Kadimastem

Company Kadimastem

Drug Name AstroRx®

Technology hPSCs (Human Pluripotent Stem Cells)

Orphan Drug Status YES - FDA

Website https://www.kadimastem.com/

Multiple studies have demonstrated that astrocytes play a crucial role in ALS disease progression. In ALS patients, the astrocytes malfunction and attack the neurons.

Kadimastem's innovative approach is to use fully differentiated astrocytes as a regenerative medicine therapy for ALS – AstroRx® to slow the progression of the disease and improve the patient's life quality and life expectancy. AstroRx® is administered directly into the patient’s spinal cord fluid by a standard intrathecal injection under local anesthesia. Pre-clinical studies in ALS animal models showed that AstroRx® is a safe treatment that provides systemic support to damaged motor neurons, increasing the life expectancy and motor functions.

Stem cells are defined by two important characteristics: They are able to renew themselves through cell division, and, under certain physiological or experimental conditions, they can differentiate into (turn to) tissue or organ specific cells. In some organs, such as the gut and bone marrow, stem cells often repair and replace damaged tissues. In other organs, such as the pancreas and the heart, stem cells only rarely divide.

4-5 days after fertilization, the pre-embryo, called blastocyst, has an inner mass of cells that can give rise to any cell type (except the placenta).

Human embryonic stem cells (hESC) lines are derived from this inner cell mass. These cells are called pluripotent due to their potency to differentiate into many (pluri) cell types.

Induced pluripotent stem cells (iPSC) are pluripotent stem cells that are artificially made from “regular” adult cells (for example: a skin cell). Inserting specific genes into a cell can cause a the cell to become pluripotent. iPSC share many similar characteristics with hESC, among them unlimited proliferation potential and the ability to differentiate to all the cell types comprising the body. Kadimastem uses both pluripotent cell sources (hESC and iPSC) for deriving its differentiated cells.

Collectively these cells are called human pluripotent stem cells (hPSCs).