- A Food and Drug Administration (FDA) advisory panel voted in favor of a novel treatment for a blood cancer that commonly affects children.
- The one-time treatment involves tweaking a patient’s immune cells to attack cancer. It put 83 percent of patients into remission in initial testing.
- Side effects can be serious, treatment is expensive, and the FDA still needs to formally approve specific drugmakers’ treatments.
A treatment for leukemia, a common childhood blood cancer, could become the first gene therapy available in the U.S. now that a Food and Drug Administration (FDA) advisory panel voted 10-0 on Wednesday in favor of it. Although the FDA still has to decide whether to approve specific drugmakers’ treatments, this initial approval is being heralded as “a major advance,” said panel member Dr. Malcolm A. Smith of the National Cancer Institute. He said the treatment is “ushering in a new era.”
Developed by the University of Pennsylvania and Novartis Corp, the therapy known as CAR-T could be the first of a wave of treatments custom-made to target a patient’s cancer. The approach involves removing immune cells from a patients’ blood, reprogramming them to create an army of cells that can zero in on and destroy cancer cells and injecting them back into the patient’s bloodstream. Doctors call it a “living drug” since it permanently alters cells that continue to multiply in the body to fight the disease.
The FDA’s vote came after lengthy discussion and impassioned pleas from the fathers of two young patients whose lives were saved by the therapy. “Our daughter was going to die and now she leads a normal life,” said Tom Whitehead, of Philipsburg, Pennsylvania. His daughter Emily, now 12, was the first child to receive the experimental therapy, five years ago. “We believe when this treatment is approved, it will save thousands of children’s lives around the world.”
The FDA is expected to approve at least one CAR-T therapy from the drugmaker Novartis in the next few months. This particular treatment would be for patients aged 3 to 25 with a blood cancer that accounts for a quarter of all cancers in children under age 15: Acute lymphoblastic leukemia (ALL). Novartis is seeking the FDA’s approval specifically for patients would whose disease has spread or failed to respond to standard treatment. This happens to more than 600 patients in the U.S. each year, leaving them with limited options that are more toxic than CAR-T therapy, and slim survival rates.
In a key test on CAR-T, results were far better than chemotherapy and even newer types of cancer drugs. Of the 52 patients whose results were analyzed, 83 percent had complete remission, meaning their cancer vanished. Most patients suffered serious side effects, including fever and hallucinations, which are often intense as the body’s revved-up immune system goes on the attack. Eleven patients died, four from side effects and seven from their leukemia. (During the patient testing, the whole process took about 16 weeks on average, which can be too long a wait for some desperately ill patients, the FDA advisers noted. Drug company officials said they can now produce a treatment and get it to a patient in about three weeks.)
The long-term side effects of CAR-T treatment remain unknown. It’s also unclear if patients whose cancer goes into remission will be cured or will have their cancer return eventually. The FDA panel recommended that patients who get the treatment be monitored for 15 years.
Although CAR-T therapy is only given once, it’s likely to cost hundreds of thousands of dollars. Typically, cancer patients take one or more drugs until they stop working, then switch to other drugs, so treatment — and side effects — can go on for years.
Other biotech and pharmaceutical companies are developing types of gene therapy to treat solid cancers and rare gene-linked diseases, and a few products have been approved elsewhere — one for head and neck cancer in China in 2004 and two in Europe, most recently GlaxoSmithKline’s Strimvelis. That was approved last year for a deadly condition called severe combined immunodeficiency and launched with a $670,000 price tag.
While these numbers are staggering, it’s a small price to pay for technology that literally saves lives.