Headquarters: Torrance

Founder-Chief Executive: Dr. Yutaka Niihara

Business: Treatment of sickle cell disease.

Dr. Yutaka Niihara has devoted nearly a quarter-century to developing drug therapy that will relieve the pain of millions of sickle cell anemia patients around the globe. Clinical trials are scheduled to wrap up in December and his company plans to make the drug available by prescription next year. But it’s too early to celebrate.

“I’m very pleased, but I cannot be too overjoyed because you never know until the very end,” Niihara said.

He developed the drug therapy in the 1990s with Charles R. Zerez and Kouichi R. Tanaka at LA BioMed, which has granted a license to Emmaus Medical Inc. for the treatment.

Niihara traces his interest in the disease to a hematology-oncology fellowship he did at Harbor-UCLA Medical Center starting in 1989. Many of his patients were hospitalized with complications of sickle cell anemia, a genetic disease that occurs in about 1 of every 500 African-Americans.

Most of the sickle cell research at the time focused on cures or prevention. But Niihara and his colleagues at LA BioMed took another route.

“We looked at it from a simplified point of view as to how we could treat the effects of this gene,” he said.

The disease causes too much oxidation of red blood cells, transforming plump, disc-shaped cells into hard, crescent shapes. The deformed cells stick together and block blood flow, causing organ damage and pain.

After Zerez came up with a way to measure oxidation levels within the red blood cells, researchers focused on reducing that oxidation. They developed a treatment based on a type of glutamine that reduced pain and can be given orally with almost no side effects, making it safe even for children and pregnant women.

Niihara and his team requested government funding and got grants from the National Institutes of Health and the Food and Drug Administration’s Office of Orphan Products Development, which promotes research into drugs to treat diseases that pharmaceutical companies do not consider potentially lucrative enough to develop. Emmaus has also raised $40 million, mostly from private investors. A recent $7.5 million private placement attracted some institutional investors as well.

If approved, the drug would be the first new therapy for sickle cell patients on the market in 20 years and it would serve a fully diagnosed but highly underserved patient population. According to the latest figures, more than $1 billion is spent treating the 100,000 sickle cell patients in the United States annually.

Niihara is not sure whether Emmaus will form a partnership with a bigger company, be acquired or even handle production and distribution itself.

“Our main goal is to choose the right option so this medication will get not only to patients in the United States and Europe but also to Africa, India, the Middle East and South America,” said Niihara, who retired from UCLA in 2009 but still volunteers as an instructor and runs a laboratory at LA BioMed.

The reward for a lifetime of work?

“I do talk to a lot of sickle cell patients who were on the earlier trials,” he said, “and they are quite happy.”

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