#175 - Matt Kaeberlein, Ph.D.: The biology of aging, rapamycin, and other interventions that target the aging process

Sep 13, 2021 Episode Page ↗
Overview

Dr. Matt Kaeberlein, a globally recognized researcher on aging, discusses defining aging, the relationship between aging and disease, and the potential of molecules like rapamycin for lifespan extension. He shares insights from the Dog Aging Project and explores mTOR, sirtuins, and NAD precursors.

At a Glance
20 Insights
2h 40m Duration
12 Topics
7 Concepts

Deep Dive Analysis

Defining Aging: Molecular vs. Functional Perspectives

Relationship Between Aging, Functional Decline, and Disease

Impact of Curing Diseases vs. Targeting Biological Aging

Rapamycin as a Geroprotective Agent: Human Studies

The Role of Inflammation in Aging and Immune Function

Challenges of Clinical Trials for Aging Interventions

Periodontal Disease as a Potential Clinical Trial Endpoint

Biomarkers of Aging and Epigenetic Clocks

The Dog Aging Project: Studying Rapamycin in Pet Dogs

mTOR Complex 1 (mTORC1) vs. mTOR Complex 2 (mTORC2)

TORIN2: A Catalytic mTOR Inhibitor and its Potential

Sirtuins, NAD, and NAD Precursors (NR/NMN)

Hallmarks of Aging

A framework describing the molecular and cellular damage that occurs during aging, including mitochondrial dysfunction, telomere shortening, cellular senescence, DNA damage, stem cell fatigue, protein misfolding, deregulated nutrient sensing, intracellular communication, and epigenetic modulation. These contribute to functional declines and age-related diseases.

Functional Definition of Aging

An appreciation for the observable declines across every organ system in the body that accompany aging, such as frailty. These functional declines often precede the clinical diagnosis of age-related diseases and significantly impact quality of life.

mTOR Complex 1 (mTORC1)

One of two protein complexes involving mTOR, largely responsive to nutrient levels. It regulates processes like autophagy, mRNA translation, and metabolism. Rapamycin specifically inhibits mTORC1 by binding to FKBP12 and disrupting the complex.

mTOR Complex 2 (mTORC2)

The second protein complex involving mTOR, with different partner proteins and distinct functional roles compared to mTORC1. While rapamycin primarily inhibits mTORC1, chronic high-dose rapamycin can have feedback effects leading to mTORC2 inhibition.

TORIN2

A catalytic (ATP competitive) inhibitor of mTOR, meaning it directly inhibits the kinase activity of mTOR. Unlike rapamycin, TORIN2 is designed to inhibit both mTORC1 and mTORC2, and its effects on aging are currently under investigation.

Sirtuins

A class of NAD-dependent enzymes, primarily deacetylases, that remove acetyl groups from other proteins. Their activity requires and consumes NAD, and is inhibited by NADH, making the NAD:NADH ratio a proxy for sirtuin activity. They are implicated in aging, but their role in lifespan extension in mammals is mixed.

NAD Precursors (NR/NMN)

Molecules like nicotinamide riboside (NR) and nicotinamide mononucleotide (NMN) that can be converted into NAD within cells. The idea is that boosting NAD levels, which decline with age, could enhance sirtuin activity and positively impact aging, though reproducibility and efficacy in humans are still debated.

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How do scientists like Matt Kaeberlein define aging?

Aging can be defined from a molecular perspective, focusing on cellular damage like mitochondrial dysfunction and telomere shortening, or from a functional perspective, observing declines across organ systems like frailty. Matt primarily focuses on the biological aspects, acknowledging social aspects are also important for quality of life.

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Does treating individual diseases significantly extend human lifespan?

No, curing individual age-related diseases like all cancers or heart disease has a relatively small impact on overall life expectancy (e.g., ~3 years for all cancers). In contrast, interventions targeting the fundamental biology of aging could hypothetically extend healthy lifespan by much greater amounts.

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Is rapamycin safe for human use at doses relevant for aging interventions?

Clinical trials with rapamycin and its analogs (like Everolimus) at lower, intermittent doses (e.g., 5mg once a week) in older adults have shown very low incidence of adverse events, comparable to placebo. This challenges the common misconception that rapamycin is too unsafe for aging applications.

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Why are pet dogs a valuable model for studying human aging?

