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Packard Center for ALS Research at Johns Hopkins

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ALS Alert Newsletter | June

ALS in the U.S.A. — the Stats and More

The new National ALS Registry looks like it has the right stuff.

New National Registry

We’re all too aware of the classic “black boxes” of ALS — the core blank spots in our knowledge of how the disease starts and progresses and, of course, the maddening unknowns about how to cure it.

But other crucial questions also go unanswered: How many people in this country have ALS? Is there something in the environment that predisposes someone to get it? Does lifestyle affect the risk of ALS? How about having infections or other illness at the same time? Can that change survival?

And it’s these latter mysteries — the relatively lower hanging fruit — that are finally being responsibly addressed in a large-scale, concerted effort that kicked off in October of 2008 when Congress passed the ALS Registry Act and approved close to $16 million to fund it.

Actually, the drive for the program began several years before that, when members of ALSA, MDA and other patient advocacy bodies realized the huge value that having more accurate disease demographics would offer “black box” researchers like Packard Center investigators.

Lucie Bruijn“Before the bill was signed,” says Lucie Bruijn, ALSA’s Chief Scientist and a frequent Packard consultant, “our effort went into getting the government to start the project and into making sure the right people would be brought on to design something scientifically useful.” The advocates held up the existing Veteran’s Administration databases, for example, as a place to start.

The resulting act mandated a national, government-based program to settle questions of ALS’s incidence (how many new cases each year) and prevalence (how many U.S. citizens have it) as well as to collect patient data that could shed light on causes and risk. A quieter goal is to raise patients’ and physicians’ knowledge of the disease, either directly or through helpful links — payback of sorts for signing on to the Registry.

Run by the government’s Agency for Toxic Substances and Disease Registry (ATSDR), a sister organization of the Centers for Disease Control, the database-making program took several years to shape before it officially opened for patient signup this past October. “There was, admittedly, frustration on the part of patients that this didn’t happen faster,” ATSDR’s Project Leader D. Kevin Horton explained on a recent MDA-sponsored webinar. “But that was for a good reason,” he says. “It takes time to test the methods we used. We had to be sure of accuracy…And we’ve done our homework.”

The Chief Concerns

The two main concerns, says Bruijn, have centered, and still do, on ensuring a large enough number of reports from people throughout the country — across all states and walks of life — and on making sure that people reporting genuinely have ALS. Early pilot “surveillance” studies in three states plus the nationwide HMO, Kaiser Permanente, convinced the Registry’s designers that their project was do-able.

As anyone who’s checked the present Registry site can see, data are mostly pulled in from several sources: patients who type in their particulars at the website and large, existing medical databases. The latter come from the VA, Medicare and Medicaid. Having different sources, of course, increases Registry entries. But they’re useful for another reason. “There’s had to be a system of checks and balances to help verify patient responses,” Bruijn explains.

Cross-checking avoids entering a patient more than once. “We still encourage patients to self-register, even if they’re not sure if they’re on other existing databases,” Horton says.

More From the Registry

Researchers already know that the sort of epidemiological approach the Registry takes can shine light in dark corners. Two years ago, for example, the Department of Veterans Affairs classed ALS as a “service-connected disease” based, in part, on a Harvard study that showed American military are at greater risk of ALS. The raw data were from a bank of demographics collected by the American Cancer Society.

It seems reasonable, then to try to answer basic questions of incidence, prevalence and risk. But Registry planners also plan to tackle slightly more nuanced topics such as the connection between early ALS and other motor neuron disorders sometimes confused with it. And beyond the original goals, Bruijn says, once the patient privacy-protected data are open to scientists in a few years, “we can start thinking of creative ways to ask how ALS progresses to different parts of the body, for example, or the role of upper vs. lower motor neurons in the disease.”

Researchers have still another wish list, Bruijn adds. “The scientific community has been pushing for the Registry as a way to increase participation in clinical trials.” Already, the Northeast ALS Consortium, a planning and clinical trials advocacy body with close ties to Packard, has weighed in, she adds. And Horton says the ATSDR has plans in that direction that “will be in place by late summer.”

Last, Bruijn and others have held a hope that the Registry’s movers-and-shakers would build a biorepository onto the project — a place to store saliva, blood plasma and other tissue samples. “It could speed our discovery of a biomarker for ALS," she says, "not to mention help the search for genes that increase susceptibility to the disease.” That may be coming. Later this year, the ATSDR starts a small-scale feasibility project for collecting and storing ALS patient samples.

But for now, the prime work is publicity. Thousands of patients nationwide need to sign onto the Registry for it to work. “It’s only as good as everyone who registers,” Bruijn emphasizes.

Have you signed up yet?

ALSO In this Issue

Q & A on The National Registry

Kevin Horton, project leader for the National ALS Registry gives us an update.

2011 Fiesta 5K
Now that the 5K Dust Has Settled . . .
Is it our best ever? Yes.

5K Live
An Insiders Look at the Packard Center's Signature Event
Hear from our runners, team captains and staff - see why 2,300 participants said olé to the 2011 Fiesta 5K.

Read our Q&A to see where the ALS Registry is headed.

Sign up for the National ALS Registry at:

For more about the Registry, check this ALSA site:

-Marjorie Centofanti

Johns Hopkins School of Medicine

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