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Can Food be Medicine?

January 28, 2020
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As we all know gut issues are common in Rett Syndrome and are often difficult to manage. Furthermore, evidence is emerging that gut health in general can contribute to symptoms in various diseases. To try to better understand the role of gut health in Rett and provide dietary interventions that may be helpful we are partnering with Beth McCormick’s team at University of Massachusetts Medical School (UMass) on an exciting new study called, Rett Syndrome - Microbiome Study for the Advancement of Novel Nutritional Supplements that will be starting later this spring. This 2-part study will help define the gut microbiome of Rett Syndrome individuals and potentially improve gut health and wellbeing with a dietary supplementation.

The gut microbiota, defined by their genetic material (the microbiome), maintain the proper environment in the GI tract, and play a critical role in establishing the gut-brain axis (GBA). The GBA is a complex communication system between the gut and the brain that has multiple effects on cognitive function and behavior. Evidence of the GBA arose more than 20 years ago from the observation that antibiotic treatment dramatically improved cognitive function in patients with hepatic encephalopathy (impaired brain function as a result of liver dysfunction) compared to those who received a non-antibiotic treatment.

Recent research suggests that gut microbiota influence symptoms in a wide range of disorders. For example, autistic patients as well as mouse models of autism exhibit microbiota alterations. Autistic-like behaviors in mice can be induced and rescued by modifying gut microbiota. Though research in humans with autism is still preliminary, improvements in symptoms have been observed after antibiotic treatment, fecal transplant, and dietary changes. Could something similar happen in Rett? These data suggest we may be able to influence symptoms in Rett syndrome as well with something as simple as a supplement or other dietary change.

The goal of our study is to comprehensively characterize the gut microbiota in Rett and identify which supplements are best suited to improve microbial health in a targeted manner. There are no clinic visits and this study is open to girls 6 years of age and older with a documented MECP2 mutation considered causative for Rett Syndrome. We are also looking for healthy siblings to participate as well. During the study, weekly stool samples will be collected (due to this aspect of the study we are accepting participants only from the USA to avoid potential issues or delays with customs), and a food diary and bowel movement diary will be kept. Quality of health and Rett symptom assessments will be reported weekly by the caregiver to a certified nutritionist who will also be available for nutritional questions and/or concerns.

We’re excited to be leading this important study with UMass with the hope that it will enable the identification of the Rett microbiome profile and allow for the development of novel dietary supplementation to improve gut health as well as other symptoms of Rett Syndrome.

If you’re interested in participating in this study or would simply like to learn more please fill out the form below or email Sharon Lynch. As we prepare to launch the study we will also keep the community updated via our website and clinicaltrials.gov.

$40M