March 25, 2024
- A new article has explored how arginine vasopressin (AVP) could be a potential treatment for ASD and bipolar disorder. The authors discuss AVP’s role in the body and suggest that a lack of AVP in the brain might lead to social difficulties and fluid imbalances in some people with ASD. They also highlight that it is crucial to ensure these conditions come from a natural lack of AVP, not from medications affecting water intake or AVP levels. Additionally, the authors suggest that in the event of positive outcomes in AVP research, copeptin, a molecule synthesized alongside AVP, may serve as a precise diagnostic tool for identifying AVP deficiency in individuals with ASD.
- Jaguar Gene Therapy is pioneering the first human gene therapy trial aimed at tackling the genetic anomaly responsible for one type of autism spectrum disorder and Phelan-McDermid syndrome (PMS), an autism-related condition for which there are currently no treatments available. Their therapy, JAG201, targets the SHANK3 gene associated with autism and delivers a functional version of the gene via a one-time injection into the brain’s cerebral ventricles. Preclinical studies in rodents and primates have shown promising results, suggesting potential improvements in neurobehavioral, cognitive, and motor function abnormalities. With approximately 1% of ASD patients having SHANK3 mutations and the possibility of underestimation due to limited genetic testing, this therapy represents a critical advancement in addressing the unmet needs of this patient population.
- A national claims-based study from Taiwan revealed that individuals with ASD face a higher risk of all-cause mortality compared to their age- and sex-matched counterparts. This increased mortality rate persisted across different subgroups, including sex, age, and the presence or absence of intellectual disability (ID). Significant causes of death included cardiovascular, respiratory, accidents, and neurological disorders, with higher mortality rates observed in most categories for individuals on the spectrum compared to neurotypical controls, except for cancer. Individuals with autism and concurrent ID showed higher mortality risks in neurological, respiratory, and gastrointestinal categories and accidents, but their risk of suicide was lower. Women on the spectrum had higher mortality risks across most categories compared to men with autism.
- PaxMedica, Inc., a biopharmaceutical company specializing in neurological disorders, has unveiled its latest company presentation, emphasizing its strategic focus, recent strides in ASD treatments, and dedication to addressing the autism community’s needs. Their new presentation highlights PaxMedica’s pipeline, notably featuring their lead investigational drug, PAX-101 (also known as Suramin), designed to target ASD’s core symptoms. The Naviaux Lab at the University of California, San Diego, led by Robert K. Naviaux, MD, Ph.D., discovered the unique properties of suramin and made it the first in a new class of drugs that could be used to test the cell danger response (CDR) hypothesis for the origin and treatment of ASD. The laboratory demonstrated that suramin improved primary symptoms of ASD as well as metabolic and gastrointestinal irregularities associated with the disorder in both animal models and a limited human clinical trial.