Pet dogs share the human environment, exhibit significant genetic and phenotypic diversity (including lifespan differences across breeds), age about 7 times faster than humans, and develop nearly all the same age-related diseases and functional declines as people, making them a powerful translational model.

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What is the difference between mTOR Complex 1 (mTORC1) and mTOR Complex 2 (mTORC2)?

mTORC1 is primarily responsive to nutrient levels and regulates processes like autophagy and protein synthesis. Rapamycin specifically inhibits mTORC1. mTORC2 has different partner proteins and functions, and is typically only inhibited by rapamycin at higher, chronic doses or by catalytic inhibitors like TORIN2.

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Do NAD precursors like NR and NMN effectively slow aging or improve health?

While there is some preclinical literature suggesting benefits from NAD precursors, the results are inconsistent and difficult to reproduce across different studies and labs, including the NIA's Interventions Testing Program. Their efficacy in humans is not yet robustly established, though they are generally considered safe.

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Why is it difficult to conduct clinical trials for aging interventions?

It's challenging because regulators are hesitant to approve trials in healthy individuals for a non-disease endpoint like 'aging,' and true lifespan studies in humans are impractical. There's also a lack of validated biomarkers of aging to measure intervention efficacy over shorter timeframes.

1. Prioritize Biological Aging Interventions

Focus on interventions that target the fundamental biology of aging for a more significant and broad impact on healthy lifespan, rather than treating individual diseases in isolation. This approach is estimated to add decades of healthy life, unlike single-disease cures.

2. Start Prevention Early

Begin prevention efforts as early as possible to mimic the healthy aging trajectory observed in centenarians, who experience a significant phase shift in disease onset.

3. Consider Once-Weekly Rapamycin Dosing

For individuals considering rapamycin, once-weekly dosing (e.g., 5-8mg, potentially with cycling periods like 8 weeks on, 6 weeks off) is a common self-experimentation protocol, though it lacks robust long-term clinical trial data.

4. Rapamycin Safety at Low Doses

Be reassured that rapamycin (and similar mTOR inhibitors like Everolimus or RTB101) at doses considered for anti-aging purposes do not show significant side effects, especially compared to widely used drugs like statins.

5. Lower ApoB & Improve Metabolic Health

Implement strategies (lifestyle, nutrition, or pharmacological) to lower ApoB and improve metabolic health (e.g., regulating glucose and insulin) to dramatically reduce the risk of atherosclerosis.

6. Fasting for Inflammation Reduction

Consider incorporating fasting into your routine, as it appears to reduce chronic inflammatory signaling, which is a critical factor in many age-related declines and diseases.

7. Evaluate Benefits & Risks

When considering any intervention, carefully weigh its potential beneficial effects against the associated risks and potential side effects, as every drug has a dose-dependent response.

8. Understand Aging as Progressive Decline

Recognize that normal aging is a progressive, chronic decline in function, not a healthy state, especially for typical older adults (e.g., 65-70 years old). This perspective should inform the acceptable level of risk for interventions aiming to slow or reverse this decline.

9. Maintain Good Oral Health

Prioritize good oral hygiene and address periodontal disease, as it is highly prevalent in older adults and linked to an increased risk of dementia, cardiovascular disease, and diabetes, likely through inflammatory pathways.

10. Caution with Epigenetic Clocks

Do not rely on current epigenetic clocks or telomere length measurements as definitive or helpful biomarkers for true biological age, as they can be easily manipulated by short-term interventions and lack robust biological explanations.

11. Sirtuins & NAD Precursors: Limited Evidence

Be aware that while sirtuins and NAD precursors (NR, NMN) show ‘a ton of smoke’ for anti-aging benefits in some studies, the evidence for their robust and reproducible efficacy, especially for lifespan extension, is mixed and not as strong as for mTOR inhibitors like rapamycin.

12. NAD Precursors: Safe but Unproven

NAD precursors like NR and NMN are considered very safe with minimal downside (primarily cost); however, their upside for anti-aging effects in humans is currently unclear and lacks definitive clinical trial evidence.

13. Do Not Use Rapamycin Before 25

Avoid using rapamycin in developing individuals, specifically before the age of 25, as its effects on development are not fully understood and could be detrimental.

14. Recognize Functional Declines as Early Aging

Pay attention to functional declines in various organ systems as early indicators of aging, often preceding the diagnosis of overt age-related diseases.

