1st gene therapy OKd for cancer
FDA approves treatment for childhood leukemia
“This affords us the opportunity to potentially cure kids that really had no hope in the past.” Dr. Michelle Hermiston, UCSF physician
The Food and Drug Administration on Wednesday approved the first gene therapy treatment for cancer, green-lighting Novartis Pharmaceuticals’ cell therapy treatment for childhood leukemia.
The regulatory approval had been widely expected but came earlier than analysts’ predictions. It signals that other drug companies developing cell-therapy treatments may also be able to commercialize what medical experts say is a revolutionary new form of medicine.
The therapy extracts a patient’s cells from his or her blood, sends them to a manufacturing site to genetically modify them, and then re-infuses them into the person’s body to attack cancer cells. Cell therapy is the first kind of treatment that uses a patient’s own engineered living cells to kill cancer cells. The idea is to target the disease in more precise and nuanced ways than conventional treatments.
Novartis’ therapy, called Kymriah, was approved for the treatment of childhood B-cell acute lymphoblastic leukemia — a cancer of the blood and bone marrow. This type of leu-
kemia is the most common childhood cancer, affecting about a third of all children with cancer. The National Cancer Institute estimates that 3,100 patients 20 years old or younger are diagnosed with acute lymphoblastic leukemia each year.
Novartis said Kymriah will cost $475,000 for one course of treatment.
The survival rate for children with acute lymphoblastic leukemia is 90 percent, but the rates plummet to 10 to 20 percent among children who relapse after a bone marrow transplant. It is this subset of patients who are eligible for cell therapy, which has been shown to have a response rate of 70 to 80 percent.
“This is really exciting, because this affords us the opportunity to potentially cure kids that really had no hope in the past,” said Dr. Michelle Hermiston, a pediatric hematology oncologist at UCSF who has not used Novartis’ therapy but has worked with a patient on a similar type of treatment in a clinical trial.
Cell therapy for oncology has proved effective in some blood cancers, but has yet to show as much efficacy in solid tumors. The reason the earliest regulatory approvals for the therapy is for leukemia — and, soon, lymphoma — is because these types of cancer affect the body’s B cells, which make antibodies that help the immune system fend off infections. In cell therapy, the genetically modified cells are programmed to attack B cells — both the cancerous B cells and normal B cells — which the body can live without.
Gilead Sciences, the Foster City biopharmaceutical giant, this week announced a $12 billion deal to acquire Kite Pharma, a pioneer in cell therapy to treat a type of non-Hodgkin’s lymphoma called diffuse large B-cell lymphoma. Kite is expected to receive approval for the therapy, KTE-C19, in November.
Several other biopharma firms — including Juno Therapeutics, Fate Therapeutics, Bluebird Bio and Cellectis — are in various stages of development for cell therapies.
“It opens up the field generally to additional strategies and technologies in this space to improve on a couple indications that have been good responses so far,” said analyst David Nierengarten, head of health care equity research at Wedbush Securities.