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Page 8 The Skanner September 20, 2017 News NHLBI: Exploring Research Directions in Hope of a Widely Available Cure for Sickle Cell Disease pain management, and improve bone marrow transplant procedures to increase the body’s ac- ceptance of donor stem cells. One promising strat- egy that is being stud- ied in a clinical trial – a study that tests how well the United States don’t have a relative who is a full genetic match and able to be a donor. The National Heart, Lung, and Blood Insti- tute (NHLBI), a leader of federally funded re- search efforts on sickle cell disease, is actively “ Bone marrow transplants can eliminate sickle cell dis- ease, ridding them of pain- ful and debilitating symp- toms, and the need for a lifetime of pain medications and blood transfusions PHOTO COURTESY OF ED UTHMAN VIA FLICKR exploring several safe and effective treatment options, and leading sev- eral initiatives designed Peripheral blood smear of sickle cell disease cells that do not sickle as do the cells in those with sickle cell disease—the characteristic that gives the disease its name. Un- fortunately, many people with sickle cell disease in to find a widely avail- able cure for sickle cell disease. The institute supports many research efforts that yield new therapies, optimize cur- rent treatments, enhance a new medical approach works in people – is half-match bone marrow transplants. Bone mar- row transplants can eliminate sickle cell dis- ease, ridding them of painful and debilitating symptoms, and the need for a lifetime of pain medications and blood transfusions. This half- match approach could make bone marrow transplants accessible to more people living with sickle cell disease. The NHLBI also com- pleted the Transcranial Doppler with Transfu- sions Changing to Hy- droxyurea (TWiTCH) trial. Based on earlier research that was fund- ed by NHLBI, regular blood transfusions have become the standard of care for reducing the risk DIANA GRIB VIA WIKICOMMONS S eptember is not just “ b a c k - t o - s c h o ol ” month; it’s National Sickle Cell Aware- ness Month—a month selected to call attention to a life-long illness af- fecting around 100,000 Americans, the majori- ty of whom are African American or Hispanic. Sickle cell disease is accompanied by severe pain attacks and poor ox- ygen delivery through- out the body that can cause critical damage to organs. Currently, the only cure is hematopoi- etic stem cell transplan- tation (HSCT)—a costly procedure in which stem cells are taken from the bone marrow or blood of a healthy donor, and then injected into the recipi- ent to generate red blood of stroke in children with sickle cell disease. The TWiTCH study found that daily treatment with hydroxyurea— an oral medicine to help reduce or prevent several com- plications of sickle cell disease – is as effective as blood transfusions at reducing blood flow velocities in the brain, a key risk factor for stroke. NHLBI is committed to additional studies that can prevent strokes and other sickle cell-related complications. Another area of prom- ise for sickle cell dis- ease treatment is gene editing, which involves changing the DNA. An NHLBI research group recently showed that correcting the sickle mu- tation in hematopoietic A career you can be proud of. stem cells from patients with sickle cell disease, and then transplanting them in mice, resulted in enough normal hemo- globin to have a potential benefit. Another recent study in a patient showed that replacing the sickle mutation by gene inser- tion (gene therapy) re- sulted in complete clin- ical remission of sickle cell disease. However, longer follow-up in more patients is required to confirm the long-term safety and effectiveness of gene therapy for sickle cell disease. NHLBI is also conduct- ing early research using small molecule drugs. In one recent NHLBI-fund- ed study, researchers found a small molecule that binds to hemoglobin and increases its ability to bind to oxygen; this could reduce sickling of red blood cells. Since small molecules can be easily administered, the hope is that this ap- proach could become a cost-effective, widely available treatment for sickle cell disease both in developed and devel- oping countries. Howev- er, this approach has not been tested in humans yet. Over the next decade, NHLBI is committed to conducting and funding innovative research on sickle cell disease. Sickle cell patients who partic- ipate in research studies are critical partners in discovering potential therapies and new ap- proaches that improve our understanding of sickle cell disease. Al- though new preventive and treatment strategies might take years to devel- op, patients should take heart that today there are effective treatments that can help reduce symp- toms and prolong life. To find out more about how you or a loved one can participate in a clini- cal trial, go to clinicaltri- als.gov and search sickle cell disease. Make The Skanner part of your daily routine Being a carpenter isn’t just a job. 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