US EditionOff-Label Drug Helps One Boy with Autism Speak, Parents Say. But Experts Want More Data
The FDA is fast-tracking leucovorin, a targeted vitamin therapy shown to improve language and social skills in some autistic children with cerebral folate deficiency, based on limited trials.
- Recently, the Food and Drug Administration began approving leucovorin as a treatment for cerebral folate deficiency , a neurological condition affecting some children with autism.
- Researchers observed over the past 15 to 20 years that some children with autism have folate receptor autoantibodies blocking folate transport, and a 2016 randomized controlled trial found high-dose folinic acid improved verbal communication.
- Leucovorin is a biologically active folinic acid derivative used in cancer chemotherapy for decades, and clinicians should assess cerebrospinal fluid folate or folate-receptor antibody testing to monitor treatment.
- Some children with confirmed folate pathway issues may see language and social gains with leucovorin, but families should expect possible irritability, hyperactivity, GI symptoms, or rare behavioral worsening.
- Researchers urge larger, confirmatory trials and National Institutes of Health funding despite five blinded controlled studies and three randomized controlled trials showing positive speech effects.
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Off-label drug helps one boy with autism speak, parents say. But experts want more data
By Céline Gounder, KFF Health News Caroline Connor’s concerns about her son’s development began around his first birthday, when she noticed he wasn’t talking or using any words. Their pediatrician didn’t seem worried, but the speech delay persisted. At 2½, Mason was diagnosed with autism. Related Articles Record attendance marks 2025 Regional Home Garden Contest Awards celebration in Pottstown FDA approves another …
Experts Share What Parents Need To Know About Leucovorin and Autism
The FDA is approving leucovorin to treat some kids with autism. Experts explain what families need to know.GettyImages/ULROKey PointsThe FDA is in the process of approving leucovorin to treat some children with autism.Leucovorin may help some autistic children with folate deficiency improve their language and social skills.Experts stress that more research is needed, and this treatment is not for every child.The Food and Drug Administration (FDA…
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