- MDA has awarded funding to help support eight new ALS research projects.
- The ALS grants are part of 44 new grants awarded by MDA, funding research into almost half of the diseases in MDA's program.
MDA has awarded eight new grants totaling nearly $2.5 million to fund research into the causes of, and potential therapies for, ALS.
The grants were reviewed and approved by the MDA Board of Directors in December 2010, based on recommendations from the MDA Scientific and Medical Advisory Committees. Funding began Feb. 1, 2011.
Motor nerve terminals: Ellen Barrett at the University of Miami (Florida) Miller School of Medicine, received $297,102 to study the dependence of motor nerve terminals (the part of the motor neuron, or nerve cell, that conveys signals from the brain to muscles) on the “energy factories” that power cells, called mitochondria. Barrett’s work may reveal potential for a combination of treatments aimed at preserving both motor neuron terminals and cell bodies to effectively slow ALS disease progression.
Phase 2 human clinical trial: Research fellow James Berry at Massachusetts General Hospital in Boston was awarded $180,000. Berry is a member of the study team currently planning a phase 2 clinical trial in people with familial ALS of the experimental drug ISIS-SOD1-Rx, made by Isis Pharmaceuticals of Carlsbad, Calif. The trial is planned as a follow-on study to a current phase 1 trial of the drug. Berry’s work could lead to a phase 3 clinical trial of ISIS-SOD1-Rx in people with familial ALS.
3-D model of the human spinal cord: MDA awarded $347,094 to François Berthod, of the Department of Surgery, Laval University, in Quebec, Canada. Berthod is developing a three-dimensional model of the human spinal cord using nervous system cells obtained from the tissues of people with ALS. The model is expected to improve understanding of the causes and progression of sporadic (noninherited) ALS.
SOD1-mitochondria interactions: MDA awarded $180,000 to Adrian Israelson at the University of California, San Diego in La Jolla. Israelson will focus on the ways in which mutant misfolded SOD1 protein (found in some forms of ALS) associates with mitochondria, and whether and how those interactions affect mitochondrial function, possibly leading to motor neuron death.
Changes in mitochondria: MDA awarded $350,133 to Michael Miller at the University of Alabama, Birmingham. Miller will study the ways in which gene mutations in familial ALS may disrupt a signaling mechanism necessary for mitochondrial function and, in turn, affect motor neuron health.
New iPSC research model: Alysson Muotri at the University of California, San Diego in La Jolla, was awarded $362,466 to support development of a new ALS research model. The model will be made using sophisticated induced pluripotent stem cell (iPSC) technology that will allow scientists to use the genomes (DNA) of ALS-affected individuals to generate a stem cell line. Populations of neural cells can then be matured from this line and isolated for study.
Motor neuron development: Shanthini Sockanathan at Johns Hopkins School of Medicine in Baltimore received $396,000 to study motor neuron development in ALS. Sockanathan and colleagues will use a combination of approaches to focus on the mechanisms underlying regulation, initiation, extent and rate of motor neuron differentiation (maturation).
SOD1-TDP43 comparison: MDA awarded $330,000 to Jiou Wang at Johns Hopkins University in Baltimore, to support comparison of two types of familial ALS — those associated with the SOD1 protein and those associated with the TDP43 protein. The investigators will examine the molecular mechanisms underlying the toxicity triggered by flawed SOD1 and TDP43 genes in a nematode (worm) research model of ALS.
For more details about these new grants, visit MDA’s research page and click on Grants at a Glance.