Alzheimer’s Prevention Drug Trial

May 16, 2012

Transcript:

Ted Simons: The Phoenix-based banner Alzheimer's institute is leading a groundbreaking new study. It involves testing an experimental drug on otherwise healthy people who have genetic mutation that makes it highly likely that they will begin to suffer from Alzheimer's symptoms by the age of 45. Here to talk about the test is Dr. Pierre Tariot, director of the banner Alzheimer's institute. thanks for joining us.

Pierre Tariot: Thank you.

Ted Simons: this is fascinating. Let's talk about this drug to be studied. Promising?

Pierre Tariot: Very promising. It has a name. It's a kind of immunotherapy, a way to stimulate the body's immune system to fight off bad proteins that cause Alzheimer's disease.

Ted Simons: does it prevent or delay onset or both?

Pierre Tariot: That's a great question. It's being studied now in people who already have the crippling symptoms of Alzheimer's disease. What we're proposing for the first time in history is an unprecedented effort to treat people who are DESTINED with 100 PERS certainty to develop the illness before any symptoms begin.

Ted Simons: and this is absolutely fascinating. These people that will be tested are an extended family from Colombia?

Pierre Tariot: We're mostly studying people in Colombia, some in the United States. There's an extraordinary group of people numbering as many as 5,000 who are distantly related to ancestors who carry this very rare causative mutation. Flash forward a few hundred years, 5,000 living within about 100 miles of each other, perhaps a third carry this mutation and will develop the disease unless we can do something to stop it.

Ted Simons: How was extended family found?

Pierre Tariot: Our colleague, when I say our, I'm referring to my friend and colleague Dr. Eric Reimann, who is co-director of the Alzheimer's prevention initiative with me, connected with this wonderful neurologist in Colombia, Francisco LAPERA, who beginning about 20 years ago realized there was a large number of families dealing with these issues. Over time studied them, eventually got some cutting edge genetic testing done and this unique mutation was found. He has been following them for years. They have been waiting for years for this kind of step to be taken.

Ted Simons: Now, how will the tests be conducted?

Pierre Tariot: We are enrolling people into a registry down there. We already have almost 1500 enrolled. Go through a similar process in the United States. As soon as we have approval from the appropriate government agencies, we will ask people if they want to participate. Those who do will be screened for appropriateness and then they will be given a shot under the skin every two weeks. The shot will either contain the active treatment or a placebo in the group of people who are carriers of the mutation. Then we'll also include people who are noncarriers who will only get the placebo and the reason we have that element to the design is because people right now don't want to know. Don't want to know.

Ted Simons: I was going to ask about the ethical considerations here. Not only people knowing and not knowing but giving placebos to folks DESTINED to have this.

Pierre Tariot: if we knew the treatment worked we wouldn't do the study. It's mandatory to compare active treatment with placebo. Show that you can see a difference. Actually ironically using placebo will probably give us the answer sooner. We can detect very subtle loss of memory and thinking ability now. We have already developed that tool. So if the treatment blocks that subtle decline we know we're on to something. We'll get that answer pretty quickly.

Ted Simons: talk about how these people will be tested. I'm imagining cognitive tests. Will there be clinical tests, the brain?

Pierre Tariot: You got it all. We will be doing very sensitive tests of memory and thinking, asking how they are functioning, we're very interested in safety. We'll be asking about that we'll be Doan brain scans to look at brain structure, how the brain uses fuel, sugar, and how the proteins are being deposited or not deposited and finally looking at spinal fluid and blood chemistry. A whole bunch of out comes.

Ted Simons: the plaque in the brain can that be looked at? Can you see that?

Pierre Tariot: Thanks to work done at banner and by my colleague Eric Reimann and the rest of the team, that technology now exists. So we're looking to see if we can prevent that from happening.

Ted Simons: any side effects with this drug?

Pierre Tariot: The reason we chose this one is that it seems to be remarkably safe and well tolerated. So far in tests with people there have not been any major adverse events. Never say never. We have to be very scrupulous in following people, but it looks promising in that regard.

Ted Simons: So when you begin to administer the shots, at what age do you start?

Pierre Tariot: We're starting in Colombia in folks age 30 and above who are in these families. That puts them within 10 to 15 years of developing the usual symptoms of Alzheimer's disease. Obviously there will be people older who will also be enrolled and they will be even closer.

Ted Simons: Well, -- will there be people enrolled who have symptoms already?

Pierre Tariot: No. This study will focus on people who are cognitively healthy. The whole idea is can we block these devastating manifestations from happening in the first place.

Ted Simons: That's got to be tough for some of the folks, especially with early onset to think there's a test but they can't be part of it.

Pierre Tariot: there are treatment studies going on all over the world for people already affected including with this therapy. There isn't one at the moment in Colombia.

Ted Simons: The cost. What cost are we looking at here?

Pierre Tariot: North of $100 million.

Ted Simons: my goodness. Where is the money coming from?

Pierre Tariot: So banner health has a foundation on behalf of the banner Alzheimer's institute and some remarkable donors have banded together to provide about $15 million in support to help make sure that this is handled in a particular way. Yesterday the government announced $16 million grant through the national institutes of health. The rest of the tab will be coming from our wonderful pharmaceutical partner, GENENTEC in San Francisco.

Ted Simons: maker of the drug.

Pierre Tariot: maker of the drug.

Ted Simons: The tests will start when?

Pierre Tariot: Well, the planning started four years ago. The grant writing has been ongoing. The planning has been intense. We now go through a period of very, very rigorous government approval. That will take many months. We hope to start treating the first individuals within a year or so.

Ted Simons: And it sounds like this could be somewhat open ended. Is it an open-ended test? Do you know how long this will last? If someone is not developing -- when do you know they won't develop symptoms with this drug?

Pierre Tariot: We actually think that we can get an initial read after each person has been treated for as little as two years. We're going to take a preliminary look then. We're quite confident that by the time people have been treated for five years we'll definitely know whether we're on to something or whether we should bark up another tree.

Ted Simons: last question here. From a distance, this sounds remarkable. This sounds like an opportunity literally of a lifetime. You say this is a big deal. How big a deal this is?

Pierre Tariot: This is unprecedented, something like this has never been attempted before. It definitely puts Arizona on the map. Puts banner on the map. We're very excited about the possibilities and we're also very humbled at the responsibility that we face.

Ted Simons: it's a very encouraging report. We'll keep an eye on it and hope to have you back soon. Thanks for joining us.

Pierre Tariot: Thank you very much.