U.S. approves second gene therapy for blood cancer

In News:

  • U.S. regulators approved a second gene therapy for a blood cancer, a one-time, custom-made treatment for aggressive lymphoma in adults.
  • The Food and Drug Administration allowed sales of the treatment from Kite Pharma.
  • It uses the same technology, called CAR-T, as the first gene therapy approved in the U.S. in August, a treatment for childhood leukemia from Novartis Pharmaceuticals.
  • In just several decades, gene therapy has gone from being a promising concept to a practical solution to deadly and largely untreatable forms of cancer.
  • The treatment, called Yescarta, will cost $373,000 per patient, according to drugmaker Gilead Sciences. Kite became a subsidiary of Foster City, California-based Gilead this month.

About CAR-T

  • CAR-T treatment uses gene therapy techniques not to fix disease-causing genes but to turbocharge T cells, immune system soldiers that cancer can often evade.
  • The T cells are filtered from a patient’s blood, reprogrammed to target and kill cancer cells, and then hundreds of millions of copies are grown.

Other Info:

  • Returned to the patient, all the revved-up cells can continue multiplying to fight disease for months or years. That’s why these immunotherapy treatments are called “living drugs.”
  • “Today’s approval of Yescarta is a very significant advance for lymphoma patients and for the cancer community as a whole.
  • Kite’s therapy is for patients with three types of aggressive, or fast-growing, large B-cell lymphoma.
  • The most common one accounts for about a third of the estimated 72,000 new cases of non-Hodgkin lymphoma diagnosed each year.


  • Yescarta, also known as axicabtagene ciloleucel, was approved for patients who have already been treated with at least two cancer drugs that either didn’t work for them or eventually stopped working.
  • Yescarta is not a benign treatment, though — three people died after getting the treatment, which can cause serious side effects.
  • The FDA is requiring Kite to do a long-term safety study and train hospitals to quickly spot and handle those reactions.
  • In the key test, Yescarta was given to 101 patients. About 72% saw their cancer shrink and about half showed no sign of disease eight months later.


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