- Scientists developed a method to create cancer-fighting cells inside the body directly
- Current CAR-T therapy involves costly, lengthy lab reprogramming of immune cells
- The new approach treated leukaemia, multiple myeloma, and solid tumours in mice
In a major medical breakthrough, scientists have developed a method to create cancer-fighting cells directly inside the body, potentially replacing the current complex and expensive process of engineering them in a laboratory. Researchers at the University of California, San Francisco (UCSF), working alongside Gladstone Institutes, Duke University, and the Innovative Genomics Institute, successfully integrated a large DNA sequence at a precise, specific site in human T cells (a type of immune system cell).
Up until now, CAR-T cell therapy has been the primary treatment for certain blood cancers. In this procedure, doctors extract a patient's immune cells, ship them to a laboratory where they are reprogrammed to fight cancer. Afterwards, they are shipped back for infusion into the patient's bloodstream.
However, the procedure is highly expensive, costing around $400,00 to $500,000 whilst taking weeks. It also requires patients to undergo chemotherapy to clear space in their bone marrow for the new T cells, a punishing process that some cannot tolerate.
Justin Eyquem, the senior author of the new paper and an associate professor of medicine at UCSF, stated that developing cancer-fighting cells inside the body could be the "beginning of a big wave of new therapies that will be truly transformational and save a lot of lives".
"When you manufacture these cells outside the body, you can do a lot of quality control to make sure you only end up with re-engineered T cells," said Eyquem. "We can't do that inside the body, so we really needed to optimise the approach upfront to avoid genetically altering any other cells."
Eyquem highlighted that there has been a big push in the medical field in recent years to attempt to produce these cells in the body directly.
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Cancer Treated In Mice
In the study published in the journal Nature, scientists used the method to successfully treat aggressive leukaemia, multiple myeloma, and even a solid tumour in mice with humanised immune systems.
"By integrating a CAR transgene into a T cell-specific locus, we generate therapeutic levels of CAR T cells in vivo in humanised mouse models. These findings offer a pathway to more efficient, precise and widely accessible T cell therapies," the researchers stated.
It is also the first time that researchers used a "dual-particle" system where the new DNA only activates once it is correctly placed. This ensures that only the intended T cells become cancer fighters, significantly increasing safety and precision compared to earlier experimental attempts
Scientists are hopeful that the new method may lead to an off-the-shelf therapy, like a vaccine, that could one day be given to anyone with the same condition.
