PHILADELPHIA – Researchers from the Perelman School of Medicine at the University of Pennsylvania and The Children's Hospital of Philadelphia (CHOP) will present the latest advances from their studies of personalized cellular therapies for blood cancers during the 58th Annual Meeting of the American Society of Hematology (ASH) in San Diego.
Highlights include early results from a study of chimeric antigen receptor (CAR) T cells engineered to target B-Cell Maturation Antigen (BCMA) in patients with multiple myeloma, and a 100 percent response rate among relapsed/refractory acute lymphoblastic leukemia (ALL) patients treated with a humanized version of CAR T cell therapy who had not received any type of personalized cellular therapy previously. Investigators will also present results from the first global multisite study of the cellular therapy known as CTL019 in pediatric and young adult patients with relapsed/refractory ALL, showing a high level of efficacy and a similar safety profile to that shown in the first trials of the therapy at CHOP. The team will present results from more than 50 patients treated at 25 sites across the world.
The latest study results build on Penn's work as leader in CAR T cell therapies, dating back to the start of the first Penn clinical trial of CTL019 for patients with chronic lymphocytic leukemia in 2010. The research team is led by Carl June, MD, the Richard W. Vague Professor in Immunotherapy in the department of Pathology and Laboratory Medicine in the Perelman School of Medicine at the University of Pennsylvania, and Director of the Center for Cellular Immunotherapies in Penn's Abramson Cancer Center.
Additional abstracts will also be presented related to animal studies to improve efficacy of CAR T cell therapies and decrease side effects associated with the treatments, and research on biomarkers to distinguish between sepsis and cytokine release syndrome following treatment with these personalized cellular therapies.
Clinical research highlights presented during the ASH meeting will include:
- Efficacy and Safety of CTL019 in the First US Phase II Multicenter Trial in Pediatric Relapsed/Refractory Acute Lymphoblastic Leukemia: Results of an Interim Analysis (Abstract #2801)
- Analysis of a Global Registration Trial of the Efficacy and Safety of CTL019 in Pediatric and Young Adults with Relapsed/Refractory Acute Lymphoblastic Leukemia (ALL) (Abstract #221)
- Efficacy of Humanized CD19-Targeted Chimeric Antigen Receptor (CAR)-Modified T Cells in Children and Young Adults with Relapsed/Refractory Acute Lymphoblastic Leukemia (Abstract #217)
- Treatment with Chimeric Antigen Receptor Modified T Cells Directed Against CD19 (CTL019) Results in Durable Remissions in Patients with Relapsed or Refractory Diffuse Large B Cell Lymphomas of Germinal Center and Non-Germinal Center Origin, "Double Hit" Diffuse Large B Cell Lymphomas, and Transformed Follicular to Diffuse Large B Cell Lymphomas (Abstract #3026)
- Chimeric Antigen Receptor Modified T Cells Directed Against CD19 (CTL019) in Patients with Poor Prognosis, Relapsed or Refractory CD19+ Follicular Lymphoma: Prolonged Remissions Relative to Antecedent Therapy (Abstract #1100)
- B-Cell Maturation Antigen (BCMA)-Specific Chimeric Antigen Receptor T Cells (CART-BCMA) for Multiple Myeloma (MM): Initial Safety and Efficacy from a Phase I Study (Abstract #1147)
- Pilot Study of Anti-CD19 Chimeric Antigen Receptor T Cells (CTL019) in Conjunction with Salvage Autologous Stem Cell Transplantation for Advanced Multiple Myeloma (Abstract #974)
Editor's note: The University of Pennsylvania has licensed technologies involved in this trial to Novartis. Some of the scientists involved in these trials are inventors of these technologies. As a result of the licensing relationship with Novartis, the University of Pennsylvania receives significant financial benefit, and these inventers have benefitted financially and/or may benefit financially in the future. Additional disclosure information is available in the meeting abstracts.