ASH 2019: Early Results With Multiplexed Genetic Engineering in Treating Multiple Myeloma
Posted: Monday, December 9, 2019
Early results of a phase I trial presented at the 2019 American Society of Hematology (ASH) Annual Meeting & Exposition in Orlando, Florida (Abstract 49), suggest that a novel multiplexed genetic engineering approach in patients with multiple myeloma and sarcoma appears to be clinically safe and feasible. According to the study authors, this is the first-ever clinical trial of this treatment in humans in the United States, and the preliminary results provide optimism for its clinical future.
“This trial is primarily concerned with three questions: Can we edit T cells in this specific way? Are the resulting T cells functional? And are these cells safe to infuse into a patient? These early data suggest that the answer to all three questions may be yes,” Edward A. Stadtmauer, MD, FACP, of the University of Pennsylvania, Philadelphia, stated in a Penn Medicine press release.
The approach was tested on three patients: two patients with multiple myeloma and a single patient with myxoid/round cell liposarcoma. The investigators used CRISPR/Cas9 technology editing to remove three genes and then infuse the cells back into the patients and target the antigen NY-ESO-1.
The treatment approach was considered to be well tolerated by all three patients, with no reports of neurotoxicity or cytokine-release syndrome. After 60 days, one patient experienced disease progression. A second patient, who received 1 unit of packed red blood cells, achieved stable disease after 90 days. It was too early to evaluate the third patient. According to peripheral blood and tumor biopsies in the two evaluable patients, the investigators observed in vivo expansion, stable persistence, and tumor targeting, with no serious side effects.
Disclosure: For full disclosures of the study authors, visit ash.confex.com.