Halofuginone dosage and evidence
Written by Aaron CuhaReviewed Sep 2026
The short answer
Halofuginone has barely been tested in people.
Evidence grade for Halofuginone: Preliminary. The rule: studied in people, but no published study has had people take it. The sum on this page: 3 human studies where nobody was given it, 1 registered with no result. A grade counts how much has been done to people, not whether it works.
- People given it
- 0
- Human studies
- 0
- Found nothing
- 0
Dose
No human dose established
No published study on this page has given this compound to people and reported a result.
3 human studies where nobody was given it, 1 registered with no result
2 effects reported, 2 unwanted, by people using it, not a trial
Goal pages: Fat loss and metabolism, Ageing and longevity
In plain English
In plain English, from Aaron
This is how I would explain it to a friend. The evidence, with the numbers, is further down the page.
Halofuginone is a vet drug used against gut parasites in animals. It was once tried as a cancer drug and as a drug for a muscle disease in boys. Both efforts stopped. It is not approved for people anywhere.
What people take it for
- A longer life, after a mouse study.
- Weight loss, after a study in mice and pigs.
- Less scarring and swelling.
What studies in people showed
Very few people have taken it. 24 cancer patients took it by mouth. Nausea and vomiting set the top dose. For long-term use the advice was 0.5 mg a day, with anti-sickness drugs. Some had bleeding problems, and the drug could not be ruled out. A study in 17 boys with a muscle disease was stopped, and no results were posted. A cream was tried on the skin of a few people. No healthy person has taken it in a study. Now animals. In male mice, the typical lifespan rose 8.8 percent. Female mice got nothing. In fat mice and pigs, it caused weight loss.
What people report
Reports, not study results
The good
- We have not collected user reports on this one yet.
The bad
- Nausea, vomiting and tiredness in the cancer study. These set the dose limit.
- Bleeding problems in several cancer patients. The drug could not be ruled out as the cause.
Where these come from: The side effects are from the cancer study on this page. We have no user reports.
My bottom line
The mouse result is one of the better ones. But in people, nausea capped the dose and some bled. I would not put a vet drug in my body on one mouse study.
An opinion, not a finding. I am a coach, not a doctor.
What it has been measured to do
Animal and lab work, not yet tested in people
Real results, but from animals or cells, not people. What works in a mouse often does not work the same way in a person. Nobody can patent this, so nobody would ever earn back the cost of a study in people. That is usually why there is none. The record on this one is set out below.
- Living longer1 animal or lab study
- Losing weight1 animal or lab study
- Feeling less hungry1 animal or lab study
- Cell energy and mitochondria1 animal or lab study
32 of the 36 indexed goals have no study of any kind behind Halofuginone
No study in people, animals or cells on this page reports a result for these. That does not prove Halofuginone does nothing for them. It does mean anything you have heard about Halofuginone and one of these did not come from a study cited here.
Show full evidence
What is Halofuginone?
Halofuginone has barely been tested in people. 24 cancer patients took it by mouth in an early safety study, and nausea capped the dose. There were also small skin cream studies, and a muscular dystrophy program in boys that was stopped. In male mice, it made typical lifespan 8.8 percent longer in the 2021 run of the NIA mouse lifespan program. That is one of the bigger male gains the program has seen in a single run. Female mice saw no change.
Halofuginone is a lab-made cousin of febrifugine, the active part of the Chinese herb Dichroa febrifuga. Vets use it against gut parasites in animals. In the 2000s a company tried to make it a cancer pill called tempostatin. The idea was to block scar tissue and the new blood vessels tumours need. Later it was tried as HT-100 for Duchenne muscular dystrophy. Neither became an approved medicine. No halofuginone product for people is in the FDA's approved drug list.
There is very little data in people. In a European early safety study, 24 patients with advanced solid tumours took it by mouth over 106 rounds of treatment, at 0.5 to 3.5 mg a day. The most they could stand for a short time was 3.5 mg a day. Nausea, vomiting and tiredness set that limit. For long-term use the advice was 0.5 mg a day, with anti-sickness drugs. Several patients had bleeding problems, and the drug could not be ruled out as the cause. A study of a skin cream in AIDS-related Kaposi sarcoma lowered type I collagen in treated spots. Too few patients could be assessed to say whether it helped. One patient with chronic graft versus host disease got better on the cream in 1999. The Duchenne program (HT-100) enrolled 17 boys in an early study and ran two follow-on studies. All three are listed as TERMINATED, with the reason given as "dosing stopped". No results were posted.
