Small-Molecule Inhibitors of Human Galactokinase for the Treatment of Galactosemia and Cancers

Lactose, found in dairy products and other foods, is comprised of two simple sugars, glucose and galactose. In galactosemia, where galactose is not properly metabolized, build-up of toxic compounds, such as galactose-1-phosphate, can lead to liver disease, renal failure, cataracts, brain damage, and even death if this disorder is left untreated. Currently, the only treatment for galactosemia is elimination of lactose and galactose from the diet, but in some cases this is not sufficient to avoid long-term complications from the disorder.

This technology describes selective small-molecule inhibitors of human galactokinase, which inhibit the first step in galactose metabolism. These compounds could be used to treat galactosemia by eliminating the build-up of toxic metabolites in brain, liver and other tissues, and could form the basis for the first effective treatment for this disorder.

These inhibitors are also promising candidates for the treatment of certain cancers, such as PTEN/AKT misregulated cancers. The inventors have already shown that the inhibitors are cytotoxic for several cancer cell lines.

Potential Commercial Applications: Competitive Advantages:
  • Treatment of galactosemia
  • Treatment of certain cancers, such as PTEN/AKT misregulated cancers
  • There is currently no effective treatment for classic galactosemia, where dietary restriction cannot prevent long-term complications in some cases.
  • Cancer therapeutics based on these inhibitors are predicted to have minimal side-effects.

Development Stage:
  • Early-stage
  • In vitro data available


Matthew Boxer (NCATS)  ➽ more inventions...

Intellectual Property:
U.S. Pat: 9,447,087 issued 2016-09-20
U.S. Pat: 10,471,061 issued 2019-11-12
US Application No. 14/346,400
US Application No. 16/152,735

Collaboration Opportunity:

The National Center for Advancing Translational Sciences is seeking statements of capability or interest from parties interested in collaborative research to further develop, evaluate or commercialize Small-Molecule Inhibitors of Human Galactokinase for the Treatment of Galactosemia and Cancers. For collaboration opportunities, please contact the NCATS Office of Strategic Alliances at

Licensing Contact:
Ami Gadhia,
Phone: 301-827-7159

OTT Reference No: E-240-2011-0
Updated: Apr 20, 2016