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Jeffrey D. Rothstein, M.D, PhD., the Director of The Robert Packard Center for ALS Research
Jeffrey D. Rothstein, M.D, PhD., the Director of The Robert Packard Center for ALS Research


The Robert Packard Center for ALS Research is an organization founded in 1999 which seeks slow or cure amyotrophic lateral sclerosis, or Lou Gehrig's Disease. Although the Center operates within the Johns Hopkins School of Medicine in Baltimore, its scope is international.

ALS is a neuromuscular disease characterized by a progressive degeneration of motor nerve cells that causes muscles to weaken, then waste away (atrophy). Intellect and the five senses remain. It crosses all socio-economic, race and ethnic lines, occurs spontaneously and has no cure. Although traditionally a disease of middle age, recent clinical observations show an increase in younger patients. This potential trend, plus evidence ALS affects military personnel at twice the rate of the general population, warrant further study.

Contents

[edit] Research

Hopkins-sponsored research on how to stop ALS, repair its damage or even prevent it is carried out under a unique blueprint that’s taken shape as The Robert Packard Center for ALS Research at Johns Hopkins. The Packard Center is the only institution of its kind dedicated solely to the disease. Over 100 scientists from Hopkins, other universities, government labs and biotech companies work together, sharing insights and materials to understand the biology of ALS and find a cure.

Housed at Hopkins’ East Baltimore campus, the Center is supported largely through private philanthropic funding, as well as more traditional grant support from the National Institutes of Health. It began as the brainchild of the Neurology department’s ALS researchers, especially the Center’s present director, Jeffrey Rothstein. They met with a small group of patients and philanthropists as well as forward-thinking Neurology administrators, to open the Packard Center in 2000.

By emphasizing teamwork and sharing—with no need to wait until lab results are published—by requiring progress, meeting milestones and inviting open collaboration, and also by carefully selecting the most promising areas of research and top scientists in those areas, the Packard Center has advanced the field dramatically.

Its scientists have helped uncover new genes for familial ALS. They’re actively working on genetic risk factors—reasons why patients with sporadic ALS may be susceptible to the disease. They’ve developed a necessary variety of animal models for testing therapies and exploring ALS biology. Also, they’ve shed light on nerve repair, uncovered natural, built-in nerve protective systems, advanced stem cell therapy and are studying natural ways to counter muscle atrophy. Concerned about more immediate help for patients, Center scientists have screened potential new drugs and are ushering them down the testing pipeline. The first five years have seen more than a dozen clinical trials for candidate drugs or other therapies.

Finally, Packard Center scientists have become world-respected in increasing understanding of what ALS does to cells. Both that knowledge, along with the Center’s drug discovery program, extend insight and treatment possibilities for other neurodegenerative diseases including Parkinson’s disease, Alzheimer’s disease, spinocerebellar ataxia, Huntington’s disease, multiple sclerosis and the peripheral neuropathies.


[edit] Achievements

1993

Hopkins scientists advance excitotoxicity theory of ALS

1994

Hopkins scientists learn how SOD1 mutation causes cell injury.

1999

A cluster of scientists opens the Center for ALS Research

2001

First rat model of ALS developed, with Wyeth labs

Mutant SOD1 kills neurons in fALS subtype

Astrocytes implicated in ALS process

First use of "doctored" stem cells to protect motor neurons (ALS models)

Nervous system inflammation advances ALS (models)

2002

First ALS2 animal model developed

Key discovery that bad cell environment advances motor neuron death

Discovery that healthy glial cells protect against ALS

ALS disrupts cells' internal movements

First use of human embryonic stem cells to protect model rats from neurodegeneration.

2003

Neuron death begins well before ALS symptoms

Motor neuron death begins at nerve endings in muscle

First major screening of FDA- approved existing drugs yields ceftriaxone

2004

ALS4 mouse model created

New technique developed to study motor neurons' neighbor cells

Inflammation/microglia are key players in ALS process

ES stem cell-derived motor neurons form whole, working circuits

Antisense technique slows disease in animal fALS models

Agent pleiotrophin enhances motor neuron regrowth

VEGF greatly lengthens lifespan of ALS mice

2005

Dynactin mouse model of ALS created

Zebrafish model of ALS created

ALS spreads from within motor neurons to neighboring cells

ALS appears to start early in motor neurons

Mutant, toxic SOD1 protein kills motor neurons from outside cells.

First massive screen for sporadic ALS genes begins

2006

Fine-tuning of glutamate receptor structure goes awry in ALS, making motor neurons hypersensitive

Muscle involvement in ALS occurs earlier than suspected

[edit] Fiesta 5 K

The center launched its in augural Fiesta 5 K in May 2007. The race has been well recieved by the Baltimore community and is widely attended by ALS patients, cartakers, and families.

In 2008 the race had over 800 participants and raised more than $ 180,000. O.J. Brigance, former linebacker for the Baltimore Ravens and ALS patient served as honorary chair for the event.

[edit] References

http://www.alscenter.org/

http://support.alscenter.org/site/TR?fr_id=1050&pg=entry

http://www.hopkinsneuro.org/team_member.cfm/expert/Jeffrey_Rothstein


[edit] External links

en:Amyotrophic lateral sclerosis