HJBR Nov/Dec 2023

54 NOV / DEC 2023 I  HEALTHCARE JOURNAL OF BATON ROUGE ONCOLOGY DIAL GUE COLUMN ONCOLOGY EARLY in my training, a practice-changing trial in glioblastoma (GBM) was reported out. GBM is almost universally fatal. At the time, patients had a life expectancy of about a year. This new treatment extended their lives by ten weeks over the previous standard of care. As a young MD/PhD student in my lab years, I was skeptical that this was a meaningful improvement. Years later, I had the opportunity to meet a prominent physician-scientist, now a Nobel laureate, who was also a GBM researcher. He admitted the same skepticism I had when he first saw those trial results. However, his mind was changed when his wife was diag- nosed with GBM and underwent this very treatment. The extra time she was afforded meant she was there for important mile- stones, like their children’s graduations. As a practicing oncologist, I see examples every day of howmeaningful tenmore weeks can be. Ten more weeks can be seeing your team win a championship; witnessing the birth of your child or grandchild; or walking your daughter down the aisle at her wedding. Those ten weeks are made possible by clinical research. Meaningful advancements in clinical research According to the American Cancer So- ciety, cancer death rates in the U.S. have decreased 33% over the last three decades. This remarkable achievement is a testament to the progress we have made, largely due to cancer research. Clinical research in cancer looks at various aspects of the cancer journey — from surveil- lance and early detection of cancer to specific therapies and post-treatment survivorship. Observational studies examine facets such as epidemiology, disparities, care delivery, and patient outcomes without direct impact on the treatment of individual patients. In- terventional clinical trials, however, assess therapies for safety and efficacy by admin- istering them to patients. These can be done in multiple settings: preventative or defini- tive treatments; pre- or post-surgery (neo- adjuvant and adjuvant); first-line or subse- quent-line; or in metastatic or earlier stage. Interventional studies are the most directly associated with improvements in cancer care. The phases of clinical trials Clinical trials move through multiple phases, but the most relevant are Phases 1 through 3: • Phase 0 studies encompass the pre- clinical work, often in cells and animal models, that build the knowledge base to justify proceeding with a Phase 1 trial. • Phase 1 trials focus on establishing the safety of an agent or combination. The typical goal is to select an appropriate dose for later phase trials. Sometimes, these are first-in-human studies, and, generally, every participant is treated with the investigational agent(s). • Phase 2 trials continue safety valida- tion of the investigational agent(s), while looking for early signs of efficacy to jus- tify a larger Phase 3 trial. • Phase 3 trials are designed to prove the efficacy of the investigational agent(s), typically in a randomized controlled trial (RCT) against the standard of care. These are often registrational studies used to apply for an FDA-approved la- bel indication for the agent(s). • Phase 4 studies, also known as post- marketing studies, continue to assess the safety and efficacy of the agent(s) after the approval of a labeled indication. Clinical trials, especially earlier phase tri- als, can provide patients with access to ad- ditional options for treatment that would otherwise be unavailable to them. However, earlier phases of drug development mean more complexity in conducting studies. Phase 1 clinical trials: challenges and rewards Phase 1 trials are a challenge to undertake, and not many centers are equipped to run them. The demands on both patients and staff TEN MORE WEEKS: Improving Cancer Outcomes Through Clinical Trials