The Pitt Hopkins Research Foundation announces its first-ever human clinical trial to attempt to eradicate the severe gastrointestinal effects of Pitt Hopkins syndrome (PTHS), a rare disorder caused by a mutation of the TCF4 gene (18 chromosome) and often classified on the Autism spectrum. In partnership with Arizona State University's (ASU) Autism/Asperger's Research Program, a trial is scheduled to begin early 2020 focused on Microbiota Transfer Therapy (MTT), also known as a fecal microbiota transplant.
"This trial is a huge milestone for our young foundation," said Audrey Davidow Lapidus, president of the Pitt Hopkins Research Foundation. "Not only do we hope the trial will bring relief for our children, it signals the continued growth of the Pitt Hopkins Research Foundation as a serious player in the medical research field."
Subscribe to our e-Newsletters
Stay up to date with the latest news, articles, and events. Plus, get special offers
from American Pharmaceutical Review – all delivered right to your inbox! Sign up now!
The trial will be led by Prof. James Adams Ph.D, director of ASU's Autism/Asperger's Research Program. According to his research, there are "abnormal gut microbiota in individuals with Autism Spectrum Disorders (ASD), suggesting a link between gut microbiome and autism-like behaviors. Modifying the gut microbiome is a potential route to improve gastrointestinal (GI) symptoms in children with ASD, and fecal microbiota transplant could transform the dysbiotic gut microbiome toward a healthy one by delivering a large number of commensal microbes from a healthy donor."
Their research noted Autism symptoms were reduced by nearly 50% two years after fecal transplant in their participants, and consequently attracted the Pitt Hopkins Research Foundation's attention. They approached Prof. Adams to propose another trial, this time with participants living with PTHS in which 75% experience at least some GI discomfort, with many suffering from debilitating constipation and low motility.
"Our research team and I are honored to be working with the Pitt Hopkins Research Foundation, and we hope that our microbiota transplant therapy is able to improve the chronic constipation and other gut problems in children with PTHS," said Prof. Adams.