A2A3 aims to donate funds to research to be used directly and immediately for cure-driven research. The A2A3 Board receives guidance from research experts as well as reports from current recipients regarding the use of donated funds for past and future research work in order to select recipients and allocate funds. Current recipients of A2A3 donations to ALS research are described below.
- ALS Therapy Development Institute (ALS TDI)
ALS TDI is a nonprofit biotechnology company started in 1998 by James Heywood when his 29-year old brother Stephen was diagnosed with ALS. Starting in the basement of Jamie’s parents, ALS TDI has grown into the world’s largest research and development program focused exclusively on ALS. Thirty leading scientists and technicians work in a state-of-the-art laboratory in Cambridge Massachusetts. They run an open research program, sharing their research findings on a daily basis via the web with patients, doctors and the research community. Their goal is to put themselves out of business as soon as possible by finding – or helping others to find – a treatment and cure for ALS.2011 Update from ALS-TDI:
This year so far, the ALS Therapy Development Institute (ALS TDI) has added five entirely new potential therapeutics to its pipeline. It has also advanced 12 other potential treatments forward in the drug screening
and discovery process. All of this information is provided in real-time on the Institute’s website at www.als.net/ourresearch. These updates demonstrate the methodical and relentless progress made by the ALS
TDI research team in testing potential therapeutics against numerous biological pathways that likely play a role in ALS disease progression. In addition to advancing therapeutic projects, the Institute has validated a
potentially crucial new tool in the battle against ALS; a viral vector which targets muscle tissue specifically. This will accelerate the study of potential treatments that aim to target muscle pathology in ALS.
Thanks to donations from groups like A2A3, ALS TDI was able to purchase a brand-new and important piece of equipment in its lab – a “FACS” machine, which uses florescence to quantify the numbers of different cell types in a given sample. This crucial piece of equipment, installed in the lab in January, has already yielded direct results for the research team and has become an important tool used every day. In addition, the Institute, has also developed a novel and innovative constant monitoring device used to augment the current measures used to track progression of disease in the animal. The project, developed in partnership with the husband of an ALS patient, used infrared heat sensing technology to deliver highly sensitive information on the movement changes of mice being treated with highly experimental therapeutics. Such monitoring helps scientists to identify potentially subtle changes in movement associated with disease treatments which may otherwise go unnoticed by the naked eye.
Our work would not be possible without the commitment and support of the A2A3 members, volunteers and supporters. Every single event and every single dollar accelerates the pace of research in our mission to discover a treatment to slow/stop ALS. Our work is a collaboration of hope and science. The A2A3’s passionate outreach activities provide HOPE to the ALS community and make the SCIENCE possible.
- University of Michigan’s Program for Neurology Research and Discovery
In 2000, a group of neurologists and neuroscientists came together to form what is now known as the Program for Neurology Research and Discovery. Under the direction of Eva L. Feldman, M.D., Ph.D., Russell N. DeJong Professor of Neurology, the group has undertaken research in some of the most complex and challenging fields of disease. From ALS (Lou Gehrig’s disease) to the nerve damage that disables people with diabetes, they have made medical discoveries that are bringing treatments to patients. With a combination of mind power and technical resources found in few other places, the Program for Neurology Research and Discovery is leading the way in translating laboratory discoveries into new patient therapies.2011 Update from U of M:
Dr. Eva Feldman has been treating patients with ALS and hunting for a cure for this terrible disease her entire medical career. She knows the heartache of having to tell patients and their loved ones that no effective treatment exists.
Now, there may finally be hope.
The director of both the A. Alfred Taubman Medical Research Institute and the Program for Neurology Research & Discovery at the University of Michigan, Dr. Feldman is currently leading the first human clinical trial of a stem cell treatment for ALS. This potential breakthrough is the product of years of painstaking labatory study and represents the first time that stem cells have been injected into the human spinal cord.
“I have seen hundreds of patients with ALS over the past 20 years, says Dr. Feldman. “Until now, I could not offer them much in the way of hope. Now, for the first time I can. That is a wonderful thing.”
The trial is still in Phase 1, which tests the safety of the procedure. So far, 11 ALS patients have undergone this revolutionary therapy, with no serious side effects. Feldman hopes to begin Phase 2 of the trial, which begins to evaluate the effectiveness of the treatment, within two years.
At the same time, her team of scientists is working to improve the ability of the stem cells to deliver ife-preserving proteins to the nerve cells under attack in ALS. They are also adapting this stem cell model to the treatment of other nerve disorders, including Alzheimer’s disease. In addition, they are working to establish induced pluripotent stem cell lines from the skin cells of ALS patients. Among other potential uses, at some point in the future they may be injected back into the spinal cords of the donor patients, in the procedure Dr. Feldman is currently pioneering. The patients won’t need to undergo immuno-suppressant therapy since these cells come from their own tissue.
Dr. Feldman is exploring another new frontier of science, studying the role of epigenetics in the origins of ALS. She believes that environmental toxins may produce epigenetic modifications – factors that cause genes to express themselves in nonstandard ways – that may give rise to the onset of the disease in certain cases. Her laboratory has discovered distinct epigenetic signatures in postmortem tissues from the spinal cords of ALS patients.
Her team is working with physicians across the state to conduct environmental exposure surveys of ALS patients throughout Michigan. These same patients are donating blood for the identification of epigenetic signatures that correlate with their environmental exposures. This research has the possibility to revolutionize our scientific and clinical approach to ALS by providing new insights into the origins of ALS, new potential treatments and new biomarkers that will help doctors diagnose the disease earlier.