S. Biomedics said that the Ministry of Food and Drug Safety has approved a clinical trial of its embryonic stem cell-derived spinal cord injury treatment. It is the first clinical trial of a locally developed embryonic stem cell-derived treatment.
The company plans to evaluate the safety and exploratory efficacy of the treatment in patients with ASIA Impairment Scale (AIS)-A and AIS-C within 60 days of nerve injury at Severance Hospital from the first half of 2020.
Spinal cord injury is a disease in which the trauma to the spinal cord, the central nerve in the spinal cord, causes paralysis of the entire body or lower body. The disorder currently has no treatment options for regenerating damaged nerves. Therefore, patients with spinal cord injuries usually live with lifelong disability and often have second and third overlapping disorders.
According to the company, there are more than 70,000 patients with spinal cord injury in Korea.
“The development of treatment will improve neurological function in many spinal cord injured patients who have severe limb paralysis,” the company said. “Embryonic stem cells, which are the basis of the therapeutic agent, are stem cells made from fertilized eggs, which are capable of infinite proliferation and differentiating into cells constituting all tissues, according to culture condition.”
However, treatments derived from the stem cells have a tumor risk and require technology to differentiate into the cells of interest, the company noted, adding that such reasons have made it hard to develop or commercialize a treatment using stem cells.
The recently approved PSA-NCAM+NPC (polysialylated neuronal cell adhesion molecule positive neural progenitor cells) uses low molecular weight compounds to control two specific signaling pathways -- bone morphogenetic protein (BMP) and activin/nodal signaling pathways.
Therefore, the therapeutic agent actively blocks the differentiation of endoderm and mesoderm cells from all pluripotent embryonic stem cells and has an efficient and universal method of differentiating neurons to induce differentiation of ectoderm neurons at high yield, it said.
“As the approval is the first of its kind for embryonic stem cell-derived cell therapy, we are aiming to develop therapeutics that cover the entire nervous system,” said Cho Myoung-soo, head of research at S. Biomedics. “Other therapeutics developed by combining differentiation methods using embryonic stem cells are expected to show visible results soon.”