Rare disease news

FDA approvals, research breakthroughs, clinical trials, and advocacy updates

Curated and summarized by AI for patients and caregivers

🔍
AllDrug approvalsClinical trialsResearchGrants & fundingAdvocacy & policyPipeline
Show:All newsBreaking onlyImportant & breaking
Date:7 days30 days90 daysAll time

8 articles from the last 90 days matching "immune system"

Clinical trialCLINICALTRIALS5 days ago

Trial Now Recruiting: Novel Genetic Disorders of the Immune System (NCT02257892)

Researchers are looking for adults and children with certain immune system disorders to join a study. The immune system normally helps your body fight infections, but in some people it doesn't work properly, causing frequent infections and other health problems. This study wants to understand why some immune systems fail and how to help people with these conditions. Relatives of affected people may also be able to join.

WHY IT MATTERSThis trial is actively recruiting 500 participants with four specific genetic immune disorders (PI3KCD, CTLA4, STAT3GOF, and MAGT1 deficiency) — if you or a family member has one of these diagnoses, you may be eligible to enroll now and contribute to understanding these rare conditions.
You can act on thisPI3KCD deficiencyCTLA4 deficiencySTAT3 gain-of-function disorderRead →
Clinical trialUNITERAREApr 15

New Recruiting Trial: Temozolomide and Survivin Long Peptide Vaccine (SurVaxM) for the Treatment of Patients With Progressing Metastatic Neuroendocrine Carcinomas

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.
You can act on thisneuroendocrine carcinomametastatic neuroendocrine carcinomaRead →
Clinical trialUNITERAREApr 5

New Recruiting Trial: Recombinant Glycosylated Human Interleukin-7 (CYT107) for the Treatment of Kaposi Sarcoma in Participants With HIV and Immune Non-Response (REGIMENKS HIV)

Researchers are testing a new drug called CYT107 to help people with HIV who have Kaposi Sarcoma (a type of cancer) and whose immune systems aren't responding well to treatment. CYT107 is designed to boost the immune system to help fight the cancer. This is a Phase 2 trial, meaning it's testing whether the drug works and is safe in a larger group of people. The trial is now accepting patients and will start in April 2026.

WHY IT MATTERSThis trial offers a potential new treatment option for people with HIV-related Kaposi Sarcoma who haven't responded to standard immune-boosting treatments, addressing a significant gap in care for this vulnerable population.
You can act on thisKaposi SarcomaHIV/AIDSRead →
Clinical trialUNITERAREApr 5

New Recruiting Trial: Natural History of Dysregulation and Aging of the Immune System in People With Trisomy 21 With and Without Thymectomy

Researchers are looking for people with Down syndrome (Trisomy 21) to join a study about how their immune system works differently and ages over time. Some participants will have had their thymus gland removed, while others won't have, so scientists can compare how this affects immune health. The study will help doctors understand why people with Down syndrome often get more infections and have other immune-related health problems.

WHY IT MATTERSThis trial is now recruiting people with Down syndrome to help researchers understand immune system problems that affect this population — findings could lead to better treatments for infections and other immune conditions common in Trisomy 21.
You can act on thisTrisomy 21Down syndromeRead →
Clinical trialUNITERAREApr 5

New Recruiting Trial: Evaluating the Safety and Tolerability of Baricitinib in Patients With Job Syndrome With Lupus-Like Disease and/or Atopic Dermatitis

Researchers are testing a drug called baricitinib to see if it is safe and works well for patients with Job syndrome who also have lupus-like disease and/or atopic dermatitis (severe itchy skin). Job syndrome is a rare immune system disorder that makes it hard for the body to fight infections and causes skin problems. This is an early-stage study that will check for side effects and how well the drug works.

WHY IT MATTERSThis Phase 1 trial is now actively recruiting patients with Job syndrome complicated by lupus-like disease and/or atopic dermatitis — a rare combination that has limited treatment options and is being studied by the National Institute of Allergy and Infectious Diseases.
💬 Ask your doctorJob syndromeAtopic dermatitisLupus-like diseaseRead →
Clinical trialCLINICALTRIALSMar 26

New Clinical Trial: Atezolizumab and Bevacizumab in Treating Patients With Rare Solid Tumors (NCT03074513)

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.
💬 Ask your doctorAppendix AdenocarcinomaHuman Papillomavirus-Related Anal Squamous Cell CarcinomaHuman Papillomavirus-Related Cervical Squamous Cell CarcinomaRead →
Clinical trialCLINICALTRIALSMar 26

New Clinical Trial: Nivolumab and Ipilimumab in Treating Patients With Rare Tumors (NCT02834013)

Researchers are testing two cancer-fighting drugs called nivolumab and ipilimumab together to treat patients with rare types of cancer. These drugs help the body's immune system recognize and fight cancer cells. The trial is currently active but not accepting new patients at this time.

WHY IT MATTERSThis trial tests a combination immunotherapy approach across 50+ rare tumor types, offering potential treatment options for patients with uncommon cancers that have limited standard therapies available.
👁 Watch this spaceAcinar Cell CarcinomaAdenoid Cystic CarcinomaAdrenal Cortical CarcinomaRead →
Clinical trialCLINICALTRIALSMar 26

New Clinical Trial: Talimogene Laherparepvec and Nivolumab in Treating Patients With Refractory Lymphomas or Advanced or Refractory Non-melanoma Skin Cancers (NCT02978625)

Researchers are testing a combination of two cancer-fighting treatments called talimogene laherparepvec and nivolumab in patients with rare lymphomas (blood cancers) and skin cancers that didn't respond to previous treatments. Talimogene laherparepvec is a modified virus that helps the immune system fight cancer cells, while nivolumab is an immunotherapy drug that removes the brakes on the immune system. This study involves 68 patients and is currently enrolling participants.

WHY IT MATTERSThis trial offers a potential new treatment option for patients with refractory (treatment-resistant) rare lymphomas like mycosis fungoides and Merkel cell carcinoma, which have very limited options after standard therapies fail.
💬 Ask your doctorAnaplastic Large Cell Lymphoma (ALK-Negative)Anaplastic Large Cell Lymphoma (ALK-Positive)Merkel Cell CarcinomaRead →

Get personalized rare disease news

Follow your conditions to see news about the diseases that matter to you — FDA approvals, trial openings, and research breakthroughs.

Create free account →Browse diseases