During a Monday press conference about various autism topics, federal health officials noted the potential for an existing cancer drug to double as therapy for the neurodevelopmental condition.
FDA Commissioner Marty Makary, National Institutes of Health (NIH) Director Jay Bhattacharya, and Centers for Medicare and Medicaid Services Administrator Mehmet Oz discussed research suggesting that leucovorin, a prescription vitamin used to treat cancer and anemia, is effective in off-label use for children with autism.
The FDA plans to approve prescription leucovorin as a "potential treatment for speech-related deficits associated with ASD," according to a press release from HHS. The agency will update the label for leucovorin to indicate treatment for cerebral folate deficiency, which has been associated with autism.
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After the label update, state Medicaid programs will be able to cover the drug for autism treatment, the release stated.
The NIH will also launch new research initiatives into leucovorin's safety and effectiveness.
Studies have shown that leucovorin could help some non-verbal children gain the ability to speak.
Leucovorin functions similarly to folic acid, helping to restore folate levels in patients who may have difficulty absorbing or using it, according to Katy Dubinsky, a New York pharmacist and the CEO and co-founder of Vitalize, a private supplement company.
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"Leucovorin is FDA-approved to reduce the toxic effects of high-dose methotrexate in cancer treatment and to prevent or treat certain types of anemia caused by folate deficiency," Dubinsky previously told Fox News Digital.
While leucovorin isn’t yet prescribed for the treatment of autism, some families have found that its off-label use has led to significant improvements in autistic children’s communication skills, experts say.
Richard E. Frye, M.D., Ph.D., a behavioral child neurologist in Arizona, has studied the drug in his research of experimental treatments for children with autism spectrum disorder.
"Leucovorin is a special form of vitamin B9 that is water-soluble and does not build up in your body," he previously told Fox News Digital.
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"Folates have been found to be safe long-term for many years and that is why we supplement our food with folate."
Leucovorin's main benefit for children with autism is improvement in language, Frye said, but other advantages may include enhanced social function, reduced repetitive behaviors and stronger attention skills.
"We do find that leucovorin helps a substantial number of children with autism," he said. "Language is one of the most obvious things, so that is what we measured in our clinical trial."
Dr. Marc Siegel, clinical professor of medicine at NYU Langone Health and Fox News' senior medical analyst, also spoke to Fox News Digital about why the drug may have this positive impact.
"Central folate deficiency is a lack of folate in the brain, which can interfere with neurological function," he said earlier this year. "A high percentage of children with autism spectrum disorder have been found to have antibodies against folic receptor alpha in the brain."
Leucovorin is converted to folate in the brain by a different pathway, Siegel said.
"Small studies are showing that it improves neurological function and communication skills in kids with ASD, but larger studies need to be done."
The fact that leucovorin has already been used long-term helps to reduce the associated risk, doctors say.
"One of the advantages of using leucovorin is that it's been used for decades to protect the body from the effects of chemotherapy, so we know what dose to use and any side effects it may produce," Frye told Fox News Digital.
However, some children do become "extremely hyperactive," do not respond to leucovorin and need alternative treatments, the doctor noted.
It’s important for families to work with a medical professional when exploring the use of leucovorin for autism, he advised.
"Leucovorin is given at much higher doses than regular vitamins, so it’s very important to work with a doctor knowledgeable in how to prescribe it," Frye said.
While leucovorin is available at pharmacies, Frye said some children need a compounded form because they are sensitive to some of the additives in commercial products.
"Our goal is to get leucovorin approved by the FDA for autism so it can be widely prescribed to children as one of the first treatments once they are diagnosed," he previously said.
Dubinsky agreed physicians may prescribe the "widely used and well-tolerated drug" for autism treatment based on their "clinical judgment and emerging evidence" — but also stressed the importance of clinical trials.
"FDA approval would provide stronger medical backing, making it a recognized treatment rather than just an off-label option," she told Fox News Digital.
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"This could lead to insurance coverage, standardized dosing guidelines and broader acceptance in the medical community."
Siegel added that leucovorin is a "safe drug" and should be considered for large-scale use based on the findings of recent research.
Frye said there is "no magic bullet" for autism, with many children benefiting from a combination of multiple treatments.
"It is unlikely that leucovorin will be used alone — it must be used with behavioral and speech therapy," he said. "We believe that leucovorin can improve outcomes for these therapies."
Andy Shih, Ph.D., chief science officer of Autism Speaks in New York, noted that every child’s experience with autism is unique.
"There is little evidence to suggest that the benefits observed [in individual cases] can be generalized to the broader autistic community," he previously told Fox News Digital.
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"While some small studies have explored leucovorin as a potential treatment for people with autism, larger controlled clinical trials are needed to determine its efficacy and safety. We urge families to consult with their doctors before considering off-label treatments."
Dubinsky added that if larger studies confirm leucovorin’s effectiveness, it could become a more accessible treatment for children with autism who may benefit from it.
