The FDA recently granted NAP, the experimental drug, with orphan drug classification and pediatric rare disease designation to treat ADNP syndrome. The condition is a rare developmental abnormality that can cause various symptoms, including intellectual disability and an autistic spectrum disorder.
According to a new study, this experimental drug can also be used to treat symptoms of autism, intellectual disability, and Alzheimer's disease. This would be a considerable development for the Autism Spectrum Disorder Therapeutics Market. It would provide treatment for patients to overcome different types of symptoms and disabilities associated with the disease.
The present study provides evidence that NAP successfully treated a wide range of symptoms of ADNP syndrome. The disease is caused due to mutations within the ADNP gene, necessary for cerebral development and safeguarding cerebral brain cells. Various studies have linked ADNP syndrome to Alzheimer's disease and some types of mental disorders, developmental delays, and autism.
NAP constitutes a small portion of the normal ADNP protein. Researchers previously discovered that treating human nerve cells with ADNP syndrome with NAP improves their function in a laboratory test tube. They wanted to see if NAP could help with the syndrome by using a model with the most harmful mutation. This allowed them to see how the brain develops and help with behavioral issues.
The study used objective methods to investigate behavior and electrical activity and to identify select protein amounts in the brain in mice with ADNP conditions. They discovered that mice with ADNP syndrome had many adverse consequences. Such symptoms include higher neonatal death rates soon after birth, slower growth and abnormal locomotion, especially in females, and poor voice communication.
During cerebral examinations, additional results were discovered: A small number of synapses (the points of contact between nerve cells) were found in young mice. Further, the team discovered impaired electrophysiological activity indicating a low potential for normal cerebral arousal and Tau protein precipitates (aggregates) similar to those found in the brains of elderly Alzheimer's disease patients.
This cutting-edge technology will treat many symptoms and limitations associated with orphan diseases.