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!
Umrao Monani, PhD, has been awarded $150,000 for his research project, “Exploring mechanisms of action of a novel SMA modifier.”
Meet Dr. Monani
Dr. Monani is a founding member of the Columbia University Center for Motor Neuron Biology & Disease and a Professor of Neurology at Columbia University Irving Medical Center. He directs the Colleen Giblin Research Laboratories, which are dedicated to developing safe, reliable, and effective treatments for a variety of pediatric neurological diseases.
Dr. Monani began to study SMA as a graduate student when he identified the key differences between the SMN1 and SMN2 genes. These findings served as the basis for some of the first mouse models of SMA. His current research focuses on why, at the molecular level, low SMN protein causes SMA.
Recently, Dr. Monani and his colleagues discovered that a synaptic chaperone protein suppresses disease severity in an SMA mouse model. Dr. Monani’s Cure SMA grant will support his lab’s efforts to determine how this “modifier protein” lessens SMA severity. Notably, they seek to determine if the suppression of disease severity occurs in an SMN-independent manner. Elucidating this could contribute to the understanding of how low levels of SMN protein cause SMA.
Dr. Monani’s research may also reveal if modifier proteins can enhance the effects of existing SMA treatments to yield better results for patients.
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.