Originally published on March 11, 2014.
Cure SMA is leading the way to a world without SMA by advancing a comprehensive research program, of which basic research is a key component. Cure SMA has awarded 79 basic grants for a total of $9,507,612 in the past 10 years. Dr. Ebert at the Medical College of Wisconsin will receive $95,000 to explore the role of neuronal support cells called astrocytes in SMA.
Astrocytes and Oxidative Stress in SMA
Objective: We aim to determine if astrocytes directly contribute to oxidative stress and motor neuron death in an in vitro model of SMA. Astrocytes are a type of support cell for motor neurons in the central nervous system.
Research Strategy: We propose to use astrocytes and motor neurons derived from SMA patient and control induced pluripotent stem cells to study mitochondrial function and markers of oxidative stress to assess cell health, as well as measure cell death.
Significance: These studies will provide additional mechanistic insight into the contribution of astrocytes to disease pathology in SMA, which could have important implications for therapeutic development.
Meet Dr. Ebert:
Who are you?
I am a native Midwesterner with a PhD in neuroscience focusing on understanding molecular processes leading to neuronal loss .
How did you first become involved with SMA research?
I became involved in SMA research in 2007 when we began to establish a human stem cell model for SMA. We were the first to generate and characterize SMA patient-derived stem cells as a model for understanding features of the disease.
What is your current role in SMA research?
My lab focuses on understanding molecular pathways that lead to motor neuron loss. Reduced SMN clearly causes motor neuron loss, but my lab aims to understand how losing SMN expression in other cell types, specifically glial cells, negatively impacts the whole neural system. Glial cells play a critical role in neuron health and function, so we are interested in learning more about how reduced SMN changes the glial cells’ supportive properties.
2014 Cure SMA Research Mission and Funding
The overall mission at Cure SMA is a world without SMA. Making this happen requires scientific research investments. Thus, our main research goal at Cure SMA is to accelerate the discovery of an effective treatment and cure for SMA by funding and advancing a comprehensive research program, including:
• Basic Research to reveal the best ways of making SMA drugs,
• Drug Discovery to make new drugs,
• Developing Clinical Trial infrastructure to help test new drugs, and
• Clinical Care Research to improve the quality of life in SMA patients.
In addition to the basic research funding announced in this edition of Compass, Cure SMA will invest in more research in 2014 to advance our vision of a world without SMA. Please be on the lookout for two upcoming Requests for Proposals (RFPs) for additional research funding from Cure SMA in 2014 in the following research areas: 1) Drug Discovery – Overseen by the Translational Advisory Council, and 2) Clinical Care – Overseen by the Medical Advisory Council.