United States approves gene therapy for cancer from Israeli-founded company

Glen Mclaughlin
October 20, 2017

19, 2017 The U.S. Food and Drug Administration has approved the second gene therapy for use in the United States.

Yescarta is the second CAR-T treatment approved by the FDA.

Gilead shares stand at US$80.01 in NY.

Gilead/Kite's drug is approved to treat adult patients with what's known as large B-cell lymphoma after two or more previous treatments.

Yescarta, formerly KTE-C19, is a CD19-directed genetically modified autologous T cell immunotherapy that will be manufactured specifically for each individual patient.

"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", FDA Commissioner Dr. Scott Gottlieb said. He called the approval a "milestone". Patients must be informed of these risks before receiving the therapy. Each treatment of the T-cell therapy is customized using a patient's own immune cells, starting with T cells collection via apheresis, shipment to a manufacturing facility, and genetic modification of the cells to express a vehicle specific to CD19, which is commonly over-expressed in B cell malignancies allowing the modified immune cell to target and kill the lymphoma cell. The gene therapy, known as CAR-T, involves removing a person's T cells, tweaking them in a lab to express certain auto proteins, then injecting the stronger cells back into the patient. The cells are then placed back into the body where they zero in on and destroy cancerous cells.

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In a key trial of 100 patients cited by the FDA, Yescarta resulted in complete remission for 51 percent of patients. Yescarta is not a benign treatment, though: Three people died after getting the treatment, which can cause serious side effects. This syndrome causes symptoms such as fever, nausea, chills, tachycardia, and headache.

Other potential side effects include serious infections, low blood cell counts and a weakened immune system.

The drug was approved along with a risk evaluation strategy that requires hospitals and clinics that prescribe the drug to be specially certified and trained.

Noting the FDA's commitment to "supporting and helping expedite" the development of gene therapies, Gottlieb said, "we will soon release a comprehensive policy to address how we plan to support the development of cell-based regenerative medicine". "That policy will also clarify how we will apply our expedited programs to breakthrough products that use CAR-T cells and other gene therapies". Currently, patients with large B-cell lymphoma in second or later lines of therapy have poor outcomes and greater unmet need, since almost half of them either do not respond or relapse shortly after transplant.

Arie Belldegrun, the Israeli-American founder of Kite Pharma, referred to the promising research when commenting on Gilead's purchase of Kite in August. "We believe this is only the beginning for vehicle T therapies".

Dr. Fred Locke, an oncologist and researcher at Moffitt Cancer Institute in Tampa, Florida, estimates that there are more than 10,000 adults who might benefit from these treatments, whereas the pediatric approval was initial geared for only about 600 patients a year.

Other reports by MaliBehiribAe

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