Peds group 'advises against leucovorin for autism'

 Faced with soaring demand for leucovorin for use in children with autism, the American Academy of Paediatrics (AAP) has said there is "limited evidence" for its safety and effectiveness.

Parents of children with autism spectrum disorder (ASD) have been clamouring for access to the drug – a form of vitamin B9 also known as folinic acid – since President Trump claimed that it could be a treatment for speech-related deficits associated with autism.

Those comments, made at the same time as Trump also linked autism to childhood vaccinations and the painkiller acetaminophen in a press conference last month, were followed by the FDA saying it had "initiated the approval" of leucovorin calcium tablets for cerebral folate deficiency (CFD), which causes autism-like symptoms.

Leucovorin was originally developed by GSK under the Wellcovorin brand to prevent side effects in people treated with some forms of chemotherapy, but is currently available as a generic.

After the FDA's announcement, which alluded to the rapid increase in autism diagnoses in the last 20 years, GSK said it would file for approval of the drug in the CFD indication, having withdrawn it from the market in the late 1990s.

The rise in autism diagnoses has been repeatedly held up by the Trump administration and HHS under Secretary Robert F Kennedy Jr as an epidemic caused by exposure to some environmental factor, although scientific experts have attributed it to increased awareness, broader diagnostic criteria, and improvements in screening and detection.

The AAP's newly-issued interim guidance advises against the use of the drug in autistic children, whilst acknowledging that "small studies of leucovorin have suggested potential benefits to communication and behaviour for some autistic children with CFD."

That is somewhat in line with the FDA's position, which has also said that use of the drug more broadly in ASD would be premature, but the comments at the press conference appear to have driven a surge in demand.

Kristin Sohl, a member of the AAP's Council on Children with Disabilities Executive Committee and past chair of its Autism Subcommittee, explained that the reason leucovorin is not being recommended more broadly in autism is that "we know that autistic children deserve the same level of evidence to support interventions for them as anyone else."

The guidance points to big holes in knowledge about the use of leucovorin in autism, including "questions about who may benefit, what dosing and monitoring are appropriate, and what the long-term safety profile looks like."

Paediatric prescribing clinicians should be prepared to answer families' questions and engage in shared decision-making, according to the AAP. They should have "compassionate, science-based conversations about the family's goals, the current evidence on leucovorin and its potential risks and benefits."

https://pharmaphorum.com/news/medical-group-advises-against-leucovorin-autism