The NY Times recently reported that a possible cure for sickle cell disease is currently undergoing drug trials. The disease is genetic, meaning it is an error in the DNA code that causes red blood shells to assume a sickle shape, and the patient may experience blood clots, strokes and painful episodes due to the genetic variant. It is most common in African based populations.
To date, the only cure was a risky and expensive bone marrow transplant, however, a new gene replacement treatment is showing signs it can actually cure the disease. While this cure may be expensive, it is a cure, not a treatment.
There are many genetic variants that can cause diseases. Many of these variants are a result of a gene error that is common in certain populations.
This new cure is encouraging because if their next trial is a success, it may prove that the disease is not only curable, but that the technology behind it can be used to cure other genetic diseases.
Check out this article from the NY Times
These Patients Had Sickle-Cell Disease. Experimental Therapies Might Have Cured Them.
Success against sickle-cell would be “the first genetic cure of a common genetic disease” and could free tens of thousands of Americans from agonizing pain.
By Gina Kolata Jan. 27, 2019
Scientists have long known what causes sickle-cell disease and its devastating effects: a single mutation in one errant gene. But for decades, there has been only modest progress against an inherited condition that mainly afflicts people of African descent.
With advances in gene therapy, that is quickly changing — so much so that scientists have begun to talk of a cure.
In a half-dozen clinical trials planned or underway, researchers are testing strategies for correcting the problem at the genetic level. Already a handful of the enrolled patients, who have endured an illness that causes excruciating bouts of pain, strokes and early death, no longer show signs of the disease.
Among them is Brandon Williams, 21, who lives with his mother in Chicago. Because of his sickle-cell disease, he had suffered four strokes by age 18. The damage makes it hard for him to speak. His older sister died of the disease.