Ramesh Jayaraman’s Post

❗A landmark FDA decision for cellular therapy For the first time, a therapy based on mesenchymal stromal cells (MSCs) has been approved by the FDA, for the treatment of acute graft-versus-host disease (GvHD) in paediatric patients. Ryoncil, an allogeneic bone marrow-derived MSC infusion, saw positive results in clinical trials. Over half of the 54 participants experienced either a complete or partial response after 4-8 weeks of infusions. Dr Diana HERNANDEZ, Anthony Nolan’s director of immune and advanced therapies, responded to the news: “This is a positive step forward for MSC-based therapies, and we’re especially excited to see an advanced therapy approved for the treatment of GvHD – a potentially devastating disease that urgently needs new treatments. At Anthony Nolan Research Institute we investigate the potential of MSCs as immunomodulatory therapies, and our Cell Therapy & Laboratory Services provide umbilical cord tissue to groups who are working to develop their own MSC-based therapies for a range of regenerative and immunomodulatory applications.” Learn about how we can support your MSC research and development: https://bit.ly/4gFsJoy Read the full FDA statement: https://bit.ly/3P2dSIT

📃Scientific paper: Proliferative glomerulonephritis with monoclonal IgG deposits in an adolescent successfully treated with daratumumab Ref.: Springer, 2024 Abstract: There is no specific treatment for proliferative glomerulonephritis with monoclonal IgG deposits (PGNMID), a disease that is very rare in the pediatric population. We report the case of a 15-year-old boy who presented with mildly reduced kidney function and nephrotic syndrome. Kidney biopsy revealed PGNMID with monoclonal deposits of IgG3 with kappa light chain restriction. Flow cytometry showed a significant CD38 plasma cell population in the peripheral blood in the absence of other signs of hematological malignancy. The patient was treated with a 6-month course of daratumumab, a monoclonal antibody targeting CD38. There was a significant reduction in proteinuria and normalization of kidney function. Based on positive experience with adults, daratumumab should also be studied in children with PGNMID. Continued on ES/IODE ➡️ https://etcse.fr/IKl ------- If you find this interesting, feel free to follow, comment and share. We need your help to enhance our visibility, so that our platform continues to serve you.

📃Scientific paper: Proliferative glomerulonephritis with monoclonal IgG deposits in an adolescent successfully treated with daratumumab Abstract: There is no specific treatment for proliferative glomerulonephritis with monoclonal IgG deposits (PGNMID), a disease that is very rare in the pediatric population. We report the case of a 15-year-old boy who presented with mildly reduced kidney function and nephrotic syndrome. Kidney biopsy revealed PGNMID with monoclonal deposits of IgG3 with kappa light chain restriction. Flow cytometry showed a significant CD38 plasma cell population in the peripheral blood in the absence of other signs of hematological malignancy. The patient was treated with a 6-month course of daratumumab, a monoclonal antibody targeting CD38. There was a significant reduction in proteinuria and normalization of kidney function. Based on positive experience with adults, daratumumab should also be studied in children with PGNMID. Continued on ES/IODE ➡️ https://etcse.fr/IKl ------- If you find this interesting, feel free to follow, comment and share. We need your help to enhance our visibility, so that our platform continues to serve you.

Exciting news for patients with inflammatory and immune conditions! The FDA has approved Ryoncil (remestemcel-L-rknd), the first Mesenchymal Stromal Cell (MSC) therapy, for pediatric steroid-refractory acute Graft Versus Host Disease (GVHD). This landmark approval offers new hope for patients and their families! 🚀 A major step forward for the field of cell therapy and regenerative medicine! #CellTherapy #RegenerativeMedicine #Innovation #FDAApproval https://lnkd.in/gDNkM6pY

