Written by Miranda Helus
When most people hear about amyotrophic lateral sclerosis (ALS), they probably will remember the Ice Bucket Challenge, the viral campaign that took the Internet by storm in the summer of 2014 and was largely responsible for reviving public recognition of ALS. Launched by the ALS Association to promote awareness and raise funding for research, individuals posted videos of themselves pouring ice water over their heads on social media and donated to the organization. Within a month of its initial launch, the ALS Association received $22.9 million in donations [1]. Before the Ice Bucket Challenge, ALS was not a publicly well-known disease, despite official reports that more than 6,000 Americans are diagnosed with ALS annually [2].
ALS is a degenerative neurological disorder in which the motor neurons that control voluntary muscle movement deteriorate, resulting in muscle weakness and loss of control over voluntary muscle movement [3]. Early symptoms include muscle spasms, twitches, and muscular spasticity and atrophy; as ALS progresses, individuals experience difficulties in movement, chewing or swallowing, speaking, and breathing [3]. Serious and even life-threatening complications arise such as malnutrition and weight loss, muscle paralysis, dementia, increased risk of pneumonia, neuropathy, and respiratory failure [3]. Most ALS deaths occur within three to five years from the onset of symptoms, although about 10% of ALS patients survive for at least ten years [3].
Although no cause has been identified, common risk factors of the disease are suggested to be genetic mutations or environmental factors such as exposure to certain toxins [3]. Other risk factors include age (elderly individuals between ages 55 to 75 are at the highest risk), gender (men have a slightly higher risk), and race or ethnicity (ALS is more likely to develop in non-Hispanics and Caucasians) [3]. Interestingly, in about 90% of ALS cases, individuals develop ALS despite not having any clear risk factors or family history of the disease; only about five to ten percent of cases are familial [3].
Unfortunately, no cure has been developed for ALS. However, treatments such as medications, speech and physical therapy, and nutritional and breathing support can help to manage symptoms and improve daily life [3]. Only two medications have been approved to directly treat ALS: riluzole and edaravone. Riluzole decreases glutamate levels to reduce motor neuron damage and slow the progression of ALS [3], but observed side effects include nausea, vomiting, bladder problems, and flu-like symptoms [4]. In May 2017, the FDA approved edaravone, an intravenous drug that has been shown to lower the rate of functional decline in ALS patients by one-third [2]. Research projects have also gone underway such as the Genomic Translation for ALS Clinical Care (GTAC), a collaborative effort to better understand how genetics affects clinical symptoms and funded using donations from the Ice Bucket Challenge [5].
Even though progress is slow, significant steps have been made towards developing more effective treatments, especially in recent years. Who would have known that a bucket of ice could have a large impact on the healthcare community?
References:
[1] Eder K. 2014. ALS Receives Recognition from CDC Report, Ice Bucket Challenge. Pharmacy Times.
[2] Grover N. 2017. FDA Approves ALS Treatment for the First Time in More Than 2 Decades. TIME.
[3] 2013. Amyotrophic Lateral Sclerosis (ALS) Fact Sheet. National Institute of Neurological Disorders and Stroke.
[4] Vermes K. 2016. Biohaven Receives Orphan Drug Designation for ALS Treatment. Pharmacy Times.
[5] 2015. Biogen, the ALS Association and Columbia University Medical Center Collaborate to Drive Understanding of Genetic Influence in ALS. Pharmacy Times.