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Repositioning of Small Molecule for ALS Treatment
Yeda R&D Co. Ltd Israel flag Israel
Abstract ID: 1632
Amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig\'s disease) is a progressive neurodegenerative disease that leads to paralysis, due to the death of motor neurons in the...
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Introduction/Background

Amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig's disease) is a progressive neurodegenerative disease that leads to paralysis, due to the death of motor neurons in the spinal cord and brain. There is no known cause, cure or effective treatment for the disease. RilutekŪ (riluzole) is the only approved drug prescribed for the treatment of ALS. However, Rilutek does not improve patient quality of life or survival.

Aims/Hypothesis

There is a need for an effective drug for ALS, a disease for which no current effective drug or treatment are available.

Results

Prof. Hornstein and his team have discovered that microRNAs (miRNA) are globally downregulated in motor neurons of sporadic and familiar ALS patients and in ALS cell-culture model. Remarkably, they demonstrated that the deleterious effect of ALS-causing mutations on miRNA processing could be reversed by Enoxacin, an antibiotic known to increase miRNA levels via upregulation of Dicer1, a key player in miRNA maturation.

The proposed technology is based on the discovery that microRNAs (miRNA) are globally downregulated in motor neurons of sporadic and familiar ALS patients and in cultured neurons, which express ALS-causing mutant forms of TDP-43, FUS or SOD1. Prof. Hornstein and his team further revealed that while mature miRNAs were downregulated, the levels of their cognate pre-miRNA precursors were upregulated by ectopic expression of ALS-causing mutant TDP-43 or FUS. Finally, they have demonstrated that the deleterious effect of the ALS-causing mutations on miRNA-processing could be reversed by Enoxacin, a Fluoroquinolone antibiotic, known to increase miRNA levels via enhancement of Dicer1 activity, which plays a key role in miRNA maturation.

Conclusion

This discovery establishes Dicer1 as a new target for ALS drug development and holds a potential for an effective treatment for ALS.

Relevance/Opportunity

Please enquire quoting reference 1632 regarding licensing or codevelopment partnerships.
FEATURED
Last Updated May 2015
Technology Type THERAPEUTIC
Phase of Development PRECLINICAL
CORPORATION