FDA approves leucovorin for rare genetic disease ‘cerebral folate deficiency,' not autism

The Food and Drug Administration (FDA) on Tuesday, March 10, approved the prescription drug leucovorin for a rare genetic disorder which affects approximately 1 in 1 million people.

The approval rejects all previous claims of the Trump administration about the benefits of drugs against autism.

The approval marks a stark departure from the White House briefing six months ago, when FDA Commissioner Dr Marty Makary and HHS Secretary Robert F. Kennedy Jr. stood alongside President Donald Trump supporting claims of leucovorin as a potential breakthrough for thousands of autistic children.

As reported in a study published in The Lancet, a 71% raise in paediatric prescriptions has been noticed, leaving some families desperate to find the drug and others turning to unregulated supplements.

However, the speculations come to an end on Monday, March 9, when senior officials confirm that there’s no evidence of leucovorin being effective against autism.

The rare disease against which it is proven beneficial is cerebral folate deficiency (CFD), which is a rare but treatable paediatric neurological syndrome.

In this disease, folate (vitamin) levels become low in the brain/ cerebrospinal fluid but remain normal in the blood, which causes developmental regression, seizures, ataxia, and cognitive decline.

It starts around 4-6 months. The disease is usually treated with folinic acid.

Common symptoms include irritability, sleep issues, delayed development, slowed head growth, speech difficulties, and movement issues.

A retired professor at Albert Einstein College of Medicine, Dr I. David Goodman, who studied this rare condition, stated that there’s no overlap between this genetic disorder and autism.

He also mentioned that the leucovorin has already been used to treat this rare deficiency since 2009.