This medical video focuses on the new drug to help those who suffer from sickle cell disease.
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Jennifer Matthews: Gloria Gilliam's home is filled with love -- and her nieces and nephews. Gloria Gilliam: They gave me a reason not to go to the hospital, a reason to stay home. Jennifer Matthews: Gloria has sickle cell disease. At times, the pain can be unbearable. Gloria Gilliam: Honest to God, it felt like hot oil being poured down through my blood vessels. Jennifer Matthews: Despite all her medication, the pain still comes. Jennifer Matthews: Molecular biologist Joe DeSimone, hopes a new drug will help. It turns on genes that have been turned off. The goal is to replace the abnormal adult hemoglobin, which causes the red blood cells to change shape, with fetal hemoglobin, which turns off after birth. Dr. Joe DeSimone: There are people naturally occurring in the population that have high expression of fetal hemoglobin that have very, very mild disease. Jennifer Matthews: DeSimone treated patients with infusions of the cancer drug decitabine and saw success. Dr. Joe DeSimone: Even the patients that did not respond to hydroxyurea, the gold standard used in the practice of treating patients, all of those have responded. Jennifer Matthews: For Gloria, a life without pain would mean independence for the first time. Gloria Gilliam: It would be very difficult to wake up in the middle of the night in that kind of pain and not have another soul in the house. Without sickle cell disease, I wouldn't have to worry about that. Jennifer Matthews: When she jokes she could send the kids to their home when she is had enough. This is Jennifer Matthews reporting.