In brief
President Donald Trump's administration has controversially approved leucovorin, previously a generic drug, as a new treatment for autism. While some doctors are prescribing the medication, critics, including prominent autism researchers, point to a lack of substantial evidence supporting its efficacy. Calls for more research on the medication escalate as the administration seeks to treat a challenging brain disorder, with many cautioning against premature endorsement without rigorous clinical trials.
WASHINGTON (AP) — When President Donald Trump’s administration announced it would repurpose leucovorin, an established drug as a treatment for autism, it surprised many experts—including the health official who initially proposed it.
Dr. Richard Frye, a child neurologist from Arizona, had been discussing a tailored version of leucovorin with federal authorities, assuming that more extensive research would be necessary.
“We were genuinely surprised that they approved it so swiftly without extensive studies or data,” Frye remarked, reflecting on the rapid rollout of the guiding announcement on autistic treatments which critics claimed could amplify unverified therapies.
Government spokespersons remained silent on the concerns raised following the announcement.
Leading autism organizations quickly distanced themselves from the decision, labeling the supporting studies as weak and minimal. Psychiatrist David Mandell from the University of Pennsylvania expressed doubts about leucovorin, declaring, We lack substantial evidence that it effectively treats autism symptoms.
Despite these cautions, Frye and his colleagues argue the medication merits further investigation, particularly concerning patients with potential folate deficiencies that may impact autism.
“Moving forward without established guidelines could lead to unpredictable outcomes,” cautioned Dr. Lawrence Gray, a specialized pediatrician. “Positive results from small-scale studies do not always replicate in larger populations.”
The potential of leucovorin
The case for leucovorin began over two decades ago, generated by initial research correlating low folate levels in some autism patients.
In 2018, Frye noted improved language skills among children with autism taking leucovorin in a small trial, yet replication has proven variably successful in international studies.
Discussions with the FDA gained momentum after Frye and colleagues formed the Autism Discovery Coalition, hoping to promote innovative therapies within the Trump administration. Yet the FDA's latest announcement, indicating general approval rather than endorsement of a revised version, may alter the financial landscape for researchers and those seeking pharmaceutical advances.
As dialogue continues, the overarching consensus among experts emphasizes the need for rigorous trials to discern the efficacy of leucovorin in autism treatment.
“While we explore new approaches, we must remain anchored to rigorous scientific evaluation,” Gray concluded.
