#281 ‒ Longevity drugs, aging biomarkers, and updated findings from the Interventions Testing Program (ITP) | Rich Miller, M.D., Ph.D.

Dec 4, 2023 Episode Page ↗
Overview

Dr. Rich Miller, Director of the Paul F. Glenn Center for Biology of Aging Research at the University of Michigan, provides an update on the Interventions Testing Program (ITP). He discusses successful lifespan-extending drugs in mice like rapamycin and 17α-estradiol, notable failures such as resveratrol, and the discovery of novel aging rate indicators.

At a Glance
19 Insights
2h 23m Duration
16 Topics
6 Concepts

Deep Dive Analysis

Overview of the Interventions Testing Program (ITP)

Superiority of UMHET3 Mice for Aging Research

ITP Study Design: Outcomes, Metrics, and Power Analysis

Challenges and Process of Drug Formulation and Delivery

Notable ITP Successes: Rapamycin and its Impact

Healthspan Measures and Collaborative Research Programs

Distinguishing Aging Rate Indicators from Biomarkers of Aging

Key Aging Rate Indicators: UCP1, Macrophages, BDNF, DCX, GPLD1

Importance of Proteomics over Transcriptomics in Aging

Future Frontiers: Bridging Aging Rate Indicators from Mice to Humans

Causes of Death in ITP Mice and Drug Effects

17-alpha-estradiol: Mechanisms of Life Extension and Sex Differences

Unsuccessful Drugs: Resveratrol, Metformin, and Nicotinamide Riboside

Over-the-Counter Successes: Meclizine and Astaxanthin

Fisetin and the Senescent Cell Hypothesis

Optimism for Future Discoveries and Program Impact

Interventions Testing Program (ITP)

A National Aging Institute initiative that evaluates potential life-extending interventions, primarily drugs, in mice. It aims to identify compounds that slow aging and extend healthy lifespan, using a genetically heterogeneous mouse model across three independent research laboratories.

UMHET3 Mice

A genetically heterogeneous mouse model used by the ITP, derived from four different inbred strains. Unlike standard inbred mice, UMHET3 mice are not genetically identical, better reflecting human genetic diversity and providing more robust, reproducible results for aging research.

Aging Rate Indicators (ARIs)

Measures that indicate how quickly an organism is aging, akin to a speedometer. These are distinct from biomarkers of aging (odometers) because they can be detected even in young adults and change consistently in the same direction across various slow-aging models.

Cap-Independent Translation

A mechanism of protein synthesis where ribosomes bind to messenger RNA (mRNA) at sites other than the 5' cap. This allows for selective translation of certain proteins, independent of mRNA transcription levels, and plays a significant role in molding the proteome in slow-aging mice.

Xenobiotic Metabolism Enzymes

Enzymes, primarily in the liver, that process foreign compounds like drugs. These enzymes often exhibit radical differences between sexes, leading to variations in drug absorption, conjugation, and excretion rates, which can impact drug concentrations and efficacy.

Senescent Cells

Cells that have stopped dividing and accumulate with age, often secreting inflammatory factors. While their existence is acknowledged, the guest expresses skepticism about their uniform definition and the extent of their role as a primary driver of aging, noting that they are often rare even in aged tissues.

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What is the primary purpose of the Interventions Testing Program (ITP)?

The ITP's main goal is to identify drugs that can slow aging and extend the lifespan of mice, providing insights into the biology of aging and potential pathways for human longevity interventions.

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Why is the UMHET3 mouse model superior to the commonly used C57 Black 6 (B6) mouse model in aging research?

UMHET3 mice are genetically heterogeneous, meaning no two are identical, which better reflects human genetic diversity and leads to more generalizable results. In contrast, B6 mice are inbred and homozygous, making them prone to specific diseases and less representative of a diverse population.

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How does the ITP define and measure maximal lifespan in its studies?

