Gene therapy - 'a true living drug' - gets FDA endorsement

This advisory committee hearing was the last major regulatory milestone before the agency decides in September whether or not to approve the treatment, which would make Novartis' CAR-T therapy the first-ever gene therapy treatment approved by the FDA in USA markets.

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).

Novartis has lagged some other big drugmakers in the first wave of immunotherapy drugs that are revolutionizing cancer treatment. Called CAR-T, this treatment procedure involves taking out immune cells from a cancer patient's blood then "reprogramming" them to produce cells that can hone in and destroy cancer cells. Cripe was a member of the FDA advisory panel that voted Wednesday to support the drug's approval.

Cancer experts who advise government regulators are reviewing what could be the first gene therapy approved in the U.S.

This particular treatment, which has the tongue-twisting name tisagenlecleucel, is made by Novartis and it's one of several being developed by drug companies that want to try to standardize the approach as much as possible so it can be patented and licensed. The drugmaker is seeking approval to use the one-time treatment for children and young adults with advanced leukemia. The treatment requires the extraction of a patient's own infection-fighting T-cells, which are then genetically engineered to recognize and fight the cancer cells affecting the patient, and infused back into the bloodstream.

But the FDA advisers did not seem very concerned about the roadblocks and the meeting finished with a unanimous yes vote more than an hour ahead of schedule.

Twelve-year-old Emily Whitehead is the success story.

"When other organizations, including the (National Institutes of Health), considered gene therapy too risky, ACGT believed in the science and funded us when no one else would".

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While many patients can be cured with existing treatments, around 15%-20% see their disease return, and most of these relapsed patients do not survive.

Assuming this treatment is approved, it will be a first for the FDA.

Variations in quality attributes, potency, and transduction efficiency of Novartis' biological cell therapy CTL019, however, did not translate into changed safety or efficacy, according to statistical analyses. In a news release, the drugmaker said that it "continues to invest in the necessary infrastructure for the potential commercialization of CTL019, including manufacturing and the establishment of a network of certified treatment centers".

Millions T-cells (a type of immune cell) are from removed the patient's blood.

Side effects, however, can be severe, and include spiking fever and crashing blood pressure.

"Now that people see there is a path to approval at the FDA, that usually gets people off the sidelines", explained Brad Loncar, founder of a cancer immunotherapy exchange-traded fund.

Other companies are also pursuing CAR-T cell strategies but have struggled with major complications. Success would also help advance a cancer-fighting technique that scientists have been trying to ideal for decades and lift the broader field of cell therapy.

CAR-Ts can also pose serious risks, including a potentially life-threatening inflammatory condition.

(Copyright © 2015. All Rights Reserved.)

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