ALS Science

The Packard Center for ALS Research is devoted solely to seeking treatments and a cure for ALS through science. Through targeted projects, fast funding, and a collaborative research model, we’re able to accelerate progress toward effective therapies and treatments for ALS.

The ALS research funded by the Packard Center is divided into two categories: ALS Science and ALS Therapeutics. Each is dependent upon the other. The science helps guide us in discovering new ALS therapies, and our current therapeutic collaborations currently involve more than 50 neuroscientists, neurologists and cell biologists engaged in ALS research projects.

Strategic Approach

A board of external experts (Scientific Advisory Board) meets regularly to discuss the ALS research program and determine which scientific programs must be started to answer the most critical questions regarding ALS. This group also prioritizes therapeutic projects that can benefit patients most in the short term.

Quick Funding

Grants are short, specifically targeted to outstanding investigators capable of getting the job done, and are funded in a timeline of two to six weeks. Compare that to the peer review process of NIH, which takes a minimum of nine months to obtain funding and at least a month or more just to prepare a grant.

Collaborative Process

To advance ALS Science, all funded investigators must attend a monthly meeting to review their data among colleagues. This ensures rapid refinement, critique, better science and better progress. Failure to meet attendance records results in immediate withdrawal of all funds – investigators must show a dedication to the process and willingness to share information well before publishing.

Outstanding Scientists

Grants are by no means limited to Hopkins investigators; in fact, nearly 70 percent of the grants now go to non-Hopkins ALS scientists. This ensures that the best people to attack specific ALS therapeutics or pathogenic questions are assigned the task.

New ALS Research Projects

Novel Yeast and Zebrafish Models to Explore the Role of FUS/TLS in ALS
Using yeast cells as easy-to-study model systems, two scientists hope to learn how and why FUS/TLS protein forms clump within cells and how that’s related to motor neuron death... Learn More >>

Zebrafish Model as a Screening Tool to Identify Modifying Genes in Amyotrophic Lateral Sclerosis
Using a zebrafish model to study the mechanism of ALS, a gene which rescues the motor abnormalities induced by mutant SOD1 called ephA4 has been identified... Learn More >>

Generation and Integration of New Motor Neurons in the Adult Spinal Cord of Zebrafish
By identifying the molecules involved and showing how the mechanism of motor neuron regeneration in zebrafish can be controlled, our studies will inform future therapies aimed at rescuing or replacing diseased motor neurons in ALS... Learn More >>

Generation and Characterization of Wildtype and ALS-linked TDP-43 Mice
The recent discovery of mutations in a gene called TDP-43 (TAR DNA-binding Protein), linked to both sporadic and familial ALS, provides the opportunity to elucidate the underlying cause of sporadic ALS... Learn More >>