Itraconazole
Itraconazole is an antifungal medication that has been studied as a medication for prostate cancer and lung cancer in a small number of patients.
How can itraconazole help you? What the research says
We summarize the clinical evidence for each medical benefit here. We begin with our assessment of the strength of evidence within each category, followed by a brief summary of individual studies or reviews of several studies. In assessing the strength of evidence, we consider the study design, number of participants, and the size of the treatment effect (how much outcomes changed with treatment).
To see more details, click the plus sign to the right of any section.
Our assessments of evidence for each medical benefit fall into one of these categories:
- Strong evidence: consistent, significant effects in several large (or at least one very large) well designed clinical studies or at least two meta-analysesa statistical analysis that combines the results of two or more research studies; the results of smaller research studies addressing the same or similar questions can be analyzed as though they are one bigger, more powerful study of clinical studies of moderate or better quality (or one large meta-analysis) finding similar results
- Good evidence: significant effects in one large or several mid-sized and well-designed clinical studies ( randomized controlled trialsa study design in which people are randomly assigned to either an experimental group or a control group to compare the outcomes from different treatments; an RCT is considered a strong design for determining a therapy’s effects with an appropriate placebo or other strong comparison control or observational studies that control for confounds)
- Modest evidence: significant effects in at least three small but well-designed randomized controlled trials (RCTs), or one or more well-designed, mid-sized clinical studies of reasonably good quality (RCTs or observationala type of study in which individuals are observed or certain outcomes are measured, but no attempt is made to affect the outcome (for example, no treatment is given); an example is a study that records people’s diets, but doesn’t try to alter their diets, and looks for patterns of disease or other outcomes related to different foods studies), or several small studies aggregated into a meta-analysis
- Preliminary evidence: significant effects in small or poorly designed clinical studies OR conflicting results in adequate studies but a preponderance of evidence of an effect
- Weak evidence: one or more case studies, supported by animal evidence OR small treatment effects of limited clinical significance OR studies with no controls OR weak trends of effects
- Insufficient evidence: preclinical evidence only OR clinical studies with such poor or unclear methodology that no conclusion can be drawn OR conflicting findings across clinical studies with no preponderance of evidence in one direction; conflicting evidence occurs when studies find conflicting effects (positive effect vs no effect or negative effect) with the same treatment and the same general study population (same cancer type, for example)
Learn more about how we research and rate therapies and practices in How We Rate Therapies ›
Improving treatment outcomes
Is itraconazole linked to improved survival? Is it linked to less cancer growth or metastasis? Does it enhance the anticancer action of other treatments or therapies? We present the evidence.
People with progressive non-small cell lung cancer who were treated with itraconazole showed a better response to chemotherapy in two small studies.
Preliminary evidencesignificant effects in small or poorly designed clinical studies OR conflicting results in adequate studies but a preponderance of evidence of an effect (this is the CancerChoices definition; other researchers and studies may define this differently) of better response to treatment, longer progression-free survivalthe time during and after treatment of a disease that a patient lives without disease progression (worsening), and longer overall survival among people with non-small cell lung cancer treated with itraconazole in addition to chemotherapy.
- Higher rate of response to treatment and longer progression-free survival, though no evidence of an effectoverall, one or more studies did not demonstrate that a treatment or intervention led to an expected outcome; this does not always mean that there is no effect in clinical practice, but that the studies may have been underpowered (too few participants) or poorly designed. Larger, well-designed studies provide more confidence in making assessments. on overall survival, among people with advanced non-small cell lung cancer treated with itraconazole in addition to platinum-based chemotherapy, compared to chemotherapy alone, in a small RCTrandomized controlled trial, a study design in which people are randomly assigned to either an experimental group or a control group to compare the outcomes from different treatments; an RCT is considered a strong design for determining a therapy’s effects1Mohamed AW, Elbassiouny M et al. The effect of itraconazole on the clinical outcomes of patients with advanced non-small cell lung cancer receiving platinum-based chemotherapy: a randomized controlled study. Medical Oncology. 2021 Feb 9;38(3):23.
- Longer overall survival, and a weak trendan apparent change due to a therapy, close to but not achieving full statistical significance (this is the CancerChoices definition; other researchers and studies may define this differently toward longer progression-free survival, among people with progressive nonsquamous non-small cell lung cancer treated with itraconazole in addition to pemetrexed (chemotherapy), compared to chemotherapy alone, in a small RCT of 23 people2Rudin CM, Brahmer JR et al. Phase 2 study of pemetrexed and itraconazole as second-line therapy for metastatic nonsquamous non-small-cell lung cancer. Journal of Thoracic Oncology. 2013 May;8(5):619-23.
People with prostate cancer that was resistant to hormone therapy taking a higher dose of itraconazole showed slower progression than people taking a lower dose in one study.
Preliminary evidencesignificant effects in small or poorly designed clinical studies OR conflicting results in adequate studies but a preponderance of evidence of an effect (this is the CancerChoices definition; other researchers and studies may define this differently) of longer progression-free survivalthe time during and after treatment of a disease that a patient lives without disease progression (worsening) and slower rise in prostate-specific antigen (PSA) among people with metastatic castration-resistant prostate cancer (MCRPC) taking a higher dose of itraconazole.
