Nearly every kidney cancer treatment available today was studied in clinical trials before being approved for wider use. Some research studies test new ways to diagnose kidney cancer, while others test new medications, combinations of medications, surgeries, or other treatments. All share the common goal of improving outcomes for people living with kidney cancer.

In this article, we’ll cover recent advances in kidney cancer clinical trials and why you or your loved one might want to consider joining one. We’ll also cover the possible benefits and potential risks of joining a clinical trial for kidney cancer.

The Role of Clinical Trials for Cancer

Clinical trials, also called interventional studies, are research studies that explore new ways to diagnose, prevent, or treat cancer by testing them in people with a specific condition.

Often, researchers set other criteria for who can participate. Known as eligibility criteria, these rules can include:

• Specific age ranges
• Cancer stages
• Whether a person has certain conditions or treatments in their medical history

Clinical trials for cancer are important parts of cancer research. They can play a pivotal role in finding ways to diagnose, prevent, or treat cancer. Because of clinical trials, people with many types of cancer now have more treatment options and better prognoses (outlooks) than ever before.

There are a few different types of clinical trials that help researchers answer questions about cancer:

• Prevention trials — Look for new ways to prevent cancer or lower risk
• Screening trials — Explore new ways to detect cancer as early and accurately as possible
• Treatment trials — Test new cancer treatments or new combinations of existing treatments
• Supportive or palliative care trials — Study new ways to improve the quality of life for people living with cancer or going through cancer treatment

Once a clinical trial is complete in the United States, the study results may be submitted to the U.S. Food and Drug Administration (FDA) for experts to review. The FDA then decides whether to approve the test or treatment for broader use.

Recent Advances in Kidney Cancer Clinical Trials

Clinical trials for kidney cancer help researchers find new treatments and more effective ways to use existing treatment options.

Thanks in part to advances from clinical trials, about 75 percent of people with kidney cancer live at least five years after diagnosis. This is called the five-year relative survival rate, which compares people with kidney cancer with people in the general population.

In many cases, a drug or treatment being tested for kidney cancer isn’t yet available outside a clinical trial because it hasn’t been approved by the FDA yet. But new kidney cancer treatments continue to become available because of clinical trial research.

Following is the most recent kidney cancer research news.

Belzutifan for Advanced Clear Cell Renal Cell Carcinoma

In 2023, the FDA approved belzutifan (Welireg), a targeted therapy, for adults with advanced renal cell carcinoma (RCC) with a clear cell component who had previously received certain other treatments.

Clear cell RCC is the most common type of kidney cancer, and many cancer cells in this type of tumor have mutations (changes) in a gene called von Hippel-Lindau (VHL). This gene typically helps prevent tumor growth, but a VHL gene mutation can make tumors more likely to grow.

Belzutifan blocks a protein involved with VHL called HIF-2 alpha. In clinical trials, clear cell RCC tumors shrank in people with VHL disease.

Belzutifan was also the first drug ever approved to treat RCC in people with VHL disease. It was approved for this use in 2021.

Ongoing clinical trials are now testing belzutifan in combination with other targeted therapies and immunotherapy.

Pembrolizumab for Renal Cell Carcinoma After Nephrectomy

In 2021, kidney cancer drug trials led to the approval of pembrolizumab (Keytruda), a type of immunotherapy drug called a PD-1 immune checkpoint inhibitor. The drug is indicated for people with RCC who have a high risk of kidney cancer recurrence (returning) after nephrectomy (surgery to remove part or all of the affected kidney).

In clinical trials, pembrolizumab helped people with clear cell RCC live longer after surgery.

Treatments meant for after surgery, called adjuvant treatments, can come with serious side effects. For instance, pembrolizumab caused side effects in 20 percent or more of clinical trial participants. These side effects included:

• Rash
• Fatigue
• Diarrhea
• Itchiness
• Hypothyroidism (low thyroid hormones)
• Musculoskeletal pain

It’s always important to discuss the possibilities with your doctor before trying adjuvant therapies for clear cell RCC after nephrectomy.

Lenvatinib Plus Pembrolizumab for Advanced Renal Cell Carcinoma

Also in 2021, the FDA approved combination therapy with the drugs pembrolizumab and lenvatinib (Lenvima) as a first-line treatment for advanced RCC in adults. Lenvatinib is a targeted therapy.

Combinations of targeted therapy and immunotherapy, such as lenvatinib plus pembrolizumab, are commonly used in the treatment of advanced kidney cancer. Ongoing clinical trials are studying more of these combinations to treat clear cell RCC, papillary RCC, and other kidney cancer types.

Tivozanib for Relapsed Advanced Renal Cell Carcinoma

In March 2021, the FDA approved a targeted therapy drug called tivozanib (Fotivda) to treat relapsed or refractory (unresponsive to treatment) advanced RCC in adults who have already been treated with at least two systemic (whole-body) therapies.

Tivozanib is a kinase inhibitor, which means it blocks a specific enzyme that helps RCC grow.

How To Join a Clinical Trial

You can ask your doctor about clinical trials and whether you might qualify for one at any point during your kidney cancer treatment.

People with newly diagnosed RCC may be eligible for some clinical trials, and people with stage 4 kidney cancer, also called metastatic kidney cancer, may be eligible for others.

Ask your doctor about available options based on your kidney cancer stage, your cancer type, your age, and any treatments you’ve tried in the past.

Possible Benefits of Joining a Clinical Trial

Joining a clinical trial can have benefits for you or your loved one with kidney cancer, as well as for people diagnosed with kidney cancer in the future. Some clinical trials may give you access to a new treatment that isn’t yet approved or widely available.

Although there’s no guarantee that a treatment in a clinical trial will help you, finding new treatments and prevention strategies for kidney cancer can help people in the future access more effective care as they navigate life with kidney cancer. Kidney cancer clinical trials can also help researchers learn more about the disease and the most effective ways to treat it.

Clinical trials also go through multiple phases to protect the safety of participants. If researchers discover dangerous side effects or determine that a treatment isn’t effective, the trial doesn’t progress to the next phase. Researchers carefully observe participants through every phase, and the trial happens in a controlled setting.

It’s important to note that you can decide to leave a clinical trial at any time, for any reason.

Researchers also work hard to protect your privacy. Your individually identifiable health information will be kept private. Researchers will tell you ahead of time how information from the trial will be used and may ask for your permission to use your health information in specific ways.

Possible Risks of Joining a Clinical Trial

Even though researchers carefully plan clinical trials and observe participants closely, joining a clinical trial can still come with risks. You might not see any improvement in your prognosis, or you might experience side effects from a treatment being tested.

In some cases, participating in a clinical trial takes more time and requires more visits to a cancer center than undergoing the standard treatment for your type of kidney cancer.

Clinical trials for cancer rarely use placebos (inactive medications designed to compare results with the active medication). Instead, they compare the medication being tested with the current approved standard therapy.

In rare cases when a placebo is given to some people, the research team will tell you it’s a possibility. But in almost all oncology clinical trials, you’ll receive an active treatment of some kind.

Talk to Your Kidney Cancer Specialist

If you have kidney cancer and are interested in learning more about clinical trials, talk to your kidney cancer specialist about current research opportunities. Your doctor can connect you with clinical trials near you and help you determine if you’re eligible.

Join the Conversation

On MyKidneyCancerTeam, people share their experiences with kidney cancer, get advice, and find support from others who understand.

Have you participated in a clinical trial for kidney cancer? Let others know in the comments below.