SoCal researchers develop breakthrough sickle cell treatment

TORRANCE, Calif. (KABC) -- Sickle cell disease is a painful blood disorder that predominately affects people of African descent. Until now, doctors had to rely on treating it with medications that come with a long list of devastating side effects.

But local researchers with the Los Angeles Biomedical Research Institute in Torrance have just received FDA approval for a new treatment that will drastically cut down on those side effects and improve the lives of many patients.

Juanita Gougis is one of those patients. The 28-year-old, from Inglewood, has spent most of her life doubled over in pain. She said she missed out on a lot of activities -- school and family gatherings and sports. Hospital visits became part of her routine.

But today, instead of checking into a hospital, Gougis now gets to visit with the researcher who turned her painful and debilitating disease into a manageable condition.

The medication is called "Endari," and it's the first drug ever to be FDA approved for sickle cell anemia in children, and the first drug in 20 years for sickle cell in adults.

But it began as a nutritional supplement, known as L-glutamine.

"I heard it was pretty healthy, and with no side effects," said Gougis.

In a four-month study, of 230 patients, Endari resulted in a 25 percent decrease in pain episodes and 33 percent fewer hospitalizations.

Dr. Yukata Niihara is the CEO of Emmaus Life Science and a LA BioMed investigator. He said they started to see improvement in the blood cells. He explained that in sickle cell disease, blood cells become hard and elongated, and take on a sickle shape.

L-glutamine improves the production of a key antioxidant called NAD. This helps the cells circulate better. "It reduces the stickiness of red blood cells," Niihara said.

While you can get L-glutamine over the counter, Niihara said it often contains impurities. Endari is a standardized, medical-grade treatment.

"We looked into a glutamine that was so pure, you could inject it into the blood vessels," Niihara said.

Juanita takes her high-dose supplement in a daily smoothie. She's grateful that the medication has allowed her to feel normal again, and visit the hospital once a year, instead of once a month.

She's able to swim again and go to college, and her mother said she's thankful she doesn't have to see her daughter in pain anymore.

"She's happy to see me just participate in life again", said Gougis.