July 5, 2008
Local Medical Firm Tackles Rare Diseases
By Melissa Evans
After years of clinical trials and research, Dr. Yutaka Niihara came up with a treatment for sickle cell anemia that was so simple he almost didn't believe it.
L-Glutamine, one of the most common amino acids produced in the body and found abundantly in protein-rich foods, bloated the sickle- shaped cells that cause immense pain for thousands of people who suffer from the disease.
"It's such a simple and safe treatment," said Niihara, who made the discovery while working as an investigator at the Los Angeles Biomedical Research Institute near Torrance in the late 1990s. "There are virtually no side effects."
Making medical discoveries, however, and getting treatments to the public are typically separated by years - a reality Niihara is grappling with at his Torrance-based pharmaceutical company, Emmaus Medical Inc.
The 8-year-old company, which focuses on developing drugs to treat rare diseases, last month launched its first drug - medical grade L-Glutamine to treat people with a condition called "short bowel syndrome."
It affects fewer than 20,000 people nationally, causing starvation due to the removal or absence of the small intestine. Without this organ, people die of malnutrition because their bodies don't absorb nutrients in food.
Researchers found that the L-Glutamine, in combination with a drug called Zorbtive, restores the body's ability to process and store nutrients. The treatments are expensive (about $600 a month for the prescription L-Glutamine alone), but far less costly and invasive than intravenous feeding - the only option for those with short bowel syndrome.
(Because it is FDA-approved, private and public insurance companies will now pick up the cost of the drug, marketed under the name NutreStore.)
The launch of the drug was a major coup for Emmaus, which Niihara founded in 2000 on paper to secure grants for research. Just this year, the company moved into offices on Western Avenue and now has a staff of 14.
Niihara and his staff have managed to attract dozens of venture capitalists, secure grant funding from the Food and Drug Administration and the National Institutes of Health and raise money from his friends.
Developing a single drug costs upward of $100 million, given the length of time it takes to run clinical trials and secure government approval - a fact that makes it difficult for small pharmaceutical companies to survive, those involved say.
Emmaus has benefited from its close ties to the Torrance-area research institute, where Niihara still serves as a part-time researcher.
One of the goals of LA BioMed is to support these types of companies in order to make research tangible for the benefit of the public, said Kenneth Trevett, director of the institute.
"Our underlying mission is to commercialize discovery so that it can be used in patient-care settings," he said.
LA BioMed has facilitated the start of four other small spin-off companies created by its researchers, most of them in the South Bay. The institute helps the companies primarily with grant writing and other personnel costs.
Niihara is hoping to secure government approval for use of his earlier discovery with L-Glutamine to treat sickle cell patients sometime in 2009.
Because he was a researcher at LA BioMed at the time of the discovery, the institute holds the patent, but the company would be licensed to sell the drug. Emmaus would then pay a small royalty to LA BioMed on profits from the drug.
All other profits would go back toward research and development of other drugs.
Niihara said they have about five other drugs at the conceptual or developmental stage for treatment of a number of diseases and conditions, including cancer.
His real passion, however, is sickle cell anemia, a condition caused by a mutation in genes of those with African roots. The red blood cells mutated into C-shaped cells to ward off malaria, a disease spread by bugs prevalent in the continent of Africa.
The odd-shaped cells, however, don't circulate properly throughout the body, causing pain, infections and damage to organs. The average life span of those with this condition is in the mid- 40s.
Researchers found that compounds in L-Glutamine bind to the cells, enlarging them and helping the cells flow more easily throughout the body. Studies on this finding are in final clinical stages.
L-Glutamine is already available commercially in most drug stories, but patented pharmaceutical grade would be concentrated and the dosages would be more accurate, researchers say. With government approval, the treatment would also be covered by insurance.
Eventually, Niihara said he hopes to bring the treatment to Africa, where sickle cell anemia is far more widespread.
"It is more profitable in the United States, but the prevalence is much less," he said. "That is one of the realities of medicine. With a treatment like this, so simple and inexpensive, I think it could help a lot of people."
THE POWER OF
Torrance-based Emmaus Medical Inc. has developed a medical-
grade version of the amino acid to treat "short bowel syndrome," a condition afflicting fewer than 20,000 people.
The company is also working to use the amino acid, found naturally in the body, to treat sickle cell anemia, a disease that more commonly afflicts people who trace their ancestry to Sub- Saharan Africa.
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