Gene therapy for rare diseases continues to make headlines. The latest clinical trial involving gene therapy is one for Duchenne muscular dystrophy (DMD). That trial recently dosed its first patient at the Nationwide Children’s Hospital in Columbus, Ohio.
Interestingly, the trial is being partially funded by patient advocacy groups. Parent Project Muscular Dystrophy (PPMD), along with smaller DMD advocacy groups –Team Joseph, Team Saij, The Fund for Pete’s Sake, Rashad’s family, and the Nicholoff family – have provided $2.2 million (U.S.) towards this trial being led by Drs Jerry Mendell and Louise Rodino-Klapac.
In a news release, Pat Furlong, president and chief executive officer of PPMD said, “This is a monumental day in the Duchenne community, a day that 24 years ago when we started Parent Project Muscular Dystrophy, we didn’t think possible. We are so grateful to the drive and passion of Dr. Mendell, Dr. Rodino-Klapac, and the Nationwide Children’s team. We will learn a lot from this study, including answers to questions around the production of sufficient virus, understanding and preventing an immune response, and how to deliver gene therapy systemically. But for now, we are celebrating the first dosing of a Duchenne patient with microdystrophin gene therapy and we are celebrating the bravery of the little boy and his family participating in this breakthrough trial.”
Interestingly, the advocacy groups are not the only key stakeholders in this trial. So is the pharmaceutical industry. Sarepta Therapeutics, Inc., the company that developed the first FDA approved exon-skipping drug (Exondys 51) for DMD patients, is also a financial backer of the trial.
Douglas Ingram, Sarepta’s president and CEO said, “Sarepta is the world leader in precision genetic medicine to treat DMD, spanning RNA-targeted therapies, gene therapy, and gene editing. In service of our mission to profoundly improve the lives of those with DMD, we look forward to the preliminary results from this study by mid-2018 and, results permitting, stand ready to aggressively invest in bringing this therapy to reality and to the DMD community.”
These advocacy groups are not the only ones investing in clinical trials and partnering with the pharmaceutical industry. And if done well, it can provide a lot of funds for the advocacy group in the future. For example, the Cystic Fibrosis Foundation invested millions of dollars towards the development of Vertex Pharmaceuticals’ drug Kalydeco. In return, the advocacy group was given a percent of the royalties after the drug was approved. In 2014, the Cystic Fibrosis Foundation sold their rights to future Kalydeco royalties for $3.3. billion.
Yes, you read that correctly — $3.3. billion.
The smart advocacy groups are not only helping patients manage their symptoms and educating the public about specific diseases, they are also investing in drug development and clinical trials. If done correctly, it is a good investment.
Is your advocacy group investing wisely?