound healing and pain reduction two of the most debilitating aspects of RDEB. Unlike traditional therapies focused only on symptom management, ZEVASKYN delivers a corrected COL7A1 gene to the skin, promoting true biological repair.

Zevaskyn may open the door to more personalized and efficient treatments for uncommon genetic illnesses as research advances and availability increases.

References

Zevaskyn approval letter, US Food and Drug Administration, available from https://www.fda.gov/media/186513/download?attachment

Bischof J, Hierl M, Koller U. Emerging Gene Therapeutics for Epidermolysis Bullosa under Development. Int J Mol Sci. 2024 Feb 13;25(4):2243. doi: 10.3390/ijms25042243. PMID: 38396920; PMCID: PMC10889532.

Luis Soro, Do, Cynthia Bartus, Stephen Purcell et al, Recessive Dystrophic Epidermolysis Bullosa A Review of Disease Pathogenesis and Update on Future Therapies, May 2015, Volume 8, Number 5, The Journal of Clinical and Aesthetic Dermatology. https://pmc.ncbi.nlm.nih.gov/articles/PMC4445895/pdf/jcad_8_5_41.pdf

Hon KL, Chu S, Leung AKC. Epidermolysis Bullosa: Pediatric Perspectives. Curr Pediatr Rev. 2022; 18(3):182-190. Doi: 10.2174/1573396317666210525161252. PMID: 34036913.

Gurevich I, Agarwal P, Zhang P, Dolorito JA, Oliver S, Liu H, Reitze N, Sarma N, Bagci IS, Sridhar K, Kakarla V, Yenamandra VK, O’Malley M, Prisco M, Tufa SF, Keene DR, South AP, Krishnan SM, Marinkovich MP. In vivo topical gene therapy for recessive dystrophic epidermolysis bullosa: a phase 1 and 2 trial. Nat Med. 2022 Apr;28(4):780-788. Doi: 10.1038/s41591-022-01737-y. Epub 2022 Mar 28. PMID: 35347281; PMCID: PMC9018416.

Abeona Therapeutics (2025). Zevaskyn (prademagene zamikeracel): Product Information and VIITAL Study Results. https://www.abeonatherapeutics.com

Koutsoukos SA, Bilousova G. Highlights of Gene and Cell Therapy for Epidermolysis Bullosa and Ichthyosis. Dermatol Ther (Heidelb). 2024 Sep;14(9):2379-2392. Doi: 10.1007/s13555-024-01239-4. Epub 2024 Aug 7. PMID: 39112824; PMCID: PMC11393223.

Phase 3, Open-label Clinical Trial of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB), ClinicalTrials.gov ID NCT04227106, https://clinicaltrials.gov/study/NCT04227106

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

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FDA Grants Accelerated Approval to Avmapki Fakzynja Co-Pack for KRAS-Mutated Low Grade Serous Ovarian Cancer (LGSOC) Targeting RAS/MAPK Pathway

May 16, 2025

by official.ajinkya11@gmail.com with No Comment New Drug Approval

Written By: Shital Gaikwad (M.Pharm Pharmacology) and Shital Doifode (M.Pharm Pharmacology)

In a significant progress in ovarian cancer treatment, the U.S. Food and Drug Administration (FDA) has granted accelerated approval to Avmapki Fakzynja Co-Pack (avutometinib capsules and defactinib tablets) for the treatment of adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who have received prior systemic therapy. This approval represents the first and only FDA-approved treatment specifically for this rare subtype of ovarian cancer, marking a significant step forward in the fight against RAS/MAPK pathway-driven tumors. The approval is granted to Verastem Oncology, a biopharmaceutical company dedicated in the advancement of new treatments for patients with RAS/MAPK pathway-determined cancers. The FDA confirmed that this approval is under the accelerated approval, dependent on tumor response rate and duration of response; however, continuous approval for this indication will be subject to the results of the confirmatory trial. The FDA has also granted this combination Orphan Drug Designation.

Background and Need for New Treatments

Low-grade serous ovarian cancer (LGSOC) is a rare and recurrent form of epithelial ovarian cancer that mainly affects younger women and has a poor response to conventional chemotherapy. Approximately 30%–60% of LGSOC tumors carry KRAS mutations, activating the RAS/MAPK pathway and promoting uncontrolled tumor growth. KRAS mutation leads to continuous activation of the RAS/MAPK pathway.

There has been no FDA-approved treatment particularly targeting KRAS-mutated recurrent LGSOC, and patients have had limited treatment options like surgery, chemotherapy hormonal However each treatment options had certain limitations like low sensitivity to chemotherapy, high recurrence rate, toxicity of hormonal therapy and lacking of options of personalising treatment. This need highlighted the urgent demand for a targeted, effective, and tolerable treatment for this patient population.

Avmapki Fakzynja Co-Pack: A First-in-Class Combination

Avmapki Fakzynja Co-Pack is a novel oral combination therapy that includes:

Avutometinib: Avutometinib is an inhibitor of MEK1 that promotes the formation of inactive RAF/MEK complexes, thereby blocking RAF-mediated phosphorylation of MEK1/2. RAF and MEK are key components in the RAS/RAF/MEK/ERK (MAPK) signaling pathway. Avutometinib suppresses phosphorylation of MEK1/2 and ERK1/2, as well as the growth of tumor cell lines with KRAS mutations. Additionally, avutometinib treatment leads to an increase in phosphorylated focal adhesion kinase (FAK) levels in cancer cells.

Defactinib: Defactinib is a selective inhibitor of focal adhesion kinase (FAK) and proline-rich tyrosine kinase 2 (Pyk2), both part of the FAK family of non-receptor tyrosine kinases. It has been shown to block FAK autophosphorylation in cancer cells in vitro and in mouse xenograft models.

This synergistic effect of both drugs leads to enhanced tumor response by concurrently inhibiting both tumor cell proliferation and survival mechanisms, making it a breakthrough option for KRAS-mutant LGSOC.

Clinical Trials and FDA Approval Basis

The accelerated approval of Avmapki Fakzynja Co-Pack was based on the results of the Phase II RAMP 201 clinical trial, which evaluated the safety and efficacy of avutometinib, a RAF/MEK inhibitor, alone and in combination with defactinib, a FAK inhibitor, in patients with recurrent low-grade serous ovarian cancer (LGSOC), particularly those with KRAS mutations.

Study Design

RAMP 201 (NCT04625270) was a multicenter, randomized, open-label Phase II study. It enrolled patients aged 18 years or older with histologically confirmed LGSOC or peritoneal cancer, measurable disease per RECIST 1.1 criteria, and disease progression or recurrence following at least one prior systemic therapy in the metastatic setting. The trial included patients with both KRAS-mutated and wild-type tumors. The primary objective was to assess the overall response rate (ORR) of the treatments.

Key findings from the trial:

Efficacy: In patients receiving the combination of avutometinib (3.2 mg twice weekly) and defactinib (200 mg twice daily), the confirmed ORR was 31%, with a complete response rate of 2% and a partial response rate of 29%. Among patients with KRAS-mutated tumors, the ORR was higher at 44%, compared to 17% in those with KRAS wild-type tumors.

Progression-Free Survival (PFS): The median PFS for the combination therapy was reported as 12.9 months, with longer durations observed in patients carrying KRAS mutations.

Confirmatory Trials

The continuous approval of this combination therapy is reliant on confirmatory evidence from the ongoing Phase III RAMP 301 trial, which will further evaluate clinical benefit in a broader population, including patients with and without KRAS mutations.

If the clinical benefit is confirmed, this would harden the combination as the standard of care for this difficult-to-treat cancer.

Side effects

Common (≥25%) Adverse Reactions (including lab abnormalities):

Elevated creatine phosphokinase, nausea, fatigue, increased AST and ALT, rash, diarrhea, musculoskeletal pain, edema, low hemoglobin, high bilirubin and triglycerides, low lymphocytes, abdominal pain, dyspepsia, acneiform dermatitis, vitreoretinal disorders, high alkaline phosphatase, stomatitis, itching, visual impairment, low platelets, constipation, dry skin, shortness of breath, cough, urinary tract infection, and low neutrophils.

Impact

The accelerated approval of Avmapki Fakzynja marks an important step in personalized oncology, especially for women fighting recurrent LGSOC with KRAS mutations, in view of few effective conventional treatment options.

This is the first FDA-approved therapy targeting KRAS-mutated LGSOC, reflecting a significant advancement in precision medicine. It also validates the potential of combining MEK and FAK inhibition to address tumor growth and resistance mechanisms.

Expert Opinion from Investigators

Dr. Rachel Grisham of Memorial Sloan Kettering Cancer Center and Weill Cornell Medical College, New York, and lead investigator of the RAMP 301 study, stated:

“This approval marks a much-needed therapeutic option and establishes this combination as the new standard of care for women with recurrent LGSOC harbouring a KRAS mutation.”

Prof. Susana Banerjee of The Royal Marsden NHS Foundation Trust and Institute of Cancer Research, National Cancer Research Institute (NCRI), London also noted:

“To see this combination advance from early trials to become the first-ever FDA-approved therapy for LGSOC is inspiring and opens a new chapter in treating RAS/MAPK-pathway-driven cancers.”

Conclusion

The FDA’s accelerated approval of Avmapki Fakzynja Co-Pack introduces a much-needed targeted therapy for patients with KRAS-mutated recurrent low-grade serous ovarian cancer. With its novel dual-mechanism approach and promising clinical results, this combination therapy offers new hope for improving survival and quality of life in a population with limited options for LGSOC. However, its continuous approval and use depend on the success of the ongoing phase III trial. As precision oncology advances, the success of treatments like Avmapki underscores the importance of genetic profiling and personalized treatment strategies in overcoming rare and resistant cancers.

