August 31, 2017

"We've never seen anything like this before" about the "living drug" , tailor-made to each patient

Historic 'living drug' gets go-ahead

The US has approved the first treatment to redesign a patient's own immune system so it attacks cancer.
The regulator - the US Food and Drug Administration - said its decision was a "historic" moment and medicine was now "entering a new frontier".

The "living drug" is tailor-made to each patient, unlike conventional therapies such as surgery or chemotherapy.
It is called CAR-T and is made by extracting white blood cells from the patient's blood. The cells are then genetically reprogrammed to seek out and kill cancer. The cancer-killers are then put back inside the patient and once they find their target they multiply.

Dr Stephan Grupp, who treated the first child with CAR-T at the Children's Hospital of Philadelphia, said the new approach was "enormously exciting" --"We've never seen anything like this before."

That first patient had been near to death, but has now been cancer-free for more than five years. Out of 63 patients treated with CAR-T therapy, 83% were in complete remission within three months and long-term data is still being collected. However, the therapy is not without risks....

US clears breakthrough gene therapy for childhood leukemia

Made from scratch for every patient, it's one of a wave of "living drugs" under development to fight additional blood cancers and other tumors, too. Novartis Pharmaceuticals set the price for its one-time infusion of so-called "CAR-T cells" at $475,000, but said there would be no charge for patients who didn't show a response within a month.

"This is a brand new way of treating cancer," said Dr. Stephan Grupp of Children's Hospital of Philadelphia, who treated the first child with CAR-T cell therapy — a girl who'd been near death but now is cancer-free for five years and counting. "That's enormously exciting."

CAR-T treatment uses gene therapy techniques not to fix disease-causing genes but to turbocharge T cells, immune system soldiers that cancer too often can evade. Researchers filter those cells from a patient's blood, reprogram them to harbor a "chimeric antigen receptor" or CAR that zeroes in on cancer, and grow hundreds of millions of copies. Returned to the patient, the revved-up cells can continue multiplying to fight disease for months or years.

It's a completely different way to harness the immune system than popular immunotherapy drugs called "checkpoint inhibitors" that treat a variety of cancers by helping the body's natural T cells better spot tumors. CAR-T cell therapy gives patients stronger T cells to do that job.
Posted by Jill Fallon at August 31, 2017 1:49 PM | Permalink