San Diego Business Journal

International Stem Cell Corp. is entering a human clinical trial to test if the company’s patented stem cell line will repair the brains of Parkinson’s patients.

The clinical trial will be the world’s first human trial to test stem cell therapies in patients suffering from Parkinson’s disease, according to ISCO’s chief scientific

officer Ruslan

Semechkin, son of CEO Andrey Semechkin.

ISCO is best known for patenting a new class of stem cells called human parthenogenetic stem cells, one of only three classes of stem cells. HpSCs are pluripotent stem cells, meaning they self-replicate and have the potential to differentiate into most cells in the body. This means that under the right circumstances, a stem cell may be able to replace ailing or destroyed tissue in the body with cell-based therapies.

Pluripotent stem cells are normally derived from human embryos or human fetal tissue, however, Semechkin said that ISCO’s line of hpSCs differ from embryonic stem cells in that their derivation does not require fertilized eggs or the destruction of viable human embryos.

Semechkin said ISCO’s class of stem cells have the potential to make cells and tissues that are less likely to be rejected during transplantation — a common issue with other human stem cells.

ISCO has created a new stem cell product called human parthenogenetic neural stem cells, or hpNSCs. HpNSC’s are built out of the company’s hpSC line, and are self-renewing multipotent cells that are precursors for the main cell types of the central nervous system. The company intends to use this cell line to help repair damaged tissue in the brains of Parkinson’s patients.

The Unmet Need

Parkinson’s disease is a chronic movement disorder of the central nervous system that often causes tremors, rigidity and slowness in those who suffer from it. The disorder is progressive, meaning that symptoms continue and worsen over time.

An estimated seven to 10 million people worldwide live with Parkinson’s disease, with as many as one million of those in the U.S.

The standard of care for the treatment of Parkinson’s is a drug called levodopa or L-dopa, but oral dosing of L-dopa has flaws. During treatment, the timing and amount of L-dopa that is absorbed into the bloodstream varies widely. This variability results in the emergence of “off episodes” — or periods of time during which Parkinson’s disease symptoms re-emerge despite the L-dopa treatment. These off episodes, which increase in frequency and severity during the course of the disease, are experienced by a majority of Parkinson’s patients and are considered one of the greatest unmet medical needs facing Parkinson’s patients.

Attracted to Site of Injury

Neural stem cells work to repair the brain in several ways. The cells are attracted to the site of injury and, in response to signals released by the damaged tissue, release a range of molecules that reduce inflammation, prevent further damage and trigger the recovery process.

Parkinson’s disease patients lack a chemical in the brain called dopamine, which helps control muscle movement. Neural stem cells have the ability to make and replace the dead and dying dopamine-producing cells in patients, replacing the missing neurotransmitter and reversing the symptoms.

ISCO’s human trial follows two promising animal studies that looked at the company’s neural stem cell treatment: a 9-month nonclinical study involving 300 rats and a 12-month study involving 22 monkeys. The studies tested safety and efficacy of ISCO’s new stem cell product human parthenogenetic neural stem cells in animals.

The animal studies confirmed that the new line of neural stem cells was effective, safe and well-tolerated for the treatment of Parkinson’s, according to the company.

Finding a Partner?

Once the company generates efficacy and safety data in humans, Semechkin said ISCO will likely partner with a large pharmaceutical company to get the company through clinical trials.

“There’s a lot of interest about the program, but Big Pharma is kind of concerned about the stem cell field in general,” Semechkin said.

There are still no FDA-approved cell therapies available to patients, according to Semechkin. The biggest concern surrounding stem cell therapies is tumor formation in patients, but Semechkin said ISCO’s animal trials showed no evidence of tumor formation or abnormal tissue in animals that received the human cell transplants.

The company is not entirely dependent on a Big Pharma partnership for funding as it has two existing streams of revenue. Sales revenue for a clinical-stage company is somewhat rare, but ISCO pulls in several million dollars per year from its Lifeline Cell Technology business (which offers cell types to other researchers) and its Lifeline Skin Care business (which sells skin care products with stem cell extracts).

Combined, the business units brought in $7 million in 2014.

INTERNATIONAL STEM CELL CORP.

CEO: Andrey Semechkin

Revenue: $7 million in 2014; $6.1 million in 2013

Net loss: $12.5 million in 2014; $10.5 million in 2013

No. of local employees: 45

Headquarters: Carlsbad

Year founded: 2006

Company description: ISCO is focused on the therapeutic applications of human parthenogenetic stem cells and the development and commercialization of cell-based research and cosmetic products

Key factors for success: Promising nonclinical data, more than one revenue stream that helps fund clinical research