Collaboration will further evaluate novel stem cell-derived product for the treatment of fatal neurodegenerative disease by regulating genes relevant to disease progression
California Stem Cell, Inc. (CSC) and ALS Therapy Development Institute (ALS TDI) are pleased to announce an extension and expansion of their collaboration aimed at advancing a potential stem cell therapy for ALS (amyotrophic lateral sclerosis). This effort will build on work that has already been completed as part of this on-going partnership to understand how stem cells, and their derivatives, may be applied to treatment of this fatal neurodegenerative disease.
In this round of experiments, ALS TDI will use a CSC high-purity line of stem cells, called MotorGraft™, to deliver gene expression modifying payloads to specific locations within the central nervous system. The Institute's "Knowledgesphere", a database of information on gene and protein expression changes related to disease, contains several gene candidates to transfect into stem cells and deliver into the body. The initial phase of this new collaboration will confirm the expression of these genes in vivo. Once expression is confirmed, the collaborators will initiate the second phase of this new collaboration, which calls for several full efficacy experiments in order to measure for any potential ameliorating disease. Members of the CSC science team will travel to ALS TDI to administer the stem cells as part of each experiment.
"As part of our mission, we feel it prudent to work in this arena and will do our part to help advance the understanding of how stem cells could be used to aid in the slowing or stopping of ALS disease progression. We were encouraged by the previous round of experiments with California Stem Cell and are excited to build on the knowledge gained," said Steve Perrin, Ph.D., chief executive officer and chief scientific officer of ALS TDI.
The two groups previously worked together to conduct experiments aimed at transplanting high purity human stem cell progenitor motor neurons, into the spinal cords of SOD1 mice, which are genetically engineered to exhibit the neuronal deterioration and mimic ALS. The results of those studies showed that the cells survive, integrate, and display markers of motor neurons when transplanted into the SOD1 mouse model.