Friday, January 4, 2013

Hope, fear, money and the regulation of stem cell therapies

If you were diagnosed with a life-threatening or life-impairing disease and told there was no proven therapy that could help you, wouldn’t you think about turning to an unproven therapy? That’s the dilemma facing many patients with diseases ranging from multiple sclerosis to arthritis, and many are turning to unproven and unregulated stem cell therapies to get the help they can’t find anywhere else. Sometimes with disastrous consequences.

We have written in the past about the dangers posed by stem cell tourism, going abroad to get stem cell therapies not available here because they haven’t undergone clinical trials to prove they are either safe or effective, and because they haven’t been approved by the FDA. Now Business Week magazine is taking an in-depth look at how it is not just overseas clinics that offer these therapies, but that it is becoming increasingly common here in the US. The reporter focuses on one company, showing how it tried to offer a variety of non-FDA approved stem cell therapies in Texas.

The article looks at the story from both a medical and business perspective and highlights some of the problems facing consumers in trying to decide if the therapies on offer will help them:
Stem cells hold enormous promise, but promise isn’t proof, and anecdotal evidence isn’t science.

“Stem cells seem so seductive,” says George Daley, a founder of the Harvard Stem Cell Institute, an organization that supports research and its clinical applications. “It’s easy to be told how they work and think they can help you. But we know from centuries of experience with medicine that anecdote is a very unreliable way of making medical advances. Medicine has been misled since the time of the leeches.”
The reporter follows a number of patients who have turned to companies offering unproven and unregulated stem cell therapies. In some cases the patients say the therapies have helped; in others, the patients have ended up suing the companies because they feel they were given a treatment that never had a chance of working.

At the heart of this is a mix of hope, fear, and money. Many of these therapies can cost tens of thousands of dollars, and are not covered by insurance. That’s a very tempting market for companies who don’t want to wait for FDA approval to bring in cash-paying customers. Business Week writes:
"But people who want to make money don’t want to wait to make money,” says Paul Knoepfler, an associate professor at the University of California at Davis School of Medicine who conducts research with induced pluripotent and cancer stem cells. “And there’s a lot of patients who don’t want to wait, either."
Knoepfler, who is a CIRM grantee, blogs regularly on the topic of unproven stem cell therapies. There are no easy answers or quick solutions to this problem. Good science, proving that these therapies work and are safe, takes time. Many patients may feel they don’t have the luxury of time. And there are all too many companies out there willing to take advantage of those fears and cash in on them.

K.M.

1 comment:

  1. At least they're trying to help...

    January 7, 2013

    BrainStorm’s Clinical Trial is Fast-Tracked to Phase IIa,
    Representing a Major Milestone for the Company

    NEW YORK & PETACH TIKVAH, Israel—January 7, 2013-- BrainStorm Cell Therapeutics (OTC.QB: BCLI), a leading developer of adult stem cell technologies for neurodegenerative diseases, announced today that the Israeli Ministry of Health has fast-tracked its current Phase I/II ALS safety trial to a Phase IIa dose-escalating trial, significantly expediting the company’s clinical development program and saving valuable time. The trial, which will evaluate the safety and preliminary efficacy of BrainStorm’s NurOwn stem cell therapy candidate, will be launched immediately at the Hadassah Medical Center in Jerusalem.


    The Ministry of Health approved acceleration to a Phase IIa trial based on evaluation of only 12 of the initial cohort of 24 patients in the company’s Phase I/II trial at Hadassah. In the Phase IIa trial, the second group of 12 patients will receive combined intramuscular and intrathecal administration of NurOwn cells in three cohorts, with increasing doses. The study participants, who have already been recruited, will be followed for three to six months after transplantation.

    “Acceleration to Phase IIa will save us critical time, enabling us to proceed much more quickly with achieving our goal of developing an effective treatment for ALS,” commented Dr. Adrian Harel, BrainStorm’s CEO.

    Professor Dimitrios Karussis, Principal Investigator of BrainStorm’s Phase I/II trial at Hadassah, commented: “Based on the positive results we have seen for the first 12 patients, we are eager to begin the next stage of clinical testing, and are delighted that the Ministry of Health has given us the green light to proceed to Phase IIa.”

    http://www.brainstorm-cell.com/index.php/news-a-events/company-news/234-january-7-2013

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