Rich Horgan, founded Cure Rare Disease to find a cure for his brother’s Duchenne muscular dystrophy and for other patients fighting rare, fatal diseases.
Cure Rare Disease, a nonprofit biotechnology research organization on a mission to develop precision medicine for rare diseases, has achieved a major fundraising milestone, with over $400,000 in donations received through a collaboration with a generous community of Twitch creators.
Cure Rare Disease is directing the funds raised by the online gaming community to its research program, which is developing customized therapeutics for those who have been diagnosed with rare, genetic diseases with no treatment or cures, starting with Duchenne muscular dystrophy (DMD).
You have a very personal reason for having founded Cure Rare Disease, could you share with us this story?
The story behind Cure Rare Disease is very personal. My younger brother, Terry, has Duchenne muscular dystrophy (DMD). It’s a genetic disease that causes muscles to degenerate over time, including cardiac and pulmonary muscles essential for life. Due to DMD, Terry has limited mobility but continues to be in good spirits as he fights against this invisible enemy. DMD is one of over 7,000 rare diseases impacting over 30 million Americans, and is ultimately a fatal disease that has no effective treatment for the population, let alone a cure. In fact, only 5% of rare diseases have any treatment.
I founded what is today Cure Rare Disease to develop a medicine for Terry and others with rare diseases like DMD because, as a family, we were not satisfied hearing from most doctors that all we could do was go home and love him with what time we were given. And so, we are changing the paradigm of how rare and ultra-rare diseases are treated. We are showing that developing individualized medicines for rare diseases like DMD is not only possible but also an effective way to combat diseases which are too rare to attract traditional drug development interest. For that reason, and others, Cure Rare Disease is structured as a nonprofit biotechnology company that measures itself based on patients helped rather than profits generated.
You grew up playing video games with your brother Terry, what games did you both play?
Very much so! Growing up Terry and I were – and still are – huge fans of the Star Wars titles including the MMO Star Wars Galaxies along with the classic FPS titles like Battlefield. My brother has since moved into titles like Escape from Tarkov, though we still both enjoy classic games. Video games are an amazing equalizer and outlet for both people impacted by rare diseases as well as for people who just love to game.
What inspired the idea of using the power of the gaming community to raise funds?
A rare disease diagnosis often comes with physical challenges, so given our long history of gaming, it became clear that gaming could provide an equal playing field for people of all abilities. Since the gaming community has long been so welcoming of people with rare diseases like Terry, who can’t play a game of basketball like others may be able to, it felt like a natural fit for raising awareness and support for our mission. In fact, there are many creators who are impacted by rare diseases. We hope to help make the community an even more welcoming place for people impacted by rare diseases to share their stories and feel welcomed.
Thanks to platforms like Twitch and the emergence of streaming competitions and creators, we felt that fundraising within this welcoming community was an opportunity we could not miss.
Could you share the concept of how gaming ambassadors can assist with the process of raising funds?
To inspire the gaming community to raise money for the cause, Cure Rare Disease created an ambassador program and sought out up-and-coming talent who are Twitch partners and affiliates. The ambassadors, who entertain their community with a variety of content, share a passion for Cure Rare Disease’s mission with their communities via live streams, social media posts, and Discord chats to inspire donations.
Our ambassadors also get exclusive access to Cure Rare Disease merchandise, in-game codes, chances to win gaming systems, and other fun perks. Moreover, they are part of a community that is effecting real change for patients impacted by rare diseases for which there are no cures. We think it’s very powerful for creators, from all backgrounds, to have a hand in literally changing the future for not just one patient, but their family, their community and for the rare disease community more broadly.
Some gamers have been ultra-successful having raised significant sums of money, could you share some of these success stories?
We’re fortunate to have a great network of ambassadors who have all pitched in to raise money to develop individualized medicines that will allow other gamers to keep on playing. Our aim is not simply to apply a band-aid solution, but rather to treat the disease at its root cause – a permanent fix to fatal diseases – through the use of CRISPR and other innovative technologies. Our ambassadors play a wide range of games and have their own charity streams or participate in organized events, like our upcoming Game for Cures streaming marathon from July 16 – 25th 2021.
In terms of success stories, we’ve had creators fundraise at varying levels – from Charitymon Project raising several thousand dollars, to Catsen who raised $7,020, and to Moist Critical who topped $150,000. Every dollar raised brings more awareness and more support to families battling for their loved one’s life.
Cure Rare Disease will soon be hosting its own charity streaming events, could you share some details on upcoming events?
Our upcoming charity streaming marathon, Game for Cures, is set for the weekends of July 16-18 and July 23-25. The six-day, two-weekend marathon will benefit Cure Rare Disease and our mission to develop therapies for kids fighting fatal diseases. We have a number of great streamers on board. People interested in learning more should visit www.cures.gg and interested streamers can sign up at this link. We are offering fun incentives for streamers who raise differing amounts. It’s really important to us that everyone who participates is recognized and thanked for their efforts!
Most of us would have felt a sense of hopelessness at the situation, yet you took the opposite approach and met with researchers, clinicians, and regulatory experts to fully and deeply understand all the work that was being done around Terry’s disease. What made you believe that you could make a difference?
I don’t come from a particularly wealthy or influential background – I put myself through business school. But nonetheless, a family who is impacted by rare disease only has two options: fight or stand idly by. Standing by wasn’t an option for us. So, I did what I could do in the place where I was. Fortunately for me, the place where I happened to be at the time was Harvard Business School. I began to think bigger, and as I made connections with these world-renowned researchers and clinicians, I realized that we have a golden opportunity to create an organization – a movement – to find a cure. We needed to rethink how medicines are developed for small populations (rare and ultra-rare diseases) since the traditional drug development paradigm relies on large populations to spread high development costs around (high cholesterol and Lipitor, for example). However, by definition, rare diseases impact fewer patients but are no less important than chronic conditions like diabetes. In realizing that it was possible to usher a life-saving drug from creation, development and through approval – even for one person – I knew there could be an answer here for not only my brother but also thousands of others impacted by rare diseases. Thus, Cure Rare Disease was born.
A drug using CRISPR technology is being developed, could you share some details on this?
CRISPR technology is a gene editing tool that allows us to make changes in a patient’s DNA including the upregulation of missing proteins. In the case of Terry and those with DMD, we will get the body to produce more of a missing protein (the dystrophin protein for DMD, specifically) similar to one that Terry lacks, which causes the loss/weakening of muscle tissue.
We’ve seen great success on correcting patient cells in a dish as well as in animal models and are expecting that Terry will be one of the first DMD patients who will be dosed with a medicine using CRISPR technology. We are both excited as well as extremely cautious.
Beyond Terry, we have 15 drugs under development – most of which will use CRISPR to help the patients impacted. We’re excited by the idea of proving this framework out and scaling up from here. We’re able to move quicker because of the streaming community and the incredible work they do!
Is there anything else that you would like to share with our community?
Cure Rare Disease, and our dozens of families waiting for an individualized therapy, are so grateful to the streaming community for their help and support. Without their generosity, we would not be where we are today.
For readers interested in learning more about our streaming program, they can visit us online. Similarly, we accept donations of all sizes online as well at https://www.cureraredisease.org/donate. Lastly, we invite creators to reach out and participate – it takes a community to win the battle against disease and we welcome all to join the fight. This is truly a cause where one person can make a world of difference.