What is new research focusing on?
Arnsten: There are many new approaches in the pipeline. Early intervention is one big priority. We need effective treatments with benign side effects so we can catch the disease early — maybe even before people start showing symptoms — and slow it down. My lab is researching the toxic actions caused by inflammation that contribute to Alzheimer’s. The goal would be to have a treatment you could use very early – once the test indicates risk even if the patient has no symptoms — that is also remarkably safe. You want to be able to use this with a patient who is, say, 50 years old, because the process can start when you’re still young.
Why does it take so long for discoveries in the lab to become medications people can take?
Arnsten: In many ways, Alzheimer’s researchers have had to invent the field, and innovations from disciplines such as genetics, cell biology, neuroscience, spectroscopy, and brain imaging have all been necessary to figure out what was changing in the brain and why. There appear to be multiple drivers of brain pathology, for example, where inflammation may contribute greater risk in some people than in others, which makes things more complex. But it also offers more opportunities for different kinds of treatments.
This type of translational science is necessarily slow, as it takes time to unravel the many factors that initiate and drive the pathology. And once you have discerned a possible therapeutic target, it takes great time and expense to determine that a treatment is effective and safe in patients.
What are some of the more notable new breakthroughs in the field?
Arnsten: One key recent breakthrough is a new blood biomarker that can detect the beginnings of tau pathology [accumulation of the tau protein in the brain], which is a hallmark of Alzheimer’s disease. This signal of emerging pathology in the brain can be seen long before one can use PET imaging to see later stage tau pathology in the brain. This new blood biomarker will also allow us to track whether a new treatment is working.
There are many new, and likely better, treatment strategies also in early stages of testing that will likely not come to fruition if Congress cuts the NIH [National Institutes of Health] budget. This would be a tragedy for so many patients and their families, and would also be very short-sighted, as the financial burden of caring for patients by the federal government is enormous.
In my lab, we’ve worked for 20 years to understand some of the early changes that especially afflict the neurons that generate memory and higher cognition, and we have identified a compound that we think can stop these early, toxic effects of inflammation with few side effects. But now, due to NIH budget cuts, we can’t get the funding to continue. These cuts will be devastating to so much research, and the field can’t just bounce back from them, because they will destroy so much of the research pipeline, hurting our health and also the U.S. economy. In the past, Congress understood the importance of NIH-funded research to American strength; we hope that rational strategies can still prevail.