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Impressive results in a trial evaluating CAR-T in 15 autoimmunity patients. #cartcelltherapy #cartcells #cart #clinicalresearch #clinicaltrials #treatment #autoimmunedisease #autoimmunity

Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare disease with sudden onset symptoms, including nerve damage affecting movement, sensation, speech, breathing, and heart rhythm. Treatments such as glucocorticoids, plasmapheresis, and intravenous gamma globulin (IVIG) can help manage symptoms but cannot completely eradicate the disease. However, a research team has reported using BCMA-CD19 bispecific CAR-T cells to treat relapsed/refractory CIDP, restoring the balance of immune responses by temporarily and profoundly eradicating B cells and plasma cells. After being treated with this therapy, one patient made significant progress in functioning according to the INCAT disability and MRC scores. Remarkably, almost complete recovery of muscle power was observed 180 days after administration of CAR-T, along with the ability to walk again. This study, therefore, highlights the evolution of patients' symptoms after treatment and confirms the safety of CAR-T cell therapy for CIDP.?

Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare disease with sudden onset symptoms, including nerve damage affecting movement, sensation, speech, breathing, and heart rhythm. Treatments such as glucocorticoids, plasmapheresis, and intravenous gamma globulin (IVIG) can help manage symptoms but cannot completely eradicate the disease. However, a research team has reported using BCMA-CD19 bispecific CAR-T cells to treat relapsed/refractory CIDP, restoring the balance of immune responses by temporarily and profoundly eradicating B cells and plasma cells. After being treated with this therapy, one patient made significant progress in functioning according to the INCAT disability and MRC scores. Remarkably, almost complete recovery of muscle power was observed 180 days after administration of CAR-T, along with the ability to walk again. This study, therefore, highlights the evolution of patients' symptoms after treatment and confirms the safety of CAR-T cell therapy for CIDP.?

CD19 CAR-T therapy continues to show great promise in autoimmune diseases. Notably in this small cohort of patients with SLE, SSc and inflammatory myositis, 14 of 15 patients experienced clinical remission without additional immunosuppression. "Schett first began reporting findings with CAR-T treatment a couple years ago, with a publication in Nature Medicine describing positive results in five SLE patients followed for up to a year... This week, he gave new results for a total of 15, with diagnoses of inflammatory immune myositis (n=3) and systemic sclerosis (n=4). These were also the subject of the report from last November, but with additional follow-up. All of the SLE patients remain in clinical remission, Schett said. As well, all of those with systemic sclerosis have shown marked reductions in European Scleroderma Trials and Research Group Activity Index (EUSTAR AI) scores, with no need for corticosteroids or immunosuppressants. Meanwhile, two of the three myositis patients have also shown durable remission. The exception is one patient with a strong initial response who then relapsed about 16 months after treatment. While the precise reason for the late failure isn't clear, Schett noted that myositis is heterogeneous and it may be that some forms will be more responsive to CAR-T therapy than others." #medicalresearch #cellulartherapy #autoimmunedisease #rheumatology

"Aging in one organ can trigger a domino effect, ultimately leading to multi-organ failure" A fascinating study reveals that liver-induced senescence can spread throughout the body, triggering multi-organ dysfunction. The research highlights TGFβ signaling as a crucial driver of this systemic transmission, opening the door to potential therapies aimed at preventing multi-organ failure in severe liver diseases. https://lnkd.in/eYMrVJZu

The article explored the dual role of the #TGF-β signaling pathway in the development of aortic diseases and evaluated the clinical potential of TGF-β targeted therapy. ? Find out more: https://lnkd.in/gaCWP2df #IJDDP #aorticaneurysm

Proud to share our recent work on AMACR deficiency!?We show that this rare peroxisomal single enzyme deficiency can be considered as an adult slowly progressive disease with a predominant neurological phenotype. The main signs comprise retinitis pigmentosa, neuropathy, ataxia and cognitive decline. Recognition of the trident-like hyperintensity in the pons on FLAIR or T2-weighted images may facilitate prompt diagnosis. Check it out here:

What's new in our latest Pediatric & #Rare Liver Diseases News? 1?? GLI celebrates the repurposed use of Livmarli for pediatric rare liver disease #patients 2?? Learn about the latest treatment developments in #autoimmunehepatitis 3?? The U.S. FDA approves the first gene therapy for rare pediatric disease! And so much more! ?? Read all the updates about the cutting-edge technology in #RareLiverDisease that could improve prompt #diagnosis, ongoing feedback, and effective interventions: https://lnkd.in/g5-_iWpt #LiverHealth #PatientAdvocacy

it is an burning topic of discussion to put a new insight in various neurodiseases :How nature and nurture conspire to regulate brain development and plasticity, similarly the balance between neuroinflammation and neuroimmune process are most important part of discussion in the pathogenesis of various neuroinflammatory diseases