Research Objective:
This 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.
This grant to Dr. Ko at the University of Southern California will help in understanding 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:
-The cause of SMA in now known. Which means that treatments can be developed that correct the underlying cause of the disease rather than just reduce symptoms.
-A back-up gene for SMA has been identified. Which means a straightforward drug target is already in the body: a built-in switch for new therapies to work on.
Using this knowledge, there are now 3 clinical trials testing new SMA therapies, and an additional 10 programs in earlier stages of the drug development pipeline.
