Families of Spinal Muscular Atrophy Awards New Research Funding of $75,000 to Dr. Chien-Ping Ko at the University of Southern California.
December 17, 2012.
Families of SMA is dedicated to creating a treatment and cure for Spinal Muscular Atrophy by funding and advancing a comprehensive research program. This new research funding will support a project at the University of Southern California to better understand the role of astrocytes in SMA disease pathology.
Description of FSMA Funded Project: The role of glia cells in SMA. Objective: The project will investigate the involvement of a type of glial cells called astrocytes in SMA pathogenesis. Astrocytes are star-shaped glia located in the central nervous system (CNS) that hold neurons in place, get nutrients to them, and digest parts of dead neurons. It has been discovered that astrocytes can communicate with neurons and modify the signals they send or receive. This means astrocytes have the potential to be involved in the processing of information and signaling that occur at the synapse. Research Strategy: The researchers will use genetically engineered mice to restore or reduce SMN expression selectively in astrocytes. They will also use cell-based assays to study the mechanisms of neuron-astrocyte interactions in SMA.
Earlier this year, FSMA's Advisory Boards met to evaluate new research funding for 37 basic research grant applications and 7 drug discovery projects for SMA. The organization is planning to award $1.4 Million in new research funding over the next few months. This new round of research funding will be allocated into three areas: 1) Basic Research to understand the disease and provide ideas for drug making, 2) Drug Discovery to develop new SMA therapies, and 3) Clinical Research to help test new drugs effectively and to improve care for patients. Click here to see more details on the process.
Our research progress provides us all with hope that one day we will live in a world without SMA. Your gift will make that hope a reality. Click here or the image below to donate and support Families of SMA research and services.
Who are you? My name is Chien-Ping Ko. I was born in Taiwan and came to the US in 1971. I received my PhD in physiology from Washington University in St. Louis, and did my postdoctoral training in neurobiology at the University of Colorado and then the National Institutes of Health. Since 1981, I have been a faculty in the Section of Neurobiology, Department of Biological Sciences, University of Southern California, Los Angeles, CA.Click here for Dr. Ko's webpage. How did you first become involved with SMA research? I have long been interested in how neuromuscular junctions (NMJs) work, form and maintain. In the middle of 2000s, my research group became interested in whether and how NMJs might be involved in motor neuron diseases. We first studied amyotrophic lateral sclerosis (ALS), a late-onset motor neuron disease that affects adults. By reading literatures on various motor neuron diseases, we were intrigued by the possibility of NMJ involvement in spinal muscular atrophy (SMA). Given that the genetic basis of SMA has been much better characterized and various animal models are available, I thought that my expertise in the NMJ might contribute to better understanding of whether and how defects in NMJs and other nerve connections (called synapses) may play a role in SMA. What is your current role in SMA research? We have recently found that NMJs in several muscles (such as neck muscles) are severely defective and that synapses from proprioceptive sensory neurons onto motor neurons in the spinal cord are drastically reduced in a mouse model mimicking the severe type of SMA. We are now using NMJs in these vulnerable muscles and central synapse loss as benchmarks to test in vivo the effectiveness of certain candidate compounds in this severe mouse model. We are also characterizing NMJs and central synapses in several new mouse models that are less severe. In addition, we are testing whether and how glial cells may play a role in the pathogenesis of SMA. Our ultimate goal is to better understand the fundamental biology of synaptic defects and hope to find treatments to restore these defects in SMA.
This grant to Dr. Ko at the University of Southern California will help us understand the role of support cells that interact with motor neurons in SMA. This will help answer the key basic research question of what tissues are affected by reduced SMN protein?
The basic research that Families of SMA has funded, through 145 research grants to 75 institutions around the world, has delivered major discoveries: -We know the cause of SMA. Which means we can develop treatments that correct the underlying cause of the disease rather than just reduce symptoms. -We identified a back-up gene for SMA. Which means we have a straightforward drug target already in the body: a built-in switch for new therapies to work on. Using this knowledge, we now have 3 clinical trials testing new SMA therapies, and an additional 10 programs in earlier stages of the drug development pipeline.