Breakthrough ALS

For over a decade, researchers and scientists have been relying on the ALS Therapy Alliance’s expertise and funding to advance their studies of amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease. 2015 marks the 14th year of our annual Breakthrough ALS fundraising campaign (formerly known as Researching a Cure).

The ALS Therapy Alliance’s ongoing grant award process is overseen by the organization’s board of award-winning researchers and scientists, as well as corporate executives and individuals who strive to learn more about the neurodegenerative disease, its cause and possible cure.

The organization has raised more than $30 million over the past 13 years thanks to its partnership with CVS/pharmacy.

The ALS Therapy Alliance is designated by the IRS as a not-for-profit 501 (c) (3) organization.

Points of Interest

The ALS Therapy Alliance (ATA) was established in 2000 to facilitate ALS research projects and collaborations among a diverse group of scientists and clinicians at multiple institutions to cure ALS. This represents a unique collaborative enterprise that spans multiple laboratories, universities and disciplines.

The ALS Therapy Alliance is fortunate to have a Board of Directors comprised of talented clinicians, scientists and business associates who are committed to the organization’s mission.

Ongoing research is being conducted in the fight against amyotrophic lateral sclerosis (ALS) by our team of clinicians, doctors and scientists at top hospitals and universities.

There are many ways you can help support the research being conducted by the ALS Therapy Alliance to find a cure for amyotrophic lateral sclerosis (ALS), otherwise known as Lou Gehrig’s disease.

What is ALS?

Amyotrophic lateral sclerosis (ALS) is an adult-onset disease that usually begins between the ages of 40 and 70. Men and women of all ethnic and racial groups are generally affected equally, but the cause is unknown.

There is no single diagnostic test for ALS, and as a result, the diagnosis is often delayed. Several tests must be conducted to exclude the possibility of other similar, but treatable, diseases.

ALS: what it is and what the name really means

ALS is a rapidly progressing neurological disorder in which most motor neurons in the spinal cord and brain degenerate, typically over three to five years. When the motor nerve cells degenerate, many types of voluntary movement are impaired and ultimately lost.

The name amyotrophic lateral sclerosis is Greek in origin. A means “no” or “negative,” myo refers to muscle, and trophic stands for nourishment. So, amyotrophic means “no muscle nourishment.” Lateral sclerosis refers to the fact that the sides of the spinal cord appear scarred, or sclerosed, in late-stage ALS.

ALS: the initial onset and symptoms

Initially, symptoms of ALS may include isolated muscle weakness, muscle twitching, cramping and stiffness of muscles, unusual fatigue and clumsiness, or difficulty swallowing and speaking.

Although the sequence of emerging symptoms and the progression rate for the disease differ from person to person, most muscle in a person with ALS will ultimately weaken and become paralyzed.

Many functions other than movement are spared in ALS. For example, a patient’s thinking ability, bladder and bowel function, sexual function, sight, hearing, smell, taste and touch are unaffected. When the muscles in the diaphragm and chest wall fail, patients cannot breathe and most will die from respiratory failure, usually three to five years after the symptoms begin. Fortunately, there are excellent respiratory support systems to forestall this problem.

ALS: the real cause remains a mystery

The cause of most cases of ALS is unknown. About 10% of cases run in families, and in some of these cases there are defined genetic defects that are known to trigger the disease. For example, some familial cases arise because of mutations in a gene that makes a free radical absorbing protein, known as superoxide dismutase (SOD1). Other cases arise because of mutations in genes (called FUS/TLS and TDP43) that make proteins that bind to a genetic material in cells known as RNA.

Another gene implicated in familial ALS makes a protein called angiogenin. The causes of non-familial ALS are not defined. We do know that in both familial and non-familial ALS, several factors seem to play a role in motor neuron degeneration, including high levels of the neurotransmitter glutamate, exposure to adverse environmental factors such as infections or poisons, insufficient energy generation by brain cells, inflammation in the spinal cord, and slowing of the transport of substances in long neuron processes known as axons.

ALS: new treatments are being developed

There is no cure for ALS. A single drug called riluzole is approved for use in ALS, but at best it modestly slows the disease. ALS Therapy Alliance researchers are dedicated to discovering new treatments for ALS. Several experimental drugs are in trials and in development in ALS research laboratories.

Though they are not ALS specific, and there are no therapies that slow the basic process of degeneration of motor nerves, other treatments, such as feeding and breathing aids, relieve some of the symptoms of ALS. In addition, some drugs are helpful for problems like fatigue, muscle cramps, depression and sleep disturbance.