Thirty-eight new grants support research in ALS, central core disease, spinal muscular atrophy, muscular dystrophy, and other diseases in MDA's program
MDA has awarded 38 new research grants totaling more than $14 million and covering more than a dozen neuromuscular diseases.
MDA's Board of Directors met in Los Angeles July 16, where it reviewed and approved the new grants based on recommendations from the MDA Scientific and Medical Advisory Committees. Grants were scored and recommended for approval based on the capabilities of the applicant, the scientific merit of the project, and the proposal's relevance to developing treatments for the disease. The effective start date for all grants was July 1, 2010.
The grants support research into ALS, Becker muscular dystrophy, Duchenne muscular dystrophy, central core disease, congenital myasthenic syndrome, Charcot-Marie-Tooth disease, Emery-Dreifuss muscular dystrophy, inclusion body myopathy, limb-girdle muscular dystrophy, mitochondrial myopathy, myotonic muscular dystrophy, spinal bulbar muscular atrophy, spinal muscular atrophy and muscular dystrophies in general.
Below are highlights of all the new grants; for more information see Grants at a Glance, a detailed slideshow covering each new grant individually.
These new grants are in addition to some 330 research projects already being funded by the Association. MDA conducts two grant-funding cycles annually, and most grants are for multiple years.
Muscular dystrophies in general
Kurt Beam, professor in the department of physiology and biophysics at the University of Colorado School of Medicine in Denver, received $303,438 for research into a process called "excitation-contraction coupling," which is responsible for the contraction of muscle cells necessary for voluntary movement and breathing.
MDA awarded $330,000 to Ju Chen, professor of medicine at the University of California, San Diego, for research into the role of a protein called Cypher in skeletal muscle function and in diseases such as myofibrillar myopathy (MFM) and late-onset distal myopathy.
MDA awarded $160,000 to Dawn Cornelison, assistant professor in the division of biology at the University of Missouri-Columbia, for research into the role of a protein, syndecan-4, in muscle tissue repair after damage.
ALS (amyotrophic lateral sclerosis)
MDA awarded 10 grants totaling nearly $3.5 million to research projects focused on uncovering the causes of, and developing therapies for, ALS. The new grants went to investigators at labs in the United States, Canada and Israel.
The newly funded projects include research into the role of the immune system in the disease; early-stage (presymptomatic) biological markers ("biomarkers"); gene therapy; the molecular causes of degeneration of motor neurons (muscle-controlling nerve cells); combined-drug therapy; the function of the TDP43 gene and its interactions with other genes; and risk factors and modifiers of disease onset in SOD1-related genetic ALS.
For more on these grants, see "MDA Awards Nearly $3.5 Million in New ALS Grants."
Zolt Arany, assistant professor in medicine at Beth Israel Deaconess Medical Center, part of Harvard Medical School in Boston, received $352,188 for research into the role of blood vessels in skeletal muscle metabolism.
MDA awarded $408,915 to Carmen Bertoni, assistant professor of neurology at the University of California, Los Angeles, to continue research into DNA repair strategies using small molecules called single-stranded oligonucleotides, or ssODNs.
Michele Calos, a professor in the department of genetics at Stanford University School of Medicine in Stanford, Calif., received $200,000 to develop a new stem-cell-based therapy.
MDA awarded $179,327 to researcher Sean Forbes at the University of Florida in Gainesville for research into the effects of impaired blood flow to muscles lacking the dystrophin protein.
Bernard Jasmin, vice dean of research at the University of Ottawa in Canada, received $360,000 for research into using small molecules called "exercise mimetics" to increase levels of the structural protein utrophin in DMD.
MDA awarded Ashok Kumar of the University of Louisville School of Medicine in Kentucky $349,206 for continued study of the molecular mechanisms that underlie disease onset and progression.
