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Why is the study needed?
The FDA classifies fecal microbiota from healthy humans as a biological drug (like human blood), and it is not yet approved by the FDA for general use. The FDA currently only allows FMT for patients with recurrent C. Difficile infections (which causes life-threatening diarrhea and kills 29,000 Americans per year). The FDA only allows FMT for people with autism as part of a research study like ours.
We hope that this Phase 2 study will successfully improve both autism and GI symptoms in the participants, and also help bring us closer to drug approval.
What are Phase 1, 2, 3 trials?
The FDA classifies MTT as a drug, and drugs need Phase 1, 2, and 3 trials to achieve drug approval in order to be available as a therapy.
We completed a Phase 1 trial, which is primarily for safety and tolerability, and found that MTT is generally safe and well-tolerated. It appeared to result in great long-term improvements in both gastrointestinal symptoms and autism symptoms, but it was an “open-label” study (everyone was treated), so there was some placebo effect.
We are currently conducting a Phase 2 trial, which is a randomized, double-blind, placebo-controlled study, which is designed to prove effectiveness. The placebo group will eventually be switched to the real treatment.
If this study is positive, we hope it will lead to a larger Phase 3 trial, and eventual approval by the FDA.
Why is this study expensive? The vancomycin costs approximately $500/patient, and the microbiota costs about $2500/patient. Although this may seem high, the treatment seems to be very effective and long lasting.
How will my donation be used? Your donation will go directly to support the costs of this study, in an account overseen by Profs. Adams and Krajmalnik-Brown. Arizona State University is waiving the normal 50% overhead it charges on research grants since these funds are classified as gifts.
What if I want to make a major donation? The ASU Foundation staff and Profs. Adams and Krajmalnik-Brown are willing to answer questions from any major donors ($1,000+)
Original Phase 1 Study: Read Here
Two-year follow-up for Phase 1 study: Read Here
Link to press release: Read Here
Information on how to apply for Phase Two Study for Adults: We currently have openings in the adult study, and hope to treat more patients when we raise additional funds. The application for the study is at https://autism.asu.edu/research-studies We hope to enroll more patients in another 6 months or so, if we are successful with our fundraising.
Information on another Child study: We hope to conduct a new Phase 2 study for children with ASD as soon as we have funding for it. Link coming soon.
Again, many thanks for considering to support these important studies to help children and adults with autism.
Prof. James B. Adams, PhD – autism researcher and autism dad
Prof. Rosa Krajmalnik-Brown, PhD – microbiome expert
Arizona State University