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OP-ED: Science lessons

Rare disease and the search for COVID-19 cures

Medical background of aligned coronavirus test tubes labelled Covid-19 with blood samples viewed from above full frame in a flat lay still life conceptual of the pandemic

DEPOSIT PHOTOS

At the end of March, our South Plainfield-based company, PTC Therapeutics, which is committed to finding new and innovative therapies for rare and ultra-rare disorders, marked its 22nd anniversary. Given this difficult time, it wasn’t really a cause for celebration, but rather reflection on our two-decade journey. That led me to see parallels to the challenges New Jersey and our nation face in the current battle against COVID-19.

All New Jersey residents and businesses have a tough road ahead, but based on our own company’s history over more than two decades, I can tell you this: In the fight against devastating disease, there can be hope even in the darkest times.

Twenty-two years ago we began efforts to develop effective treatments for patients with Duchenne muscular dystrophy, or DMD. Today, we have expanded that research to bring hope to patients living with other rare disorders. We have four drugs on the market and more than 800 employees globally. But no successful company in the business of drug discovery, investigation and delivery gets there without real failures and wrong turns along the way.

We often say that the path we’ve followed with our patients and their families has never been a straight line. That’s the nature of science. Sometimes, it felt like for every step forward, we took two steps back. For any company that tries to develop an idea into the successful delivery of a life-altering treatment, there is an understanding that most of the time, you will fail. Until you succeed.

In the COVID-19 context, as you hear the latest statistics on hospitalizations, deaths and treatments that offer initial promise but then fail, it is critical to remember that the path to fighting disease is never a straight line.

Moreover, while our company is successful, we have yet to be profitable. This is primarily because a large portion of our revenue is reinvested back into research and development, to continue to bring innovation, new treatments and better lives to patients and families.

And therein lies another lesson in the fight against COVID-19. We should all recognize the need for resources to fund this mission as we work every day to reach our goal. Legislators in New Jersey and nationally should understand that these past weeks have shown that you cannot effectively develop and deliver treatments after the crisis has hit. Investment in R&D, testing and clinical trials must be a priority in good times and bad. Having steady resources for further drug discovery is a priority.

The reality is this: Seeking cures, finding answers and saving lives takes funding. This means facing economic realities, to be sure, but never in a manner that erodes your commitment to the patients at the heart of your mission. All of us should understand that, for our work to succeed, governments, payers and private companies need to take steps and make investments not just to save some of the patients suffering from this disorder, but to protect all groups and demographics. No one – old, young, rich or poor – should be considered expendable.

Right now, our country and the world feel the pressure of an unprecedented challenge. In the rare disorder community, families face serious challenges every day. We’ve spent two decades getting to know patients and their families, and the tragedies they face in the absence of effective treatment. Every day, you feel as if you’re in a race against time as you pursue the goal of bringing life-altering drugs to patients.

How do you succeed? The key has been to make smart assessments of a given situation, define the best solution, then monitor progress and remain flexible enough to change course if needed. Then we begin the process again the very next day. In our nation’s current COVID-19 crisis, you’ll see teams doing this across New Jersey and around the globe. As in our work, this may seem like a long journey, and there will be steps forward and steps back. Success in confronting any disease is lived day by day and moment by moment.

We should take time to honor the spirit of all those who have dedicated their lives and resources to finding treatments in the fight against COVID-19. Then let’s roll up our sleeves and get back to work finding solutions for tomorrow.

Stuart Peltz is CEO of PTC Therapeutics Inc., a biotechnology company based in South Plainfield.

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