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Study · Animal study (in vivo) · mouse · 2026

7 results

Elamipretide Improves Mitochondrial Function in Mitochondrial Trifunctional Protein-Deficient Mice and Human Fibroblasts.

Vieira Neto E, Wang M, Szuminsky AJ, Ferraro L, Basu S, Zhao X, Karunanidhi A, Wang Y, Vockley J

Journal of inherited metabolic disease1 Jan 2026Cited by 3 (Europe PMC)

PMID41500837 (opens in a new tab)PMCIDPMC12779498 (opens in a new tab)DOI10.1002/jimd.70132 (opens in a new tab)PepBankPUB-41500837
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Summary

Mitochondrial trifunctional protein (TFP) deficiency is an inherited disorder of long-chain fatty acid β-oxidation (FAO). TFP is a heteromeric enzyme composed of two α and two β-subunits. Despite early detection and dietary treatment, TFP deficiency patients often develop hypoglycemia, rhabdomyolysis, cardiomyopathy, and peripheral neuropathy. Degenerative retinopathy and milder peripheral neuropathy occur in patients with an isolated deficiency of the αTFP subunit of long-chain 3-hydroxyacyl-CoA dehydrogenase (LCHAD) activity. Triheptanoin treatment improves most complications, but not peripheral neuropathy and retinopathy. Notably, TFP also carries a fourth enzymatic function involved in cardiolipin remodeling, which we previously found to be impaired in TFP/LCHAD deficiency. We therefore tested whether elamipretide, a synthetic cardiolipin-binding peptide, could improve mitochondrial function and cardiolipin levels in βTFP-deficient mice and patient-derived fibroblasts. Mice were treated with elamipretide delivered by osmotic minipump and challenged with treadmill exercise or cold stress after fasting. βTFP-deficient mice treated with elamipretide showed improved exercise endurance, but cold tolerance was not altered. Liver mitochondria from male βTFP-deficient mice demonstrated improved FAO-ETC enzyme activities. However, cardiolipin content and composition were unchanged. In patient fibroblasts, elamipretide produced a possible genotype-dependent increase in mitochondrial bioenergetics and a reduction in ROS. These results support a mechanism in which elamipretide stabilizes between FAO enzymes and ETC complexes, thereby improving mitochondrial function independently of changes in cardiolipin levels. Elamipretide thus emerges as a potential therapeutic agent for TFP/LCHAD deficiency, warranting further preclinical studies.
CC BYText from Europe PMC. Highlighted sentences are the source of results.

Results from this study7

7 results from studies that gave the peptide. No result from this study comes from people.

  1. 01AnimalAnimal study (in vivo)Efficacy outcome

    In betaTFP-deficient mice, elamipretide improved exercise endurance in a treadmill challenge.

    In plain wordsIn mice with a fat-metabolism disorder, elamipretide improved how long they could run on a treadmill.

    Peptide
    Elamipretide (SS-31)
    Outcome
    IncreaseExercise endurance
    Study

    mousen: not reported in source

    betaTFP-deficient mice
    Outcome group
    Exercise and physical function · Metabolic and weight
    Studied dose
    Dose not stated · osmotic minipumpAs given in this study (mouse).

    Source sentences

    Summary
    βTFP-deficient mice treated with elamipretide showed improved exercise endurance, but cold tolerance was not altered.
    2 context sentences
    Summary · context
    Mice were treated with elamipretide delivered by osmotic minipump and challenged with treadmill exercise or cold stress after fasting.
    Summary · context
    We therefore tested whether elamipretide, a synthetic cardiolipin-binding peptide, could improve mitochondrial function and cardiolipin levels in βTFP-deficient mice and patient-derived fibroblasts.
  2. 02AnimalAnimal study (in vivo)Efficacy outcome

    In betaTFP-deficient mice, elamipretide did not alter cold tolerance after fasting.

    In plain wordsIn mice with a fat-metabolism disorder, elamipretide did not change how well they tolerated cold.

    Peptide
    Elamipretide (SS-31)
    Outcome
    No significant changeCold tolerance after fasting
    Study

    mousen: not reported in source

    betaTFP-deficient mice
    Outcome group
    Exercise and physical function · Metabolic and weight
    Studied dose
    Dose not stated · osmotic minipumpAs given in this study (mouse).

    Source sentences

    Summary
    βTFP-deficient mice treated with elamipretide showed improved exercise endurance, but cold tolerance was not altered.
    2 context sentences
    Summary · context
    Mice were treated with elamipretide delivered by osmotic minipump and challenged with treadmill exercise or cold stress after fasting.
    Summary · context
    We therefore tested whether elamipretide, a synthetic cardiolipin-binding peptide, could improve mitochondrial function and cardiolipin levels in βTFP-deficient mice and patient-derived fibroblasts.
  3. 03AnimalAnimal study (in vivo)Mechanism

    In male betaTFP-deficient mice, elamipretide improved FAO-ETC enzyme activities in liver mitochondria.

    In plain wordsIn male mice with a fat-metabolism disorder, elamipretide improved the activity of energy-producing enzymes in liver mitochondria.

