Clinical Trials in Specific Therapeutic Areas: Insights and Developments
Scientific research is a global endeavor, with thousands of clinical trials in progress at any given time. Researchers focus on the most common health conditions, such as cancer or heart disease, as well as rare diseases with only a handful of known cases.
Table of Contents
Breakthrough Research in Cancer
New Research on Preventing Cardiovascular Disease
Expanding Patient Access to Rare Disease Studies
Learn More About Clinical Trials
Researchers use clinical trials to make new discoveries about a wide variety of health conditions. Some studies look at the most common conditions, like cancer and heart disease, to try to reduce the harm those conditions cause. Other researchers focus on finding new treatments for rare diseases. All studies rely on individuals who agree to participate in the research. Their role in studies may be as simple as giving a blood sample. Others agree to be among the first people to try a new treatment protocol for cancer.
Regardless of the medical specialty, clinical trials like this are critical to major medical breakthroughs. Clinical trial participants help researchers learn new information about health conditions. That information leads to new treatments that improve people’s lives.
Breakthrough Research in Cancer
Oncology is a field that has seen tremendous advancements in recent years. A greater understanding of cancer genomics and the value of immunotherapies and targeted therapies has advanced cancer treatment dramatically. In the past several years, oncology clinical trials have led to breakthroughs in treating cancers that were once considered deadly.
In 2023, researchers published findings from the SORAYA study, a trial testing the efficacy of Mirvetuximab soravtansine (MIRV) for ovarian cancer. The Phase 2 stage of the trial found dramatic benefits for participants who received the treatment. As a result, the FDA granted accelerated approval for the new protocol. This approval allowed the doctors to prescribe the protocol for patients who met the treatment criteria. Additional findings from the Phase 3 MIRASOL trial, a double-blind study with 453 participants, show that MIRV is effective in contributing to overall survival and reducing the risk of disease progression or death compared with chemotherapy. This is especially true for patients with folate receptor alpha (FRα)-high, platinum-resistant ovarian cancer.
Another promising cancer study is trying to find a way to stop the spread of cancer within the body. Researchers are looking at the effects of a drug called digoxin on circulating tumor cells (CTCs). Investigators believe CTCs are responsible for the metastasis, or spread, of cancer. This Phase 1 trial has examined whether participants with advanced or metastatic breast cancer who take digoxin show a dissolution of CTC clusters, with results to follow.
New Research on Preventing Cardiovascular Disease
Heart disease is one of the most common conditions in adults. Prevention has been a priority, with doctors recommending lifestyle changes to reduce the possibility of developing heart disease. New research is examining other factors that may increase risk.
Cardiovascular researchers are looking at factors that may identify cardiovascular disease (CVD) risks and lead to methods for preventing CVD. Cardiovascular clinical trials involve recruiting patients and their family members to give blood samples for genetic testing. The research may help doctors understand what aspects of genetics, anatomy, and biology may contribute to CVD development.
In one study, the Mayo Clinic partnered with the National Health Service in England to collect data from people with known arrhythmogenic ventricular cardiomyopathy (AVC) and their blood relatives. The goal is to identify biomarkers that predict the disease's risk, likely onset, and progression. The study is ongoing, with participant enrollment open.
A second Mayo Clinic study is looking at biomarkers that are present in adults hospitalized for congestive heart failure. Investigators are asking people to submit blood and urine samples. They’ll use the samples to look for what proteins are present. The findings may offer insight into what biomarker measures correlate to heart failure risk. The main portion of the study will wrap up in December 2024.
Expanding Patient Access to Rare Disease Studies
Clinical trials for rare diseases—defined as diseases occurring in fewer than 5 out of 10,000 people—have always posed challenges for researchers. Researchers are currently exploring how personalized medicine, such as gene editing, treats rare diseases. Designing the studies can be difficult because there are so few potential participants. Some preclinical work can be done in animal studies, but that cannot always identify potential safety or efficacy issues in human trials.
Researchers are now exploring how to use real-world data about drug effects to better predict outcomes before moving to human trials. Data sharing between drug makers and researchers could open up new ways to model treatment outcomes after animal trials but before human trials. The modeling may identify and mitigate possible side effects before enrolling people in clinical trials.
In addition, rare disease researchers are exploring new ways to structure human trials. Some experts believe that some research can rely on single-arm trials with no placebo group. This increases the sample of patients receiving the intervention. It also mitigates ethical concerns about withholding a promising treatment from a subset of participants. In a single-arm trial, all study participants have access to the treatment.
Learn More About Clinical Trials
Studies on common conditions and rare diseases are possible because of the critical help of people who participate in clinical research. If you want to learn more about clinical trials and how to participate, connect with Studypages and sign up for our free Pulse Newsletter. Our platform helps you become part of real clinical studies and play a role in the advancement of healthcare research.