Development of a Spermine Prodrug
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Dr. Phanstiel discusses exciting developments with a “prodrug” that he’s been working on for a year and a half.
A prodrug is a compound that the patient’s body converts into a medication.
He first describes the polyamine pathway as it relates to SRS. Polyamines are organic compounds. They are present in all cells in our bodies. The polyamine pathway converts putrescine into spermidine, and converts spermidine into spermine. Healthy cells need the right levels of all three polyamines.
SRS patients have a mutation in the SMS gene. The SMS gene manufactures an enzyme called spermine synthase. This enzyme manufactures a polyamine called spermine. Individuals with SRS don’t have enough spermine.
Dr. Phanstiel hopes to treat SRS with a prodrug. This prodrug will introduce spermine into the cells. It’s non-toxic. He has shown that the prodrug can improve spermine levels in cells from an SRS patient. The next step is to test in SMS mice.
Dr. Phanstiel acknowledges the contributions of Dr. Mukund Tantak. Dr. Tantak developed the chemistry for the prodrug. The Greenwood Genetic Center and the Snyder-Robinson Foundation provided funding for this project.
About the Presenter
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Otto Phanstiel, PhD is Professor of Medical Education at the University of Central Florida College of Medicine, Orlando. His specialty is medicinal chemistry and he carries out research on the development of new drugs including compounds which target the polyamine transporter activity. These drugs starve cancers of key polyamines which limits their growth and blocks the spread of metastatic cells to other tissues. In his presentation, he will discuss development of methods to correct the imbalance in polyamines in cultured cells from SRS patients.
Otto obtained a PhD. in Organic Chemistry from the University of Florida and is another past chair of the GRC on Polyamines.