Stem cells to treat autism: success or wishful thinking?

Six months after eight-year-old Ari Goodman received stem cell therapy at a clinic in Panama, he appears to be winning his battle with the autism he has coped with since he was 18 months old. Since his first treatment last May, Ari is showing significant signs of improvement. According to his mother, Clara Klein Goodman, he is able to read simple words, answer questions, and hold conversations with his parents.

“I feel like we are getting Ari back,” said Clara in a Nov. 8 phone interview. “We can see that the wheels are turning. We are not out of the woods yet, but having this treatment and seeing how well he has done is giving me hope that anything is possible.”

As the U.S. Food and Drug Administration has not yet approved the use of stem cells for people with autism, the family had to travel to the Stem Cell Institute in Panama City, Panama. For the Goodmans and others of limited means, the expensive procedure — not covered by insurance — is difficult to afford. After a story on the Goodman’s struggle appeared in NJJN last year, Clara and her husband, Daniel, raised $15,000 for the treatment and associated costs. The early results have shown such promise, they are trying to raise another $15,000 for a second round of stem cell therapy at the same clinic. 

The Goodmans asked Rabbi Aaron Gruman, the religious leader of their synagogue, Congregation Toras Emes, an Orthodox congregation in East Windsor, to assist with the fund-raising efforts. He immediately agreed; tax-deductible contributions toward Ari’s treatment go through Toras Emes.

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“We are a supportive group and, like the Jewish community at large, when a challenge comes our way we ask how we can meet it,” Gruman said. “Right now the focus is the next set of treatments and what can we do to help them.”

Yet despite the positive reviews of the Goodmans and other families, the scientific community is less optimistic. The FDA has yet to complete a 2012 study it began on using umbilical cord stem cells for people with autism, but there is much skepticism among those in the scientific community about the treatment’s effectiveness. 

Paul Knoepfler, a professor of cell biology and human anatomy at the University of California School of Medicine in Davis, told NJJN, “To date, I’m not aware of any conclusive data showing that stem cells are successful in treating autism…. A very small number of FDA-approved studies are ongoing,” but, he said, they are in their early days, so it’s “too soon to say what they will find.”

Notwithstanding the doubts of much of the medical community, others have claimed encouraging results similar to the Goodmans’ from the controversial treatment. The official website of the Stem Cell Institute, which did not return multiple requests for comment, includes praise for the embryonic stem cell infusions, written, the institute says, by parents whose children have received the treatment and seen great improvements to their quality of life.

Among the other families reporting successes similar to that of the Goodmans is that of Kenneth Kelley of Bangor, Maine. Kenneth began stem cell treatments in 2009 at the age of eight, five years after he was diagnosed with severe autism. “He could not have a conversation. It was pretty scary. He would scream a lot and antagonize his younger sister, who was just three years old,” said his mother, Marty. “We were ready to put him in an institution.”

After six stem cell treatments, he began to read. “At this point his only major issue is a speech impediment,” she told NJJN.

A documentary she made and posted on YouTube, Ken’s Journey to Recovery, begins with an angry, screaming two-year-old and ends with a charming pre-adolescent boy buying a snack at a local grocery store and thanking the clerk before playing a game of Monopoly. Four years since Kenneth’s last treatment, Marty said, she plans to take him to Panama one more time “to see if we can bring him little further.” 

Three years ago, Amanda Fannon’s son, Evan, then three, was diagnosed with autism. 

“Before the treatments he had extreme hyperactivity. He was nonverbal. He couldn’t communicate or understand questions. He couldn’t function on day-to-day things like brushing his teeth,” Amanda told NJJN. 

Evan and his mother went to the institute in Panama for the first time in June of 2015, and within two weeks after returning to their Minnesota home in a suburb of Minneapolis, his speech improved. That September, at age five, he was reevaluated and upgraded from “severe” to “moderate” autism. 

They revisited the institute in June 2016, and Evan’s speech and his ability to understand has improved, according to Amanda. The Fannons hope to return to Panama a third time, “but we are working on the finances right now. It is very expensive.”

A major donor to stem cell research is The Marcus Foundation, which makes large-scale grants to find “innovative therapies” for treating autism and other severe diseases, as well as to many Jewish causes. Among the foundation’s grant recipients is Duke University; the Duke medical center has been testing stem cell treatments for children under six who have autism. In a statement to NJJN, foundation chair Bernard Marcus — a Newark native — said he could not discuss the clinical trial at Duke, but added that he and his colleagues “are very hopeful that it will be successful. We await the results and hope they are positive.”

Since 2002 the International Society for Stem Cell Research has been amassing data from 4,100 scientific professionals researching the effectiveness of stem cell therapies. Jack Mosher, the organization’s scientific affairs manager, was dubious that the use of umbilical cord stem cells could treat the symptoms of autism. “Scientifically speaking it is hard to understand how it could work,” he told NJJN.

When asked about the variety of success stories reported, Mosher said, “I understand and respect patients and parents of patients who go through difficult experiences with autism, but anecdotal evidence doesn’t really tell us anything. 

“If I were a parent of someone with autism, I could probably appreciate that perspective more,” he said. “But from an analytical scientific perspective, I think that always has to be balanced with data. In scientific and medical communities the way we determine whether something is effective is based on clinical trials and studies that aren’t just anecdotal.”

But try telling that to a parent who has seen the benefits firsthand. 

“God doesn’t give you more than you can handle,” Clara Goodman said. “We feel it was divine intervention that led us down this path toward finding help for Ari.”