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Exciting news in cell therapy - FDA announced approval of Ryoncil, an allogeneic (donor) bone marrow-derived mesenchymal stromal cell therapy for the treatment of steroid-refractory acute graft-versus-host disease in pediatric patients. This is the first approval of an MSC cell therapy.

Revolutionizing Autoimmune Disease Treatment with CAR-T Cell Therapy CAR-T cell therapy is emerging as a groundbreaking approach in treating autoimmune diseases, offering new hope for patients with conditions like lupus, multiple sclerosis, and rheumatoid arthritis. This innovative therapy precisely targets autoreactive B and T cells, the key drivers of autoimmune disorders. Recent clinical trials have shown impressive results, demonstrating significant symptom improvement and disease control that often surpasses traditional treatments. For example, in multiple sclerosis, CD19-targeting CAR-T cells have been explored in clinical trials. One study reported increased walking distance in MS patients treated with CAR-T cells, with minimal side effects observed. Additionally, preclinical studies have shown promise using CAR-engineered regulatory T cells (CAR-Tregs) targeting myelin oligodendrocyte glycoprotein (MOG) to suppress autoimmune responses in MS models. Clinical trials for systemic lupus erythematosus (SLE) have also yielded promising results. In one case, a patient treated with CD19-targeting CAR-T cells achieved clinical remission with no signs of relapse for up to 18 months. Another trial using dual CD19 and BCMA-targeting CAR-T cells in SLE patients reported deep B cell depletion and normalization of complement levels. These examples highlight the potential of CAR-T cell therapy to offer durable remission without frequent dosing. As research progresses, CAR-T cell therapy has the potential to transform autoimmune disease treatment, offering new hope and improved quality of life for millions of patients worldwide. #CARTcellTherapy #AutoimmuneDiseases #ImmunologyInnovation #MultipleSclerosisTreatment #LupusResearch #PrecisionMedicine #CellularTherapy #BiotechAdvances #ImmunotherapyBreakthrough #AutoimmuneRemission Reference: Li, Y.R., Lyu, Z., Chen, Y., Fang, Y., & Yang, L. (2024). Frontiers in CAR-T cell therapy for autoimmune diseases. Trends in Pharmacological Sciences, 45(9), 839-857. https://lnkd.in/eBBp3kf9

Great news for the MSC field! FDA Approves First Mesenchymal Stromal Cell Therapy to Treat Steroid-refractory Acute Graft-versus-host Disease! https://lnkd.in/d7k6b9Fc #MSCs #GvHD

There are several merits of #decentralized #manufacturing in #cell therapy: * Reduced #VeintoVein Time: Speeds up the process from cell collection to reinfusion, crucial for timely patient treatment. * #CostEfficiency: Lowers logistics and transportation costs by producing therapies closer to patients. * #ImprovedAccessibility: Enables more hospitals and clinics to offer advanced cell therapies, increasing patient access. We are now starting to see data reflecting it as #Orgenesis recently announced promising early clinical results for its CD19 CAR-T therapy, ORG-101, which is designed with a decentralized production model in mind. The therapy has shown impressive complete response rates of 82% in adults and 93% in pediatric patients with #CD19+ #Acute Lymphoblastic Leukemia. Additionally, the incidence of severe Cytokine Release Syndrome (CRS) was notably low, at 2% in adults and 6% in pediatric patients. This #decentralized approach aims to make CAR-T therapies more accessible and cost-effective by streamlining production and reducing treatment costs. #celltherapy #manufacturing #PointofCaremanufacturing #PatientAccess #CD19 #Leukemia #Orgenesis #CART #Decentralized #ImprovedAccessibility

Bristol Myers Squibb and Biotechs Push Boundaries in Autoimmune Disease Treatment with CAR-T Cell Therapy Exciting advancements are unfolding in the treatment of autoimmune diseases! At the recent American College of Rheumatology annual meeting, Bristol Myers Squibb (BMS), Cabaletta Bio, and Kyverna Therapeutics shared promising clinical data on their innovative cell therapies. 🔬 Bristol Myers Squibb presented the first data from their clinical trial using an experimental CAR-T cell therapy, adapted from their approved blood cancer treatment Breyanzi but with a streamlined five-day manufacturing process. Remarkably, all seven lupus patients treated showed clinical responses after just one month of follow-up. In a noteworthy case, one patient became pregnant shortly after treatment, suggesting the therapy doesn't impair fertility—a significant consideration for many patients. 💡 Rheumatologist Georg Schett highlighted the rapid growth in this field: "Two years ago at the ACR, nobody spoke about cell therapy. And now this is really something everybody knows about." 🤝 Cabaletta Bio reported that eight patients, including four with lupus, were able to discontinue immunosuppressants after receiving their cell therapy. 🌐 Kyverna Therapeutics announced they've treated over 50 patients across more than 15 autoimmune conditions with their experimental CAR-T cell therapy. Focusing on diseases like lupus nephritis, systemic sclerosis, and multiple sclerosis, they've seen positive responses, with patients no longer requiring immunosuppressants post-treatment. Importantly, Kyverna reported no severe cases of cytokine release syndrome or neurotoxicity (ICANS) across their patient group. 🚀 With industry veterans like Warner Biddle joining Kyverna as CEO, there's a clear momentum towards advancing these therapies through clinical trials and regulatory approvals. Biddle emphasized the shift towards focusing on trials that "can get us to the first BLAs and getting access to a broader set of patients." The Bottom Line: These developments represent a significant leap forward in treating autoimmune diseases. Using CAR-T cell therapy—a method previously successful in oncology—could revolutionize patient outcomes and quality of life. Let's keep an eye on these groundbreaking efforts that bring hope to millions affected by autoimmune conditions.

