Relaxed scientific protocols and standards within the Department of Health and Human Services have led to an increase in clinics offering experimental stem cell treatments to parents of children with severe autism. Despite being technically unapproved by the Food and Drug Administration, parents are shelling out tens of thousands for treatments that claim to improve language and social skills and reduce problem behaviors.
Operating ‘beyond the bounds of FDA approval’
Although there is a lack of FDA approval and little evidence of its efficacy, stem cell treatments for autism are being steadily provided across the country. Children with autism “as young as 18 months old” are getting “unapproved stem cell treatments” at clinics in Florida, Texas and elsewhere, “part of a growing market operating beyond the bounds of FDA approval,” said The Guardian.
The procedure involves sedating a child before administering intravenous doses of millions of stem cells, “commonly derived from human umbilical cords harvested at birth,” said The Guardian. Sometimes the doctors providing the treatment have “no scientific expertise in autism or child development.” Instead, they have “entered the booming stem cell sector,” billing the procedures as “regenerative medicine” for children, “some of whom have severe disabilities.”
As stem cell clinics “multiply across America,” they are “finding an influential ally in the U.S. Health Secretary Robert F. Kennedy Jr.,” said The Guardian. Kennedy’s influence could lead to new policy, said Paul Knoepfler, a stem cell biologist and unofficial watchdog of stem cell clinics, to the outlet. The FDA has not taken action in the last 18 months. This could mean a “big change coming from the FDA very soon, backing off oversight of birth-related stem cells.”
Several clinics prominently cite an early Duke University study involving 25 autistic children that “suggested possible improvements following umbilical cord stem cell infusions,” said Trial Site News. But a “larger and more rigorous follow-up trial” involving 180 children failed to “demonstrate significant improvements in core autism symptoms compared with placebo controls.” Similar results emerged from a “placebo-controlled study conducted by Sutter Health.” This has led researchers to conclude that the “evidence does not currently support routine use of stem cell therapies for autism outside formal clinical research settings.”
Up until now, Americans seeking stem cell therapies for autism have looked abroad to places where they are approved and federally regulated or operating in grey areas. That has fed a flourishing multibillion-dollar industry of “stem cell tourism” in places such as “Mexico and Panama” and “as far afield as Abu Dhabi,” said The Guardian.
But most European countries limit the use of stem cell injections to clinical trials, and they are not an approved treatment for autism. In January, the Indian Supreme Court ruled that stem cell therapy cannot be used as a clinical treatment for autism spectrum disorder, making it clear it is “not only unethical but amounts to medical malpractice,” said Indian network NDTV.
When ‘hope outpaces evidence’
The biggest lesson from this story is “not political,” said Trial Site News. “It is human.” Parents seeking out unapproved stem cell therapies are “not irrational.” Most are “navigating difficult realities” with “limited options and enormous responsibility.” The danger emerges when “hope outpaces evidence.” The appropriate response is “neither unquestioning enthusiasm nor reflexive dismissal” but rather “rigorous clinical research, transparent reporting, long-term safety monitoring and honest communication with families.”
After RFK promised to find a cure for autism last year, some people were “appalled and fearful,” clinical social worker Jennifer Cork said at Psychology Today. However, plenty of families whose children require substantial support were “relieved that someone in a position of power was finally talking about their struggles.” The issue is that these families “don’t need untried, expensive treatments.” They need “affordable therapies, respite care and adequate accommodations that they don’t have to fight for.” They also need Kennedy to “remember that autistic people are human beings, not lab rats.”
Desperate parents are putting their faith in untested hands
