Dear Autism Community,

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.

 

Sincerely,

Prof. James B. Adams, PhD – autism researcher and autism dad

Prof. Rosa Krajmalnik-Brown, PhD – microbiome expert

Arizona State University

Profs. James Adams, Rosy Krajmalnik-Brown, and Dae Wook Kang

Quotes from families in our adult treatment study

"Our son's GI issues appear to be entirely gone.  Also, his autism symptoms have noticeably improved in the short time since the end of the treatment.  We expect even greater improvement in the future!  Sure wish we could have done this when he was diagnosed at age 3."

"The most immediate effect of the research and treatment is that my son can eat many more foods without the gastric problems such as gut pain, constipation and then diarrhea.  I have also noticed more communication and expressive language.  I believe over time there may be more improvement in his autistic behaviors."

"My adult son  is now able to have "normal" back and forth conversations with neighbors and extended friends and family. At Christmas he sat with us and chatted with everyone. He previously would have walked back out the door upon our arrival to go walking to relieve anxiety, and would essentially be gone during the entire visit. His transformation continues to be incredible at the 6 month mark! "