Wednesday, May 22, 2013

Italians allow untested use of stem cells

Today, the Italian government approved an untested use of stem cells that advocates say could be the last hope for some critically ill people. However, despite a lot of hope on the part of families—and some celebrities—there’s no evidence that the cells work or are even safe.

Lawrence Goldstein of UCSD wrote about the issue last week in the San Diego Union Tribune. He’s part of CIRM-funded research into possible therapies for Alzheimer’s disease and Lou Gegrig’s disease (ALS) and knows the long path from having a good idea to testing that idea and showing that it actually works. He writes:
As scientists and doctors who study devastating diseases like Alzheimer’s, we are sometimes asked by patients why, when things seem hopeless, they shouldn’t resort to untested drugs or unproven therapies. “I’m already so badly off,” they say, “how could it be worse?”
It can always be worse.
An unproven drug or therapy may not kill, but what if it caused incurable pain? What if it did nothing at all but render your family bankrupt? Questionable stem cell treatments often cost tens of thousands of dollars, not just in faraway countries but across the border and in San Diego where they may be offered under various guises.
The EMBO Journal carried a commentary about the case earlier this month arguing that allowing unproven therapies could harm patients.
This case raises multiple concerns, most prominently the urgent need to protect patients who are severely ill, exposed to significant risks, and vulnerable to exploitation. The scientific community must consider the context—social, financial, medical, legal—in which stem cell science is currently situated and the need for stringent regulation.
The Italian government did, at the last minute, accept amendments to ensure that the cells will only be available in the context of clinical trials. What this means is that scientists will follow up with people receiving the cells to see if there were side effects and whether their condition improved.

Follow up is required in clinical trials in the United States and other countries before allowing a new therapy to become widely available. However, in most cases research groups have to provide evidence that their approach is likely to be safe and is worth testing in people before those trials begin. The Italian group submitted no such evidence.

Nature wrote about the approach, being promoted by the Stamina Foundation:
Stamina claims to have treated in the past six years more than 80 patients with diseases ranging from Parkinson’s disease to muscular dystrophy. Many of the patients have been young children. In the therapy, mesenchymal stem cells are extracted from the bone marrow of the patients, manipulated in the laboratory and re-infused into the patients.
Yesterday we wrote about trials testing stem cell-based therapies for heart disease. As we saw, some approaches that looked promising turned out not to be as effective as people had hoped. Others are starting to show promise. That’s the reason for carrying out careful clinical trials. Without testing different approaches it’s impossible to know which actually work.



  1. Funny thing about the self centered research establishment that requires donations, stock offerings, grants, etc. while they earn and burn both money and time is they think people seeking these experimental treatments have an abundance of time and have other options. The patients recognize the risks. Clinical trials are going no where. The drugs that come out have significant side effects and the costs are obscene. Maybe if the industry was not so worried about protecting profits and patents more cures would be available to the general public...I see more people making money than people being saved. The gern fiasco was the last straw for many years?...and then what? for Dr. Goldstein's objections...we're already in pain and most of us are already want to help? Do something tangible. As for the Italian law let's see what happens...there might be some researchers out there who really want to help mankind...maybe they will now. If I understand correctly the Centre of Excellence on Neurodegenerative Diseases at the University of Milan use stem cells and are highly regarded. This law might even push them to use their expertise in cases where compassionate use is appropriate.

  2. Over 600 patients asking stem-cell treatments

    The law passed on Wednesday budgets three million euros for an 18-month stem-cell therapy research programme sponsored by the health ministry and set to begin July 1. The Higher Institute for Health (ISS), a state health institution for research and education, will coordinate research, and work with the Italian pharmaceutical agency AIFA and the National Transplant Centre.

  3. stem cells bad but this is fine???????????

    MS Patients Choose Death Risk With Biogen Idec, Inc. (Massachusetts) (BIIB)'s Potent Drug Treatment

    5/23/2013 8:26:23 AM

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    Kari Antin, a 40-year-old financial analyst in Minneapolis, knows she’s taking a life-or-death risk with every monthly infusion of Biogen (BIIB) Idec Inc.’s Tysabri. She is among the 55 percent of multiple sclerosis patients who harbor a potentially lethal virus that can be reactivated by the drug, allowing it to creep from the kidneys to the brain, where it destroys cells that protect the nerves. The risk soars after two years of treatment. Still, Antin insists on sticking with the drug she credits with restoring her health.

    It’s a dilemma faced by almost 60,000 multiple sclerosis patients worldwide who continued on Tysabri beyond the recommended two years. It’s also new territory for Biogen, as the number of patients pushing the time limit increases, and a concern for prescribing doctors who increasingly find patients resolute about staying on the potent medicine that reduces the risk of relapse by 68 percent.