References

U.S. Food and Drug Administration. (2025, May 8). FDA grants accelerated approval to the combination of avutometinib and defactinib for KRAS-mutated recurrent low-grade serous ovarian cancer. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-combination-avutometinib-and-defactinib-kras-mutated-recurrent-low

Verastem Oncology. (2025, May 8). Verastem Oncology announces FDA approval of Avmapki Fakzynja Co-Pack for the treatment of KRAS-mutated recurrent low-grade serous ovarian cancer [Press release]. https://verastem.com/news/fda-approval-avmapki-fakzynja

ClinicalTrials.gov. (n.d.). A study of avutometinib (VS-6766) and defactinib in patients with recurrent low grade serous ovarian cancer (RAMP 201) (NCT04625270). National Library of Medicine. https://clinicaltrials.gov/study/NCT04625270

National Cancer Institute. (2024). Low-grade serous carcinoma of the ovary. https://www.cancer.gov/pediatric-adult-rare-tumor/rare-tumors/rare-gynecologic-tumors/low-grade-serous-carcinoma

Highlights of Prescribing Information, Avmapkitm Fakzynjatm Co-Pack, available from https://www.verastem.com/pdf/avmapki-fakzynja-co-pack-full-prescribing-information.pdf

FDA Grants Accelerated Approval to Avmapki Fakzynja Co-Pack, Drugs.com,available from https://www.drugs.com/newdrugs/fda-grants-accelerated-approval-avmapki-fakzynja-co-pack-avutometinib-capsules-defactinib-kras-6516.html

Susana N. Banerjee et al. ENGOT-ov60/GOG-3052/RAMP 201: A phase 2 study of VS-6766 (RAF/MEK clamp) alone and in combination with defactinib (FAK inhibitor) in recurrent low-grade serous ovarian cancer (LGSOC).. JCO 40, TPS5615-TPS5615(2022). DOI:10.1200/JCO.2022.40.16_suppl.TPS5615

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

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Japan First-in-the-World to approve Elevidys (Delandistrogene Moxeparvovec), one-of-the Costliest Gene Therapy Drug for Duchenne Muscular Dystrophy (DMD) in Children less than 4 years of age

May 15, 2025

by official.ajinkya11@gmail.com with No Comment New Drug Approval

Written By: Pragati Ekamalli (B.Pharm)

Reviewed By: Vikas Londhe M.Pharm (Pharmacology)

In a groundbreaking step forward for genetic medicine, Japan has become the first country in the world to approve the use of Elevidys (delandistrogene moxeparvovec), a gene therapy created by Sarepta Therapeutics, for children under 4 years old diagnosed with Duchenne muscular dystrophy (DMD). DMD is a rare and life-threatening genetic disorder that leads to the progressive weakening and loss of muscle function, typically appearing in early childhood and worsening over time. Elevidys works by delivering a modified gene to help produce a version of the dystrophin protein, which is missing or defective in children with DMD. This approval represents a major milestone in the global effort to treat genetic disorders and could bring new hope to families affected by DMD. It also highlights Japan’s leadership in adopting cutting-edge medical treatments that may slow disease progression and improve quality of life for young patients.

Understanding Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a rare, inherited disorder that mainly affects boys due to its X-linked pattern of inheritance, meaning the faulty gene is carried on the X chromosome. The condition results from mutations in the DMD gene, which is responsible for producing dystrophin a protein critical for maintaining the strength and stability of muscle fibers. In the absence of functional dystrophin, muscles are unable to protect themselves from everyday stress, leading to ongoing damage, muscle fibres breakdown, and replacement by fat and scar tissue. Symptoms usually begin in early childhood, often with difficulty walking, running, or climbing stairs. As the disease progresses, most affected children lose their ability to walk by the age of 12 to 14. In later stages, DMD can lead to serious complications involving the heart and lungs, often reducing life expectancy into the twenties or thirties without advanced medical care.

Understanding Dystrophin Protein

Dystrophin is large structural protein (427kDa) present in cytoplasmic face of the sarcolemma. Dystrophin protein plays a crucial role in maintaining the integrity and function of muscle cells including skeletal muscle and cardiac muscle.

Dystrophin is act like an anchor which link internal cytoskeleton of a muscle fibre to the surrounding extracellular matrix by dystrophin-glycoprotein complex. This complex stabilizes muscle fibre during contraction. Dystrophin protein is coded with DMD gene on X-Chromosome and it is one of the largest human genes. Any mutation or deletion of DMD genes affects the dystrophin protein production, either less or completely diminished.

Elevidys: A Novel Gene Therapy Approach

Elevidys is an innovative gene therapy that uses an adeno-associated virus (AAV) as a delivery system to transport genetic material into the body. Specifically designed for Duchenne muscular dystrophy (DMD), Elevidys delivers a shortened but functional version of the dystrophin gene, called micro-dystrophin, directly into muscle cells. Although smaller than the full-length gene, micro-dystrophin includes the critical domains needed for the protein to support muscle fibre structure and function. By enabling cells to produce this essential protein, Elevidys targets the root cause of DMD — the absence of functional dystrophin. Administered as a single intravenous infusion, this therapy offers the promise of a one-time treatment that may slow disease progression and improve muscle strength, potentially transforming the standard of care for children living with DMD.

Japan’s Regulatory Approval

On August 14, 2024, Chugai Pharmaceutical Co., Ltd., a member of the Roche Group, submitted a regulatory application to Japan’s Ministry of Health, Labour and Welfare (MHLW) seeking approval for Elevidys, a gene therapy for Duchenne muscular dystrophy (DMD). The application specifically targeted ambulatory boys aged 3 to 7 years who do not have deletions in exons 8 and/or 9 of the DMD gene and who are not immune to the AAVrh74 viral vector used to deliver the treatment. This submission was supported by data from the global Phase III EMBARK study, which evaluated the safety and effectiveness of Elevidys. The trial did not meet its primary endpoint improving motor function as measured by the North Star Ambulatory Assessment (NSAA), however clinically meaningful benefits were observed in key secondary outcomes, including faster time to rise from the floor and improved 10-meter walk speed. Despite the mixed results, Japan’s MHLW granted priority review to the application and ultimately approved Elevidys for use in children less than 4 years of age. With this decision, Japan became the first country to authorize Elevidys for this younger age group, reflecting its proactive approach to approving innovative therapies for rare and serious diseases like DMD. This landmark approval offers new hope for patients and families affected by this devastating condition.

Global Context

Before Japan’s historic approval, the U.S. Food and Drug Administration (FDA) granted Elevidys accelerated approval in June 2023 for ambulatory children aged 4 through 5 years with Duchenne muscular dystrophy (DMD). The FDA’s decision was based on evidence that the therapy successfully prompted the production of micro-dystrophin, the shortened version of the dystrophin protein essential for muscle health. However, this approval was conditional, with continued authorization depending on the confirmation of meaningful clinical benefits in ongoing follow-up trials. Japan’s more inclusive approval for children under 4 years old not only marks a significant expansion of access to this gene therapy but also sets a global precedent. It signals to other regulatory agencies the potential value of early intervention in DMD. As new clinical data emerges and confirmatory studies continue, more countries may follow suit, potentially reshaping the global treatment landscape for this devastating disease and offering hope for earlier and more effective management of DMD in young patients.

Conclusion

The approval of Elevidys in Japan for children younger than 4 years of age marks a major breakthrough in the treatment of Duchenne muscular dystrophy (DMD). Unlike traditional therapies that mainly manage symptoms, Elevidys directly targets the genetic root of the disease by introducing a functional version of the dystrophin gene. This approach offers the potential not just to slow the progression of muscle degeneration, but to meaningfully alter the course of the disease, particularly when administered early in life. The decision by Japan’s health authorities highlights the critical role of early intervention and paves the way for improving long-term outcomes for children affected by DMD. Moreover, this milestone emphasizes the value of ongoing scientific research, clinical trials, and international collaboration in developing transformative therapies for rare and life-threatening conditions.

References

1. Sarepta Therapeutics Announces Approval in Japan of ELEVIDYS, a Gene Therapy to Treat Duchenne Muscular Dystrophy, 13 May 2025, Sarepta Therapeutics https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-approval-japan-elevidys-gene

2. A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) (EMBARK), NCT05096221, ClinicalTrials.gov, available from https://clinicaltrials.gov/study/NCT05096221

3. Roche announces new results from EMBARK demonstrating significant sustained benefits of Elevidys in ambulatory individuals with Duchenne muscular dystrophy (DMD), 27 January 2025, https://www.roche.com/media/releases/med-cor-2025-01-27

4. Gao QQ, McNally EM. The Dystrophin Complex: Structure, Function, and Implications for Therapy. Compr Physiol. 2015 Jul 1; 5(3):1223-39. Doi: 10.1002/cphy.c140048. PMID: 26140716; PMCID: PMC4767260.

