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Our Approach

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Our Research Vision

From the beginning, those who started the scientists of the Robert Packard Center for ALS Research at Johns Hopkins have believed the key to research for the cure is synergy. While all the Center's individual projects have merit – they're all worthy of funding – but the trail to success can only be forged by the sessions where scientists share ideas, successes, and even difficulties. 

Most NIH-funded  program-based projects require hours of collaboration in the planning stages. Yet that typically drops off once project 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 that are required of Center scientists, as well as 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 or animal 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 or otherwise affect 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' grantees' progress twice a year. In addition, other selected experts offer their expertise at the Center's monthly meetings, annual retreat, and symposium.

ALS Strategy

Sharper focus may be the key to finding a therapy for ALS. Historically, research efforts have been fragmented. While scientists worldwide have advanced knowledge of the disease, it’s often in a piecemeal fashion. Packard works under a different plan: choose the best investigators in different aspects of ALS pathology and assign their work as part of a broad but rational attack. That plan has four aspects that our Center facilitates.

Recruit Committed ALS Investigators Worldwide

The Packard Center comprises consists of a group of committed scientists throughout the world who tackle key questions on the cause and treatment of ALS. To support their studies – sometimes work not directed specifically toward ALS – the Center provides expertise, advanced technology and a collaborative spirit.

Integrate Investigative Knowledge

The Center's Scientific Advisory Board steers investigation into novel areas with the potential to advance ALS research. That board, the Center director and science director, as well as outside collaborators, suggest a rightthe best course for testing the potential therapies that result.

Foster Discourse and Collaboration

Since its the beginning, the Center has held monthly meetings for its scientists, offering updates on new approaches to ALS and the chance to brainstorm and evaluate each others' work. Researchers present highlights of each year's work at an annual symposium.

Channel Basic Science Findings into Clinical Investigations

All of the Center's studies are meant to translate from bench to bedside in rapid time. Even basic science studies have begun with an eye onto clinical trials at a later date. Because many of our physician-researchers also staff Johns Hopkins ALS Clinic, a well-respected facility at the East Baltimore campus, recruiting for trials is relatively easy.

Together, we can reach that goal.

Steven Finkbeiner

Our Experts

Gladstone Institutes, UCSF
Two recently discovered genes that have been associated with both familial and sporadic forms of ALS encode the related proteins TDP43 and FUS cause neuron death in ALS.
Meet Our Experts