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
Blood-based biomarkers for Sanfilippo syndrome progression and treatment
Grant Summary Institution: University of California, Los Angeles Primary Investigator: Gal Bitan, Ph.D./Professor of Neurology Duration: 24 months Start Date: September 2021 Type of Sanfilippo studied: Types A, B, C, and D Types of Sanfilippo that could benefit: Types...
Identifying Disease Modifiers in Drosophila Models of Sanfilippo (MPS IIIA)
Grant Summary Institution: Clemson University, South Carolina, U.S. Primary Investigator: Trudy Mackay, PhD, Director, Clemson Center for Human Genetics and Self Family Endowed Chair of Human Genetics Duration: 24 months Start Date: January 2022 Type of Sanfilippo...
Discovery & validation of translational biomarkers for Sanfilippo childhood dementia
Page reviewed by: Dr. Cara O'Neill, FAAP and Jan Kaslin, PhD Page last updated: February 18, 2022 For web accessibility options: Click/tap the floating blue icon on the right. Grant Summary Institution: Monash University, Victoria, Australia Primary Investigator: Jan...
Targeting Heparan Sulfate Proteoglycans as a Novel Therapeutic Strategy for Sanfilippo
Grant Summary Institution: University of Naples Federico II, Naples, Italy Investigator: Luigi Michele Pavone, Associate Professor of Biochemistry, Department of Molecular Medicine and Medical Biotechnology, Medical School, University of Naples Federico II Duration:...
Exploring the role of heparan sulfate and dopamine as disease modifiers in Sanfilippo
Grant Summary Institution: Telethon Institute of Genetic Medicine (TIGEM), Pozzouli, Italy Principal Investigator: Elvira De Leonibus, PhD, of TIGEM and the Italian National Research Council Duration: 24 months Start Date: July 1, 2021 Type of Sanfilippo studied: A...
Newborn screening pilot ScreenPlus that includes Sanfilippo Type A and B
When children with a rare disease are diagnosed at birth, they have the greatest opportunity to receive timely, approved treatments or participate in clinical trials that can provide them better quality of life and less pain and suffering. Early diagnosis and...
Strengthening the rationale for the use of the “molecular tweezer” CLR01 in the treatment of Sanfilippo
Cure Sanfilippo Foundation has co-funded a research grant with Sanfilippo Children's Foundation (Australia) and H.A.N.D.S consortium, an international network of Type C families and groups. This grant is to Associate Professor Alessandro Fraldi at CEINGE - Advanced...
Investigating NDST1 inhibitors as target for substrate reduction therapy for Sanfilippo
Identifying a novel class of substrate reduction therapy drugs for the mucopolysaccharidoses that inhibit N-deacetylase/N-sulfo-transferase (NDST) Cure Sanfilippo Foundation has co-funded a one-year research project with Sanfilippo Children’s Foundation (Australia) to...
Exploring anti-fungal immunity and the airway microbiome in Sanfilippo syndrome (MPS III)
Cure Sanfilippo Foundation is funding a two-year project by Neta Shlezinger, PhD, at the Koret School of Veterinary Medicine in Israel, in collaboration with Dr. Annick Raas-Rothschild, Associate Professor and Director of Rare Diseases Institute at Sheba...
Creation of plasmid and efficacy study for MPS IIIC clinical trial
Cure Sanfilippo Foundation has funded New York-based Phoenix Nest, Inc. The funding supports creation of plasmid and the required in-vivo efficacy study in the Sanfilippo Type C (MPS IIIC) mouse model. These and other steps will enable the project to be evaluated by...
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...
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...
Facilitating and Enhancing CNS Entry of Systemically Delivered AAV Vectors for MPS IIIA, IIIB in Mice
Uniform gene delivery to the central nervous system (CNS) remains one of the most intractable challenges to the development of effective gene therapies for a wide range of neurological diseases, including MPS III (Sanfilippo Syndrome). While the brain microvasculature...
Cure Sanfilippo Co-Funds Research Into Treating Behavioral Symptoms of MPS III
Cure Sanfilippo Foundation has again collaborated with Sanfilippo Children's Foundation (Australia) to fund a new research project by Dr. Elvira De Leonibus at the Telethon Institute of Genetics and Medicine (TIGEM) in Italy. The research project aims to understand...
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...
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...
Funding Development of Nematode & Fly Disease Models and Screening Compounds
Cure Sanfilippo Foundation is funding new research to develop nematode and fly disease models and screening compounds for Sanfilippo Syndrome Type A (MPS IIIA) and Type B (MPS IIIB).
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...
Re-positioning of FDA-Approved Compounds in MPS III Cell Models
Cure Sanfilippo Foundation is funding search to explore re-positioning FDA-approved compounds using cell-based, high-content screening in Sanfilippo Syndrome (MPS III) cell models. Awardee: Diego Medina, PhD- Telethon Institute of Genetics and Medicine (TIGEM)-...
New Therapeutic Approach to Treat Central Nervous System in Sanfilippo
Target Amyloid Aggregation as a New Therapeutic Approach to Treat the Central Nervous System in Sanfilippo SyndromeUpdate: April 2021 Cure Sanfilippo Foundation has co-funded a translational research grant to build upon this research. Learn more. Update: April 2020...
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...
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
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...
Neural And Mesenchymal Stem Cell-Mediated Gene Therapy for Sanfilippo Syndrome Type B
Grant Summary Institution: University of Florida Primary Investigator: Coy Heldermon, MD, PhD Duration: 5.75 years Start Date: March 2017 Type of Sanfilippo studied: Types BCure Sanfilippo Foundation is co-funding this gene therapy-approach project with Sanfilippo...
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...
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...
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...
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...
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...
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...
Mesenchymal Stem Cells to Produce Sulfamidase for Intrathecal Delivery
Cure Sanfilippo Foundation awarded Jan Nolta, PhD, Director of UC Davis Stem Cell Program, University of California Davis, Institute for Regenerative Cures to engineer mesenchymal stem Cells (MSCs) to produce sulfamidase for intrathecal treatment of Sanfilippo...
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...
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...