5. Venugopal V, Pavlakis S. Duchenne Muscular Dystrophy. [Updated 2023 Jul 10]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2025 Jan-. Available from: https://www.ncbi.nlm.nih.gov/books/NBK482346/

6. Chugai Receives Regulatory Approval for “ELEVIDYS” as a Gene Therapy Product for Duchenne Muscular Dystrophy in Japan, 13 May 2025, Chugai Pharmaceuticals,https://www.chugaipharm.co.jp/english/news/detail/20250513181500_1160.html

“Microplastics in Human Tissues: Insights from Nature Medicine and NEJM Studies”

May 14, 2025

by official.ajinkya11@gmail.com with No Comment Research

Written By Lavanya Chavhan B.Pharm

Reviewed and Fact Checked By Vikas Londhe M.Pharm (Pharmacology)

Microplastics and nanoplastics (MNPs) tiny particles resulting from the degradation of plastic materials have rapidly become a focus of global health research. Two landmark studies published in Nature Medicine and The New England Journal of Medicine (NEJM) offer critical insights into the bioaccumulation and health consequences of these particles in human tissues. Together, they present an urgent call to re-evaluate the long-term implications of environmental plastic pollution.

Brain Accumulation and Neurological Concerns: Findings from Nature Medicine

A study published in Nature Medicine (April 2025) examined the presence of MNPs in human tissues, with a focus on liver, kidney, and brain samples from autopsies conducted in 2016 and 2024. The researchers used advanced analytical techniques including pyrolysis gas chromatography mass spectrometry (Py-GC/MS), electron microscopy, and Fourier-transform infrared spectroscopy to identify and quantify plastic particles.

Key Findings

Ubiquitous Presence: MNPs were detected in all three organs, but brain tissues exhibited the highest concentrations, 7 to 30 times greater than liver or kidney.

Polyethylene (PE) Dominance: PE accounted for approximately 75% of the plastic content in brains.

Temporal Trends: There was a significant increase in plastic concentrations between 2016 and 2024, reflecting rising environmental exposure.

Dementia Link: Brains of individuals with documented dementia had markedly higher levels of MNPs up to 26,076 µg/g suggesting a possible connection with neurodegenerative processes

Tissue Localization: Nanoplastics were found in brain parenchyma, immune cells, and cerebrovascular walls, raising questions about blood-brain barrier integrity and inflammatory responses.

Conclusion: Although the study does not establish causality, it strongly suggests that chronic exposure to MNPs may be associated with neurological vulnerability. The findings warrant deeper exploration into how plastic particles reach and affect brain tissue, particularly in individuals with compromised neurological health.

Cardiovascular Risks and Atheroma Infiltration: NEJM Study

The NEJM study (March 2024) provided the first direct clinical evidence linking MNPs to cardiovascular risk. It was a multicenter, prospective study involving 304 patients undergoing carotid endarterectomy for asymptomatic carotid artery disease.

Key Findings:

MNP Detection: In 58.4% of patients, polyethylene was found within atherosclerotic plaques. Polyvinyl chloride was also detected in 12.1%.

Nanoplastics Inside Plaques: Electron microscopy revealed jagged-edged particles (<1 µm) embedded in foam cells and extracellular plaque matrix.

Increased Cardiovascular Events: Over a 34-month follow-up, patients with MNP-positive plaques were at 4.5 times greater risk of myocardial infarction, stroke, or death compared to those without detectable plastics.

Inflammation Correlation: Elevated levels of interleukin-1β, IL-6, TNF-α, and CD68 were observed in MNP-positive plaques, suggesting heightened inflammatory responses.

Conclusion: The presence of MNPs in vascular tissues appears to be more than incidental. It correlates with both histological inflammation and clinically significant cardiovascular outcomes. This raises red flags about MNPs as emerging risk factors in heart and vascular diseases.

Implications for Human Health and Future Research

The convergence of findings from brain and vascular tissues paints a concerning picture:

Bioaccumulation is Organ-Specific: MNPs preferentially accumulate in certain tissues, potentially due to local physiological characteristics like vascular density or barrier permeability.

Health Impact is Multisystemic: While one study implicates MNPs in neurodegeneration, the other links them to cardiovascular events two of the most pressing areas in public health.

Mechanistic Gaps Remain: Both studies acknowledge the limitations in establishing causality. The exact pathways of MNP absorption, translocation, and clearance in humans remain poorly understood.

Another small study presented in American Heart Association conference

At the American Heart Association’s Vascular Discovery Scientific Sessions held in Baltimore on April 22, 2025, researchers presented compelling evidence linking microplastics to vascular disease. The study, led by Dr. Ross Clark from the University of New Mexico, analyzed carotid artery plaques from individuals who had experienced strokes, mini-strokes, or transient vision loss. Findings revealed that these plaques contained microplastics at concentrations 51 times higher than those in healthy arteries. Notably, polyethylene and polyvinyl chloride were the predominant plastics detected. While the study did not establish a direct causal relationship, it highlighted a strong association between microplastic accumulation and symptomatic arterial disease, underscoring the need for further research into the potential health impacts of environmental plastic exposure.

Conclusion

As plastic production continues to grow rapidly across the globe, recent studies serve as a strong warning that we must take urgent action. This includes putting better regulations in place, increasing scientific research, and raising public health awareness. Tiny plastic particles known as microplastics and nanoplastics are now found in the air we breathe, the food we eat, and even in our bodies. These particles may carry harmful chemicals and can travel through the blood stream to vital organs. Over time, long term exposure to them could increase the risk of serious health problems, including disease of the brain, heart, lungs and more.

To truly understand how these plastics affect our health, we need experts from many different fields to work together. Toxicologists can study how the plastics interact with our cells, neurologists can explore their impact on the brain and nervous system, cardiologists can look into heart-related effects, and environmental scientists can track how plastics move through ecosystems and into our bodies. Only through this kind of teamwork can we begin to understand plastic’s full impact on human health now and in the future.

Reference

Alexander J. Nihart, Marcus A. Garcia, Eliane El Hayek et al, Bioaccumulation of microplastics in decedent human brains, nature medicine, Volume 31 , April 2025, 1114–1119, https://doi.org/10.1038/s41591-024-03453-1

2. R. Marfella, F. Prattichizzo, C. Sardu et al, Microplastics and Nanoplastics in Atheromas and Cardiovascular Events, N Engl J Med 2024;390:900-10. DOI: 10.1056/NEJMoa2309822

3. Micronanoplastics found in artery-clogging plaque in the neck, American Heart Association Meeting Report , Scientific Conferences & Meetings, Stroke News & Brain Health, 22 April 2025 available from https://newsroom.heart.org/news/micronanoplastics-found-in-artery-clogging-plaque-in-the-neck

Vyayama in Ayurveda: Principles, Benefits & Daily Routine for Physical Exercise

May 13, 2025

by official.ajinkya11@gmail.com with No Comment Lifestyle

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

In Ayurveda, the ultimate aim is to maintain the health of a healthy individual and to cure the disease of the sick. To achieve this, our ancient texts emphasize the importance of Dinacharya (daily regimen) and Ritucharya (seasonal regimen). Among these daily practices, Vyayam (exercise) holds a significant place. While modern fitness often emphasizes intensity and physique, Ayurveda focuses on personalized, moderate, and mindful movement that harmonizes with nature and individual constitution (Prakriti).

In this article, we explore the principles of Vyayama in Ayurveda, its numerous benefits, and how to integrate it into a balanced daily routine

Importance in Modern Life

Vyayama refers to controlled and purposeful physical activity that enhances physical stamina, mental clarity, and overall well-being. According to classical Ayurvedic texts such as the Charaka Samhita and Ashtanga Hridaya, Vyayama is considered an essential part of Dinacharya (daily routine) and a pillar of health maintenance.

In today’s fast-paced lifestyle and predominantly sedentary routines, regular exercise becomes crucial for maintaining overall health. Lack of physical activity has led to a surge in lifestyle disorders (LSDs) such as

Obesity

Diabetes type 2

Heart disease

PCOD (Polycystic Ovarian Disease), especially in women

Key Principles of Vyayama in Ayurveda

Moderation is the Key

Ayurveda advises that one should exercise only until half of their physical strength is expanded. Sign that you should stop include shortness of breath, excessive sweating, or fatigue. Overexertion can aggravate Vata and Pitta doshas, leading to imbalance.

Customize to Your Prakriti (Constitution)

Vata types (light, dry, and mobile): Gentle exercises like yoga, walking, or tai chi are ideal.

Pitta types (hot, intense, and focused): Cooling and non-competitive activities like swimming or hiking are recommended.

Kapha types (heavy, slow, and steady): More vigorous exercise like running or aerobics can be beneficial.

Time of Day

The best time for Vyayama is during the early morning hours (6 AM to 10 AM), when Kapha dosha is dominant. Physical activity at this time helps stimulate metabolism and energizes the body for the day.

Seasonal Considerations
Exercise intensity should vary with the seasons:

Winter (Hemanta/Shishira): Strength is at its peak, moderate to vigorous exercise is suitable.

Summer (Grishma): Strength is lowest opts for light exercises to avoid heat exhaustion.

Monsoon (Varsha): Prone to joint stiffness, mild stretches or indoor yoga is best.

Benefits of Vyayam as Described by Acharya Vagbhata

Acharya Vagbhata, one of the classical scholars of Ayurveda, beautifully elaborated the benefits of regular exercise

Enhances Digestion (Agni Deepana): Exercise kindles Agni (digestive fire), aiding in better metabolism and nutrient absorption.

Promotes Lightness (Laghavam): Regular movement removes heaviness from the body and mind, creating a sense of ease and vitality

Improves Circulation and Immunity (Bala Vriddhi): Exercise improves circulation and enhances Ojas (vital essence), the foundation of immunity.

