The first FDA-approved treatment using CRISPR may see hundreds of African Americans cured of sickle cell disease.
Making headlines without end for years, but only approved for trials, a CRISPR product is now finally on the shelves, so to speak, and this iteration of the treatment is called Casgevy and manufactured by Vertex Pharmaceuticals.
CRISPR edits the genetic code of the body’s own stem cells to treat conditions that arise from defective or mutated genes. In the case of sickle cell, Homo sapiens evolved this genetic change to protect themselves against the malaria parasite of the African continent.
However the disease that sometimes results from the sickle cell gene, which affects over 100,000 Americans, can cause debilitating pain and shortened lifespan.
“It’s been really remarkable how quickly we went from the actual discovery of CRISPR, the awarding of a Nobel Prize, and now actually seeing it being an approved product,” said Dr. Alexis Thompson, chief of the division of hematology at Children’s Hospital of Philadelphia, who has previously consulted for Vertex.
Until now, the only previous treatment for sickle cell disease was a bone marrow transplant from a donor which carried large risks such as rejection of the immune system. It’s also difficult to find a matching donor.
MORE CRISPR NEWS: FDA Clears First Study of CRISPR Gene-Editing in Human Patients
While classified as a one-time treatment, the process involves many individual appointments, which themselves are painful, but NBC spoke to one woman who feels as if she’s been given a second shot at life, and can now run and go to the gym; things she couldn’t do before.
“I’m just like a regular person. I wake up and do a 5K. I lift weights. If I wanted to swim, I can swim. I’m still trying to know how far I can stretch it, like what are all the things I can do,” said 29-year-old LaRae Morning.
MORE CRISPR NEWS: Aggressive Leukemia Disappears in 13-Year-old Girl Who was First to Receive New CRISPR Treatment
She would recommend the treatment to anyone who has the option, despite the painful administration process. Winning the Nobel Prize in chemistry in 2020, CRISPR is new, and state-of-the-art. What it isn’t, though, is cheap, and Casgevy costs $2.2 million.
Because it can help prevent years of medical care, NBC reports at least some of the cost will be covered by insurance.
SHARE This Groundbreaking Moment In The Next Stage Of Medicine…