Study led by Israeli researchers could help patients with autism, alzheimer’s

Youths and maidens alike, old and young together. 

Psalms

148:

12

(the israel bible)

December 8, 2021

3 min read

Drug repurposing involves medications that have already been approved by the US Food and Drug Authority (FDA) and other regulatory bodies or received permission for being an experimental drug for a certain condition and using them for very different medical conditions. 

 

Aside from the fact that developing a new drug costs a huge amount of money, finding existing medications that can have a beneficial effect on different diseases speeds up the time that patients must wait to get help. 

 

Tel Aviv University (TAU) researchers developed a novel lab model to assess the effect of an experimental drug on symptoms related to autism and intellectual abilities in young people and determine if it also benefits elderly patients with Alzheimer’s disease. 

 

They found that a short protein segment called NAP remedies a broad spectrum of symptoms including slow development, poor communication and brains with a relatively small number of synapses, low electrical activity, and protein aggregate similar to those found in Alzheimer’s patients.

 

An extensive international study led by TAU scientists found that an experimental drug that has been awarded by the FDA with “orphan drug” status (because only a relatively small number of patients suffer from it) for future treatment of a rare development disorder can also treat a variety of symptoms relating to autism, intellectual disability and Alzheimer’s disease. 

 

NAP was discovered by MD/PhD student Dr. Gideon Carmon’s in the lab of Prof. Illana Gozes of the department of human molecular genetics and biochemistry at TAU’s Sackler Medical School. The FDA granted the experimental drug with orphan drug designation and pediatric rare disease designation for treatment of a rare developmental disorder called ADNP syndrome, which can cause a variety of symptoms. Among them, the hallmark features are intellectual disability and autism spectrum disorder.

 

Their article was published in the prestigious journal Biological Psychiatry under the title “Novel ADNP Syndrome Mice Reveal Dramatic Sex-Specific Peripheral Gene Expression With Brain Synaptic and Tau Pathologies.” 

 

In the current study, a team of researchers led by Gozes developed an innovative mouse model and found that NAP can be effective in treating a broad spectrum of symptoms of ADNP syndrome, which is caused by mutations in the ADNP gene essential for brain development and protecting cerebral brain cells. 

 

ADNP syndrome causes a wide variety of signs and symptoms. Its hallmark features are intellectual disability and autism spectrum disorder, which is characterized by impaired communication and social interaction. Previous studies showed that ADNP syndrome is related to Alzheimer’s disease and certain types of mental disabilities. 

 

Ramot, TAU’s technology commerce company, has filed a number of patent applications to protect the technology and its implementation and, in collaboration with Gozes, is raising funds to finance further clinical research. Ramot is also in discussions regarding commercial collaboration with pharmaceutical companies. “We’re excited by this new discovery and believe that this is groundbreaking technology that will remedy a variety of symptoms and disabilities in a broad spectrum of orphan diseases,” said Prof. Keren Primor Cohen, the CEO of Ramot.

 

Gozes explained that “NAP, in fact, comprises a short segment of the normal ADNP protein. We previously found that treatment using NAP corrects the function of human nerve cells afflicted with ADNP syndrome in a laboratory test-tube. In this study, we sought to examine the efficacy of NAP in treating various aspects of the syndrome using a model with the most harmful mutation, which allowed us to view brain development and facilitate remedying of behavioral problems.”

 

The study, which examined a model using mice with ADNP syndrome, used objective methods to analyze behavior, electrical activity and to further identify select protein contents in the brain. The researchers found that the mice suffering from ADNP syndrome showed a broad spectrum of pathological outcomes, including increased rates of neonatal death immediately after being born, slowed development and irregular walking, mostly among females, as well as poor voice communication. 

 

Cerebral examinations showed a small number of synapses – the points of contact tween nerve cells – impaired electrophysiological activity demonstrating a low potential for normal cerebral arousal, as well as precipitates (aggregates) of the Tau protein in young mice, similar to those in the brains of elderly Alzheimer’s disease patients.

 

For most of these symptoms, the researchers examined the effect of the future medicinal substance NAP – made of a short and normal segment of the ADNP protein, the same protein that is impaired because of the mutation. “In the past,” said Gozes, “we found that NAP corrects impaired functioning of ADNP that has mutated in the nerve cell model in the culture. We now examined its effect in animals modeling the ADNP mutation. To our amazement and joy, we discovered that treatment using NAP normalizes the functioning of these mice for most of the symptoms.” 

 

Gozes concluded that “this study is an important milestone on the way to developing drugs that will help children with autism stemming from genetic mutations, as well as Alzheimer’s patients.”

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