Sickle cell disease affects mainly people with an African background but it’s a genetic disease that can be passed down to anyone, regardless of race, color, gender, or socioeconomic background. Experimental therapies may have found a cure to a debilitating disease.
According to the U.S. National Library of Medicine, sickle cell disease affects nearly 1 in 13 African Americans and 1 in 365 Africans.
Those affected will have to have inherited it from both parents and will experience painful swelling of the hands and feet, have a yellow-ish undertone on their skin, and have fatigue from anemia.
Anyone with sickle cell will face serious complications – everything from infections to organ damage. And the only cure was bone marrow and stem cell transplantation. But those methods usually have such terrible side effects, they are only reserved for the most extreme cases.
But now, a cure may be in sight.
A handful of patients who have suffered with this disease for years no longer show signs of it. According to a New York Times article, it will be at least three years before a cure could be ready for the public. The idea that a genetic disease could be cured in our lifetime used to be a dream.
This new therapy could save thousands of lives and improve hundreds of thousands. It also opens up the gateway to curing other genetic diseases that have destroyed people’s lives.
The annual cost of having this disease is up to $10,000 for children and up to $30,000 for adults. Most people find it hard to hold down jobs because the disease is extremely painful at times and causes strokes, anemia, and an early death.
Sickle cell disease is still a mystery to many.
Those with only one gene of the mutation inherited from one parent has resistance to malaria that their counterparts, sick or healthy, don’t have. Early in life, the people with the resistance to malaria have a serious genetic advantage, especially in malaria-ridden countries.
When gene therapy became a viable cure, the first disease scientists looked at was sickle cell because everyone affected had the same mutation and it was only one gene that needed fixing. But it’s easier said than done.
Scientists had to figure out a way to affect only the red blood cells that were changed because of the gene and at specific times.
The new trials require the patients to have their stem cells removed from their bone marrow, the scientists genetically modify them, and then they are infused back into their bloodstreams. The hope is that they will create healthy red blood cells in the patients’ bone marrow.
Scientists are trying three different methods to cure sickle cell.
In the first method, they are attempting to introduce a virus that carries a viable replica of a healthy hemoglobin gene into the patient’s body. The virus will then transfer it into the patient’s own stem cells.
Scientists tried this method before, but it was only recently that they found viruses big enough to hold the gene but small enough to be effective in a patient’s body.
The second method looks at different types of hemoglobin and how to use them to our advantage. There is “fetal hemoglobin” which we have when we’re fetuses but are then shot off in place for “adult hemoglobin” after we are born.
Scientists are looking for the gene that turns off the fetal hemoglobin and turns on the adult hemoglobin so they can use fetal hemoglobin to fill in the gaps that are affected by sickle cell. Researchers and scientists believe that fetal hemoglobin not only works just as well as adult hemoglobin but are also not affected by sickle cell disease.
The third strategy is gene editing using the crispr method. Cripsr is almost like a copy/paste tool but for genes. The idea behind this is simply editing the sickle cell gene and completely getting rid of it.
Regardless of what method is tried and proven, it could mean an improved quality of life for hundreds of thousands of people. Nobody should have to live with this kind of disease – a debilitating illness that attacks people medically, financially, and psychologically. Dealing with illness takes a mental toll on them and their families.
There have been multiple success stories and we can expect sickle cell disease to be a thing of the past in just a few short years.
Also published on Medium.