FDA approves new cancer therapy

Most patients suffered serious side effects.

Yet it also causes collateral damage.

Novartis is also testing the drug in diffuse large B-cell Lymphoma, the most common form of non-Hodgkin lymphoma, as is Kite.

"The therapy is being given to people who would be dead if they didn't receive it, and they would be dead within a pretty short period of time", said Edmund Waller, director of Emory University's stem cell and immune therapy division.

In a clinical trial, 79 percent of patients given the Novartis therapy were alive a year later.

Neither the cancer therapy nor the follow-up is cheap.

The Switzerland-headquartered company wants the chimeric antigen receptor T-cell (CAR-T) therapy approved in relapsed or refractory B-cell acute lymphoblastic lymphoma (ALL) in children and young adults. To use the technique, the removed cells must be frozen and shipped to a Novartis plant for thawing and processing.

Many studies and trials have been conducted on the living drug, most of which have yielded positive results. The antibodies are spun out of donated blood, and infused into patients, whose risk of infection quickly returns to normal levels. Novartis' therapy is not identical to the CAR-T cells used in those trials, which were administered in adults, but the deaths cast a pall over the entire field. In light of the drug's side effects, the FDA advisory panel viewed this as a favorable risk-benefit balance. "Coverage determination will be made on clinical grounds". Cripe was a member of the FDA advisory panel that voted Wednesday to support the drug's approval. CAR-T cells are built with proteins where they seek out cancer protein cells in the body that they destroy.

The committee has asked Novartis to commit to 15 years of follow-up, given the therapy is following a fast-track approval and no long-term data is available yet. The drugmaker is seeking approval to use the treatment for patients aged 3 to 25 with a blood cancer called acute lymphoblastic leukemia whose disease has spread or failed to respond to standard treatment.

This super charges the T-cells to attack B-cells, which are immune cells that turn malignant in leukemia. However, the drug company said they can now produce the treatment and get it to a patient in about three weeks. "This is a complex product", Bryan said in opening the meeting. That could create problems if demand surges because of Novartis's cancer treatment.

The approval of the new treatment has potential consequences that extend well beyond treatment of leukemia. "Shire continues to explore ways to address patient demand today and as it looks toward the future".

While the permanence of the manipulated cancer killers is good for eliminating the blood malignancy, it's bad for the immune system.

The global CAR-T market is estimated to hit $8.5 billion by 2028, according to a report released in February by Coherent Market Insights. "It's bad that it might be permanent". In essence, the patients T-cells are taken out of the body and re-engineered to target and kill only cancerous cells in the body.

Data from the USA multicenter trial and single site trial assessing the safety and efficacy of CTL019 among pediatric and young adult patients with r/r B-cell ALL also backed the recommendation and the biologics license application (BLA).

After decades of setbacks and disappointments in efforts to fix, replace, or change genes to cure diseases, several companies are near the finish line in a race to bring CAR-T and other types of gene therapy to patients.