Chris Lorson, professor of veterinary pathology, and molecular biology and immunology at the University of Missouri in Columbia, was awarded an MDA research grant totaling $300,000 over three years to optimize an approach currently in clinical trials for spinal muscular atrophy (SMA) by delivering an enhanced form of drug combined with other potential synergistic therapies. This work could provide evidence for an innovative combinatorial approach to SMA that would address a broad range of patient needs.

What inspired you to study SMA?

I have been working in the SMA field since 1997 as a post-doctoral fellow in Dr. Elliot Androphy’s lab when I received the MDA’s Robert G. Sampson Neuromuscular Research Named Fellowship Award. The interaction with the SMA community and the direct connection to translation research was inspiring and that connection is something that hope I impart to my lab today.

What is your area of focus within the SMA disease field?

Recently we have been focusing upon translational approaches to increase SMN protein. In this project funded by the MDA, we will be investigating whether the co-administration of an antisense oligonucleotide (ASO) and other drugs that improve the SMA phenotype can be combined for an even greater therapeutic effect.

Why is your area of focus important?

This project focuses upon a novel compound we have developed in the lab (an ASO targeting an important regulatory region called “Element 1”). We are interested in moving this type of compound into the clinic and the ability to combine this compound with other drugs that target other disease manifestations would be important as it moves towards the clinic.

Why is it important that MDA continue to fund research in SMA?

SMA is exceptionally well suited for therapeutic approaches that target SMN2; however, there currently is no FDA-approved drug for SMA. A number of exciting clinical trials are underway for gene therapy, small molecules or ASOs, but SMA is a complex disease that will likely require multiple approaches to address all of the symptoms.

What do you feel people impacted by SMA can have the most hope about with respect to research right now?

The SMA community as a whole (patients and families as well as researchers/clinicians) have watched as the SMA field transitioned from basic research, to translational research, and now into clinical trials. For a “rare” disease, the number of therapeutic opportunities are truly amazing. Importantly, however, there is an enormous amount of work to be done and none of it can happen fast enough.

To learn more about how MDA research is accelerating treatments and cures for SMA, please visit mda.org.