FDA Approves First CAR-T Cell Therapy

Doctors

The FDA-approved drug Kymriah has been called a "living drug" because involves using genetically modified immune cells from patients to attack their cancer, according to NPR.

It has already been used to treat children dying from leukemia, who are now cancer free.

Wednesday's announcement will "change the face of modern medicine and drug development", FDA Commissioner Dr. Scott Gottlieb said at a news briefing.

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

ALL is a potentially fatal cancer of the blood and bone marrow. About 20 percent of the 3,500 pediatric and young adult patients diagnosed every year with ALL in the United States relapse or do not respond to conventional treatment. But they have not altered the actual DNA of the patients' cells like Kymriah is touted to do.

CAR-T treatment is different from the popular immunotherapy drugs that treat cancers by helping the body to spot tumors easily. We now have proof that it is possible to eradicate cancer by harnessing the power of a patient's own immune system.

Other trials with CTL019 therapy are also underway in the Abramson Cancer Center for adult ALL patients and those with CLL and non-Hodgkin lymphoma. Even so, the approval has industry experts and cancer doctors excited.

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

However, Anderson stressed that "this approval only pertains to a small population of children [with ALL]". It is estimated that only 600 patients per year would be eligible for the therapy due to how rare the condition is, according to the article.

Amazingly, of the 63 patients who received CAR-T therapy, 83 percent were in complete remission within three months. It can cause high fevers and flu-like symptoms, and can be a life-threatening complication.

So, hospitals and clinics that offer Kymriah will have to have special certifications.

And then there's the issue of cost.

Kymriah allows patients, for whom first line drugs failed, another chance.

Most patients with ALL recover through other treatments such as radiation, chemotherapy and stem cells. They'll also have to be less toxic, so more people can consider it instead of only those who have very few other options. Nevertheless, researchers caution that, in many respects, it's still early days for vehicle T cells and other forms of ACT, including questions about whether they will ever be effective against solid tumors like breast and colorectal cancer.

The FDA-approved CAR-T therapy administers a customized treatment that uses a patient's own T cells, which plays an important role for a person's immunity system.

Breakthrough medicine does not come cheap, with the company Novartis charging $475,000 for the therapy, but the results speak for themselves.

"The science has reached a point of superiority, where enough of the components of these endeavors have worked out", he explained. These challenges include commercialisation, market access, patient and physician adoption, cost/benefit discussions and reimbursement, as well as manufacturing scale up. "We can deliver on the original promise". The treatment is the first of its kind approved in the U.S. "ISCT will continue to drive to support all elements of the sector to make sure this happens".

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