A medical institute at the forefront of new treatments

The Roskamp Institute in Sarasota, Florida is a cutting-edge medical institution seeking answers to debilitating and sometimes fatal conditions like Alzheimer’s and traumatic brain injury. In this segment, we learn about research that’s at the forefront of new treatments.

TRANSCRIPT

The Roskamp Institute in Sarasota, Florida, is a cutting-edge medical institution, seeking answers to debilitating and sometimes fatal conditions, like Alzheimer's and traumatic brain injury.

In this segment, we learn about research that's at the forefront of new treatments.

Here's the story.

The institute is a standalone biomedical research establishment funded by the NIH, the Department of Defense, the Veterans Administration, and the Roskamp Foundation.

And the goal of the institute is to find new treatments for newer psychiatric disorders.

Our research has been primarily on Alzheimer's disease, which has really been the disease that we've moved furthest forward with, 'cause it's the one we've been working on the longest.

But there are also many other research programs here at the institute, including programs on traumatic brain injury, Gulf War illness, post-traumatic stress disorder.

The institute provides tours to the public as a way to educate the community about the team of scientists and their work.

We have molecular biology, genetics, chemistry, proteomics, pathology, and we can move forward with that into the clinic very easily because we have this translational aspect.

We have our own clinic, and then we have a lot of clinical collaborators.

It's a massive effort that is part of the larger work of hundreds of institutions and pharmaceutical companies to find cures for Alzheimer's and other neurological disorders that affect millions of people worldwide.

One of those patients is Suki.

My mom has vascular dementia.

She suffered two large strokes, or two bigger strokes and several ischemic strokes in between.

She was forgetful.

Things on the stove were left on.

Neighbors noticed that she got lost walking.

And so then she started living with me.

Suki had always been independent, and Nancy made every effort possible to accommodate her mother and her needs.

I was able to create an environment at our home so that she could be as independent and could be home with the animals that she loves so much.

My mom did not particularly care for house pets when she was fully cognitively functioning.

Now, having the two rescues in the home all the time -- and now I have my grandpuppy at home -- she gets along famously with them.

In fact, she misses them when she's not with them.

And just recently, we attended my older daughter's wedding, and my mom had a blast.

She danced.

She interacted with so many people.

And, so, those types of things I really am so thankful for.

Today, patients and their families are relying on hope, but the team at Roskamp is feverishly working to turn that hope into solutions.

Their most important work has been in Alzheimer's and the discovery of the amyloid gene.

Once we found those genetic errors in the amyloid gene, it became apparent that amyloid, the buildup of amyloid, was really an early step in what was resulted in degeneration of neurons.

So that threw a focus on amyloid and ways to perhaps reduce amyloid and thereby mitigate the risk for the disease.

So we and others started on a long journey to find treatments that did exactly that.

We just finished a large multi-country, multi-site study of a drug that we developed here at the institute which targets amyloid.

And we've been using that in early-stage and middle-stage disease Alzheimer's.

The drug is Nilvadipine, which was originally developed for the treatment of high blood pressure.

And we had been finding, in the laboratory, that Nilvadipine was capable of stopping amyloid production by cells.

And we tested it in mouse models of Alzheimer's disease and showed that it improved cognition in those mouse models and, also, it reduced pathology.

So we saw less amyloid in the brains of those mice.

The research then moved to human studies, which were completed in the summer of 2017.

What we suspect from other studies and what we hope to see in this study is that if we get in early enough, we can reduce the rate of progression of the disease, and maybe with higher doses or slightly different versions of the drug, we can put the disease in hold, in a holding path.

The Roskamp Institute has taken a fruitful approach to developing research and implementing studies that lead to new treatments.

There's a huge amount of collaboration and integration of thought and ideas across all of the scientists and clinicians in the institute.

And things are not siloed, so it won't be my project or Dr. Paris' project.

If there's a project happening, we bring whoever is needed to the table.

A key person who is often brought to the table is Dr. Keegan, the clinical director at the institute.

You can't just be in your silo working on traumatic brain injury and not think about, 'Well, what's happening in a neurodegenerative disease, like Parkinson's or Alzheimer's, or an inflammatory disease, like multiple sclerosis?'

You kind of have to look at how there's overlap.

Today, the process of research moves along much more quickly.

The use of computers has made it possible to test ideas at an amazing speed.

Years ago, we would have had to screen each individual drug before we could decide whether they were useful or not.

I mean, physically screen them.

We would have to make maybe 5 million drugs and test every one.

Now we can screen in silico, on the computer, 5 million drugs in an afternoon.

And the Roskamp Institute has been very agile in managing the entire drug-development process.

We have this model where the not-for-profit institute partners with for-profits or even spins off for-profits in certain situations, and then we find for-profit dollars to get through that very expensive pre-clinical testing before we enter the clinic.

And that's been a successful strategy for us.

The future of research here is promising.

The expertise and the talent and the passion that we have here at the institute makes me very confident that we are going to achieve some key goals in the coming years.

Many of the techniques that we used are applicable to a wide range of diseases.

So my hope for the future is -- we do much, much more of what we're already doing.

I think there's lots of promising research, and I have hope for that.

But I don't want to not enjoy and appreciate where I am with my mom.

I know there's certain things that have been lost, but there's also, you know, a lot of things that we can enjoy in the present.