In light of the COVID-19 pandemic and the unprecedented changes it has brought with it, we wanted to share an overview of how we are carrying forward the mission of Prion Alliance during this unusual period. First and foremost, we hope you are safe and healthy. As of this writing, on April 21, 2020, the COVID-19 pandemic has claimed more than 150,000 lives (that we know of), infected nearly 2.5 million people (that we know of), and redefined daily life for billions more. It’s extraordinary and sobering how quickly the world has changed before our eyes.
Before I talk about our science and how we are working to carry it forward, I want to give a quick shout-out to our colleagues at Broad, MGH and beyond who are directly applying their skills to addressing the pandemic — be it by creating COVID-19 testing capacity, performing research on the virus, or seeing patients in the clinic. We’re proud to know so many people who are stepping up amidst the whirlwind to generate data and provide care — both of which are so desperately needed at this time.
For Eric and I, as for many, contributing to the pandemic response means staying away from our workplace to help protect those who need to be there. So the key question becomes how to best keep our quest for a prion disease therapeutic moving forward while working remotely, which we have now been doing for about a month.
If you’d asked me before I was a scientist, I might have said that there was something inherently contradictory about asking a biomedical researcher to work from home. Isn’t the whole point to be in the lab? Well, yes and no, as it turns out.
The answer is most clearly yes for animal studies — the most critical feature of our drug development work, at this stage. These studies aren’t substitutable with off-site activities, and have long timelines that can’t be paused — and in the meantime, they’re often rate-limiting for generating the data that could eventually get us to clinical trials. Of the many kinds of science we’ve done to date, this is the domain where disruption is the scariest. But we’ve been fortunate to be partnered on these studies with three amazing teams: the Broad comparative medicine staff, the neuroscience team at Ionis Pharmaceuticals, and our scientific collaborators at McLaughlin Research Institute. All three teams are finding ways, as essential personnel, to safely navigate this crisis while keeping key research moving forward.
It’s been humbling and inspiring to see just how deeply prepared for emergencies these scientists already were, and had to be. We are so grateful for their foresight, dedication and resilience.
We’ve gotten a number of questions about whether Ionis is still onboard to develop an ASO for prion disease. Rest assured that they are. Ionis has publicly confirmed its commitment to its programs (you can read a company statement on Ionis’s website), and as described in this message to the prion disease community, that commitment includes continuing to drive forward towards a prion disease ASO. Mission-critical activities continue at the company, and everyone is focused on how to move forward efficiently, without losing time to the pandemic.
There are, of course, other aspects of Eric’s and my research that we simply aren’t able to advance at this time. All of our cell culture and small molecule experiments have been paused. While this is disappointing, we take comfort in the fact that these experiments were exploratory, and weren’t rate limiting for drug development. On the clinical side, our clinical research study at Mass General Hospital has paused study visits, as the hospital seeks to minimize exposure risk while brings all possible resources to bear on COVID-19 care and testing. We’re grateful to have had the opportunity to build our cohort and momentum before the pandemic struck, and look forward to bringing back participants, both old and new, once it’s safe to. We’re also fortunate that we had just recently collected, analyzed and written up a first chapter of data from this study. When Eric and I discussed when to publish a first analysis from what we hope will be a long-running study, he made the case that you should share early and often, in part because you just never know what obstacles might crop up down the road. I don’t think he was picturing the circumstances we now find ourselves in — but he was right.
Off-site, Eric and I are doing what we can to support the people supporting the animals. We’re also working, more broadly, on all of the non-labwork activities that make the right labwork possible: planning experiments, analyzing and visualizing data, writing up results to share them with the world. We’re diving into long-postponed literature searches that will inform our future directions. And we’re in communication every day with collaborators at various sites in Cambridge/Boston and beyond. Some are able to work in their labs right now helping to move our joint science forward. Others are in touch to devise next steps for whenever circumstances allow.
In summary, like huge numbers of people around the world, we’re adapting as best we can. While we miss being in a building buzzing with scientific activity, we’re incredibly fortunate to have the ability to be productive off-site for now, and to know that our most critical studies are being capably carried forward. As the months progress, we, like everyone else, will have to continuously reassess what makes the most sense for our work and mission. But know that the mission hasn’t taken a backseat — we’re still as focused as we’ve ever been on advancing an effective therapeutic for prion disease.
Stay safe and well, and thank you for your support. We can’t fully predict how the world will have changed when we finally put this pandemic behind us, but it seems reasonable to assume that there are hard times to come for virtually every nook and cranny of the global economy. We have always been a scrappy little enterprise, and we will continue to scrap our way forward. Thank you for being part of this journey.