The United States Food and Drug Administration has just announced the very first approval for a cell-based gene therapy as a potential cancer treatment in the US. This week the FDA approved the drug Kymriah, which scientists call a “living drug” because it actually uses genetically modified human immune cells from patients, cells which attack cancer cells. Primarily, it has been approved to treat adults and children who have (the blood and bone marrow cancer) lymphoblastic leukemia.
“This is a brand new way of treating cancer,” explains Dr. Stephan Grupp of Children’s Hospital of Philadelphia. He was the first doctor to treat a child with the new CAR-T cell therapy. The patient—a little girl—was very near death, but this treatment has proven successful and she has now been cancer free for five years!
CAR-T treatment works by using gene therapy techniques to activate T-cells—almost supercharge them—instead of attempt to fix the disease. Generally, cancer cells are pretty good at dodging T-cells, which are usually very proficient at killing invaders. The therapy, then, involves filtering these immune system soldiers from the blood and then, essentially, reprogram them as “chimeric antigen receptor” cells that actually target cancerous cells.
That is the good news.
The bad news—or, rather, the rub, at least—is that Novartis reports it would probably charge upwards of $475,000 for each treatment, but you must also consider that each treatment has to be made from scratch and customized to the patient. Also, the company essentially issued a money-back guarantee, saying there would be no charge if the patient did not show any improvement within the first month of treatment.
All that in mind, FDA Commissioner Scott Gottlieb remains optimistic, saying, “We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer.”
Analysts said the eventual pricing of the Novartis treatment could be an advantage for Kite.
Since these therapies are “unbelievably effective” for leukemia, Novartis’ pricing power is high, said Thomas Shrader, biotechnology analyst at Stifel. That means Kite could piggyback off Novartis’ price, even though its therapy is aimed at non-Hodgkin’s lymphoma, which has a lower response rate to the therapy than leukemia does.
Scientists around the country also are trying to make CAR-T therapies that could fight more common solid tumors such as brain, breast or pancreatic cancers — a harder next step.