Emerging Breakthroughs in Neurodegenerative Disease Treatments: Kavita Nair, PhD, FAAN

Cutting-edge therapies are reshaping the outlook for neurodegenerative diseases, says Kavita Nair, PhD, FAAN, professor of neurology and clinical pharmacy, University of Colorado Anschutz. From potential first-in-class oral medications for multiple sclerosis (MS) to groundbreaking drugs that target the root causes of Alzheimer disease, researchers and clinicians stand on the verge of significant advancements.

This transcript was lightly edited for clarity; captions were auto-generated.

Transcript

What are some important medicines that are in development for neurodegenerative diseases?

With MS, we are currently waiting to get approval for the first BTK [Bruton tyrosine kinase] inhibitor in oral medication, and it's a little unique. MS has about 22 approved medications, which is kind of an embarrassment of riches, but I'm so grateful that patients have so many choices. This medication is unique in the sense that it's a brain penetrant, it's an oral medication, and it does treat a more progressive form of MS, or we hope that the indication for treatment will be for a more progressive form of MS that's not characterized by relapses or attacks after those relapses subside.

There's no cure for MS; we just try to manage and keep the disability progression at bay as long as possible. This form of MS is a little bit more insidious, a little bit more silent, where the relapses are less frequent, or not there at all, but the disability progresses, which means patients still start to maybe lose some function in their mobility and different aspects of their functioning. That's one potential drug we're waiting to hear about approval [for].

In Alzheimer disease, which is probably, at least in my view, one of the more exciting areas, because for several decades, there were just symptomatic drugs used to treat the symptoms. We've already had 2 disease-targeting drugs, the anti-amyloid therapies, and now we're waiting at the end of this year to hear the results of the semaglutide, the GLP-1 [glucagon-like peptide-1] one, to see what they have to say. That's going to be a little bit of a game changer. And then we have another anti-amyloid therapy manufactured by Genentech that is starting its phase 3 trials that's a little different from the current anti-amyloid therapy in that it might actually have some ability to cross a blood-brain barrier through some unique mechanisms.

We also have products that are going to potentially be able to deal with the tau proteins, which are developed in the brain. All of the products that I talked about, with the exception of GLP-1, attack the amyloid plaques. I think in the next 2 to 3 years, we're going to see some unique products and drugs that are going to attack different aspects of the Alzheimer pathology. I'd say a very exciting time for a very devastating disease.