New Treatment Option for Certain Children with Autism
New Treatment Option for Certain Children with Autism
Exciting advances in autism research are opening new doors for families. The FDA has recently updated the labeling for leucovorin—a form of vitamin B9—to include treatment of cerebral folate deficiency in children with autism spectrum disorder. This development offers hope for improved speech, language, and developmental outcomes in eligible children.
Understanding Cerebral Folate Deficiency
Cerebral folate deficiency is a condition where folate (vitamin B9) levels in the brain are low, even when blood folate levels are normal. The brain needs folate for proper development and function, particularly for making neurotransmitters that help brain cells communicate.
This condition can lead to: delays in speech and language development, difficulty with verbal communication, motor skill challenges, cognitive delays, and behavioral difficulties. Importantly, cerebral folate deficiency affects only some children with autism—not all. This is why proper testing and diagnosis are essential before considering this treatment.
The Connection to Autism
Research shows that some children with autism spectrum disorder have cerebral folate deficiency. This represents a treatable biological factor that may contribute to autism symptoms in certain children. Studies suggest that 12-20% of children with autism may have cerebral folate deficiency. When these children receive appropriate treatment, many show improvements in language skills, communication abilities, and overall development.
How Leucovorin Treatment Works
Leucovorin is a special form of folate that can cross from the blood into the brain more easily than regular folic acid supplements. When given to children with confirmed cerebral folate deficiency, leucovorin helps restore adequate folate levels in the brain by: providing the brain with the specific form of folate it needs, supporting neurotransmitter production, promoting healthy brain cell function and communication, and potentially improving developmental progress.
Research shows that leucovorin treatment may help improve verbal communication and language skills in eligible children, particularly when started early. Some children show progress within weeks to months of starting treatment, though individual responses vary.
Who Might Benefit from This Treatment
Leucovorin treatment is specifically designed for children who have both autism and confirmed cerebral folate deficiency. Your child might be a candidate for testing if they have: autism spectrum disorder diagnosis, significant speech and language delays, limited verbal communication, history of regression in language or motor skills, or family history of autoimmune conditions.
Testing and Diagnosis
Diagnosis involves specialized testing including blood work and lumbar puncture to measure folate levels in cerebrospinal fluid, testing for folate receptor autoantibodies, and comprehensive developmental assessment. Our experienced pediatric team can guide you through the evaluation process.
What to Expect with Treatment
If cerebral folate deficiency is confirmed and treatment is recommended, leucovorin is typically given as a daily oral medication. Treatment may need to continue for many months to years. Regular monitoring and follow-up are essential to assess response and adjust treatment as needed.
Important Considerations
This treatment is not appropriate for all children with autism. It is specifically for those with confirmed cerebral folate deficiency. Work closely with your pediatrician and specialists to determine if testing and potential treatment are right for your child.
Frequently Asked Questions
What is the new treatment option for autism?
Recent advances include FDA-approved medications for specific autism-related symptoms, behavioral therapies like Applied Behavior Analysis (ABA), and emerging treatments targeting specific genetic markers. Your pediatrician can discuss which options may be appropriate based on your child's specific needs and symptoms.
At what age can autism be diagnosed?
Autism can be reliably diagnosed as early as 18-24 months, though some signs may be apparent even earlier. Early diagnosis and intervention lead to better outcomes, so it's important to discuss any developmental concerns with your pediatrician during well-child visits.
What are the early signs of autism in toddlers?
Early signs include limited eye contact, delayed speech, repetitive behaviors, difficulty with changes in routine, intense focus on specific objects or topics, and challenges with social interaction. However, every child is different, and only a qualified professional can make a diagnosis.
Will my insurance cover autism treatment?
Many insurance plans cover autism-related therapies, including behavioral therapy, speech therapy, and occupational therapy. Coverage varies by state and plan, so check with your insurance provider about specific benefits and required authorizations.
How effective are behavioral therapies for autism?
Research shows that early, intensive behavioral interventions like ABA therapy can significantly improve communication, social skills, and adaptive behaviors in children with autism. The most effective programs are individualized, start early, and involve family participation.
Can children with autism attend regular schools?
Many children with autism can attend mainstream schools with appropriate support services. Schools must provide an Individualized Education Program (IEP) or 504 plan to ensure your child receives necessary accommodations and services to succeed academically and socially.
Need Personal Guidance?
This article provides general information. For questions specific to your child's health, please call our office or book an appointment online.