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FDA approves first new drug to treat sickle cell disease in more than 20 years

Michael Loiacono has sickle cell disease, a condition diagnosed at birth. It causes normally disc-shaped red blood cells to instead be sickle-shaped -- causing a host of health issues. (CBS Austin)

On the surface, Michael Loiacono looks like a healthy 30-year-old man.

"It's not a disease that people passing would know is something that afflicts me," he said. "But on your good days, you're operating at 30-40 percent energy capacity as other people."

Michael has sickle cell disease, a condition diagnosed at birth. It causes normally disc-shaped red blood cells to instead be sickle-shaped -- causing a host of health issues.

"Over time, that leads to a lot of difficulties with the tissues that are involved in that they simply don't get a lot of blood flow chronically so they don't work very well," said Dr. Beth Hellerstedt, a hematologist-oncologist at Texas Oncology.

The symptoms affected Michael at a young age.

"As a kid, playing sports, being subjected to high heat, or jumping in an extremely cold pool, doing things that for a lot of kids feel like a mundane kid activity had serious and sometimes dire consequences," he said.

Those consequences are incredibly painful episodes.

"Imagine being a child, every time you go to bed you don't know if you're going to wake up that night screaming and in excruciating pain and having to be shipped to the hospital as soon as possible," he said.

The FDA just approved a new drug called Endari could offer some relief. Dr. Hellerstedt said it could reduce the frequency of those painful incidents.

"Every single measure that we can do to be of assistance is going to be an improvement for these patients in their quality of life," she said.

This is the first new drug approved for sickle cell in more than 20 years. Michael, a husband and father to a young son, is excited to see if Endari will work for him.

"I was delighted to get the news," he said. "I probably had half a dozen of my friends text me the day that it came out."

He hopes it's a new step toward a more normal life for him and of more new treatments to come.

"Ideally, we'd like to find something to eradicate sickle cell, but this is a great step in the right direction and more funding, research, and resources dedicated to this I think would have a profound impact," he said.

To learn more about sickle cell disease and Endari, visit the Sickle Cell Association of Texas' website.

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