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A lesson on leucovorin

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Two pediatricians, a child neurologist and a child psychiatrist walk into the ASF weekly science podcast to discuss the safety, efficacy and appropriateness of leucovorin, the drug that the HHS is fast tracking through the FDA approval process. Does it work? Is it safe? What should I do or know when I talk to my doctor?

Here is a link to the statement by the Society of Developmental and Behavioral Pediatrics: https://sdbp.org/sdbp-statements-regarding-leucovorin-tylenol-and-autism/

Here are the four studies mentioned:

Here is a requested correction to one of the papers where a calculation error was made:

https://pubpeer.com/publications/987569A781B9A602DCE7358D4513A0

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Genetic therapies in store for neurodevelopmental disorders

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Gene therapies have been in the news lately. They are being used to help individuals who have a genetic variant linked to a disorder or disease, including but not limited to: spinal muscular atrophy, carbamoyl phosphate synthetase 1 (CPS1) deficiency, diabetes and some types of cancers. What is the promise in rare genetic forms of neurodevelopmental disorders and autism? This week, scientists from Jaguar Gene Therapy discuss their ongoing studies in Phelan-McDermid Syndrome and how gene therapies hold promise for treating neurological impairments caused by a known genetic variant. The interview provides basic information of what a gene therapy is, how it works, how it is used and what is monitored during these treatments.

https://www.nih.gov/news-events/news-releases/infant-rare-incurable-disease-first-successfully-receive-personalized-gene-therapy-treatment

https://jaguargenetherapy.com

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Do Transcranial Magnetic Stimulation and Direct Current Stimulation help people with autism? The latest science here.

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Two therapies that are meant to alter brainwave activity, called Transcranial Magnetic Stimulation and Transcranial Direct Current Stimulation are receiving a lot of attention for potential efficacy in treating autism. They are non-invasive, which means treatment is provided on the scalp. While results vary, the overall evidence does not support these two interventions in helping to treat core autism features. However, as TMS is approved for depression and OCD, people should ask their doctors about these potential treatments if they suffer from these conditions. Learn more in the articles below:

https://link.springer.com/article/10.1007/s00787-024-02635-z

Click to access nihms-1934887.pdf

https://academic.oup.com/cercor/article-abstract/34/13/8/7661139?redirectedFrom=fulltext&login=false

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Are Naturalistic Developmental Behavioral Interventions controversial?

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While NDBIs are generally considered beneficial, they still face controversies – do they actually work and does that translate to an improved quality of life for the family? This week’s #ASF podcast interviews Molly Reilly and Jinwei Song of @UConn to dive into these issues, as well as the role of the caregiver in the intervention and how their influence affects the outcome. References below.

https://pubmed.ncbi.nlm.nih.gov/38719439

https://journals.sagepub.com/doi/epub/10.1177/13623613241227516

https://link.springer.com/article/10.1007/s10803-023-06198-x

Websites: PRT, ESDM, JASPER, EMT, SocialABCs, and Pathways
Books/Manuals: ESDM, PRT, JASPER, EMT

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Can we solve the pandemic problems around diagnosis and intervention?

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This week’s #ASFpodcast highlights a few articles from the Journal of Autism and Developmental Disorders this week which examined the tolerability and efficacy of online diagnostic procedures and interventions, from the perspective of both parents and clinicians. They seem to work about the same, although there were some caveats. For many reasons, online and telehealth options are here to stay, and more needs to be done to improve their accuracy, acceptability, feasibility and effectiveness. These early studies are promising though, and lead the way to even more improvements to help make them a viable option for families in the future.

https://link.springer.com/article/10.1007/s10803-022-05435-z

https://link.springer.com/article/10.1007/s10803-022-05576-1

https://link.springer.com/article/10.1007/s10803-022-05554-7

https://link.springer.com/article/10.1007/s10803-022-05580-5https://link.springer.com/article/10.1007/s10803-022-05607-x

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ABA studies in ASD are not perfect

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ABA and ABA – inspired behavioral therapies like Naturalistic Developmental Behavioral Interventions are the most commonly used in autism. They have become more sophisticated in design, using control groups and implementing other ways to minimize bias. But when you put all of the data together around ABA-type interventions in a pile, are they effective? Do they work? It turns out, just like everything, these studies are not perfect. But even using the strictest of criteria, the results are positive for the support of social communication skills. Of course, there are nuances and details, you can hear about them in this podcast.

https://pubmed.ncbi.nlm.nih.gov/31763860/

https://pubmed.ncbi.nlm.nih.gov/33482692/

THE FACTS BEHIND APPLIED BEHAVIOR ANALYSIS
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Managing autism practice from a distance: it’s called ECHO

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Extension for Community Healthcare Outcomes (ECHO) pairs specialist centers with community providers to help them manage cases and empower them with knowledge to help their patients locally. It is done over the computer, which is especially relevant today. The goal is to help clinicians and physicians manage patients when local expertise is not available. Micah Mazurek of University of Virginia recently published a randomized study about the efficacy of this program, and is a special guest on the podcast to explain what it is and how it can be used to help doctors help each other and their families.

https://jamanetwork.com/journals/jamapediatrics/article-abstract/2762007