FDA Approves World’s First Gene-Editing Drug — Despite Side Effects

For the first time in history, there’s hope that the debilitating illness known as sickle cell disease may be treatable — even cured.

The Food and Drug Administration announced Friday that two gene therapies for sickle cell disease have been approved following years of clinical trials.

Sickle cell disease is an inherited blood disorder caused by a mutated gene that affects hemoglobin, a protein in red blood cells, according to Johns Hopkins Medicine.

Because those crescent or sickle-shaped blood cells can’t effectively carry oxygen through the body, people with sickle cell disease face a lifetime of organ failure, anemia, strokes and excruciating pain.

Victoria Gray, 38, was diagnosed with sickle cell disease as a young child. She tried to learn everything she could about the disorder in grade school.

“It left me in a lonely place,” Gray told USA Today. “All I learned was I was doomed to die.” Indeed, most people with sickle cell disease die by their early 50s.

After countless hospital stays and prescriptions for powerful pain medications, by the time she was 32, Gray told her doctor that she was too exhausted to go on. “I told him I couldn’t live like this anymore.”

But advances in genetic research over many years now offer some hope to the people who have been living with sickle cell disease, most of whom are Black.

One gene therapy, developed using CRISPR, a Nobel Prize-winning gene editing tool, is known as Casgevy and was developed in a partnership between Vertex Pharmaceuticals and CRISPR Therapeutics.

The other treatment was created by Bluebird Bio of Somerville, Mass. Known as Lyfgenia, it uses a gene therapy technique to add a healthy hemoglobin gene to patients’ DNA.

Both of the treatments involve lengthy procedures and high doses of chemotherapy, potent enough to cause infertility.

The Vertex therapy, for example, requires patients to have stem cells harvested from their bone marrow. The cells are then sent to a facility where they are edited using CRISPR technology. Then, the newly-edited bone marrow cells are infused back into the patient’s body during a months-long hospital stay.

Despite the high-tech medical miracles promised by gene therapy, getting access to these treatments is almost as arduous as the disease itself.

There are only a handful of US medical centers authorized to provide the treatments. Each patient’s cells must be individually edited. The chemotherapy and other procedures are so onerous that not everyone can tolerate them.

Moreover, the treatment comes with a multimillion-dollar price tag and potential insurance-approval nightmares, The New York Times reports.

But living with the disease is also hugely expensive: People with sickle cell disease can cost $1.7 million with commercial insurance over a patient’s lifetime, and those with the illness can expect to pay roughly $44,000 in out-of-pocket charges during their lives.

Nonetheless, for patients like Gray, the obstacles to treatment are worth surmounting. Her chemotherapy treatments left her weakened and with mouth sores. She also lost her hair.

But five years after her lengthy treatment began, Gray is now able to work full-time after finishing a clinical nursing degree.

“My life has really been transformed,” she said. “I get to live life as a normal person.”

About eight months after her treatment, Gray woke up and thought her body had gone numb because she didn’t feel any pain. She started pinching her face and thighs before realizing that the lack of pain meant her treatment was working.

“I can dream again,” Gray said. “I had stopped dreaming and hoping for anything. Now I have big dreams and big plans.”