Augusta, GA (WJBF)—Happening now at a local hospital—a study that is the first of its kind, designed to cure a deadly disease.
The Medical College of Georgia and Augusta University Health are part of a national trial studying if bone marrow transplants should be given to all patients suffering from Sickle Cell Disease. MCG is one of about 35 centers across the country enrolling people ages 15 to 40 in the program. The goal is to cure patients suffering from Sickle Cell Disease and allow them to have a longer life expectancy.
Sickle Cell is an inherited disease affecting roughly 70,000 people in the United States, typically those of African or Mediterranean descent. The bone marrow in Sickle Cell patients produces red blood cells that carry oxygen poorly. The cells have an odd, sickle shape that bang into blood vessel walls injuring them. They also pile up blocking blood flow and eventually, this destroys major organs and bones.
Dr. Jeremy Mark A. Pantin is a principal investigator for the study. He explains this trial is for sicker patients. “We want to be able to offer the option of cure to our patients who struggle the most with this chronic disease,” says Dr. Pantin.
For the transplant, doctors use chemotherapy to wipe out the patient’s bone marrow and replace it with a donor’s bone marrow. Siblings tend to be the first donor choice because there is the best chance for a successful transfer. After the transplant, the patient stays in the hospital for at least 2 weeks while the new blood cells multiply and the immune system gears back up. Afterwards, doctors will monitor the group for 2 years to see what percentage stays “disease free.”
Dr. Pantin says the goal of this study is to allow patients to shed the complications from the Sickle Cell Disease. “You want to take away patients’ suffering and help them have a longer and healthier life,” describes Dr. Pantin.
To be eligible, they must have experienced at least one of the disease’s most serious consequences. These include a stroke; at least 2 episodes of acute chest syndrome in the last 2 years; or 3 or more pain crises per year in the last 2 years that required medical attention. Exclusionary criteria include a history of substance abuse, pregnancy or breastfeeding and uncontrolled infection in the six weeks before enrollment.
Dr. Pantin describes that the people, to whom this study is geared, are typically prone to higher risks from this kind of procedure. “You have to balance the patients’ complications through the course of their illness and the reality that patients generally have a reduced life expectancy, with the promise of being cured of the disease, but not without some risk,” Dr. Pantin says.
However, a transplant can effectively cure a disease that can result in regular hospitalization, an increasing reliance on powerful pain medication, an increased stroke risk, particularly for children, and an average life expectancy in the mid-40s.
Additionally, there are serious financial benefits from ditching the disease. Dr. Pantin says, 5 years of medical treatment costs roughly $1.2 million for a person suffering from Sickle Cell.
MCG has screened 6 people and enrolled 1. Dr. Pantin says the woman enrolled will be the first person to receive this kind of transplant in Augusta. Her sister was her bone marrow match.
The goal for the study is to enroll 200 patients across the country.