Q) I haven’t heard much about ALS since the ice bucket challenge went viral. Did all the money that was raised lead to any new treatments?
A) Every non-profit organization dreams of staging a fund raiser like the ice bucket challenge that leveraged various social media platforms back in July and August of 2014 to go viral.
In time, more than 17 million people posted videos online, including such dignitaries as former President George W. Bush and billionaire Bill Gates, to raise over $115 million in as little as two years. This campaign was a god-send as amyotrophic lateral sclerosis (better known as ALS or Lou Gehrig’s disease after the legendary New York Yankee who passed away from this disease in 1941) is one of the most devastating diseases we deal with today.
It also far more common than most people suspect, an unfortunate consequence of the fact that it is usually so aggressive most do not live long after diagnosis. ALS is a progressive neuro-degenerative disease that attacks cells in the brain and spinal cord that control our voluntary muscle movements such as moving our fingers, talking or chewing our food. As the impulses from our nervous system fade, the muscles begin to weaken then twitch before eventually wasting away until a point is reached where the patient, no longer to eat, speak or even breathe without medical intervention, can no longer sustain life.
The number of people living with ALS in Canada is estimated to be about 2,500 to 3,000 which makes it sound like a rare disease but this is, as I mentioned earlier, more of a misperception due to the fact that it kills so quickly that most people do not know anyone living with this disease.
Its actual prevalence is estimated at about 1 in 400 people which makes it about as common as multiple sclerosis. Only one person in 10 with ALS is likely to live for more than a decade making our recently departed physicist Stephen Hawking a medical marvel in that he lasted for more than 50 years with the condition.
ALS is most commonly diagnosed in people in their 50’s and 60’s although it has been known to rarely strike younger adults (Hawking was 21). Traditionally, it is tough to diagnose because its initial symptoms are so subtle and common they are often attributed to stress, a poor diet or a lack of sleep.
Usually, the first symptoms noticed (which occurs long after the disease starts attacking the brain as we have many redundancies in our body that can compensate for the lost cells for quite some time) involve muscle weakness in the arms and legs that can lead instability or stiffness. Less commonly the muscles of the face (leading to a slurring of speech and swallowing difficulties) or trunk may be affected first.
Eventually, nearly all voluntary muscles (i.e. the muscles you consciously control) are affected. Patients usually retain control of their bladder and bowels until very late in the disorder but feeding tubes become common fairly early on and become the only option for maintaining nutrition. Fortunately, eye movement is also preserved for a long time which allows the affected to continue to communicate with the help of gaze technology via a computer long after they are no longer able to speak.
Even as their physical bodies decline, half of those affected remain mentally sharp, perhaps a dubious blessing considering the circumstances. Fortunately, while there still is not a cure or treatments that do much more than preserve life for a few months more, all the money raised by fund raisers such as the Ice Bucket Challenge have allowed researchers to greatly enhance their understanding of this disease.
While the cause or causes of ALS still remain unclear, researchers are studying the genes in patients and engineered mice in the hopes of developing drugs or stem cells that will significantly slow the disease or even stop its progression. One recent study in mice at Stanford University suggested that stopping a protein called ataxin-2 seemed to foster resistance to ALS. The mice that were treated lived for hundreds and hundreds of days as opposed to just a month in the untreated ones.
Another sign of progress was the approval of a second drug to treat ALS called Radicava (edaravone) in May of 2017. Radicava is a new type of treatment providing an alternative to the pre-existing drug known as Rilutek which was released years earlier and never made much of an impact.
Radicava is given by intravenous infusion every 2 weeks and aims to reduce a process known as oxidative stress that occurs within our cells. Most patients eventually are able to receive their infusions in the comfort of their own homes. The drug has not been a massive break-through (a shame since it costs more than $140,000 US a year) but some patients do tend to see significant improvements in their quality of life as long as they start on the drug early enough (disease duration less than 2 years) Not great news, but progress nonetheless which hopefully will spur future treatments that will have even greater impacts on the course of this disease in the future.
For more information about this or any other health care related question, contact your pharmacist.