Promising clinical trials for Alzheimer's

Carrie Richardson is 44 years old. Arms out straight.

At 41, she developed early-onset Alzheimer's disease. The condition is caused by rare genetic mutations that essentially guarantee a person will develop the disease. But you always know you can count on me.

Her mother, Mary Salter of Montgomery, Alabama, knows the toll of the disease all too well. Seven so far. I've lost seven family members, from the ages of 37 to 44.

Including her son, Bryan, who died last year. Remember the first time we came to WashU? Bryan was with us.

Mary, Carrie, and Carrie's daughter Hannah have been coming to the Washington University School of Medicine in St. Louis-- Doing OK? Yep.

--to participate in some of the world's first clinical trials in Alzheimer's prevention. It's the least I can do to help. I feel like I couldn't live with myself if I didn't do it. Now we have a chance to change the course of the disease in a way we've never been able to do before.

Dr. Randy Bateman leads international clinical trials at WashU Medicine. Wow, this is a big operation here. One reason for Dr. Bateman's optimism-- for the first time, there's finally success treating mild Alzheimer's with medication that removes amyloid plaques, the protein deposits that build up on the outside of nerve cells in the brain, interfering with memory and thinking. These are all amyloid plaques.

These drugs have been shown to slow cognitive decline. The people who had the plaques removed are 30% better than the people who didn't have the plaques removed. But it does not reverse the dementia--

No. --even at that stage? It doesn't stop the dementia. The dementia still continues, but at a slower rate. So, of course, the next question is, what would happen if you started the treatment even earlier? Even earlier. Exactly. Even before symptoms.

That's the whole point. If you look at the people who are treated in these trials, the people who are at the earlier stages, the earlier stage you go, the better off they do. Some of them actually have been stable. And so what this suggests is that timing is critically important. Within a particular family with early-onset Alzheimer's, symptoms usually start at about the same age. So researchers can figure out when to start treatment to try to prevent the disease. So this is a way to test the hypothesis that if you start treating people before they have symptoms, it'll make a difference. Absolutely it is. So think of it this way. In these people, we have near 100% certainty they will get Alzheimer's disease dementia, and we know about when they're going to get it. We'll take the proteins from a patient--

Though these rare mutations account for less than 1% of Alzheimer's patients, the lessons learned about when to start treatment may apply to everyone. Jake Heinrichs is a Broadway theater electrician from Brooklyn who carries one of the early-onset genes. The disease claimed his grandmother, uncle, father and brother. All in that same age range. Which was around-- Symptoms in the 40s and dying in the 50s. The word "Alzheimer's" for you must have-- growing up, I imagine-- had-- It was a death sentence. No drug allergies?

Heinrichs started getting treatment in Dr. Bateman's study in 2013. Though he's definitely getting an antibody called lecanemab now, for the first seven years, he did not know if he was receiving an antibody or placebo. I was at an age where I should have been showing signs. And I am now at an age that I should probably not even be alive. Do you see what Mama and I put in? Yeah, we hooped-- we hooped it.

His wife is Broadway director Rachel Chavkin. She says over the past three years, he will occasionally repeat a question, but otherwise has shown no cognitive decline since she fell in love with him 20 years ago. Jake is now 51, which is the age that his father was when he died. Touch my finger. Touch your nose. Back and forth.

The Trump administration's budget cuts have stopped or delayed millions of dollars in Alzheimer's research. The situation has already had an impact on Dr. Bateman's trials. And so it's a precarious time. And research is not like building a building or painting a wall, where you can start and then stop for a few years and then go back and resume what you were doing. It's much more like feeding a baby. And that if you stop doing that for a few weeks, it's irrecoverable.

With federal research money being threatened, what do you see as the potential side effect of decreasing that investment? It delays the breakthrough. The data these young researchers have to consider different ideas is so much better than even five years ago. So this is the time where you'd think, wow, let's double down on this disease.

That's exactly what Bill Gates is doing. Drop the shield.

The billionaire Microsoft co-founder is using his wealth and influence to bring together industry and government to tackle Alzheimer's. How much have you already invested personally? I put in a bit over $300 million. And I'm going to stay very involved in this. So these are what the samples are stored in.

At a time of such possibility, uncertainty about funding is threatening momentum.

It's quite up in the air. If you talk to researchers, they're dealing with the uncertainty of, should they hire young people, or buy new equipment? And so, hopefully in the next several months, this gets resolved and we can go full speed ahead.

Gates has spent a considerable amount of his fortune to improve public health. And for him, it so happens Alzheimer's is personal. Bill Gates Sr. was an attorney and philanthropist who died five years ago at the age of 94. Your father had Alzheimer's. And it strikes me that even being Bill Gates didn't protect your family from that. He was lucky enough to have 24-hour care, but there was no medicine, nothing that could bring his cognition back.

Gates is supporting research to improve early diagnosis, treatment, and patient enrollment in trials. And no surprise, he sees a huge role for advanced computing. Where does AI come in? OK. All of this-- That got a smile from you.

Yeah. That's-- It's-- well, AI is going to impact everything we do. The biology of the brain is so complex that AI's ability to take large amounts of data and find meaning in that data means that this research is going to go a lot faster. Show me how you screw in a light bulb.

The early-onset clinical trials are expected to continue for at least another five years, involving hundreds of people at 40 sites around the world. If successful, it could usher in routine screening for Alzheimer's with a simple blood test, just as we do now for high cholesterol and diabetes. And I can imagine our viewers, looking at this right now, saying to each other, do I want to be tested? or, how can I get tested? And right now in medicine, we don't test people without symptoms because the treatments aren't yet proven to work in people without symptoms.

Finding effective treatments is a hot area of research, and may include giving cocktails of medications that target not only amyloid but other possible culprits, such as brain inflammation and a damaging nerve cell protein called tau. And so if this trial holds up and we can continue to demonstrate benefit year-after-year that these people are protected, then I actually think that will predict what's going to happen in the near future in ongoing prevention trials, that they will be positive, they will work, they will give people years of dementia-free life. You had MRIs before? Many, many. Many, many?

So Jake Heinrichs undergoes regular MRIs, physicals-- Listen to your heart.

--memory testing, spinal taps, and PET scans to monitor his brain function and see if he can prevent an almost certain genetic destiny. You know, your dad's a little bit of a hero. Did you know that?

Four-year-old Sam Heinrichs is living proof of what can come from a belief in science and a leap of faith. What made you finally say, you know what, even though there's a 50% chance he might get the gene, we're going to go ahead and try to have a baby. Because I've been part of this study, and it seems to be effective, it gave me hope that this scourge of Alzheimer's is something that we're not going to fear in the future. Anything could happen. But right now, I have hope.