We recently announced a computational Challenge, the ALS Prediction Prize Challenge, with Prize4Life to better predict the progression of disease in ALS patients. Having a better understanding of how this deadly neurological disease, also known as Lou Gehrig’s disease, progresses could lead to breakthroughs in treatment and quality of life for patients. We recently spoke with Dr. Melanie Leitner, Chief Scientific Officer for Prize4Life, about the Challenge.
Hello Dr. Leitner. First of all, can you tell us a bit about ALS, who it typically strikes, and its impact on patients?
Amyotrophic Lateral Sclerosis, also known as Lou Gehrig’s disease in the US and Motor Neuron Disease in other parts of the world, is a progressive fatal neurodegenerative illness that attacks motor neurons. When motor neurons die, the ability of the brain to control muscle movement is lost, leading to paralysis. Unable to function, muscles atrophy. Eventually, all muscles under voluntary control are affected and patients in time lose their ability to walk, talk, swallow, and breathe. During this terrible process, the mind typically remains intact, observing the loss of each function. When the diaphragm and chest muscles fail, patients stop breathing on their own. The majority of people with ALS die of respiratory failure within 2 to 5 years of noticing the symptoms.
The ALS Association estimates that there are about 30,000 people living in the US today with ALS. Ironically, ALS strikes as many or more people than some other better known neurodegenerative diseases, such as Huntington’s Disease or Multiple Sclerosis, but because ALS is so rapidly fatal, at any given time there are fewer people living with ALS.
People of all races and ethnic backgrounds are susceptible to ALS, which typically strikes people between the ages of 40 and 70, but it is known to affect younger and older people as well. The disease occurs slightly more often in men than women.
This Challenge is focused on developing accurate and predictive indicators for disease progression. Why is this so important?
The question of accurately predicting disease progression is important for two key reasons. First, as an ALS patient or caregiver of a patient, it is frustrating that, unlike other diseases, when you’re diagnosed with ALS there is currently no way of knowing at the outset if you’re going to be like Lou Gehrig and have a very rapid course to your disease or if you’re going to be one of the rare individuals like Stephen Hawking who live more than 10 years with the disease.
Second, being able to accurately predict disease progression would make it easier to design and conduct effective ALS clinical trials. Right now, because researchers can’t tell whether a given ALS patient will live for two, five, or ten years, clinical trials require observing a large number of patients over a long period of time to determine whether or not a potential new therapy is effective.
Are there no current predictors of ALS disease progression? Read more