In observance of World Sickle Cell Day, Rare Disease Report®
spoke with Beverly Francis-Gibson, president and CEO of Sickle Cell Disease Association of America (SCDAA), and Mark Diamond, vice president of commercialization of Emmaus Life Sciences, on their newly announced partnership in an effort to combat sickle cell disease.
The partnership between the nonprofit and the pharmaceutical company will aim to help create and facilitate a symbiotic relationship between patient advocacies, patients, and physicians.
Sickle cell disease is characterized by red blood cells that are hard, sticky and C-shaped (like the farm tool, a “sickle). The potentially fatal rare genetic condition affects an estimated 95,000 Americans – both adults and children. The misshapen sickle cells clog smaller blood vessels, result in excruciating pain, and put patients at an increased risk for infection acute chest syndrome and stroke.
“In spite of sickle cell disease, which is the number 1 inherited genetic disorder, having been around for a very long time, there is a tremendous lack of understanding of sickle cell disease in the community and among healthcare professionals,” said Diamond. “Patients need to be educated on how to best manage their disease and prevent sickle cell crisis from happening.”
Through Emmaus’ funding, Diamond went on to explain his hope of seeing appropriate developments made in educational materials to help inform the community. He also expressed his hopes for providing those educational materials in multiple languages in order for everyone to have the ability to understand the most pertinent information pertaining to sickle cell disease since a variety of nationalities are affected by the disease.
In addition, by providing online tools to build capacity and better assist the sickle cell community, the partnership will assist in strengthening the SCDAA’s community-based member organizations. Ensuring families have access to educational resources in order to acquire knowledge of the disease, giving needed support services, hosting summer camps, supporting research, and advocating on behalf of families for adequate treatment are some of the primary functions that will be assisted in SCDAA’s member organizations.
“This partnership is a wonderful opportunity for the sickle cell community to get technical support, more resources, information, and education on the disease in addition to clinical information and research that is happening to help them access new or developing drugs that can help them with their illness,” commented Francis-Gibson. “This is a huge opportunity that we think will have a wide impact across the country.”
Additionally, the partnership will assist in helping usher treatments and to encourage sickle cell patients to participate in clinical trials, which Francis-Gibson explained many sickle cell patients have had reservations on.
“The major challenge is many of the patients we work with are skeptical and hesitant of participating in clinical trials because of previous, historical things that have happened,” she said. “I have been hearing that many of our patients don’t want to be treated as guinea pigs in a laboratory, and they feel that, if companies want them to be involved in clinical trials, then they need to be involved in it way before the drug is being developed. They want to have more of a say in the process and understand the pros and cons of the clinical trials in a way that they can understand it.”
However, with Emmaus having produced Endari, the first sickle cell drug approved in nearly 20 years last year, Diamond believes more treatments for sickle cell disease are only further encouraged. He claimed with over half a dozen therapies currently in clinical development in various stages, the future looks bright.
“The other big issue with sickle cell disease is patient compliance. For some reason, it’s a very challenging group of patients to deal with. Very often, they don’t follow the doctor’s instructions, they don’t take their medications like they’re supposed to, and that’s when they run into trouble,” he said. “If a patient is feeling ok one day, they may think everything is fine, but then all of a sudden, they can hit with sickle cell crisis and they can get into trouble, wind up in the hospital, and spiral out of control.”
In close, Diamond stressed the importance of team work “The important thing is to prevent and avoid sickle cell crisis, and the way to do that is to make sure patients understand how to manage their disease and that they form partnerships with their physicians as well as local sickle cell advocacy so that they, we, work as a team to combat this disease.”