Reduces Fat and Toxins (Medo Hara): Moderate exercise burns excess meda dhatu (fat tissue) and helps eliminate ama (toxins)

Sharpens the Mind (Medha and Smriti Vriddhi): Physical movement clears mental fog, improving concentration and memory

Enhances Mood and Sleep: Balanced Vyayama helps manage stress and anxiety, leading to better emotional stability and restful sleep

Who Should Avoid Exercise?

Vyayam is contraindicated in the following conditions

Individuals with Vata- or Pitta-dominant disorders

People suffering from Ajirna (Indigestion)

Elderly persons above the age of 80

Children below 12 years of age

After heavy meals

During fever or illness

According to Ayurveda, there is the certain age limit for exercise but according to modern science No matter your age, it’s never too late to exercise or start exercising. Talk to your doctor to get started.

Guidelines for Practicing Exercise

Exercise should be performed by those who have good strength and have consumed unctuous (Snigdha) food.

In Vasanta Ritu (spring) and Shita Ritu (winter), it is advised to do only half of one’s full capacity due to the varying energy dynamics of the seasons.

After exercise, Abhyanga (body massage) should be performed to relax and soothe the body.

Over-Exercise Side Effects

Despite its benefits, excessive Vyayama can cause certain side effects, these side effects include

Trishna—excessive thirst

Kshaya—Tissue depletion

Shwāsa – Breathlessness

Raktapitta—Bleeding disorders

Shrama—Fatigue

Kāsa – Cough

Jvara—Fever

Chardi – Vomiting

Glāni – Dizziness or faintness

Conclusion

Incorporating Vyayama into daily life, tailored to individual constitution, season, and current health status, is a holistic approach to maintaining health and preventing disease. Ayurveda’s emphasis on moderation, mindfulness, and personalization ensures that physical exercise contributes to overall balance and well-being.

Reference

Vagbhat Sutra sthan Chapter 2 / 9-12

The Vital Role of Physical activity in preventing Lifestyle diseases – Caritas

Hospital & Institute of health science

Anupama Krishnan, Blessymol EV. Vyayama. In: Deole Y.S., eds. Charak Samhita New Edition. 1^st ed. Jamnagar, Ind: CSRTSDC; 2020. https://www.carakasamhitaonline.com/index.php?title=Vyayama&oldid=44682.

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How MIT’s SLIM Microcrystal Injection Technology is Transforming Long-Acting Treatments for HIV and Contraceptives

May 13, 2025

by official.ajinkya11@gmail.com with No Comment Research

Medically Reviewed By Vikas Londhe M.Pharm (Pharmacology)

MIT scientists have developed a self-injectable, long-acting drug delivery system using microcrystals. Learn how this innovation could transform contraception and chronic disease treatment.

Imagine a single injection that lasts for months, delivered through a tiny needle you can use yourself, no surgery, no hospital visit. Thanks to a groundbreaking innovation from researchers at Massachusetts Institute of Technology (MIT), that future is now one step closer.

MIT researchers have introduced a groundbreaking innovation that could make this vision a reality. Called SLIM; Self-aggregating Long-acting Injectable Microcrystals this new drug delivery system offers extended release, smaller needle size, and high drug potency, all packed into a single self-administered injection.

A New Way to Take Medicine: Once Every Few Months

Researchers at MIT and Harvard-affiliated Brigham and Women’s Hospital have developed a new drug delivery system called SLIM, short for Self-aggregating Long-acting Injectable Microcrystals.

Instead of needing large needles or surgery, SLIM allows medicine to be injected just under the skin through a tiny needle. Once injected, the medicine automatically forms a small solid “implant” that slowly releases the drug over several months.

This method is especially promising for medications like birth control and HIV medicine where convenience, privacy, and reliability are essential

Global Health Impact

For millions worldwide especially women in under-served regions SLIM could mean:

No need for surgical implants or repeated clinic visits

Empowered self-administration of essential medications

Lower costs and better access to long-term contraception

This new technology is published in the journal Nature Chemical Engineering. The technology also aligns with public health goals from organizations like the WHO and the study is co-funded by Gates Foundation.

How It Works: Tiny Crystals, Big Impact

SLIM works by suspending drug particles called microcrystals in a liquid. When injected into the body, this liquid mixes with the body’s water and causes the particles to clump together into a compact, slow-dissolving depot.

Less pain, smaller needles: Unlike other long-acting injections that require thick, painful needles, SLIM uses fine needles as small as 30 gauge (about the width of a human hair).

Fewer additives: Most long-acting shots rely on lots of added slowly degrading polymers such as polylactic acid or polycaprolactone to slow down drug release. SLIM only needs a tiny amount, reducing risks of side effects and injection pain.

Why It Matters: Especially for Women and Low-Resource Settings

In many parts of the world, women may not have easy access to clinics for repeated contraception or medications. Self-injectable treatments could empower them to take control of their health privately and conveniently.

This is a big step beyond current injectables like Depo-Provera, which lasts 3 months but can’t match the long-lasting protection of surgical implants like Nexplanon. SLIM offers the best of both: long-lasting protection with no surgery required.

A Breakthrough for Contraceptive Delivery

The researchers tested SLIM using levonorgestrel, a common hormonal contraceptive. In lab studies on rats, the SLIM formulation:

In early tests, the SLIM injection worked really well. After being injected, it formed a small, solid implant under the skin that stayed in place. It released the medicine much more slowly up to 5.6 times slower than current options, helping it last longer. The implant stayed strong and stable for over 3 months, and the injection was easy enough to be done at home using a regular small syringe. This means that women could get fewer injections while still having reliable, long-term birth control.

Bottom Line: Why This Matters for the Future of Medicine

The researchers plan to test SLIM with other drugs, especially those that are hard to dissolve in water, like many modern medications.

They’re also studying how to fine-tune drug release and improve safety for human use.

Because of its compact, stable design, SLIM could become a game-changer for treating chronic conditions like mental health, HIV prevention, or cancer with fewer doses and better adherence.

The SLIM system could redefine how we take medicine offering safer, longer-lasting, and more comfortable treatment through self-administered injections. It’s a promising leap toward better access and better outcomes, especially in global health.

Long-acting injectables are a proven way to boost adherence, especially for chronic conditions. But current systems are limited by: Large, painful needles, Viscous, hard-to-inject formulations and Poor suitability for self-administration.

SLIM overcomes these barriers with smart design: high drug loading, low viscosity, and small injection size all while delivering long-lasting effects.

Reference

Feig, V. R., Park, S., Rivano, P. G., et al. (2025). Self-aggregating long-acting injectable microcrystals. Nature Chemical Engineering, 2(3), 209–219. DOI: 10.1038/s44286-025-00194-x

Engineers develop a better way to deliver long-lasting drugs, MIT News, Massachusetts Institute of Technology, 24 March 2025

Owen A, Rannard S. Strengths, weaknesses, opportunities and challenges for long acting injectable therapies: Insights for applications in HIV therapy. Adv Drug Deliv Rev. 2016 Aug 1;103:144-156. Doi: 10.1016/j.addr.2016.02.003. Epub 2016 Feb 23. PMID: 26916628; PMCID: PMC4935562.

McEvoy, Joseph. (2006). Risks versus benefits of different types of long-acting injectable antipsychotics. The Journal of clinical psychiatry. 67 Suppl 5. 15-8.

National Research Council (US) Committee on Population. Contraception and Reproduction: Health Consequences for Women and Children in the Developing World. Washington (DC): National Academies Press (US); 1989. 4, Contraceptive Benefits and Risks.Available from: https://www.ncbi.nlm.nih.gov/books/NBK235069

Source of the Video: Giovanni Traverso (YouTube) _Self-Aggregating Long-Acting Injectable Microcystals

Disclaimer: The video is for educational and informational purposes only.

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Novel Oncolytic Virus Therapy Uses Transplant Rejection Pathway to Kill Cancer

May 10, 2025

by official.ajinkya11@gmail.com with No Comment Clinical Trails

Medically Reviewed By Vikas Londhe M.Pharm (Pharmacology)

Introduction

In a breakthrough that bring together virology, immunology, and genetic engineering, researcher from State Key laboratory of Targeting Oncology, China have developed a novel oncolytic therapy using a genetically modified Newcastle Disease Virus (NDV) that tricks the immune system into treating cancer cells as if they were foreign organ transplants. This approach, which uses the body’s natural “hyperacute rejection” mechanism typically seen in organ xenotransplants, showed notable results in early clinical trials for patients with advanced, treatment-resistant cancers.

Hyperacute rejection is a type of organ transport rejection that happens within minutes to hours after the transplant. It occurs when the recipient’s immune system already has antibodies that quickly recognize the new organ as foreign and attack it. This rapid immune response causes severe damage to the transplanted organ, often leading to its failure almost immediately.

Xenotransplant is a medical procedure where cells, tissues, or organs are transplanted from one species to another for example, transplanting a pig’s heart into a human.

The Innovation: NDV-GT and the aGal Trigger

The therapy uses a recombinant NDV that expresses the porcaine α1, 3-galactosyltransferase (a1, 3GT) gene, resulting in the presentation of αGal antigens on infected tumor cells. In humans, the αGal antigen is recognized as foreign due to the evolutionary loss of the α1, 3GT gene. Our immune systems naturally produce large amounts of anti-αGal antibodies, primarily from exposure to gut microbiota. This makes the immune system especially reactive to any cell displaying αGal.

By engineering NDV to express this foreign antigen on tumor cells, scientists effectively turned cancer cells into targets of a powerful immune cascade similar to organ rejection. This results in rapid tumor destruction through complement activation, thrombosis, and immune cell infiltration.

