An Experimental Drug for Alzheimer’s is Being Tested to Treat Autism

Professor Illana Gozes and her team at Tel Aviv University recently developed NAP (CP201). It is an experimental drug that has been proven to be effective in the treatment of ADNP syndrome.

According to an article in the journal Israel21, over twenty years ago Dr. Gozes discovered the neuroprotective protein ADPN that is essential for brain development. Notably, she discovered that ADPN is associated with autism.

Dr. Gozes also associated ADPN with schizophrenia and Alzheimer’s.

Professor Gozes is a highly regarded neuroscientist with expertise in tauopathy and its critical role in Alzheimer’s disease. The doctor and her team were joined in their efforts by major research institutions across Europe.

Last year, Israel21 announced Dr. Gozes’ groundbreaking findings of ADNP protein accumulations in the brains of patients with Alzheimer’s.

About ADNP Syndrome

The most common symptoms of ADNP syndrome (activity-dependent neuroprotein) are impaired social interaction, developmental delays, speech impediments, motor dysfunctions, communication (autism spectrum disorder), and intellectual disability.

The ADNP protein was found to be the leading cause of a mutated gene resulting in the ADNP syndrome. It was after discovering the ADNP protein that the team realized that autism can be determined by genetics. That if at birth the child has one mutation in a critical gene, that child will develop autism.

About the NAP (CP201) Discovery

NAP is an experimental drug that was invented at the University of Tel Aviv to treat Alzheimer’s disease. Dr. Gozes explained that NAP is actually a fragment of the ADNP protein.

NAP was tested on nerve cells replicating the ADNP syndrome and the mutation-induced Alzheimer’s symptoms. The NAP-induced ADNP protein then bound to the cells causing the damaged cells to return to normal function.

The experiment was a success. NAP protected against ADNP mutations.

Dr. Gozes pointed out that some autism-related disorders show similar symptoms in the brain. Therefore, the researchers are optimistic that patients with these related disorders will benefit from the NAP treatment.

Postmortem Examination

Dr. Gozes’ study examined tissues from a seven-year-old Croatian boy’s postmortem brain. The child had been diagnosed as having ADNP syndrome associated with autism.

The team examined the brain of this young child and found deposits of tau in his brain tissues. The child’s brain was compared to that of a thirty-one-year-old adult who did not have preexisting conditions.

Dr. Gozes and the researchers realized that ADNP is an important protein as it prevents electrical blockades, significantly reduces motor disability, and offers significant neuroprotection.

These results were published in the journal Translational Psychiatry.

Orphan Drug Status

The FDA granted NAP an “orphan drug” status qualifying its sponsor for incentives that include tax credits for qualified clinical tests and also development incentives.

Dr. Gozes is the chief scientific officer at Israel’s Coronis Neurosciences, a specialty pharmaceutical company. The company is currently sponsoring an early phase clinical trial for children who have been diagnosed with ADNP.

For years researchers have concentrated on the Tau and amyloid deposits in Alzheimer’s patients’ brains. Dr. Gozes points to their new study which moves away from that theory. They are focusing on regulatory proteins and their mutations in Tau that damage neurons’ structure.

Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia four years ago. He was treated with a methylating agent While he was being treated with a hypomethylating agent, Rose researched investigational drugs being developed to treat relapsed/refractory AML.

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