FDA approves ACGT-funded cancer treatment

US clears first 'living drug' for childhood leukaemia

Tisagenlecleucel, a cell-based gene therapy, is approved in the United States for the treatment of patients up to 25 years of age with B-cell precursor ALL that is refractory or in second or later relapse.

On Wednesday, Swiss pharmaceutical giant Novartis made history as the first company to win Food and Drug Administration (FDA) approval for a groundbreaking new type of cancer treatment known as CAR-T.

On Twitter, FDA commissioner Scott Gottlieb called the approval historic. Patients are often subjected to multiple treatments, including chemotherapy, radiation therapy, targeted therapy or stem cells, but only 10 % are still alive 5 years later.

"While both external and Novartis' quantitative assessments of these values indicate that a cost-effective price could be $600,000 to $750,000, we recognize the importance of this paradigm-shifting therapy and are setting the price at $475,000 for this one-time treatment", Dana Cooper, a spokesman for Novartis, said in an interview with OncLive.

David Mitchell, president of Patients For Affordable Drugs, said: "While Novartis's decision to set a price at $475,000 per treatment may be seen by some as restraint, we believe it is excessive".

"Not only does Kymriah provide these patients with a new treatment option were very limited options exist, but a treatment option that has shown promising remission and survival rates in clinical trials", he added.

Other severe side effects can pop up and require hospitalization.

Of more than 60 ALL-suffering subjects in a clinical trial for Kymriah, 83 percent were cancer-free within three months.

The FDA granted Kymriah Priority Review and Breakthrough Therapy designations. The therapy involves harvesting a patient's T-cells and then genetically modifying them to hold potent molecules called chimeric antigen receptors (CARs). Kite, which was recently acquired by Gilead Sciences, is expected to hear back from the FDA about its treatment for aggressive B-cell non-Hodgkin lymphoma by November.

"This transformational therapy for patients is the result of true collaboration between industry, academia, healthcare professionals, patients and caregivers", said Bruno Strigini, CEO, Novartis Oncology.

Kymriah will carry a boxed warning because of the treatment's potential to cause deadly side effects, including neurological complications and what's known as cytokine release syndrome, a systemic reaction triggered by the destruction of the cancer cells.

"The only potentially curative option for these pediatric and young adult patients is allogeneic stem cell transplant, which costs $500,000 to $800,000 for the first year", Cooper said. Satwani - who hasn't delivered the CAR-T therapy but is heading up the program at Columbia, one of the 32 sites Novartis will use - said patients would need to be monitored closely for about a month, meaning they'd need to be less than a two-hour drive from their hospital.

Since its founding in 2001, ACGT has allocated approximately $29 million in grants to USA and Canadian cancer gene therapy research projects. Penn and Novartis are also investigating the next generation of vehicle therapies for multiple myeloma, and for solid tumors, through trials in glioblastoma, mesothelioma, and ovarian and pancreatic cancer. "The lessons learned from the clinical trials of auto T-cell operations will assist us in being able to provide to the larger population". And a year ago, another company, Juno Therapeutics, said five people in its clinical trials had died, all from cerebral edema.

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