May 23, 2023 – A new study led by Prof. Haitham Amal and his team at the Hebrew University of Jerusalem (HU), discovered a direct connection between Nitric Oxide (NO) levels in the brain and autism spectrum disorder (ASD).

The new Hebrew University study was featured in the prestigious journal, Advanced Science. It was generously funded by AFHU donors the Satell Family Foundation and the Neubauer Family Foundation, as well as grants from the Eagles Autism Foundation, the U.S. Department of Defense (DoD), the Israeli Science Foundation (ISF), the National Institute of Psychobiology in Israel (NIPI), the Israeli Council for Higher Education, and the Berettler Centre for Research in Molecular Pharmacology and Therapeutics.

NO is a multifunctional signaling molecule and a neurotransmitter that plays an important role in physiological and pathophysiological processes.

The study demonstrated that ASD indicators increase as NO increases in the brain. Conversely, in cases where levels of NO in the brains of murine models of autism were lowered in a proactive and controlled manner, autism indicators and behavior decreased accordingly.

Millions of people worldwide are diagnosed with ASD annually. According to the U.S. Center for Disease Control and Prevention, autism is the most common developmental disorder in the U.S. with one in 36 children on the spectrum. In Israel, more than 30,000 children up to the age of 18 have been diagnosed with ASD.

“Our research showed – in an extraordinary way – that inhibiting the production of NO, specifically in brain neuron cells in biological models for autism, causes a decrease in autistic symptoms,” says Prof. Amal, head of the HU Amal Lab, professor at the School of Pharmacy, Institute for Drug Research, Faculty of Medicine. “By inhibiting the production of NO in laboratory models, they became more social, and less repetitiveness was observed in their behavior. Additionally, the models showed interest in new objects and were less anxious. Lastly, the decrease in NO levels led to a significant improvement in neuronal indices.”

Photo courtesy of Dr. Haitham Amal

In addition to several ASD biological models, the study results include tests conducted using human stem cells and clinical blood samples from children with low-functioning autism.

“This research is a significant breakthrough in autism research with the first direct connection made between an increase in the concentration of NO in the brain and autistic behavior, Prof. Amal says. “This discovery can have implications on the relationship of NO with other neurological diseases, such as Alzheimer’s, or psychiatric diseases including schizophrenia and bipolar disorder. I am hopeful that with our new understanding of the NO mechanism, we can begin to develop therapeutic drugs and help millions of children and adults living with autism around the world.”

The Amal Lab members that led this study were Dr. Manish Tripathi, Shashank Ojha, Maryam Kartawy, and Wajeha Hamoudi.

Recently, Amal co-founded and is Chief Scientific Officer at Point6 Bio Ltd., a Hebrew University portfolio company. He is also partnering with a NASDAQ-traded U.S. pharmaceutical company to research and develop ASD drugs. Prof. Amal was recently awarded a $400,000 Eagles Autism Foundation Grant, the first researcher outside the U.S. to receive this prestigious grant.