Deep into 2020, the strangeness of this year has not let up. The COVID-19 pandemic continues to define daily life around the world, and we can only begin to glimpse the path by which science may lead us out of this. We hope that you are staying healthy, staying safe, and sometimes, somehow, finding ways to stay connected to the people you love and the things that make you happy.
We’ll share a bit about our past several months, but first, an announcement: donations to Prion Alliance will be matched 1:1 up to $20,000 through the end of 2020! Especially in this year of profound uncertainty, from the bottoms of our hearts, thank you to our matching donors for this incredible gesture of support.
Donations can be made through PayPal, or by check at:
P.O. Box 391953
Cambridge, MA 02139
Worth noting: donations to charities like Prion Alliance will work a little differently this year. Under the CARES Act, part of the federal government’s pandemic relief program passed in March, taxpayers will be able to take a deduction of up to $300 for charitable donations made in 2020 when filing taxes in spring 2021, even if you take the standard deduction rather than itemizing your personal deductions.
For all of the challenges of this year, we’re proud to report that the fight against prion disease has continued. Most tangibly, our ASO program has reached some exciting new milestones. As many of you know, for years we’ve been working with Ionis Pharmaceuticals to help develop an antisense oligonucleotide, or ASO, drug for prion disease. We are thrilled to share that ION716, the human version of this drug, is now officially listed in Ionis’s pipeline. And in September, Dr. Anne Smith, who leads clinical development for ION716 at Ionis, announced to the patient community that the goal is for first-in-human trials to launch in 2021. You can hear Dr. Smith give an eloquent, lay-friendly overview of the whole ASO program in this video recorded by our terrific allies at the CJD Foundation.
There is still lots of work to do — and in some ways, the closer we get to the clinic, the higher the stakes become. But it’s worth taking a moment to reflect on how far we’ve come. ION716 is poised to become the first drug ever developed and tested specifically for prion disease with a major pharmaceutical partner. In the meantime, we’re continuing to work to make it possible for this drug to be tested not just for its ability to treat active disease, but for its ability to delay or prevent disease in healthy people at risk. If we’re successful in forging this preventive path, that will be another first — one that may be a useful model for other diseases, too.
Beyond this headlining news, we’ve been busy setting up new projects and collaborations in the lab, and look forward to sharing more about these as they develop. We’ve also managed to continue to spread the word about our work at virtual conferences, summits and grand rounds sessions. A special focus this year has been expanding our outreach to the neurology community — to make the most of coming opportunities, we’ll need their help improving prion disease diagnosis, and updating the information that reaches patients and families about potential trials, treatments, and genetic testing. If you’d like to hear a bit of what this sounds like, or are just looking for a friendly voice to brighten up another day of social distance, you can listen to Sonia’s two-part interview with the “Neurology Podcast,” which aired on Sept 28 and Oct 5, and is available on Apple Podcasts (1, 2) or Spotify (1, 2).
We miss you. Our quest draws its energy from our community — as do we, as people — and as much as we have to be grateful for in 2020, there’s no denying that this has been a strenuous year. Our baby Kavari, now eight months old, shows all signs of being as social a creature as his big sister and parents before him. It’s poignant to see how much joy he draws from the few faces he’s able to see.
May 2021 be a year of hope and progress, for all of us.
with love, Sonia and Eric