CALABASAS HILLS, Calif., July 16 /PRNewswire/ -- The ALS Association
(ALSA), the only national not-for-profit organization dedicated solely to the
fight against amyotrophic lateral sclerosis (ALS), today announced it has
launched a major research project that would significantly reduce the time
taken to identify the genes that cause ALS, more commonly referred to as Lou
Gehrig's disease. ALS is a fatal disease that attacks the motor neurons in
the brain and spinal cord and has no known cure. The $1.5 million project is
a collaboration among world-renown geneticists and scientists. This study
represents the largest research grant ever given by The ALS Association for a
single project and is among the largest ever in Lou Gehrig's disease research.
"The discovery of more genes that link to ALS is crucial for our
understanding of disease mechanisms and may provide new targets for therapy,"
said Lucie Bruijn, Ph.D., science director and vice president, The ALS
Association. By accelerating the process of discovering genes linked to Lou
Gehrig's disease, researchers hope to provide an important new roadmap for
scientists to use to speed the discovery of new therapies and cures for ALS
and other diseases.
About the Research Project
Human geneticists and scientists will apply technology used in the Human
Genome Project to find new genes and mutations linked to familial (passed
within families) ALS. The research project is to be executed in three phases.
The first phase involves development of BAC (bacterial artificial chromosome)
libraries from familial ALS patients to enable the sequencing of large regions
of the chromosome linked to familial ALS. Sequencing of the BAC libraries
will form the second phase of the project. As many sequence variations may be
present both in normal and disease populations, sequence changes (mutations)
unique to the patient population must be identified -- this represents the
third phase of the project.
"This international collaboration to identify new genes linked to familial
ALS is among the most exciting and potentially informative projects we have
initiated," said Dr. Tom Maniatis, professor of molecular and cellular
biology, Harvard University and head of The ALS Association's Cure ALS
Advisory Committee. "We are very fortunate to have the outstanding human
geneticists who have identified new ALS loci (regions), working closely with
scientists who played central roles in sequencing the human genome. If
successful, this effort will not only identify ALS genes associated with
currently identified familial loci, but will advance the technology required
to identify additional ALS genes in the future."
The discovery of the Cu/Zn superoxide dismutase 1 (SOD1) gene on
chromosome 21 linked to 20 percent of familial ALS, almost a decade ago and
more than a century after ALS was first identified, was a significant
milestone in ALS research. The results from this new study are expected to
advance the overall understanding of Lou Gehrig's disease. It will enable the
development of animal models scientists can use to test a variety of disease
hypotheses and therapies and even find a cure for this disease.
"There is no doubt that discovery of new causes of familial ALS will
illuminate our understanding of non-familial (sporadic) ALS. It is even
possible the new insights will be relevant to other brain degeneration
diseases," said Dr. Robert Brown of the Massachusetts General Hospital.
Sporadic ALS accounts for 90 percent of all ALS cases.
"Using the same technology as that used in the Human Genome Project
enables us to more systematically and rapidly sequence through large regions
of the genome to identify genes linked to disease. It's more efficient and
cost effective," says Dr. Eric Lander, director of the Whitehead Institute
Center for Genome Research, who will spearhead this effort. "Also, using
genomic DNA from ALS patients is the most direct and reliable way to identify
A distinctive characteristic of the project is the collaboration it has
fostered among some of the finest minds in the field of human genetics.
Scientists participating in this effort include Dr. Robert Brown of
Massachusetts General Hospital; Dr. Guy Rouleau of Montreal General Hospital;
Dr. Jackie de Belleroche of Imperial College, Charring Cross Hospital, London,
England; and Dr. Teepu Siddique of Northwestern University. In addition,
Dr. Pieter J. de Jong, Children's Hospital Oakland Research Institute, will
generate BAC libraries from cell lines prepared by GMP Companies, Inc.
Dr. Eric Lander of the Whitehead Institute Center for Genome Research, will
direct the sequencing phase of the project.
About ALS Research
The ALS Association is the largest private source of funding for
ALS-specific scientific research in the world. This gene identification
project will broaden The ALS Association's already diverse research portfolio.
The ALS Association continues to aggressively seek new and under-researched
areas, knowing fully the risks involved, but at the same time realizing the
importance of such an approach in order to find effective therapies and,
eventually, a cure for this dreaded disease.
The ALS Association has initiated a number of promising new directions in
ALS research, ranging from stem-cell research, to the development of new
animal models for ALS, to the establishment of cell-based assays and screening
for potential drugs to treat ALS.
Amyotrophic lateral sclerosis (ALS), more commonly referred to as Lou
Gehrig's disease, is a fatal, neurodegenerative disease that attacks nerve
cells and pathways in the brain and spinal cord. When these cells die, the
ability of the brain to start and control all voluntary muscle control and
movement dies with them. Patients in the later stages of the disease are
totally paralyzed, yet in most cases, their minds remain sharp and alert.
About The ALS Association
The ALS Association is the only national not-for-profit voluntary health
organization dedicated solely to the fight against ALS through research,
patient support, advocacy, and public awareness. The Association and its
widespread network of volunteer-led Chapters and support groups, along with
its certified ALS clinics, wage battle against the disease. For more
information about ALS and The ALS Association, visit www.alsa.org .
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SOURCE The ALS Association