The 65th annual meeting of the American Society of Hematology (ASH) began with a significant development as the FDA approved two gene therapies for sickle cell disease, marking a culmination of a decade of clinical trial work and three decades of basic science. Vertex Pharmaceuticals and Bluebird Bio, the manufacturers of these therapies, presented updated data at ASH.
While the FDA approvals took the spotlight, ASH23 featured other noteworthy studies, including research exploring the potential of CAR-T cell therapy in treating autoimmune diseases and an investigation into the disparities between clinical trials and the real-world effectiveness of drug regimens. Additionally, Pfizer conducted a study using social media to understand the challenges faced by individuals with sickle cell disease.
Unlocking the Potential of CAR-T Cell Therapy for Autoimmune Diseases
A small academic study in Germany presented new results at ASH, suggesting that CAR-T cell therapy, primarily used for blood cancers like leukemia and lymphoma, may be effective in treating autoimmune diseases. The study included 15 patients with lupus and other autoimmune conditions who have all remained in complete remission since receiving CD19 CAR-T cells. These engineered immune cells not only target cancer cells but also misfiring B cells responsible for inflammatory immune attacks in diseases like lupus.
The study followed patients for a median of 15 months after CAR-T infusions, during which all patients experienced positive outcomes and ceased taking immunosuppressive drugs. While some patients experienced mild cytokine release syndrome, no serious treatment-related adverse events were reported. Researchers hope that this approach could serve as an alternative to autologous stem cell transplants for life-threatening autoimmune diseases.
Controlled clinical trials are a powerful tool for drug researchers, but they may not fully reflect real-world patient experiences. Alissa Visram, a doctor at the Ottawa Hospital Research Institute in Canada, conducted research comparing the “efficacy” observed in clinical trials to the “effectiveness” of multiple myeloma drug regimens in the real world. The study found that six standard drug regimens performed worse in real-world settings than in Phase 3 clinical trials.
Real-World Treatment Outcomes and Patient Perspectives on Sickle Cell Disease Care
Median progression-free survival and overall survival were significantly shorter in the real world for these treatments. The findings underscore the importance of understanding treatment effectiveness outside of clinical trials, as these results can influence policymaking, treatment decisions, and patient care. Interestingly, one regimen, Bristol Myers Squibb’s Pomalyst with the steroid dexamethasone, performed better in the real world than in trials, possibly due to similar prior treatment exposures between real-world and trial controls.
Individuals with sickle cell disease often encounter obstacles when seeking proper care, particularly during acute pain crises. These crises may necessitate emergency room visits and potent painkillers like opioids, which can lead to misconceptions by healthcare providers and reluctance to prescribe medication. To gain insights into these challenges, researchers at Pfizer turned to social media, analyzing over 45,000 posts on platforms such as Instagram and TikTok related to sickle cell disease. The study aimed to capture candid patient experiences in a format where people are more likely to express themselves freely.
The analysis revealed common themes, including a “lack of empathy from healthcare providers” and discrimination in seeking care. Patients often felt that their symptoms were dismissed or underestimated by physicians. This innovative approach of utilizing social media offers a quantifiable method to understand patient perspectives, especially when traditional data collection methods are limited by geography or administrative burdens.
In conclusion, ASH23 showcased significant advancements in the treatment of sickle cell disease with FDA approvals of gene therapies, explored the potential of CAR-T cell therapy in autoimmune diseases, highlighted the disparities between clinical trials and real-world treatment effectiveness, and utilized social media to gain insights into the challenges faced by sickle cell patients. These studies contribute valuable knowledge to the field of hematology and patient care.
Resource: Biopharmadive, December 09, 2023
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