Cure SMA Awards $140,000 Grant to Sara Custer, PhD, of Indiana University

By Cure SMA | Published On January 29, 2015

Audrey Lewis founded Families of SMA, now Cure SMA, 30 years ago. Audrey recognized early on the importance of attracting new and talented researchers to SMA, with the hope that they would commit their careers to developing a treatment and cure for SMA.

Cure SMA honors Audrey’s legacy with the Audrey Lewis Young Investigator Award, which is periodically given to younger researchers working in the SMA field. The goal is to make a positive impact on the early phase of a talented researcher’s career, enabling them to focus on the SMA field. The first award was given in 2011 to Dr. George Mentis of Columbia University for his project, "SMA as a progressive synaptic disease."

The recipient of the 2015 Audrey Lewis Young Investigator Award is Sara Custer, PhD, of Indiana University. Dr. Custer is a senior post-doctoral researcher, working towards leading her own SMA research lab in the future. She will receive $140,000 for her project, "Gene changes in a NSC-34 model of SMA."

Individuals with SMA don’t correctly produce survival motor neuron protein (SMN protein) at high enough levels. Dr. Custer’s project will examine how these low SMN levels affect the genes in motor neurons.

Meet Sara Custer

Who are you?

I have a PhD from the University of Washington in Neurobiology and Behavior and I study hereditary neurodegenerative diseases in cell culture and mouse models.

How did you first become involved with SMA research?

I became involved with SMA after moving to Indianapolis and joining the research lab of Dr. Elliot Androphy. I had previously worked with some type II and III patients at an amazing therapeutic equestrian facility in Woodinville, WA. Equestrian therapy is great for your core and for your spirit!

What is your current role in SMA research?

I am interested in learning more about the basic biology of SMA and how other proteins can influence the health and maintenance of motor neurons. We use a combination of cell models and animal models to address these questions. The more we know about the cellular environment in SMA, the more targets we have to aim at for therapeutic intervention.

How will this study work?

Using a motor neuron cell model of SMA, we will determine the gene changes caused by low SMN and also examine the biological consequences of these changes on motor neuron biology and SMA pathology.

What is the significance of your study?

Determining the gene changes caused by low SMN protein levels in our cells should identify genes that are specifically important for motor neuron health. This will reveal new drug targets and thus new avenues for therapeutic intervention in SMA, beyond the SMN protein. These pathways could be critical to motor neuron health and may also be relevant to multiple motor neuron diseases.

Basic Research Funding

This grant to Dr. Custer is part of $640,000 in basic research funding that we'll be announcing over the next few weeks. We'll profile each of the researchers who've received a grant, and share how their work can benefit those affected by SMA.

Basic research is the first step in our comprehensive research model. We fund basic research to investigate the biology and cause of SMA, in order to identify the most effective strategies for drug discovery.

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