In mice, the NIA's lifespan program fed halofuginone from 7 months of age. Typical (median) male lifespan rose from 794 to 864 days, an 8.8 percent gain. Survival to old age, measured at the age when only 10 percent of mice are usually still alive, rose 6.9 percent. Females showed no change (median up 0.2 percent). The paper's main text does not give the dose in the food. It is in the extra tables. Separately, a 2025 study found that halofuginone caused weight loss in obese mice and in pigs. It did this by raising two hormones, GDF15 and FGF21, through a cell stress alarm.
How does Halofuginone work?
Halofuginone blocks an enzyme that loads the amino acid proline onto the cell's protein-building machinery. Adding extra proline undoes the block. The cell reads this as a shortage of amino acids and switches on a starvation alarm, one branch of its wider stress response. In a dish, in mouse and human immune cells, this stops one type of inflammatory T cell (Th17) from forming. Its older label, a blocker of type I collagen, is a knock-on effect of the same thing. The mouse lifespan program picked it because eating fewer amino acids makes several species live longer, and halofuginone copies that signal with a drug. In people, the only data on how it works come from skin. The cream lowered collagen gene activity in skin samples from one graft versus host patient. It lowered type I collagen in Kaposi sarcoma spots. None of it showed up in the blood after using the cream. The 2025 finding that it cuts food intake and raises GDF15 and FGF21 is in mice and pigs. Nothing like it has been measured in a person.
What have studies in people found about Halofuginone?
Very little. 24 cancer patients took it by mouth in one early safety study. It was used as a cream in one small Kaposi sarcoma study and in one graft versus host patient. 17 boys with Duchenne muscular dystrophy took it by mouth in a program that was stopped with no published results. No healthy person has taken it in a published study, and no study has looked at ageing.
- Early safety study by mouth, 24 patients with advanced solid tumours, 0.5 to 3.5 mg a day. Nausea, vomiting and tiredness set the top dose at 3.5 mg a day. The advice for long-term use was 0.5 mg a day with anti-sickness drugs. Several bleeding events, cause unclear.
- Duchenne (HT-100): an early study in 17 boys plus two follow-on studies. All are listed as terminated with "dosing stopped" as the reason, and no results posted. A study that stopped and never reported is itself worth knowing.
- Skin cream in AIDS-related Kaposi sarcoma: less type I collagen in treated spots. Too few people could be assessed to say whether it helped.
- One graft versus host patient used the cream for 6 months: the skin made less collagen and the neck turned better on the treated side. Both went back after stopping. None of the drug showed up in the blood.
- No published study has given halofuginone to a healthy adult, and none has looked for any effect on ageing.
What this does not tell you: Nothing here shows benefit in a person for any use. The long-term dose people could stand (0.5 mg a day) was set by nausea and vomiting, and the cancer study recorded bleeding events. Nobody knows whether the mouse dose that extended lifespan matches anything a person could take. The public record does not explain why the Duchenne program stopped.
The state of the research
The National Institute on Aging runs a mouse lifespan program called the Interventions Testing Program. It tests each compound at three labs at the same time: The Jackson Laboratory, the University of Michigan and UT Health San Antonio. The mice are a mixed breed, so a result is less likely to be a quirk of one strain. Males and females are both tested and judged separately. The three labs' results are then pooled. Every result gets published, whether or not the compound worked. It runs on public money from the National Institute on Aging, with no drug company money. In the 2021 run, halofuginone made typical (median) male lifespan 8.8 percent longer and survival to old age 6.9 percent higher. It did nothing in females. Of the three wins in that run, this male result was the strongest by the statistics. The gain in late survival matters. That is what separates a possible anti-ageing drug from one that only stops early deaths. It has been tested once, at one dose and starting age.
Nobody knows what it does in people because both human programs stopped. The tempostatin pill never got past the early study, where nausea held the long-term dose to 0.5 mg a day. The Duchenne program passed between three small companies. It ended with all three studies listed as terminated and never reported. The public record says "dosing stopped" and nothing more, so we cannot say why. Halofuginone is an old molecule with cheap vet versions, so no company can own it and pay for new human studies. Without them, the mouse result stays a mouse result. That cuts both ways. No trials does not mean it fails in people. It also means any harm goes unmeasured.
The evidence, in charts
Fig. 1a · how many people
25people, across 2 studies in people cited here
| Study | People | Share |
|---|---|---|
| European Journal of Cancer 2006 | 24 | 96% |
| Transplantation 1999 | 1 | 4% |
One study holds 96% of these people, so the total rests more on one study than its size suggests. Counts are the number of people each study on this page says it signed up. That includes studies that only watched people and gave nobody the compound. 2 more studies in people give no headcount and are not counted here. Studies still signing people up are left out, and only studies cited on this page are counted.
