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Aichi Medical University and Toray Innovate Basic Technique to Assess Efficacy of Novel Drug Candidates to Treat ALS

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May 29, 2023

Aichi Medical University
and Toray Industries, Inc.


Tokyo, Japan, May 29, 2023 – Aichi Medical University and Toray Industries, Inc., announced today that they have created a basic technique to evaluate the efficacy of novel drug candidates to treat amyotrophic lateral sclerosis (ALS). This advance stems from their joint research into treating this illness. 

The new technique should enable very precise ALS drug therapy efficacy assessments and predictions, enhancing and accelerating drug discovery R&D.

Also known as Lou Gehrig’s Disease, ALS is an intractable disease that damages motor neurons and causes generalized muscle atrophy and weakness. The average life expectancy after onset is three to five years. There are around 10,000 sufferers in Japan (see note 1).

The disease progression patterns among individuals and pathologies are diverse. The resulting difficulty in creating experimental models (note 2) for diverse pathological conditions has accordingly hampered ALS drug discovery.

Analysis of data from the Japanese Consortium for Amyotrophic Lateral Sclerosis Research (note 3), which collects patient clinical and genetic information, reveals four disease progression pattern groups and the proportions of sufferers in them (note 4). 

To establish their new technique, Aichi Medical University and Toray efficiently produced motor neurons derived from induced pluripotent stem (iPS) cells (note 5) from 30 patients according to their representations in the four groups. As well as enabling novel drug candidate assessments and predictions for patient pathological patterns, their technique should lead to new ALS therapies offering efficacy for specific patient groups.

Aichi Medical University and Toray collaborated under a Japan Agency for Medical Research and Development-led project called GAPFREE (for Funding for research to expedite effective drug discovery by Government, Academia and Private partnership) (note 6). 

The two partners will enhance their technique and leverage in driving drug discovery research advances to provide efficacious ALS medications as swiftly as possible to assist patients, their families, and other stakeholders. They will also help develop new drugs by cultivating partnerships and open innovation with pharmaceuticals companies working on neurological diseases.



 

 


Notes
  1. Estimate based on number of medical beneficiary certificate holders for specific medical expenses for ALS, a designated intractable disease, that the Ministry of Health, Labour and Welfare has disclosed as an example for health administration reporting.
  2. Experimental models use animals or cells reproducing human disease conditions and are for evaluating the efficacy of novel drug candidates before administration to people.
  3.  The Japanese Consortium for Amyotrophic Lateral Sclerosis Research at Aichi Medical University aims to elucidate the pathophysiology of ALS and develop therapies for it by analyzing the clinical and genetic information of sufferers. The consortium has collected medical information on the disease progression patterns of more than 2,000 individuals in Japan.
  4. Watanabe H, et al. J Neurol Neurosurg Psychiatry. 2016; 87: 851-858.
  5. iPS cells can be generated from human blood and other cells and propagate and change into other cell types in the body. It is possible to use iPS cells from ALS patients to reproduce disease states in dishes. This could help with evaluating the efficacy of novel drug candidates. (Reference papers: (1) Imamura K, et al. Sci Transl Med. 2017; 9: eaaf3962 and (2) Fujimori K, et al. Nat Med. 2018; 24: 1579-1589)
  6. Overview of Japan Agency for Medical Research and Development project 
    Project name: Funding for research to expedite effective drug discovery by Government, Academia and Private partnership
    Research and Development theme: Development of novel therapeutic drugs for amyotrophic lateral sclerosis using a large-scale disease registry and iPS cell technology 
    Research period: April 2019 to March 2023