The regional leader in groundbreaking pediatric stem cell transplant and cell therapies, Manning Family Children’s, is making strides to eliminate illnesses like sickle cell anemia for good. Dr. Benjamin K. Watkins leads as director of the program, the only one of its kind in the Gulf South region, working to offer life-changing treatments and the specialized infrastructure to change the lives of Louisiana residents whose conditions might otherwise be ignored or under-treated.
Dr. Watkins, a nationally recognized expert in Pediatric Hematology Oncology with particular expertise in stem cell transplantation and cellular therapies, completed his medical degree and pediatric residency from the University of Arkansas for Medical Sciences (UAMS). He then completed a pediatric hematology/oncology fellowship at Emory University School of Medicine and a blood and marrow transplant fellowship at Seattle Children’s Hospital and the Seattle Cancer Care Alliance. Now, through his work at Manning Family Children’s, Dr. Watkins is focused on addressing the systemic disparities inherent to sickle cell disease and offering hope for a cure for this devastating disease.
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“Sickle cell disease is the most common inherited blood disorder in the country, affecting approximately three thousand Louisiana residents alone, one of the highest in the country”, says Dr. Watkins. “This disease affects every organ of the body, with patients suffering from chronic pain, strokes, heart failure, and shortened life span. Yet, despite the breadth of this suffering, a chronic lack of research has led to few advancements in treatment. Sickle cell disease almost universally affects Black and African American patients, and this disease has been stigmatized and lived in the shadows for far too long.”
For comparison, research into cystic fibrosis, a condition that predominantly affects white patients, has received almost thirty times the amount of funding as sickle cell, despite only affecting a third of the patients nationwide. Other hindrances to care include proximity to accredited facilities, with rural areas often unable to receive adequate preventative care through regular screenings to help prevent life-threatening complications. New cutting-edge therapies have recently become available and are beginning to shine a light on this disease. Through stem cell transplant and the advent of innovative gene therapies being offered by facilities like Manning Family Children’s, with FDA approval finalized in December of 2023, Dr. Watkins hopes to greatly increase the ability for patients to receive treatment through focused attention and targeted innovation.
“These therapies are only available to patients with the most aggressive forms of the disease, as they require intense chemotherapy for success,” explains Dr. Watkins. “Stem cell transplant has been developed slowly over the course of nearly seventy-five years and has proven to be a cure for sickle cell disease, with outcomes improving every year. Gene therapy has also just recently emerged as an option to treat this disease. By collecting stem cells from a patient and adding or editing a gene to eliminate the sickling of cells before returning it to the patient, we are able to offer them a new lease on life. While these advanced treatments may only be offered to the most severely affected people, we hope that the spotlight that is now being shown on these therapies will shed light on all people with sickle cell and result in improved care for all.”
In March of 2024, Manning Family Children’s became the only authorized treatment facility for two different gene therapies for sickle cell in Louisiana. Now, Dr. Watkins and his team are scheduled to begin collecting stem cells from their first gene therapy patient in the coming weeks, taking a massive step in the equitable treatment of sickle cell disease and giving hope for a cure to a disease that has often left people feeling hopeless.
