First Human Stem Cell Trials

Earning the United States Food and Drug Administration’s (USDA) approval, UC Irvine researchers will be the first to test an embryonic stem cell treatment on humans in clinical trials.
Hans Keirstead, co-director of the UCI Sue and Bill Gross Stem Cell Research Center, headed the research responsible for earning the USDA’s nod by using stem cells to cure paralysis in rats. This was accomplished by transforming the stem cells into oligodendrocytes, a form of spinal cord cells that maintain communication in the central nervous system of vertebrates.
Gabriel Nistor, a postdoctoral scholar employee in the UCI Department of Anatomy and Neurobiology, aided Keirstead in developing a technique that would turn human embryonic stem cells into oligodendrocytes. The approach utilized stem cells to replace a natural insulation known as myelin. Myelin allows the central nervous system to run smoothly through facilitating electrical conduction. When the amount of myelin is insufficient in a vertebrate, communication in the central nervous system is weakened and paralysis can occur.
Although UCI researchers will be the first to treat humans using embryonic stem cells, according to Nistor, this will not be the first time that humans have been treated in trials using stem cells. This is because previously neural stem cells derived from fetal brains have been used in USDA-approved trials. However, the addition of embryonic stem cells in clinical trials is significant as these cells are pluripotent, whereas adult neural stem cells are merely multipotent. The terms pluripotent and multipotent refer to the range of cell types into which stem cells can grow. While pluripotent cells can become nearly any tissue type, multipotent cells are limited by what type of tissue they turn into.
For instance, adult neural stem cells would only be able to grow into tissue found in the central nervous system. Because embryonic stem cells are not bound to specific tissue types, there is more room for innovation.
“[For testing] embryonic stem cells, I don’t know any clinical trial. This is the very first one. It’s a pioneering trial,” Nistor said.
In order to reach the point of clinical trials, funding was needed, which was partially supplied by the Geron Corporation. According to Nistor, the relationship between researchers and the corporation improved the work being done with stem cells beyond financial terms.
“Our collaboration with the corporation took a step forward by putting the quality of the industry in our work, in a translatable approach with quality insurance … [their guidelines were] more appropriate for industry standard than [just] lab books in [a] university,” Nistor said.
According to Vice Chancellor for Research Susan Bryant, outside of private funding, federal and state funding must also be utilized to benefit stem cell research.
“The National Institute of Health [NIH] would be the main place where it’s funded, and it’s a small program and is very limited in the amount of cell lines that can be used,” Bryant said.
The reason for this is due to a presidential order issued in 2001. According to Bryant, the NIH budget has also been declining in recent years, making funding increasingly difficult. Likewise, state resources have similarly been reduced.
“The California Institute for Regenerative Medicine has been giving out funding for stem cell research for the last four years and it’s now getting into the same budget difficulties that the rest of the state is,” Bryant said.
Regardless of funding issues, the ball is now rolling in regard to embryonic stem cell clinical trials. Nistor compared his work to early diabetes studies conducted in the 1960s. Although patients used in clinical tests experienced allergic reactions from substances such as bovine insulin, the findings from these experiments led to further studies to develop safer medicines. While Nistor cannot foresee the exact results of the clinical trials, he believes that the lone fact that clinical trials are occurring is historical.
“It’s important to have a start regardless if the outcome is good, or unknown or uncertain,” Nistor said. ” A clinical trial might fail. It doesn’t really matter. It’s a starting point.”