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Challenges of ALS

Amyotrophic lateral sclerosis (ALS) is a devastating and rapidly progressive neurodegenerative disease characterized primarily by motor neuron loss resulting in progressive weakness, disability, and eventually death. Disease heterogeneity and historically poor survival rates make ALS a clinically challenging disease to research and trial.1
 
ALS clinical challenges

Challenges of ALS

Amyotrophic lateral sclerosis (ALS) is a devastating and rapidly progressive neurodegenerative disease characterized primarily by motor neuron loss resulting in progressive weakness, disability, and eventually death. Disease heterogeneity and historically poor survival rates make ALS a clinically challenging disease to research and trial.1
ALS clinical challenges
Epidemiology
 

ALS Prevalence: The Most Common
Adult-Onset Motor Neuron Disorder2

ALS incidence and prevalence in the United States
An estimated 5000 people in the United States are diagnosed with ALS each year with a prevalence of at least 16,000.3
how often ALS is diagnosed
On average, a new case of ALS is diagnosed every 90 minutes.4
The relatively low prevalence is potentially understated due to the rapid progression of ALS.1

The Prevalence of ALS Increases by Age

Proportion of ALS diagnoses by age group (years)5

prevalence of ALS by age chart
ALS prevalence chart

ALS Risk Factors Among US Population5

  • Men develop ALS more frequently than women (1.56:1)
  • White, non-Hispanics are the majority of diagnoses (79%)
  • 60-69 years and 70-79 years are the most common ages of onset
  • Familial ALS (a hereditary form) occurs in 5% to 10% of cases
  • Several additional risk factors for ALS have been hypothesized, including chemical exposure, occupational exposure, military service, infectious agents, nutritional intake, physical activity, and trauma
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Survival Rates
 

ALS Prognosis: ALS Is Incurable and Can Progress Rapidly3,5

  • The majority of patients die within 2 to 5 years of the date of diagnosis
  • 50% live longer than 2 years
  • 10% live longer than 5 years
  • Rates of disease progression among ALS patients vary
  • Patients who receive a diagnosis at an earlier age have a slightly better prognosis
The variable rate of ALS progression makes disease prognosis difficult.6
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Clinical Challenges
 

ALS Continues to Pose Numerous Clinical Challenges1

Disease Rarity: ALS is a rare and rapidly progressive motor neuron disease, and, even in multicenter trials, patient enrollment can be slow. Recruitment remains an obstacle for all clinical trials.

Disease Heterogeneity: The rate of progression of ALS varies widely among patients. Some patients die within one year; others live more than a decade.

Lack of Biomarkers: To date, there remains a lack of reliable biomarkers that are exclusive to ALS for predicting disease progression and prognosis.

Numerous Outcome Measures: A multitude of outcome measures are used to provide evidence of clinically meaningful change in ALS clinical trials, such as survival time and functional status. Each of these measures has its strengths, but none are perfect. See 4 of the most common outcome measures here.
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NEXT: RETHINKING ALS
 
References: 1. Berry JD, Cudkowicz ME. New considerations in the design of clinical trials for amyotrophic lateral sclerosis. Clin Investig (Lond). 2011;1(10):1375-1389. 2. Basic Facts about MND. Motor Neurone Disease Association website. https://www.mndassociation.org/about-mnd/what-is-mnd/basic-facts-about-mnd/. Accessed May 19, 2020. 3. Who gets ALS? ALS Association Rocky Mountain Chapter website. http://webco.alsa.org/site/PageServer/?pagename=CO_1_WhoGets.html#:~:text=The incidence of ALS is two per 100,000 people, and, cases occur without family history. Accessed August 21, 2020. 4. What is ALS? ALS Therapy Development Institute website. http://www.als.net/what-is-als/. Accessed May 19, 2020. 5. Mehta P, Kaye W, Raymond J, et al. Prevalence of Amyotrophic Lateral Sclerosis — United States, 2015. MMWR Morb Mortal Wkly Rep. 2018;67:1285–1289. 6. Simon NG, Turner MR, Vucic S, et al. Quantifying disease progression in amyotrophic lateral sclerosis. Ann Neurol. 2014;76(5):643-657.