The ITP uses a statistical test (Wang-Allison test) that looks at the proportion of treated versus control mice still alive when 90% of the total pooled population has died, rather than relying on the age of the very last mouse to die.

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Why do some drugs, like rapamycin and canagliflozin, show sex-specific differences in efficacy or concentration in mice?

These differences are likely due to sex-specific variations in xenobiotic metabolizing enzymes in the liver, which can lead to different rates of drug absorption, conjugation, and excretion, resulting in varying blood concentrations between male and female mice.

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What does it mean that rapamycin can extend lifespan even when started in middle-aged mice?

This suggests that even at an advanced age (equivalent to 55-60 in humans), there are still ongoing aging processes that are susceptible to drug intervention, indicating that some age-related damage is not irreversible and can be slowed down.

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What are 'aging rate indicators' and how do they differ from 'biomarkers of aging'?

Aging rate indicators (ARIs) are like a speedometer, measuring how quickly an organism is aging, and can be observed in young adults. Biomarkers of aging are like an odometer, indicating how much aging has occurred, and are typically only useful when an animal or person is already old.

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Why is it important to study proteins (proteomics) rather than just RNA (transcriptomics) when investigating aging and drug effects?

RNA levels are poorly correlated with actual protein levels (only about 30% correlation in aging studies), and it is proteins that perform functions in the cell. Focusing solely on RNA misses 70% of the changes that control protein levels, leading to an incomplete or potentially misleading understanding of biological processes.

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What is the ITP's current understanding of 17-alpha-estradiol's mechanism of action?

The mechanism is still obscure, but it significantly extends lifespan in male mice, even beyond females, and does not benefit females. It does not bind well to traditional estrogen receptors, suggesting an alternative target or pathway, possibly involving the production of specific estriol family steroids in males.

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Why did resveratrol fail to extend lifespan in ITP studies, despite widespread public interest?

Resveratrol did not extend mouse lifespan in ITP studies, and its status as a sirtuin activator and its efficacy in other models have been widely questioned. The amount found in red wine is negligible for any effect, and much of its initial hype has been disproven by rigorous scientific inquiry.

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What were the surprising findings regarding over-the-counter drugs Meclizine and Astaxanthin in ITP studies?

Both Meclizine (an antihistamine for seasickness) and Astaxanthin (a food dye/antioxidant) significantly increased the lifespan of male mice by about 10%. This is notable as they are the first over-the-counter winners, though their mechanisms and effects on maximum lifespan require further investigation.

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What did the ITP learn from testing Fisetin, a purported senolytic drug?

Fisetin did not extend the lifespan of male or female mice in ITP studies. Furthermore, it did not appear to remove senescent cells (as measured by markers like P16) from the brain, liver, muscle, or kidney tissues, suggesting it failed to act as a senolytic in this context.

1. Prioritize Functional Anti-Aging Outcomes

When evaluating anti-aging interventions, prioritize evidence of retained youthful function (e.g., improved muscle strength, hearing, cognition, bone health) in addition to lifespan extension, as this confirms a true anti-aging effect rather than just disease postponement.

2. Prioritize Proteomics in Research

Focus on collecting and analyzing proteomic data over solely transcriptomic data in research, as protein levels are poorly correlated with mRNA levels (~30% correlation), and proteins are the functional molecules in cells.

3. Study Non-Transcriptional Proteome Molding

Explore non-transcriptional pathways like differential RNA translation and selective protein degradation (e.g., chaperone-mediated autophagy) that significantly impact protein levels independently of mRNA transcription.

4. Choose Heterogeneous Mouse Models

For biomedical research, especially in aging, use genetically heterogeneous mouse models (e.g., UMHET3) instead of inbred strains (e.g., C57 Black 6) to improve translational relevance and avoid misleading results from a single genotype.

5. Verify Drug Formulation & Bioavailability

Before initiating long-term drug studies, rigorously test the drug’s concentration in the administered form and its presence in target tissues (e.g., liver, plasma) via pilot administration to ensure proper dosing and biological effect.

