FDA to consider approving a treatment to cut down sickle cell symptoms

(NBC, KYMA/KECY) - The Food and Drug Administration (FDA) is considering the approval of a treatment that could cut down the severity of sickle cell symptoms.

"This therapy is transformative. They will feel so better, but it is not yet curative," said Dr. Julie Kanter, Director of the University of Alabama at Birmingham (UAB) Adult Sickle Cell Center.

The FDA is considering two treatments. The first is called exa-cel. We all have a hemoglobin switch in our bodies, but it doesn't always work.

"What this therapy does is that it literally cuts that DNA where that switch is located and turns the fetal hemoglobin or baby hemoglobin gene back on, and then that person can produce that healthy hemoglobin and the sickle hemoglobin, which really decreases the number of pain crisis and painful events they have."

Dr. Julie Kanter, Director of the UAB Adult Sickle Cell Center

Second treatment and drawbacks

The second is lovo-cel, which Dr. Kanter is currently conducting trials on that one at UAB. It alters the patient's stem cells, fixing the genetic problems that cause sickle cell.

"It is truly transformative. It is really amazing...their hemoglobin just about comes to normal and their pain crisis goes away. It's an incredibly valuable therapy," Dr. Kanter shared.

Dr. Kanter says the treatment is a great option for patients but there are some drawbacks.

"It's not just a pill that you take. It's an incredibly long process...In total, it takes almost a year for that whole procedure to take place. After that year, though, people feel fantastic but it certainly isn't easy and it's expensive," Dr. Kanter explained.

Dr. Kanter reminds patients exa-cel and lovo-cel aren't cures, but doctors believe it does reduce crisis symptoms.

The new treatment is up for FDA approval. It's a gene therapy called LentiGlobin.