MDA Awards 10 Grants Aimed at Stopping ALS

by Amy Madsen on Wed, 2010-09-01 09:34

MDA has awarded 10 grants totaling nearly $3.5 million to fund 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.

MDA’s Board of Directors met in Los Angeles July 16, where it reviewed and approved the new grants based on recommendations from the Association’s Scientific and Medical Advisory Committees (SAC and MAC). Grants are 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 diseases in MDA’s program. The effective start date for the grants was July 1, 2010.

Following is a listing of all grants approved by MDA’s Board:

  • Studying pre-symptomatic familial ALS: Michael Benatar, associate professor of neurology and epidemiology at Emory University in Atlanta, received $525,000 to continue research into early-stage FALS (familial ALS). Benatar’s group will study presymptomatic individuals with mutations in SOD1 and other ALS susceptibility genes, such as TDP43 and FUS. The team aims to better define the presymptomatic stage of familial ALS and identify environmental factors that might modify the age of disease onset among genetically susceptible individuals. Benatar co-directs the MDA Clinic at Emory University.
  • Genes that interact with TDP43: Daniela Zarnescu, assistant professor in neuroscience at the University of Arizona in Tucson, received $375,000 to conduct gene and drug discovery research in a drosophila fruit fly model that carries a mutation in the TDP43 gene. Mutations in this gene are associated with human ALS. Zarnescu’s group will conduct genetic screening tests on a TDP43 fruit fly model of ALS in order to identify genes that interact with TDP43, some of which may be involved in disease causation or progression.
  •  Studying function of TDP43 gene: Oliver Hobert, professor of biochemistry and molecular biophysics at Columbia University, New York, was awarded $374,511 to study the TDP43 gene, mutations in which can cause ALS. Using the invertebrate C. elegans (nematode, a type of worm) model, Hobert’s team will study the TDP43 gene in order to determine its function and possible interaction with other genes.
  •  Combined cell/gene therapy in ALS: Daniel Offen, head of the neurology laboratory at Tel-Aviv University, Israel, received $359,700 for research into a combined cell and gene therapy approach for ALS. Offen’s research team will engineer progenitor cells, a type of immature cell that forms muscle, to express various combinations of proteins that support motor neuron health. The investigators will observe the cells’ effects on disease in an ALS research mouse model and in human muscle cells.
  • TDP43’s effect on motor neurons: Wilfried Rossoll, assistant professor at Emory University in Atlanta, received $358,653 for research into the effects on motor neurons of TDP43 protein, which has been implicated in ALS. Preliminary data generated by Rossoll’s team suggests that TDP43 plays a role in mRNA processing (one of the steps in the cell’s protein-making process) in the long axons (fibers) that conduct electrical impulses from nerve cells to muscle cells. The team will test the hypothesis that decreased levels of TDP43 in mRNA-protein complexes in these axons may contribute to the degeneration of motor neurons in ALS.
  • Studying motor neuron degeneration: Shanthini Sockanathan, associate professor of neuroscience at the Johns Hopkins University School of Medicine in Baltimore, was awarded $347,832 for research into the molecular causes of motor neuron degeneration in diseases including ALS. Sockanathan’s team will study the physical and biological characteristics of the GDE2 research mouse model (which lacks the GDE2 protein) and monitor the progression of motor neuron degeneration in older animals.
  • Identifying a possible ALS risk factor: MDA awarded $345,000 to Jean-Pierre Julien, professor at Laval University, Canada, for study into variants of a protein called chromogranin B in ALS. In previous work, Julien’s group discovered that chromogranins interact specifically with mutant (flawed) forms of SOD1. (SOD1 mutations are known to be a cause of ALS.) The aim of the current project is to describe the exact mechanism by which a chromogranin B variation called P413L may act as a risk factor for ALS and as a modifier of disease onset.
  • Can T cells protect motor neurons?: MDA awarded $330,000 to Stanley H. Appel, chair of the department of neurology at the Methodist Neurological Institute (MNI) in Houston, to study the ability of immune system T cells to protect motor neurons in ALS. Project plans include the transplantation of various T cell types into ALS research mouse models in order to determine which of the cells are most helpful to neurons. Appel is a member of MDA’s Board of Directors, chairs the MDA Medical Advisory Committee and co-directs the MDA Neuromuscular Clinic in Houston.
  • Role of toxic TDP43 in ALS development/progression: Research scientist Brian Freibaum at St. Jude Children’s Research Hospital in Memphis, Tenn., was awarded $180,000 for research into the mechanism by which toxic TDP43 protein leads to the development and progression of some forms of ALS. Freibaum has planned a three-tiered approach to uncover the protein’s role in the disease using various tissues of fruit flies engineered to carry the human TDP43 gene; human cell lines and mouse motor neurons; and a fruit fly research model.
  • Clearing excess glutamate in ALS: Research scientist Dena Jacob at Thomas Jefferson University in Philadelphia, was awarded $180,000 for research into increasing the effectiveness of drugs in ALS-affected cells.

For more information, check out Grants at a Glance.

Amy Madsen
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