By: CBC News
U.S. approves genetically engineering blood cells as childhood leukemia treatment
In Canada, scientists working on protocol that is affordable for the public health-care system
Opening a new era in cancer care, the U.S. Food and Drug Administration on Wednesday approved the first treatment that genetically engineers patients’ own blood cells into an army of assassins to seek and destroy childhood leukemia.
The CAR-T cell treatment developed by Novartis and the University of Pennsylvania is the first type of gene therapy to hit the U.S. market — and one in a powerful but expensive wave of custom-made “living drugs” being tested against blood cancers and some other tumours, too.
FDA called the approval historic.
“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 harbour a “chimeric antigen receptor” 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.
‘We are exploring a more Canadian solution’
Today’s FDA approval is important because it means this immunotherapy approach to cancer is finally ready for the clinic — to join surgery, radiation, and chemotherapy in the cancer fighting arsenal. But in practice, it won’t change cancer treatment in the short term, especially not for Canadian patients.
Right now, the CAR-T therapy will only be available in specially selected centres in the U.S. which have been equipped and trained to do the complicated procedure of extracting a patient’s cells, and genetically modifying them, reprogramming them to attack cancer, and then infusing them back into the patient’s body. And the price tag of half a million dollars US, not including supportive care, will put the treatment out of reach for many patients and their families.
But a network of Canadian scientists are busy working out a made-in-Canada protocol that would be affordable for the public-health system. John Bell, scientific director of BioCanRX and a senior scientist at the Ottawa Hospital, said his group is working to get the new CAR-T therapy infrastructure up and running in Canada.
“We hope to get a human trial going next year or early the following year using this exact strategy. There are two steps. First you have to manufacture the virus product which is used to reprogram the immune cells which are used in the therapy. We’re in the throes of doing that now. The second step is to harvest cells from [the] patient, infect them with the virus and put them back in the patient. We hope to have that optimized and ready to go next year.” – Dr. John Bell, Scientific Director, BioCanRx