Via Scoop.it – ALS Lou Gehrig’s Disease
In people with ALS, the motor nerves deteriorate leading to muscle weakness and ultimately paralysis. In hopes to stop this neurodegeneration in its tracks, researchers are looking towards substances called neutrophins including BDNF, GDNF, IGF-1 and VEGF to keep nerves healthy and plugged into muscles. But delivering these protective substances safely at the right place, at the right time and at the right dose has turned out to be extremely difficult to do. And, scientists remain unsure which one of these neurotrophins is the best choice for people with ALS to protect the motor nerves from further deterioration. Studies suggest that moderate aerobic exercise such as stationary bicycling or treadmilling might have the potential to help keep muscles and nerves healthy longer in people with ALS by increasing levels of many of these protective substances in the brain and spinal cord. What’s more, a moderate workout might even help fight the disease by boosting energy supplies, removing damaged proteins and reducing inflammation. Neurologists nevertheless remain reluctant to recommend specific exercise routines for their patients. There simply is not enough clinical evidence according to experts to indicate which routines are safe and offer the most benefit to people with ALS. New clinical trials promise to change that by putting exercise to the test in people with ALS.
It was a meeting of the minds. On March 8th, 9th, and 10th, Packard Center grantees gathered at the Baltimore Marriott Waterfront to present and discuss their research findings from the past year. Unlike previous years, the symposium’s format integrated bench science with clinical research findings. As Packard Science Director Piera Pasinelli noted at the start of the first day, lab results and clinical trials inform each other. When each perspective learns from the other, Pasinelli said, scientists will move more quickly towards finding promising therapeutic targets to develop into drugs for ALS. “The goal of Packard has always been to bring new discoveries forward and closer to the clinic as quickly as possible,” said Pasinelli. “By encouraging these partnerships and exchange of ideas between clinicians and bench scientists, we want to take the most promising scientific ideas to the attention of the clinicians faster. Because basic and clinical science inform each other, this exchange is also beneficial to refine strategies in the lab and in the clinic.” Perhaps the most significant breakthrough in ALS science in the past year has been the discovery of the repeat expansion on chromosome 9. Known as C9ORF72, it was co-discovered by Packard Scientist Bryan Traynor and is the most common genetic cause of familial ALS in North America. The identification of hundreds of copies of a repeating six DNA base pair sequence was a milestone, but like many important scientific discoveries, it raised more questions than it answered.
A $25 million gift has enabled Johns Hopkins to establish a new center to develop novel therapies for the neurodegenerative disease known as amyotrophic lateral sclerosis, Lou Gehrig’s disease, or ALS. Much of the center’s research will focus on using stem cells individually derived from ALS patients to develop new model systems to investigate how nerve cells degenerate, as tools to screen new drug therapies, and to develop stem cell therapies as transplants to potentially slow or reverse the disease. The new center, dedicated March 21 and formally known as the Michael S. and Karen G. Ansari ALS Center for Cell Therapy and Regeneration Research at Johns Hopkins, is named for its benefactors: Michael and Karen Ansari. Michael Ansari is the founder, chairman and CEO of M.I.C. Industries. The gift, representing a five-year commitment, will fund a variety of efforts that aim to eventually cure ALS. The disease targets motor neurons, a type of nerve cell that controls muscle movement, and affects about three out of every 100,000 individuals. “Despite knowing about this disease for decades and the large number of clinical trials that have been completed, we still have little in our arsenal to treat it,” says Nicholas J. Maragakis, M.D., an associate professor of neurology at the Johns Hopkins University School of Medicine, co-medical director of the ALS Clinic and director of the new center. “We are now able to think out of the box about this disease. The goals of a center will focus on the use of stem cells as tools to foster aggressive programs in discovering the underlying mechanisms behind what causes ALS and rapidly translating these discoveries to the patients in our clinic.”
Prize4Life, a non-profit organization dedicated to accelerating the discovery of treatments and a cure for Amyotrophic Lateral Sclerosis (ALS), is pleased to announce the appointment of Roger D. Kornberg, PhD, to its Scientific Advisory Board. “Prize4Life is delighted to welcome such an accomplished and talented scientific mind,” said Avi Kremer, co-founder and Chief Executive Officer of Prize4Life. “Roger Kornberg is an excellent addition to our Scientific Advisory Board, which provides a high level of expert knowledge and wise counsel to Prize4Life and is integral to the success of our important mission.” Dr. Kornberg is currently a professor of structural biology at Stanford University School of Medicine, where his lab has continued to elucidate the complex process by which DNA is unraveled, read, and “transcribed” into RNA. Although his research is not directly related to ALS, Dr. Kornberg believes “Prize4Life’s mission of finding treatments and a cure for ALS is imperative. I look forward to joining the organization’s impressive assembly of scientific advisors. Through its innovative model, Prize4Life can produce real results for ALS patients.”
From Sunday morning to Tuesday evening last week, there was a lot of talk of MND research going on in Liverpool. The reason for this ‘hotspot’ of discussions was due to the annual meeting of an international consortium of MND researchers taking place at the University of Liverpool. The 10th International Consortium on SOD1 and ALS (ICOSA) meeting took place last weekend (4 – 5 March). In 2001, five laboratories came together to form ICOSA, where the aim was to share knowledge to design better-informed experiments to understand the rare, inherited SOD1 form of MND. MND Association grantee, Prof Samar Hasnain was one of its founding members. Success of this philosophy of sharing knowledge prior to publication has resulted in several leading groups joining the effort, looking at other causes of inherited MND too. A tradition of ICOSA meetings is to hold an open meeting for sharing latest results with a wider audience, following their closed meeting. Thus, on Tuesday 6 March, an open meeting was held to allow the exchange of the latest results and ideas between ICOSA members and the UK MND research community.
A Ben-Gurion University researcher and her colleagues have identified a chemical compound that may lead to prolonged lifespans for patients with Amytotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. Prof. Esther Priel and her team published an article in the February edition of EMBO Molecular Medicine Journal, in which they demonstrate how their compound can induce an increased production of the enzyme telomerase in the bodies of laboratory mice and in human stem cells. The increased amount of telomerase slows the progression of ALS, also known as Lou Gehrig’s disease. It will take at least 10 years to develop the compounds into a drug, she predicted. In the meantime, however, the team is producing positive results in animals, she said. While ALS remains a rare disease in Israel and throughout the world, Priel stressed just how debilitating it becomes to those who have it. “There are not a lot of people, fortunately, who have this disease, but it’s a terrible disease because they become paralyzed, and the head is working very well, so they are aware of what is going on with their body,” she said. “It is a devastating disease and there is no cure for it.”
ALS Research News 