Clinical trialCLINICALTRIALSApr 15
Researchers are testing a cancer drug called nivolumab in patients with rare tumors that have a specific marker called PD-L1. This is a Phase 2 trial that will include up to 28 patients with many different types of rare cancers who haven't responded well to standard treatments. The study will last up to 12 months and measure how well the drug works.
WHY IT MATTERSIf you have one of the 43 rare tumor types listed and your cancer has high PD-L1 expression, this trial offers access to an immunotherapy that may work regardless of where your cancer started.
Clinical trialUNITERAREApr 15
Researchers are testing a new cancer treatment that combines two approaches: a chemotherapy drug called temozolomide and a vaccine called SurVaxM that trains the immune system to fight cancer cells. This trial is for patients with neuroendocrine carcinomas (rare cancers in hormone-producing cells) that are spreading and getting worse despite other treatments. The study is now accepting patients and will run through 2026.
WHY IT MATTERSThis is one of the first trials testing an immunotherapy vaccine specifically for metastatic neuroendocrine carcinomas, offering a potential new option for patients whose cancer has progressed on standard treatments.
ResearchPUBMEDApr 10
Doctors from around the world created a network called the International Neuroblastoma Risk Group to share information about neuroblastoma, a type of cancer that starts in nerve cells. They combined data from over 25,000 patients to help researchers understand the disease better and develop better treatment plans. This teamwork model shows how rare cancer research can be improved when hospitals and countries work together.
WHY IT MATTERSPatients with neuroblastoma now benefit from standardized treatment approaches and risk classifications developed through this international collaboration, which means more consistent and potentially better care regardless of where they receive treatment.
ResearchPUBMEDApr 1
Researchers studied whether the amount of cancer in a patient's body before treatment affects how well two immunotherapy drugs work together. They looked at 722 patients with rare cancers who received nivolumab and ipilimumab (two drugs that help the immune system fight cancer). The study wanted to understand if patients with smaller tumors do better than those with larger tumors when treated with these combination drugs.
WHY IT MATTERSIf baseline tumor size predicts treatment response in rare cancers, doctors could better counsel patients on expected outcomes and identify which patients might benefit most from this dual immunotherapy approach before starting treatment.
ResearchPUBMEDMar 26
Scientists are developing tiny particles called exosomes that can deliver cancer-fighting medicines directly to rare cancer tumors. These natural particles act like delivery trucks, carrying drugs to cancer cells while reducing damage to healthy cells. Early research shows this approach could help rare cancer patients who currently have few treatment options.
WHY IT MATTERSPatients with rare cancers often lack targeted treatments and face delayed diagnoses—exosome-based therapies could provide new options by delivering drugs more effectively to tumors while causing fewer side effects.
ResearchPUBMEDMar 26
Researchers studied how cancer tumors respond differently to a two-drug immunotherapy treatment (ipilimumab plus nivolumab) in patients with rare cancers. Some patients had some tumors shrink while others grew at the same time—called 'mixed response.' This study looked at 438 patients to understand how this mixed response affects how long patients survive and whether the cancer comes back.
WHY IT MATTERSUnderstanding mixed response patterns helps doctors better predict which rare cancer patients will benefit most from dual checkpoint inhibitor therapy and may improve how treatment success is measured beyond just tumor size.
Clinical trialCLINICALTRIALSMar 26
Researchers are testing a combination of two immunotherapy drugs in patients with four types of rare cancers: neuroendocrine tumors, biliary tract cancers, ovarian clear cell carcinoma, and tumors with high microsatellite instability. This Phase 2 trial involves 240 patients and is based on earlier research showing these cancers may respond well to this type of treatment.
WHY IT MATTERSPatients with these rare cancers now have access to a targeted immunotherapy combination that showed promise in earlier studies, potentially offering a new treatment option for cancers that historically have limited therapeutic choices.
Clinical trialCLINICALTRIALSMar 26
Researchers are testing whether two cancer-fighting drugs called atezolizumab and bevacizumab work better together for treating rare cancers. These drugs help the body's immune system recognize and attack cancer cells. The study includes 133 patients with various uncommon solid tumors and is currently enrolling participants.
WHY IT MATTERSThis trial offers patients with rare cancers like Merkel cell carcinoma, nasopharyngeal cancer, and mesothelioma access to a combination immunotherapy treatment that may not be available outside of clinical research.
Clinical trialCLINICALTRIALSMar 26
Researchers are testing a drug called cabozantinib-s-malate to treat children and young adults with rare cancers, including sarcomas (muscle cancers), Wilms tumor (kidney cancer), and other solid tumors that have come back after treatment or didn't respond to initial therapy. The drug works by blocking proteins that help tumors grow and form new blood vessels. This phase II trial has enrolled 109 patients and is no longer recruiting new participants.
WHY IT MATTERSIf your child has a recurrent or treatment-resistant sarcoma, Wilms tumor, or other rare pediatric solid tumor, this completed trial data may help inform whether cabozantinib could be an option to discuss with their oncologist.
Clinical trialCLINICALTRIALSMar 26
Researchers are testing whether combining three cancer drugs—nivolumab, ipilimumab, and cabozantinib—can help treat rare cancers of the bladder, kidney, prostate, and other urinary system organs. This phase 2 trial is actively recruiting 314 patients to see if this drug combination works better than current treatments. The study is being run by the National Cancer Institute.
WHY IT MATTERSThis trial offers patients with rare genitourinary cancers (like collecting duct carcinoma, kidney medullary carcinoma, and rare bladder variants) access to a novel three-drug combination that may be more effective than standard treatments currently available.