🔊 CGT Alert: The U.S. Food and Drug Administration (FDA) has approved #Mesoblast’s cell therapy, Ryoncil (remestemcel-L-rknd), for the treatment of steroid-refractory acute graft-versus-host disease (SR-aGVHD) in pediatric patients aged two months and older. ☝ This marks the first approval of a mesenchymal stromal cell (MSC) therapy for this condition. ☝ Ryoncil is derived from the bone marrow of healthy adult donors and contains MSCs, which can differentiate into various cell types and play multiple roles in the body. This approval is significant as SR-aGVHD is a serious and life-threatening complication that can occur after allogeneic hematopoietic stem cell transplantation (allo-HSCT), where the donor’s immune cells attack the recipient’s tissues. #Celltherapy #FDAapproval #Mesenchymalcells #GVDH

🎉Another BCMA CAR-T therapy for multiple myeloma hits the market in China! - Short-term efficacy ✨ 💪In recent years, CAR-T cell therapy has achieved tremendous success in treating hematologic malignancies. Over the past 11 years, China has seen a surge in clinical trials evaluating the safety and efficacy of CAR-T therapy. China has now surpassed the United States as a major force in CAR-T clinical research. 🌟 🚀In the field of multiple myeloma (MM), China has gained regulatory approval from the NMPA for two fully human BCMA CAR-T therapies for the treatment of relapsed/refractory MM (R/R MM). These therapies are Equecabtagene Autoleucel and Zevorcabtagene Autoleucel. 💉 🩺While both therapies target the same BCMA and share a common co-stimulatory domain, there are some fundamental differences between them. Zevorcabtagene Autoleucel includes patients with stable disease after previous treatment, whereas Equecabtagene Autoleucel focuses on patients with relapsed or progressive MM. 🎯 📈Looking at the short-term efficacy, the median time to peak CAR-T cells in peripheral blood is 12 days for Equecabtagene Autoleucel and 14 days for Zevorcabtagene Autoleucel. This indicates that the former can rapidly exert its effect, potentially laying the foundation for achieving clinical remission faster and better. 💥 📆Clinical trial results seem to support this notion, with Equecabtagene Autoleucel showing a median time to response (TTR) of 15 days, compared to 29 days for Zevorcabtagene Autoleucel. At the 3-month follow-up, the complete remission rates were 40.3% for Equecabtagene Autoleucel in the registered clinical trial FUMANBA-1 1b/2 phase, surpassing the rate of 28.3% observed in the registered clinical trial LUMMICAR-1 phase 2 for Zevorcabtagene Autoleucel. 📊 💉It's worth noting that the infusion dose for Equecabtagene Autoleucel is 1x106 cells/kg, while the total infusion dose for Zevorcabtagene Autoleucel is 150x106 cells, which translates to double the dose of Equecabtagene Autoleucel for patients with a weight of 70kg-75kg. Additionally, the reinfusion dose for Zevorcabtagene Autoleucel is not fixed and needs to be increased to 1.8x108 cells for patients weighing ≥80kg. ⚖️ 🌈With the increasing availability of these groundbreaking BCMA CAR-T therapies, the landscape of multiple myeloma treatment continues to expand, offering more possibilities and choices. 🔬 🎉🎉To assess whether the condition is suitable for CAR-T or clinic therapy, you can submit Advanced Medicine in China for preliminary evaluation! WhatsApp: 137 1795 9070 Email: doctor.huang@globecancer.com #BCMACART #MultipleMyeloma #Innovation #CART #CARTTherapy #chinesemedical

