Department of Neurology Developing New Treatments for ALS

May 12, 2008

New treatments for amyotrophic lateral sclerosis, or ALS, one day may be possible using progenitor cells from a patient’s own nervous system, according to investigators in the Jim Himelic Neuromuscular Research Laboratory at The University of Arizona College of Medicine.

Progenitor cells, which are found in the brain and spinal cords of adults, have the potential to develop into new nerve cells that could replace those lost to ALS as well as to other neurologic diseases such as Parkinson’s and Alzheimer’s Diseases.

ALS (also called “Lou Gehrig’s disease” after the famous New York Yankee who lost his life to the disorder) is a progressive, fatal neurodegenerative disease affecting adults. The disease, which typically occurs randomly in the population, causes a loss of muscle function by affecting the nerve cells in the brain and spinal cord that control movement, eventually leading to death.

According to the Muscular Dystrophy Association, every day an average of 15 people are newly diagnosed with ALS – more than 5,000 people per year – and ALS currently affects as many as 35,000 Americans. Unfortunately, the average life expectancy following diagnosis typically is two to five years.

UA researchers are developing novel techniques to activate cells called progenitors – found in the brain and spinal cord of adults – to form new nerve cells. During human development from the embryonic, or the first eight weeks, through the fetal, or eight weeks to birth, periods, these progenitors produce almost all of the nerve cells in the brain and spinal cord.

Once this task is complete, progenitors generate other brain cells but do not make new nerve cells. The goal of UA researchers is it to “re-train” these progenitors – which remain present in the adult brain and spinal cord – to produce new nerve cells. In this manner, researchers hope to replace the nerve cells that have died in ALS patients with new functional nerve cells, potentially slowing or possibly even reversing the course of the disease. If successful, this approach also may be used to replace other types of nerve cells that are lost in other neurologic diseases.

Using endogenous progenitor cells that already are present in the patient’s own nervous system avoids many of the scientific and political hurdles associated with using exogenous – another person’s – cells. In addition to the technical difficulties of transplanting cells throughout the brain and spinal cord, exogenous cells potentially can be rejected by the recipient’s immune system and also could inadvertently introduce a foreign cell that may contain a virus.

The UA’s ALS research is funded by the Jim Himelic Foundation, known as JHF, established in 2000 by the family, friends and colleagues of Jim Himelic, a friend of the Tucson community and much-respected juvenile court judge who died from ALS in February 2000.

JHF has already has raised $492,000 toward a goal of $1 million in seed money for ALS research at the UA College of Medicine. Seed money allows researchers to fund the preliminary research necessary for obtaining larger government and private grants to further their studies.

The funds have allowed the UA to hire cellular biologist Jonathan Flax, UA research assistant professor of molecular and cellular biology in the University’s department of neurology, and one of 10 individuals in the country conducting progenitor cell research for the treatment of ALS.

JHF also is working with neurologist Dr. Katalin Scherer, UA assistant professor of neurology, to apply ALS research in a clinical setting. Scherer is fellowship trained in clinical neurophysiology and board certified in electrodiagnostic medicine. Her clinical and research interests include ALS as well as other adult and pediatric neuromuscular diseases.

“We are dedicated to laboratory and clinical-based research and to the comprehensive care of patients with neurologic disease, including ALS,” says Bruce M. Coull, professor and head of the UA Department of Neurology. “Our hope is that this cutting-edge research will eradicate ALS within our lifetime.”