ten years ago, CAR T cell therapy changed the world of cancer treatmentoffers a personalized approach to patients with hematologic cancers, including leukemia.
However, it is costly and time consuming to provide professional care to patients. It can take up to two months to harvest a patient’s T cells and reprogram them into cancer-fighting cells. beginner For many young patients with advanced cancer.
These patients “don’t have months or years to live,” said Dr. Mohamed Kalfan Dhabaja, director of blood and bone marrow transplantation and cell therapy at the Mayo Clinic in Florida. “They have weeks.” Kharfan-Dabaja was not part of the new study.
A new approach to CAR T (chimeric antigen receptor T cell) therapy aims to significantly reduce its turnaround time. Instead of reprogramming each patient’s cells, researchers explore the safety of using universal or ‘off-the-shelf’ CAR T cells from other patients, pre-programmed to fight cancer. These cells will be further fine-tuned using another genetic modification technique — Crisper — to avoid being rejected by the patient’s own immune system.
Scientists at University College London, UK, tested the safety of an experimental approach in six children, mostly infants, with progressive leukemia.The study was published Wednesday in the journal Science Translational Medicine.
Conventional treatments, such as chemotherapy, were already ineffective in the six children in this Phase 1 clinical trial. The goal was to determine whether the new approach is safe. Scientists will determine whether it works in future large-scale studies.
“These are very difficult children to treat,” said study author Wasim Qasim, professor of cell and gene therapy at Great Ormond Street Children’s Hospital in London.
The goal of new therapeutic approaches is not necessarily curative. The alternative is to bring the patient into remission so that he or she is fit enough for a bone marrow stem cell transplant.
The new approach is a “bridge to transplant,” said Dr. Stephen Gottschalk, director of the Division of Bone Marrow Transplants and Cell Therapy at St. Jude Children’s Research Hospital in Memphis. “If you’re not in remission before transplant, your chances of recurrence are probably 80%.”
Gottschalk, who was not involved in the trial, called the new research “exciting.”
It takes about 12 days to modify the harvested T cells with CRISPR, a genetic tool that lets you cut and paste pieces of DNA, and prepare them for use, Qasim said. Wait times can be even shorter for most patients. Only the time required to thaw prepared frozen T cells.
“If we want these treatments to be available globally, off-the-shelf products will be the way to deliver them,” said Qasim.
The ‘off-the-shelf’ approach also ensures that there are enough T cells for treatment. “These infants are so small that it is very difficult to actually collect the cells,” Gottschalk said.
Children in the trial experienced no dangerous side effects from the experimental treatment. One specific side effect associated with CAR T therapy was cytokine storm, and it can be fatal. This occurs when the body’s immune system is disrupted in response to treatment.
Four children eventually died, not due to treatment or cytokine storm. Nine months after treatment and her 18 months, two of hers are in remission and are living well, Kassim said.
Early results are encouraging, said Chris Mahadeo, Ph.D., associate professor of pediatric patient care at the University of Texas MD Anderson Cancer Center in Houston.
“The idea is to give all these children a full life,” said Mahadeo, who was not involved in the study. “That’s the promise we’re working on. “
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