First Ever Cancer Gene Therapy For Leukemia Gets Approved By US Regulators

An innovative cancer treatment for childhood leukaemia that uses patients’ genetically altered immune cells to fight the diseases was approved on Wednesday, 30 July by FDA (Food and Drug Administration) and called it a historic action.

The first gene therapy to hit the U.S market made from scratch for every patient is one of a wave of ‘living drugs’ under development to fight additional blood cancers and other tumours as well.

As per the report by AP, Novartis Pharmaceuticals set the price for its one-time infusion of ‘CAR-T cells’ at $475,000 and said that the patients who did not show a response within a month would not be charged.

Dr Stephan Grupp of Children’s Hospital of Philadelphia, who treated the first child with CAR-T cell therapy said, “This is a brand new way of treating cancer, a girl who had been near death but now is cancer-free for five years and counting, that is extremely 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 then filter those cells from a patient’s blood, reprogram them to harbour a “chimeric antigen receptor” or CAR that zeroes in on cancer, and grow hundreds of millions of copies. These revved-up cells can continue multiplying to fight diseases for months or years.

FDA Commissioner Scott Gottlieb said, “We are entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack deadly cancer,” adding that the new technologies like gene and cell therapies hold out the potential to transform medicine and cure intractable illnesses.

Novartis and the University of Pennsylvania developed the first CAR-T version which has been approved for use by hundreds of patients a year who are desperately ill with acute lymphoblastic leukaemia. Each year in U.S, more than 3,000 children and young adults get struck by it.

The new CAR-T therapy might replace bone marrow transplants that cost more than half a million dollars, for some patients, said Grupp who led the Novartis study. He said, “I do not want to be an apologist for high drug prices in the U.S. But if it is the last treatment they need, that is a really significant one-time investment in their wellness, especially in children who have a whole lifetime ahead of them.”

As reported by The Washington Post, in an important study of more than five dozen patients, researchers reported, more than 80% of those who got the therapy went into remission.

A pediatric oncologist at Memorial Sloan Kettering Cancer Centre in New York, Kevin Curran has called the new treatment ‘exciting for pediatric oncology’ and said he expected it would make bone marrow transplants obsolete.

Novartis executives have said that by the end of the year they hope to have 20 certified medical centres ready within a month. They further said that the company is collaborating with the federal Centres for Medicare and Medical Services to develop a pricing model based on how well the drug works.

A patient-assistance and a travel expenses program have been set up by Novartis which can be used to help families who lack insurance or have coverage that falls short of this treatment.