Clinical trials start for ALS drug developed at TAU

Stem-cell research used in experimental drug to treat Lou Gehrig’s disease in clinical trials at Hadassah University Medical Center.

By JUDY SIEGEL
Stem Cells 311 (photo credit: (University of Louisville Medical School)
Stem Cells 311
(photo credit: (University of Louisville Medical School)
Clinical trials using stem-cell technology from Tel Aviv University research to treat Lou Gehrig’s disease have begun at Hadassah University Medical Center in Jerusalem’s Ein Kerem. It is hoped that, as in animal models, the drug will prevent further degeneration in the brain and delay the progress of motor dysfunction in victims of the disease.
BrainStorm Cell Therapeutics, a TAU spinoff company that was licensed to develop the experimental drug called NurOwn, also reached an agreement recently with Massachusetts General Hospital, which will subsequently begin trials on patients in collaboration with the University of Massachusetts Medical School. A number of researchers working within the company have spearheaded the invention involved, and a number of the researchers working are TAU graduates.
Amyotrophic lateral sclerosis (ALS) – also referred to as Lou Gehrig’s disease after the famous New York Yankees baseball player who was struck by it – is a form of motor neuron disorder caused by the degeneration of neurons located in the ventral horn of the spinal cord and the cortical neurons. It results in the paralysis of a patient’s limbs and organ function, while brain function remains normal.
Developed by Prof. Daniel Offen and Prof. Eldad Melamed of TAU’s Sackler Faculty of Medicine and the Felsenstein Medical Research Center in Petah Tikva, the technology is now a patentpending process that takes stem cells from a patient’s own bone marrow and causes them to differentiate into astrocyte-like cells, which are responsible for the well-being of the brain’s neurons.
The cells release neurotrophic factors, or neuroprotectants, which have been shown to play a key role in reducing the progress of ALS.
The research has just appeared in the Stem Cells Reviews and Reports journal and a number of other publications.
This stem cell technology, says Offen, represents 10 years of development.
Inspired by advances in embryonic stem cell research and its huge potential – but trying to bypass the ethical and safety issues – he and his fellow researchers turned to stem cells derived from a patient’s own bone marrow.
After coaxing the cells to differentiate into astrocytelike cells, whose natural function is to guard the brain’s neurons and prevent deterioration, the researchers began testing the concept in animal models.
“In the mouse model,” Offen explains, “we were able to show that the bone marrow- derived stem cells prevent degeneration in the brain following injection of selective neurotoxins.”
According to the researchers, the technology they used is a uniquely successful method for differentiating bone marrow stem cells into astrocyte-like cells without manipulating the genetic material of the cell itself.
They are the first team of researchers to demonstrate the efficacy of this technology in vivo in various models of neurodegenerative diseases.
The ongoing clinical studies are aimed at evaluating the safety and the efficacy of this treatment, says Offen. Because the original cells are drawn from the patients themselves, he adds, the body should have no adverse reactions.
Although the current study targets ALS, these cells have the potential to treat a broad range of neurodegenerative conditions, including Parkinson’s and Huntington’s diseases.
For many diseases of this kind, the current available treatments only attempt to alleviate the symptoms of these diseases rather than repair existing damage.