We don’t do a lot of reporting on medical breakthroughs here but this seems pretty significant. The FDA has approved the first “living drug” that uses genetically modified immune cells to target cancer. From NPR:
The FDA approved Kymriah, which scientists refer to as a “living drug” because it involves using genetically modified immune cells from patients to attack their cancer.
The drug was approved to treat children and young adults up to age 25 suffering from a form of acute lymphoblastic leukemia who do not respond to standard treatment or have suffered relapses.
The disease is a cancer of blood and bone marrow that is the most common childhood cancer in the United States. About 3,100 patients who are 20 and younger are diagnosed with ALL each year.
The NY Times notes the approval came after an impressive trial of the drug:
The approval was based largely on a trial in 63 severely ill children and young adults who had a remission rate of 83 percent within three months — a high rate, given that relapsed or treatment-resistant disease is often quickly fatal…
The first child to receive the therapy was Emily Whitehead, who was 6 and near death from leukemia in 2012 when she was treated, at the Children’s Hospital of Philadelphia. Now 12, she has been free of leukemia for more than five years.
To customize Kymriah for individual patients, white blood cells called T cells will be removed from a patient’s bloodstream at an approved medical center, frozen, shipped to Novartis in Morris Plains, N.J., for genetic engineering and multiplying, frozen again and shipped back to the medical center to be dripped into the patient. That processing is expected to take 22 days.
As you would expect, this kind of novel, custom medicine isn’t cheap. The treatment, which only has to be given once costs $475,000. That sounds insane to me but Novartis points out the other option for treatment, a bone-marrow transplant, costs up to $800,000. What’s really exciting about this to a layman like myself is the promise it seems to hold out for the future. The BBC reports:
Dr David Maloney, medical director of cellular immunotherapy at the Fred Hutchinson Cancer Research Center, said the FDA’s decision was a “milestone”.
He added: “We believe this is just the first of what will soon be many new immunotherapy-based treatments for a variety of cancers.”
This particular therapy has worked best against blood cancers and not as well against solid tumors, but we’re still at the very beginning of this. The technology will improve, expand, get cheaper and better as we go. If you know anyone who has cancer, that’s exciting news.
Even as it stands today, it’s hard to imagine how much this means to the 600 or so patients a year, many of them children, who may turn to this new treatment. Below is a 2012 video about Emily Whitehead, the first child to receive this therapy. And here’s Emily’s website showing how she is doing today.