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Vaccine Being Tested To Prevent Dementia In People With Down Syndrome

BOSTON (CBS) - Local researchers are testing an experimental vaccine that may not only prevent Alzheimer's disease in people with Down syndrome, but in those without Down syndrome as well.

People with Down syndrome are at high risk of developing Alzheimer's-like dementia at a very young age.

Michael Clayburgh, 29, has Down syndrome, but that doesn't hold him back.

"Michael has three jobs," says Nancy Novelline Clayburgh, Michael's mom.  "He works at Target, McDonald's and Dunkin Donuts."

Michael Clayburgh
Dr. Brian Skotko and Michael Clayburgh (WBZ-TV)

He's also quite the Special Olympics athlete, but perhaps Michael's greatest contribution will be a scientific one.

"He could be a major factor in finding the cure to Alzheimer's," explains Nancy.

People with Down syndrome have an extra copy of chromosome 21.  One of the genes on that chromosome can lead to plaque build-up in the brain.

"That goop in the brain when it starts to come together can really disrupt neuronal function and lead to the dementia that's associated with Alzheimer's," explains Dr. Brian Skotko, director of the Down Syndrome Program at Massachusetts General Hospital.

He says up to 50 percent of people with Down syndrome will develop dementia by age 50.  He worries about his 38-year-old sister, Kristin, who also has Down syndrome.

Dr. Brian Skotko
Dr. Brian Skotko and his sister Kristin (WBZ-TV)

"Because of her I wanted to become a doctor, and one day I wanted to be able to do research for and with people with Down syndrome," says Dr. Skotko.

He is studying a vaccine designed to harness the body's own immune system to gobble up plaques and prevent the onset of dementia.

Mass. General and two other centers are recruiting up to 24 patients in a Phase I clinical trial testing its safety. Michael is one of those patients. He gets an injection every one to two months followed by blood tests and MRIs.

The study is being funded, in part, by a nonprofit organization called LuMind RDS Foundation.  CEO Hampus Hillerstrom's 4-year-old son, Sebastian, has Down syndrome.

"If my son could start taking it early on and delay the onset of Alzheimer's by decades, it would be wonderful," says Hillerstrom.

Dr. Skotko agrees.

"On a personal level there is nothing I want more for my patients and my sister as to one day have a vaccine, have a cure, to prevent Alzheimer's disease from happening," says Dr. Skotko.  "They work too hard.  They have too much potential. They're too wonderful people to have it all go away due to Alzheimer's."

And if Michael does develop Alzheimer's, Nancy says they will cross that bridge when they come to it.

"Hopefully, it will be 10 to 15 years from now," she says, "And this vaccine will be available to him and he'll be all set. Let's hope."

If all goes well with the clinical trials, Dr. Skotko says this vaccine could become available in the next 10 years or so, not only to try to prevent Alzheimer's disease in patients with Down syndrome, but in the general population as well.

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