Exenatide induces frataxin expression and improves mitochondrial function in Friedreich ataxia.

Igoillo-Esteve M, Oliveira AF, Cosentino C, Fantuzzi F, Demarez C, Toivonen S, Hu A, Chintawar S, Lopes M, Pachera N, Cai Y, Abdulkarim B and 9 more

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Rai M, Marselli L, Marchetti P, Tariq M, Jonas JC, Boscolo M, Pandolfo M, Eizirik DL, Cnop M

JCI insight30 Jan 2020Cited by 57 (Europe PMC)

PMID31877117PMCIDPMC7098728DOI10.1172/jci.insight.134221PepBankPUB-31877117

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Summary

The text of this summary is not shown here, because the article license does not allow reuse. Read it at the source (opens in a new tab)

Text rights: Publisher copyright. Results still quote short attributed sentences.

Results from this study10

7 results from studies that gave the peptide. 1 result from people.

01HumanNon-randomized trialMechanism
In a pilot trial in Friedreich ataxia patients, exenatide modestly induced platelet frataxin over treatment.
In plain wordsIn patients with Friedreich ataxia, exenatide modestly increased frataxin in blood platelets during a pilot trial.
Peptide
Exenatide (exendin-4)
Outcome
IncreasePlatelet frataxin induction
Study
n: not reported in source
Outcome group
Receptor activity and mechanism · Nervous system and cognition
Studied dose
Dose not stated · 5 weeksAs given in this study (human).
Source sentences
Summary
The frataxin-inducing effect of exenatide was confirmed in a pilot trial in Friedreich ataxia patients, showing modest frataxin induction in platelets over a 5-week treatment course.
1 context sentence(design, population, supporting numbers)
Summary · context
Here we examined the metabolic, neuroprotective, and frataxin-inducing effects of glucagon-like peptide-1 (GLP-1) analogs in in vivo and in vitro models and in patients with Friedreich ataxia.
Report a problemFND-3187711710
02AnimalAnimal study (in vivo)Efficacy outcome
In frataxin-deficient mice, exenatide improved glucose homeostasis.
In plain wordsIn mice deficient in frataxin, exenatide improved blood sugar regulation.
Peptide
Exenatide (exendin-4)
Outcome
MixedGlucose homeostasis
Study
mousen: not reported in source
Frataxin-deficient mice
Outcome group
Glycemic control · Metabolic and weight
Studied dose
Dose not stated
Source sentences
Summary
The GLP-1 analog exenatide improved glucose homeostasis of frataxin-deficient mice through enhanced insulin content and secretion in pancreatic β cells.
1 context sentence(design, population, supporting numbers)
Summary · context
Here we examined the metabolic, neuroprotective, and frataxin-inducing effects of glucagon-like peptide-1 (GLP-1) analogs in in vivo and in vitro models and in patients with Friedreich ataxia.
Report a problemFND-3187711701
03AnimalAnimal study (in vivo)Mechanism
In frataxin-deficient mice, exenatide enhanced pancreatic beta-cell insulin content.
In plain wordsIn mice deficient in frataxin, exenatide increased insulin content in pancreatic cells.
Peptide
Exenatide (exendin-4)
Outcome
IncreasePancreatic beta-cell insulin content
Study
mousen: not reported in source
Frataxin-deficient mice
Outcome group
Glycemic control · Metabolic and weight
Studied dose
Dose not stated
Source sentences
Summary
The GLP-1 analog exenatide improved glucose homeostasis of frataxin-deficient mice through enhanced insulin content and secretion in pancreatic β cells.
1 context sentence(design, population, supporting numbers)
Summary · context
Here we examined the metabolic, neuroprotective, and frataxin-inducing effects of glucagon-like peptide-1 (GLP-1) analogs in in vivo and in vitro models and in patients with Friedreich ataxia.
Report a problemFND-3187711702
04AnimalAnimal study (in vivo)Mechanism
In frataxin-deficient mice, exenatide enhanced pancreatic beta-cell insulin secretion.
In plain wordsIn mice deficient in frataxin, exenatide increased insulin release from pancreatic cells.
