Stem cell treatments surging into clinic
More than ever before, stem cell therapies appear poised to transform medicine — potentially curing heart disease, diabetes and paralyzing injuries, among other ailments.
But it’s also clear that such innovations will be very expensive.
How the government, insurers and patients will pay for what could be a flood of these new treatments drew the attention of more than 700 biomedical and health-care executives Tuesday at the 2014 Stem Cell Meeting on the Mesa.
The annual conference, held on La Jolla’s Torrey Pines Mesa, will run through Thursday. It brings together the business and academic worlds of cell therapy, including but not limited to stem cell treatments.
In California alone, 131 clinical trials are taking place with stem cells, according to Clinicaltrials.gov, a government website that tracks clinical trials. Patients are being treated for conditions such asblindness from retinal diseases, HIV, leukemia, sickle cell disease,stroke and aging of skin.
The recent proliferation of clinical trials marks great progress toward the ultimate goal of getting new treatments to patients, said stem cell researcher Jeanne Loring, who directs the Center for Regenerative Medicine at The Scripps Research Institute in La Jolla.
“It’s been a sea change from last year,” said Loring, who is working with some colleagues in planning their own stem cell trial to treat Parkinson’s disease.
They’re developing replacement neurons grown from artificial embryonic stem cells called induced pluripotent stem cells. The process begins with cells derived from the skin of patients to be treated.
Stem cell therapy reached a milestone this summer when San Diego’s ViaCyte gained federal approval to test its treatment for Type 1 diabetes. The company is scheduled to treat the first patient at UC San Diego in a few weeks.
Participants in the clinical trial will be implanted with devices containing pancreatic “islet” progenitor cells that mature to make insulin and other hormones to regulate blood sugar. The progenitor cells are cultivated from human embryonic stem cells, then encased in a semipermeable layer to keep them in place and protect them from destruction by the immune system.
This trial is mainly meant to assess the device’s safety, said ViaCyte chief executive Paul Laikind, who discussed the company’s efforts at the conference Tuesday.
ViaCyte is especially important in another sense: It represents much-needed evidence of advancement for the taxpayer-funded California Institute for Regenerative Medicine, which has contributed hundreds of millions of dollars to stem cell projects — including $55 million to ViaCyte.
The treatment could provide a “functional cure” for Type 1 diabetes, Laikind said. That cure would last only as long as the insulin-producing cells function; those cells are expected to live for a few years. At that point, the old device could be taken out and another implanted, Laikind said.
To get reimbursement for such breakthroughs, companies would need to show strong scientific evidence, said a panel of health insurers and payment specialists.
If the product is demonstrably superior to what’s currently available, cost won’t be an obstacle to reimbursement, said Nicholas Anderson, a medical technology analyst with Health Economics and Outcomes Research. But please make your data understandable, Anderson told companies.
“Some of the worst data we get is when scientists publish the papers,” Anderson said. “Because it’s 2 percent and 90 percent, cap the value of P squared R squared — all this jargon that doesn’t answer the question (of effectiveness). … If you can’t demonstrate that, we can’t write a check.”
Across the Pacific, Japan is surging as a major power in stem cell research, said Scripps Research’s Loring and Paul Knoepfler, a stem cell scientist at UC Davis.
On Sept. 12, a Japanese woman with macular degeneration received an implant of new retinal tissue that was grown from induced pluripotent, or IPS, cells. It was the first time that IPS cells had been tested in a person.
The cells were first developed by Japanese researcher Shinya Yamanaka, who shared a Nobel Prize in 2012 for his work.
“I’m in touch with the people doing the work in Japan, and it’s just great to see people doing this,” Loring said.
Japan has streamlined its clinical trial system, said Gil Van Bokkelen, chairman and CEO of the Cleveland-based biopharmaceutical company Athersys.
Just one well-designed clinical trial that shows safety and “a meaningful basis for therapeutic effectiveness” can be enough to get marketing approval, Van Bokkelen said at the conference.
The rapidly aging population of Japan gives that country a strong incentive to develop regenerative medicine, Van Bokkelen said.
Knoepfler said the U.S. Food and Drug Administration, which has its own accelerated procedures for pioneering therapies, could apply them to stem cell treatments.
“One really interesting question is to what extent are stem cell therapies going to be allowed to go into those fast-track categories,” Knoepfler said. “In Japan, things are moving very fast.”http://www.utsandiego.com/news/2014/oct/07/stem-cell-meeting-mesa-2014/2/?#article-copy