Fig. 1b · evidence mix
4 of 8 sources here are studies in people. The rest are not.
- Study in people · done in people450%
- Animal study · done in animals225%
- Lab (cells in a dish) · cells or tissue225%
Counted from the list of sources on this page. It counts only what we cite, so there may be more studies out there. A review counts once, however many studies it sums up.
Fig. 2 · evidence over time
The studies cover 8 different years, 1999 to 2026, counted from the list of sources on this page.
Fig. 3 · legal status at a glance
US
Not approved
UK
Not approved
AU
Not approved
CA
Not approved
Not approved in any of the four countries shown. This is what the law says, not a verdict on the science. See Legal status below for the full text and any notes.
Fig. 4 · how much does what
Nobody has tested different amounts in people
This chart would show how the effect changes as the amount goes up. For this compound, nobody has measured that in people. So we leave it empty. We will not borrow an animal chart and pretend it works the same in people.
Fig. 5 · what the molecule is
- Kind of compound
- Small molecule
- Molecule weight
- Not on file
- Half-life
- None on file
- Chain length
- None on file
No amino acid sequence is on file for Halofuginone, which is expected: a small molecule is not built from amino acids.
The studies we cite
- Study in people2006Phase I and pharmacokinetic study of halofuginone, an oral quinazolinone derivative in patients with advanced solid tumours
This is a dose-finding study in cancer patients, and it is mostly a list of harms. 24 patients took halofuginone by mouth at 0.5 to 3.5 mg a day across 106 treatment courses. The most tolerated briefly was 3.5 mg a day, limited by nausea, vomiting and fatigue. For ongoing use the recommended amount dropped to 0.5 mg a day, with anti-sickness drugs alongside. Several patients had bleeding problems that could not be ruled out as drug-related.
24 patients, 106 courses, 0.5 to 3.5 mg a day by mouth. Acute maximum tolerated dose 3.5 mg a day (nausea, vomiting, fatigue); recommended chronic dose 0.5 mg a day with 5-HT3 antagonists. Several patients had bleeding complications in which causality could not be excluded. Linear pharmacokinetics with large between-patient variability. EORTC study.
- Study in people2011Phase II AIDS Malignancy Consortium trial of topical halofuginone in AIDS-related Kaposi sarcoma
The cream changed the skin chemistry, and the trial could not say whether it helped. Each patient had halofuginone applied to some lesions and the plain base to others, without knowing which was which. Type I collagen fell only where the drug went, and a second marker did not move. Too few people completed the study to judge whether lesions improved. A collagen measurement is not a patient getting better. The National Cancer Institute supported it.
Blinded intrapatient vehicle-controlled topical study. Type I collagen fell significantly only in halofuginone-treated lesions; MMP-2 unchanged. Too few evaluable participants to assess efficacy. NCI-supported consortium trial.
- Study in people1999Topical treatment of cutaneous chronic graft versus host disease with halofuginone: a novel inhibitor of collagen type I synthesis
One person. That is the whole study. A patient with chronic graft versus host disease affecting the skin had the ointment applied to one side of the neck daily for six months. Collagen production fell and neck rotation improved on the treated side, and both reverted after stopping. None of the drug could be detected in the blood. A single case can generate an idea. It cannot establish that a treatment works.
One patient. Daily ointment on one side of the neck for 6 months reduced skin collagen synthesis and improved neck rotation on the treated side; effects reversed after stopping. No plasma halofuginone detectable. A single case, not a trial.
- Study in people2016Safety, tolerability and pharmacokinetics of single and multiple doses of HT-100 in Duchenne muscular dystrophy
The Duchenne programme stopped and never reported. 17 boys were enrolled in an early safety study of HT-100, and the registry records the trial as terminated, with the reason given as dosing stopped. No results were posted. Two follow-on studies were terminated as well, also without results. A halted trial with no published outcome is the least informative thing a drug can leave behind, and it is what this one left.
Phase 1b, 17 boys enrolled, sponsor Processa Pharmaceuticals (earlier Halo Therapeutics and Akashi). Status TERMINATED, reason recorded as "dosing stopped". No results posted. Two extension studies (NCT01978366, NCT02525302) are also terminated without results.
