Dr Choi on Advances With Cell Therapy in Hematologic Malignancies

“We have to think [about] what tools are out there. What tools can help us really advance these therapies and get to our goal of ending or eradicating cancer?”

Yeong “Christopher” Choi, PhD, MBA, senior vice president of Industry Partnerships, an associate professor of oncology in the Department of Medicine, and the technical director of the GMP Facility in the Center of Immunotherapy at Roswell Park Comprehensive Cancer Center, discussed the ongoing development of biomedical technologies—including CRISPR editing and CAR T-cell therapy—for patients with hematologic malignancies and other diseases.

One of the most transformative developments in the field of biomedical research has been the advent and application of CRISPR-based gene, Choi began. CRISPR represents a major technological advancement with broad potential across multiple domains, including the management of hematologic malignancies, solid tumors, regenerative medicine, and autoimmune diseases, he stated. These innovations are part of a larger collection of advancements in oncology, he noted.

Notably, the cell-based gene therapy exagamglogene autotemcel (exa-cel; Casgevy) was one of the first 2 gene therapies to receive FDA approval in December 2023, for the treatment of patients with sickle cell disease ages 12 years and older with recurrent vaso-occlusive crises. Exa-cel was also the first FDA-approved therapy utilizing CRISPR/Cas9 genome editing technology to modify patients’ hematopoietic stem cells. CRISPR/Cas9 can be used to cut targeted areas of DNA, enabling the removal, addition, or replacement of DNA. The modified hematopoietic stem cells are then transplanted back into the patient.

The overarching goal of developing these therapeutic platforms is to improve treatment outcomes and ultimately eradicate disease, Choi emphasized. As such, understanding their contribution to achieving these therapeutic objectives will better inform their integration and application in clinical practice, according to Choi. Overall, enhanced cellular therapies, such as armored CAR T-cell therapies and CRISPR gene editing, are promising and exemplify the potential of next-generation modalities to transform clinical care, he concluded.