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:

Expansion of Department of Defense Grant (Cardiac)General SupportDr. Elizabeth McNallyNorthwestern University
DMD ClinicGeneral SupportN/AUniversity of Massachusetts, Worcester
Pandemic relief for DMD FamiliesGeneral SupportHope For GusN/A
CRISPR/CasPreclinical drug developmentEric OlsonUniversity of Texas Southwest
DMD ClinicGeneral SupportN/AUniversity of Massachusetts Memorial

Our Past Funding:

GalGt2 Gene Therapy TrialPreclinical drug developmentDr. Kevin FlaniganColumbus Children’s Hospital
VBP15 Dissociative Steroid DrugClinical trialEric Hoffman, PhDReveraGen
Repeat Dosing Gene TherapyPreclinical drug developmentDr. Keith FosterUniversity of Reading
Folistatin Gene TherapyPreclinical drug developmentDr. Jerry MendallNationwide Children’s Hospital
Program to Consolidate ResearchResearch SupportN/ACharley’s Fund
Myomics Drug ScreeningPreclinical drug developmentBrian Tseng, MD, PhDMassachusetts General Hospital
Gene Therapy ResearchPreclinical drug developmentJeff Chamberlain, PhDUniversity of Washington
Laminin 111Preclinical drug developmentBradley Hodges, PhDProthelia
Nutraceutical (THI), Skeletal Muscle FunctionPreclinical drug developmentHannele Ruohola-Baker, PhDUniversity of Washington
Zebra Fish Screening of FDA Approved PharmaceuticalsPreclinical drug developmentDr. James DowlingUniversity of Michigan
Preparation of Halofuginone for Phase I/II Clinical TrialPreclinical drug developmentMarc BlausteinHalo Therapeutics
Carmeseal (P188), Cardiac and Skeletal Muscle FunctionPreclinical drug developmentBruce Markham, PhDPhrixus Pharma/Coalition Duchenne
Development of Dystrophin Independent TherapyPreclinical drug developmentLouis Kunkel, PhDBoston Children’s Hospital/Jett Foundation
Anti-fibrotic (Relaxin) Skeletal Muscle and Diaphragm HistologyPreclinical drug developmentJeffrey Chamberlain, PhDUniversity of Washington
Nutraceutical (Quercetin) Respiratory and Cardiac FunctionPreclinical drug developmentJoshua Selsby, PhDIowa State University
Nutraceutical (THI), Skeletal Muscle FunctionPreclinical drug developmentDawn Lowe, PhDUniversity of Minnesota
Duchenne DashboardProject management softwareCarlo Rago, PhDOpenOnward
Halofuginone Phase II Clinical TrialMarc Blaustein Halo Therapies
ISOFEN Phase II Clinical TrialEmilio Clementeni MD, PhDUniversity of Milan
Duchenne Muscular Dystrophy Conference (Portland, OR)Research ConferenceChris JamesRaceMD
New Biomarkers for DMDBasic ScienceJulie Saba MD, PhDChildren’s Hospital of Oakland, RaceMD
General Clinic SupportClinic SupportJett Clinic for Neuromuscular DisordersMassachusetts

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