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Research & Grants

Research Turns Hope For A Cure Into Action

The key to turning hope into action is tirelessly supporting and accelerating scientific research and projects that move us closer to treatments or a cure for Sanfilippo Syndrome.

At any given moment, there are 5-10 projects in need of funding. New ideas to investigate, promising research that needs to be taken to the next step, or a clinical trial ready to start.

Your support helps these projects happen as swiftly as possible in a race against time.

Scientific Research That Has Been Funded

Thanks to your support, Cure Sanfilippo Foundation funds scientific research exploring multiple avenues for a treatment/cure to Sanfilippo Syndrome.

Each is chosen because of its promise for moving forward with the possibility of treating children in clinical trials.

At this stage, we can’t be sure of what therapeutic treatments will work best (or work at all), which is why we support many different options that each have promising potential. We prioritize those that are proving viable, and need additional funding to continue moving these forward to bring to children.

Funded Scientific Grants

Re-Purposing FDA-Approved Inflammation Drug for Sanfilippo

Cure Sanfilippo Foundation is sponsoring an “Open-label Study of Anakinra in MPS III” clinical trial in collaboration with The Lundquist Institute (formerly LABiomed), and Sobi (Swedish Oprhan Biovitrium AB). This study is an open-label, single-arm, no placebo or...

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Neural Stem Cells for Sanfilippo Syndrome

Cure Sanfilippo Foundation has committed funding for a multi-year grant to Karl Johe, PhD, of ReMotor Therapeutics, Inc., to explore the therapeutic benefit of transplanting human neural stem cells engineered to overexpress and secrete SGSH into the brain of patients...

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Cure Sanfilippo Funds CRISPR Gene Editing of Neural Stem Cells

Cure Sanfilippo Foundation is excited to grant funding to LA BioMed for the advancement of Dr. Michelina Iacovino’s work in the field of regenerative medicine for the treatment of Sanfilippo Syndrome (MPS III). This innovative work will utilize CRISPR gene editing to...

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Cure Sanfilippo Foundation Funds New Stem Cell Research

Cure Sanfilippo Foundation is pleased to support the work of Dr. Jan Nolta’s lab in collaboration with biotech partner Neuralstem. This project aims to develop an off-the-shelf neural stem cell therapy for patients with Sanfilippo Syndrome (MPS IIIA).As the Director...

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Cure Sanfilippo Announces Support for Autophagy Research

Cure Sanfilippo Foundation is pleased to award funding support to Dr. Scott Selleck and the Pennsylvania State University (Penn State) to study the cellular process of autophagy as a central mechanism in MPS (mucopolysaccharidosis) disease progression in the central...

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Creating a Zebrafish Model of Sanfilippo Syndrome Type A

Grant Summary Institution: Australian Regenerative Institute at Monash University in Melbourne, Australia Investigator: Dr. Jan Kaslin and team Start Date: December 2018 Types of Sanfilippo that benefit: A Research Stage: TranslationalGrant Update: May 2021 "An...

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Mesenchymal Stem Cells for Sanfilippo Syndrome

Engineering Mesenchymal Stem Cells (MSCS) to Produce Sulfamidase for Intrathecal Treatment of Sanfilippo SyndromeAwardee: Jan Nolta, PhD, Director of UC Davis Stem Cell Program, University of California Davis, Institute for Regenerative CuresStart Date: August 2017

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New Therapy for Six Mucopolysaccharidoses

Update: December 2020 Cure Sanfilippo Foundation has co-funded a grant to continue Prof. Andreas Schulze's work on the development of substrate reduction therapy drugs for Sanfilippo and other similar conditions. Learn more.Cure Sanfilippo Foundation is co-funding...

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Research Strategy for Pre-Existing AAV Antibodies

Cure Sanfilippo Foundation has awarded a grant to Haiyan Fu, PhD, at Nationwide Children's Hospital in Columbus, OH, to research strategies for overcoming a patient's pre-existing AAV (Adeno-Associated Virus) antibodies so gene therapy using AAV is accessible to...

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Re-Purposing of FDA-Approved Drugs for LSD

Re-Purposing of FDA-Approved Drugs for the Treatment of LYSOSOMAL STORAGE DISEASES (LSD)Project is funded by Cure Sanfilippo FoundationProject Summary: The study aims to identify FDA-approved compounds that activate the clearance of pathologic lysosomal accumulation...

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Research Extension: Intranasal Delivery of Sulfamidase

Cure Sanfilippo Foundation is funding an extension of research by Jeff Esko, PhD, Co-Director Glycobiology Research & Training Center of  University of California San Diego School of Medicine, to explore intranasal delivery of sulfamidase as a therapy for...

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Synaptic Dysfunction in Sanfilippo Type C

Cure Sanfilippo Foundation has awarded a grant to Alexey Pshezhetsky, PhD, of St. Justine University Hospital (Montreal, CA) to explore the synaptic dysfunctions in Sanfilippo Syndrome Type C.Project Summary: The study’s major objective is to understand the...

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Testing Compounds on MPS III Fibroblasts

Cure Sanfilippo Foundation has awarded a grant to Alexey Pshezhetsky, PhD, of St. Justine University Hospital (Montreal, CA) to conduct compound testing on MPS III fibroblasts.Project Summary: Results of two previously-published studies in which MPS IIIA fibroblasts...

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Intranasal Delivery of Sulfamidase for Sanfilippo

Cure Sanfilippo Foundation is funding research by Jeff Esko, PhD, Co-Director Glycobiology Research & Training Center of University of California San Diego School of Medicine, to explore intranasal delivery of sulfamidase as a therapy for Sanfilippo...

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Support for AAV9 Gene Therapy for MPS IIIA and MPS IIIB

Cure Sanfilippo Foundation made a grant to Nationwide Children's Hospital (Columbus, OH) to conduct gene therapy to treat Sanfilippo Type A (MPS IIIA) and Type B (MPS IIIB). Project Summary: Funding support of staff to enable necessary clinical trial preparatory steps...

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Intravenous AAV9 Gene Therapy for MPS IIIA and MPS IIIB

Cure Sanfilippo Foundation awarded a grant to Abeona Therapeutics, a privately-traded company that is managing license & toxicology/drug production for the AAV9 gene therapy clinical trial at Nationwide Children’s Hospital. Project Summary: Support of formal GMP...

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