By Akira Kyles, Special to the AFRO
The National Institutes of Health (NIH) announced, Sept. 13, the launch of an initiative to speed up the development of a better cure for sickle cell disease.
NIH is the nation’s medical research agency that provides money to scientists to conduct studies to reduce illness and disabilities. The initiative is being led by NIH’s National Heart, Lung and Blood Institute (NHLBI).
Sickle cell is a disease caused by a single genetic mutation that causes a person’s red blood cells to form an abnormal, sickle shape. The sickled cells can clog the blood vessels and deprive cells of oxygen, according to NIH’s press release.
The National Institutes of Health (NIH) announced a new initiative to speed up the research in finding a cure to sickle cell led by NIH’s National Heart, Lung and Blood Institute (NHLBI).
“Our scientific investments have brought us to a point where we have many tools available to correct or compensate for the defective gene that causes sickle cell disease. We are now ready to use these tools to speed up our quest for a cure,” said Gary H. Gibbons, M.D., director of NIH’s NHLBI in a press release.
The current focuses of the initiative are gene therapies for sickle cell patients to create genetic-based cures. “That’s when you correct the gene that makes the sickle red cell but there are many other treatments,” said Traci Mondoro, Ph.D., chief of the Transitional Blood Science and Resources Branch. “So, the gene therapies, genetic-based cures for instance, wouldn’t be great for an older person who has sickle cell because they might not be able to tolerate the therapy. So, what this initiative is doing is trying to help scientists who are still developing things in the lab, that haven’t gotten into humans yet, move them further along.”
According to Morondo, the NIH has a five to 10-year goal for the initiative. “That doesn’t sound very fast, however, if people were doing this without the initiative, it would take them that long to raise the money or get the kind of team together that they would need to pay for studies and work with the FDA to move it forward,” said Mondoro. “So, we’re providing, not just money to people, but we’re providing expertise and other things like cells or chemicals that they might otherwise have to pay for to move this forward.”
Currently the only known cure for sickle cell is a bone marrow transplant, which is rare for patients to find a sibling match. A different cure would open a wider net for sickle cell patients. There is treatment such as hydroxyurea and pain medicine.
Hydroxyurea increases fetal hemoglobin, the protein within the red blood cells that people make as fetuses, in the body and decreases the stickiness of the red blood cells to the blood vessels.
“Fetal hemoglobin is protective, it decreases the sickling of the red blood cells, so they can move easily through the blood vessels,” said Courtney Fitzhugh, M.D., Lasker Clinical Research Scholar.
Due to the pain that sickle cell anemia causes, patients are given strong pain medication which can be hard for them to get. “Sickle cell is a public health crisis because a lot of people can’t get the pain medication that they need,” said Mondoro. “You have to take some pretty strong pain meds and sometimes their doctor or the emergency room doctor may tell them that they look fine and don’t need opioids or other strong pain medications. People with sickle cell aren’t addicted to drugs, they need them for pain control.”
With strides in medicine and research, the future for sickle cell patients looks optimistic. “I have been taking care of patients with sickle cell disease since 1997 and there has been so much excitement over the last few years.” said Fitzhugh. “There are a lot of new drugs that are being studied. This is a very good time, there’s a lot more research going to sickle cell disease. So, there’s a lot of hope for the future for patients that have sickle cell disease.”