Wednesday, August 28, 2019

Pressures for Stem Cell Profits and Cures: A Case from Japan with Implications for California

A stem cell treatment in Japan for spinal cord injury is raising a ruckus about ethics, efficacy and billion-dollar searches for cures and profits.

The matter involves a therapy called Stemirac and Sapporo Medical University. The treatment is now available to the public in Japan with most of its $140,000 cost covered by Japan's national health insurance program. 

The most recent overview of Stemirac came yesterday on a site called "Undark" in an article written by Amos Zeeberg. In the piece, Arnold Kriegstein, director of the stem cell program at UC San Francisco, called Stemirac  "essentially an unproven therapy."  Bruce Dobkin, a UCLA neurologist, was reported as saying "the results briefly reported in the media may suggest the treatment doesn’t even work."

(Undark's site says it is a "is a non-profit, editorially independent digital magazine exploring the intersection of science and society."  Its publisher is Deborah Blum, a former colleague of this writer at The Sacramento Bee. Blum is now director of the Knight Science Journalism Program at MIT.) 

In the piece yesterday about Stemirac, Zeeberg wrote, 
"It’s arguably the world’s most ambitious approved stem cell treatment and should have been a cause for celebration: a long-awaited breakthrough for the field of regenerative medicine — using modern biological tools to repair the body — and a harbinger of more impressive medicines."
He continued, 
Arnold Kriegstein
UCSF photo
"Instead, the therapy has been met with a heated debate. On one side, many experts have slammed Stemirac’s approval in uncommonly direct terms, saying there isn’t enough evidence to show it is effective or even safe. The treatment went through an expedited approval unique to Japan: After short, small clinical trials that suggest safety and efficacy, regulators can approve stem cell treatments on a conditional basis — allowing use of the treatments for seven years, while sponsors gather additional evidence to support a full approval.
"Critics also say Japan’s approach is far too soft — that early approvals allow patients to take experimental therapies that could be ineffective or dangerous, at a high cost to both patients and insurance providers. 'This is essentially an unproven therapy,' said Arnold Kriegstein, a stem cell researcher at the University of California, San Francisco. 'I’m very surprised this is happening in a country like Japan.'..."
The Undark article continued, 
Bruce Dobkin
UCLA photo
"What’s more, UCLA neurologist Bruce Dobkin told Undark, the results briefly reported in the media may suggest the treatment doesn’t even work. Dobkin pointed to previous trials testing other potential treatments for spinal cord injuries, and the Stemirac findings 'are exactly the results we found in patients in randomized controlled trials — in the control groups,' he said. That is, patients injected with Stemirac seemed to do as well as patients who got placebos in these earlier trials. He says people who have recently suffered spinal cord injuries, like Kusachi, the injured high diver (mentioned in Zeeberg's piece), and the others in the Stemirac trial, often have significant natural improvement over the next several months — exactly the period covered by the trial. It’s possible the patients were simply healing naturally, he says, but without a control group and double-blinding, it is hard to tell."
Undark's article captures many of the issues surrounding the development of stem cell therapies including the pressure to generate results for patients and profits for companies -- not to mention prestige and praise for researchers.

California's $3 billion stem cell program feels that pressure as well. It is running out of cash and would more than welcome a breakthrough in one of its 56 clinical trials -- one that would stimulate California voters to provide more billions for the state's nearly 15-year-old stem cell research effort.
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