The ALS Association Accelerates Search for Lou Gehrig's Disease Genes

Unprecedented Research Effort Mirrors The ALS Association's

Aggressive Search for a Cure

Jul 16, 2002, 01:00 ET from The ALS Association

    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
 ALS-linked genes."
     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.
     About 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 .
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SOURCE The ALS Association