Promising stem-cell based drug for treatment of ALS receives patent

The therapeutic drug is safe for administration and helpful in slowing disease progression, as well as increasing life expectancy and the overall quality of life for ALS patients.

THE HADASSAH-UNIVERSITY Medical Center campus is seen in Ein Kerem. (photo credit: MOSHE SHAI/FLASH90)
THE HADASSAH-UNIVERSITY Medical Center campus is seen in Ein Kerem.
(photo credit: MOSHE SHAI/FLASH90)
Drug company Kadimastem received a patent from the Israel Patent Office on Tuesday for the promising new drug AstroRx to treat Amyotrophic Lateral Sclerosis (ALS). The new medication uses embryonic stem cells to create healthy nervous system cells, which can support malfunctioning brain and spinal cord tissue in patients suffering from this neurodegenerative disease.
Results of its most recent clinical trial indicated that this therapeutic drug is safe for administration and helpful in slowing disease progression, as well as increasing life expectancy and overall quality of life in ALS patients. 
Kadimastem CEO Asaf Shiloni explained excitedly that, "the patent granted by the Israel Patent Office is a significant step forward towards bringing AstroRx to market for the benefit of ALS patients worldwide.
"We aim to have a pre-IND [Investigational New Drug] meeting with the US FDA in the near future, with the goal of setting in motion the next stage of clinical trials as soon as possible," he said. "In the meantime, we shall continue our groundbreaking research and development programs for both our ALS and diabetes products.”
ALS is a crippling and fatal disease that causes a progressive degeneration of motor neuron cells. These cells travel from the brain down through the spinal cord to control muscular movement in the body. When they don't function properly, an individual suffering from the disease slowly loses his or her ability to move, eat, speak and eventually breathe. There is no cure for the disease, but Kadimastem's innovative drug can slow the rate of its progress and vastly improve quality of life. 
Kadimastem's bio-pharmaceutical company develops their medicinal therapies using Human Embryonic Stem Cells (hESCs) to create specific cell types that patients with the disease lack.
With AstroRx, the company has mastered a method of turning pluripotent, or immature stem cells into astrocytes, cells in the spinal chord and brain. The astrocyte cells are then injected directly into the cerebrospinal cord fluid (CSF) of the patients. Healthy astrocytes are crucial in treating the disease, because they support the functioning of the nervous system and help repair damage to the brain and spinal chord.
The company's technology is groundbreakingly important as it can reduce the cost of treatment and avoids genetic modification of parent astrocyte cell populations in the disease's treatment and slowing of its progression. 
AstroRx just completed Phase 1/2a of its clinical trial and found the treatment to be safe for use and encouraging in its effectiveness. There were no serious side effects in the six-month period following its administration to the Cohort B test group. 
According to the ALS Functional Rating Scale-Revised (ALSFRS-R), the rate of ALS disease progression in Cohort B patients decreased from -1.43/month to .78/ month, a decrease of almost a half (45%).
"The results after six months of follow up are encouraging, as they suggest a clinically meaningful signal of effect for a period of three months by a single administration of AstroRx® and confirm the safety of AstroRx®," explained Dr. Marc Gotkine, a leading researcher in the trial and head of Hadassah Medical Center's ALS.
The trial was conducted at Hadassah Medical Center, in Jerusalem's Ein-Kerem. The drug has been recognized by the FDA as an orphan drug for the treatment of rare diseases. 
Kadimastem is traded on the Tel Aviv Stock Exchange (KDST).