Preclinical Success in Monkeys

Using CRISPR-Cas9 technology, the researcher created a realistic liver cancer model in cynomolgus monkeys. The intravenous administration of NDV-GT in these monkeys led to remarkable tumor regression with many tumors disappearing entirely. The therapy not only lysed the cancer cells but also caused thrombotic blockage of tumor blood vessels mimicking hyperacute rejection seen in xenograft failures.

Immune profiling revealed activation of both innate and adaptive immune responses, including CD4+ and CD8+ T cell infiltration, increased expression of granzyme B and perforin, and reduced tumor angiogenesis markers. Importantly, no significant toxicity or organ damage was observed.

Clinical Trial Results

In a phase I interventional clinical trial (ChiCTR2000031980), 20 patients with various advanced, treatment-resistant cancers, including liver, lung, rectal, esophageal, breast, and cervical cancer and melanoma, were treated with intravenous NDV-GT. The results were prominent:

90% Disease Control Rate (DCR): 1 complete response, 6 partial responses, and 11 cases of stable disease.

No severe adverse events: Mild side effects, no cytokine release syndrome, and no detectable viral shedding.

Durable response: Severe patients experienced long-term stabilization o remission lasting up to 36 months.

One standout case involved a patient with advanced hepatocellular carcinoma and lung metastases. After 1.5 months of NDV-GT treatment, most tumors had disappeared, and serum tumor markers dropped significantly. Another patient with metastatic ovarian cancer achieved a partial remission for over a year.

Mechanism of Action

NDV-GT’s effectiveness lies in its dual mechanism :

Direct Oncolysis: NDV naturally prefers and destroys tumor cells while sparing healthy ones.

Immune Amplification: The engineered Gal expression triggers complement-mediated cytotoxicity and T-cell driven immune cascade, simulating a rapid rejection response that targets the tumor as a foreign organ.

This dual action also alleviated the immunosuppressive tumor microenvironment, making tumors more susceptible to further immune attack.

Safety and Implications

Despite the robust immune activation, NDV-GT treatment was well tolerated. No significant increase in neutralizing antibodies or adverse immunogenic effects was observed. The lack of toxicity and the specific targeting of tumor tissue underscore its promise for broader clinical use.

Conclusion

NDV-GT represents a groundbreaking shift in oncolytic virotherapy. Bby mimicking a hyperacute rejection response. This therapy turns the body’s natural defense mechanism against tumors in a way that is both powerful and safe. As phase II-III trials are being prepared, this immune- tricking virus could redefine treatment paradigms for refractory and metastatic cancers.

Reference

1. Liping Zhong, Lu Gan, Bing Wang et al, Hyperacute rejection-engineered oncolytic virus for interventional clinical trial in refractory cancer patients, Cell, Volume 188, Issue 4P1119-1136.E23February 20, 2025.

2. Tricking the Immune System: A New Approach to Targeting Cancer Cells by Mimicking Pig Organs, Insight, 22 Jan 2025, available from https://oncodaily.com/insight/immune-system-229351

freepik__conceptual-3d-model-of-a-human-cell-membrane-with-__67322

Magnesium for Stress and Brain Health: How This Essential Mineral Supports Mental Wellness

May 10, 2025

by official.ajinkya11@gmail.com with No Comment Lifestyle

Medically Reviewed By Dr. Bhavana Pandav Wagh (MBBS, MD-Psychiatry)

Introduction

Magnesium is an essential mineral that our body needs to function properly, but many people don’t get enough of it. This nutrient helps with a wide range of important processes, such as muscle movement, nerve function, and keeping our heart rhythm steady. When we don’t have enough magnesium in our system, it can lead to problems like tiredness, muscle cramps, trouble sleeping, and even low mood or anxiety. What makes it more challenging is that the symptoms of magnesium deficiency are very similar to the effects of stress like feeling tired, irritable, or anxious. Because of this overlap, it’s important to ensure we get enough magnesium through our diet or supplements, especially during stressful times. Taking steps to improve magnesium levels can support physical and mental health, helping us feel more balanced and energized overall.

The idea of a “vicious circle” between magnesium and stress, first explained by researchers Galland and Seelig in the early 1990s, highlights how closely these two are connected. They discovered that stress doesn’t just affect the mind—it also impacts the body by causing it to use up more magnesium. Over time, this can lead to a magnesium deficiency. But the cycle doesn’t stop there. When magnesium levels are low, the body becomes even more sensitive to stress, making a person feel more anxious, tense, or overwhelmed. This creates a loop where stress leads to magnesium loss, and low magnesium levels make stress harder to handle. Breaking this cycle is important for maintaining both mental and physical health, and it can often start with improving magnesium intake through food or supplements, especially during high-stress periods.

Stress is a natural reaction that helps us respond quickly to challenges or dangers, but in today’s fast-paced world, it has become a constant part of many people’s daily lives. While short-term stress can sometimes be helpful like pushing us to meet deadlines or stay alert chronic stress can take a serious toll on the body and mind. Prolonged stress can weaken the immune system, increase the risk of heart problems, disturb sleep, and contribute to anxiety or depression. Because of its wide-ranging effects, managing stress is more important than ever. Simple practices like regular exercise, mindfulness, proper sleep, and maintaining a balanced diet including nutrients like magnesium can make a big difference. Taking care of stress not only improves our mood and energy but also supports long-term health and well-being.

Why Magnesium?

Magnesium is an essential mineral that plays a key role in keeping the body functioning smoothly. It is the second most abundant mineral found inside our cells, after potassium, and is involved in hundreds of important chemical reactions. These reactions help the body produce energy, build proteins, and manage the genetic material in our cells. Magnesium is especially important for the health of our muscles and nerves, helping them work properly and stay in balance. It also supports a steady heartbeat and acts like a natural relaxant by helping control how calcium enters our cells. In addition, magnesium helps maintain the balance of other key minerals like sodium and potassium, which are necessary for proper fluid balance and nerve signals. By supporting all these functions, magnesium plays a vital role in overall health and stability of the body’s systems.

Magnesium’s Role in Stress and Brain Health

Enhancing Serotonin Production

Magnesium plays an important role in helping the body make serotonin, a chemical often called the “feel-good” hormone because it helps regulate mood, sleep, and emotions. To produce serotonin, the body uses an enzyme called tryptophan hydroxylase, and magnesium is needed for this enzyme to work properly. Without enough magnesium, the process of making serotonin may slow down. In addition to supporting serotonin production, magnesium also helps serotonin receptors in the brain work more effectively. This means that not only is more serotonin available, but the brain can also use it better. Together, these effects help promote a more stable mood, reduce feelings of anxiety, and support overall mental well-being.

Regulating Excitatory and Inhibitory Neurotransmission

Magnesium helps keep the brain’s communication system in balance by controlling the activity of important brain chemicals called neurotransmitters. One of these chemicals, glutamate, is an excitatory neurotransmitter that stimulates brain cells. While some stimulation is necessary for learning and memory, too much glutamate activity can over stimulate brain cells and lead to damage or increased anxiety. Magnesium plays a protective role by blocking NMDA receptors specific sites in the brain that glutamate uses to send signals. By doing this, magnesium prevents neurons from becoming overly excited, helping to maintain a healthy balance between stimulation and relaxation in the nervous system. This balance is essential for clear thinking, emotional stability, and overall brain health.

GABA Enhancement

Magnesium also plays a key role in supporting the brain’s calming system by enhancing the action of GABA (gamma-aminobutyric acid), the main inhibitory neurotransmitter. GABA helps quiet down brain activity, promoting relaxation, reducing anxiety, and supporting restful sleep. Magnesium supports GABAergic transmission by helping GABA bind more effectively to its receptors in the brain. This strengthens GABA’s calming effects, making it easier for the brain to manage stress and stay balanced. When magnesium levels are low, GABA function may be weaker, leading to increased feelings of nervousness or restlessness. By ensuring adequate magnesium, the brain can better regulate its excitability, promoting a sense of calm and emotional stability.

Modulating the Stress Response

Magnesium plays a crucial role in helping the body manage stress by regulating the activity of the hypothalamic-pituitary-adrenal (HPA) axis, which is the central system responsible for the stress response. When we experience stress, the HPA axis triggers the release of hormones like ACTH (adrenocorticotropic hormone) and cortisol, which prepare the body to respond to challenges. However, if this response remains active for too long, it can lead to harmful effects such as anxiety, fatigue, and even weakened immunity. Magnesium helps keep this system in check by reducing the release of ACTH and cortisol, preventing an exaggerated or prolonged stress response. By calming the HPA axis, magnesium supports a more balanced reaction to stress and helps the body return to a relaxed state more quickly.

Source: Dr. Eric Berg DC (Youtube) _ Magnesium’s effect on mood: Anxiety and Depression

Disclaimer: Video is for educational and informational purposes only.

Promoting Neuroprotection

Magnesium plays a protective and nourishing role in brain health by increasing levels of brain-derived neurotrophic factor (BDNF), a protein that supports the growth, survival, and repair of neurons. BDNF is essential for maintaining healthy brain function, enhancing learning, memory, and overall cognitive performance. Higher levels of BDNF are also linked to improved mood and emotional resilience, while low levels are often found in individuals with depression and other mood disorders. By boosting BDNF production, magnesium helps protect brain cells from damage and supports their ability to adapt and recover an effect that has been shown to resemble the action of antidepressant treatments. This makes magnesium a valuable nutrient not only for brain function but also for long-term mental health and emotional well-being.