6. Rapid Euthanasia for Research Integrity

Employ rapid euthanasia methods (e.g., quick CO2 gas fill) to minimize stress and physiological changes (e.g., adrenaline, glucose, pH) that could confound biochemical and molecular analyses of tissues.

7. Adjust Dosing for Sex Differences

Consider sex-specific differences in drug pharmacokinetics; adjust dosing or administration duration for each sex (e.g., lower dose, stop earlier) to optimize efficacy and avoid toxicity.

8. Screen Drugs Using Aging Rate Indicators

Utilize aging rate indicators (ARIs) as a cost-effective screening tool for potential anti-aging drugs, testing their ability to shift ARIs in young adults within months, which may predict lifespan extension more efficiently than full lifespan studies.

9. Nominate Drugs for ITP Testing

If you are a researcher with a potential life-extending drug, submit an application to the ITP explaining its rationale and safety for testing in mice, as they annually select candidates for lifespan extension studies.

10. Test Drugs in Middle-Aged

When a drug shows efficacy in young adults, further test its effects in middle-aged subjects to determine if benefits extend to later life stages, which is a key goal for anti-aging interventions.

11. Access ITP Tissue Repository

Researchers can request tissues from ITP-treated mice (both winners and losers) for specific organ-specific functional or pathological outcome tests by submitting a request detailing the tissue needed and the research plan.

12. Fund Longevity Research via ITP

Philanthropists interested in longevity research can directly support the Interventions Testing Program (ITP) by setting up independent arrangements or foundation awards to its university sites, thereby increasing funding and accelerating critical longevity research.

13. Combine Rapamycin and Acarbose

Explore the combination of rapamycin and acarbose for potentially synergistic longevity benefits, as this combination yielded the largest lifespan increase (29% in male mice) observed by the ITP.

14. Rapamycin Benefits in Later Life

Consider rapamycin for longevity benefits, as studies show it can significantly extend lifespan in mice even when initiated in late middle age (20 months old, equivalent to 55-60 human years) without diminishing its efficacy.

15. 17-alpha-estradiol for Male Longevity

Consider 17-alpha-estradiol for male longevity, as it significantly extends lifespan in male mice even when initiated in middle age (16-20 months old), without affecting female longevity.

16. Acarbose Benefits in Middle Age

Consider acarbose for longevity, as it extends lifespan in mice (better in males) even when initiated in middle age (16-20 months old), though starting earlier yields greater benefits.

17. Meclizine for Male Longevity

Consider meclizine (an over-the-counter seasickness drug) as a potential longevity intervention, as it significantly increased lifespan by about 10% in male mice.

18. Astaxanthin for Male Longevity

Consider astaxanthin (a food dye/supplement) as a potential longevity intervention, as it significantly increased lifespan by about 10% in male mice.

19. Exercise for Cognitive Enhancement

Engage in regular exercise to potentially improve cognitive function, as exercise increases GPLD1 production in the liver and fat, which is correlated with enhanced cognition.

If you want to see if your drug works, you sort of have to test it on people who are not identical genetically to one another. Yet that sort of thing, which is so obvious in human analogies, is ignored by nearly all mouse scientists.

Rich Miller

The drugs that we consider, we have four of these now, that give more than a 10 percent increase in lifespan in terms of proportional change of healthy lifespan are doing about three times better than some hypothetical drug that abolished cancer in people or abolished heart attacks in people.

Rich Miller

The inevitable conclusion, which I would have bet a lot of money against, is that by the time you're 20 months of age in a mouse, which is sort of like 55 or 60 years old in a person, damage will have occurred that's irreversible. But apparently there's still some further stages of that process that occur afterwards, after we started to administer the drug at 20 months of age, which are dependent on aging, and the drug inhibits.

Rich Miller

RNA is very poorly correlated with protein, and it's the protein that counts. There's a lovely pair of studies from the Jackson labs... the correlation between the RNA and the protein was 30%.

