A Simpler Approach to Treating Sickle Cell Disease

July 21, 2013

Millions of people worldwide suffer from sickle cell disease, a genetic disorder that can cause severe pain, both acute and chronic, along with damage to various organs. The patients have a significantly shortened life span as a result. This hereditary condition affects about 100,000 people in the United States and several million in Africa, many of whom will die before they reach adulthood.

At Emmaus Medical Inc., we set out to alleviate the suffering caused by sickle cell disease by using a relatively simple approach based on the amino acid, L-glutamine. While the logic for the mechanism of its action was well explained, it was not always easy to convince people that such a simple therapy could help. We have come a long way since then. Now, we are conducting a Phase III clinical trial of our oral L-glutamine therapy, which is only one of four drugs that have been approved by the Food and Drug Administration for Phase III trials for sickle cell disease. We expect to conclude the trials by year-end and, under the fast-track status granted by the FDA, may be eligible for priority review.

The Phase III trials mark an important milestone in our research that began more than two decades ago. Early on, we found that L-glutamine tends to make sickle cells healthier. Here’s how: Research shows that sickle cells are more susceptible to oxidation damage than normal red blood cells, but at the same time have an increased capacity to make one of the most potent anti-oxidant substances. But there was an issue; there were not enough raw materials to produce this substance. Therefore, we simply decided to try giving sickle cells more raw materials to produce this anti-oxidant substance. That’s where L-glutamine came in. The evidence clearly demonstrated that sickle cells use glutamine to produce the substance.

In our studies, we found that L-glutamine treatment reduces the tendency of sickle cells to become sticky and block small blood vessels. This is important because much of the suffering endured by sickle cell patients is due to the fact that the sickle-shaped red blood cells tend to adhere to the cells that line capillaries and block them. These blockages cause all the problems, including painful crises and organ failures associated with the disease. Patients receiving L-glutamine in our Phase II clinical trials, which were completed in 2008, experienced fewer painful sickle cell crises and had a lower rate of hospitalization. No major side effects were noted. The current Phase III trials involve more than 200 patients at more than 30 sites around the United States.

We set out to help people suffering the effects of sickle cell disease live better lives. The treatment is not meant to be a cure but we are hoping that this simple daily treatment will prevent the terrible ramifications of sickle cell disease. After decades of research, we hope to be able to finally reach that goal in the near future.

Niihara-Final_2.29.2012About Dr. Yukata Niihara

Yutaka Niihara, M.D., M.P.H., is Chief Executive Officer of specialty pharmaceutical company Emmaus Medical Inc. and principal inventor of the patented L-glutamine therapy for the treatment of sickle cell disease. Dr. Niihara is also a Clinical Professor of Medicine, Division of Medical Oncology/Hematology at David Geffen School of Medicine at UCLA. You can learn more about Emmaus Medical here.

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