TREATING ONCO-PEDS WITH NOVEL THERAPY

A new type of cancer therapy recently approved by the U.S. Food and Drug Administration for children with leukemia who run out of options is changing medical practice and has triggered excitement in the normally reserved world of cancer science. The therapy, called Kymriah, comes from Novartis. It is the first in a novel class of treatments known as

CAR-Ts, chimeric antigen receptor T cells in which a patient’s own immune cells are withdrawn, engineered to target cancer cells, and then infused back into the body.

As a Novel drug, some patients experience side effects for the Kymriah. One in ten people died last month which have risen regarding how much researchers understand about CAR-Ts and how far the therapy still has to improve to become widely used. Kymriah is one of the most expensive drugs, so far priced about $475,000 for onetime treatment and that price could climb still higher to treat other cancers, industry leaders said last week. The Food and Drug Administration of U.S. is expected to approve Kymriah for use in some non-Hodgkin lymphoma patients. Researchers are also wondering whether some patients require boosters to keep the cancer cells under control after initial treatment.

The other major issue is, of course, safety. Although side effects with CAR-T are often less when compared with chemotherapy, the most surprising thing is that the cytokine release syndrome (CRS) an activation of the immune system which leads to death in rare cases. Doctors hope the cytokine response will be reduced if CAR-Ts are delivered earlier, before patients have been heavily treated with chemotherapy and when their immune systems are still relatively healthy. Drug companies’ hopes that CAR-Ts will eventually work as well on solid tumours as they do on liquid ones but that remains as a dream for the moment.

 Solid tumours by definition are much harder; these tumours are more intense and have defence mechanism than blood cancers, making it tougher for CAR-Ts to get through. And they lack a distinctive marker that would allow only cancer cells and not crucial healthy ones to be targeted and killed. Companies and researchers are now trying CAR-Ts in combination with other treatments such as the other major type of immune therapy, called checkpoint blockades.