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.