Research

The Isaac Foundation has provided over $2 million in research grants for MPS related projects around the world. Some of these projects include:

• Kommodo Therapeutics’ investigation of Engineering Memory T Cells as a platform for Long-Term Enzyme Replacement Therapy in Lysosomal Storage Disorders (https://pubmed.ncbi.nlm.nih.gov/38712248/)
• Dr. Brian Bigger’s project on brain-targeted stem cell gene therapy for MPS II, which moved into a first of its kind clinical trial in 2023 and recruitment is ongoing (https://www.manchester.ac.uk/about/news/groundbreaking-gene-therapy-trial-for-hunter-syndrome-opens/)
• Dr. Alberto Auricchio’s research into gene therapy for MPS VI, one of our earliest and longest running projects. We began funding research of gene therapy in cats within the lab (https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3581049/pdf/hum.2012.179.pdf) and eventually this translated into the first human trial of gene therapy for MPS VI (with Principal Investigator Dr. Nicola Brunetti). Phase I and II of the trial are now complete (https://clinicaltrials.gov/study/NCT03173521) and expansion is being planned.
• Dr. Calogera Simonaro’s foundational work discovering the influence of inflammation on bones and joints of patients with MPS diseases (https://www.pnas.org/content/107/1/222.long). 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 (https://clinicaltrials.gov/ct2/show/NCT03632213).