The Sickle Cell Cure Foundation, Inc. (SCCF) is a non-profit 501(c)(3) research foundation incorporated in 2006. SCCF chose the PHF Research Park as its world headquarters to conduct translational research using patented treatments aimed at curing two of mankind’s most devastating diseases: malaria and its allied blood disorder, sickle cell disease (SCD). The Bill & Melinda Gates Foundation recently awarded a Grand Challenge Exploration grant to SCCF to conduct its animal safety and efficacy trials. SCCF aims to raise additional funds for human clinical trials required for FDA approval. The therapeutics offered by SCCF are inexpensive, non-invasive, and easy to deliver; and SCCF’s mission fits the Gates’ central philanthropic principle that “all lives have equal value”.
SCD is a disease that co-evolved with malaria among peoples living in equatorial regions where mosquitoes are endemic and serve as vectors for the malaria parasite. In the US, over 70,000 individuals are afflicted with SCD. Globally, an estimated 880,000 die from malaria and 340,000 from SCD annually, with the great majoriy of these deaths being children under the age of five years. SCD afflicts many in western and central African countries, as well as people of Portuguese, Spanish, French Corsican, Sardinian, Sicilian, Italian, Greek, Turkish, Cypriot, Israeli, Arab, and Indian decent. While one copy of the sickle gene confers resistance to the malaria parasite, carrying two copies can result in death. Large public health measures have limited the spread of malaria in developed countries. In contrast, SCD has been neglected. The wide spread occurrence of SCD and recent resurgence of malaria motivated the World Health Organization in May 2006 to list SCD as a neglected disease warranting intensified research. SCCF is mounting a global effort to address this global health problem.
Gene Regulation Therapy (GRT) is the overall research approach employed by SCCF. GRT does not destroy genes, alter them, nor implant them in the patient. Rather, SCCF has discovered how to manipulate “toggle switches” that reactivate the body’s own fetal hemoglobin production. Medical literature has amply documented that the presence of fetal hemoglobin in adult red blood cells at concentrations of 30% or more confers a natural resistance to SCD…and also to malaria. Requiring no refrigeration, the GRT for SCD is natural, non-invasive, and reversible. To maintain its benefits, the patient only requires periodic “recharge” doses to keep the toggle switch “on.” Similar GRT-responsive, “onoff” genetic relationships are the subject of translational research and development. GRT promises effective clinical treatments for many debilitating disorders including diabetes, Parkinson’s, Huntington’s, liver diseases, and certain cancers. SCCF’s business plan for global distribution of Gene Regulation Therapy for SCD estimates a 95% drop in the cost of standard Western treatments. Whereas current treatments only address the symptoms of SCD, SCCF’s approach corrects the primary cause. For the world’s poor majority, SCCF’s business model uses cross-subsidy pricing to deliver the cure to Africa pharmaceutical outlets for as little as US $12.00 per person per year.