How clinical trials are transforming cancer care

Targeted therapies, immunotherapy, and precision medicine represent some of the most significant advancements in cancer treatment in recent years. These approaches, born out of rigorous research and testing, have reshaped the combat against cancer, offering new hope and improved outcomes.

· Targeted therapies: Targeted therapies aim to inhibit specific molecules or pathways involved in tumour growth and survival. Unlike traditional chemotherapy, which indiscriminately kills rapidly dividing cells, targeted therapies are designed to selectively target cancer cells while sparing normal tissues. Key discoveries in targeted therapy include the development of tyrosine kinase inhibitors (TKIs) for cancers driven by aberrant signalling pathways, beginning with the epidermal growth factor receptor (EGFR) mutations in non-small cell lung cancer with expansion now to other targets and tumours.

· Immunotherapy: Immunotherapy harnesses the power of the immune system to recognise and eliminate cancer cells. By unleashing the body's natural defences against cancer, immunotherapy has emerged as a game-changing approach for treating a wide range of malignancies, including melanoma, lung cancer, and bladder cancer. The next generation of immunotherapy approaches include antibody drug conjugates, CAR T cell constructs, tumour infiltrating lymphocytes (TILs) and vaccines. Clinical trials for an innovative pancreatic cancer vaccine, for example, are underway.

· Precision medicine: Precision medicine tailors treatment strategies to the unique genetic makeup and molecular characteristics of patients. By identifying specific biomarkers or genetic alterations driving cancer growth, precision medicine allows for targeted interventions that maximise efficacy and minimise toxicity. Clinical trials have shown that matching patients with targeted therapies based on their molecular profiles can lead to higher response rates and longer progression-free survival compared to conventional chemotherapy.

Innovations in cancer therapy, whose safety and efficacy have been validated by clinical trials, have ushered in a new era of hope and progress. Targeted therapies, immunotherapy, and precision medicine have transformed the treatment landscape, offering personalised, effective, and less toxic options for patients facing this devastating disease.

While clinical trials play a pivotal role in advancing medical science, conducting these trials poses numerous challenges and barriers. Fewer than 5% of patients participate on a trial and in some parts of the world, less than 1% of patients are enrolled. Recruiting participants for clinical trials is often a major bottleneck, delaying the completion of studies, compromising their statistical power, and depriving patients of novel therapies with potentially enhanced benefit. Moreover, navigating regulatory requirements is another significant challenge as trials are performed globally but the burden of ensuring safety and providing informed consent for participants is significant. The complexity and variability of regulations across different jurisdictions can add to administrative burdens and delays. Educating patients about the possible benefits and risks of participation in a given study and ensuring this information is available to all patients irrespective of where they are receiving care remains a critical need.

Funding constraints are an additional barrier, particularly for investigator-initiated studies or research in niche therapeutic areas with limited commercial interest. Securing adequate funding for trial initiation, implementation, and dissemination of results is essential for sustaining scientific progress and ensuring the viability of clinical research endeavours.

Despite the challenges and barriers, opportunities exist to enhance trial efficiency and accessibility through innovative approaches and collaborative initiatives. Harnessing advances in technology, such as electronic data capture, telemedicine, and decentralised trial models, can streamline trial operations, reduce administrative burden, and enhance patient engagement. These technologies can also increase access globally.

Moreover, ethical considerations lie at the heart of clinical trial design and implementation, ensuring that research endeavours uphold principles of respect. Obtaining informed consent is a cornerstone of ethical clinical research, ensuring that participants are fully informed about the nature of the study, its potential risks and benefits, their rights as research participants, and setting context about how the investigational therapy may fit into reasonable options for their particular clinical situation.

Protecting patient privacy and confidentiality is also paramount in clinical research, particularly in the era of electronic health records and big data analytics. Researchers must implement robust data security measures to safeguard sensitive patient information against unauthorised access, disclosure, or misuse.

With advancements ranging from sophisticated surgical methods to precise therapy and groundbreaking immunotherapy strategies, the options to treat cancer have never been more extensive. Robust clinical trial design coupled with innovative therapy will continue to improve the way cancer is treated.

This article is authored by Dr. Paul Sabbatini, MD - senior vice-president for clinical research and MSK gynaecologic oncologist.