tiptrot.com October 23, 2017

Childhood Leukemia Will Finally Have An FDA Approved Treatment

01 September 2017, 01:34 | Nichole Osborne

First gene therapy coming to US after FDA approval

Novartis' CAR-T gene therapy, the first approved by FDA, to be priced based on cancer patients' outcomes

The FDA's "historic action" targets acute lymphoblastic leukemia (ALL), considered the most common childhood cancer in the U.S.; some 3,100 patients under the age of 20 are diagnosed with the bone marrow and blood disease each year. ACGT provided an additional $800,000 of funding in 2008.

Novartis estimates about 600 patients a year would be eligible for the treatment, which belongs to a class of drugs known as vehicle T-cell therapies.

This represents the first approval of this form of gene transfer therapy and as such has given rise to the International Society for Cellular Therapy (ISCT), the global professional society of clinicians, researchers, regulatory specialists, technologists and industry partners in the cell therapy sector, revising its forecast for the CAR-T and cell therapy industries. "It's a very exciting time".

"We're very comfortable" with the price, said Bill Hinshaw, head of the company's USA cancer business.

In clinical trials, CAR-T therapies have shown remarkable efficacy against blood cancers. Salvage therapies for children with relapsed ALL are highly toxic and only work in about 50 percent of relapsed patients. Satwani - who hasn't delivered the CAR-T therapy himself but is heading up the program at Columbia, one of the 32 sites that Novartis will use - said patients need to be closely monitored for a month or so, meaning they'll need to be less than a two hour drive away from their hospital.

"We're entering a new frontier in medical innovation with the ability to reprogram a patient's own cells to attack a deadly cancer", says FDA Commissioner Scott Gottlieb in a statement. "The therapies that we're using to treat children 20 years from now will emerge from the investment in science that we make today".

Although the price of the therapy is high by any standard, Cooper stated that Novartis is "committed to doing everything we can to ensure that children and young adults who can benefit from [tisagenlecleucel] have access to the therapy and we are working with payers to ensure they fully understand the value of [it] and provide coverage for patients". The treatment is also known as CAR-T. In trials, the therapy achieved remission in 83% of patients.

He added: "We believe this is just the first of what will soon be many new immunotherapy-based treatments for a variety of cancers".

His current research is focused on similar reprogramming of cells but for the detection of lung cancer cells.

The approval of tisagenlecleucel was based on phase II results from the single-arm, global ELIANA trial of 63 patients who received a single dose of tisagenlecleucel.

However, the treatment can have serious side effects like infections, low blood pressure, kidney injury and other effects. The treatment, which only has to be given once costs $475,000.

On Wednesday, the FDA also expanded approval for another drug, tocilizumab, to treat CRS in patients 2 and older.

The Novartis-Penn Center for Advanced Cellular Therapeutics (CACT) opened in 2016 and hosted Vice President Joe Biden at the launch of his Cancer Moonshot initiative, cementing Penn's role as an global innovator in the development and manufacturing of personalized cellular therapies.

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