Recent findings about sickle cell disease show a lower concentration of drugs could be effective for treatment, according to University of Minnesota researchers.
Research released by the University of Minnesota’s Department of Biomedical Engineering on March 13 found the molecular development of sickle cell disease is significantly less efficient than previously thought. The team said this development means a lower concentration of drugs would treat the disease.
Sickle cell disease is a group of genetic and chronic blood disorders that mutates blood cells causing the blockage of blood vessels. The process begins with hemoglobin, a protein that carries oxygen throughout the body, bonding to itself and forming rods that result in the misshaping and stiffening of the cell.
Previously, treatment was thought to need a one-to-one ratio of medication to hemoglobin — an unrealistic dose, due to the toxicity of the drugs.
Researchers found the molecular origin of sickle cell disease occurs at a fast, yet inefficient rate.
“It would be analogous to saying: if I stacked up 100 Legos … 96 of them would fall off and only four would stay on,” said University biophysics researcher David Wood. “I would have to stack up another 100 to get another four to stay. So [the molecular process is] only about 4 percent efficient.”
Wood said the immediate impact of their research may lead to the development of new drugs. The first case of sickle cell disease was reported in 1910, yet only two FDA-approved drugs are currently available, Stephen Nelson, a physician with Children’s Minnesota, wrote over email.
“[Sickle cell disease patients] have chronic tissue damage and frequent infections because their spleens and their immune system don't work properly,” Wood said. “Some of these people are in the hospital ... several times a week with really severe pain crises."
Sickle cell patients may be undertreated for their pain by doctors, who could be reluctant to prescribe opioids due to the opioid crisis, according to Nelson.
“There is a misconception that sickle cell patients are drug-seeking and that they may be ill-equipped to appropriately manage opioids,” Nelson wrote via email.
Wood said their research used the highest resolution measurements ever performed on the molecular processes of sickle cell disease to track the speed and efficiency of the cells.
“The technology of the cameras and microscopes that we're using is a lot better, which allowed us to collect higher quality data,” said researcher Brian Castle.
Wood said he hopes their research provokes other scientists to pay attention to the molecular process of diseases, which has been typically ignored in previous research.
Although there are other options for sickle cell treatment, such as blood transfusions, gene therapy and stem cell transplants, they may not be accessible to the largest population of sickle cell patients.
“The bulk of the people who have sickle cell disease live in Sub-Saharan Africa; there's like 12 million people in Sub-Saharan Africa, and they're not going to be getting gene therapy,” Wood said. “I would argue that … a medication that they can take twice a day and that would basically prevent most of their symptoms might be the right answer.”