Combating Oxidative Stress

Magnesium also plays an important role in defending the brain against oxidative stress, which occurs when there is an imbalance between harmful molecules called free radicals and the body’s ability to neutralize them. Free radicals can damage brain cells, leading to inflammation, aging, and the development of neurological disorders. Magnesium helps reduce the production of these harmful molecules and supports the body’s natural antioxidant defenses. By maintaining adequate magnesium levels, the brain is better protected from oxidative damage, which helps preserve mental clarity, reduce inflammation, and lower the risk of conditions like depression, cognitive decline, and neurodegenerative diseases. In this way, magnesium acts as a shield for brain health at the cellular level.

Conclusion
Maintaining sufficient magnesium intake is essential for supporting mental well-being, managing stress, and protecting overall brain health. From aiding in serotonin production and calming the nervous system to regulating the body’s stress response and protecting neurons from oxidative damage, magnesium plays multiple vital roles. It helps balance excitatory and inhibitory neurotransmission, boosts brain-protective factors like BDNF, and acts as a natural stress buffer by stabilizing hormone levels. Inadequate magnesium can lead to a cycle of stress sensitivity and neurological imbalance, making it crucial to include magnesium-rich foods such as leafy greens, nuts, seeds, and whole grains or supplements when needed. By ensuring optimal magnesium levels, we not only enhance brain function and mood but also strengthen the body’s resilience against stress and long-term mental health challenges

References

Magnesium, Fact Sheet for Health Professional, National Institute of Health, US Department of Health and Human Services, Available from https://ods.od.nih.gov/factsheets/Magnesium-HealthProfessional/

Al Alawi AM, Majoni SW, Falhammar H. Magnesium and Human Health: Perspectives and Research Directions. Int J Endocrinol. 2018 Apr 16;2018:9041694. doi: 10.1155/2018/9041694. PMID: 29849626; PMCID: PMC5926493.

Pickering G, Mazur A, Trousselard M, Bienkowski P, Yaltsewa N, Amessou M, Noah L, Pouteau E. Magnesium Status and Stress: The Vicious Circle Concept Revisited. Nutrients. 2020 Nov 28; 12(12):3672. Doi: 10.3390/nu12123672. PMID: 33260549; PMCID: PMC7761127.

The Vicious Circle between Stress and Magnesium; how stress increases magnesium loss and deficiency increases susceptibility to stress, 21 June 2024, Grassroot Health Nutrients Research Institute.

Yaribeygi H, Panahi Y, Sahraei H, Johnston TP, Sahebkar A. The impact of stress on body function: A review. EXCLI J. 2017 Jul 21;16:1057-1072. Doi: 10.17179/excli2017-480. PMID: 28900385; PMCID: PMC5579396.

Houston MC, Harper KJ. Potassium, magnesium, and calcium: their role in both the cause and treatment of hypertension. J Clin Hypertens (Greenwich). 2008 Jul;10(7 Suppl 2):3-11. Doi: 10.1111/j.1751-7176.2008.08575.x. PMID: 18607145; PMCID: PMC8109864.

Sidsel-Marie Glasdam, Stinne Glasdam, Günther H. Peters, Chapter Six – The Importance of Magnesium in the Human Body: A Systematic Literature Review, Editor(s): Gregory S. Makowski, Advances in Clinical Chemistry, Elsevier, Volume 73, 2016, Pages 169-193, https://doi.org/10.1016/bs.acc.2015.10.002

Cuciureanu MD, Vink R. Magnesium and stress. In: Vink R, Nechifor M, editors. Magnesium in the Central Nervous System [Internet]. Adelaide (AU): University of Adelaide Press; 2011. Available from: https://www.ncbi.nlm.nih.gov/books/NBK507250/

Forrest H. Nielsen, Chapter 31 – Relation between Magnesium Deficiency and Sleep Disorders and Associated Pathological Changes, Editor(s): Ronald Ross Watson, Modulation of Sleep by Obesity, Diabetes, Age, and Diet, Academic Press, 2015, Pages 291-296, https://doi.org/10.1016/B978-0-12-420168-2.00031-4.

Sartori SB, Whittle N, Hetzenauer A, Singewald N. Magnesium deficiency induces anxiety and HPA axis dysregulation: modulation by therapeutic drug treatment. Neuropharmacology. 2012 Jan; 62(1):304-12. Doi: 10.1016/j.neuropharm.2011.07.027. Epub 2011 Aug 4. PMID: 21835188; PMCID: PMC3198864.

Maryam Afsharfar, Mansour Shahraki, Mansour Shakiba, The effects of magnesium supplementation on serum level of brain derived neurotrophic factor (BDNF) and depression status in patients with depression,Clinical Nutrition ESPEN, Volume 42, 2021, Pages 381-386, https://doi.org/10.1016/j.clnesp.2020.12.022

Zheltova AA, Kharitonova MV, Iezhitsa IN, Spasov AA. Magnesium deficiency and oxidative stress: an update. Biomedicine (Taipei). 2016 Dec; 6(4):20. Doi: 10.7603/s40681-016-0020-6. Epub 2016 Nov 17. PMID: 27854048; PMCID: PMC5112180.

Moringa Oleifera Benefits: Why This Super Food is taking over Modern Nutrition?

May 8, 2025

by official.ajinkya11@gmail.com with No Comment Food Wellness

Written By: Pragati Ekamalli, B.Pharm

Clinical Studies Reviewed By: Vikas Londhe M.Pharm (Pharmacology)

Final Review By:

Samruddhi Jadhav B.Sc-Microbiology, MSc-Nutrition and Food Science (Clinical & Holistic Nutritionist)

Introduction

Over the past decade, there has been a growing craze of recognizing nutrient-rich plants as superfoods, following the popularity of chia seeds; the latest addition of plant in to this trend is moringa (Moringa Oleifera). Although moringa is an ancient plant with an exceptional nutritional profile, some of its nutritional values were unknown in the past, and hence, this plant was hidden from the diet and nutrition industry. Moringa is now often considered a ‘miracle tree’ or ‘tree of life’ and is at the vanguard in modern nutritional discussions.

What is Moringa Oleifera?

Moringa also known as ‘drumstick’ or ‘horseradish tree’ is a multipurpose tropical tree. It is largely used for food and has abundant medicinal and agricultural uses. Moringa is nutritious, fast-growing and drought-tolerant plant. It is ancient plant, rediscovered in the 1990s and its cultivation has since become popular in South Asia, particularly the Himalayan region, and is also cultivated across Africa, South America, and Southeast Asia, where it is among the most economically valuable crops. All parts of the plant have been used traditionally for centuries; however, the leaves have been gaining popularity in the modern diet and nutrition industry.

Powerhouse of Nutrition

Moringa leaves are rich in vitamins, minerals, and plant-based proteins. Moringa contains all nine essential amino acids, making moringa a rare source of complete plant-based proteins. Moringa is richly packed with several vitamins, including vitamin A (beta-carotene), vitamin C, vitamin E, and several B vitamins like vitamin B1 (thiamine), vitamin B2 (riboflavin), and vitamin B3 (niacin). Moringa is packed with minerals like

calcium, iron, magnesium, potassium, and zinc. Moringa leaves are known for their antioxidant potential, the powerful antioxidants like quercetin, chlorogenic acid, and beta-sitosterol, which are responsible for combating oxidative stress and inflammation. It is also low in fat and contains no harmful cholesterol.

Health Benefits

“Numerous studies have already been conducted, and some are still ongoing, to understand the health benefits of moringa. The hype around moringa is not speculative, but it is backed by scientific research.” However, in our research, it is found that, as moringa is a plant and no active substance has yet been isolated to perform clinical trials on humans, most of the studies were performed in animal models only, where different extracts were used for activity.

Skin Health

For skin health, moringa offers an array of benefits. Its antioxidant properties help protect skin cells from free radical oxidative damage. As per one study, moringa slows down signs of aging such as wrinkles and loss of elasticity. Clinical studies have shown that topical application of moringa extracts can improve skin firmness and hydration. Additionally, its anti-inflammatory and antimicrobial properties make it effective in treating acne, eczema, and minor wounds. By reducing inflammation and promoting collagen synthesis, moringa supports faster skin healing and a clearer complexion.

According to one detailed study performed in China and published in Frontiers in Nutrition, moringa leaf extracts possessed promising anti-elastase and anti-collagenase activities.

Another study published in ‘Biomedicine and Pharmacotherapy’ related to the stem extract of the moringa plant shows protective effects against epidermal oxidative stress injury induced by hydrogen peroxide in keratinocytes by UVB radiation in mice.

Hair Health

In hair care, moringa is equally beneficial. It nourishes the scalp and strengthens hair follicles due to its rich content of amino acids, iron, and vitamins, which are vital for hair growth.

One study published in the Pharmacognosy journal found that the seed oil of moringa possesses anti-alopecia activity. The compounds like phytosterol and fatty acid are responsible for the anti-hair fall effect. Moringa’s anti-hair fall effect works by inhibiting the 5α-reductase enzyme, thus preventing the formation of dihydrotestosterone (DHT) and its binding to androgen receptors in hair follicles. This action helps prevent hair loss. Additionally, other components such as tocopherols function as antioxidants, protecting the scalp and promoting healthy blood circulation, which aids in delivering essential nutrients to the hair. This action can be beneficial in maintaining scalp health and reducing dandruff and itchiness.

