New gene therapy worth the price of a house

KITE PHARMA — Cell therapy specialists treat blood cells from a patient, to be processed for axicabtagene ciloleucel, otherwise known as Yescarta. (NBC News)

The Food and Drug Administration recently approved a new personalized treatment for cancer on Oct. 18. Yescarta, a chimeric antigen receptor (CAR) T-cell therapy, is both a risk and a lifesaver since it holds dramatic side-effects in some patients and is costly. Doctors proclaim that Yescarta will be used as a last-resort treatment for specific cancer types.

Yescarta, which is the generic name for axicabtagene ciloleucel, has been the second gene therapy approved by the FDA for U.S. cancer patients. This is a form of gene therapy because doctors would take certain cells from a patient’s immune system, and genetically modify them to attack and kill cancer cells. These cells are then placed back inside the body to fight the disease. This is an effective treatment for malignancies since it uses the patient’s immune system to attack cancer cells and to keep cancer cells in remission.

The FDA claimed that “Yescarta is approved for use in adult patients with large B-cell lymphoma after at least two other kinds of treatment failed.” Dr. David Maloney, medical director of cellular immunotherapy at Fred Hutchinson Cancer Re- search Center, said, “There will likely be thousands of lives saved in the next few years because of it.” Diffuse large B-cell lymphoma is ranked as the most common type of non-Hodgkin lymphoma. About 72,000 people are diagnosed with this disease each year, but the new therapy can only be limited toward a fraction of these patients.

Dr. Steven Rosenburg developed this treatment at the National Center Institute. At the NCI, the therapy was licensed to Kite Pharma, and once the treatment produced successful results, Gilead Science had bought this treatment from Kite.

More specifically, Gilead Sciences, which will market Yescarta, had priced the treatment at $373,000. However, one cancer patient and founder of Patients for Affordable Drugs, David Mitchell, said, “As a cancer patient, I look forward to the potential of CAR-T drugs. But drugs don’t work if people can’t afford them, and no American should pay $373,000 for a drug that taxpayers helped invent.”

Problems arose with this treatment because half the patients who were treated suffered from deadly flu-like symptoms. Patients also suffered from nerve damage since their immune system was disturbed during the treatment. Other side-effects involved serious infections, low blood cell counts and a weakened immune system.

Only hospitals and clinics that dispense Yescarta need to be specially certified. The staff who prescribe, dispense or ad- minister Yescarta are required to know when to recognize and manage cytokine release syndrome and nervous system toxicities.