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May 2007MDA NEWS RESEARCH NEWS Pass it along! |
MDA NEWS
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Scott Stafne, 34, of Woodbury, Minn., loved to ski. Then one day in August 2004, when he and his wife were expecting their first child, he learned he had ALS – Lou Gehrig’s disease. The story of how their lives changed dramatically is presented on MDA’s ALS Division Web site May 7 … and is one of many such stories.
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Every day during May, MDA’s ALS Division will present an account of a different person with the devastating adult disease amyotrophic lateral sclerosis. May is the country’s 16th annual ALS Awareness Month, during which MDA is focusing on raising public awareness of the disease.
MDA is the world’s leader in ALS research and services. During May, public service announcements and news stories about MDA’s ALS program will appear in news media, and MDA offices across the country are hosting educational seminars and special events.
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Be sure to read the profiles in strength, perseverance, humor and love in the “ALS: Anyone’s Life Story” online feature. And when you share those wonderful stories, know that MDA-funded researchers continue their relentless pursuit of treatments and cures for this destructive, fatal disease, and they’re getting results.
Evidence Mounts for Immune System
Role in ALS
Researchers in the Don Cleveland laboratory at the University of California-San Diego have found new evidence implicating the immune system in Lou Gehrig’s disease.
Team members, including MDA-supported Severine Boillee, discovered that mouse motor neurons that carry mutated genes known to cause ALS are a component of the immune system. Findings suggest therapies that interfere with that aspect of the immune system could be beneficial in treating ALS.
Two New ALS Publications Debut
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MDA has just produced two new publications: a brochure describing MDA’s ALS Division program, and “Milestones in ALS Research,” which tracks scientific developments in understanding the disease since its discovery in 1874.
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A special edition of the MDA/ALS Newsmagazine describes the Association’s ALS program in depth.
Augie’s Quest ‘Bash’ Raises Millions
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The second annual Black & Blue Bash for Augie’s Quest raised more than $2 million for ALS research during a March 30 gala event in San Francisco. More than 1,000 supporters attended.
Augie and Lynne Nieto,
co-chairpersons of MDA’s ALS Division, hosted the event. To
date, MDA’s Augie’s Quest research initiative has raised
more than $9.9 million and funded groundbreaking MDA
research efforts into ALS.
RESEARCH NEWS
Drug Restores Muscle Function
in
Mice with MD
In lab tests, a drug for the first time restored normal muscle function in mice that have the animal equivalent of Duchenne muscular dystrophy.
MDA helped fund the mice studies conducted at the University of
Pennsylvania in Philadelphia using a new drug called PTC124 developed
by PTC Therapeutics of South Plainfield, N.J. The study’s
findings are particularly relevant because results are about to
be released from related human trials, also supported by MDA.
Researchers Isolate New Cause of CMT
A new cause of Charcot-Marie-Tooth disease has been identified by a team including MDA grantee Robert Baloh at Washington University in St. Louis and Alan Pestronk, director of the MDA clinic at the university.
They found that mutations in the gene for mitofusin 2 (MFN2) cause 19 percent to 33 percent of type 2 CMT (which results from damage to nerve fibers that run between the spinal cord and body extremities).
A logical next step, the researchers say, is to develop mice with
MFN2 defects so therapies for the condition can be sought in the
laboratory.
Motor Neurons Respond to Stress Differently
Motor neurons (the nerve cells that operate muscles) don’t seem capable of reacting to outside stress as well as other types of cells, according to studies by MDA grantee Heather Durham at Montreal Neurological Institute. Motor neurons die or work improperly in diseases such as ALS and spinal muscular atrophy.
Durham and other researchers say if this shortcoming could be remedied,
it might lead to therapeutic measures for ALS.
Team Increases Injections of
Cells in DMD,
Becker Study
MDA-supported researcher Jacques Tremblay at Laval University in Quebec has been studying the effects of injecting muscle precursor cells (MPCs) into the muscles of males over 18 who have Duchenne or Becker muscular dystrophy.
When Tremblay injected MPCs into boys with DMD, dystrophin (the protein lacking in the disease) was produced in eight of nine cases.
In future trials, the researchers will inject subjects with many
more cells per square centimeter at one time.
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