FDA panel recommends approving 1st gene-altering leukemia treatment

Lydia Fleming
July 14, 2017

Experts say the approval will pave the way for a new era of gene-based medicines, which has shown promise in treating a wide range of diseases. Novartis plans further filings for CTL019 in the United States and EU later this year, including applications with the FDA and European Medicines Agency (EMA) for the treatment of adults with r/r diffuse large B-cell lymphoma (DLBCL). "We believe when this treatment is approved, it will save thousands of children's lives around the world".

Acute lymphoblastic leukaemia comprises approximately 25% of cancer diagnoses among children under 15 years old and is the most common childhood cancer in the US.

Fatalities due to cerebral oedema have occurred with other CAR-T therapies however - notably Juno Therapeutics' JCAR015 which was discontinued in March and another candidate from Kite Pharma - and as a result the safety of CTL019 had been expected to come under close scrutiny. Ultimately, it is up to the FDA to decide if it should approve the treatment or not.

"When fully commercialized, this [CTL019] therapy will no doubt save the lives of many children and young adults who have had no other effective therapy for relapsed and refractory B-cell acute lymphoblastic leukemia [ALL]", said Dr. John Maris, a pediatric oncologist at The Children's Hospital of Philadelphia. That happens to more than 600 patients in the US each year. The CAR-T space should start to heat up once other companies start to win FDA approval for their CAR-T therapies. This is the most common type of cancer among children, according to the National Cancer Institute. Most patients suffered serious side effects but almost all recovered. Clinical trials of the medically advanced treatment were held in February 2016 by a group of scientists, when several dozen blood cancer patients, who barely had months to live, were administered the drug.

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Described in the meeting as a "living drug", CTL019, also known as tisagenlecleucel, involves a patient's own T-cells being harvested and genetically altered so they attack cancer.

The approval of the drug is likely, especially in light of the positive results of experiments in the field of cell modification to fight diseases.

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 during the meeting in Silver Spring, Maryland.

In this pivotal study informing the committee's decision, roughly half of 68 patients receiving the drug experienced this complication, though none died from it.

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"Because CTL019 is an investigational therapy, the safety and efficacy profile has not yet been established", the Novartis statement said. There might be a risk that it could cause the growth of new malignancies over time, but the advisory panel said the risk was low and that the mitigation proposal presented by Novartis were adequate. Novartis is now poised to be the first, and it is working on similar types treatments for another type of leukemia, as well as multiple myeloma and an aggressive brain tumor.

CAR-T therapy is a high risk approach, and little is known about any long-term toxicity effects of treatments.

First developed by the University of Pennsylvania, CTL019 uses the 4-1BB co-stimulatory domain in its vehicle 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.

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