15. Prioritize Minimizing Side Effects

In any treatment, especially for long-term use or in vulnerable populations (like pets or healthy individuals), prioritize strategies that minimize the likelihood of side effects, such as optimizing dosing frequency.

16. Value Social Aspects of Aging

Recognize that social aspects are extremely important for quality of life as people get older, intersecting significantly with biological aging.

17. Peter Atiyah’s Rapamycin Protocol

Peter Atiyah takes 5-8mg of rapamycin once weekly, having previously cycled it (8 weeks on, 6 weeks off), but notes he is ‘flying blind’ without biomarkers to guide this decision.

18. Matt Caberlin’s Frozen Shoulder Protocol

Matt Caberlin used 8mg of rapamycin once weekly for eight weeks to resolve severe frozen shoulder, attributing the improvement to rapamycin’s anti-inflammatory effects (he explicitly states this is not an encouragement for others to do the same).

19. Advocate for Regulatory Changes

Support efforts to change regulatory environments (e.g., FDA) to allow clinical trials of geroprotective drugs in healthy individuals, using quantitative functional endpoints, and to address the challenges of off-patent drugs.

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You can't argue that biological age is the single greatest risk factor for every major cause of death and disability in developed countries. That is just a fact.

Matt Kaeberlein

The impact on life expectancy is actually from curing disease is actually quite small.

Matt Kaeberlein

There's no evidence actually for significant side effects from rapamycin monotherapy, everolimus monotherapy, or RTB 101 at the doses that people are talking about using in this context. That's just blatantly false.

Matt Kaeberlein

I would not call a 65 year old, a typical 65 or 70 year old healthy. They're not, they're functionally impaired.

Matt Kaeberlein

I don't think I will ever understand aging fully. And I don't think the field will, at least in any timeframe that I can expect to experience, right? But I also believe that we don't have to understand it fully to be able to have an impact on the biology of aging through interventions.

Matt Kaeberlein

I think that the underlying theme that seems to be similar about all of these things that work in mice is if you look in tissues of aged mice at inflammatory cytokines, P16, P21, markers of senescence, they seem to be tamped down by all of these, these interventions, which might explain the functional improvements that we see from using these interventions in aged mice.

Matt Kaeberlein

Rapamycin Treatment for Frozen Shoulder

Matt Kaeberlein
  1. Take 8 mg of rapamycin once a week.
  2. Continue treatment for 8 weeks.
15-30%
Lifespan extension in mice from various interventions Routine now for several different interventions to slow aging in mice.
~3 years
Increase in life expectancy for a typical 50-year-old woman if all cancers were cured Based on actuarial analysis, showing limited impact of single disease cures.
~3 years
Increase in life expectancy for a typical 50-year-old woman if heart disease was cured Based on actuarial analysis, showing limited impact of single disease cures.
~7 years
Increase in life expectancy for a typical 50-year-old woman if both cancer and heart disease were cured Roughly additive, still a small impact compared to targeting aging itself.
~20 years
Hypothetical increase in life expectancy from targeting biological aging in humans (extrapolated from mice) With the added benefit of these extra decades being spent in relatively good health.
5 mg once a week
Rapamycin dose in Everolimus (RAD001) study for immune function Showed improved vaccine response with no significant increase in adverse events in older adults.
~2/3
Prevalence of periodontal disease in older adults Two-thirds of older adults have or will get periodontal disease, which is linked to other age-related conditions.
At least 7 years old
Age for dogs to enter the Dog Aging Project (Triad) study To ensure dogs are middle-aged and aging rapidly enough for effects to be observed within the study timeframe.
40-100 pounds
Weight range for dogs in the Dog Aging Project (Triad) study Selected because larger dogs age faster and develop age-related diseases more quickly than smaller dogs.
15%
Target lifespan change detectable by the Dog Aging Project (Triad) study The study is statistically powered to detect this change in lifespan within a three-year window.
3 years
Duration of the Dog Aging Project (Triad) clinical trial Designed to be feasible for observing lifespan effects in dogs.
350 dogs
Sample size for the Dog Aging Project (Triad) clinical trial Randomized into two groups: 175 placebo and 175 rapamycin-treated.
0.15 mg/kg once a week
Rapamycin dose for dogs in the Dog Aging Project (Triad) study Chosen based on previous observations and to minimize side effects.
55-60 days
Lifespan of Lee syndrome mouse model A mouse model of a severe childhood mitochondrial disease, significantly extended by rapamycin.