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In This Issue:

Drug Trinity Shows Unexpected Strength
Jean-Pierre Julien—his “cocktail” does wonders for ALS mice.

From Iceland: A New Way to Decode ALS Genes
In Iceland, a country of roughly 300,000 citizens, ALS is pretty much unknown. If you wanted to find genes tied to that disease, it’s an unlikely spot for a search. But from that country may come, if not the genes themselves, a superior way to track them down.

Getting to the
Heart of It

With ALS, many of the simplest questions remain unanswered. ‘That just won’t do,’ say Center scientists.

One Step Closer to the Bedside
The Basics Bolster Stem Cell Therapy.

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From Iceland: A New Way to Decode ALS Genes

photo - Jonathan Glass convinced deCode to hunt ALS genes.  
Jonathan Glass convinced deCode to hunt ALS genes.

In Iceland, a country of roughly 300,000 citizens, ALS is pretty much unknown. If you wanted to find genes tied to that disease, it’s an unlikely spot for a search. But from that country may come, if not the genes themselves, a superior way to track them down. In March, a Packard Center scientist announced a project with an Icelandic company noted for its high success rate in finding genes for more common diseases.

Neurologist Jonathan Glass aims to isolate genes that either cause ALS or that may predispose people to get it. He has Center funding for his work with deCode Genetics, a genomics firm that, so far, has singled out genes involved in more than 20 illnesses. DeCode specializes in widespread diseases such as Parkinson’s, stroke, heart disease and schizophrenia.

The company takes advantage of Iceland’s small population—one that, because of centuries of relative isolation, has less genetic variation than many other countries. The underlying principle is that it’s easier to find a needle in a haystack if the haystack’s small and uniform.

“But for ALS research,” says Glass, “we’re more interested in the gene-spotting tools deCode’s developed than in the Icelandic gene pool.” DeCode has isolated more than 20,000 markers—distinctive patterns in the human genome—that serve as reference points. By collecting a person’s DNA and highlighting the markers, you get a distinctive genetic fingerprint, Glass says. The hope is to find that similar fingerprints pop up again and again in American ALS patients but not in those without the disease. That would signal a specific stretch of DNA as suspect, one that might carry a gene that needs to be investigated.

This summer, deCode allied itself with the Emory University School of Medicine, where Glass is a clinician-researcher, to test their findings for common diseases on a U.S. gene pool. “Even though ALS isn’t what you’d call common,” Glass says, “we approached deCode about doing it, and now we have a side project that will involve other Packard Center clinician-scientists and their patients across the country.”

ALS doesn’t look like a genetic disease, he says, aside from rarer inherited versions. “Yet even sporadic disorders like Alzheimer’s have genes that predispose people to the disease. We’d like to see if that’s the case here. It would put us that much closer to therapy.”

The next task is to collect blood samples nationwide from 400 patients and their parents. Parental DNA is particularly necessary to tie specific stretches of the molecule to ALS. “We’ll let people know when that starts,” says Glass.

Next > Getting to the Heart of It
With ALS, many of the simplest questions remain unanswered. ‘That just won’t do,’ say Center scientists.


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Special Features:

Vantage Point
With approaches to ALS therapy, progress seems to move one step forward and a half step back.

On Center
Runners, Walkers Make Feet Fly to Help the Center.

Rich Soil for a Blooming Friendship
“About six years ago, I ran into Laura and I could tell something wasn’t right,” says Coleman, who now directs the New York State Trial Lawyers Association. “When I heard later she had ALS, I felt a bit strange in writing a personal letter out of the blue, but I sent it anyway.”

Insider's View
Noah Lechtzin, M.D., is a Hopkins pulmonologist who sees ALS clinic patients regularly.

From the Clinic
Lora Clawson, M.S.N., C.R.N.P., manages Johns Hopkins’ ALS clinic, including its clinical trials. In this column she answers typical patients’ questions.

A Friend Indeed
Ride for Life Keeps Center Rolling.

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