    Peptide
    Elamipretide (SS-31)
    Outcome
    IncreaseLiver mitochondrial FAO-ETC enzyme activities
    Study

    mousen: not reported in source

    betaTFP-deficient mice
    Outcome group
    Mitochondrial function · Metabolic and weight
    Studied dose
    Dose not stated · osmotic minipumpAs given in this study (mouse).

    Source sentences

    Summary
    Liver mitochondria from male βTFP-deficient mice demonstrated improved FAO-ETC enzyme activities.
    1 context sentence
    Summary · context
    We therefore tested whether elamipretide, a synthetic cardiolipin-binding peptide, could improve mitochondrial function and cardiolipin levels in βTFP-deficient mice and patient-derived fibroblasts.
  4. 04AnimalAnimal study (in vivo)Mechanism

    In betaTFP-deficient mice, elamipretide did not change cardiolipin content.

    In plain wordsIn mice with a fat-metabolism disorder, elamipretide did not change the amount of cardiolipin, a mitochondrial fat.

    Peptide
    Elamipretide (SS-31)
    Outcome
    No significant changeCardiolipin content
    Study

    mousen: not reported in source

    betaTFP-deficient mice
    Outcome group
    Mitochondrial function · Metabolic and weight
    Studied dose
    Dose not stated · osmotic minipumpAs given in this study (mouse).

    Source sentences

    Summary
    However, cardiolipin content and composition were unchanged.
    2 context sentences
    Summary · context
    Mice were treated with elamipretide delivered by osmotic minipump and challenged with treadmill exercise or cold stress after fasting.
    Summary · context
    We therefore tested whether elamipretide, a synthetic cardiolipin-binding peptide, could improve mitochondrial function and cardiolipin levels in βTFP-deficient mice and patient-derived fibroblasts.
  5. 05AnimalAnimal study (in vivo)Mechanism

    In betaTFP-deficient mice, elamipretide did not change cardiolipin composition.

    In plain wordsIn mice with a fat-metabolism disorder, elamipretide did not change the make-up of cardiolipin, a mitochondrial fat.

    Peptide
    Elamipretide (SS-31)
    Outcome
    No significant changeCardiolipin composition
    Study

    mousen: not reported in source

    betaTFP-deficient mice
    Outcome group
    Mitochondrial function · Metabolic and weight
    Studied dose
    Dose not stated · osmotic minipumpAs given in this study (mouse).

    Source sentences

    Summary
    However, cardiolipin content and composition were unchanged.
    2 context sentences
    Summary · context
    Mice were treated with elamipretide delivered by osmotic minipump and challenged with treadmill exercise or cold stress after fasting.
    Summary · context
    We therefore tested whether elamipretide, a synthetic cardiolipin-binding peptide, could improve mitochondrial function and cardiolipin levels in βTFP-deficient mice and patient-derived fibroblasts.
  6. 06LabIn vitro (cells)Mechanism

    In patient-derived fibroblasts, elamipretide produced a possible genotype-dependent increase in mitochondrial bioenergetics.

    In plain wordsIn lab-grown cells with the gene defect, elamipretide may have raised mitochondrial energy output, possibly depending on the gene change.

    Peptide
    Elamipretide (SS-31)
    Outcome
    IncreaseMitochondrial bioenergetics in patient fibroblasts
    Study

    humann: not reported in source

    Patient-derived fibroblasts (TFP/LCHAD deficiency)
    Outcome group
    Mitochondrial function · Metabolic and weight
    Studied dose
    Dose not stated

    Source sentences

    Summary
    In patient fibroblasts, elamipretide produced a possible genotype-dependent increase in mitochondrial bioenergetics and a reduction in ROS.
    1 context sentence
    Summary · context
    We therefore tested whether elamipretide, a synthetic cardiolipin-binding peptide, could improve mitochondrial function and cardiolipin levels in βTFP-deficient mice and patient-derived fibroblasts.
  7. 07LabIn vitro (cells)Mechanism

    In patient-derived fibroblasts, elamipretide reduced reactive oxygen species.

    In plain wordsIn lab-grown cells with the gene defect, elamipretide lowered levels of reactive oxygen species, harmful by-products of metabolism.

    Peptide
    Elamipretide (SS-31)
    Outcome
    DecreaseROS in patient fibroblasts
    Study

    humann: not reported in source

    Patient-derived fibroblasts (TFP/LCHAD deficiency)
    Outcome group
    Mitochondrial function · Metabolic and weight
    Studied dose
    Dose not stated

    Source sentences

    Summary
    In patient fibroblasts, elamipretide produced a possible genotype-dependent increase in mitochondrial bioenergetics and a reduction in ROS.
    1 context sentence
    Summary · context
    We therefore tested whether elamipretide, a synthetic cardiolipin-binding peptide, could improve mitochondrial function and cardiolipin levels in βTFP-deficient mice and patient-derived fibroblasts.

AI wrote and checked these results. AI can make mistakes, so check important results against the source.

Version history

PepBank keeps every version of the data on this study. Status changes from Retraction Watch, Europe PMC and Crossref are listed with their source.

  1. Version 2Current

    10 Oct 2026, 08:19 UTC

    Study results updated

  2. Version 1

    9 Oct 2026, 02:47 UTC

    First added · source update from Europe PMC

Subjects

Terms as the source lists them.

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