Grant title: "In Vitro Studies of Cellular Interactions and Trophic Factor Receptor Function in a Cell Culture Model of ALS." (Co-funded by the Murray Abrams Memorial Research Fund). This project uses the highly technical advantage of studying neurons in a culture dish to understand what is going wrong inside the individual motor neurons and to test potential therapeutics that can keep motor neurons healthy.

Grant title: "A Novel Neuron Survival-Promoting sPLA2 Inhibitor as Treatment for ALS." (Co-funded by ALSA.) Inflammatory signals have been discovered in the spinal cord of people with ALS and in the animal model of ALS. Previous trials with anti-inflammatory drugs have not helped, but they also may not have been the right kind of drug. We are collaborating with Tim Cunningham of the Neurobiology Dept. at Drexel, who has developed a potent anti-inflammatory agent that shows effectiveness in other neurological conditions such as stroke and multiple sclerosis. We want to test this drug on our mouse model of ALS, and eventually, in our PALS.

This is highly speculative and early work, but we are developing plans with a biotechnology company in California to figure out how to get stem cells to grow into motor neurons and reconnect with muscles in the mouse model of ALS.

Grant title: "Genetic Analysis of ALS in Inbred Transgenic Mice." (Co-funded by MDA). This has been our main project for several years. The onset and progression of ALS is very different in different people, even if they are carrying the same mutation that causes ALS. This suggests to us that other genes may affect how (or whether) one gets ALS. The same is found in mice. We are mapping the genome of the mouse to look for the "good" genes that can slow down or stop the degeneration.

Probably one of the simplest but most important things we do is to maintain this colony of mice expressing mutant SOD1, so that researchers working on other diseases can easily switch to do pilot projects on ALS. In addition, we have bred a unique mouse with very few copies of the mutant SOD1 gene, making it a model for onset later in life and slow progression of disease.

Using the G93A Mouse in Pre-clinical Trials of Potential Therapeutics. There are many promising compounds that may have clinical relevance in ALS. The first step in evaluating these compounds is to test them in the mouse model of ALS. But how do you tell if a mouse is affected by ALS? Here is a description of the clinical criteria we use to assess the effects of the ALS-causing SOD1 mutation on mice: Lab Research