Approaching the clinic: nutritional support for patients with Angelman Syndrome

Quick Overview

In this presentation from the 2017 FAST Science Summit, Mike McCandless, Jessica Duis, and Donna Herber discuss their work on developing a ketone ester supplement for patients with Angelman Syndrome. They explain that the supplement is designed to provide nutritional support and make dietary interventions easier for patients. The team discusses the background and basic science behind the supplement, as well as the upcoming clinical trial at Vanderbilt University. They emphasize that the supplement is not a drug, but a food-based intervention, and they hope to make it available to patients as soon as possible.

Introduction

This talk discusses the work of Mike McCandless, Jessica Duis, and Donna Herber from Disruptive Nutrition. The team is focused on developing a ketone ester supplement for children with Angelman Syndrome. The talk provides background information on the company and its research, as well as details about the clinical trial they are conducting at Vanderbilt University.

Background

In 2011, Mike McCandless’s mother was diagnosed with cancer, which led him to research the potential benefits of a ketogenic diet. He connected with researcher Dominic D’Agostino and funded research in his lab, which yielded promising results in animal models. McCandless then brought on Donna Herber, a scientist, to help navigate the medical research and determine the best path forward. They decided to focus on Angelman Syndrome and began presenting their findings at conferences.

The Clinical Trial

The team at Disruptive Nutrition is conducting a clinical trial at Vanderbilt University to study the safety and tolerability of their ketone ester supplement in children with Angelman Syndrome. The trial will last for 16 weeks and will involve clinical monitoring both at home and in the clinic. Participants will be randomized to receive either the supplement or a placebo, and the study will evaluate the effects on seizures, cognition, and mobility.

Benefits and Next Steps

The team believes that their supplement can make the ketogenic diet more manageable for children with Angelman Syndrome. They hope to provide a convenient and effective nutritional support option that can improve seizure control and potentially have other positive effects on behavior and development. Once the trial is complete and the supplement is deemed safe and well-tolerated, it will be made available to the public.

Conclusion

The work of Disruptive Nutrition aims to provide nutritional support for children with Angelman Syndrome through a ketone ester supplement. The team is conducting a clinical trial to evaluate the safety and tolerability of the supplement and hopes to make it available to the public in the future. Their goal is to improve the quality of life for children with Angelman Syndrome and make the ketogenic diet more accessible and manageable for families.