By Rich Horgan
“Go home and love them for as long as you can,” are difficult words to hear from a doctor after learning about a loved one’s rare, fatal disease diagnosis. When my brother, Terry, was diagnosed with Duchenne muscular dystrophy (DMD) – a rare, fatal muscle-wasting disease – we could not accept his diagnosis as a death sentence.
Years later, when I was attending Harvard Business School, I began to network with leaders in health care and biotechnology and soon realized that a treatment for my brother’s DMD could be possible through new CRISPR-based gene therapy, a process to edit an individual’s DNA to correct a problem in their body. I founded a nonprofit that became Cure Rare Disease and gathered an interdisciplinary team of academics, scientists and clinicians who together could create and dose this biotech therapy.
While other organizations working to develop a cure for rare diseases are for-profit enterprises, I opted to run Cure Rare Disease as a nonprofit, one that focuses on the patient experience and keeps families closely involved. Today, we have grown into an international organization with a network of families across North America, including the LaSaracina family of Warren, and collaborators around the globe.
Thanks to this collaborative effort, we are planning to dose Terry with a treatment in the coming months once we’ve completed final tests and received approval from the U.S. Food and Drug Administration. He’ll be one of the first people in the world to be dosed with a CRISPR treatment. This form of gene therapy seeks to stop the impacts of DMD by editing Terry’s DNA to correct the amount of a specific protein his body produces. Tests to date have been promising, and we expect that the treatment will stop the weakening of his body’s muscles – especially those of his lungs and heart – and may return some strength and function to his arms, heart and diaphragm.
Terry’s experience and those of Cure Rare Disease families are not unique. In fact, according to the National Institutes of Health, 1 in 10 Americans live with a “rare” disease – which include many kinds of cancers, skin diseases and neurological diseases. Even “ultra-rare” diseases can impact tens of thousands of Americans.
In the case of DMD and other types of muscular dystrophy, specific differences in an individual’s genetic code makes their disease unique and can make seeking out clinical trials and hope difficult. This is where we want to be the solution. Our work is personalized to the patient, but our framework is being tailored to the other children in our drug development pipeline. We’re committed to developing a treatment specific to them that will treat – and hopefully cure – their rare disease.
As you can imagine, this is an extremely complex and expensive process. It involves years of research, testing and specialized manufacturing. But we fully intend on being able to continue through our nonprofit structure – if we can get help.
While we’re always open to new donors joining our thousands of existing donors, we’ve launched “Strive for Five” as a means to reach one million people and ask them each to give just $5. This $5 million campaign is a grassroots effort that calls for not only a donation but includes an ask to share the campaign with five friends who will do the same. If you are interested in keeping the movement going in your community, consider donating at www.cureraredisease.org/five, and asking for support from your friends via social media, email, text or by sharing Terry’s story.
Rich Horgan is the founder and president of Cure Rare Disease, a nonprofit biotechnology research organization on a mission to develop precision medicine for rare diseases.
