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Kadimastem Approached the FDA Regarding Its ALS Treatment
Oct. 6, 2014
The company recently reported positive results in its first pre-clinical trial for the treatment of ALS.
Ness Ziona, Israel, 6 October 2014, Israeli biotechnology company Kadimastem announces today that it approached the FDA regarding the cellular treatment it is developing for ALS. This is the initial approach the company is making to the FDA. In the framework of talks with the FDA, Kadimastem intends to consolidate a preliminary outline for its continuing trials for this indication.
Recently, the company reported positive results in a pre-clinical trial it conducted. In the trial, the efficacy of injecting support cells (astrocytes), produced by the company in a unique technology, into the spinal fluid of ALS model mice, was tested. This model is highly significant in predicting the treatment's activity in humans.
The results of the trial showed an increased life expectancy in the mice treated, as well as a significant improvement of their motor (muscle) function, compared to the untreated mice. The efficacy of the treatment was also demonstrated in other indices indicating a delay in disease onset. Injections into the spinal fluid are standard procedure performed routinely in hospitals around the world. The company found that injections into the spinal fluid enable the even dispersion of cells throughout the central nervous system, thereby establishing the method of cell penetration in future treatment of patients.
Professor Michel Revel, the company's Chief Scientist, noted: "This is an important step in the company's progress towards the development of cellular therapy for ALS patients. Kadimastem is developing an industrial product, and is designing the cell production process while ensuring the required safety and reliability regulations. The early contact with the regulatory authorities is vital for the success of the development".
Yossi Ben-Yossef, the company's CEO, noted: "Since the publishing of the initial results of the pre-clinical trial, we are operating to advance the process with the FDA. Upon receiving the FDA's feedback, we will pursue the clinical development of cellular treatment for ALS".
Kadimastem (www.kadimastem.com) (TASE: KDST) uses human stem cells that are differentiated to create medical solutions for diabetes and degenerative diseases of the nervous system and for drug screening. The company’s technological platform enables the differentiation of stem cells into a range of functional human cells, including neuron-supporting cells in the brain as well as pancreatic cells that secrete insulin – beta cells.
Kadimastem relies on technology that was initially invented at the Weizmann Institute in Prof. Michel Revel’s laboratory, and which is now being further developed and advanced in the company’s labs at the Weizmann Science Park. Kadimastem is developing two types of medical applications: A. Regenerative medicine, the purpose of which is to repair and replace tissues and organs damaged by disease using healthy cells grown in laboratory conditions, for example, transplantation of insulin-secreting pancreas cells to treat insulin-dependent diabetes, or transplantation of healthy brain-supporting cells to improve survival of nerve cells and treat ALS; B. Drug screening platforms which use functional human cells and tissues to discover new medicinal drugs.
ALS (Amyotrophic Lateral Sclerosis) is the most severe of a group of neurodegenerative diseases that damage motor neurons, and is incurable. In the United States it is also known as Lou Gehrig's disease, after the American baseball player who was affected by the disease in the 1930s. The disease causes paralysis and death, with average survival after diagnosis of only 2-5 years. Currently, there is no cure or significant drug treatment for ALS, except one drug which prolongs the patient's life only by a number of months. The Ice Bucket Challenge, in which Kadimastem participated, has unprecedentedly raised awareness of the disease worldwide.