- Animal study2026Extension of lifespan by epicatechin, halofuginone and mitoglitazone in male but not female genetically heterogeneous mice
In mice. In the 2021 run of the NIA mouse lifespan program, halofuginone was fed from 7 months of age. The typical male lived 864 days against 794, an 8.8 percent gain. Survival to old age rose 6.9 percent. There were 154 treated males and 298 untreated ones. Females did not change (typical lifespan up 0.2 percent). Public NIA money paid for it.
Interventions Testing Program 2021 cohort, treatment from 7 months. Male median lifespan 864 versus 794 days (+8.8 percent, log-rank p = 0.002), 90th percentile survival +6.9 percent (p = 0.01); 154 treated males against 298 controls. Females: median +0.2 percent, p = 0.75. NIA funded.
- Animal study2025The clinical antiprotozoal drug halofuginone promotes weight loss by elevating GDF15 and FGF21
In obese mice and in pigs. Halofuginone cut how much the animals ate, raised the energy they burned, and caused weight loss. It worked by raising two hormones, FGF21 and GDF15. Mice bred without those hormones did not respond. Note that the paper calls halofuginone FDA-approved for scleroderma. The FDA's approved drug list does not back that up.
In diet-induced obese mice and in pigs, halofuginone suppressed food intake, raised energy expenditure and produced weight loss via integrated stress response induction of FGF21 and GDF15; both hormones were required in knockout mice. Animal only. Note that the abstract describes halofuginone as FDA-approved for scleroderma, which the FDA approved drugs database does not support.
- Lab (cells in a dish)2012Halofuginone and other febrifugine derivatives inhibit prolyl-tRNA synthetase
In a dish. This study found what halofuginone grabs inside the cell: the enzyme that loads the amino acid proline. Adding extra proline undid the effect. That one target explains why this family of drugs blocks scarring, inflammation and malaria.
Identified glutamyl-prolyl-tRNA synthetase as the molecular target; inhibition reversed by exogenous proline. Explains the anti-fibrotic, anti-inflammatory and anti-malarial activities of the febrifugine family through one mechanism.
- Lab (cells in a dish)2009Halofuginone inhibits TH17 cell differentiation by activating the amino acid starvation response
In a dish, then in mice. Halofuginone stopped mouse and human Th17 cells, a type of inflammatory immune cell, from forming. It did this by switching on the cell's amino acid starvation alarm, and extra amino acids undid it. In mice, it protected against a lab model of brain and nerve inflammation.
Selectively blocked mouse and human Th17 differentiation in culture via the amino acid starvation response, rescued by excess amino acids, and protected mice from experimental autoimmune encephalomyelitis. Cell and mouse data.
Common questions
Has Halofuginone been tested in people?
Not in a completed trial cited here. 3 human studies where nobody was given it, 1 registered with no result. Halofuginone has barely been tested in people.
What is the Halofuginone dosage?
No human dose established. No published study on this page has given this compound to people and reported a result. 3 human studies where nobody was given it, 1 registered with no result
Is Halofuginone FDA-approved?
Not approved for human use. No halofuginone product appears in the FDA approved drugs database. A version taken by mouth (tempostatin) and a version for Duchenne muscular dystrophy (HT-100) never reached approval. The Duchenne studies are recorded as terminated (stopped early). Used in animals to treat infections with single-celled parasites. The lifespan result is in male mice.
What did the ITP find for halofuginone?
In male mice fed from 7 months of age, typical lifespan rose 8.8 percent (864 against 794 days). Survival to old age rose 6.9 percent, pooled across three labs. Female mice showed no change. It has been tested in one run only.
Has halofuginone been given to people?
Yes, but rarely. 24 cancer patients took it by mouth in an early safety study. Nausea and vomiting held the long-term dose to 0.5 mg a day, and some patients had bleeding problems. Small skin cream studies exist in Kaposi sarcoma and in one graft versus host patient. A Duchenne muscular dystrophy program in 17 boys was stopped and never reported results.
Is halofuginone approved anywhere for humans?
No. It is a vet drug for gut parasites. The FDA's approved drug list has no halofuginone product, and neither human program reached approval. A 2025 paper calls it FDA-approved for scleroderma. The FDA's list does not back that up.
How does it work?
It blocks an enzyme the cell needs to use the amino acid proline. That switches on the cell's amino acid starvation alarm. In a dish, that blocks one type of inflammatory immune cell and the making of collagen. In mice and pigs it raises the hormones GDF15 and FGF21 and causes weight loss. The mouse lifespan program chose it as a drug that copies eating fewer amino acids. In people, the only thing measured is skin collagen in samples.