Source: Freepik.com

Source: Isagenix® International (YouTube) _ Moringa Benefits Documentary, “The Miracle Tree”

Diabetes Mellitus

Moringa is believed to help in managing diabetes by lowering blood sugar levels, improving insulin sensitivity, and reducing inflammation. Several studies have been published regarding the anti-diabetic potential of moringa, including various parts of the plant. On retrospective review published in the journal Nutrients, the studies conducted in animals for antidiabetic effects, and some human trials as well. Most of the animal models show a significant reduction in glycemia and glucose tolerance test. However, as DM is a chronic disease, long-term studies are needed. On the contrary, human trials are not consistent with animal studies, and most of the trials do not show an effect on glycemia; only two studies have shown the anti-diabetic effect. Hence, more detailed and deep research is needed to establish this connection.

Rheumatoid Arthritis

Moringa oleifera contains bioactive compounds like quercetin, kaempferol, and chlorogenic acid, which exhibit strong anti-inflammatory properties. A study published in the Journal of Pharmaceutical Sciences and Research shows that Moringa extract reduced inflammation in rat models of arthritis by decreasing inflammatory markers like TNF-α and IL-1β. Rheumatoid arthritis involves dysregulation of the immune system. Some compounds in Moringa may modulate immune responses. One study shows that Moringa seed extract modulated both humoral and cell-mediated immune responses in mice, published in the Indian Journal of Physiology and Pharmacology.

Bone Health

Moringa potentially reduces bone loss associated with oxidative stress and inflammation. Incorporating moringa into the diet may support bone health and help prevent conditions like osteoporosis, especially in aging populations. Moringa oleifera leaf extract has been shown to significantly enhance bone regeneration in critical-sized mandibular defects in rabbits, its osteogenic effects by stimulating osteoblast differentiation, reducing oxidative stress and inflammation, and inhibiting osteoclast activity. These findings support Moringa’s potential as a cost-effective, natural osteopromotive agent for bone regeneration.

Cardiovascular Health

Moringa leaves are rich in antioxidants like quercetin, chlorogenic acid, and beta-carotene. These compounds help reduce oxidative stress and inflammation, both key contributors to cardiovascular disease. Some animal and human studies have shown that Moringa can help reduce total cholesterol, low-density lipoprotein (LDL), and triglycerides. Moringa exhibits mild diuretic properties and vasodilatory effects, which can help lower blood pressure.

Eye Health

Moringa leaves are a notable source of beta-carotene. Gram for gram, moringa leaves can have more beta-carotene than carrots, depending on their preparation and freshness. One study investigated the moringa stem’s effect on cataract formation in mice induced by oxidative stress. The result showed that Moringa stem alleviates oxidative stress-induced cataract formation, and the mechanism of the effect is predominantly related to its improvement of the endogenous antioxidant system in the lens.

Anaemia

Moringa leaves are an excellent source of iron and vitamin C (which enhances iron absorption). Animal studies have shown that moringa leaf extracts can boost red blood cell count and hemoglobin levels. Another study showed that supplementation with moringa leaf powder has shown improvements in hemoglobin levels in children and pregnant women with anemia.

Functional Constipation

Moringa has positive effect on functional constipation and can cure functional constipation. One detailed study has been published in journal frontiers in microbiology showed that moringa significantly reduced constipation symptoms like delayed defecation, low fecal water content, and inhibited intestinal motility. High and medium doses of MOAE shortened the time to first stool, increased fecal number and water content, and improved gastrointestinal transit rate. Moringa restored the balance of gut hormones and neurotransmitters disturbed by loperamide.

Other health conditions

Moringa exhibits a collective therapeutic potential against asthma, bacterial infections, and liver diseases. Its anti-inflammatory and bronchodilator effects help alleviate asthma symptoms by reducing airway inflammation and improving respiratory function. The plant’s potent antimicrobial compounds, including isothiocyanates and flavonoids, combat a broad spectrum of bacterial pathogens, enhancing immune defense. Additionally, moringa demonstrates hepatoprotective activity by reducing oxidative stress and inflammation in the liver, thus supporting detoxification and tissue repair. Together, these multifaceted actions position moringa as a promising natural remedy for managing respiratory, infectious, and hepatic conditions.

Due to its combined properties such as boosting metabolism, improving gut health, providing antioxidants, and offering essential nutrients moringa is commonly used by many people as a natural aid for weight loss.

Conclusion

In conclusion, moringa stands out as a powerhouse of nutrition and a valuable addition to a balanced diet. Rich in essential vitamins, minerals, antioxidants, and amino acids, it supports immune function, reduces inflammation, and may help regulate blood sugar and cholesterol levels. Whether consumed as a fresh leaf, powder, or supplement, moringa offers a natural and accessible way to enhance overall health. As scientific interest in moringa continues to grow, its traditional uses are increasingly supported by modern research, making it not just a super food, but a symbol of how nature and science can work together for better health

References

Pareek A, Pant M, Gupta MM, et al, Moringa oleifera: An Updated Comprehensive Review of Its Pharmacological Activities, Ethnomedicinal, Phytopharmaceutical Formulation, Clinical, Phytochemical, and Toxicological Aspects. Int J Mol Sci. 2023 Jan 20;24(3):2098. Doi: 10.3390/ijms24032098. PMID: 36768420; PMCID: PMC9916933.

Lakshmipriya Gopalakrishnan, Kruthi Doriya, Devarai Santhosh Kumar, Moringa oleifera: A review on nutritive importance and its medicinal application, Food Science and Human Wellness, Volume 5, Issue 2, 2016, Pages 49-56, https://doi.org/10.1016/j.fshw.2016.04.001.

Islam Z, Islam SMR, Hossen F, et al, Moringa oleifera is a Prominent Source of Nutrients with Potential Health Benefits. Int J Food Sci. 2021 Aug 10; 2021:6627265. Doi: 10.1155/2021/6627265. PMID: 34423026; PMCID: PMC8373516.

Ali A, Akhtar N, Chowdhary F. Enhancement of human skin facial revitalization by moringa leaf extract cream. Postepy Dermatol Alergol. 2014 May; 31(2):71-6. Doi: 10.5114/pdia.2014.40945. Epub 2014 Apr 22. PMID: 25097471; PMCID: PMC4112252.

Xu Y, Chen G and Guo M (2022) Potential Anti-aging Components From Moringa oleifera Leaves Explored by Affinity Ultrafiltration With Multiple Drug Targets. Front. Nutr. 9:854882. doi: 10.3389/fnut.2022.854882

Yu Zhou, Wushuang Yang, Zhiqun Li, et al, Moringa oleifera stem extract protect skin keratinocytes against oxidative stress injury by enhancement of antioxidant defense systems and activation of PPARα, Biomedicine & Pharmacotherapy, Volume 107, 2018, Pages 44-53, https://doi.org/10.1016/j.biopha.2018.07.152

Korassa YB, Saptarini NM, Mustarichie R, Hendriani R. The Potential of Moringa (Moringa Oleifera Lamk) Seed Oil as Anti-Alopecia. Pharmacogn J. 2022;12(1): 379-387

Vargas-Sánchez K, Garay-Jaramillo E, González-Reyes RE. Effects of Moringa oleifera on Glycaemia and Insulin Levels: A Review of Animal and Human Studies. Nutrients. 2019 Dec 2; 11(12):2907. Doi: 10.3390/nu11122907. PMID: 31810205; PMCID: PMC6950081.

Alessandro Leone, Sara Di Lello, Simona Bertoli, Moringa oleifera leaf powder enhances glycemic control in sahrawi women with type 2 diabetes: Findings from a 3-month unblinded randomized controlled trial, PharmaNutrition, Volume 31, 2025, 100434, https://doi.org/10.1016/j.phanu.2025.100434.

Vanitha Reddy P, Asna Urooj , Sudha Sairam et al, Hypocholesterolemic Effect of Moringa oleifera Polyphenols in Rats Fed High Fat-Cholesterol Diet, Mal J Nutr 23(2): 473 – 478, 2017

Ghalia Shamlan, Doha M. Al-Nouri, Arwa A. Antiarthritic, anti-inflammatory activity of Moringa peregrina seed oil and leaves in Freund’s complete adjuvant-induced arthritis in rats, Journal of King Saud University – Science, Volume 33, Issue 3, 2021,101350, https://doi.org/10.1016/j.jksus.2021.101350

Mahdi HJ, Khan NAK, Asmawi MZB, et al,. In vivo anti-arthritic and anti-nociceptive effects of ethanol extract of Moringa oleifera leaves on complete Freund’s adjuvant (CFA)-induced arthritis in rats. Integr Med Res. 2018 Mar;7(1):85-94. Doi: 10.1016/j.imr.2017.11.002. Epub 2017 Dec 23. PMID: 29629295; PMCID: PMC5884001.

Elsadek, N.A., Aboukhadr, M.A., Kamel, F.R. et al. Moringa oleifera leaf extract promotes the healing of critical sized bone defects in the mandibles of rabbits. BDJ Open 10, 22 (2024). https://doi.org/10.1038/s41405-024-00201-y

Alia F, Putri M, Anggraeni N, Syamsunarno MRAA. The Potency of Moringa oleifera Lam. as Protective Agent in Cardiac Damage and Vascular Dysfunction. Front Pharmacol. 2022 Jan 24;12:724439. Doi: 10.3389/fphar.2021.724439. PMID: 35140601; PMCID: PMC8818947.

