Biogen
Idec (NASDAQ: BIIB) announced today that it has created a new
research consortium in collaboration with several leading academic
research centers that will leverage a range of scientific techniques and
disciplines to identify new approaches to treating amyotrophic lateral
sclerosis (ALS). It is anticipated that coordinating research and
sharing results across a number of different disciplines will greatly
accelerate the understanding of the mechanism of this disease and the
development of new targets and approaches to treatment.
Through the Consortium, each participating lab will undertake 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. This level of
collaboration will enable researchers to apply others’ learnings to
their own efforts in near real time.
“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, Ph.D., senior vice president, chief scientific
officer, for Biogen Idec and Professor of Cell Biology, 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. We are hopeful that the assembly of this extraordinary group
will allow unprecedented insights and define a new level of synergy
between academic research and Biogen Idec.”
Biogen Idec has committed more than $10 million over three years to fund
research projects by members of the Consortium. 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.
The members of the Consortium were identified based on their core
scientific and technical skills. Expertise in ALS was not a primary
requirement for participation, although the group includes some of the
most renowned neuroscientists and ALS researchers in the world.
Members of the Consortium are:
Pietro De Camilli, M.D., 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’s research as part of this Consortium 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, Ph.D., Bert and Natalie Vallee Professor of
Molecular Pathology, Harvard Medical School. Dr. Harper’s work as part
of the Consortium will focus on profiling the organization 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, Ph.D., 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's work is focused on the mechanisms of motor neuron growth,
survival and cell death and how these are regulated in human disease.
His research as part of the Consortium is to 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, M.D., Sterling Professor of Genetics and
Professor of Pediatrics, Yale School of Medicine; Investigator, Howard
Hughes Medical Institute, Yale University School of Medicine. Dr.
Horwich’s research as part of the Consortium is focused 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, Ph.D., 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’s work as
part of the Consortium is to 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, Ph.D., President; Carson Family Professor;
Head of the Laboratory of Brain Development and Repair, The Rockefeller
University. Dr. Tessier-Lavigne’s laboratory investigates how neural
circuits in the brain form during embryonic development. As part of the
Consortium, Dr. Tessier-Lavigne’s lab will investigate cell death
pathways and determine how they contribute to motoneuron degeneration
and disease progression in ALS.
Spyros Artavanis-Tsakonas, Ph.D. will oversee the Consortium on
behalf of Biogen Idec and will lead research efforts for the company and
his lab at Harvard University. Dr. Artavanis-Tsakonas’s Consortium
research focus will employ high throughput screens in Drosophila
(often called “fruit flies”) 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 Commitment to ALS Research
Biogen Idec is committed to advancing the understanding and treatment of
ALS. The company is working with researchers around the globe to explore
ways to create better outcomes for people with ALS.
Biogen Idec is investigating multiple compounds in ALS, with two
compounds in preclinical research. In 2012, the company entered into a
research collaboration with Duke University and Hudson Alpha Institute
to sequence the genomes of up to 1,000 people with ALS over the next
five years in an effort to gain a deeper understanding about the
fundamental genetic causes of the disease. Duke and Hudson Alpha will
work with several world-class researchers who have deep expertise and
experience with ALS and the genes associated with the disease.
In addition, Biogen Idec has sponsored 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.
The company has also contributed $500,000 to the University of
Massachusetts Medical School ALS Champion Fund. The funding will
increase awareness of ALS and support basic and clinical science
research into potential treatments for ALS and other neurodegenerative
diseases.
About ALS
Amyotrophic lateral sclerosis, also known as Lou Gehrig's disease and
motor neuron disease, is a universally and rapidly fatal
neurodegenerative disorder characterized by progressive muscle weakness
and wasting. ALS affects adults in the prime of life and creates a
substantial burden for caregivers. Worldwide incidence of ALS is
approximately two people per 100,000. Only one drug has been approved
for the treatment of ALS, and it typically extends survival by two to
three months. Life expectancy after the onset of symptoms is usually
three to five years. Though mutations in several genetic factors result
in ALS, the precise mechanistic cause is not yet known.
About Biogen Idec
Through cutting-edge science and medicine, Biogen Idec discovers,
develops and delivers to patients worldwide innovative therapies for the
treatment of neurodegenerative diseases, hemophilia and autoimmune
disorders. Founded in 1978, Biogen Idec is the world’s oldest
independent biotechnology company. Patients worldwide benefit from its
leading multiple sclerosis therapies, and the company generates more
than $5 billion in annual revenues. For product labeling, press releases
and additional information about the company, please visit www.biogenidec.com.
Biogen Idec Safe Harbor Statement
This press release contains forward-looking statements, including
statements about the development of potential therapies for ALS. These
forward-looking statements may be accompanied by such words as
"anticipate," "believe," "estimate," "expect," "forecast," "intend,"
"may," "plan," "will" and other words and terms of similar meaning. You
should not place undue reliance on these statements. Drug development
and commercialization involve a high degree of risk. Factors which could
cause actual results to differ materially from current expectations
include the risk that adverse safety events may occur, regulatory
authorities may require additional information or may fail to approve
any potential new therapy, product reimbursement may be limited or
unavailable, there may be problems with manufacturing processes,
intellectual property rights may not be adequately protected, and the
other risks and uncertainties that are described in the Risk Factors
section of Biogen Idec Inc.’s most recent annual or quarterly report and
in other reports Biogen Idec Inc. has filed with the SEC. These
statements are based on current beliefs and expectations and speak only
as of the date of this press release. Biogen Idec Inc. does not
undertake any obligation to publicly update any forward-looking
statements.
