Each year, Cure SMA invites scientists from around the world to submit funding proposals for basic research projects that address specific unanswered questions in SMA biology. Our Scientific Advisory Board then ranks the submitted proposals on both their scientific merit and relevance to Cure SMA’s research priorities. Funding is awarded to the highest ranked projects.
In 2024, Cure SMA awarded a total of $740,000 to six scientists to pursue these objectives!
Yongchao Ma, PhD, has been awarded $100,000 for his research study, “Targeting Fascin regulation of mitochondrial DNA leakage and neuroinflammation as a treatment strategy for SMA.”
Meet Dr. Ma
Dr. Ma is an Associate Professor of Pediatrics, Neurology, and Neuroscience at Northwestern University Feinberg School of Medicine. He also holds the Children’s Research Fund Endowed Professorship in Neurobiology at Ann & Robert H. Lurie Children’s Hospital of Chicago and the Stanley Manne Children’s Research Institute.
During his post-doctoral training, Dr. Ma studied the development of spinal cord motor neurons and why they degenerate in SMA patients. His current research focuses on understanding how genetic defects in mitochondria, the power houses of cells, contribute to motor neuron degeneration in SMA.
Prior studies have shown that a protein called “Fascin” supports mitochondrial function. In SMA mice, low levels of Fascin are associated with mitochondrial dysfunction and unhealthy motor neurons. With his research grant from Cure SMA, Dr. Ma and his lab members will use mouse models to investigate this relationship. They will attempt to determine the mechanisms through which Fascin plays a role in inducing neuroinflammation and motor neuron degeneration in SMA.
Findings from this research may reveal new targets for novel SMA therapeutics.
Cure SMA’s top basic research priorities currently include:
- Learning more about how the survival motor neuron protein (SMN), which is expressed at low levels in SMA, is expressed and functions.
- Understanding the ways the SMN protein affects motor neurons and other cells in the body.
- Developing new treatments and advancing existing drug research, focusing on add-on therapies which work independently of SMN.
- Finding new research tools that can be used to learn more about disease progression and treatment response in SMA.
Thank You!
Special thanks to the Concepcion Family, Nunemaker Family, Weisman Family, Luke 18:1 Foundation, and Dhont Foundation for their generosity to Cure SMA in our quest to invest in basic research that will ultimately drive the next generation of SMA treatments.