The FDA has initiated the approval of a drug, leucovorin (folinic acid), that could help kids who have "developmental delays with autistic features."
This isn't necessarily a treatment for autism, by the way.
From the FDA:
The U.S. Food and Drug Administration today initiated the approval of leucovorin calcium tablets for patients with cerebral folate deficiency (CFD), a neurological condition that affects folate (a vitamin essential for brain health) transport into the brain. Individuals with cerebral folate deficiency have been observed to have developmental delays with autistic features (e.g., challenges with social communication, sensory processing, and repetitive behaviors), seizures, and problems with movement and coordination.
...
The FDA is working with GSK, the innovator of Wellcovorin (leucovorin calcium), on a process to include the essential scientific information needed for the safe and effective use of these drug products for adults and pediatric patients with CFD. As the New Drug Application (NDA) holder for this medicine, GSK has preliminarily agreed to work with the FDA on this relabeling effort.
FDA Commissioner Dr. Marty Makary says one of the causes of autism (at least some disorders that look like autism) "is that the receptor on the blood-brain barrier that allows folate to be transported into the brain is blocked by an antibody ... so the autoimmune reaction blocks the receptor of folate, starving the brain of this critical vitamin." He says this medication, leucovorin, "bypasses that blocked receptor," and studies say "two-thirds of kids with a folate deficiency will show an improvement in their autism symptoms."
Again, this isn't a treatment for autism, necessarily. However, studies have shown that a majority of autistic children (58%-76%) have this folate deficiency. Do the math on that and with leucovorin we could see reduced autism symptoms in a decent number of autistic children (I am not a scientist or a mathematician, so I'm not going to pretend that my math checks out).
Let's hope and pray that this drug can get approval and improve lives.
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