yw YN . (NAPSA)—In 2000, a mysterious CURE SICKLE CELL. disease was making 3-year-old Mercy Mendozasoill that her grandmother bought a burial plot for her in thelittle town in Honduras where they lived. tors] didn't know much,sothey treated Anup[his brother] the way they treated me, andit was fatal” said Dasu, whose parents eventually brought him to the United States for treatmentandcare. Around the same time in Des Moines, Iowa, Cassandra Trimnell became the first child diagnosed with sickle cell disease through that state’s a Ths tne has come. operwedisease. cance seilcal Swelling, pain and immobility were fast erodingher health.Finally, a doctor who had beentrained in the United States recognized her condition: It wassickle cell disease, an inherited blooddisorder. ‘Thirteen years earlier in India, then 4-month-old Kirti Dasu was diagnosed with the same condition, as eventually washis brother. But while Kirti, now 30, survived, his brotherdid not. “[The doc- = p n> ‘#OuringSCD_ About1 in every 365 African American babiesis born with sickle cell disease. @ Although about one in every 365 African-American babies is born with sickle cell disease. perhaps an ancestor who lived some 7,300 years ago in whatis now the Saha- ran Desert. The child was born with a genetic mutation that has endured over 250 generations because it protects against malaria, a majorkiller, then as now. It also causes deformed,sickle-shaped red bloodcells. A child developssickle-cell disease if he or she inherits two faulty copiesofthe gene, one from each parent. ..there can be a cure. The time has come. “Throughthis initiative, researchers will be taking advantage of the growing number of tools they now have to correct or compensate for the defective gene that causes this pain- newly implemented newbornscreening others with the condition know, the ful disease,” said Keith Hoots, M.D., director of NHLBIDivision of Blood most others in the African-American community, she didn’t knowshe had the sickle cell trait until her beautiful baby can clog the bloodvessels and deprive cells of oxygen. In turn, this lack of oxygen wreaks havoc on the body, dam- added, is to find more than one cure because patients responddifferently, depending on their ages and other worked hard andgot the care Cassandra needed. potentially leading to premature death. “Fifteen minutes from now, I could transplants—the only cure for sickle cell disease to date—tend to be more successfulin children, but only a few patients can find suitable donors. program. Her mom wasstunned.“Like was born,’ said Trimnell, 31, but she As Mendoza, Dasu, Trimnell and @ results can beserious. Thesickled cells aging organs, causing severe pain, and ‘The experiences of Mendoza, Dasu havea crisis with chestpain or be unable and Trimnell—oncontinents thousands of miles apart—turned outto belife changing:Each is now fierce advocate for sickle cell disease awareness and education. Their stories speak to the complex journeyofthe sicklecelltrait. They also underscore a little-known truth—thatsickle cell disease, the most commoninherited blood disorder, has a far-reaching impact on a surprisingly diverse swathof the global population. The rare condition affects at least 100,000 people in the United States— mainly African Americans, but also Hispanics and Asians—and 20 million worldwide. They share not only a diag- nosis but, according to new research, to breathe,’ said Mendoza. “There are timesI have to go to the hospital, and I don’t think I'm going to make it” ‘The National Institutes of Health (NIH)spends some $100 million annu- ally onsicklecelldisease research, which hasled to pain-reducingtreatments such as hydroxyureaandalsolaid the ground- work for genetic approachesto potential cures,such asthe genetic editing of bone marrowcells. To speed up this genetic research, NIH’s National Heart, Lung, and Blood Institute (NHLBI) in Septem- berlaunchedthe Cure Sickle CellInitiative, an effort designed to movethe most promising of the genetic therapies into clinicaltrials within five to 10 years. Diseases and Resources. Thegoal, he factors. For example, bone marrow “There may neverbejust one universal cure,” hesaid. ‘Theinitiative spells hope for Men- doza, Trimnell and millions of people living with the condition. Andit gives Dasu another reason to continue advocating for new discoveries that help people like him. In 2017, Dasu received a bone marrowtransplant and he has madeanastonishingrecovery. “Tm good!”said Dasu.“Now,for the first time, I mean it when I say ‘good’ Notjust ‘not too muchpain; but ‘good” His hope,hesaid, is that new therapies will makeit possible for others to say the same.