In recent years, CAR-T cell therapy targeting BCMA has emerged as a groundbreaking treatment for multiple myeloma, offering new hope to patients. At the 2024 EBMT Annual Meeting, Professor Qiu Lugui's team presented the latest results from the FUMANBA-1 study on China’s first BCMA-targeted CAR-T therapy, Iquilencel (CT103A). BCMA (B-cell maturation antigen) is a key target for multiple myeloma (MM). Soluble BCMA (sBCMA) in the blood reflects tumor burden and can interfere with BCMA-targeted therapies. Iquilencel was designed to minimize this impact through its carefully selected single-chain variable fragment (scFv). The FUMANBA-1 phase II study in Chinese patients with relapsed/refractory multiple myeloma (RRMM) showed that Iquilencel induces deep and durable responses, with a complete response (CR) rate of 82.4% and a 12-month progression-free survival (PFS) rate of 85.5%. This study explored whether baseline serum sBCMA levels affect clinical outcomes following Iquilencel infusion. ### Study Methods and Results The study measured serum sBCMA levels and monitored CAR transgene copy numbers. Baseline sBCMA levels were classified into high (≥225.1 ng/mL) and low (<225.1 ng/mL) groups. Results showed no significant differences in CAR-T cell expansion, AUC during the first 28 days, or cell persistence between the high and low sBCMA groups. Patients with high baseline sBCMA levels had overall response rates (ORR) and ≥CR rates of 100% and 80%, respectively, compared to 97.8% and 84% in the low sBCMA group. There were no significant differences in minimal residual disease (MRD) negativity rates, 18-month sustained MRD negativity rates, PFS, and overall survival (OS) between the two groups. ### Conclusion The FUMANBA-1 study indicates that Iquilencel's efficacy is not influenced by baseline sBCMA levels, making it a promising treatment option for RRMM patients. Its unique properties enable Iquilencel to remain effective and persistent regardless of sBCMA levels. Professors Qiu Lugui and Li Chunrui highlighted that Iquilencel can overcome high baseline sBCMA challenges, providing significant and lasting responses for RRMM patients. These results support Iquilencel as an ideal treatment choice for RRMM, offering hope for more effective and long-lasting outcomes. ����To assess whether the condition is suitable for CAR-T or clinic therapy, you can submit Advanced Medicine In China for preliminary evaluation! WhatsApp: +8613717959070 Email: doctor.huang@globecancer.com #EBMT2024 #CAR_T #MultipleMyeloma #Iquilencel #EquecabtageneAutoleucel #sBCMA #CancerResearch #Immunotherapy #MedicalBreakthrough #Biopharmaceuticals

🌟 New CAR T-Cell Therapy Offers Hope for CNS Lymphoma Patients! 🌟 Central nervous system lymphomas (CNSL) have long posed significant treatment challenges due to their aggressive nature and high relapse rates. Traditional therapies often fall short, leaving patients with limited options. However, the recent approval of anti-CD19 CAR T-cell therapy is a game-changer! 🚀 This innovative treatment could revolutionize the landscape for CNSL patients, offering targeted approaches that aim to improve survival rates while minimizing side effects. 🔍 Key Insights: - CNSL Treatment Variability: Outcomes differ based on disease origin. - High Relapse Rates: Traditional therapies often lead to significant toxicity. - Promising Future: Ongoing clinical trials are crucial in establishing the efficacy and safety of CAR T-cell therapy. As research progresses, we remain hopeful that this breakthrough will lead to more personalized and effective treatment strategies in the fight against CNSL. 👉 Click on the link for more details on this exciting development! #CARtherapy #CNSL #CancerTreatment #ClinicalResearches #HealthcareInnovation #Immunotherapy #Pharma #MarketAccess #MarketAccessToday

It is such a great new before holidays FDA Approves First Mesenchymal Stromal Cell Therapy to Treat Steroid-refractory Acute Graft-versus-host Disease Steroid-refractory acute graft-versus-host disease is a serious and life-threatening condition that can occur as a complication of allogeneic hematopoietic (blood) stem cell transplantation (allo-HSCT). In allo-HSCT, a patient receives hematopoietic stem cells from a healthy donor to replace their own stem cells and form new blood cells, a procedure often done as part of treatment for certain types of blood cancers, blood disorders or immune system disorders. “Steroid-refractory acute graft-versus-host disease can have significant, wide-ranging health consequences, including damage to multiple organs, reduced quality of life and risk of death in affected patients,” said Nicole Verdun, M.D., director of the Office of Therapeutic Products in CBER. “The FDA remains dedicated to helping address the urgent unmet needs of individuals with debilitating and deadly diseases, and today’s approval is an important step in that effort.” “We are pleased that FDA has accepted our BLA resubmission for review, and look forward to the potential approval of RYONCIL for children with SR-aGVHD,” said Mesoblast Limited CEO Dr. Silviu Itescu FDA Accepts Mesoblast’s Biologics License Application (BLA) for Ryoncil® in Children With Steroid-Refractory Acute Graft-Versus-Host Disease (SR-aGVHD) https://lnkd.in/eMvKG5C4 https://lnkd.in/e6MG6JRB