Research Projects Funded

The Isaac Foundation has provided over $1 million in research grants for MPS related projects around the world. Some of these projects include:

  • Dr. Calogera Simonaro’s foundational work discovering the influence of inflammation on bones and joints of patients with MPS diseases (article). This project led to a novel therapy called Pentosan Polysulfate that is currently in clinical trial for MPS VI.
  • Dr. Roberto Giugliani’s clinical trial evaluating whether the drug Losartan can help reduce aortic root dilation in MPS VI and MPS IVA (trial).
  • Dr. Alberto Auricchio’s research into gene therapy for MPS VI. This was one of our earliest and longest running projects, which began with investigating gene therapy in cats within the lab (article) and eventually translated into the first human trial of gene therapy for MPS VI. The trial now is nearing completion (trial).
  • Dr. Brian Bigger’s project on brain-targeted stem cell gene therapy for MPS II (article). This project is moving into clinical trial in 2021.