First off, we have exciting news to share: donations to Prion Alliance will be matched 1:1 up to $75,000 through the end of June! As always, you can donate through Paypal, or mail us a check at Prion Alliance, PO Box 391953, Cambridge, MA 02139. Thank you for your support!
It is hard to believe that only six months have passed since our last update. As COVID vaccination rates rise, the world is changing right before our eyes, yet again — perhaps even heading towards that long-awaited “new normal.” We hope you are healthy and well, and that as summer deepens, the spirit of renewal finds its way to your doorstep in ways large and small.
On this end, it has been an interesting time. Followers of cureffi.org will be well acquainted with the “pivot” in our ASO collaboration with Ionis, publicly announced in April. Essentially, the previous front-runner ASO drug candidate, ION716, that was progressing towards human clinical trials has been replaced with a new ASO candidate. Still the same kind of drug, still with the same goal: lowering the amount of prion protein in the brain — but the exact molecule doing the job will be different. In the long run, we hope this is good news: the decision is motivated by a desire for the best possible drug to reach human patients. But this swap does set the clock back. While we had originally hoped that clinical trials could launch in 2021, 2022 now looks more realistic.
What can we make of this wrinkle? Drug development is a notoriously difficult business, and it’s a rare program that doesn’t experience at least one if not many such setbacks along the path to the clinic. Indeed, many observers would view this development as small potatoes. The program is still on track, Ionis has restated their commitment to our disease, and we’ve lost only a year or so in what was always going to be a multi-year process. But that’s not how it feels — not to us, and not to anyone for whom this disease is personal. If you’ve seen prion disease with your own eyes, if you fear that it may come for someone in your home, time matters. It’s the only thing that matters.
So we are here to reaffirm our commitment and optimism that things are moving forward. But we’re also here to affirm the anguish that any delay costs us, as a community. We feel it every day. Realism demands that we cultivate patience with the insanely complicated, demanding and imperfect process of drug development. But it is equally our moral imperative to ensure that we bring to the table only the exact minimum amount of patience required, and not a drop more. It’s a delicate dance.
We are searching, always, for ways to future-proof, and to leverage what we’ve learned so far into new and diverse weapons against prion disease. Our work with ASOs, even were it to suddenly cease today, has provided a scientific foundation from which we now see the future differently. Lowering prion protein in the brain works. So as we hustle to support ASO development as best we can, we are also exploring other drug types that may be able to achieve this end in different ways. Each will offer a unique set of pros and cons. Ideally, each will represent an independent shot on goal. While it’s early days, it feels good to charging ahead in new directions.
Concurrently, our lab is growing. We are now a team of five, looking to expand to six in the coming months. The growth is exciting, daunting, and necessary. We are lucky to have a great team, and we are also more aware than ever of what it takes to sustain a scientific enterprise over the long term. Thank you to everyone able to donate in support of our science — your donations remain an incredibly important piece of the endlessly complex logistical puzzle of running our lab.
Finally, a note on the home front. Kavari turned one in March, and is now toddling upright in an ever more convincing impersonation of a real person. When the whole family laughs, he laughs too, a bit wildly, and longer than anyone else. It feels like he’s joining the team. Daruka, in the meantime, is approaching her fourth birthday. The other day on our way to daycare, she informs me, “Today, I’m not going to school, I’m going to my lab.” “What will you do there?” I ask her. “Help my mouses,” she replies.
Thank you for everything.
Sonia and Eric