MDA grants scientist $300K for research into role of SMN protein
3-year grant could lead to new stem cell treatments for SMA
The Muscular Dystrophy Association (MDA) has awarded one of 37 new research grants to a professor studying the role of survival motor neuron or SMN — the protein that’s missing in people with spinal muscular atrophy (SMA) — in disease development and treatment.
Winning the three-year grant — in the amount of nearly $300,000 — is Umrao Monani, PhD, who specializes in the field of pediatric neurology at Columbia University Irving Medical Center in New York.
This year’s MDA grants total $8.4 million, all of which will go toward research into a range of neuromuscular diseases, ranging from amyotrophic lateral sclerosis and Charcot-Marie-Tooth disease to muscular dystrophy and SMA.
“MDA is proud to support the most innovative research that will lead to novel treatments for a range of neuromuscular diseases,” Sharon Hesterlee, PhD, MDA’s chief research officer, said in a press release detailing the various grants.
MDA research grants have helped develop gene therapies for SMA, MD
SMA is caused by a lack of SMN, a protein involved in the maintenance of motor neurons. Without enough SMN, motor neurons — the nerve cells that send signals to muscles to control movement — die off, causing muscles to weaken and waste away over time.
Earlier work in mice has suggested that a lack of SMN protein in muscles also may contribute directly to the development of SMA symptoms. However, how this happens is unclear.
Monani’s team will use the grant to explore the role of SMN protein in muscles, focusing specifically on stem cells.
Muscles are home to a type of stem cells from which muscle cells can develop. These stem cells are normally inactive, but when muscles are injured, they become active to give rise to new muscle cells to replace the damaged ones.
If successful, the results are expected to cast light on the role of those stem cells in SMA and on how the SMN protein works to keep muscles healthy — which could lead to new treatments for the disease.
The team previously had found that a lack of SMN prevents a group of proteins, called SNARE complex, from assembling in motor neurons. The SNARE complex is important for nerve cells to send signals to other nerve cells.
Innovative and strategic grant funding … has now led to the first ever gene therapy treatments for Duchenne muscular dystrophy and spinal muscular atrophy.
The MDA has awarded a total of 35 grants in SMA for research over a period of more than a decade. The award to Monani appeared to be the only one in SMA this cycle.
Overall, this most recent grant cycle strengthened the organization’s commitment to advancing research into neuromuscular diseases.
“Innovative and strategic grant funding … has now led to the first ever gene therapy treatments for Duchenne muscular dystrophy and spinal muscular atrophy,” said Donald S. Wood, PhD, MDA’s president and CEO.
“We are pleased to invest an additional $8.4 million during this grant cycle to continue to accelerate advancements in treatments to empower the families we serve,” Wood said.