Published: Fri, July 14, 2017
Medical | By Dorothy Lyons

FDA panel recommends approval for experimental leukemia treatment

FDA panel recommends approval for experimental leukemia treatment

Patient advocacy groups praised the FDA's findings and the impact the new treatment could have on patients with ALL.

The vote came after lengthy discussion and impassioned pleas from the fathers of two young patients whose lives were saved by the therapy.

The treatment, called CTL019, genetically tweaks a patient's own immune system cells into what scientists call "a living drug" to battle a form of acute lymphoblastic leukemia (ALL), The New York Times reported. "We believe when this treatment is approved, it will save thousands of children's lives around the world". Children with cancer are closely monitored for years in clinical practice, said Timothy Cripe, chief of the hematology/oncology division at the Nationwide Children's Hospital.

Gene therapy CTL019 teaches the immune system to recognize cells that fail and destroy them.

Kite Pharmaceuticals has another CAR-T drug up for FDA priority review for the treatment of lymphoma.

More than 600 patients in the USA each year reach that critical stage.

Studies have shown that CAR-T therapies can produce lasting remissions in such cases.

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In a test conducted, gene therapy results exceeded chemotherapy, and even newer types of cancer medicines. In one key trial of tisagenlecleucel, which started in 2015, 82.5% of 63 patients experienced overall remissions.

The main side effect of CTL-019 is cytokine release syndrome, a blistering immune reaction to the drug and the detritus of dying cancer cells. For example, the engineered T cells could be used as an "adjuvant" or additional therapy after a previous treatment has wiped out all but the last traces of cancer, said Richard Maziarz, a blood cancer specialist at Oregon Health and Science University in Portland. "Our hope is that these results will lead to widely available treatments for high-risk B-cell leukemia and lymphoma, and perhaps other cancers in the future". The cells are then sent back to the patient's treatment centre. The whole testing process took 16 weeks on average, which FDA advisers noted could be too long for severely ill patients.

Patients who receive the treatment will be entered in a registry and tracked for 15 years.

In May, Novartis signaled its interest in also developing allogeneic auto T-cell therapies, when it entered a nonexclusive license agreement with Celyad to use its USA patents to produce allogeneic vehicle T cells, in a deal that could generate up to $96 million in up-front and milestone payments for the Belgian biotech.

CAR-T stands for chimeric antigen receptor T-cell therapy. Its treatment was for a type of leukemia, and it was working on similar types of treatments in hundreds of patients for another form of the disease, as also multiple myeloma and an aggressive brain tumour. Questions also remain about the drug's possible long-term side effects. About 85% are cured by chemotherapy, but those whose cancer returns have few options.

First developed by the University of Pennsylvania, CTL019 uses the 4-1BB co-stimulatory domain in its auto to enhance cellular responses as well as persistence of CTL019 after it is infused into the patient, which may be associated with long-lasting remissions in patients. At the University of Texas M.D. Anderson Cancer Center, one team of researchers is turning to umbilical cord blood for inspiration. That was approved past year for a deadly condition called severe combined immunodeficiency and launched with a $670,000 price tag.

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