#ChimericAntigenReceptor T-cell (CAR-T) therapy targeting CD19 has significantly advanced the #treatment of relapsed or refractory B-cell Acute Lymphoblastic #Leukemia (B-ALL) in adults. The U.S. Food and Drug Administration (FDA) has approved brexucabtagene autoleucel (brexu-cel) for this indication. 10 Key Takeaways: 1. Efficacy in Relapsed/Refractory Cases: CAR-T therapy has shown substantial effectiveness in adult patients with relapsed or refractory B-ALL, offering a viable treatment option where traditional therapies have failed. 2. FDA Approval: Brexucabtagene autoleucel (brexu-cel) is approved by the FDA for adult patients with B-ALL, marking a significant milestone in treatment availability. 3. Impact of Disease Burden: The success of CAR-T therapy is influenced by the patient's disease burden at the time of treatment, with lower disease burden correlating with better outcomes. 4. Durability of Remission: While many patients achieve remission, ongoing research is focused on understanding and enhancing the durability of these responses. 5. Safety Profile: CAR-T therapy can be associated with adverse effects, including cytokine release syndrome and neurotoxicity, necessitating careful patient monitoring. 6. Patient Selection Criteria: Identifying appropriate candidates for CAR-T therapy involves evaluating factors such as overall health, disease stage, and prior treatments. 7. Manufacturing Challenges: The production of CAR-T cells is complex and time-sensitive, requiring efficient manufacturing processes to meet clinical demands. 8. Future Directions: Research is ongoing to develop next-generation CAR-T therapies with improved efficacy, safety, and applicability to a broader patient population. 9. Combination Therapies: Combining CAR-T therapy with other treatment modalities is being explored to enhance therapeutic outcomes. 10. Access and Cost Considerations: Efforts are underway to address the high costs and accessibility challenges associated with CAR-T therapy to make it more widely available to patients in need. #CARTCellTherapy #BCellALL #LeukemiaTreatment #Immunotherapy #FDAApproval #Oncology #CancerResearch #PatientCare #MedicalInnovation #Hematology Source: https://lnkd.in/dSkmAMV4

Pappas Capital portfolio company Minoryx Therapeutics has met the primary endpoint in its pivotal trial for pediatric patients with cerebral Adrenoleukodystrophy. https://lnkd.in/eBd-8r76

🚨 Big Moves in Autoimmune Cell Therapy 🚨 Bristol Myers Squibb just unveiled exciting early data for its CAR-T therapy in autoimmune diseases at the American College of Rheumatology annual meeting, highlighting the growing momentum in this space. - All seven lupus patients treated so far showed clinical responses, with improvements seen as early as one month. - A notable case: one patient became pregnant post-therapy, with no lupus flares during pregnancy—suggesting the treatment doesn’t impair fertility and can provide durable disease control. - Safety is under close watch. BMS reported one case of transient grade 3 ICANS, which fully resolved, as the field navigates risks tied to this cutting-edge approach. BMS joins Cabaletta Bio and Kyverna Therapeutics in advancing cell therapy for autoimmune diseases, transforming it into one of biopharma’s most exciting frontiers. As George Schett noted: “Two years ago, nobody spoke about this. Now it’s on everyone’s radar.” What excites you most about the potential of cell therapy in autoimmune diseases?

🚨Large Data Set Sheds Light on Rate of CAR-T Side Effects🚨 “Cytokine release syndrome (CRS) is the most commonly observed adverse event associated with chimeric antigen receptor T-cell (CAR-T) therapy. However, true rates of CRS have been difficult to ascertain due to the small sample sizes of CAR-T therapy clinical trials. A new analysis based on Medicare data may offer a more reliable picture of CRS occurrence—and road map for strategies to prevent it.” #cancernews #oncologynews #oncology #cancercare #cancerresearch https://lnkd.in/eUKEsP9N

BRAF V600 mutation is indeed found in approximately 5-10% of pediatric high-grade gliomas (pHGGs). Targeted therapies using BRAF inhibitors (e.g., dabrafenib) and MEK inhibitors (e.g., trametinib) have shown promise in clinical trials. Despite challenges such as high drug costs in resource-limited settings like India, incorporating targeted therapies should be prioritized in relapsed or refractory cases. In the past two years, we identified BRAF V600E mutations in 3 out of 25 pediatric high-grade glioma cases, with one patient experiencing relapse. Integrating NGS in brain tumor diagnostics should therefore be a fundamental approach to enable precise, mutation-driven treatment planning and improve outcomes. #braintumor#PeditrichGG Dr. Rahul Bhargava. Shrinidhi Nathany Sunisha Arora SWATI BHAYANA https://lnkd.in/g8ivfhCk

Acelyrin announced additional data from its phase 2 clinical trial as well as the design for its phase 3 LONGITUDE program of lonigutamab in thyroid eye disease (TED) patients. Lonigutamab is a subcutaneously (SC) delivered humanized IgG1 monoclonal antibody targeting the insulin-like growth factor-1 receptor (IGF-1R). Furthermore, the characteristics of lonigutamab that enable subcutaneous delivery also enable the potential for longer-term dosing and allow the potential to minimize exposures relative to IV therapy which the company believes will “improve depth and durability of clinical response.” Read More: https://ow.ly/7yEa50UBLmz