Is Leucovorin a New Treatment for Autism?

 

Recently, the FDA announced that it has begun the approval process for a medication called leucovorin for a condition known as Cerebral Folate Deficiency (CFD).

Because CFD can include symptoms that look like autism, many families are asking:

“Is this a new treatment for autism?”

Let’s walk through this carefully and clearly.

What Is Leucovorin?

Leucovorin (also called folinic acid) is a prescription medication.

It is a special, active form of folate — a vitamin that helps the brain grow and function properly.

It has been used safely for many years for:

• Supporting certain cancer treatments
• Treating specific types of anemia
• Preventing medication side effects

It is not a supplement. It is a prescription medication.

What Is Cerebral Folate Deficiency (CFD)?

Some children have difficulty transporting folate into the brain. 

Even if blood folate levels are normal, the brain may not receive enough. This is called Cerebral Folate Deficiency (CFD).

Children with CFD may have:

• Severe speech delay
• Developmental delay
• Loss of previously learned words (regression)
• Seizures
• Movement coordination problems
• Autism-like features

Leucovorin may help bypass this transport issue.

Does This Mean My Child Must Have CFD to Benefit?

Not necessarily — but this is important.

Research suggests that children who benefit most often:

• Have significant language delay
• Lost words during early development
• Have certain folate-related antibodies
• Have seizures or autoimmune features

Some studies show that children without confirmed CFD may still improve — but the response is less predictable.

Leucovorin is not helpful for every child with autism.

It is most likely to help a specific subgroup.

Do We Need Lab Testing First?

There are two approaches:

1. Testing First

Some providers order blood tests to look for folate receptor antibodies.
If positive, this strengthens the case for treatment.

2. Monitored Trial

In some situations, if testing is not available or affordable, a carefully monitored medication trial may be considered.

More advanced testing (like spinal fluid testing) is usually reserved for complex neurological cases and is not routine.

The decision depends on:

• Your child’s developmental history
• Severity of symptoms
• Medical history
• Family history
  
  

What Kind of Assessment Is Needed?

Before considering any medication, it is critical to understand:

• Does your child truly meet criteria for autism?
• Are there overlapping conditions (ADHD, anxiety, language disorder, learning disorder)?
• Is there a regression history?
• Are there seizure concerns?
• What is the pattern of strengths and weaknesses?

At MindWeal, we use a comprehensive psychiatric evaluation that:

• Reviews developmental history in detail
• Examines language profile
• Assesses attention, behavior, and emotional symptoms
• Identifies possible medical or metabolic red flags
• Clarifies diagnosis

This helps determine whether your child fits the subgroup that may benefit — rather than using a one-size-fits-all approach.

Medication decisions should always follow a clear diagnostic understanding.

What Does the Research Say?

So far, there have been:

• 2 high-quality clinical trials showing improvement in language
• Several smaller supportive studies

These studies suggest leucovorin may improve verbal communication in some children with autism who have specific biological markers.

However:

• It does not cure autism
• It does not improve all autism symptoms
• It has not been approved specifically for autism

The research is promising but still developing.

Is It Safe?

Leucovorin has been used safely for decades.

Most children tolerate it well.

Possible side effects may include:

• Increased activity
• Irritability
• Trouble sleeping

If prescribed, close monitoring is essential.

The Bottom Line

Leucovorin is:

• A prescription medication
• Supported by early but promising research
• Most helpful for a specific subgroup
• Not a cure for autism
• Not necessary for every child

If you have questions about whether this may apply to your child, your provider can help guide you through the science and decide thoughtfully.