Cyclophosphamide

Cyclophosphamide is a decades-old chemotherapy agent tried and tested for other cancers such as breast cancer, ovarian cancer and leukemia. For mesothelioma, however, it remains in the testing phases.

Researchers have already proven the safety and efficacy of cyclophosphamide in other diseases. The U.S. Food and Drug Administration (FDA) approved it for use in a number of cancers and diseases, including breast cancer and leukemia. Doctors have seen mixed results in clinical trials but remain hopeful that the drug can be implemented successfully in mesothelioma treatment regimens.

Now, researchers are studying its effects in combination with other chemotherapy drugs and in combination with immunotherapy on mesothelioma patients.

Cyclophosphamide Information

Cyclophosphamide Pills
  • Other names: Clafen, Cytoxan, Neosar
  • Manufactured by: West-Ward Pharmaceuticals
  • FDA-approved: Yes (to treat leukemia and breast cancer)
  • Used or tested in: Breast cancer, leukemia, lymphoma, mesothelioma, myeloma, neuroblastoma, ovarian cancer, retinoblastoma

How Is It Used?

Unlike traditional chemotherapy drugs that must be administered intravenously, cyclophosphamide may also be administered in tablet form, once or twice daily, or by injection. Mesothelioma patients in clinical trials usually receive the drug in the traditional intravenous manner, typically once every two weeks.

Cyclophosphamide is an alkylating agent, which means it is designed to inhibit tumor growth by interfering with the DNA of cancer cells. It can bind with DNA in mesothelioma cells. Once it does so, it prevents cell division and prompts the death of cancer cells.

It is commonly combined with other chemotherapy drugs or immunotherapy agents such as SS1P and certain vaccines.

What Are the Side Effects?

Cyclophosphamide comes with the general side effects of chemotherapy such as fatigue, weight loss, nausea and hair loss. It may cause additional side effects of abdominal pain, chest pain or shortness of breath, which usually are not severe.

However, doctors have noted one major potential side effect: an increased risk of other types of cancer. Long-term use and high doses create the greatest risk. Bladder cancer is the most common resulting cancer, which may develop years after use.

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Study Results

Researchers have had mixed results when testing cyclophosphamide in mesothelioma patients.

One study had positive results when patients underwent radiation therapy followed by a chemotherapy regimen of cyclophosphamide and doxorubicin. Adding the chemotherapy regimen caused tumors to decrease in size by about 25 percent. Overall, patients who received radiation therapy and chemotherapy survived a median of 13 months, as compared with only six months in patients who received radiotherapy alone. However, these results may be slightly skewed since chemotherapy was only given to those patients who were 70 or younger and who responded well to initial radiation therapy.

A study from 2010 combined cyclophosphamide with doxorubicin and platinum-based chemotherapy. Success was measured by progression-free survival, which is the amount of time that passes after treatment until the cancer spreads. In the three patients receiving this chemotherapy combination, median progression-free survival was only 1.5 months. This is significantly shorter than the median 12.3 months achieved by a regimen of pemetrexed and platinum-based chemotherapy.

A 2017 study combined cyclophosphamide with an immunotherapy approach known as T-cell therapy in six pleural mesothelioma patients. Of the four patients who were evaluable at publication, one showed tumor shrinkage, another’s tumor stopped growing and the other two did not respond (their cancer progressed). Researchers proved the activity of this combination against mesothelioma and may continue to study it in future trials.

Doctors see promise in cyclophosphamide for the treatment of mesothelioma, and clinical trials continue to test the drug on mesothelioma patients. Ongoing studies are testing the chemotherapy agent in conjunction with immunotherapy or other chemotherapy drugs for use in treating mesothelioma patients.

