Today, PPMD announced a $2.2 million dollar grant to Dr. Jerry Mendell, Dr. Louise Rodino-Klapac (co-PI), and Nationwide Children’s Hospital. 

Our purpose in awarding this grant is to open and accelerate the field of gene therapy – a strategy that seemed impossible as a potential treatment for Duchenne as far back as the 1990s. If successful, it will provide systemic delivery of a microdystrophin construct that targets both skeletal and cardiac muscle achieved through one IV injection. The FDA has agreed, it is time, and has approved the protocol and in mid- to late-2017, trial participants with Duchenne could receive systemic virus delivering the microdystrophin gene – a major milestone and step forward for Duchenne.

This grant is the first project of a new Gene Transfer Initiative PPMD is launching this month that will include the work Dr. Mendell and Dr. Rodino-Klapac are doing, as well as a deeper exploration of CRISPR/Cas9 technology as a potential therapeutic approach to treating Duchenne.

We will learn a lot from this study at Nationwide: questions around the production of sufficient virus, understanding and preventing an immune response, and how to deliver systemically (throughout the entire body). This grant opens doors to potentially treat almost all people with Duchenne and opens the doors for all gene transfer strategies. And CRISPR/Cas9, which could permanently correct DNA, will need to utilize this same AAV9 virus as a delivery method. Though it will take some time, we remain committed to changing the Duchenne landscape.

We believe in collaborating across stakeholder groups – patients, caregivers, researchers, clinicians, companies, and regulators – working together to move exciting new research strategies forward. On the heels of an extremely successful holiday campaign focused on exploring CRISPR/Cas9, we believe this is the moment to commit to Nationwide Children’s gene therapy work as part of our larger Gene Transfer Initiative. We remain hopeful that gene transfer will be part of the combination of therapies that halts disease progression. We plan to explore ways we can fill gaps and overcome hurdles to speed the development of this promising science.

We did not do this alone.  It takes a village and we are deeply grateful to our partners: Team Joseph, Team Saij, The Fund for Pete’s Sake, Rashad’s family, and the Nicholoff family.

It is a major step and a good one.  Fingers crossed and prayers to the universe.  The field will be opened and we believe this paves the way for all.

PPMD’s Press Release: 

Parent Project Muscular Dystrophy Awards Nationwide Children’s Hospital $2.2 Million Grant to Explore Gene Therapy in Duchenne Muscular Dystrophy

HACKENSACK, NJ – January 6, 2017 – Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), today announced a $2.2 million grant to be awarded to Jerry Mendell, MD, PhD; co-PI Louise Rodino-Klapac, PhD; and Nationwide Children’s Hospital. This grant will support Drs. Mendell and Rodino-Klapac and their team’s work in exploring gene therapy as a potential treatment for Duchenne.

Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in every 5,000 live male births.

This $2.2 million grant marks the largest single award in PPMD’s almost 23 years. PPMD has a long history of supporting early-stage, innovative research, providing funding at a critical moment in a therapy’s development. Gene therapy has been explored for years as a possible therapeutic approach to treating Duchenne. Only recently, though, have advances in science and technology made it seem like a viable treatment option for Duchenne.

According to PPMD founding President and CEO, Pat Furlong, “Gene therapy has seemed like the future for quite some time, almost like something out of science fiction. Today, the future is here. Dr. Mendell and Dr. Rodino-Klapac have unique experience in this field, bringing with them a breadth of knowledge and first-hand experience with people living with Duchenne. They, and the team at Nationwide Children’s Hospital, have spent years exploring the potential of gene therapy in Duchenne and PPMD is thrilled to provide support for the next stage in this exploration.”

Dr. Mendell and Dr. Rodino-Klapac on learning of the award, said, “We would like to thank PPMD for funding this exciting trial for Duchenne muscular dystrophy. Based on our experience, we believe we have outlined a safe approach for delivery of a gene with the potential to make a clinical difference to change the lives of people with this devastating disease. We have laid the foundation for doing this trial over the past decade and we are eager to get started. Our program is further enhanced by collaboration with Anne Connolly at Washington University.”

Dr. Mendell will build on his previous work in Spinal Muscular Atrophy (SMA), micro-dystrophin, and SGCA (sarcoglycan alpha) gene therapy trials. He and Dr. Rodino-Klapac will conduct a Phase 1/2a trial of systemic gene therapy delivery of microdystrophin in patients three months of age or older, with the primary outcome being safety.  Functional outcomes will be measured for all patients using the Bayley-III Gross Motor Scale.

The PPMD grant will support the manufacturing and clinical costs of the study for trial participants. The award will be milestone driven, based on regulatory interactions, vector manufacturing, and patient dosing and follow-up. Dr. Mendell and Dr. Rodino-Klapac have already had successful regulatory interactions laying out a path to bring this study to clinical trial and vector manufacturing is on schedule. The trial is poised to begin in mid-to-late 2017.

Later this month, PPMD is launching a Gene Transfer Initiative that will include the work Dr. Mendell and Dr. Rodino-Klapac are doing, as well as a deeper exploration of CRISPR/Cas9 technology as a potential therapeutic approach to treating Duchenne. “PPMD believes in collaborating across stakeholder groups – patients, caregivers, researchers, clinicians, companies, and regulators – working together to move exciting new research strategies forward. On the heels of an extremely successful holiday campaign focused on exploring CRISPR/Cas9, we believe this is the moment to commit to Nationwide Children’s gene therapy work as part of our larger Gene Transfer Initiative which we look forward to rolling out in the coming weeks,” says Furlong.

The $2.2 million grant to Nationwide Children’s Hospital, Dr. Mendell, and Dr. Rodino-Klapac was made possible in part by the generous support of additional Duchenne foundations, including: Team Joseph, Team Saij, The Fund for Pete’s Sake, Rashad’s family, and The Nicholoff family. PPMD is grateful for their partnership.

To learn more about PPMD’s research investments, please visit the website.

About Parent Project Muscular Dystrophy

Duchenne is a fatal genetic disorder that slowly robs people of their muscle strength. Parent Project Muscular Dystrophy (PPMD) is the largest most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne muscular dystrophy—our mission is to end Duchenne.

We invest deeply in treatments for this generation of people affected by Duchenne and in research that will benefit future generations. We advocate in Washington, DC, and have secured hundreds of millions of dollars in funding. We demand optimal care, and we strengthen, unite and educate the global Duchenne community.

Everything we do—and everything we have done since our founding in 1994—helps people with Duchenne live longer, stronger lives. We will not rest until every person has a treatment to end Duchenne. Go to www.ParentProjectMD.org for more information or to learn how you can support our efforts and help families affected by Duchenne. Follow PPMD on Facebook, Twitter, and YouTube.

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