ALS - Amyothropic Lateral Sclerosis
Every 90 minutes someone is diagnosed with amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disorder. Each case is different, and the community of people with ALS and the care providers involved with ALS TDI share their experiences to help those newly diagnosed and their families. Advancements in ALS research have provided significant new insights into ALS, how to stratify those diagnosed, and develop effective treatments and cures.
ALS, also known as Lou Gehrig's disease, Charcot's disease, and motor neuron disease (MND), attacks certain cells in the brain and spinal cord needed to keep our muscles moving. Early signs and symptoms of ALS include:
- muscle cramps and muscle twitching
- weakness in hands, legs, feet or ankles
- difficulty speaking or swallowing
The senses, including hearing, sight, smell, taste, and touch, are not affected by ALS.
There is no single diagnostic test for ALS. However, experts in the disease, usually neurologists specializing in neuromuscular diseases, are very capable of diagnosing ALS. In some cases, they might order additional tests if the diagnosis is not clear. These include:
- electromyography and nerve conduction
- magnetic resonance imaging (MRI)
- genetic tests
- muscle biopsy
- spinal tap
- blood and urine tests
Most people with ALS live 2-5 years after their first signs of disease. About 10% of people with ALS survive at least 10 years. This variable rate of disease progression makes prognosis difficult to predict and therapies challenging to develop.
Currently, there are two medications that have been approved by the FDA as treatments for ALS; Riluzole and Radicava. People diagnosed with ALS are encouraged to speak with their doctors about these approaches. While neither were found in clinical trials to be broadly disease modifying (stopping disease) in all people with ALS, many people taking them do experience some impact on their disease progression (which can be monitored in part through participation in ALS TDI's Precision Medicine Program).
This urgent unmet medical need for effective treatments for this devastating and fatal disease is the basis for the research and drug development effort at the nonprofit biotech organization, the ALS Therapy Development Institute.