FDA finds little evidence that a generic drug can help people with autism
WASHINGTON– The Food and Drug Administration on Tuesday approved a generic drug for a rare brain disorder, while walking back suggestions from President Donald Trump and other administration officials that the drug held great promise for people with autism.
The agency said it approved leucovorin for children and adults with a genetic disorder that limits the supply of folate, a form of vitamin B, to the brain. FDA officials estimate that this ultra-rare disease affects fewer than one in a million people in the United States.
This is a major step back from statements made at a White House news conference in September, when Trump and FDA Commissioner Marty Makary announced the drug was being studied to benefit autistic patients, some of whom suffer from a form of brain vitamin deficiency.
“That could be 20, 40, 50 percent of children with autism,” Makary said at the news conference.
The White House event followed promises by Health Secretary Robert F. Kennedy Jr. to determine the cause of autism by September.
But senior FDA officials told reporters Monday that their review was narrowed to focus on the strongest evidence, which only supported the drug’s use by patients with the rare mutation that affects folate levels in the brain.
FDA officials also pointed out that a study supporting the drug’s use for autism was withdrawn earlier this year.
Leucovorin is a synthetic metabolite of folate, essential for successful pregnancy and recommended for women before conception and during pregnancy. The current FDA label covers the use of leucovorin to reduce the side effects of certain chemotherapy drugs and treat a rare blood disease.
Patients affected by the disease targeted by Tuesday’s approval suffer from movement disorders, seizures and other neurological problems that can resemble symptoms of autism.
But professional medical societies say it’s far from clear whether the drug helps people with autism.
The American Academy of Pediatrics does not recommend routine use of leucovorin in children with autism, including those with a brain disease known as cerebral folate deficiency. Some trials in this subgroup of patients “suggest a potential benefit,” the group says, but the research comes from small-scale studies.
Unresolved questions about the drug have not stopped American doctors from prescribing it.
An article published in The Lancet last week found that leucovorin prescriptions for children aged 5 to 17 were 71% higher than normal in the three months immediately following Trump’s press conference in late September. And families of autistic children have reported difficulty getting their prescriptions filled in recent weeks.
FDA officials told reporters that the agency was allowing imports of the drug by foreign drugmakers to help increase supply. The drug’s original manufacturer, GSK, has no plans to relaunch its version of the drug.
Trump officials initially decided to review the drug after speaking with an Arizona-based neurologist who prescribes the drug to autism patients and runs an online education company focused on the experimental treatment.
The theory behind the use of this medication is that some autistic people have specific antibodies that block folate from entering the brain. But the Autism Science Foundation and other groups note that non-autistic relatives of people with the disorder often have the same antibodies, suggesting they are not a factor in the disease.
Although there is no single cause of autism, most researchers say science points to genetic and environmental factors playing a role.
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