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Prostate Cancer Research

Johns Hopkins prostate cancer experts lead the world in clinical research on new surgical, medical, radiation and experimental treatments for prostate cancer. For more information on clinical trials in prostate cancer being conducted at Johns Hopkins, visit our Clinical Trials page.


  • Checkpoint inhibitors: Checkpoint inhibitors are drugs that lift the “brakes” on a patient’s immune system so that it can fight back against cancer cells. These drugs have been approved to treat melanoma, kidney and some forms of lung cancer. Researchers at our Prostate Cancer Multidisciplinary Clinic and  the Bloomberg-Kimmel Center for Cancer Immunotherapy, are involved in several clinical trials of these drugs for prostate cancer. The immunotherapies under study include the pembrolizumab (Keytruda) and nivolumab (Opdivo), which target the immune checkpoint protein PD-1; and ipilimumab (Yervoy), which targets the checkpoint protein CTLA-4.
  • Immunotherapy and other treatments: In preclinical studies, Johns Hopkins scientists have seen some promising results combining radiation and stimulation of the immune system that could be useful in treating cancer that has advanced into tissues near the prostate; other possibilities include adding a checkpoint inhibitor to chemotherapies or hormonal therapies in men with advanced prostate cancer.
  • Combined immunotherapies: A phase 2 clinical trial at the Kimmel Center is looking at how patients with the AR-V7 mutation and metastatic castration-resistant cancer respond to a combination of ipilimumab and nivolumab.
  • Other immunotherapies: In another Phase 2 Kimmel Center clinical trial, researchers including Channing Paller, M.D. are testing the effects of the combination of an oral immunotherapy drug called galunisertib and enzalutamide versus enzalutamide alone for metastatic castration-resistant cancer.

Natural Remedy Research

  • Mistletoe: A Phase 1 clinical trial of intravenous mistletoe extract for prostate and other tumor types by Paller and colleagues is planned for late 2016-2017 to evaluate the safety and ability of the extract to shrink tumors.
  • Intravenous Vitamin C: Paller is leading a Phase 2 clinical trial looking at how the combination of the chemotherapy drug docetaxel and intravenous doses of vitamin C might lower PSA levels in patients with metastatic castration-resistant cancer. Intravenous vitamin C has been shown to produce compounds like hydrogen peroxide that attack cancer cells.

Hormone Research

  • High-dose testosterone: Sam Denmeade, M.D.  leads two Phase 2 clinical trials of high-dose testosterone treatment in men with metastatic castration-resistant cancer: the TRANSFORMER study, which compares high-dose treatment with enzalutamide; and the RESTORE study, which is looking at how well high-dose testosterone therapy re-sensitizes patients to ezalutamide or abiraterone.
  • BET inhibitors: An oral BET inhibitor (drugs that have been tested in cancers like AML and multiple myeloma) are being tested alone and in combination with enzalutamide in a Phase1/2 trial for metastatic castration-resistant cancer.
  • Antiandrogen drugs: A Phase II trial planned for early 2017 will look at the experimental oral drug EPI-506, which blocks part of the androgen receptor in men with metastatic castration-resistant cancer, with and without the AR-V7 mutation

Chemotherapy Research

  • Johns Hopkins is the only facility in the world that offers clinical testing for the AR-V7 genetic variant. A 2015 study by Emmanuel Antonarakis, M.D. and others suggests that chemotherapy drugs such as docetaxel may benefit men with castration-resistant cancer. Researchers are using the AR-V7 mutation to guide clinical trials of other drugs and combinations of drugs (see immunotherapy).
  • Although the BRCA2 genetic mutation is most well-known for its role in increasing the risk of breast and ovarian cancer in women, this mutation is found in up to 5 percent of men with metastatic prostate cancer as well. Antonarakis says several studies of olaparib, a type of drug called a PARP inhibitor, will be used in upcoming clinical trials for men who have mutations in 15 different DNA repair genes like BRCA2. The drug will be tested in patients with earlier prostate disease who have elevated PSA levels after prostectomy, and in comparison with enzalutamide for advanced prostate cancer patients.

Oligometastatic Cancer Research

Men with advanced prostate cancer who have five or fewer metastases are called oligometastatic patients. At most cancer centers, treatments for these patients are considered palliative rather than curative. Johns Hopkins clinicians take a more aggressive approach in treating these patients:

  • One clinical study led by Kenneth Pienta, M.D., and aimed at men with five or fewer metastases combines a period of hormone therapy and the chemotherapy drug docetaxel, followed by a radical prostectomy, and then targeted stereotactic radiotherapy to the metastases sites.
  •  The ORIOLE Phase 2 clinical trial led by Phuoc Tran, M.D., for men with one to three bone metastases, will compare outcomes in men who receive stereotactic radiation targeting the metastases with men who receive no radiation or hormone therapy.

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