Scientists make breakthrough in ALS treatment

BGU researcher, colleagues identify compound that may prolong life for Amytotrophic Lateral Sclerosis patients.

ESTHER PRIEL 390 (photo credit: BGU)
(photo credit: BGU)
A Ben-Gurion University researcher and her colleagues have identified a chemical compound that may lead to prolonged lifespans for patients with Amytotrophic lateral sclerosis (ALS) and other neurodegenerative diseases.
Prof. Esther Priel and her team published an article in the February edition of EMBO Molecular Medicine Journal, in which they demonstrate how their compound can induce an increased production of the enzyme telomerase in the bodies of laboratory mice and in human stem cells. The increased amount of telomerase slows the progression of ALS, also known as Lou Gehrig’s disease.
Telomerase has already been proven to prolong life in such cases, and in 2009, scientists Elizabeth H. Blackburn, Carol W. Greider and Jack W. Szostak won the Nobel Prize in Physiology or Medicine for their discovery of how telomerase protects chromosomes.
Priel, based in Ben-Gurion University’s Department of Microbiology and Immunology, worked principally with her colleagues Prof. Aviv Gazit – formerly of the Hebrew University – and Prof. Shimon Slavin – also formerly of the Hebrew University and now director of the International Center for Cell Therapy & Cancer Immunotherapy in Tel Aviv – as well as a team of student researchers. About a month before its article was published, the group also secured an over-$1 million grant for their patented breakthrough treatment for neurodegenerative diseases, BGU said.
BGN Technologies, BGU’s tech transfer company, recently signed a research and license agreement with a private American investment fund that has agreed to invest the money to expand the team’s research over the next two years.
“This is a scientific breakthrough, since these compounds have great potential not just in fighting neurodegenerative diseases, but also other diseases related to aging,” Priel said.
The chemical compound engineered by the team increased the protein and the enzyme levels within both mice cells and human stem cells, thereby “protecting the cells from oxidative stress,” Priel told The Jerusalem Post on Sunday. The enzyme, she explained, is responsible for the lifespan of the cells as well as the tissues to which they belong, and mice whose genetics were manipulated to include this enzyme had prolonged lives.
“This is why also in potential they are good for other neurodegenerative diseases, because they are not providing any answer for the cause of the disease,” Priel said.
“They are enabling the damaged cells to protect themselves from the consequence of the damage. This is why this could be good for different diseases.”
It will take at least 10 years to develop the compounds into a drug, she predicted. In the meantime, however, the team is producing positive results in animals, she said.
While ALS remains a rare disease in Israel and throughout the world, Priel stressed just how debilitating it becomes to those who have it.
“There are not a lot of people, fortunately, who have this disease, but it’s a terrible disease because they become paralyzed, and the head is working very well, so they are aware of what is going on with their body,” she said. “It is a devastating disease and there is no cure for it.”