Peptide
Exenatide (exendin-4)
Outcome
IncreasePancreatic beta-cell insulin secretion
Study
mousen: not reported in source
Frataxin-deficient mice
Outcome group
Glycemic control · Metabolic and weight
Studied dose
Dose not stated
Source sentences
Summary
The GLP-1 analog exenatide improved glucose homeostasis of frataxin-deficient mice through enhanced insulin content and secretion in pancreatic β cells.
1 context sentence(design, population, supporting numbers)
Summary · context
Here we examined the metabolic, neuroprotective, and frataxin-inducing effects of glucagon-like peptide-1 (GLP-1) analogs in in vivo and in vitro models and in patients with Friedreich ataxia.
Report a problemFND-3187711703
05AnimalOther designMechanism
In the preclinical models, exenatide induced frataxin in beta cells and brain.
In plain wordsIn preclinical animal models, exenatide increased frataxin in pancreatic cells and brain.
Peptide
Exenatide (exendin-4)
Outcome
IncreaseFrataxin induction in beta cells and brain
Study
n: not reported in source
Preclinical beta-cell and brain models, not separated
Outcome group
Receptor activity and mechanism · Whole body
Studied dose
Dose not stated
Source sentences
Summary
Exenatide induced frataxin and iron-sulfur cluster-containing proteins in β cells and brain and was protective to sensory neurons in dorsal root ganglia.
1 context sentence(design, population, supporting numbers)
Summary · context
Here we examined the metabolic, neuroprotective, and frataxin-inducing effects of glucagon-like peptide-1 (GLP-1) analogs in in vivo and in vitro models and in patients with Friedreich ataxia.
Report a problemFND-3187711704
06AnimalOther designMechanism
In the preclinical models, exenatide induced iron-sulfur cluster-containing proteins in beta cells and brain.
In plain wordsIn preclinical animal models, exenatide increased proteins containing iron-sulfur clusters in pancreatic cells and brain.
Peptide
Exenatide (exendin-4)
Outcome
IncreaseIron-sulfur cluster-containing proteins in beta cells and brain
Study
n: not reported in source
Preclinical beta-cell and brain models, not separated
Outcome group
Mitochondrial function · Whole body
Studied dose
Dose not stated
Source sentences
Summary
Exenatide induced frataxin and iron-sulfur cluster-containing proteins in β cells and brain and was protective to sensory neurons in dorsal root ganglia.
1 context sentence(design, population, supporting numbers)
Summary · context
Here we examined the metabolic, neuroprotective, and frataxin-inducing effects of glucagon-like peptide-1 (GLP-1) analogs in in vivo and in vitro models and in patients with Friedreich ataxia.
Report a problemFND-3187711705
07AnimalOther designEfficacy outcome
In the preclinical models, exenatide protected dorsal root ganglion sensory neurons.
In plain wordsIn preclinical animal models, exenatide protected sensory nerve cells in dorsal root ganglia.
Peptide
Exenatide (exendin-4)
Outcome
MixedDorsal root ganglion sensory neuron protection
Study
n: not reported in source
Preclinical dorsal root ganglion sensory neuron models, not separated
Outcome group
Neuroprotection · Nervous system and cognition
Studied dose
Dose not stated
Source sentences
Summary
Exenatide induced frataxin and iron-sulfur cluster-containing proteins in β cells and brain and was protective to sensory neurons in dorsal root ganglia.
1 context sentence(design, population, supporting numbers)
Summary · context
Here we examined the metabolic, neuroprotective, and frataxin-inducing effects of glucagon-like peptide-1 (GLP-1) analogs in in vivo and in vitro models and in patients with Friedreich ataxia.
Report a problemFND-3187711706

Studies that did not test the peptide alone3

In this study, Exenatide (exendin-4) was a lab tool or a carrier, part of a mix, or the subject of a lab method. These results are not evidence about taking Exenatide (exendin-4).

