In June 2007, one year after a group of concerned and close individuals formed Bright Side of the Road Foundation, we established The Barry Winovich Endowed Chair for ALS Research at the Cleveland Clinic Foundation. Our goal was to raise $1.5 million over a five-year period.

We did this to provide researchers with a steady stream of income in order to find a cure for ALS (Amyotrophic lateral sclerosis, a.k.a “Lou Gehrig’s Disease”).

By January 2008, we reached a third of our goal –  $500,000 (five hundred thousand dollars).

The Bright Side Board of Directors presents the Cleveland Clinic Foundation with its second $250,000 check for the Barry Winovich Endowed Chair for ALS Research.

WHY DID WE CHOOSE THE CLEVELAND CLINIC?

The Bright Side of the Road selected the Cleveland Clinic for two main reasons: First, because it is recognized as a global leader in neuroscience research (#1 in Ohio and #5 in the nation. Second, the Clinic is located right here in Northeast Ohio.

WHO IS DR. ERIK PIORO?

Dr. Erik Pioro

The Cleveland Clinic selected its Director of the Clinic’s Center for ALS and Related Disorders, Dr. Erik Pioro, as the chair holder.  Dr. Pioro is an internationally renowned expert in ALS, and highly regarded as among the best and the brightest in ALS research.

In addition to many years of clinical care and research, Dr. Pioro collaborates with other respected professionals in cutting edge genetic research, at such places as the National Institute of Health, (NIH) and Johns Hopkins University. He has authored numerous articles, as well as a book on ALS research and clinical care, and frequently presents at international ALS symposiums.  Dr. Pioro’s and the Clinic’s Neurosciences Institute’s strong reputations put the Cleveland Clinic on the short list for pharmaceutical trials.

Given the Clinic’s breadth of expertise, Dr. Pioro and other ALS researchers have unfettered access to research across many other disciplines, such as neuroimaging, neurology, and diseases, such as multiple sclerosis, Alzheimer’s, and epilepsy.

WHAT ARE DR. PIORO AND THE CLEVELAND CLINIC DOING THAT COULD LEAD TO A CURE?

Since the middle of this decade, there have been major strides in genetic and therapeutic research that could lead to breakthroughs for ALS.  The Cleveland Clinic, through Dr. Pioro’s leadership and unrelenting commitment, is at the gateway of these possibilities.

We have identified the following avenues of research as ones we find both encouraging, and in need of financial support. (Please note: However encouraging some of these findings may be, at this time there exist no treatments for ALS that are proven, and there is no cure. Neither is the cause – or causes – understood. Our mission is one of great urgency.)

I.    BREAKTHROUGHS IN GENETIC RESEARCH

DNA study to identify human genes responsible for ALS

Given the mounting evidence that genetics, coupled with environmental triggers, could cause ALS, Dr. Pioro’s lab is extracting DNA from blood samples of ALS patients and conducting a gene array analysis to identify which genes cause or contribute to ALS.  The National Institute of Health is collaborating with Dr. Pioro and The Cleveland Clinic on this research.

DNA Study of Identical Twins

Taking this genetic pinpointing a step further, Dr. Pioro is also analyzing the genetic differences between two identical 45-year-old male twins, only one of whom has ALS.  The research here is to “fine map” their genetics and pinpoint differences that may have led to the development of ALS in one but not the other.

Genetic findings in Wobbler Mouse Model

In addition to the human studies, Dr. Pioro’s lab is using a unique mouse model — the “Wobbler” mouse — for studying the genetics of ALS, in contrast to many other labs, which have used a genetically engineered SOD1  mouse model.

Recent studies are showing that the SOD1 mouse model is not as effective, because certain genes that are pinpointed in human ALS are not present in the SOD1 mouse, whereas they are present in the Wobbler mouse.  Dr. Pioro’s Wobbler mouse research has identified certain “neuroprotective” effects of the Wlds  gene that may play a significant role in ALS.

This finding is of great interest to the ALS research community. Pinpointing this gene’s role in protecting motor neurons can lead to the development of treatments that can basically mimic this gene. Also, because the Wobbler mouse is so genetically similar to human beings, studies have identified other genes and mutations that play a role in human ALS.

Major Discovery of Possible Causal Link: “TDP-43” Gene

In 2008, neuroscience researchers found mutations of the TDP-43 protein in humans with ALS.

It is possible that this is not only present but is a cause of ALS. The abnormality of this protein is present in the Wobbler, but not the SOD1 or any other mouse model.

Coupled with this and other findings over the past several years, and by using this mouse model, the Cleveland Clinic’s genetic research is at the forefront of a breakthrough in understanding this deeply mysterious disease. As the causation puzzle unfolds, the Cleveland Clinic is well positioned to explore relevant treatments, and eventually, a cure, for ALS.

II.    NEUROIMAGING “CUTTING EDGE” TECHNOLOGY

In conjunction with the Cleveland Clinic’s Departments of Radiology, Neuroradiology, and Nuclear Medicine, Dr. Pioro’s clinical research lab has used the Clinic’s high- powered MRI imaging technology to identify and track brain regions where neurons begin to degenerate. This will enhance the Clinic’s ability to track the disease progression.  It not only enables researchers to detect ALS earlier, but to identify possible “pathways” from the brain to upper motor neurons that could be targeted for drugs to stop the dysfunction.  The evolving capabilities in neuroimaging, when combined with both genetic and drug research, place the Cleveland Clinic at the forefront of neurological research.

III.    CLINICAL DRUG TRIALS

Because of the Cleveland Clinic’s influence in the medical, research and pharmaceutical communities, and as a leading clinical research center, it has complete access to the most up-to-date drug trials and research for ALS, as well as other neurological diseases.  Of note this past year are the following drugs that are targeted for trials and are being used by patients under the FDA’s “compassionate use” policy:

• Arimoclomol – to be tested (Phase II-B) in the fall of 2008
• Ceftriaxone – an antibiotic that has been found to be neuroprotective
• Lithium – in conjunction with the only FDA approved drug for ALS (rilutec), a recent study in Italy of 44 ALS patients showed that it can reverse symptoms
• Baclofen – has shown to reverse symptoms when injected into spine

Note: Chelation is also being followed for certain patients that have sought treatment from other centers.  Chelation is a technique by which certain metals that are found to be elevated in ALS patients are reduced in the bloodstream through extraction.

CONCLUSION

The principal monies of the Barry Winovich Endowed Chair for ALS Research fund will remain intact. This will enable the fund to generate a permanent source of annual income for the chair holder, Dr. Pioro. We built in flexibility, so that some income can be paid out each year to the chair holder before we reach our goal. Not only will this income keep the research ongoing, but it will also provide seed money for projects that could lead to NIH grants.  As part of our stewardship, Bright Side of the Road Foundation will participate in the Clinic’s annual review of the chair holder’s progress in meeting established goals.

We believe that through our commitment and momentum, we are uniquely positioned to unite supporters of fighting ALS in Northeast Ohio, in order to bring us closer to a cure.  We hope that by 2009, we will reach more than 50% of our goal.

Our approach allows us:

1. To provide seed money for projects that can lead to NIH grants;
2. To be responsible stewards of the funding by participating in The Cleveland Clinic’s review of the chair holder’s progress in meeting established goals; and
3. To bring together people who are determined to find treatments and a cure for ALS throughout Northeast Ohio and beyond.

We ask you to please join us in making this hope of finding a cure for ALS a reality.