Public forum: Stem cells and key diseases

HSCI researchers are exploring options to treat diabetes, nervous system diseases, cardiovascular disease, and other degenerative diseases in a variety of ways, including stimulating existing stem cells, transplanting new cells, and targeting specific stem cell populations. At a recent public forum on “Stem Cells and Key Diseases,” moderated by Kevin Eggan, PhD, HSCI brought together three of its disease program heads to discuss the state of science in both the laboratory and in the clinic, and to identify areas of progress and potential.

Gordon Weir, MD, head of HSCI’s Diabetes Program, addressed the progress and goals of diabetes-focused stem cell research. In order to treat diabetes by increasing the number of insulin-producing beta cells in the pancreas, Weir explained, researchers are actively investigating two main strategies - how to create new cells for implantation and how to encourage existing beta cells to multiply.

Drawing an analogy between stem cell research and the development of heart transplantation surgery techniques, Kenneth Chien, MD, PhD, head of the Cardiovascular Program at HSCI, said he expects stem cell research to transform our understanding of cardiovascular disease over the next 10 years, potentially leading to new drugs and treatment strategies.

In discussing the complexity of the nervous system, Jeffrey Macklis, MD, DHST, head of the HSCI’s Nervous System Diseases Program, explained that many hundreds, if not several thousands, of different types of neurons exist in the brain. Macklis compared this diversity of nerve cell types to different types of vehicles, ranging from a sedan to a fire truck, each with a specific function and capability. Macklis stressed that researchers face the problem of determining which kind of vehicle they want and whether it is needed for a replacement part or for use in disease study. In discussing Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s disease, and other diseases of upper and lower motor neurons, Macklis profiled ongoing research at HSCI that is attempting to make both spinal and corticospinal motor neurons, as well as research aimed at studying what drugs might help preserve them or enhance their growth. Macklis emphasized that success in this field will result not only in the replacement of diseased neurons but also in the discovery of drugs for the amelioration or prevention of neuronal death and degeneration through the use of specialized nerve cells for disease study.

While each of the panel members agreed that the therapeutic use of stem cells is many years away, there was also consensus that current research in the field has and will continue to result in many useful clinical tools for disease study. Advances in stem cell research have provided researchers with an opportunity to study the progression of disease at the cellular level, resulting in increased knowledge about the nature of disease progression as well as the potential for finding novel methods of treatment.

A video of the public forum is available online at: