From Physician to Patient: Dr. Vargas’s Journey With a Clinical Trial

As a pediatric endocrinologist, Dr. Alfonso Vargas is familiar with the complexities of the human body, including all the incredible processes that happen under the radar and everything that can go wrong. His decades studying and treating conditions related to children’s hormones have taught him how important it is to take care of your body – and how crucial advancements in medicine are when something does go awry.

His experience as a healthcare provider spans the globe, from Colombia (at the Universidad Nacional de Colombia in Bogota) to Florida (at the University of South Florida College of Medicine, Tampa General Hospital, and All Children’s Hospital) to Louisiana (at the Department of Pediatrics Louisiana State University Health School of Medicine and Children’s Hospital New Orleans).

For 50 years, Dr. Vargas dedicated his life to medicine. In 2016, just months after his retirement at the age of 69, he became a patient himself when he received not his first but his second diagnosis of cancer, this time in the form of Merkel cell carcinoma (MCC). Through a set of serendipities, a groundbreaking clinical trial, and a strong system of support, Dr. Vargas took advantage of cutting-edge medicine to battle one of the rarest forms of cancer. 

Two Cancer Diagnoses, One Patient: Dr. Vargas’s Diagnoses With Lymphoma and Merkel Cell Carcinoma

In 2004, when Dr. Vargas was 56, he was diagnosed with diffuse large B-cell lymphoma, a type of blood cancer, and treated within the LCMC Health System. “I was tumor free by the end of the year and tumor cured five years later,” he explains. Tumor free means the tumors were gone, but tumor cured means there were no signs of cancers left and the cancer would not return. 

In September 2016, when he was 68, he experienced spontaneous nose bleeds. This led to testing, a referral to an oncologist at LSU Health New Orleans, and more testing. Just a month later, he was confirmed to have Merkel cell carcinoma, a rare form of skin cancer. MCC often grows at a rapid rate and spreads to other parts of the body quickly. 

Dr. Vargas’s cancer was considered stage IV because it had spread to distant parts of his skin, including his nostrils, left ear, and maxillary sinuses (a hollow space within the bones that surround the nose). 

At the time, MCC was a particularly scary diagnosis. “I was told it was more aggressive, more dangerous, and there was no treatment, no cure," he recalls. 

Serendipities and a Groundbreaking Clinical Trial for Merkel Cell Carcinoma

Merkel cell carcinoma only affects around 3,000 Americans per year. Yet, the serendipities that Dr. Vargas would encounter – and would lead him to enroll in a clinical trial at LSU LCMC Health Cancer – make it feel more prevalent. 

Years before Dr. Vargas’s diagnosis of MCC, in 2007, Al Copeland, Sr., New Orleanian and founder of Popeyes Famous Fried Chicken and Copeland’s Family of Restaurants, was also told he had this rare disease. Just months after his diagnosis, Al Copeland died, leaving one final wish to his family – to help find a cure for Merkel cell carcinoma. 

In honor of Al Copeland and his legacy, the Al Copeland Foundation was formed, leading to various initiatives surrounding cancer prevention, outreach, and clinical research and trials. In October 2016, LSU Health New Orleans and the Copelands announced the NCI clinical trial for advanced Merkel cell carcinoma – the very clinical trial that Dr. Vargas took part in. “By serendipity of life, I was the only patient of Louisiana admitted into the national study,” he says.

But this wasn’t Dr. Vargas’s first connection with the Copeland family. Years prior, he treated Al Copeland Jr.’s daughter. Now, in a way, the Copelands were providing care for Dr. Vargas. 

“Philanthropy played a very important role in LSU finalizing what they were already doing in developing these clinical trials,” explains Dr. Vargas. 

This clinical trial studied the effectiveness of an immunotherapy called pembrolizumab (commercially known as Keytruda) in treating MCC. Participants were to complete 35 cycles of pembrolizumab, and it was set to last about two years.

Dr. Vargas received his first treatment on November 2, 2016. “When I was receiving the first dose, the Copeland family was at my bedside,” he says. “They were not only very happy that the wishes of Al Copeland were being accomplished to some degree, but that the therapy was being started.”

Of course, this wasn’t Dr. Vargas’s only link to LSU LCMC Health Cancer Center, either. Not only had he recently retired from the LSU School of Medicine, he also previously worked with Dr. Augusto Ochoa, Deputy Director of the LSU LCMC Health Cancer Center and Chair of the Department of Interdisciplinary Oncology, during his time in Colombia.

In April 2018, after 18 months of the clinical trial, Dr. Vargas was declared tumor free. In November 2018, he completed the trial. Because he was given total tumor clearance for more than 6 months, he stopped pembrolizumab therapy. 

In October 2019, Dr. Vargas’s care team detected another tumor, and he restarted pembrolizumab therapy. By March 2020, Dr. Vargas was once again free of tumors. 

Clinical Trials: An Ongoing Contribution to Science 

Decades before his cancer diagnosis, Dr. Vargas committed his life to science. But in alignment with his serendipitous experiences, he continued to make advancing science a central part of his legacy through his participation in a clinical trial.  

“Being a doctor myself is part of my way of life, my philosophy, my personal beliefs,” he says. Faced with what was an incurable disease at the time, he wanted to do what he could to help others. “If I'm not going to continue with my life, I can contribute to the possibility of others benefitting from the experience that the clinical trial will give.” 

In 2018, the Food and Drug Administration (FDA) approved pembrolizumab to treat advanced Merkel cell carcinoma. During the clinical trials Dr. Vargas participated in, 64% of patients survived 3 years after their diagnosis – compared to only 10% of patients who received only chemotherapy prior to these trials. 

Clinical trials are often the spark for other discoveries, as well. “Many times, the clinical trial doesn't give you the light for the very problem you are studying, but collaterally you discover things that are beneficial for other kinds of disorders or medical disorders,” he explains. 

To date, pembrolizumab has been approved for treatment of more than a dozen kinds of cancer, some of which earned accelerated approval based on the success of patients.

A Path Forward in Science and Hope 

Dr. Vargas’s journey has not been without challenges. Despite many fortuitous moments, he faced what many people with cancer face – uncomfortable side effects, extensive disruptions to his life, and uncertainties about what comes next. He had to rely on his family and loved ones in more ways than one, and he had to put immense trust in his care team. He also requires ongoing monitoring, testing, and, when needed, treatment for tumors that have reappeared. 

Amidst it all, he has taken control of what he could, including participating in a clinical trial. From his career to his cancer diagnosis, Dr. Vargas has witnessed firsthand the impressive and swift progress of science. This time, it was an advancement that saved his life. 

CALL TO ACTION/COMMENT/SHARE: Do you have questions about clinical trials? Contact LSU LCMC Health Cancer Center to learn more about the benefits of clinical trials and how to join.