ALS Research Vision
From the beginning, those who started the Robert Packard Center for ALS Research at Johns Hopkins have believed the key to research for the cure is synergy. While the Center's individual projects have merit – they're all worthy of funding – the sessions where scientists share ideas will likely make the difference.
Most NIH program-based projects require hours of collaboration in the planning stages. Yet that typically drops off once funding begins and researchers have less incentive to work together. Although the Center supports more than 100 scientists, technicians and lab staff, its members collaborate throughout projects. The spark for this comes, in part, from regular monthly investigators' meetings – required of Center scientists – and yearly symposia. It also comes from central planning that insures that each lab works on a crucial piece of the overall puzzle.
Approaches to Disease
The work of Center scientists takes one of two approaches.
In the first, researchers look for the basic molecular or cellular cause of the disease. They use a variety of strategies, including:
- • Cloning disease-related genes
- • Isolating the proteins that faulty genes encode and clarifying their effects on normal cells
- • Creating cell models of ALS by knocking out possible key genes or adding specifically mutated ones
- • Mapping the pathways of decline in cell models
- • Identifying distinct genes that control the progression of ALS or that could somehow alter it
The second approach centers on a search for new therapies or ways of attacking the disease. The Center's clinicians work with basic researchers, moving their most promising therapies from cell and animal models on into human clinical trials. The clinicians stand at the forefront of efforts to slow, halt or cure this devastating disease.
To maintain focus and inspire advances, the Center's panel of Scientific Advisors – experts in motor neuron disease and cell death – review studies' progress twice a year. In addition, other selected experts offer their expertise at the Center's monthly meetings, annual retreat and symposium.