Exciting Research from Mast Therapeutics

Nancy Rene

Nancy Rene
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Advice from Nancy

Mast Therapeutics, a pharmaceutical company located in San Diego is doing research on Vepo-Loxamer, an advanced investigational new drug intended to treat vaso-occlusive crisis, the hallmark of sickle cell disease. Mast recently completed enrollment in a Phase 3 trial called the EPIC study. This study is evaluating whether or not this new drug can shorten the duration and reduce the severity of a crisis.

In the study, Vepoloxamer is given by infusion over two days in the hospital. The objective is to improve blood flow and oxygen delivery. You can read more and see the video on Vepoloxamer.

According to Greg Gorgas, one of the Senior Vice Presidents at Mast,

“Vepoloxamer has been described as a molecular band-aid, with a sticky middle and slippery ends. Our research suggests the middle of Vepoloxamer adheres to sticky cell surfaces while the slippery ends may help normalize or restore blood flow.”

You can hear the entire conversation with Greg by going to our podcast on Vepoloxamer.

When we asked if this drug is safe, Greg reminded us that only the FDA can say whether or not a drug is safe and available for widespread use. Mast expects the report from the EPIC study in September 2016.

Initially, if FDA approved, Vepoloxamer would likely be administered in the in-patient setting in addition to the standard of care for people hospitalized for a sickle cell crisis. We know that many times a crisis does not result in hospitalization and Mast is looking at investigating how or if it could be investigated for use in the outpatient setting.

At Axis Advocacy we will continue to follow the research on this and other drugs that may be important in the care of sickle cell patients. For a long time have had little to rely on other than hydration, hydroxyurea, and mediocre treatment in the Emergency Room. We sincerely hope that is about to change.

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