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Doctors explain battle to beat Alzheimer's

By Felipe Barral, CNN
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Reducing risks of Alzheimer's disease
  • Research in Colombia could pave the way for treatments of Alzheimer's disease
  • Dr. Kosik: The numbers of Alzheimer cases on the horizon is very worrisome
  • Dr. Tariot: I can imagine a future where we might intervene in children to prevent Alzheimer's
  • Dr. Reiman: Given the stakes and opportunity I can't think of anything more exciting

Editor's note: A small Colombian community could hold one of the keys to finding treatments for Alzheimer's disease. Watch "World's Untold Stories" January 29 and January 30.

Antioquia, Colombia (CNN) -- In the Colombian region of Antioquia, members of 28 extended families develop early-onset Alzheimer's in their 40s.

Here scientists explain how the Colombian link could provide clues to preventing the disease and achieving the dream of a world where Alzheimer's can be beaten. Initially they say their ideas were considered crazy but now the scientific community is watching closely.


Q: What's important about the Alzheimer's found in this one region of Colombia?

There's a gene here where people are getting the disease generation after generation because they are getting a mutation in one of their genes. It gives us a very big clue in being able to predict who's going to get it.

We have a large family who are related to each other. They all have the same problem and scientifically this is very powerful. This is absolutely unique to have a population that all came, probably from a single founder.

What we now have is a large group of over 5,000 people, the progeny of that first founder, many of whom suffer from Alzheimer's.

One possibility is that the individual, the family from which that first person came, that family died out. Another possibility is that the gene was here from the indigenous population and the Spanish invader then gave it to his children through the indigenous population.

Doctor: Genes key to Alzheimer's study
Testing the difficulty of memory loss
Gallery: Filling the blank
As the baby boomer bubble is moving into the age of retirement, the incidents of Alzheimer's Disease will increase
--Dr. Ken Kosik

Because there are two mountain ranges on either side of Antioquia, the people have not moved very far away and therefore the largest concentration of these families is right here.

Q: Could the Colombian experience provide a possible treatment?

We don't know the answer to that but almost all of the research going on, both in universities and pharmaceutical companies, stems from insights that not just this family, but other families like it, where we have genetic information.

To develop a program here that will ultimately result in a treatment, one needs a lot of moving parts and many of those parts (include) sophisticated technology.

The Alzheimer's field has advanced very rapidly in terms of the way we can analyze and evaluate people, all the tools we have from genes to MRI.

Q: How did you meet Francisco Lopera?

I had been working on Alzheimer's and came to give a lecture in Bogota. After the lecture, Francisco came up to me and tugged me on the sleeve and said: "I want to tell you about some people with Alzheimer's.

I confess that for the first few moments I did not realize how important what Francisco was telling me really was. But he was persistent.

It didn't take long before I realized he was saying something that was very different than anybody else was telling me, and we just jumped on the first plane from Bogota. That was about 15 years ago.

Q: What are you learning about genetic information and mutations?

One of the things that has become apparent is that in these genes that carry mutations in early onset Alzheimer's, there are many different sites within those genes that can be mutated.

It's remarkable that almost everything that we know about Alzheimer's disease... stems from a few individuals with rare mutations like those in Colombia, and they have pointed us towards the targets around which we are now designing therapies and treatments.

We have brought together the leading edges of technology and meshed that with people who are still living under developing conditions but have a disease, a genetic form of Alzheimer's disease, which holds some secrets that I think can be extraordinary helpful for everybody with Alzheimer's disease.

Filling the blanks: Dreams of a cure

Q: How urgent is finding a cure?

As the population is aging, and the baby boomer bubble is moving into the age of retirement, the incidents of Alzheimer's disease will increase.

It will increase because the greatest risk factor for Alzheimer's disease is age, and since more and more people are living to advanced age, we are seeing and will continue to see a lot of Alzheimer's disease.

The numbers of Alzheimer cases that are now on the horizon is very worrisome and I hope that we will be better prepared than we are now.


Putting the puzzle together

Q: What stage is the work at, what could be the future?

(We want to) move the field to the point where many of us are asking and answering these questions: Can we intervene before symptoms develop and can we slow this process down?

There are these stages of a good idea in science -- first everyone tells you that you're crazy, and then they say it's not crazy but here are all the things wrong with it.

Then they say well it's obvious, and then the fourth stage is where it becomes a requirement.

We're at the point where it's gone from "you're crazy" to "it might be a good idea but here are all the things that are wrong with your idea."

To me it seems like the border of a large puzzle has been completed and now we can complete the interior of the puzzle.

Will any of our therapies help an individual put the puzzle piece back in? I hope so. I'm not gonna abandon that hope at all.

But it seems more likely that if we could prevent the pieces from disappearing in the first place that... that's more likely to happen, more likely to effective.

I could imagine a future where we might intervene in children to prevent Alzheimer's disease.

Right now we'd say it's incurable, it's not untreatable but we don't know how to cure it, we don't know how to prevent.

We're doing treatments studies, we're gearing up for prevention studies. We really hope they work. But if you go all the way in the other direction, it's not inconceivable that in 50 years, we'll know how to vaccinate 12-year-olds.


Imaging techniques for Alzheimer's

Q: How does brain imaging fit into this?

In 1993 researchers identified a common gene that one in four of us have that increases a persons risk for Alzheimer's disease.

We've been able to detect some of the same brain changes we see in patients with Alzheimer's disease -- maybe 50 years before the onset of symptoms.

We've been able to track some of those changes in middle-aged people a couple of decades before the onset of symptoms.

And we estimate that instead of needing to study 50,000 people over 20 years starting in middle age to test a prevention therapy, maybe we could do the same study with just 200 people at higher risk in two years.

So, what if we could study a different prevention therapy every two years and find one that works within 12 years. Wouldn't that be great?

Q: How can you assess possible prevention therapies?

We propose the idea of doing a study every two years of a promising treatment in people who, based on their age and genetic background, are close to developing symptoms.

If after two years we see no effects on our brain imaging or biomarker measurements the study is discontinued and the family members get access to the next most promising treatment.

But if there's an effect on these biological measurements, continue the study just a little bit longer to show that it slows down some of these memory declines.

If we can do that, we would have found a treatment to reduce the risk of Alzheimer's disease.

Q: Where does the research go next?

Given the stakes and the opportunity at hand we can't think of anything more exciting.

We're beginning to see a groundswell of interest of researchers around the world who've begun to think about using biomarkers in prevention trials.

What we've proposed doing is; include all of the promising biomarkers, as expensive as that is, in the same study to determine whether they are budging in the right direction, to compare them in terms of their ability to budge in response to treatment, and to compare them in terms of their ability to predict a clinical outcome so that we could have just the right biomarker for other prevention studies.

We're excited about that.

Now is the time to launch the era of Alzheimer's prevention research, to establish the sense of urgency to address this problem starting now.