ST. LOUIS – Friday was a monumental day for Americans battling a rare but painful disease. The Food and Drug Administration has approved two new gene therapies for patients with sickle cell disease.

Studies show they are extremely effective, too. The news had patients, doctors, and advocates for those fighting the disease in the St. Louis area celebrating.

The new, one-time gene therapy treatments are for sickle cell patients who are at least 12 years of age with severe forms of the disease.

The disease affects hemoglobin, the protein in red blood cells that carries oxygen. A genetic mutation causes the cells to become crescent-shaped, which can block blood flow. That can cause excruciating pain, organ damage, and stroke.

An estimated 100,000 Americans live with the illness. St. Louisan Bonita Washington is one of them.

“It is pain that we have no control over. The pain is real,” Washington said.

Washington said the painful attacks, or episodes, people with sickle cell disease are forced to endure are almost unbearable. They often force repeated hospitalizations and strip patients of their ability to perform basic daily functions. But Washington said Friday’s news gives everyone close to the disease hope.

“To know there are people out there who see our pain and want to do something to alleviate or eradicate it, it’s wonderful,” she added.

Many people live with sickle cells, but it’s the people lost to the disease that many times make the greatest impact. Rosemary Britts’ daughter, Ronicia, died four years ago from a sickle cell. She was just 29 years old. Britts said her daughter was hopeful the new therapy would finally rid her of the disease.

“She was waiting for gene therapy to be approved,” Britts said. “I think she would be excited.”

Britts said sickle cell may have taken Ronicia’s life, but it didn’t stop her from living.

“She lived her life every day like it was her last. There was nothing she wouldn’t try to do,” Britts said. “If she wanted to skydive, I would have figured out a way to strap an oxygen tank on her and get her in a plane.” These new therapies could not only save lives but also allow more people with sickle cell disease to live fully without the restrictions often accompanied by the illness.

“To believe that this doesn’t have to be the end, I can get this treatment and live my life like anyone else,” Britts said.

These new gene therapies come with a steep price tag, potentially in the $1 million range without insurance. They also take weeks and involve chemotherapy. Until now, the only way to eradicate sickle cell disease was through a bone marrow transplant.