Projects Are Preclinical
ISCO’s lead therapeutic candidate targets Parkinson’s disease. Dr. Semechkin says he expects to complete IND-enabling studies this year. The company had a pre-IND meeting with the FDA in February, and currently is working to complete the necessary tumorigenicity, toxicology, and tolerability studies.
ISCO’s first clinical product will be human parthenogenetic neural stem cells (hPNSCs)—self-renewing multipotent cells that differentiate into key cells in the central nervous system. ISCO literature says they are “differentiating into dopaminergic neurons and expressing specific neurotropic factors to protect the nigrostriatal system of the brain.” Along with expressing brain-cell-protecting neurotrophic factors that prevent further damage, the cells can engraft and produce the dopamine necessary to prevent the symptoms of Parkinson’s disease.
Importantly, unlike other neuronal cells, hPNSCs can be cryopreserved and delivered to clinical sites in a frozen state. Clinics thaw the cells when needed, performing the quality control and viability tests just before implantation.
“Additionally, a number of other indications including stroke, traumatic brain injury, and amyotrophic lateral sclerosis can also be treated with neural stem cells, making it a very attractive product from a commercial perspective,” Dr. Craw asserts.
While this will be the first therapeutic to market, the company also has 128 patents and cross-licenses, with another 90 patents pending. Those patents and licenses span 30 patent families, covering specific hpSC lines as well as production and differentiation methodologies for research, therapeutic, and commercial applications.
Another program using ISCO’s technologies creates a human cornea from stem cells, enabling corneal tissue implants. Because corneal tissue is made of multiple cell types and structures, “This is quite an achievement,” Dr. Craw says. The company plans to commercialize this product in India through Insight Bioventures.
“The greatest need for corneal tissue is in India and China, where healthcare systems are not well developed and eye-banking is rare,” he elaborates. “The Indian population also has a slight genetic predisposition to a particular corneal disease.” In the United States, because there is an excellent network of eye banks and limited cultural resistance to the organ donation, there is no significant market for this product.
ISCO also has a program to create liver cells using its stem cell platform. “In some inherited liver diseases, such as Crigler-Najjar Syndrome, the liver lacks the ability to create a particular enzyme. In such cases, liver transplants and hepatic cell transplants have been useful,” notes Dr. Craw. “However, liver cells are not cryopreservable and do not last more than a few hours outside the body. Consequently, the major problem is obtaining sufficient donated liver cells.”
ISCO is optimistic about its “liver-like” cells. The company says they perform as well as those from donated liver cells in animal models of liver disease.