Novel leukemia therapy aims to treat sick kids before time runs out
An experimental new treatment for aggressive leukemia in children combines CAR-T therapy and CRISPR to make "off the shelf" treatments, cutting down the wait time.
A decade ago, CAR T cell therapy changed the world of cancer treatment, offering a personalized approach to patients with blood cancers like leukemia.
But getting that specialized treatment to patients is costly and time consuming. It can take up to two months to harvest a patient's T cells and reprogram them into cancer-fighters — a nonstarter for many young patients with aggressive cancer.
These patients "don't have months or years to live," said Dr. Mohamed Kharfan-Dabaja, director of Blood and Marrow Transplantation and Cellular Therapies at the Mayo Clinic in Florida. "They have weeks." Kharfan-Dabaja was not part of the new research.
First child to receive CAR T cell therapy is 10 years cancer-freeMay 11, 202206:17A novel approach to CAR T (chimeric antigen receptor T cell) therapy aims to cut down that turnaround time significantly. Instead of reprogramming each patient's cells, researchers are testing the safety of using universal, or "off the shelf," CAR T cells from other patients, preprogrammed to fight cancer. These cells are then tweaked further using another gene-modifying technology — CRISPR — to ensure they are not rejected by the patient's own immune system.
Scientists at University College London in the United Kingdom tested the safety of the experimental approach in six children — mostly toddlers — with advanced leukemia. The research was published Wednesday in the journal Science Translational Medicine.
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