- Longer progression-free survival and slower rise in prostate-specific antigen (PSA) (though faster progression than androgen deprivation therapy) among people with metastatic castration-resistant prostate cancer (MCRPC) taking 600mg itraconazole daily for 6 months while continuing androgen deprivation therapy compared to 200mg itraconazole in a small RCTrandomized controlled trial, a study design in which people are randomly assigned to either an experimental group or a control group to compare the outcomes from different treatments; an RCT is considered a strong design for determining a therapy’s effects3Antonarakis ES, Heath EI et al. Repurposing itraconazole as a treatment for advanced prostate cancer: a noncomparative randomized phase II trial in men with metastatic castration-resistant prostate cancer. Oncologist. 2013;18(2):163-73
Weak evidenceone or more case studies, supported by animal evidence OR small treatment effects of limited clinical significance OR studies with no controls OR weak trends of effects (this is the CancerChoices definition; other researchers and studies may define this differently) of slower rise in PSA among some, but not all, people with biochemically resistant prostate cancer taking itraconazole.
- A weak trendan apparent change due to a therapy, close to but not achieving full statistical significance (this is the CancerChoices definition; other researchers and studies may define this differently toward lower PSA, but only in a subset of patients who responded to itraconazole, among people with biochemically recurrent prostate cancer (rising PSA after treatment) taking 600mg itraconazole daily for at least 3 months in a small uncontrolled triala study in which a therapy is used, but without a comparison group against which to judge outcomes; an uncontrolled trial is considered a weak study design4Lee M, Hong H et al. Itraconazole as a noncastrating treatment for biochemically recurrent prostate cancer: a phase 2 study. Clinical Genitourinary Cancer. 2019 Feb;17(1):e92-e96.
- Greater than 50% reduction in PSA in one person with biochemically recurrent prostate cancer (rising PSA after surgery and radiation) taking 600mg itraconazole daily for 3 months in a case studya descriptive and exploratory analysis of a person, group, or event regarding changes observed over time; because changes due to treatment are not compared to similar changes over time without treatment, a case study is considered a weak study design5Suzman DL, Antonarakis ES. High-dose itraconazole as a noncastrating therapy for a patient with biochemically recurrent prostate cancer. Clinical Genitourinary Cancer. 2014 Apr;12(2):e51-3.
Keep reading about itraconazole
Author
Sophie received her Bachelor of Arts from the University of Cambridge, where she studied Middle Eastern languages and the philosophy of science. She then completed a premedical post-baccalaureate at the City University of New York. Before joining CancerChoices, she worked for several years at the Cornell Center for Research on End-of-Life Care, where she helped to conduct research on terminal illness and grief. Working in end-of-life research filled her with the conviction that all patients deserve free, accessible, and scientifically accurate information about the therapies available to them. While taking classes in anthropology, she also became curious about traditional medical knowledge and philosophies. These interests led her to CancerChoices. She is delighted to be part of CancerChoices’s work creating rigorous, evidence-based treatment guides for patients and physicians.
Reviewers
Laura Pole is senior clinical consultant for CancerChoices. Laura is an oncology clinical nurse specialist who has been providing integrative oncology clinical care, navigation, consultation, and education services for over 40 years. She is the co-creator and co-coordinator of the Integrative Oncology Navigation Training at Smith Center for Healing and the Arts in Washington, DC. Laura also manages the “Media Watch Cancer News That You Can Use” listserv for Smith Center/Commonweal. In her role as a palliative care educator and consultant, Laura has served as statewide Respecting Choices Faculty for the Virginia POST (Physician Orders for Scope of Treatment) Collaborative as well as provided statewide professional education on palliative and end-of-life care for the Virginia Association for Hospices and Palliative Care.
For CancerChoices, Laura curates content and research, networks with clinical and organizational partners, brings awareness and education of integrative oncology at professional and patient conferences and programs, and translates research into information relevant to the patient experience as well as clinical practice.
Laura sees her work with CancerChoices as a perfect alignment of all her passions, knowledge and skills in integrative oncology care. She is honored to serve you.
Alissa Huston received her medical degree at the University of Buffalo, and went on to complete an Internal Medicine residency at Strong Memorial Hospital. She then attended the University of Pittsburgh for her Hematology/Oncology fellowship, during which time she was involved in research involving bone metastasis in cancer.
Currently, she is an Associate Professor of Medicine at the Wilmot Cancer Institute at the University of Rochester, where her clinical efforts are focused on breast cancer. She is also the Co-Medical Director of the Pluta Integrative Oncology & Wellness Center. Her research efforts focus on understanding the effects of treatment for breast cancer upon bone health and how integrative modalities can help mitigate symptoms for cancer patients as they undergo treatment.
She is a member of the American Society of Clinical Oncology and the Society for Integrative Oncology. Her philosophy is that there is no singular approach to patient care. Even though many patients may carry the same diagnosis, each individual is affected by that illness in a different way. Patients are not perceived as just a number, but instead approached and treated as the unique individuals they represent.
Last update: May 7, 2025
Last full literature review: February 2025
CancerChoices provides information about integrative in cancer care, a patient-centered approach combining the best of conventional care, self care and evidence-informed complementary care in an integrated plan cancer care. We review complementaryin cancer care, complementary care involves the use of therapies intended to enhance or add to standard conventional treatments; examples include supplements, mind-body approaches such as yoga or psychosocial therapy, and acupuncture therapies and self-care lifestyle actions and behaviors that may impact cancer outcomes; examples include eating health-promoting foods, limiting alcohol, increasing physical activity, and managing stress practices to help patients and professionals explore and integrate the best combination of conventionalthe cancer care offered by conventionally trained physicians and most hospitals; examples are chemotherapy, surgery, and radiotherapy and complementary therapies and practices for each person.
Our staff have no financial conflicts of interest to declare. We receive no funds from any manufacturers or retailers gaining financial profit by promoting or discouraging therapies mentioned on this site.
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