Newswise — Twenty years ago, retail pioneer and philanthropist A. Alfred Taubman lost a good friend to amyotrophic lateral sclerosis — a horrifying fatal disease that's better known as Lou Gehrig's disease or ALS.
The memory of watching New York Sen. Jacob Javits slowly succumb to the nerve-killing condition has never left Mr. Taubman's mind. And that memory has motivated him to support ALS research at the University of Michigan Medical School and beyond.
Now, he has greatly increased that support, with a new $5 million gift, and the promise of even further funding when he also donates his share of the royalties from his new book, "Threshold Resistance."
The gifts will go to support ALS studies led by U-M neurologist and scientist Eva Feldman, M.D., Ph.D., whose lab has already received two $1 million gifts from Mr. Taubman. She is considered a national leader in ALS treatment and research, and heads the U-M Program for Neurology Research and Discovery.
Together with a team at University of California, San Diego School of Medicine, led by Martin Marsala, M.D., associate professor of anesthesiology, Dr. Feldman and her team will work on several scientific fronts to try to stop or slow the disease. Working at first in animals, then in ALS patients, they hope to make quick progress.
Among the weapons they will deploy against ALS are genetic tools to keep nerve cells from dying, new ways of delivering promising drugs and genes directly into nerve cells, and a potential treatment based on injecting stem cells into the spinal cord. The latter approach, which will require the use of human embryonic stem cells, will be tested at UC San Diego where Dr. Marsala has received additional funding from the California Stem Cell Initiative for his work on spinal cord injury.
"It's hard to imagine a more devastating disease than ALS," says Mr. Taubman, "and we have some of the highest incidence rates in the country right here in Michigan. Dr. Feldman and her team are doing miraculous work, and it's important that they have the resources to build on their momentum. I'm not a doctor or a scientist, but I am an optimist who believes in the extraordinary possibilities of modern medicine. This is important work that must continue."
Dr. Feldman calls the gift a major boost to research. "Mr. Taubman's generous funding allows us to venture into exciting new territory with stem cells. It gives our patients great hope that our new research with our California colleagues will translate the promise of stem cell technology into the reality of therapy for ALS patients."
Dr. Marsala, who hosted Dr. Feldman recently for several weeks of collaborative research, is an expert on grafting new cells into the spinal cord, and has studied the use of stem cells to treat spinal injuries caused by interruptions in blood flow.
The use of this technique in ALS, which involves the death of the motor neurons that send signals to the muscles and control movement, could be a new frontier in treating the disease.
Already, Marsala and Feldman have performed preliminary research in animals. The promising results from this study, which have not yet been published, form the basis for one prong of the attack on ALS that will be supported by Mr. Taubman's generous gifts.
If additional laboratory work proves successful, a clinical trial in ALS patients could begin within five years.
The other prongs of the attack involve two other laboratory-based approaches that could also lead to clinical trials. The first will develop a method to help ALS-affected nerve cells generate more of the molecules called growth factors that might keep them healthy.
Dr. Feldman and her team have already succeeded in creating a genetic snippet that can encourage cells to increase the number of times their genetic machinery reads the genetic blueprint for those growth factors. Preliminary results show that injecting that snippet, called a zinc finger transcription factor, into cells causes more of the growth factor VEGF to be produced " and enhances nerve regeneration. Further research on this approach will be made possible by the gift.
The research team also hopes to develop another genetic tool, called a silencing RNA sequence, to keep the cells of ALS patients from making mutant proteins involved in the disease.
For more information on the U-M Program for Neurology Research & Discovery, visit http://www.med.umich.edu/pnrd. Dr. Feldman is the DeJong Professor of Neurology at UMMS and directs the department's ALS clinic, which is certified by the ALS Association.
The gifts from Mr. Taubman are part of the University-wide $2.5 billion Michigan Difference campaign.
Background information on Amyotrophic Lateral Sclerosis or Lou Gehrig's disease:
Called a neurodegenerative disease because it causes nerve cells to weaken and die, ALS attacks a specific kind of nerve cell called a motor neuron " the type of cell that connects muscles to the spinal cord and brain, and transmits signals that enable us to move.
As motor neurons in the upper and lower parts of the body begin to be attacked, patients may experience painless weakness in their hands, feet, arms or legs. They may also begin to have trouble walking, speaking or swallowing. Over time, the degeneration continues " robbing each patient of the ability to move, speak and function on his or her own, and ultimately killing each patient within months or years.
ALS has its roots in genetic mutations, but many have not yet been discovered. A small minority of ALS cases are inherited by members of families affected by gene mutations. But the vast majority of the approximately 30,000 Americans who currently have the disease have a spontaneous form that can strike anyone, anywhere. Treatments have improved in recent years, but ALS still challenges doctors and scientists as one of the most baffling and tragic diseases. Only through research such as that being performed at U-M and UCSD will new options arise.