SAN FRANCISCO. Calif. - This year, more than 5,600 people in the United States will be told they have ALS. Within five years, many of those people will be robbed of their ability to work, to walk, to even talk. Until one day, they won’t even be able to breathe. Yet, doctors have found a possible new hope to slow the effects of this devastating disease.
ALS patient Mary Pat Murray told Ivanhoe, “I can carry on a conversation. I can eat. I can drink. I can have a normal (life), as normal as my life is now.”
However, Mary Pat Murray knew what ALS would eventually do to her.
Also known as Lou Gehrig’s disease, ALS destroys the nerve cells, eventually attacking every muscle in the body.
Neuroscientist and Neurologist at Washington University School of Medicine in St. Louis, Azad Bonni, MD, PhD, FRCPC, explained, “It hits people in the prime of their careers, the prime of their lives. So, it’s quite devastating.”
As of 2015, there was only one drug, Riluzole, approved to treat ALS, but Dr. Bonni said it was ineffective. His research suggests that a heart drug may slow the destruction of nerve cells in ALS patients.
“We have found one of the targets that may be important in the disease, is an enzyme that actually has been used as a target for drugs in heart disease,” said Dr. Bonni.
In ALS, cells that support nerve cells are more active than they should be and actually cause nerve damage. Studies in Bonni’s lab showed the drug Digoxin target the support cells, slowing them down. Mice in his lab lived 20 more healthy days, it doesn’t seem like a lot, but in human time, it is!
Dr. Bonni said, “We’re looking for ways to not only extend life, in these types of diseases but also improve the quality of life.”
Murray knew it might be too late for her, but with each new drug, came new hope for other people not yet diagnosed.
Drugs that target support cells may turn out not only to be beneficial for ALS patients but also those suffering from other neurodegenerative diseases such as Alzheimer’s, Huntington’s, and Parkinson’s diseases, but researchers stress more testing will need to be done before such drugs can be tried in patients. Source
BACKGROUND: ALS, or amyotrophic lateral sclerosis, was first discovered by a French neurologist Jean-Martin Charcot in 1869. It is a neurological disease that rapidly progresses and kills motor neurons, causing muscle weakness and atrophy. Commonly referred to as Lou Gehrig’s disease, there are over 12,000 people in the United States diagnosed with the disease and it is one of the most common neuromuscular diseases around the world. Many ALS patients die from respiratory failure due to their diaphragm collapsing within three to five years from the onset of symptoms. There are two types of ALS, sporadic and familial (or genetic). Only five to 10 percent of ALS cases are inherited, while 90 to 95 percent develop the disease with no known associated risk factors. Source
TREATMENTS: Currently, the only medication approved by the FDA for ALS is Riluzole, a medication that can alter the progression of the disease introduced in 1995. There are a few side effects associated with Riluzole, including dizziness and risk of liver damage, but doctors hope that future research will develop better medications or find a combination that reduces the effects. Physical therapy is also used to help with pain and mobility as well as improving a patient’s quality of life by giving them the independence to move around. Additional support and care is a necessity for ALS patients. Speech therapists, nutritionists and caregivers work in tandem to help patients who have muscle atrophy preventing them from eating or speaking properly. ALS patients retain their cognitive abilities, despite the complications from the condition, and can become depressed or anxious about their disease, so a solid support system is essential in treating ALS. Source
NEW TECHNOLOGY: Researchers at Washington University School of Medicine in St. Louis have been testing a medication called Digoxin, a medication typically used in heart patients, in the treatment of ALS. Led by Azad Bonni, MD, PhD, a Neuroscientist and Neurologist at Washington University, the study used mice models with a mutated gene that caused symptoms resembling ALS, including paralysis. Doctors found that a protein located in nervous system cells called astrocytes, sodium-potassium ATPase, was in high quantities in the ALS mice. Dr. Bonni and his team treated the mice with Digoxin, which works by inhibiting the sodium-potassium ATPase. Mice treated with the drug lived on average 15 percent longer and were healthier. (Source: Azad Bonni, MD, PhD. Source
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