Technion discovers how to reduce cognitive damage from brain disease

Researchers: This breakthrough demonstrates a potential new treatment for cognitive damages in the brain white matter.

From Right to Left: Pig’s brain; standard MRI brain scan; new MRI scan showing differences in molecular makeup in different parts of the brain (photo credit: SHIR FILO/HEBREW UNIVERSITY)
From Right to Left: Pig’s brain; standard MRI brain scan; new MRI scan showing differences in molecular makeup in different parts of the brain
(photo credit: SHIR FILO/HEBREW UNIVERSITY)
In a groundbreaking discovery, researchers from the Technion-Israel Institute of Technology, in partnership with Cincinnati Children’s Hospital Medical Center (CCHMC), found out how to rescue functional and structural connectivity, and reduce cognitive impairments of brain diseases with drug treatment.
The researchers, Prof. Itamar Kahn of the Technion’s Rappaport Faculty of Medicine in Israel and Prof. Nancy Ratner of Cincinnati Children’s Hospital Medical Center (CCHMC), claimed that such a breakthrough demonstrates a potential new treatment for cognitive damages in the brain white matter, the areas of the central nervous system. 
 
The brain white matter, contains millions of nerve fibers, or axons, that connect other parts of the brain and spinal cord and signal your nerves to talk to one another. A fatty material called myelin protects the fibers and gives white matter its color. 
 
The pioneering research, “Brain-wide structural and functional disruption in mice with oligodendrocyte-specific Nf1 deletion is rescued by inhibition of nitric oxide synthase,” was published in the official journal of the National Academy of Sciences of the United States, PNAS. Technion MD/PhD candidate Jad Asleh and Tel Aviv Medical Center neurosurgeon Dr. Ben Shofty were co-lead authors.
The brain disease Neurofibromatosis type 1 (NF1), one of the most common genetic disorders and is not limited to any person's race or sex, causes tumors in the nervous system which can grow anywhere on the body.
The research showed that treating NF1 with a drug targeted at an abnormal cellular pathway that is critical for normal white-matter formation, rescues functional and structural connectivity and is thought to potentially alleviate some of the cognitive impairments seen in NF1.
Neurofibromatosis mouse model shows impaired structural and functional brain connectivity that is rescued by targeted therapy. (Credit: Technion)Neurofibromatosis mouse model shows impaired structural and functional brain connectivity that is rescued by targeted therapy. (Credit: Technion)
According to the study, by using a cell-specific, hormonal triggered, genetically engineered mouse model, the researchers were able to confine disease manifestation to white matter producing cells and, as a result, directly examine the contribution of abnormal myelination to neurological disorders, myelination referring to an increase in the fatty envelop surrounding neuronal processes and fibers that increase the efficiency of electrical transmission.
This study emphasizes the role of abnormal white matter in brain disorders and suggests a potential targeted therapy for NF1-associated myelin abnormalities. 
Based on the results of previous work from Prof. Ratner and this study, a preliminary clinical study is currently in progress to test the safety of this drug treatment and its potential in treating the cognitive impairments in NF1 pediatric patients.