New Advances in Sickle-Cell Disease Treatment

About 1 in every 12 African Americans carries the gene mutation that causes sickle-cell disease, which ultimately affects 1 in 500 people of African descent. Sickle-cell disease can cause serious disability, and even death, in sufferers of nearly all ages. Treatments have helped to reduce the impacts of this potentially deadly condition, but so far, all efforts have worked merely to slow it down, dampening symptoms at best. But exciting new advances may have uncovered keys to turning the tables and eradicating symptoms for good.

What is Sickle-Cell Disease?

Sickle-cell disease (SDC) occurs due to a mutation that alters red blood cell shape. While it primarily affects people of African descent, Medline reports that this condition also occasionally appears in Asian, Indian, Middle Eastern and southern European families.

The trait is most common in areas where malaria is a problem, and it may even have initially developed as a faulty evolutionary strategy against the blood parasite. While people with SCD have some protection against malaria due to the malarial-inhospitable shape of their red blood cells, the difference can cause some serious health issues so it's not actually an advantage but rather a serious health condition that needs to be managed.

People with SCD commonly suffer from anemia, mainly because their red blood cells die much faster than normal ones. Their bodies can only make so many new cells to replace those lost, causing serious shortages in some cases. The resulting deficit may lead to fatigue. SCD can also cause deadly blood clots because the abnormally shaped red blood cells are more likely to tangle together and stick to blood vessel walls. Complications can include painful swelling, organ damage and sometimes even death. 

Innovative Treatment Strategies

Current treatments, which include antibiotics, pain relievers and blood transfusions, can only offset the effects. Many people also rely on a medication called hydroxyurea, which tricks the body into producing more of a different, more effective, type of red blood cell it usually stops making in infancy. However, this treatment is only helpful for some.

Good News in Sickle-Cell Disease Research

The National Institutes of Health (NIH) recently released news of two strategies that could change the face of SCD treatment for good. The first builds off of the benefits of hydroxyurea treatment, with researchers looking for other medications that are even more effective on that front; they’re also looking at ways to improve the consistency with which people take these medications, as SCD can cause memory problems that may affect medication adherence.

Another promising direction researchers are working in is gene therapy. This uses the patient’s own stem cells, which researchers alter genetically and implant back into the bone marrow. Previous attempts at using stem cell therapy have proven too risky to use on adults, but the process was also previously difficult to perform on children due to issues with donor matching and improvements are happening rapidly. Developments could make donors into recipients and that could fix some of these problems.

The NIH (National Institutes of Health) is currently recruiting volunteers with SCD for study in just this area. People who are interested can go to www.clinicaltrials.gov to sign up or check out further details.

Sickle-cell disease continues to affect far too many people despite current treatments, but researchers are working hard to find better options and some of these new ideas look promising. Soon we may see a change in the way doctors treat the condition for good. Hopefully, more reliable treatment strategies will save lives and allow more sufferers to lead normal, functional lives without fatigue, memory problems, and many of the other challenges faced by those with this condition. We'll keep you updated as we hear more.

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