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White paper: Advancing drug discovery for neurodegenerative diseases

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By Noelia Munoz-Martin, PhD

How iPSC-derived models are advancing drug discovery for neurodegenerative diseases

The incidence of neurodegenerative diseases has been rising dramatically due to increased life expectancy and an aging population. However, treatments that are currently available don't treat the root cause of the disease, but only attempt to alleviate symptoms or slow disease progression.

The FDA estimates that for every new drug approved for neurodegenerative diseases, approximately 40 have failed in clinical trials, of which many were initially seen as highly promising in preclinical settings. Under these circumstances, there seems to be a clear need to explore alternative approaches to better facilitate target identification and advance therapeutics with higher confidence throughout the pipeline. This white paper reviews the progression in drug discovery and development for Alzheimer’s disease, Parkinson’s disease and Amyotrophic Lateral Sclerosis (ALS), focusing on promising candidates identified with the help of iPSC technology.

Highlights of this white paper:

  • The complexity and epidemiology of neurodegenerative diseases demands the incorporation of alternative approaches for the development of disease-modifying therapies.

  • The incorporation of iPSC-derived models into therapeutic development for neurodegenerative diseases can increase the confidence on drug-response predictions and facilitate decision-making.

  • Several promising lead candidates identified with the use of iPSC technology are currently being investigated in clinical trials for Alzheimer’s disease, Parkinson’s disease and ALS.

Advancing drug discovery for neurodegenerative diseases

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