Treatment for autism symptoms gets patent for ASU researchers

February 1, 2022

A new treatment for autism, created by researchers at Arizona State University and their colleagues, has been granted a patent by the US Patent Office. The therapy, called Microbiota Transplant Therapy (MTT), aims to improve the chronic gastrointestinal symptoms often associated with the disorder.

Receiving patent approval for this promising treatment is also an important step toward developing a Food and Drug Administration-approved drug to treat core symptoms of autism. An early study of microbiota transplant therapy suggests it may be effective in treating both core autism symptoms and chronic gastrointestinal symptoms.

ASU Professor Rosa Krajmalnik-Brown and Assistant Professor Daewook Kang (now at the University of Toledo) in a 2017 ASU file photo. Krajmalnik-Brown, Kang and Professor James Adams are three of six co-inventors who have obtained a patent for their treatment of autism and associated symptoms.
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Rosa Krajmalnik-Brown, an ASU professor and director of the Biodesign Center for Health Through Microbiomes, and a pioneer in gut microbiome and autism research, is a co-inventor of the treatment.

“Our mission at the ASU Biodesign Center for Health Through Microbiomes is to improve human health by developing new methods to modify the microbiome. This is at the heart of our center, and it’s exciting that we have a patent to achieve this,” said Krajmalnik-Brown.

Autism now affects one in 44 children in the United States, often presenting with major problems with language, social interactions and behavior. Many people with autism have significant health issues, 30-50% of which have chronic gastrointestinal symptoms such as constipation, diarrhea and abdominal pain. These symptoms are difficult to treat and can persist for many years or even decades. Chronic pain caused by gastrointestinal distress can worsen other symptoms associated with autism, including irritability, attention deficits, behavioral problems and sleep disturbances.

“We are delighted with the approval of this patent. Our open-label pilot study and two-year follow-up study revealed major improvements in gastrointestinal and autism symptoms. Now we are conducting randomized clinical trials to fully assess the efficacy of MTT treatment,” said co-inventor James Adams. Adams is a professor at the School for Engineering of Matter, Transport and Energy, one of ASU’s seven Ira A. Fulton Schools of Engineering.

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james adams

In 2017, ASU researchers and their collaborators developed MTT to improve gastrointestinal symptoms. MTT involves pre-treatment with a special antibiotic to eliminate harmful bacteria, gut cleansing to remove remaining bacteria, and purified gut microbiota from carefully selected healthy human donors.

The treatment, similar to the fecal transplant commonly used to treat intestinal infections such as C. Difficile, involves 10 weeks of intense daily therapy. The approach is based on pioneering research by Dr Thomas Borody of the Center for Digestive Diseases in Australia, who used this method to successfully treat his autistic patients, and Professors Alex Khoruts and Michael Sadowsky of the University of Minnesota, who have developed the methods for producing purified microbiota.

In their 2017 study, Adams, Krajmalnik-Brown and their research team found that MTT reduced gastrointestinal symptoms by about 80% and initially reduced autism symptoms by about 25%. But, as an open study, they observed a placebo effect. A follow-up study of 18 participants two years after treatment ended found that most continued to see significant improvement in gastrointestinal symptoms. Additionally, an expert autism evaluator reported an almost 50% reduction in core autism symptoms.

Measurements of the participants’ microbiota at the Krajmalnik-Brown lab by Assistant Professor Daewook Kang (now at the University of Toledo) showed that although the children, aged 7 to 16 at the time of the study, initially had low diversity of gut bacteria, the diversity and presence of beneficial microbes increased and improved within two years of treatment. Based on these promising results, the FDA granted MTT “fast track” status in 2019, which means prompt review and increased assistance from the FDA.

Recent research suggests that our gut microbiomes affect brain communication and neurological health. Around the world, interest is growing in the idea that changes in the normal gut microbiota may be responsible for triggering various conditions. At ASU, a research team is exploring the use of the microbiome to treat symptoms of autism. Image by Shireen Dooling

Patent approval is also important because pharmaceutical companies can invest in conducting phase 2 and phase 3 clinical trials, which are necessary for FDA approval and release of the treatment to the public. The patent has been licensed to Finch Therapeutics, which expects clinical trials in mid-2022.

Meanwhile, the ASU team is pursuing its own Phase 2 clinical trials for adults with autism (the last participants started treatment) and children with autism (half the participants started treatment). These studies will be important to ultimately gaining FDA approval.

Patent information, as well as a complete list of contributors, is available on the US Patent Office website (US patent number 11202808).

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