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FDA panel endorses potential very first US gene therapy to treat leukemia
In this July 9, 2015, photo, provided by Novartis Pharmaceuticals Corp., human T cells belonging to cancer patients arrive at Novartis Pharmaceuticals Corp.’s Morris Plains, N.J., facility. This laboratory is where the T cells of cancer patients are processed and turned into super cells as part of a fresh gene therapy-based cancer treatment Novartis is a part of. (Brent Stirton/Courtesy of Novartis Pharmaceuticals Corp. via AP) (Novartis Pharmaceuticals Corp.)
A panel of cancer experts Wednesday unanimously endorsed a leukemia treatment that could be the very first gene therapy available in the U.S.
The Food and Drug Administration (FDA) advisory panel voted 10-0 to recommend approval of the treatment, which was devised by the University of Pennsylvania and Novartis Corp. The FDA is not required to go after the panel’s recommendation, but often does.
The one-time treatment would be used on children and youthfull adults with advanced leukemia.
The therapy could be the very first of a wave of treatments custom-made to target a patient’s cancer. Called CAR-T, it involves removing immune cells from a patients’ blood, reprogramming them to create an army of cells that can recognize and demolish cancer and injecting them back into the patient.
“This is a major advance,” said panel member Dr. Malcolm A. Smith of the National Cancer Institute. He said the treatment is “ushering in a fresh era.”
The vote came after lengthy discussion and impassioned prayers from the fathers of two youthfull patients whose lives were saved by the therapy. The one-time leukemia treatment would be for children and youthful adults with the most common form of childhood cancer, known as ALL.
“Our daughter was going to die and now she leads a normal life,” said Tom Whitehead, of Philipsburg, Pa. His daughter Emily, now 12, was the very 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.”
Novartis is seeking approval to use the treatment for patients aged three to twenty five with a blood cancer called acute lymphoblastic leukemia whose disease has spread or failed to react to standard treatment. That happens to more than six hundred patients in the U.S. each year. At that point, they have limited options — all more toxic than the CAR-T therapy — and survival chances are slender. ALL accounts for a quarter of all cancers in children under age 15.
In a key test, results were far better than chemotherapy and even newer types of cancer drugs. Of the fifty two patients whose results were analyzed, eighty three percent had accomplish remission, meaning their cancer vanished. Most patients suffered serious side effects but almost all recovered.
CAR-T therapy starts with filtering key immune cells called T cells from a patient’s blood. In a lab, a gene is then inserted into the T cells that prompts them to grow a receptor that targets a special marker found on some blood cancers. Millions of copies of the fresh T cells are grown in the lab and then injected into the patient’s bloodstream where they can seek out and ruin cancer cells. Doctors call it a “living drug” — permanently altered cells that proceed to multiply in the bod into an army to fight the disease.
During the patient testing, the entire process took about sixteen weeks, which can be too long a wait for some despairingly ill patients, the FDA advisers noted during the meeting in Silver Spring, Md. Drug company officials said they can now produce a treatment and get it to a patient in about three weeks.
Novartis said in a statement that it has long believed CAR-T therapy could “switch the cancer treatment paradigm.”
The cost of CAR-T therapy is likely to be hundreds of thousands of dollars, but it’s only given once. 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.
The treatment’s short-term side effects, including fever and hallucinations, are often intense as the bod’s revved up immune system goes on the attack. The long-term side effects of the treatment are unknown. It’s also unclear if patients whose cancer goes into remission will be cured or will have their cancer comeback eventually. The FDA panel recommended that patients who get the treatment be monitored for fifteen years.
Other biotech and pharmaceutical companies are developing types of gene therapy to treat solid cancers and infrequent gene-linked diseases. A few products have been approved elsewhere — one for head and neck cancer in China in two thousand four 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.
UniQure’s Glybera was approved for a uncommon enzyme disorder. It was used only once in five years, likely due to its $1 million-plus price tag, so uniQure is pulling it from the market.
The Associated Press contributed to this report.