Israeli scientists say they have developed a drug that slows protein deposits on the brain that are a cause of Alzheimer’s disease, and say it may also be used for treating some autism cases.
The Tel Aviv University team behind the peer-reviewed research saw an improvement in the symptoms of mice. The biggest success, they say, was a dramatic decrease in the excessive buildup of a protein in the brain called tau. The protein performs important functions in normal brains, but among Alzheimer’s patients it tends to build up excessively, and becomes misfolded and abnormally shaped.
The scientists edited the genomes of the mice to give them a rare developmental disorder called ADNP syndrome, which involves a range of symptoms, many of them close to those of Alzheimer’s.
For years, biochemist Prof. Illana Gozes of Tel Aviv University has been working on an experimental drug for ADNP called NAP, which is based on NAPVSIPQ, a snippet of a protein essential for brain formation.
Now, she has used genome editing techniques to produce mice with ADNP and examine the impact of NAP on the different symptoms. She illustrated a strong reduction in tau buildup, and said that if the same effect is seen in human trials, that could make the drug a good candidate for preventing the onset of Alzheimer’s.
“This is the first time we have examined the impact of NAP using a genome-edited animal model, and indications are that it protects against the same deposits that are found in the brains of people with Alzheimer’s,” Gozes told The Times of Israel.
“In people, the more deposits there are, the more dementia we tend to see, so this is very positive.”
Separate to the reduction of tau buildups, changes to the brain’s electrical activity that are common to Alzheimer’s patients were reduced after the administration of NAP, Gozes said.
Her team, which included Dr. Gideon Carmon, also observed a lessening of other symptoms associated with ADNP, including slowed development and problems walking.
One of the symptoms of ADNP is Autism Spectrum Disorder, and Gozes said she is hopeful that positive outcomes in the lab experiment indicate that NAP may help treat some ASD cases — those caused by ADNP, and “many” others.
“This study is an important milestone on the way to developing a drug, or drugs, that will help children with autism stemming from genetic mutations, as well as Alzheimer’s patients,” Gozes said.