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

6 articles matching "advanced cancer"

Clinical trialUNITERARE2 days ago

New Recruiting Trial: Biofeedback-Assisted Pelvic Floor Rehabilitation for Radiation-Induced Sexual Dysfunction in Cervical Cancer Survivors

Researchers are testing a new treatment to help cervical cancer survivors who have sexual problems after radiation therapy. The treatment uses biofeedback—a technique that helps you learn to control your pelvic floor muscles—combined with physical rehabilitation exercises. This trial is now accepting patients to see if this approach can improve sexual function and quality of life.

WHY IT MATTERSThis trial directly addresses a common but often overlooked side effect of cervical cancer treatment, offering cervical cancer survivors a non-medication option to restore sexual function after radiation therapy.
You can act on thiscervical cancerradiation-induced sexual dysfunction
GrantNIH REPORTERMay 11

New NIH Grant: Lysine Methylation as a Novel Regulatory Mechanism of HPV E2 Function — $209K at Unknown Institution

Scientists are studying how a virus called HPV (human papillomavirus) uses a process called lysine methylation to survive in human cells. HPV can cause several types of cancer, including cervical cancer. This research aims to understand how the virus works so doctors can develop better treatments to stop HPV infections before they turn into cancer.

WHY IT MATTERSThis basic research could eventually lead to new non-invasive treatments for high-risk HPV infections, which currently have no effective alternatives to invasive procedures and surgery.
Good to knowCervical cancerOropharyngeal cancerAnogenital cancer
GrantNIH REPORTERMay 11

New NIH Grant: Programmable Multiplexed Detection of Cell-free DNA Mutation for Liquid Biopsy — $415K at Unknown Institution

Researchers are developing a new blood test that can detect tiny pieces of cancer DNA floating in the bloodstream to catch lung cancer early. Instead of invasive procedures like biopsies or CT scans, this simple blood test could help doctors find lung cancer sooner and monitor how well treatment is working. The National Institutes of Health is funding this $415,000 research project starting in 2026.

WHY IT MATTERSFor lung cancer patients, this blood-based test could enable earlier detection when treatment is most effective and reduce the need for invasive biopsies or repeated imaging scans.
👁 Watch this spacelung cancer
ResearchNIH REPORTERMay 11

New NIH Grant: Advanced development and validation of aliquot-level visual indicators of biospecimen exposure to th — $354K at Unknown Institution

Scientists are developing a new tool to help doctors know when frozen tissue and blood samples have thawed or been stored improperly. This matters because many cancer tests, like HER2 testing for breast cancer, require samples to stay fresh and cold. If samples warm up accidentally, the test results could be wrong. This $354,000 research project will create visual indicators—like color-changing labels—that show when a sample has been exposed to unsafe temperatures.

WHY IT MATTERSPatients with breast cancer undergoing HER2 testing depend on properly preserved tissue samples for accurate diagnosis; visual thaw indicators could prevent misdiagnosis caused by specimen degradation.
Good to knowbreast cancer
🔴 BreakingDrug approvalRSSMay 8

FDA Grants Seventh Approval under the National Priority Voucher Pilot Program

The FDA approved a new drug called Bizengri that treats a very rare and aggressive bile duct cancer called NRG1 fusion-positive cholangiocarcinoma. This approval is special because it's the seventh drug to receive fast-track approval through the FDA's National Priority Voucher Pilot Program, which helps speed up treatments for the rarest diseases. This cancer forms in the tubes that carry bile from the liver, and this new treatment targets a specific genetic change found in some patients with this disease.

WHY IT MATTERSPatients with NRG1 fusion-positive cholangiocarcinoma now have access to the first FDA-approved targeted treatment specifically designed for their genetic subtype of this ultra-rare cancer.
💬 Ask your doctorNRG1 fusion-positive cholangiocarcinoma
Clinical trialCLINICALTRIALSApr 24

New Clinical Trial: Attention and Interpretation Modification (AIM) for Fear of Cancer Recurrence: An Intervention Development Study (NCT01517945)

Researchers at Memorial Sloan Kettering Cancer Center are testing a smartphone app designed to help breast cancer and rare cancer survivors deal with the fear that their cancer might come back. The app uses a technique called Attention and Interpretation Modification (AIM) to help people change how they think about cancer recurrence. The study has enrolled 252 people and is no longer recruiting new participants, but the results could help many cancer survivors in the future.

WHY IT MATTERSThis trial addresses a common but often overlooked mental health challenge for cancer survivors—fear of recurrence—by testing a personalized, accessible mobile app intervention that could be widely available if proven effective.
👁 Watch this spaceBreast CancerRare Cancers

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