Qi L, Zhou Y, Li W, et al, Effect of Moringa oleifera stem extract on hydrogen peroxide-induced opacity of cultured mouse lens. BMC Complement Altern Med. 2019 Jun 21; 19(1):144. Doi: 10.1186/s12906-019-2555-z. PMID: 31226981; PMCID: PMC6588927.

Khanam M, Sanin KI, Ara G, et al, Effects of Moringa oleifera leaves on hemoglobin and serum retinol levels and underweight status among adolescent girls in rural Bangladesh. Front Nutr. 2022 Jul 22;9:959890. doi: 10.3389/fnut.2022.959890. PMID: 35938103; PMCID: PMC9353109.

Rotella R, Soriano JM, Llopis-González A, et al The Impact of Moringa oleifera Supplementation on Anemia and other Variables during Pregnancy and Breastfeeding: A Narrative Review. Nutrients. 2023 Jun 8;15(12):2674. doi: 10.3390/nu15122674. PMID: 37375577; PMCID: PMC10301989.

Padayachee, H. Baijnath, An updated comprehensive review of the medicinal, phytochemical and pharmacological properties of Moringa oleifera, South African Journal of Botany, Volume 129, 2020, Pages 304-316, https://doi.org/10.1016/j.sajb.2019.08.021

Gao X, Yang W, Li S, et al, Moringa oleifera leaf alleviates functional constipation via regulating the gut microbiota and the enteric nervous system in mice. Front Microbiol. 2023 Dec 20; 14:1315402. Doi: 10.3389/fmicb.2023.1315402. PMID: 38192287; PMCID: PMC10773919.

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Gold Nanoparticles Show Promise for Vision Restoration, Brown University Study Finds

May 8, 2025

by official.ajinkya11@gmail.com with No Comment Research

Medically Reviewed By Vikas Londhe M.Pharm (Pharmacology)

Researchers at Brown University have developed a groundbreaking vision restoration method that could help people regain vision loss due to retinal diseases like age-related macular degeneration (AMD) and retinitis pigmentosa. These common causes of vision loss damage the retina the light-sensitive layer at the back of the eye leading to progressive blindness. The innovative approach involves using gold nanoparticles, tiny biocompatible particles that can be safely introduced into the eye. When stimulated by near-infrared (NIR) laser light, these nanoparticles activate the remaining retinal cells, allowing them to send visual signals to the brain much like healthy photoreceptors do. This emerging technology has the potential to outperform existing retinal prosthetics by offering a minimally invasive, less painful alternative that could deliver higher-quality vision restoration. With its promise of improved outcomes, enhanced safety, and greater accessibility, this cutting-edge technique could transform the treatment of retinal degeneration and provide hope for millions living with severe vision impairment or blindness.

The Science behind the Innovation

In healthy eyes, specialized cells called photoreceptors rods and cones detect light and convert it into electrical signals. These signals are then passed to other key retinal cells, such as bipolar cells and ganglion cells, which relay the information to the brain, allowing us to perceive images. However, in degenerative eye diseases like age-related macular degeneration (AMD) and retinitis pigmentosa, these critical photoreceptors degenerate and eventually stop functioning, leading to progressive vision loss and, in severe cases, blindness. Remarkably, the inner retinal cells like bipolar and ganglion cells often remain intact and functional even after the photoreceptors are lost. Building on this insight, researchers at Brown University are developing an innovative vision restoration approach that bypasses the damaged photoreceptors entirely. By using advanced technology to directly stimulate the surviving inner retinal cells, this method could restore vision in individuals affected by retinal degeneration. This novel strategy has the potential to revolutionize vision loss treatment by targeting still-viable parts of the eye, offering a powerful alternative to traditional therapies and retinal implants.

Image Source: Brown University from an article: Golden eyes: How gold nanoparticles may one day help to restore people’s vision. Image Showing: How gold nanoparticles work and how it will create visual image in the mind of patient

The research team at Brown University has developed a cutting-edge technique involving the use of gold nanorods ultra-small, rod-shaped particles made of gold delivered directly into the eye through a minimally invasive procedure called intravitreal injection. These gold nanorods are precisely engineered to absorb near-infrared (NIR) light, a safe and effective wavelength that penetrates deep into the eye without causing damage, since it is minimally absorbed by water in the tissue. Once exposed to NIR light, the gold nanorods undergo a process called surface plasmon resonance, which generates localized and highly controlled heat. This subtle thermal energy can activate surrounding retinal nerve cells in two key ways: by inducing tiny electric currents across their membranes or by triggering specialized ion channels that respond to changes in temperature. This innovative approach offers a promising new method for restoring vision by reactivating the surviving retinal cells in individuals affected by degenerative eye diseases such as macular degeneration and retinitis pigmentosa. It also holds the potential to be a safer, more precise alternative to traditional retinal implants or surgical treatments.

Promising Results in Preclinical Studies

In experimental tests with mice suffering from retinal damage, the research team injected gold nanoparticles into the animals’ eyes and projected patterned near-infrared (NIR) laser light onto the retina. These light patterns, shaped like simple images, were designed to test whether the NIR light could stimulate the remaining healthy retinal cells. Using a powerful technique called calcium imaging; the scientists observed that the bipolar and ganglion cells key players in the visual signalling pathway responded to the laser patterns in a manner similar to how they would react to natural light in a healthy retina. Additionally, brain imaging revealed increased activity in the visual cortex, the region of the brain responsible for processing visual information. This indicated that the brain was once again receiving visual input, a strong sign that sight could potentially be restored. Even more promising, the gold nanoparticles remained stable and safely positioned within the eye for several months, with no signs of toxicity or inflammation. These results highlight the long-term potential of this non-invasive, laser-activated nanoparticle therapy as a safe and effective treatment for restoring vision in people affected by retinal degenerative diseases.

Advantages over Traditional Retinal Prosthetics

This new nanoparticle based method has several key benefits compared to current retinal prosthetic devices. First, it is minimally invasive unlike traditional devices that require complex surgery to implant electrodes; this approach only needs a simple injection into the eye, a common and low-risk procedure in eye care. Second, it offers much higher resolution. Existing devices are limited because they can only stimulate certain fixed spots in the retina, often resulting in blurry or narrow vision. In contrast, the nanoparticles can be targeted more precisely, even down to individual cells, and their stimulation pattern can be changed as needed, potentially restoring a full, detailed field of vision. Lastly, because it uses near-infrared (NIR) light, which doesn’t affect visible light, this method can work alongside any natural vision a person still has, making it a more flexible and user-friendly solution.

Future Outlook

Researchers aim to transform this groundbreaking vision restoration technology into a wearable device, such as smart glasses or high-tech goggles equipped with a camera and a near-infrared (NIR) laser system. The camera would continuously capture visual input from the surrounding environment, while the NIR laser projects precise light patterns onto the retina. These patterns would activate specially designed gold nanoparticles, allowing the user to perceive visual images without the need for invasive procedures. Although more research is needed to confirm long-term safety and effectiveness in larger animal models and eventually in humans, this innovative approach could revolutionize treatment options for millions suffering from retinal diseases and vision loss. By eliminating the need for genetic modification or complex surgical implants, this technology offers a potentially safer, more accessible and cost-effective alternative to current retinal prosthetics, paving the way for broader adoption and improved quality of life for patients worldwide.

Reference:

1. 1.Jiarui Nie, Kyungsik Eom, Hafithe M. AlGhosain, ntravitreally Injected Plasmonic Nanorods Activate Bipolar Cells with Patterned Near-Infrared Laser Projection ACS Nano 2025, 19, 12, 11823–11840, https://doi.org/10.1021/acsnano.4c14061

2. Golden eyes: How gold nanoparticles may one day help to restore people’s vision, 16 April 2025, Brown University.

3. Chen F , Si P , de la Zerda A , Jokerst JV , Myung D . Gold nanoparticles to enhance ophthalmic imaging. Biomater Sci. 2021 Jan 21;9(2):367-390. doi: 10.1039/d0bm01063d. Epub 2020 Oct 15. PMID: 33057463; PMCID: PMC8063223.

4. Natarajan S. Retinitis pigmentosa: a brief overview. Indian J Ophthalmol. 2011 Sep-Oct; 59(5):343-6. Doi: 10.4103/0301-4738.83608. PMID: 21836337; PMCID: PMC3159313.

5. Giuliana Gagliardi, Karim Ben M’Barek, Olivier Goureau, Photoreceptor cell replacement in macular degeneration and retinitis pigmentosa: A pluripotent stem cell-based approach, Progress in Retinal and Eye Research, Volume 71, 2019, Pages 1-25, https://doi.org/10.1016/j.preteyeres.2019.03.001

6. Treating retinal disease with tiny gold particles, 24 April 2025, Association of optometrist, available from https://www.aop.org.uk/ot/news/2025/04/24/treating-retinal-disease-with-tiny-gold-particles

7. NEI-funded researchers test new visual prosthesis system to restore vision, 18 April 2025, National Eye Institute, available from https://www.nei.nih.gov/about/news-and-events/news/nei-funded-researchers-test-new-visual-prosthesis-system-restore-vision

All posts by : official.ajinkya11@gmail.com

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Vyayama in Ayurveda: Principles, Benefits & Daily Routine for Physical Exercise

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

Importance in Modern Life

Key Principles of Vyayama in Ayurveda

Benefits of Vyayam as Described by Acharya Vagbhata

Who Should Avoid Exercise?

Guidelines for Practicing Exercise

Over-Exercise Side Effects

Conclusion

Reference

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Gold Nanoparticles Show Promise for Vision Restoration, Brown University Study Finds

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