Vaso-Occulsive Crisis: The Future of Treating Patients with Sickle Cell Disease in Emergency Departments

Claudia Morris, MD, Childrens Healthcare of Atlanta, Department of Pediatrics, Emory University School of Medicine, has been studying arginine (Arg) as a therapeutic option for sickle cell disease (SCD) for years. Recently, her research revealed groundbreaking findings of the effects of Arg on patients experiencing a vaso-occlusive episode (VOE).

While statistically insignificant, results showed a clinically relevant decrease in total parenteral opioid (TPO) and a shortened time to crisis resolution in both intervention groups treated with Arg compared with placebo. Arginine is approved by the FDA for growth hormone stimulation, and because of that investigators have access to 50 years worth of safety information, despite the lack of use for treating VOEs.

"The challenge right now is that we have no therapies in the emergency department to treat acute pain, outside of pain medications and IV opioids," Morris said. "I'm delighted to see a growing interest in biopharma to investigate new drugs to treat pain, and so I'm confident that we're going to have a pipeline of new interventions for patients, which is terrific."

Regardless of the condition, pain medication can have long term effects, she continued. Patients with sickle cell disease experiencing a VOE are presenting to the emergency department (ED) in severe pain. While, there are multiple mechanisms at play during an episode, the acute pain is one of the main issues.

"What's really exciting about Arginine is, as a nutritional supplement it's incredibly safe," Morris explained. "And we can actually impact the total amount of IV opioids they use."

Right now, Morris and her team of investigators are working with the FDA one phase 3 data to bring this treatment to emergency departments. She also shared that her colleague Richard Onalo, MD, Department of Paediatrics, Faculty of Clinical Sciences, University of Abuja, Gwagwalada, Nigeria, had positive results from his study in Nigeria.

"The fact that he had such a positive result of oral arginine in Nigeria, is just really exciting," Morris said, "because I think that there is this option in Sub Saharan Africa where they don't necessarily have access to hydroxyurea, or some of these other new medications that may be coming down the pike, that are also very expensive."

To learn more about Dr. Claudia Morris's research, watch the rest of HCPLive's interview on arginine for sickle cell disease.