A new treatment for sickle-cell anemia, researched at LA BioMed and developed by the medical institute’s Torrance spinoff company, will hit the market in October.
Los Angeles Biomedical Research Institute celebrated Monday the launch of Endari, a L-glutamine oral powder that won Food and Drug Administration approval on Friday, after 25 years of research and tests.
“This is a breakthrough moment that we celebrate today,” county Supervisor Mark Ridley-Thomas said.
“It represents hope for millions of people around the world.”
Sickle-cell anemia, which disproportionately affects people of African descent, is a genetic mutation that turns round, free-flowing red blood cells into stiff, curved cells that clog up blood vessels and deplete the body of oxygen.
LA BioMed backed the Emmaus Life Sciences Inc. startup as the Torrance company researched the therapy and tested it in years of clinical trials. While the institute shares a campus and many research projects with Harbor-UCLA Medical Center in Harbor City, LA BioMed is a private, nonprofit entity.
“There’s been a lot of skepticism about whether this would work or not,” LA BioMed president and CEO David Meyer said. “It was a rough road and we had to, pretty much, go it alone.”
An estimated 100,000 Americans suffer debilitating bouts of pain and sickness because of the devastating blood disorder, passed down through family lines. Globally, 25 million people are believed to have the disorder, and 3 million Americans carry the gene that causes it, researchers say.
Until now, doctors have relied heavily on lethargy-inducing pain medication and hydroxyurea, which brings with it a litany of devastating side effects to treat the painful side effects of sickle-cell anemia.
Emmaus Life Sciences CEO Yutaka Niihara discovered the potential of L-glutamine — an amino acid that boosts immune function — in the 1990s while working as a hematologist-oncologist physician at Harbor UCLA Medical Center.
“Encountering patients (at Harbor UCLA) on a personal basis, I really realized they are experiencing something that is the most devastating thing we can imagine. That’s what prompted me to do something, Niihara said. “If I had known it would take 25 years to develop this, I don’t think I would have had the courage to start.”
For Juanita Gougis, an early research subject in Endari’s clinical trials, the new treatment has turned once-monthly hospital stays into once-yearly visits.
The 28-year-old Inglewood woman was diagnosed with sickle-cell anemia as a toddler. When she was five years old, the disease forced her to quit the basketball team because she couldn’t keep up with the other kids.
“I wanted to play basketball and it was very difficult,” Gougis said. “After playing, I’d have throbbing pain in my limbs and would have to go to the hospital.”
Throughout her childhood and teenage years, Gougis grew used to routine hospital stays, heavy pain medication and bouts of depression.
But, since joining the Endari trial, she’s rebounded to a nearly normal life, making only rare visits to the hospital.
“Going to the hospital less is a great relief to me,” Gougis said. “I don’t have this downward opinion of my life, where all I see in my future life is the hospital.”
Like many drugs, Endari can cause side effects, including constipation, nausea, headache, coughing and pain in the extremities, back or chest.
L.A. County Department of Health Services Director Mitchell Katz also praised the new treatment.
“I’ve been so sad as a physician that the number of treatments I’ve had to offer are so few and not as helpful as I wish,” Katz said. “From now on, every time I prescribe this to a patient of mine, I’m going to say a thank-you prayer to you for having developed it.”
