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.

Gene Regulation Therapy

We have named our approach to curing sickle cell disease Gene Regulation Therapy, and the result is called a phenotypic cure. That is to say, no genes are permanently altered; instead, we use normal bodily signals to turn off the sickle cell gene and activate another gene with the same function in its place. We have discovered a naturally occurring protein, a protein that is grown in our bodies that will shut off the sickle cell gene. This same protein will also turn on a fetal hemoglobin gene to replace the silenced sickle hemoglobin. There are a few people in the world in which this substitution of fetal hemoglobin for sickle hemoglobin occurs naturally, and these people never have the devastating effects of sickle cell disease or any other physical problems. Our recent experiments with blood from sickle cell patients have shown that this approach will work as predicted. The treatment should be easy to deliver, as a protein directly into the blood or marrow, where red cell precursors have receptors on their surface that recognize this protein and internalize it. We have also discovered a plant compound that will activate expression of this protein in human cells, and the plant compound will be tested for both safety and effectiveness in humans with sickle cell or beta-thalassemia.

General purpose:

Education and scientific research.

Specific purpose:

The purpose of the SCCF is to bring the cure that we have discovered to clinics around the world. For this goal to be accomplished costs associated with completing the patent process, validating the cure clinically, and performing safety trials in animals and humans must be paid. We seek donors as partners in this endeavor.