BETHLEHEM, Pa. — New therapies to treat sickle cell disease were approved by the U.S. Food and Drug Administration earlier this month — treatments that could be available in the Lehigh Valley next year.
Sickle cell disease is a painful hereditary blood disorder that affects Black people and certain ethnicities disproportionately.
A physician at Lehigh Valley Health Network said the health care system hopes to offer the treatment in 2024.
"I think it is a groundbreaking approval for patients who are living with this disease,” said Dr. Bradley Lash, a hematologist at LVHN. "I think sickle cell is one of the most debilitating conditions that we deal with.
“Instead of making what's considered normal hemoglobin, you make an abnormal hemoglobin called sickle and that causes the cells, under periods of stress, to get stuck in blood vessels leading to complications such as pain, strokes that start affecting people from very young ages throughout their entire life,” he explained.
Although he said there are current treatments available for the illness, those therapies require multiple treatments over time and often require the patient to have a long hospital stay. The newly approved drugs, Casgevy and Lyfgenia, are proving to be one-and-done gene therapies.
"There were two approved on the same day, they work slightly differently, but have the same kind of effect," Lash said, "taking a patient's stem cells and modifying them to basically either provide a type of hemoglobin that won't sickle or a ‘normal’ hemoglobin, so that they don't have pain."
Lash said the new therapies truly can be transformative.
"These patients from a very young age spend a significant amount of time in hospitals with pain, and it can really impact their life. So the fact that we now have a therapy that not only treats it, but may modify this and allow them to have a normal life is huge," he said.
According to the U.S. Centers for Disease Control and Prevention, about one in 13 Black babies is born with the sickle cell trait and one in 365 Black children are born with sickle cell disease.
The disorder also affects people of Hispanic, Middle Eastern, southern European and Asian Indian descent, according to the CDC.
Lash said only a few hospitals offer the new treatments now, including Children's Hospital of Philadelphia.
However, he said, LVHN is launching cellular therapeutics in early 2024, offering stem cell transplants in January or February, and then the network hopes to offer the new sickle cell gene therapy toward the end of 2024.
The FDA approval is for people over the age of 12 with recurrent pain or acute chest syndrome.
