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Foundation Supporting ALS Progression Model Study

Brain synapses illustration

(AllanAjifo, Wikimedia Commons)

31 March 2016. A study to validate computer models that predict progression and survival of patients with ALS is underway by a bioinformatics company and biopharmaceutical developer. The 3-year project is funded by a grant of nearly $500,000 from ALS Association in Washington, D.C.

ALS, or amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease, is a progressive neurological disease that attacks the nerve cells controlling voluntary muscles, such as those in the arms, legs, and face. In ALS, muscles gradually weaken and waste away, leading to individuals losing their strength and their ability to move their arms, legs, and body. When diaphragm and chest wall muscles fail, people lose the ability to breathe without ventilatory support, often leading to death from respiratory failure.

ALS patients experience varying degrees of progression of the disease, with some patients suffering complete paralysis in as few as two years, while for others the disease progression is much slower. In the early stages of the disease, it is difficult to determine whether a given patient will experience slow or fast disease progression. The ability to predict disease progression is also critical for those interested in planning ALS clinical trials for potential new treatments.

ALS Association sponsors open competitions to develop better models for predicting progression of the disease, which provide access to the Pooled Resources Open-Access Clinical Trial or PRO-ACT database that contains records from more than 8,500 ALS patients taking part in clinical studies. One of those challenge winners is David Ennist, now chief scientist at Origent Data Sciences, in Vienna, Virginia. Origent is a bioinformatics company that develops predictive models for neurological conditions, including for individual patients.

The ALS Association grant funds work by Ennist and colleagues at Origent to validate predictive models for progression of ALS built from the PRO-ACT database, with data from a clinical trial of the drug tirasemtiv, created by Cytokinetics Inc. in South San Francisco. Cytokinetics is a developer of therapies for disorders involving skeletal muscles, such as those in the respiratory system affected by ALS. Tirasemtiv is a small-molecule activator of troponin proteins that regulate calcium in skeletal and heart muscle contractions.

As reported on Science & Enterprise in July 2015, ALS Association is supporting a late-stage clinical trial by Cytokinetics testing tirasemtiv as a treatment for people with ALS to slow the decline of respiratory function. While the trial is still underway, data from the study and earlier trials will be made available to Origent to prospectively validate its ALS progression models. Those models are expected to make it possible for clinical trials of ALS treatments to require fewer patients and take less time.

“If validated,” says Ennist in a joint statement, “the models we have developed will be submitted to the FDA and may enable more nimble, cost-effective execution of ALS clinical trials, resulting in a potentially quicker path to new medicines.”

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