Kadimastem - Stem Cell to Cure Diseases

Regenerative Medicine > ALS

Stem cell therapy is emerging as a new paradigm for treating and potentially curing human neurodegenerative diseases. It involves the use of stem cell-derived living cells to replace and initiate the production of other cells that are missing or damaged due to disease or injury. At the moment, millions of patients are suffering from a wide range of neurodegenerative diseases

Based on the work of Prof. Michel Revel lab (Developer of Rebif®, Kadimastem’s Chief scientific Officer), Kadimastem is currently focusing on developing stem cell-based therapeutic solutions for Amyotrophic Lateral Sclerosis (ALS, often referred to as Lou Gehrig's disease), spinal cord injury and Multiple Sclerosis (MS). In these diseases oligodendrocyte and astrocyte cell populations, that support the proper electrical conduction of neurons, are damaged. Finding a cell source to replace and support these cells is an important unmet medical need.  

ALS, a lethal disease lacking effective treatments, is characterized by the loss of upper and lower motor neurons. The lifetime risk is approximately 1 in 2000. To date, the only 2 available effective treatments for ALS are the Food and Drug Administration (FDA)-approved agents, Riluzole (Rilutek®) which has been available since 1995 and Radicava™® (edaravone injection) which was approved by the FDA in May 2017. In clinical trials, Riluzole has been shown to be safe and provides a modest increase in median survival time by approximately 2-3 months (‎5). In clinical trials, Radicava™ has been shown to slow the decline of ALSFRS-R score by 2.5 points compared to placebo. There is currently no available treatment to stop or reverse ALS progressive course.

Although the causes of ALS are unknown and the presentation of the disease highly variable, common to all forms of ALS is the significant loss of motor neurons leading to muscle weakness, paralysis, respiratory failure and ultimately death. ALS is diagnosed after significant cell loss has occurred; a solution that would prevent further cell loss would have significant impact on survival.

Despite the relative selectivity of motor neuron cell death in ALS, studies demonstrate that glial transporters critical for the appropriate balance of glutamate surrounding the motor neurons are affected both in animal models, and in tissue from sporadic and familial ALS. The significance of non-neuronal cells in the disease process has been well characterized using SOD1 mouse models representing many of the key aspects of the human disease. In addition, transplantation using glial-restricted precursors (GRPs) that differentiate into astrocytes in SOD1 mutant rats has been shown to increase survival.

Kadimastem is focused on the development of an ALS therapy based on hESC-derived astrocyte precursor cell transplants to prevent the progression of ALS.