President Trump’s Claims About Leucovorin & Autism

President Trump has claimed that a particular medication called Leucovorin is a “gamechanger” in autism treatment, and that “it could help make our kids healthy again”. This is entirely false. Leucovorin is a useful medicine to treat a highly specific condition found in some autistic children, but it is not a treatment for autism, and it does not have a significant positive impact on the symptoms of autism.

BuDS advises that the parents of autistic children, and autistic people themselves, take no notice of Trump’s announcement. It is simply not true.

This is twice in one week that we have been forced to correct dangerous misinformation from the US President and his coterie.

What Did Trump Announce?

President Trump announced on Monday (22 September) that the US Federal Drug Administration, which regulates medicines within the USA, will be approving the medication Leucovorin for the treatment of autistic children on prescription. There has been no confirmation from the FDA (as of 23 September) that this is in fact going to happen.

What Is Leucovorin?

Leucovorin (also known as folinic acid) is a form of folic acid, also known as vitamin B9. Folic acid is essential to many critical biological processes, for brain functions, and to prevent birth defects such as spina bifida. Folic acid is normally readily absorbed by the body from food or vitamin supplements. However, research indicates that some autistic children may have a rare condition where their brain does not absorb the folic acid it needs. This may be known as cerebral folate deficiency (CFD), or folate receptor alpha autoantibodies (FRAA).

People with CFD or FRAA may benefit from taking Leucovorin, because it can bypass the block in their brain and help them absorb the folic acid they need. This Leucovorin therapy treatment has been shown in the US to reduce some of the symptoms experienced by autistic children with CFD or FRAA, such as language impairment. However, because only a few specialist clinics are using this therapy, Leucovorin therapy is still regarded as experimental and unproven, and is not part of mainstream autism treatment. Leucovorin is currently not licenced in the US as a treatment for the symptoms of autism.

The Situation In The UK

In the UK, CFD and FRAA are not routinely recognised or treated under the NHS. As a result, there is no data about how many autistic people/children may have these conditions. There are no NICE guidelines for their treatment in the context of autism, and testing for them is not part of the normal treatment pathway for autism. Testing for FRAA requires specialised work not routinely available on the NHS, and testing for CFD requires a lumbar puncture which is a high-risk procedure in children.

Leucovorin is not licenced in the United Kingdom for the treatment of the symptoms of autism. It can be prescribed off-licence, typically by hospital consultants in paediatric neurology units. Leucovorin cannot be legally sold in the UK.

Leucovorin And Autism

It is important to realise that Leucovorin does not “cure” or “treat” autism as such. It is a helpful treatment for a medical condition sometimes found in autistic people, not a treatment for autism. It improves the uptake of folic acid in autistic children who lack that vitamin, and that has some benefits to the way their brain functions. It is completely wrong to suggest that Leucovorin is a general treatment for autism, or that it will make a big difference to most autistic children. There is currently no evidence to support these wild claims.

Leucovorin has significant side effects. It commonly causes nausea, vomiting, diarrhoea, fatigue, and headaches. In the trials on autistic children in the US, it caused negative behavioural side effects such as irritability or aggression, as well as positive improvements to speech and mobility. Much more testing and trials are needed to see if Leucovorin on balance is positive or negative, and whether long-term use might be harmful. Like all medicines, there is also the risk of anaphylaxis.

Leucovorin And Folic Acid/Vitamin B9

It is important to realise that Leucovorin is not folic acid or vitamin B9. Folic acid/vitamin B9 is a nutritional supplement available over the counter in all pharmacies, and is recommended for pregnant women or people with anaemia. The body absorbs the folic acid from the nutritional supplement in the usual way. Leucovorin is a prescription-only medicine which delivers folinic acid direct to the brain, which can only be used in specific and limited circumstances under medical supervision. Taking folic acid/vitamin B9 is not a substitute for Leucovorin, because they are in fact completely different substances working in different ways.

Social media influencers are already arguing that folic acid or vitamin B9 nutritional supplements will help to “cure” or “improve” the symptoms of autism. This is not true. While Leucovorin is claimed to improve the symptoms of autism, and Leucovorin contains folinic acid (which is related to folic acid), Leucovorin is not folic acid or vitamin B9. It cannot be claimed that folic acid or vitamin B9 can do what an entirely different medical product is claimed to do. However, advocates for vitamins (and their manufacturers and sellers) are nevertheless likely to try to increase their sales – and profits – by making false claims about vitamin B9/folic acid and autism.

President Trump’s Health & Human Services Secretary, Robert F Kennedy Jr, is a long-time advocate for food supplements and vitamins as an alternative to scientific medicines. He is also a significant past investor in companies that make these food supplements and vitamins. He has long argued that folic acid deficiencies are a cause of autism, along with vaccines. There is no evidence for these beliefs, and it is extraordinary that a person holding such bizarre beliefs should be appointed to a leading health position.

Guidance for Parents of Autistic Children

In the United States, some private autism treatment clinics have been testing autistic children for folic acid deficiencies (CFD or FRAA), and where the tests are positive, using Leucovorin to help resolve that deficiency. This tends to be in children presenting with a high level of impairment. Some improvement in speaking and moving has been seen, but also an increase in irritability and aggression. Leucovorin has not been shown to be a general treatment or cure for autism, but it has been a useful treatment for a very specific condition experienced by some autistic children.

President Trump was not correct to say that Leucovorin is a “gamechanger” in autism treatment, or that “it could help make our kids healthy again”. There is no evidence that Leucovorin can make a significant difference in autistic children. No large-scale trials of Leucovorin have taken place. The manufacturers of Leucovorin do not make this claim, and it is not currently licenced, even in the United States, for the treatment of autism. It remains to be seen whether the FDA (headed by people appointed by Trump) will change the licencing of Leucovorin in the future. However, even if this occurs in the US, it is almost certain not to happen in the UK, where the NHS does not currently even test for CFD or FRAA.

BuDS recommends that the parents of autistic children do not take any action at the moment. Unless you are wealthy, there is no testing for CFD or FRAA available in the UK, and it is extremely unlikely that any NHS consultant neurologist would prescribe Leucovorin except to treat these very specific conditions. Even if Leucovorin was a miracle cure for autism (which it is not), it would still take years for it to be properly tested and made available on the NHS.

We strongly counsel against trying to buy Leucovorin online or through private doctors. It is a powerful prescription medicine, and should only be used by qualified doctors in very specific circumstances. Your child could easily be harmed if you give it to them hoping it may improve their autism. The same applies to excessive doses of vitamin B9/folic acid. There is no harm about talking to your GP or pharmacist about a food supplement for your child, and taking their advice. But giving your child high doses of vitamin B9/folic acid (or any other vitamin) can be dangerous, and is not recommended.