MDA awarded $375,000 to Louis Kunkel, director of the program in genomics at Children's Hospital Boston, for research into compounds already approved for human use that may alter disease progression. Kunkel, a longtime MDA adviser and chairman of MDA's Scientific Advisory Committee, was on the MDA-funded scientific team that in 1986 first identified the gene mutation underlying DMD.
Gordon Lynch, professor of physiology at the University of Melbourne, Australia, received $375,000 for research into strategies aimed at using proteins called "growth factors" to improve muscle function.
Josephine Nalbantoglu, associate professor in the department of neurology and neurosurgery at McGill University in Montreal, Canada, received $313,170 for research into increasing levels of the muscle protein utrophin as a therapeutic strategy in DMD.
Vihang Narkar, assistant professor at the University of Texas Health Science Center at Houston, was awarded $302,326 to study the potential therapeutic value of increasing the overall amount of a specific type of muscle called "aerobic muscle" in DMD.
Professor Steve Wilton at the University of Western Australia in Perth received $368,100 for continued research into a strategy called "exon skipping," which bypasses mutations in the dystrophin gene responsible for DMD.
Central core disease (CCD)
MDA awarded $375,000 to Francesco Muntoni, professor of pediatric neurology at University College in London, United Kingdom, for research into the molecular mechanisms underlying CCD and multiminicore myopathies.
Michael Francis, associate professor in the department of neurobiology at the University of Massachusetts Medical School in Worcester, received $330,000 for research into effects on the connection between nerve and muscle known as the neuromuscular junction.
MDA awarded $282,630 to Albena Jordanova, professor in the department of genetics at the University of Antwerp, Belgium, for research into the molecular causes of, and potential drug targets for, a recently discovered form of CMT known as dominant intermediate CMT type C (DI-CMTC).
Ji-Yeon Shin, a research scientist at Columbia University Medical Center in New York City, received $180,000 for continued study of the molecular mechanisms responsible for EDMD.
MDA awarded $310,893 to Howard Worman, professor of medicine, pathology and cell biology at Columbia University Medical Center, for research into treatments that target the underlying cellular disease process responsible for cardiomyopathy (heart damage) in people with EDMD.
Inclusion body myopathy (IBM)
Virginia Kimonis, chief of the division of genetics and metabolism at the University of California, Irvine, received $372,000 to conduct experiments designed to determine whether amelioration of symptoms is possible in IBM associated with Paget's disease of bone and/or frontotemporal dementia (IBMPFD).
MDA awarded $339,561 to Richard Cripps, professor and chair of the biology department the University of New Mexico in Albuquerque, for research into the role of the Trim32 gene in type 2H LGMD.
Renzhi Han, assistant professor of physiology at Loyola University Medical Center in Maywood, Ill., received $405,000 to study mutations in the dysferlin gene that lead to development of several types of muscle diseases known as "dysferlinopothies," including type 2B LGMD.
MDA awarded $363,672 to Carlos Moraes, professor of neurology at the University of Miami School of Medicine, to study the effects generated by an increase in functional mitochondria (the "energy factories" in cells).
Leo Pallanck, associate professor of genome sciences at the University of Washington, Seattle, received $312,699 for research into elimination of flawed cell machinery in mitochondrial myopathy.
MDA awarded $411,000 to Lee-Jun Wong, professor in the department of molecular and human genetics at Baylor College of Medicine in Houston, for research designed to produce a one-step diagnosis procedure.
Aymeric Ravel-Chapuis, a research scientist at the University of Ottawa, Canada, was awarded $179,850 to study the role of a protein called Staufen1.
MDA awarded $330,000 to Albert La Spada, chief of the division of genetics in the department of pediatrics at the University of California, San Diego, to study what causes nerve cells called motor neurons to die in SBMA and other neurodegenerative diseases such as ALS (amyotrophic lateral sclerosis) and spinal musclar atrophy.
Spinal muscular atrophy (SMA)
MDA awarded $79,277 to John Manfredi, chief scientific officer at Sfida BioLogic, Inc., in Salt Lake City, Utah, for continued research into new drug compounds that promote the growth and function of motor neurons.