A new therapy for Parkinson's disease heralds a new era in medicine
TOKYO Stem cells have enormous promise in regenerative medicine, with the potential to treat a range of injuries and diseases. The main roadblock has been ethical concerns regarding the use of human embryonic tissue. But now, new technologies are starting to come onto the market that sidestep those issues, opening the door to innovative therapies, as well as identify new drugs by screening using stem cells.
International Stem Cell Corp. (ISCO) will soon start a phase I clinical trial in Australia using the company's ISC-hpNSC, human parthenogenetic stem cell-derived neural stem cells, for the treatment of Parkinson's disease, the second most common neurodegenerative disease.
California-based ISCO pioneered the development of a new class of stem cells, produced by chemically stimulating unfertilized human oocytes (egg cells), thereby creating a host of opportunities to treat a broad range of diseases. The unfertilized eggs do not have the potential to become embryos, and no viable embryo is created or destroyed.
The Nikkei Asian Review recently spoke with Russell Kern, Ph.D., chief scientific officer of the company, about the upcoming clinical trial and new developments in regenerative medicine.
Can you elaborate on the clinical trial about to start in Australia? Parkinson's disease is a degenerative disorder and a serious disease that affects millions of people all over the world. The disease occurs when dopaminergic neurons die in an area of the brain that controls movement. Dopaminergic neurons produce an important brain chemical messenger or neurotransmitter known as dopamine. When the dopaminergic neurons die, there is a shortage of dopamine that causes the movement problems of people with Parkinson's. The clinical trial we are about to start in Australia is based on extensive preclinical studies in rodents and nonhuman primates. After transplantation of ISCO's stem cells, our studies showed a significant increase in brain dopamine levels as well as neurotrophic support and cell replacement of dopaminergic neurons. The studies demonstrated improvement in Parkinson's disease symptoms, while being safe, well tolerated and not causing severe adverse effects.
What are the characteristics of parthenogenetic stem cells? They are demonstrating excellent characteristics when compared with other classes of stem cells. For example, parthenogenetic stem cells simplify therapies in regenerative medicine by potentially circumventing problems of immune rejection by limiting mismatching between stem cell-based therapeutic products and individual patients.
In recognition of the potential of our platform, in 2015 the Japan Patent Office and the European Patent Office granted ISCO protection on our stem cell lines. The patents cover the new method of creating stem cells that combine many of the benefits of embryonic stem cells with induced pluripotent stem (iPS) cells and can be used to generate virtually unlimited quantities of human cells. Currently, at the company, we are using this technology to develop treatments for a number of indications where cell therapy has been demonstrated to be effective but where there is an insufficient supply of safe, functional cells.
Being able to produce parthenogenetic stem cells in large quantities and in a way that greatly simplifies the chances of immune matching gives us a clear advantage over other stem cell technologies, like embryonic stem cells for obvious reasons. One of ISCO's stem cell lines matches approximately 70 million people and makes it incredibly simple to immune match its stem cells.
How do you see the stem cell research in other countries? ISCO has been producing its stem cell lines for its stem cell bank and for corporate and academic customers worldwide, including in Japan.
Japan is positioning itself to play a leadership role in regenerative medicine. Since iPS cells were discovered by the Japanese scientist Shinya Yamanaka of Kyoto University, the country has been on a quest to become a leader in the field. And the new regulatory environment makes Japan the most liberalized market for stem cell therapies.
Globally, there are a number of stem cell therapies in development promising to treat conditions that traditional medicine cannot. Additional good news for patients is that cell therapies are widely discussed and interest is growing in academic and industry circles.
Interviewed by Nikkei senior deputy editor Seiichiro Mishina