The International Space Station.
The Human Genome Project.
The cure for polio.
Great feats that required different scientific breakthroughs but shared one common attribute.
Focused and intentional collaboration.
The hardest problems can only be solved this way.
Finding treatments, and ultimately cures for Facioscapulohumeral Muscular Dystrophy (FSHD), are complex problems and thus require no less focus and collaboration. In fact, collaboration started about 150 years ago.
Developing treatments for FSHD only recently started in 2019 when the first drug designed to counteract DUX4 expression entered clinical trials. But getting there started in the 1870’s, when FSHD was first noted as a disease by two French physicians following a family affected by it. The DUX4 gene was finally discovered in 1999, found to be expressed and toxic in 2007, and in 2010 the genetic mechanism causing its expression was decoded. In 2012, the gene most frequently mutated in FSHD2 was identified and seven years later, in 2019, research finally reached the clinic.
Creating a better world for everyone affected by FSHD has, and always will, require teamwork, partnerships, and collaboration across the globe. Scientific research in FSHD must build on the prior 150 years to drive innovation and better outcomes in clinical research. The clinical research pipeline, as of 2023, has grown to more than twenty biopharma companies actively involved. This is promising because we will need multiple types of therapies to ensure effectiveness in a diverse and varied patient population. We will also need to ensure patients can access these therapies once approved in a timely and cost-effective manner. Nobody wants to imagine a scenario where a therapy that could help is available, but out of reach.
Rising to the occasion demands an entire army of stakeholders working together and supporting each other toward common goals. Embracing the benefits of cooperating together and willing to accept the risks that come with breaking down silos that derail collaboration. Language, culture, time, data and resource-sharing are among the ever-present barriers to effective partnerships.
Researchers, clinicians, biopharma companies, regulators, payers, and most importantly, patients, families and those who advocate for them. All must work together with intentionality.
A Japanese poet, Ryunosuke Satoro, beautifully sums it up:
“Individually, we are one drop. Together, we are an ocean.”
In May of 2022, an initiative called Project Mercury was created by the FSHD Canada Foundation and the FSHD Society. It’s a platform purposefully designed to energize, focus and facilitate the intentional collaboration needed to address two critical challenges that could prevent people affected by FSHD from getting effective therapies. These challenges are:
- Clinical trial readiness – FSHD clinical trials of promising therapies could fail either due to lack of patients participating in them or not enough sites to conduct multiple trials globally.
- Patient access – patients not being able to access approved therapies on a timely and cost-effective basis.
The name underscores how it works:
Project = focused, goal-oriented and timely execution.
Mercury = the Roman God of speed. Because the patient is waiting.
Put it together and Project Mercury is the collaboration platform designed to speed effective therapies in FSHD.
Many of us, people and organizations, often have a natural inclination to go it alone. While this do-it-yourself spirit can help you move forward, adding in the element of collaboration will make you unstoppable.
Project Mercury is a call and a platform for:
- Researchers to share data and insights.
- Biopharma companies to engage patients, caregivers and healthcare providers more effectively in the clinical research process.
- Regulators and payers to better understand the health and financial burdens on patients and families.
- Patient advocates to super-charge their capacity for driving clinical research and speeding the delivery of therapies.
- Everyone to better understand a human being’s journey and experience living with FSHD.
Project Mercury is an ambitious initiative but with intentional collaboration and a willingness of stakeholders to share insights and resources, the global FSHD community will get this done!