In its Summer 2013 round of grants, MDA awarded 31 new grants totaling $8.5 million.
The new grants, which took effect Aug. 1, support research in 12 neuromuscular diseases under MDA’s umbrella, including: amyotrophic lateral sclerosis (ALS); Becker muscular dystrophy (BMD); central core disease (CCD); Charcot-Marie-Tooth disease (CMT); Duchenne muscular dystrophy (DMD); facioscapulohumeral muscular dystrophy (FSHD); limb-girdle muscular dystrophy (LGMD); myotonic muscular dystrophy (MMD, DM); mitochondrial myopathy; muscular dystrophies (general); oculopharyngeal muscular dystrophy (OPMD); spinal-bulbar muscular atrophy (SBMA); and spinal muscular atrophy (SMA).
Findings from many of the newly funded projects will advance science and technology for multiple diseases. The grants follow five major themes.
A number of the new grants investigate new therapeutic technologies — specifically, new types of gene therapy.
For example, two grants fund research into repairing defective genes using a process called gene editing. Another project looks at how to prevent unwanted immune reactions associated with gene therapy. Although all three projects are being conducted in DMD models, the technologies involved have potential in many other muscle diseases.
“These are ‘platform’ technologies, which, if successful, will impact not only the disease under study, but many others as well,” says MDA Vice President of Research Jane Larkindale. “Development of new platforms for drug discovery is critical to developing new drugs, and almost half of the new MDA grants have the potential to advance therapy development in more than one condition covered by MDA.”
Nearly half of the newly funded research projects test experimental treatments and/or evaluate biological processes or pathways that potentially could be modified to treat the disease.
“These grants fund less ‘why is there a problem’ research and more ‘what can we do to fix the problem’ research than ever before,” Larkindale explains.
Diseases at the forefront
As science advances, new opportunities often arise to make progress in diseases that previously had stymied researchers.
That’s the case in CCD. MDA is taking advantage of scientific advances in technology to fund three new grants that will better define the fundamental problems in CCD, and provide insights into new treatment targets.
One benefits many
“We are beginning to see a real leverage of knowledge from one MDA-funded project to another,” Larkindale notes.
For example, through its translational research program, MDA has been funding ReveraGen BioPharma to develop a gentler version of prednisone (a corticosteroid drug) for DMD. Earlier this year, MDA funded a project to study the same drug in dysferlinopathies (muscle diseases caused by mutations in the dysferlin gene, an important muscle repair protein).
“A whole new group of diseases may benefit from the money and time put into the Reveragen project,” Larkindale says.
Back to basics
Sometimes research involves stopping and taking a closer look.
“In the two new ALS grants,” Larkindale explains, “researchers are looking at ways to alter the course of the disease by going ‘back to basics’ in order to figure out what — of all the things that go wrong in ALS — can be modified.”
The two ALS projects focus on clumps of protein (aggregates) that are found in the cells of people with the disease. Both projects investigate the possibility of removing these aggregates by breaking them down — one through a cellular waste disposal process called autophagy (“awe-TOF-uh-gee”) and the other through proteins called disaggregases, whose function is to break up such clumps.
“As research evolves, new ideas come to the fore in different diseases,” Larkindale says. “MDA is committed to pursuing those new ideas in all the diseases in our program — and to maximize the progress in each of them to speed the best research in all of them.”