In a new study, researchers found deposits of the tau protein typically found in Alzheimer’s patients in tissues taken from the postmortem brain of a 7-year-old autistic child.
The child suffered from the ADNP syndrome, a mutation that causes a deficiency/malfunctioning of the ADNP protein essential for brain development.
In light of these findings, the researchers tested an experimental drug called NAP, originally developed for Alzheimer’s disease, on nerve cells in a model of ADNP syndrome with the mutation that induced Alzheimer’s-like symptoms.
The experiment was a success, with the damaged nerve-like cells returning to normal function.
The research was conducted by a team at Tel Aviv University and elsewhere.
The team explains that the current study is based on tissues taken from the brain of a 7-year-old boy with ADNP syndrome who died in Croatia.
When they compared the postmortem ADNP syndrome brain tissues to tissue from the brain of a young person without ADNP syndrome, they found deposits of the tau protein in the ADNP child, a pathology that characterizes Alzheimer’s disease.
The researchers then “treated” damaged nerve-like cells carrying an ADNP mutation similar to the deceased child’s mutation with a drug candidate called NAP.
NAP was originally intended to be used to help treat Alzheimer’s disease.
It is actually a short active fragment of the normal ADNP protein. When the team added NAP to the nerve cells carrying an ADNP mutation, the tau protein bound to the nerve cell skeleton properly, and the cells returned to normal function.
The fact that NAP treatment has been successful in restoring the normal function of neuronal-like cell models with impaired ADNP raises hopes that it may be used as a remedy for ADNP syndrome and its severe implications, including autism.
Moreover, because other genetic disorders related to autism are characterized by tau pathologies in the brain, the researchers hope that those suffering from these syndromes will also be able to benefit from NAP treatment in the future.
NAP (also called CP201) has been classified as an “orphan drug” by the US Food and Drug Administration and is currently in the preparatory stages of a clinical trial in children with ADNP syndrome through the company Coronis Neurosciences.
One author of the study is Prof. Illana Gozes.
The study is published in Translational Psychiatry.
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