08LabNot a test of Exenatide (exendin-4) aloneIn vitro (cells)Mechanism
In patient-derived cells, pooled GLP-1 analogs induced frataxin expression, without an exenatide-specific estimate.
In plain wordsIn lab tests using patient-derived cells, GLP-1 analogs increased frataxin, without a separate exenatide result.
Peptide
Exenatide (exendin-4)
Outcome
IncreaseFrataxin expression
Study
humann: not reported in source
Friedreich ataxia patient-derived induced pluripotent stem cell beta cells and sensory neurons
Outcome group
Receptor activity and mechanism · Whole body
Source sentences
Summary
GLP-1 analogs also induced frataxin expression, reduced oxidative stress, and improved mitochondrial function in Friedreich ataxia patients' induced pluripotent stem cell-derived β cells and sensory neurons.
1 context sentence(design, population, supporting numbers)
Summary · context
Here we examined the metabolic, neuroprotective, and frataxin-inducing effects of glucagon-like peptide-1 (GLP-1) analogs in in vivo and in vitro models and in patients with Friedreich ataxia.
In this study, Exenatide (exendin-4) was a lab tool or a carrier, part of a mix, or the subject of a lab method. This result is not evidence about taking Exenatide (exendin-4).
Report a problemFND-3187711707
09LabNot a test of Exenatide (exendin-4) aloneIn vitro (cells)Mechanism
In patient-derived cells, pooled GLP-1 analogs reduced oxidative stress, without an exenatide-specific estimate.
In plain wordsIn lab tests using patient-derived cells, GLP-1 analogs reduced oxidative stress, without a separate exenatide result.
Peptide
Exenatide (exendin-4)
Outcome
DecreaseOxidative stress
Study
humann: not reported in source
Friedreich ataxia patient-derived induced pluripotent stem cell beta cells and sensory neurons
Outcome group
Mitochondrial function · Whole body
Source sentences
Summary
GLP-1 analogs also induced frataxin expression, reduced oxidative stress, and improved mitochondrial function in Friedreich ataxia patients' induced pluripotent stem cell-derived β cells and sensory neurons.
1 context sentence(design, population, supporting numbers)
Summary · context
Here we examined the metabolic, neuroprotective, and frataxin-inducing effects of glucagon-like peptide-1 (GLP-1) analogs in in vivo and in vitro models and in patients with Friedreich ataxia.
In this study, Exenatide (exendin-4) was a lab tool or a carrier, part of a mix, or the subject of a lab method. This result is not evidence about taking Exenatide (exendin-4).
Report a problemFND-3187711708
10LabNot a test of Exenatide (exendin-4) aloneIn vitro (cells)Mechanism
In patient-derived cells, pooled GLP-1 analogs improved mitochondrial function, without an exenatide-specific estimate.
In plain wordsIn lab tests using patient-derived cells, GLP-1 analogs improved mitochondrial function, without a separate exenatide result.
Peptide
Exenatide (exendin-4)
Outcome
MixedMitochondrial function
Study
humann: not reported in source
Friedreich ataxia patient-derived induced pluripotent stem cell beta cells and sensory neurons
Outcome group
Mitochondrial function · Whole body
Source sentences
Summary
GLP-1 analogs also induced frataxin expression, reduced oxidative stress, and improved mitochondrial function in Friedreich ataxia patients' induced pluripotent stem cell-derived β cells and sensory neurons.
1 context sentence(design, population, supporting numbers)
Summary · context
Here we examined the metabolic, neuroprotective, and frataxin-inducing effects of glucagon-like peptide-1 (GLP-1) analogs in in vivo and in vitro models and in patients with Friedreich ataxia.
In this study, Exenatide (exendin-4) was a lab tool or a carrier, part of a mix, or the subject of a lab method. This result is not evidence about taking Exenatide (exendin-4).
Report a problemFND-3187711709

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.

Version 2Current
22 Oct 2026, 02:26 UTC
Study results updated
Version 1
21 Oct 2026, 18:59 UTC
First added · source update from Europe PMC

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Exenatide induces frataxin expression and improves mitochondrial function in Friedreich ataxia.

Summary

Results from this study10

Studies that did not test the peptide alone3

Version history

Subjects

Publication types

MeSH terms

Keywords