Biogen Idec forms ALS research consortium

Published: 2-Jan-2013

Commits more than US$10m of funding over three years

Biogen Idec of Switzerland has created a new research consortium that will identify new approaches to treating amyotrophic lateral sclerosis (ALS), also known as motor neuron disease.

The firm has committed more than US$10m over three years to fund research projects by consortium members.

This initiative will complement and extend a collaboration the company announced earlier this year with Duke University and the Hudson Alpha Institute to sequence the genomes of 1,000 people living with ALS.

Through the consortium, each participating lab will carry out a three-year research project. Researchers will meet on a regular basis to provide updates and share information and insights emerging from their research and to exchange results from ongoing efforts at Biogen Idec, which will help to guide ongoing research activities.

ALS is a very difficult disease to understand and treat

‘ALS research is a primary area of focus for Biogen Idec, but has proven to be a very difficult disease to understand and treat,’ said Spyros Artavanis-Tsakonas, senior vice president, chief scientific officer at Biogen Idec and Professor of Cell Biology at Harvard Medical School.

‘We believe that taking a holistic approach that explores the many variables involved in the development and progression of ALS will speed our ability to identify viable drug targets that can be moved into testing.’

The consortium members are:

Pietro De Camilli, Eugene Higgins Professor of Cell Biology and Professor of Neurobiology; Director, Yale Program in Cellular Neuroscience and Neurodegeneration and Repair; Investigator, Howard Hughes Medical Institute, Yale University School of Medicine.

Dr Camilli will investigate the general role of the VAP protein family in lipid regulation and endoplasmic reticulum stress response and to identify the molecular pathways that are affected by disease-causing mutations of the VAP-B protein.

J. Wade Harper, Bert and Natalie Vallee Professor of Molecular Pathology, Harvard Medical School. Dr Harper will focus on profiling the organisation of the mitochondrial interactome in response to ALS mutant proteins to determine whether expression of mutant proteins linked to ALS leads to activation or inhibition of the mitochondrial quality control system.

Christopher E. Henderson, Gurewitsch/Vidda Professor of Rehabilitation and Regenerative Medicine, Pathology, Neurology and Neuroscience; Director, Columbia Stem Cell Initiative; Co-Director, Center for Motor Neuron Biology and Disease (MNC); Co-director, Project A.L.S. Laboratory for Stem Cell Research; Columbia University.

Dr Henderson will explore the differences between motor neurons in the spinal cord

Dr Henderson will explore the differences between motor neurons in the spinal cord, which are destroyed in ALS, and those which move the eyes, which are unaffected in ALS, with the goal of identifying genes that may confer protection against the disease.

Arthur L. Horwich, Sterling Professor of Genetics and Professor of Paediatrics, Yale School of Medicine; Investigator, Howard Hughes Medical Institute, Yale University School of Medicine.

Dr Horwich will focus on the role of the SOD1 gene, which has been associated with protein misfolding and has been shown to play a role in pathology of ALS. In particular, the lab will explore whether misfolded SOD1 protein can spread from affected tissue to normal tissue using mouse models of ALS.

Lee L. Rubin, Professor of Stem Cell and Regenerative Biology; Director of Translational Medicine, Harvard Stem Cell Institute, Harvard University. Dr. Rubin’s work uses a stem cell based approach to understand neurodegenerative diseases.

Dr Rubin will develop SOD mouse or human motor neuron cell lines that capture early pathological changes and use these cellular phenotypes to identify drug-like molecules/pathways that affect disease pathology to identify potential therapeutic leads.

Marc Tessier-Lavigne, President; Carson Family Professor; Head of the Laboratory of Brain Development and Repair, The Rockefeller University.

Dr Tessier-Lavigne’s lab will investigate cell death pathways and determine how they contribute to motoneuron degeneration and disease progression in ALS.

Spiros Artavanis-Tsakonas will oversee the consortium on behalf of Biogen Idec and will lead research efforts for the company and his lab at Harvard University. He will employ high throughput screens in Drosophila to identify genes that effect ALS dependent phenotypes resulting from expression of disease causing forms of SOD1, TDP-43 and FUS, and to identify protein complexes associated with ALS-related proteins in humans and Drosophila.

Biogen Idec is investigating multiple compounds in ALS, with two compounds in preclinical research

Biogen Idec is investigating multiple compounds in ALS, with two compounds in preclinical research. Last year the company entered into the research collaboration with Duke University and Hudson Alpha Institute. Duke and Hudson Alpha will work with researchers who have experience with ALS and the genes associated with the disease.

In addition, the company is sponsoring research agreements with various academic institutions with the shared goal of understanding the common course of ALS from symptom onset to diagnosis, through treatment and life expectancy.

Biogen Idec has also contributed $500,000 to the University of Massachusetts Medical School ALS Champion Fund to increase awareness of ALS and support basic and clinical science research into potential treatments for ALS and other neurodegenerative diseases.

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