Rich Miller

People are very easy to fool. It's easy to come up with eight or 10 things that people believe because they read them on the internet or they watched them on Fox News or whatever. And they're just wrong about this. But people are very, very gullible.

Rich Miller

ITP Drug Testing and Validation Protocol

Rich Miller
  1. Receive drug nominations and evaluate biological plausibility, potential efficacy, and safety for mice.
  2. Formulate drug into food, ensuring correct dosage and stability (e.g., encapsulation for rapamycin).
  3. Conduct pilot studies for 8 weeks with mice receiving the formulated food.
  4. Measure drug concentrations in tissues (e.g., liver, plasma) from pilot mice to confirm absorption and bioavailability.
  5. Analyze mRNA changes in liver from pilot mice to confirm biological activity of the drug.
  6. If pilot studies are successful, proceed with a full lifespan experiment using 50 males and 50 females per drug at each of the three ITP sites, compared to 100 male and 100 female controls per site.
  7. Administer drugs in food ad libitum, with euthanasia at specific times (e.g., 9-10 AM) using rapid CO2 exposure to minimize stress.
  8. Measure primary outcomes: proportional hazard (median lifespan) and maximum lifespan (using Wang-Allison 90th percentile test), stratifying results by site.
  9. For successful drugs (Stage 2 studies), conduct additional healthspan assessments (e.g., grip strength, motor tasks, cognition, pathology) and collect tissues for collaborative research.
$1 million
ITP funding for each of the three sites (direct costs) Per year, total direct costs across all sites are $3 million per year.
Approximately $4.5 million
Total annual cost to taxpayers for the ITP Includes indirect costs to universities, about 50% more than direct costs.
More than 50%
Percentage of NIA budget allocated to Alzheimer's disease research Making NIA's overall budget large, but its budget for general biology of aging much smaller.
97%
Percentage of requests for aged mice to NIA for C57 Black 6 mice Highlighting the reliance on this single inbred strain in biomedical research.
50%
Percentage of genes shared between any two UMHET3 mice Similar to siblings, but a random half, ensuring genetic heterogeneity.
3%
Lifespan extension from a hypothetical cure for cancer in people (over age 50) Compared to ITP drugs providing 10-20% lifespan increases in mice.
Less than 3%
Lifespan extension from a hypothetical drug abolishing heart attacks in people (over age 50) Compared to ITP drugs providing 10-20% lifespan increases in mice.
20 months
Age at which some rapamycin-treated mice started treatment Equivalent to 55-60 years old in a person, demonstrating efficacy in late middle age.
Approximately 90%
Percentage of rapamycin digested in the stomach Required encapsulation for effective delivery to the small intestine.
3-fold higher
Blood concentration difference of rapamycin in female vs. male mice Potentially explaining greater lifespan extension in females.
3-fold higher
Blood concentration difference of canagliflozin in female vs. male mice In old females, it can be 10 times higher than in young males, suggesting toxicity.
10
Number of slow-aging mouse models used to identify aging rate indicators Includes 4 genetic mutants, caloric restriction, and 5 drugs (acarbose, canagliflozin, 17-alpha-estradiol, rapamycin, P10 overexpressors).
13
Number of identified aging rate indicators that consistently change in all slow-aging mice Including UCP1, macrophage types, BDNF, DCX, and GPLD1.
30%
Correlation between RNA and protein levels in aging tissues Meaning 70% of protein level changes are not explained by underlying mRNA transcription.
80%
Percentage of ITP mice deaths due to cancer Varies by type and sex, but neoplasia is the most common lethal injury.
29%
Lifespan increase from rapamycin combined with acarbose in male mice The largest percentage increase achieved by the ITP to date, and the first successful drug combination.
Approximately 10%
Lifespan increase from Meclizine in male mice Significant increase, but did not affect females or maximum lifespan.
Approximately 10%
Lifespan increase from Astaxanthin in male mice Significant increase, but did not affect females or maximum lifespan.