Sickle cell anemia disease blood cells; 3D illustration
September was Sickle Cell Awareness Month, designated by the U.S. Congress to increase awareness about sickle cell disease (SCD) and help boost research and development targeting the disease.
Among the companies involved in SCD research is Theris Medical. The company has launched clinical trials for an investigational orphan product, the deferoxamine intradermal drug patch, which it is developing with the University of Alabama at Birmingham. The therapy is currently in a Phase 1/2 trial.
The patch is intended to improve the quality of life for those suffering from sickle cell leg ulcers. These ulcers are a rare – incidence of between two and 7% of patients - but painful and debilitating complication of sickle cell disease.
If approved by the Food and Drug Administration (FDA) the topical patch will deliver an already existing drug – deferoxamine - directly to treat sickle cell patients’ ulcers.
The deferoxamine patch offers a potentially significant improvement in the treatment of sickle cell patients patients with ulcers, but it faces a number of logistical and access challenges, some of which are exacerbated by the ongoing Covid-19 pandemic.
SCD is a group of congenital red blood cell disorders, named sickle cell for the crescent shape of red blood cells. SCD affects millions of people worldwide. In the U.S., at least 100,000 individuals are living with the disease, which predominantly impacts people of sub-Saharan African descent.
The disease alters the structure of hemoglobin, the molecule in red blood cells that delivers oxygen to organs and tissue throughout the body. The most common SCD disorder type is sickle cell anemia. The sickle-shaped cells stick to blood vessel walls, causing blockages - called vaso-occlusion - that impede the proper flow of blood and therefore oxygen throughout the body. Severe pain crises often result. Besides premature mortality, SCD causes significant morbidity, including severe pain, anemia, organ damage, infections, and ulcers.
I spoke with Dr. Julie Kanter, director of the University of Alabama at Birmingham’s Adult Sickle Cell Program and co-director of the school’s Lifespan Comprehensive Sickle Cell Center. Kanter has been involved in the clinical trial of the deferoxamine patch.
Kanter explained that many sickle cell leg ulcer patients don’t have access to transportation to the medical treatment centers where the patch is being developed. In coordination with the University of Alabama, Theris Medical is providing car services to transport patients participating in clinical trials to their appointments.
But, if the product is approved by the FDA, transportation will still be an issue in the clinical care setting, as weekly follow-up and monitoring will be necessary for a period of time even though the patch can be self-administered.
Furthermore, Kanter pointed out that nationwide there aren’t enough hematologists who can treat sickle cell disease. Additionally, currently there aren’t any accredited centers specifically designed to care for sickle cell disease patients.
Like some orphan and most neglected diseases, historically, research and development targeting SCD has been relatively underfunded. This is not necessarily due to comparatively small numbers of patients. In fact, drug companies have devoted considerably more resources to research and development of novel drugs in certain other orphan diseases, such as cystic fibrosis - which affects fewer than 30,000 in the U.S. - than SCD.
Though long overlooked, the situation appears to be changing with respect to the life-threatening hematological disease. Compared to just a few years ago, there is presently more awareness of, and attention paid to SCD as a substantial global health issue. As a sign of positive change, for example, the federal government and the Bill & Melinda Gates Foundation recently announced they would each contribute $100 million to provide effective SCD therapy in the developing world.
Yet, for sustained improvement in SCD care, challenges regarding development of therapies, access to new products, and ensuring adequately resourced and staffed care facilities will have to be overcome.
The health disparities laid bare by the Covid-19 pandemic aren’t new. Prior to the Covid-19 outbreak, African-Americans were already considered the least healthy ethnic group in the U.S. This is partly due to Black patients being more likely to receive healthcare at under-resourced safety-net hospitals, or to be on Medicaid.
African Americans who suffer from SCD face the double whammy of resource constraints and unique challenges associated with treating the disease, which include logistical hurdles such as transportation and insurance coverage. Designing equitable access pathways to treatments like the deferoxamine patch may contribute to closing the gaps in health outcomes.