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Karen Selby, RN and Patient Advocate at The Mesothelioma Center

Karen Selby joined Asbestos.com in 2009. She is a registered nurse with a background in oncology and thoracic surgery and was the director of a tissue bank before becoming a Patient Advocate at The Mesothelioma Center. Karen has assisted surgeons with thoracic surgeries such as lung resections, lung transplants, pneumonectomies, pleurectomies and wedge resections. She is also a member of the Academy of Oncology Nurse & Patient Navigators.

Sources
  1. Aerts, J. (2011). Dendritic Cell-based Immunotherapy Combined with Low-dose Cyclophosphamide in Patients with Malignant Mesothelioma [Clinical Trial]. Erasmus Medical Center. Retrieved from http://clinicaltrials.gov/ct2/show/NCT01241682
  2. American College of Rheumatology. (2010). Cyclophosphamide (Cytoxan). Retrieved from https://www.rheumatology.org/I-Am-A/Patient-Caregiver/Treatments/Cyclophosphamide-Cytoxan
  3. Anderson, T.M. (2011). Vaccine Therapy in Treating Patients with Stage I, Stage II, or Stage IIIA Non-small Cell Lung Cancer or with Stage I or Stage II Mesothelioma [Clinical Trial]. Roswell Park Cancer Institute. Retrieved from http://www.clinicaltrials.gov/ct2/show/NCT00003974
  4. Kim, S.T. et al. (2010). The Efficacy of the Frontline Platinum-based Combination Chemotherapy in Malignant Peritoneal Mesothelioma. Japanese Journal of Clinical Oncology, 40(11), 1031-1036. Retrieved from  https://academic.oup.com/jjco/article/40/11/1031/906872/The-Efficacy-of-the-Frontline-Platinum-based
  5. Linden, C.J., et al. (1996). Effect of hemithorax irradiation alone or combined with doxorubicin and cyclophosphamide in 47 pleural mesotheliomas: a nonrandomized phase II study. Retrieved from http://erj.ersjournals.com/content/9/12/2565.full.pdf
  6. Medline Plus. (2011). Cyclophosphamide. Retrieved from http://www.nlm.nih.gov/medlineplus/druginfo/meds/a682080.html
  7. Medline Plus. (2011). Cyclophosphamide Injection. Retrieved from http://www.nlm.nih.gov/medlineplus/druginfo/meds/a611044.html
  8. National Cancer Institute. (2011). Cancer Drug Information: Cyclophosphamide. Retrieved from http://www.cancer.gov/cancertopics/druginfo/cyclophosphamide
  9. National Cancer Institute. (2011). Dictionary of Cancer Terms: alkylating agent. Retrieved from http://www.cancer.gov/dictionary?cdrid=45589
  10. National Cancer Institute. (2011). NCI Drug Dictionary: Cyclophosphamide. Retrieved from http://www.cancer.gov/drugdictionary?CdrID=39748
  11. National Cancer Institute. (2011). Pilot Study of Allogeneic Tumor Cell Vaccine with Metronomic Oral Cyclophosphamide and Celecoxib in Patients Undergoing Resection of Lung and Esophageal Cancers, Thymic Neoplasms, and Malignant Pleural Mesotheliomas [Clinical Trial]. Retrieved from http://www.clinicaltrials.gov/ct2/show/NCT01143545
  12. National Cancer Institute. (2011). SS1P and Pentostatin Plus Cyclophosphamide for Mesothelioma [Clinical Trial]. Retrieved from http://www.clinicaltrials.gov/ct2/show/NCT01362790
  13. Wiseman, C.L. (2009). Cyclophosphamide Plus Vaccine Therapy in Treating Patients with Advanced Cancer [Clinical Trial]. National Cancer Institute. Retrieved from http://www.clinicaltrials.gov/ct2/show/NCT00002475
  14. Lee, S., et al. (2017). Phase 1/2 Trial of WT1 TCR-Transduced Central Memory and Naïve CD8+T Cells for Patients with Mesothelioma and Non-Small Cell Lung Cancer. Journal of Thoracic Oncology; 12(1):S1384-S1385. doi: 10.1016/j.jtho.2016.11.1962

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