The “unwarranted hype” of stem cell therapies
Some patients feel that they just don’t have the time to wait for good evidence from long-term clinical trials to come to fruition. Take this fundraising plea from a patient with ALS, a disease in which half of patients die within three years of their first symptoms:
“I have only 32% breathing left, I can’t speak and I lose (sic) control of my hands and legs. I have where a respirator mask for 12-14 hours a day … PLEASE HELP!
Doesn’t the patient who has “delivered to 32% of breathing” have the right to try anything that could save him / her, the crowdfunding to do so, even if this treatment is administered in an unregulated clinic ? Barker fears that it is this vulnerability that is being exploited, citing potential risks including tumor formation.
But he also believes legitimate stem cell research will suffer. “If this does not work out, regulators could ban these therapies for times to come based on the belief that these clinics offer essentially the same therapies as centers that have slowly been working toward clinical trials through appropriate rational approaches.” , said Barker.
Although regulators must play a role in tandem with cross-border partnerships, there is a growing consensus that crowdfunding platforms should take responsibility as well. Campaigns for unsafe or insufficiently proven treatments continue to rise millions of dollars.
It’s true that these platforms can allow patients to explore options, stay hopeful, and cover insurance gaps, while also allowing families and friends to extend their support. But shouldn’t they also ban bad actors and stop the spread of disinformation (campaigns are shared hundreds of thousands of times on social media)?
Not necessarily, a GoFundMe suggested statement to Gizmodo earlier this year: “While we hope to be a useful resource for personal fundraising, we don’t think it’s up to us to tell them what decision to make. After a call to crowdfunding sites to rigorously review cancer appeals to protect patients from unproven or dangerous treatments (such as “ozone therapy” and vitamin infusions), GoFundMe banned users from seeking donations for treatment at some clinics in the month and now report that they are actively assessing concerns about certain stem cell campaigns.
“With these new concerns about some stem cell clinics, we are taking a thoughtful approach to approaching stem cell campaigns while continuing to provide a safe and secure place for people to fundraise for their needs and causes. We are reaching out to space experts and medical regulators to understand the latest regulatory developments and how these developments affect our customers, ”they said in a statement.
When presented with similar concerns, JustGiving has already declared: “We don’t think we have the expertise to pass judgment on this. In an email to the BBC, they claimed they made sure all crowdfunding pages followed legal requirements and claimed that “the safety and well-being of people using our platform, that they raise funds or make a donation to a cause, is always our priority “.
Tree of Hope, a UK crowdfunding organization and children’s charity, claims a different approach. Their medical committee takes advice on UK Stem Cell Foundation campaigns. “Families look to Tree of hope when all other options have been exhausted, ”said Lee Vallins, deputy general manager and head of family support for the association. “We work with amazing, albeit often desperate, parents and make sure we explore options with them, at their pace. But although they have supported a number of families in crowdfunding stem cell transplants, safeguards are in place.
“We gladly spend time with parents who call us to ask for advice on this type of therapy and quite often we turn down a request if parents are unwilling to consider recognized clinical trials or alternative treatment options,” explains Vallins. “While not one of the biggest crowdfunding organizations, we certainly strive to be one of the most ethical with our checks and balances throughout the process. “
Hunt for hope
Jay Shetty eventually received a two-hour infusion of stem cells through a cannula in his arm at Duke University. What were Shilpa’s expectations? “I knew it wasn’t a cure, I knew he wasn’t going to get away with walking and running. But I thought at least Jay would be able to sit up in six months, to be honest. Unfortunately, that didn’t happen, ”she says. “We haven’t seen any dramatic changes but he’s not as spastic, he’s more conscious, for us as a family, we think it’s big enough. It’s kind of a foundation for all physiotherapy.
But was stem cell therapy responsible for this improvement? Maybe Jay would have learned more skills growing up anyway? “It’s hard to pin down because we’ve been doing therapy for a long time,” says Shilpa.
To date, stem cell studies in cerebral palsy at Duke University Medical Center led by Joanne Kurtzberg, did not quite live up to expectations although the work is underway. In 2017, 63 children with cerebral palsy were randomized to receive treatment (a single infusion of cord blood) or a placebo. Disappointingly, the researchers found no change in the trial’s primary endpoint: motor function one year after baseline infusion. They reported better results at higher doses (highlighted in some media coverage), but Paul Knoefler at UC Davis School of Medicine is not convinced. “Based on the small study size, a high degree of variability within samples from the same groups, changes in the placebo group from what was expected and the modest nature of the possible differences with the higher dose compared to the placebo, I am not convinced that the reported effect was significant, ”he says. He also tells me that with a study by the Duke group on autism, these results “do not suggest a strong positive effect of umbilical cord cells for pediatric neurological disorders.”
Cells4Life, the cord blood stem cell bank that supported Jay’s fundraiser, still says on its website that “Duke’s trials have shown that cord blood can reverse symptoms of cerebral palsy.” (Cells4Life says its mission is to “conserve every baby’s cord blood.” Their price ranges from £ 1,495 ($ 1,816) to £ 2,090 ($ 2,538) with additional annual storage fees.)
The Shetty are not discouraged. “If we find a match and Duke opens a similar trial, I’m more than happy to pay for it,” says Shilpa. “If we have the money, we’ll keep doing it over and over again to be honest. In Jay’s case, there are a lot of empty spaces in his brain and he needs new cells.
Meanwhile, she and other parents will continue to browse the internet late at night, looking for anything that could help their children, in search of hope, calling for a cure. As long as they do, stem cell clinics will promise to answer their call.
Jules Montague is a writer and neurologist in London. His first book, Lost and found, explores what is left of a person when the pieces of their mind disappear – from dementia and brain damage to sleep disturbances and multiple personality disorders. His second book, The Diagnosis Healing, will be published next year and explore how medical diagnosis is deeply tainted by the forces of trade, imperialism and gender discrimination.
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