What We’re Funding

Gene therapies in development have the potential to help all children with Duchenne, and Columbus Children’s Hospital is on the cutting edge of gene therapy trials with their current trial of Follistatin for Duchenne.  Another very promising treatment is Galgt2. Researchers Dr. Paul Martin and Dr. Kevin Flannigan are designing a trial of Galgt2 in humans.  Dr. Martin writes, “The Overexpression of Galgt2 in the skeletal muscles of transgenic mice inhibits the development of muscular dystrophy…Galgt2 may inhibit the development of muscle pathology in mdx animals…Muscles showed a marked improvement in normalized isometric force during repetitive eccentric contractions…[Also] muscles from Galgt2 transgenic mice showed a significant decrement in normalized specific force and in hind limb and forelimb grip strength at some ages.”  

As gene therapies become viable treatments, one concern is that doctors will not be able to do more than one dose because the body develops an immune response to the viral vector. For that reason, it is important that doctors will be able to modulate that response. Dr. Keith Foster, at the University of Reading in England has been working a strategy to control immune response. Dr. Foster writes,  “…Analysis shows that we have generated novel myeloid precursor cells (MPCs) that have all the hallmarks of being cells with positive immunomodulatory properties; namely the ability to migrate out from the vasculature to sites of inflammation and damage (supplementary figure 1c) and to potentiate the legacy of long term immune tolerance via the induction and/or expansion of a resident Treg population through appropriate cytokine production…These isolated MPCs could dampen the profound dysregulated inflammation in DMD by suppressing activated T cells, and can be manipulated to specifically present antigen such that the generation of in vivo immune tolerance to AAV capsid proteins has great potential.” 

Every Duchenne parent is aware that the only approved medications for Duchenne are corticosteroids.  While a steroid keeps a child on his feet for a few extra years, the side effects are awful. They include weight gain, loss of bone density, and cataracts. Researcher Dr. Eric Hoffman’s new drug, VBP-15 may provide all the benefits of steroids without the side effects. Dr. Hoffman writes that VBP-15 is “an oral drug with mechanisms that provide efficacy through anti-inflammatory signaling and membrane-stabilizing pathways, independent of hormonal or immunosuppressive effects…VBP15 protects and promotes efficient repair of skeletal muscle cells upon laser injury, in opposition to prednisolone. Potent inhibition of NF-?B is mediated through protein interactions of the glucocorticoid receptor, however VBP15 shows significantly reduced hormonal receptor transcriptional activity. The translation of these drug mechanisms into DMD model mice improves muscle strength, live-imaging and pathology through both preventive and post-onset intervention regimens.”  

Our Current Funding:

Project	Category	Investigator/Organization	Affiliation
Expansion of Department of Defense Grant (Cardiac)	General Support	Dr. Elizabeth McNally	Northwestern University
DMD Clinic	General Support	N/A	University of Massachusetts, Worcester
Pandemic relief for DMD Families	General Support	Hope For Gus	N/A
CRISPR/Cas	Preclinical drug development	Eric Olson	University of Texas Southwest
DMD Clinic	General Support	N/A	University of Massachusetts Memorial

Our Past Funding:

Project	Category	Investigator/Organization	Affiliation
GalGt2 Gene Therapy Trial	Preclinical drug development	Dr. Kevin Flanigan	Columbus Children’s Hospital
VBP15 Dissociative Steroid Drug	Clinical trial	Eric Hoffman, PhD	ReveraGen
Repeat Dosing Gene Therapy	Preclinical drug development	Dr. Keith Foster	University of Reading
Folistatin Gene Therapy	Preclinical drug development	Dr. Jerry Mendall	Nationwide Children’s Hospital
Program to Consolidate Research	Research Support	N/A	Charley’s Fund
Myomics Drug Screening	Preclinical drug development	Brian Tseng, MD, PhD	Massachusetts General Hospital
Gene Therapy Research	Preclinical drug development	Jeff Chamberlain, PhD	University of Washington
Laminin 111	Preclinical drug development	Bradley Hodges, PhD	Prothelia
Nutraceutical (THI), Skeletal Muscle Function	Preclinical drug development	Hannele Ruohola-Baker, PhD	University of Washington
Zebra Fish Screening of FDA Approved Pharmaceuticals	Preclinical drug development	Dr. James Dowling	University of Michigan
Preparation of Halofuginone for Phase I/II Clinical Trial	Preclinical drug development	Marc Blaustein	Halo Therapeutics
Carmeseal (P188), Cardiac and Skeletal Muscle Function	Preclinical drug development	Bruce Markham, PhD	Phrixus Pharma/Coalition Duchenne
Development of Dystrophin Independent Therapy	Preclinical drug development	Louis Kunkel, PhD	Boston Children’s Hospital/Jett Foundation
Anti-fibrotic (Relaxin) Skeletal Muscle and Diaphragm Histology	Preclinical drug development	Jeffrey Chamberlain, PhD	University of Washington
Nutraceutical (Quercetin) Respiratory and Cardiac Function	Preclinical drug development	Joshua Selsby, PhD	Iowa State University
Nutraceutical (THI), Skeletal Muscle Function	Preclinical drug development	Dawn Lowe, PhD	University of Minnesota
Duchenne Dashboard	Project management software	Carlo Rago, PhD	OpenOnward
Halofuginone Phase II	Clinical Trial	Marc Blaustein	Halo Therapies
ISOFEN Phase II	Clinical Trial	Emilio Clementeni MD, PhD	University of Milan
Duchenne Muscular Dystrophy Conference (Portland, OR)	Research Conference	Chris James	RaceMD
New Biomarkers for DMD	Basic Science	Julie Saba MD, PhD	Children’s Hospital of Oakland, RaceMD
General Clinic Support	Clinic Support	Jett Clinic for Neuromuscular Disorders	Massachusetts

Hope for Gus is a 501(c)3. Our 990 is posted annually on sites like Charity Navigator.