Pipeline

We have derived a homogenous population of neural stem cells (ISC-hpNSC®) from hpSC through a chemically directed differentiation protocol (1). The ISC-hpNSC® were expanded and cryopreserved into master and working cell banks under cGMP. The cells underwent strict quality control measures testing each cell bank for cell identity, potency, purity and sterility to ensure the safety of the final product. Our clinical grade ISC-hpNSC® express the appropriate neural stem cell markers, have a normal karyotype and are clear of microbial and viral contaminants.

Parkinson’s Disease
According to the Parkinson’s Disease Foundation, an estimated seven to 10 million people worldwide live with Parkinson’s disease. As many as one million patients live in the United States, more than the combined total of people diagnosed with multiple sclerosis, muscular dystrophy, and Lou Gehrig’s disease. The total direct and indirect cost of Parkinson’s disease is estimated to be nearly $25 billion per year in the United States.

At ISCO, unlike conventional treatments for Parkinson’s disease that do not stop disease progression, the company’s ISC-hpNSC® offers to be a disease modifying treatment that could potentially slow down disease progression. The ability of our stem cells, ISC-hpNSC® to differentiate into dopaminergic neurons as well as express brain-protecting neurotrophic factors offers a new approach to treating Parkinson’s disease. We believe that a one-time transplant of ISC-hpNSC®, into the brain of Parkinson’s patients, replacing the dead and dying dopaminergic neurons and offering protection and neurotrophic support to the remaining neurons, could alleviate symptoms of the disease and prevent further deterioration.

Transplantation of ISC-hpNSC® was shown to be safe, well tolerated and effective in treating preclinical models of PD (2,3). ISC-hpNSC® promoted behavioral recovery without dyskinesias and increased striatal dopamine (DA) concentration, fiber innervation and number of DA neurons in comparison to control animals. Based on these extensive preclinical studies, the Australian Therapeutic Goods Administration and Human Research Ethics Committee have granted approval to conduct a dose escalating Phase I study.

Traumatic Brain Injury
According to the CDC, roughly 30% of all injury related deaths are caused by Traumatic Brain Injury (TBI). For survivors, lasting effects from the injury including cognitive function, memory loss, depression, and altered vision, can remain. Roughly 2.5 million emergency room visits, hospitalizations, and deaths occurred in 2010, with TBI contributing to 50,000 deaths.

In addition, TBI can cause epilepsy and patients are at risk of Alzheimer’s and Parkinson’s diseases and other brain disorders that become prevalent with age. In the acute phase of TBI, significant breakdown of the blood brain barrier is quite common with subsequent inflammation. ISC-hpNSC® provides neurotrophic support and neuroprotection by the release of neurotrophic cytokines which may dampen an inflammatory reaction and help restore the blood brain barrier. For the more chronic phase of TBI, ISC-hpNSC is capable of repairing damage from Parkinson’s disease and stroke. It is therefore possible that ISC-hpNSC® may alleviate the non-specific damage inflicted by TBI.

Ischemic Stroke
The National Stroke Association lists stroke as a leading cause of death in the United States, and a leading cause of serious, long-term adult disability. Approximately 795,000 strokes occur each year, taking a life approximately every four minutes. About 87% of strokes are ischemic. The estimated direct and indirect cost of stroke in the United States in 2010 is $73.7 billion.

Despite its prevalence, there are no medical therapies for alleviating the chronic functional impairments following stroke. Human fetal neural stem cell transplants have been shown to produce some recovery in animal models of stroke, but ethical consideration and a short supply of fetal tissue limits this approach. Our clinical grade ISC-hpNSC® have been demonstrated to be safe, and provide significant functional improvement in a stroke mouse model. ISC-hpNSC® have the ability to become a variety of neural cell types replacing the brain cells lost after a stroke. ISC-hpNSC® also provide neurotrophic support and neuroprotection by the release of neurotrophic cytokines. Additionally, ISC-hpNSC have immunomodulatory properties that reduce the inflammatory response caused after the stroke. The multiple mechanisms of action make ISC-hpNSC® an ideal therapeutic candidate for stroke.