Kadimastem - Stem Cell to Cure Diseases

Drug Discovery > ALS

Kadimastem's ALS Program 

A Breakthrough for a Regenerative Medicine Therapy

Kadimastem's innovative approach is to use terminally differentiated cells derived from human pluripotential stem cells (hESCs) as a cell source for a regenerative medicine therapy of ALS.

  • These cells can ultimately be developed as an off-the-shelf "industrial" product that can potentially treat additional types of neurodegenerative diseases (including CerebroVascular Accident (such as Stroke), Alzheimer Disease and Parkinson Disease).
  • The treatment provides biological support to malfunctioning motor neurons in the brain and spinal cord, thereby slowing the progression of the disease and improving the patients’ quality and expectancy of life.

Potential Market

Kadimastem’s product, AstroRx, has the potential to capture a large share of the market. The annual ALS cost in the US alone was estimated at $6 billion per year, which is indicative of a multibillion dollar market.
(Source: http://alliancerm.org/disease/als-and-regenerative-medicine)

Positive Results of Pre-Clinical Trials

The Test

Bio-safety experiments have been designed in compliance with the FDA and conducted on ~ 600 mice.
Proof-of-concept studies done on transgenic mice and rat models that have high copy numbers of the mutated (G93A) human SOD1 gene, modelling both familial and sporadic ALS pathophysiology.
Key takeaways
AstroRx was found safe, non-toxic, non-tumorigenic and present in the CNS of hundreds of NSG mice in biosafety experiments designed with the FDA
The proof-of-concept efficacy animal studies with rodents showed that AstroRx delays disease onset and prolongs survival and muscular activity of mice and rat models of ALS.


Key milestones for the Phase I/IIa Clinical Trials

Kadimastem is prepared to commence phase I/IIa clinical trials on 21 ALS patients
The trial will take place in the Department of Neurology of the Hadassah Medical Center, Jerusalem, Israel.
Hadassah is a world leading hospital for ALS therapy. Dr. Marc Gotkine, head of the ALS clinic, will be the principal investigator for the clinical trials.
The transplantation itself will take place at the Clinical Trial Center of Hadassah under the supervision of Prof. Yossef Caraco.

Prof. Tamir Ben Hur, world renowned expert in neurological diseases, is the lead medical advisor and a member of Kadimastem's Scientific Advisory Board.

AstroRx cells will be injected into the cerebrospinal fluid, a simple standard medical procedure done under local anesthesia.
Training of the medical staff regarding objective evaluation of ALS severity and scoring for the clinical trial was done by Prof.  Jeremy Shefner, a world-renowned ALS physician from the US.

Kadimastem's Diabetes Program

Kadimastem's cell therapy to treat diabetes is based on stepwise differentiation of hPSC towards pancreatic islet-like clusters, producing functional insulin (beta cells), glucagon (alpha cells) and other hormones. Starting from large scale hPSC cultures, Kadimastem's process drives the cells through several condition changes towards pancreatic endocrine cells. These functional hormones producing cell aggregates will be encapsulated to prevent immune rejection and mobility in the patient body, while allowing glucose sensing and insulin secretion.

Developmental stage

Using an unlimited cell source, hPSCs, we apply a stepwise differentiation protocol to produce islet-like clusters containing >50% insulin producing cells (β-cells) and about 20% glucagon producing cells (α-cells). Our in-vivo experiments have demonstrated functionality of the cells manifested by secretion of therapeutic levels of human insulin. The cells are encapsulated to prevent immune rejection and to contain the cells and prevent their mobility in the patient's body, while allowing glucose sensing and insulin secretion. Kadimastem’s therapy will be a cell containing off-the-shelf product that will be administered subcutaneously.

Potential Market

There are approximately 158 million relevant diabetes patients worldwide today1. The American Diabetes Association (ADA) estimated the total costs of diagnosed diabetes to be $245 billion annually (in the US) including $176 billion in direct medical costs and $69 billion in reduced productivity2. The global cost is estimated to be $548 billion annually and will grow to about $727 billion annually by 20353.

1. http://www.idf.org/worlddiabetesday/toolkit/gp/facts-figures
2. http://www.diabetes.org/advocacy/news-events/cost-of-diabetes.html
3. http://www.diabetesatlas.org/key-messages.html ; http://www.diabetes.org/diabetes-basics/statistics/?loc=db-slabnav