2012 MPS III Grand Challenge Research Grant Update
In February of 2012, the National MPS Society announced the availability of a $235,000 Grand Challenge Grant for the treatment of MPS III. The Society and its MPS III foundation partners, including Team Sanfilippo, combined general research dollars with MPS III research dollars to create this unprecedented award amount. The purpose of the grant was to take a large step forward in finding a treatment for MPS III. Following a recommendation by the Society’s Scientific Advisory Board, the Grand Challenge Grant was awarded to Dr. Brian Bigger in June of 2012 for his project, “Evaluation of high dose genistein aglycone in the treatment of mucopolysaccharide disease types IIIA, B and C.”
The purpose of the project was to evaluate the efficacy of treating MPS III individuals with genistein using trial participants in the UK. Being a treatment trial, a significant amount of additional funds had to be raised and UK regulatory approval obtained before the trial could be started. In February of 2013, the Society’s Board became concerned about the delays in the start of the trial and whether the project was viable. A June 2013 deadline was set to begin the project or the funds would be withdrawn. Following positive movement towards starting the trial, the Society disbursed $117,500 to the project in June 2013 with the stipulation that the remaining funds would be disbursed after one year of treating trial participants if they were showing positive results.
As of May 2014, the project is not fully funded and UK regulatory approval has not been obtained. Due to the over two year intervening period since the Grand Challenge Grant was first advertised, the Board voted on May 3, 2014 to withdraw the undisbursed $117,500 support from the genistein project and to advertise the availability of these remaining funds to ensure they are spent on the best MPS III project at this time. The genistein project can be resubmitted for consideration along with other newer projects that may now be available.