A Vitamin-Derived Drug

Xiao-kun Zhang, Ph.D.
 and Marcia Dawson, Ph.D.

Xiao-kun Zhang, Ph.D. Professor, Tumor Development Marcia Dawson, Ph.D. Professor, Tumor Development

In the late 1980s, researchers found that retinoic acid nuclear receptors (RARs) regulate genes that are sensitive to trans-retinoic acid (an oxidized form of vitamin A). Their research led to understanding how trans-retinoic acid could change some types of cancer cells to normal cells and could induce remission in patients with acute promyelocytic leukemia.

Retinoid X receptor agonist docked to the
RXR ligand-binding domain

Retinoid X receptor agonist docked to the RXR ligand-binding domain

The Dawson lab synthesized 9-cis-RA, which was later found to activate nuclear receptors RAR and RXR. SBP researcher Dr. Xiao-kun Zhang, then a postdoctoral fellow in SBP researcher Dr. Magnus Pfahl’s laboratory, discovered that 9-cis-RA can influence many diverse signaling pathways. This research helped identify Targretin®, a synthetic ligand more stable and specific than 9-cis-RA, which was designed and synthesized by Dr. Marcia Dawson’s lab.

Molecular structure for Targretin®

Molecular structure for Targretin®

In 1999, Targretin® was granted approval for orphan drug status by the FDA for systemic treatment of generalized, cutaneous T-cell lymphoma, a rare variety of non-Hodgkin lymphoma that primarily affects the skin and causes a number of deaths in the U.S. each year. Orphan drugs receive accelerated approval so that drugs with limited markets will be commercialized by pharmaceutical companies.

  Research - Cancer - Your Health: A Vitamin-Derived Drug
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