Rapamycin for Alzheimer's Disease: Off-Label Use and Ongoing Research

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• Off-Label Rapamycin Study
• Capturing Patient Experience Data
• High-Risk Alzheimer's Patients
• Rapamycin Side Effects Profile
• Important Medical Disclaimer
• Full Transcript

Off-Label Rapamycin Study

Dr. Matt Kaeberlein, MD, PhD, discusses a new grant-funded study on off-label rapamycin use. This research initiative will survey individuals taking rapamycin for various health reasons. The study specifically aims to gather data from patients using rapamycin for Alzheimer's disease or Alzheimer's disease risk reduction. Dr. Matt Kaeberlein, MD, explains the project's goals to Dr. Anton Titov, MD. The research is not a double-blind, placebo-controlled clinical trial.

Capturing Patient Experience Data

The research methodology involves comprehensive patient surveys. Dr. Matt Kaeberlein, MD, PhD, details how the study will capture perceived benefits and side effects. Patients will also be asked to share their medical and dental records. This allows researchers to create a quantitative before-and-after picture of rapamycin use. Dr. Kaeberlein tells Dr. Anton Titov, MD, that this approach can yield valuable real-world data.

High-Risk Alzheimer's Patients

A key focus involves ApoE4 homozygotes at high risk for dementia. Dr. Matt Kaeberlein, MD, PhD, is collaborating with Dr. Alan Green, a physician prescribing rapamycin off-label. Dr. Green has many patients with this high-risk genetic profile. These individuals have a much higher risk of developing Alzheimer's disease than the general population. Studying their experiences with rapamycin could provide crucial insights into potential preventive effects.

Rapamycin Side Effects Profile

A major study goal is quantifying the frequency of adverse effects in non-transplant patients. Dr. Matt Kaeberlein, MD, PhD, highlights the different usage context. Off-label users typically take 4 to 6 milligrams of rapamycin once weekly. This is a very different regimen than organ transplant patients use. Transplant patients take high daily doses alongside other immunosuppressants. Understanding the real-world side effect profile is a critical research objective.

Important Medical Disclaimer

Both Dr. Matt Kaeberlein, MD, PhD, and Dr. Anton Titov, MD, emphasize this critical point. Rapamycin remains an experimental drug for Alzheimer's disease and aging-related uses. It is a prescription medication that requires physician supervision. Dr. Kaeberlein strongly advises against self-medication. He consistently tells people who contact him that they must work with their own doctor or veterinarian. This information is for general educational purposes only and is not medical advice.

Full Transcript

Dr. Anton Titov, MD: This was my follow-on question on that, listening to this very amazing, long, and painful journey that you're talking about. There are huge patient communities—patients like me, other big patient communities. Alzheimer's takes an enormous toll on caregivers, family, and their family.

So there must be people trying rapamycin in patients with Alzheimer's disease and using it off-label, perhaps just of their own volition. Is there any research that is at least trying to analyze the data from those patients together? It happens certainly for other indications; people do that for the quantified self movement, various other things. So what do you do about that?

Dr. Matt Kaeberlein, MD: I'm glad you asked that question because we actually just got a grant from the Impetus Grant Program to do exactly that. Within the next, I don't know, three or maybe even two weeks, we will be starting a large-scale, survey-based study of people who are taking rapamycin off-label, not only for Alzheimer's disease or Alzheimer's disease risk but for a variety of potential health effects.

We are trying to capture that data both on side effects and on potential perceived benefits. We are also asking those patients to share medical and dental records with us to try to look in a more quantitative way at a before-and-after picture of rapamycin.

It's not perfect; it's not going to be a double-blind, placebo-controlled clinical trial. But I still think, as you alluded to, you can get really interesting and informative information from capturing actual patient experience in that way.

We're actually working with Dr. Alan Green, who is one of the physicians who has been at the forefront of prescribing rapamycin off-label on this project. He actually has several patients who are ApoE4 homozygotes—not necessarily with dementia or Alzheimer's disease yet, but at high risk, much higher risk than the general population of developing dementia—who are taking rapamycin.

A large number of ApoE4 homozygotes, as well. I think that group of individuals, in particular, will be very interesting to start to try to get a feel for what their experiences have been.

I view it as both potentially getting a feel for positive effects, if any—and I think we have to be clear, we don't know yet; that's why we want to do the real clinical trial—but also really trying to quantify the frequency of adverse effects.

That's where I think you can really start to get a pretty good picture from these patients. They're very different than organ transplant patients; they haven't had an organ transplant, and they're not taking high doses of other immunosuppressants.

Most of them are taking rapamycin once weekly, four to six milligrams. So it's a very different regimen than organ transplant patients. Capturing what the actual frequency of side effects is like in those patients will be valuable as well.

Dr. Anton Titov, MD: I'd like to remind our listeners that none of what we say, what you say, or anybody else's on the product can be construed as medical advice. It's purely for informational and general information purposes only.

People should always consult their physician. It's an important disclaimer and should not take anything they hear anywhere on the internet as medical advice or a call to action. I think that's just very important to remind people.

Dr. Matt Kaeberlein, MD: I agree with that. It won't surprise you to hear that I get contacted by people often wanting rapamycin for their dog or themselves. I always tell them the same thing: it's important to appreciate this as an experimental drug, at least for these uses, and it's a prescription medication.

Legally, you are obligated—if you're going to take rapamycin—to get a prescription from your veterinarian if it's for your dog, or get a prescription from your physician if it's for you. Certainly, you want to be doing that under the supervision and with the knowledge of your physician.

I agree, it's not medical advice. If you're going to do it, you want to do it under the care of a physician. I think that is really important for people to appreciate.