Potential new targets for ALS

Overview

Amyotrophic lateral sclerosis (ALS) is a devastating disease that is progressive, invariably fatal, and untreatable. Our invention features isolated Cyp4gl polypeptides with mutations at various key amino acid positions that suppress the effects and progress of ALS, as well as mutated polynucleotides that encode these polypeptides. Our invention also includes methods for treating ASL by administering a pharmaceutical or nutraceutical composition comprising one or more lipids.

Market Opportunity

Mutations in many genes with a wide range of functions and proposed mechanisms can cause familial ALS (fALS). Yet, 90% of cases of ALS present as sporadic ALS (sALS), with no family history and presumably no genetic correlation. There is substantial evidence that fALS and sALS have common underlying etiologies. Although many mouse models have been developed to study the mechanisms of ALS motor neuron death, all the candidate gene approaches to date have failed to extend the lifespan of affected mice. Thus, there is a pressing need for fundamental insights into this intractable disease, as well as methods for treating it.

Innovation and Meaningful Advantages

Our invention provides compositions and methods for treating fALS, as well as sALS cases that share several common features with fALS. In some cases, the composition comprises a suppressor Cyp4gl polypeptide; in others, the composition comprises a polynucleotide encoding an amino acid sequence of the suppressor polypeptide. In some compositions, the polypeptide is attached to the candidate agent, and in some, the candidate agent decreases the activity of Cyp4gl polypeptide.

A common feature of ALS is systemic metabolic dysregulation, especially lipid metabolism. Our invention includes methods for treating ASL by administering a pharmaceutical or nutraceutical composition comprising one or more lipids. It is likely that the Cyp4gl mutations could be used to identify a class of lipids which, when increased, or decreased, would themselves provide an ideal therapeutic index. Though research has been focused on dietary interventions in ALS, specific metabolites have not been forthcoming.

Collaboration Opportunity

We are interested in exploring 1) startup opportunities with investors and 2) research collaborations with leading pharmaceutical companies.

Principal Investigator

Robert A. Reenan, PhD

Professor of Biology

Brown University

robert_reenan@brown.edu

https://vivo.brown.edu/display/rreenan#Background

IP Information

Worldwide WO2019/104311A1; patent issued, 2019-05-31.

Worldwide WO2019/104315A1; patent issued, 2019-05-31.

Publications

Agudelo A, St. Amand V, Grissom L, Lafond D, Achilli T, Sahin A, Reenan R, Stilwell G. Age-dependent degeneration of an identified adult leg motor neuron in a Drosophila SOD1 model of ALS. Biol open 2020 Oct 21;9(10): bio049692. doi: 10.1242/bio.049692.

Contact

Andrew Bond, PhD

Senior Director of Business Development

andrew_bond@brown.edu

Brown Tech ID 2372

Website

http://brown.technologypublisher.com/technology/48647

Contact Information

TTO Home Page: http://brown.technologypublisher.com

Name: Andrew Bond

Title: Director of Business Development – Life Sciences

Email: andrew_bond@brown.edu