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Experimental Alzheimer’s drug may help children with autism, Israeli led team finds

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The international study, led by Tel Aviv University Prof. Illana Gozes of the Department of Human Molecular Genetics and Biochemistry, found that an experimental drug NAP was found to be effective in treating neurons in a model for ADNP syndrome


An international team of researchers led by Tel Aviv University (TAU) have found that an experimental Alzheimer’s drug could help children with autism.

In a recently published study led by TAU’s Prof. Illana Gozes of the Department of Human Molecular Genetics and Biochemistry, the team discovered Alzheimer’s-like characteristics in the brain tissue of a seven-year-old autistic child

The experimental drug for Alzheimer’s called NAP, developed by Gozes, was found to be effective in treating neurons in a model for ADNP syndrome. The treated damaged cells returned to normal function.

ADNP syndrome is defined as one of the 10 most common genetic syndromes found in those on autistic spectrum and is also characterized by mental impairment.

Gozes explained that the seven-year-old child, who died in Croatia, suffered from ADNP syndrome, which is a mutation that causes a deficiency in the ADNP protein. 

“This protein essential for brain development, and its characteristics can include severe developmental delay, mental disability and autism,” she said. 

Featured Image: An illustrative picture of a brain scan.
(Credit: SpeedKingz/Shutterstock)
Above Image: Prof. Illana Gozes of TAU’s Department of Human Molecular Genetics and Biochemistry. (Credit: Tel Aviv University)

When the team compared the brain tissue samples to that of a brain of a young person “who does not suffer from ADNP syndrome, we found deposits of the tau protein in the child, which is a known characteristic of Alzheimer’s disease.”

Following this discovery, the researchers then “treated” the damaged nerve cells from the child using Gozes experimental drug. 

“NAP, is actually a short section of normal ADNP protein,” Gozes said. “When we added the NAP to the neurons representing a mutation in ADNP, the tau protein started binding to the cells properly, and the cells returned to normal function.”

Gozes explained that the fact that “NAP treatment has been able to restore neuronal cell-like cells with impaired ADNP to normal functioning raises hopes that this substance may be used as a remedy for ADNP syndrome and its severe consequences, including autism.”

As part of the study, the team also aimed to broaden their understanding of the effects of the mutation that causes ADNP syndrome. 

To do this, they extracted the genetic material mRNA (RNA messenger) from the tissues of the deceased child, and analyzed 40 proteins of the deceased child encoded with mRNA. Full genetic sequencing was also performed to determine protein expression in white blood cells taken from three other children with ADNP syndrome. 

“On all the data obtained in the genetic sequences, an in-depth study was performed using advanced computational tools of bioinformatics,” the scientists said. “The data was then compared to an online database of proteins from healthy individuals, thus revealing a variety of characteristics common to sick children but very different from the normal occurrence of those proteins.”

Gozes stressed that because other genetic syndromes associated with autism are also related to tau protein in the brain, “we hope that those with these syndromes will also be able to benefit from NAP treatment in the future,” adding the team are planning to do a clinical trial in children with ADNP syndrome through the commercial company Coronis Neurosciences.

According to Gozes, “these findings mean that the mutation that causes ADNP syndrome impairs a wide range of essential proteins, many of which bind to, among other things, tau protein, and also impair its function.”

This, she said, creates various pathological effects in the brain (and other tissues) of children with ADNP syndrome. 

“The vast and in-depth knowledge we have accumulated through the present study opens the door to further, extensive and diverse research work. 

“We hope and believe that in the end we will reach the goal: drug development,” Gozes concluded.

The study was conducted in close collaboration with researchers from the Blavatnik School of Computer Science at Tel Aviv University, researchers from the Sheba Medical Center and a variety of research institutions across Europe: BIOCEV Institute of Biotechnology in the Czech Republic; Aristotle University of Thessaloniki, Greece, University of Antwerp Croatia The article was published in July 2020 in the journal Translational Psychiatry by Nature.

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