LAS VEGAS (FOX5) — The U.S. government is approving the drug leucovorin to address rising rates of autism among children, despite limited evidence that it works.
Doctors say the drug is already in high demand among desperate parents.
Leucovorin, which parents in the autism community have been calling “the little bottle of hope,” gained wide popularity last year when families of children with autism who are nonverbal began reporting that after only a few days of using the medication their children had speech. By late fall, the medication was fast-tracked for FDA approval.
Local family tries new approach
Seven-year-old Christopher Wilson has autism and has been nonverbal since birth. His father Shawn Wilson said through early intervention and behavioral therapy at Touro University’s Pierce Autism Center, he has seen improvements over the years.
“He has his own language. He has some words that he used,” Wilson said.
Jennifer McConnell, a board-certified behavior analyst at the Pierce Autism Center, explained how behavioral therapy works.
“We’re working on communication. For him to be able to have functional communication, meaning that as he expresses his wants and needs, it’s something universal that anyone can understand,” McConnell said. “The goal of the therapy is to identify a behavior that either needs to increase or decrease, and to teach a skill that will replace that behavior.”
But with language still out of reach for Christopher, Shawn wanted to try a new approach.
“I saw recently, RFK Jr., I think it was back in September or October time, he came out with that thing with the leucovorin,” Wilson explained.
Drug shows limited effectiveness
Leucovorin, an inexpensive generic drug derived from folic acid used to treat cancer patients undergoing chemotherapy, was being touted as a cure for autism able to unlock speech in nonverbal children.
“What was your hope?”
I was hoping the magic would happen in three days and he’d just start talking. You know? It didn’t happen,” Wilson said.
Dr. Devraj Chavda is one of only about a dozen pediatric neurologists in the state and Christopher’s doctor. He said these results are not uncommon, as the drug has only shown limited effectiveness.
“It’s very clear that this is not a cure, but there is a lot of misunderstanding in the community that it is. However, we do not have enough evidence or enough research to study,” Chavda said.
Chavda explained the drug warrants additional study, particularly for patients with a deficiency of folate, or vitamin B9, in the brain that may play a role in autism. But for now, he said, it should only be taken carefully.
Christopher has been on the medication for a month, but Shawn told FOX5 Dr. Chavda will discontinue it if there are no improvements by the end of month six. When asked if he would recommend to other parents, knowing what he knows now, Shawn said yes.
“You just want to do the best thing you can for your kid. You want to help them out. And you want them to have a normal life, just like we would expect,” Wilson said.
Supply and access concerns
Experts have cautioned that the two biggest challenges moving forward will be access and supply. Leucovorin is a generic, so supply could vary along with quality as different manufacturers use different additives.
The U.S. government is currently recruiting patients to take part in the largest clinical trial to study the drug’s effectiveness in autism. That trial is set to begin sometime this year.
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