Stem cell and gene therapies for cancer and other diseases used to be considered exotic. But stunning successes are fast moving them into the medical mainstream.
While only a few of these therapies have yet been approved, many more are being tested experimentally. In addition to treating otherwise fatal cancers, they may relieve sickle cell disease, restore failing hearts and even cure HIV infection.
And with mainstream success comes a mainstream worry: How will patients pay for these expensive treatments? Or to look at it another way, how much is it worth to save a life?
Drug company representatives discussed these issues Wednesday at Cell & Gene Meeting on the Mesa, an annual event in La Jolla devoted to stem cell and gene therapy.
At the end of August, drug giant Novartis marked a milestone by receiving U.S. approval for a blood cancer treatment made from the patient’s own genetically modified immune cells. The treatment, Kymriah, has rescued children and young adults who were gravely ill with acute lymphoblastic leukemia and placed them into remission.
Kymriah costs $475,000. But Novartis made an unusual guarantee: If patients don’t respond in a month, the company won’t charge for it.
However, these arrangements, like the therapies themselves, are so new that federal regulators are hesitant, said Pascal Touchon, a senior vice president with Novartis Oncology.
“The system is not organized for that,” Touchon said at a morning panel. “So when you ask for the first time whether we can do that, the answer is no. That’s the starting point.”
Novartis eventually reached agreement with the Centers for Medicare and Medicaid Services, or CMS.
The challenge now is to make general rules for such therapies, instead of making rules case-by-case, said Bob Azelby, chief commercial officer of Juno Therapeutics. Juno is developing a cancer immune therapy similar to Novartis’.
“They’re getting value for the dollars they’re spending,” Azelby said of CMS.
Cell & Gene Meeting in La Jolla began more than a decade ago as a purely scientific conference on stem cells. But it has grown as stem cell technology has been augmented with gene therapy, the delivery of disease-fighting genes.
Genetically modifying stem cells provides a virtually unlimited source of cells with useful properties. These could fight cancer, or perhaps correct a disease caused by a faulty gene.
Many of these therapies, such as Kymriah, are made from a patient’s own cells, collected, modified, grown and re-infused into the body. Such custom-made treatments are extremely expensive. They belong to a class of treatments called CAR T cell therapy.
Bluebird Bio, also represented on the panel, is developing its own version of CAR T cell therapy, in its case for another blood cancer called multiple myeloma.
In addition, Bluebird is developing gene therapies for a rare disease called cerebral adrenoleukodystrophy, and the blood disorders sickle cell disease and beta-thalassemia.
“For Bluebird … it’s ultimately putting together a value story, that allows for a dialogue” about pay-for-performance, said Jeffrey Walsh, chief financial and strategy officer.
Vericel, which grows replacement skin from a burn patient’s own skin cells, also makes such a value pitch, said Nick Colangelo, president and CEO.
“When we treat a catastrophic burn patient, an order of our skin grafts can cost a couple hundred thousand dollars,” Colangelo said. “But it’s a one-time treatment.”
Moreover, treated patients have nearly a 90 percent survival rate, he said.
“That clearly is a product that has a lot of value,” Colangelo said.
Besides helping patients and the companies that make successful therapies, treatments like Kymriah also benefit companies that supply their research tools. One of them is Thermo Fisher Scientific, which had an early collaboration with Carl June, the physician who pioneered the therapy at the University of Pennsylvania.
Thermo Fisher supplied its Dynabeads, microscopic magnetic beads that attach to specified cells using an antibody linker, said Mark Stevenson, the company’s chief operating officer.
“They help extract and amplify the cells prior to the therapy … to actually enrich the cells that you want to pull out, also that you’re able to develop and expand the correct CAR T cells,” Stevenson said. “And for Novartis we scaled up that therapy to make a successful launch.”
“It’s a very exciting time for cell therapy,” Stevenson said. “We’ve been involved in it for 10 years and we’re finally seeing the benefits coming to patients.”