Unproven Stem Cell Treatments for Autism Surge in the US Amid Regulatory Rollback Under RFK Jr.

Across the United States, children with autism as young as 18 months old are receiving unapproved stem cell infusions at a growing number of clinics, a practice that scientists say lacks robust evidence and exploits desperate families. The surge comes amid what observers describe as a retreat from enforcement by the Food and Drug Administration (search)Search term under Health and Human Services Secretary Robert F. Kennedy Jr.

The procedure typically involves sedating the child before intravenously administering millions of stem cells derived from human umbilical cords. Clinics in Florida, Texas, and elsewhere charge between $8,000 and $20,000 per infusion, often recommending repeat treatments that drive some families to take out loans against retirement savings or second mortgages.

A market operating beyond FDA approval

Paul Knoepfler, a stem cell biologist at the UC Davis School of Medicine who tracks stem cell clinics through his blog The Niche, has detected a marked decline in FDA enforcement activity. "We haven't seen the FDA taking action in the last 18 months. I think we're going to see big change coming from the FDA very soon, backing off oversight of birth-related stem cells," he said.

Arnold Kriegstein, professor of neurology at the University of California San Francisco, expressed alarm: "I'm appalled that this is being allowed to go on in the US, and that so many desperate people are being taken advantage of with a 'treatment' that in my view is completely bogus."

The FDA has approved stem cell therapies only for a narrow range of indications, such as bone marrow transplants and immune system reconstitution after chemotherapy. Autism, a heterogeneous neurodevelopmental condition with no single cellular mechanism, is not among them.

The evidence gap

Clinics marketing stem cells for autism frequently cite a small 2017 Duke University (search)Search term study in which more than two-thirds of 25 autistic children appeared to show improvements in speech, socialization, and eye contact. However, a larger, more rigorous Duke trial released in 2020—a double-blind, placebo-controlled study involving 180 children—found the opposite: the treatment "was not associated with improved socialization skills or reduced autism symptoms."

A separate placebo-controlled study by Sutter Health with 29 children reached a similar conclusion, finding minimal evidence of clinical effectiveness.

"We still don't understand exactly what goes wrong in autism, and until we have an understanding it will be impossible to design a rational approach," Kriegstein said.

Kennedy's influence and the regulatory shift

Kennedy has publicly signaled his intent to loosen federal oversight of the "wellness industry." In an interview with podcaster Gary Brecka, he said he would "end the war at FDA against alternative medicine," including the "war on stem cells," adding that the FDA should "not tell physicians what they can and cannot prescribe."

Early in his tenure, Kennedy invited Dr. Chadwick Prodromos, an orthopaedic surgeon who had been operating largely out of Antigua, to a Washington roundtable to discuss diluting federal regulations on stem cell treatments. Prodromos, who had personally administered stem cells to Kennedy for spasmodic dysphonia, is now offering umbilical cord stem cell infusions to autistic children from clinics in Naples, Florida, and Dallas, Texas.

Prodromos told The Guardian that he is operating under Florida's new law allowing unapproved stem cell therapy—specifically under the "pain management" category, asserting that "the patients we treat for autism all have documented need for pain management." However, legal expert Jeff Cohen of the Florida Healthcare Law Firm noted that Florida's statute "only speaks to stem cells, and only speaks to physicians applying them for three specific purposes"—orthopaedics, wound care, and pain management—and that autism is not one of them.

A new trial in Tijuana

In April, Tracy Slepcevic—a Kennedy appointee to the federal committee that helps steer national autism research policy—announced a "pioneering" experiment at the Autism Health Summit in San Diego. The trial, set to begin July 15 in Tijuana, Mexico, will administer umbilical stem cells to 120 children aged seven to 15 with autism diagnoses. The overall cost is estimated between $1.5 million and $5 million.

Slepcevic has partnered with Ed Clay, founder of the Cellular Performance Institute (search)Search term in Tijuana, who told the summit: "The goal definitely is to, if this works, get it approved in the United States."

Families paying to participate

Christy Holdren, mother of eight-year-old Landyn, spent $12,500 on a first round of stem cell treatment last October and is preparing to spend $15,000 more for a second dose, taking a loan against her retirement savings. "He actually looks at us and not through us, and that's huge for us," she said of perceived changes, though she acknowledged Landyn's aggressive and self-harming behavior is worsening again.

Katelyn Cook, 27, from Erie, Pennsylvania, paid $8,000 for her five-year-old son Colston's infusion. "I mean, it's not fun. But I paid $10,000 for my car, and my kid, I value him over every single thing in my life. I would spend anything to give him a better chance at life."

Alycia Halladay, chief science officer of the Autism Science Foundation, warned of collateral harm: "We've seen families drop out of evidence-based interventions like speech therapy or applied behavioural analysis therapy, or even stop medication, because they've gone for stem cells and think they no longer need it."

Safety concerns and the fringe

While stringently conducted trials have shown little safety risk from umbilical cord stem cell infusions, scientists remain concerned about the broader unregulated landscape. A Pew Charitable Trust survey documented 360 cases of side-effects including life-threatening blood infections, heart attacks, and organ damage from unapproved stem cell clinics.

On the more extreme fringe, Mike Chan, a Malaysian doctor operating mainly out of Thailand, markets five-day "autism treatment packages" for approximately $13,800 and injects autistic children with fetal stem cells extracted from slaughtered merino sheep raised in Germany. Knoepfler called such cross-species techniques "extremely worrisome. We don't know how the human body reacts to rabbit or sheep stem cells. It might trigger activity by the immune system in ways that are harmful."

Fiona O'Leary, a campaigner against pseudoscience and mother of four autistic children, summarized the situation: "Kennedy is undoing so much hard work to hold back the misinformation. Autistic kids are being experimented on. Families are spending huge amounts, and losing everything."