Arizona State University – Autism/Aspergers Research Program
Abstract:
This presentation will cover the results of a major Phase 2 clinical trial of Microbiota Transplant Therapy (MTT) for adults on the autism spectrum. This was a randomized, double-blind, placebo-controlled study for Part 1, and then in Part 2 the placebo group received a similar treatment, and the original treatment group received an extra 8 weeks of microbiota transplant. We found that MTT was generally safe and well-tolerated, and microbiota capsules were better tolerated than placebo capsules. For the primary outcome (autism symptoms), the treatment group improved more than the placebo group (medium effect). For the secondary outcome of daily stool record, the treatment group improved more than the placebo group (medium effect). The microbiome of the treatment group became more similar to the donor, with increases in certain beneficial bacteria. Overall, we believe these positive results demonstrate that MTT is generally safe and effective for treating autism and GI symptoms in adults with autism, and provide important insight into how to improve dosing to potentially improve clinical benefit.
MTT for Pitt Hopkins: We will also briefly summarize the positive results of our Phase 2 trial of MTT for children with Pitt Hopkins Syndrome, a single-gene disorder involving severe intellectual disability, severe autism and severe constipation. MTT was generally safe and well-tolerated, and resulted in substantial improvement in 6 of 6 children in that study. These results have also been submitted to the FDA for review.
Gut-Brain Axis Therapeutics Inc.: We will briefly explain the new company we founded, Gut-Brain Axis Therapeutics Inc, which we hope will be able to conduct the final studies needed to achieve approval of MTT for children and adults with autism and Pitt Hopkins.
Acknowledgements: Many, many thanks to the families who participated in these studies, and many thanks for financial support from the federal government, Finch Therapeutics, Fearfully/Wonderfully Made, BHARE Foundation, Zoowalk for Autism Research, and donations from 1000+ families – this was a very expensive study, and the support from everyone was essential for our success. We also thank the Pitt Hopkins Research Foundation for their support of our study of MTT for Pitt Hopkins.
Location: Zoom webinar (free)
Dates/Times: These are new dates/times/links.
We are going to offer identical webinars on three different dates. We will also record the webinar and make it available on our website, http://autism.asu.edu.
May 30 7:30 pm EST/ 4:30 pm PST
register at: https://asu.zoom.us/webinar/register/WN_9wdqGcPYS6-QxX7FbP1zlQ
June 6 9:00 pm EST/ 6:00 pm PST
register at: https://asu.zoom.us/webinar/register/WN_2R0PqxN_T3qgXHzAAL_q4g
June 9 2:00 pm EST/ 11:00 am PST
register at: https://asu.zoom.us/webinar/register/WN_Pd4fBT0vS_Oex3e0l6EopA
The presentations will last about 40 minutes, with about 20 minutes for Q/A. We will post a Frequently Asked Questions after the webinars on our website, http://autism.asu.edu