Dear friends and supporters,
We have been blessed. 2016 has been an amazing year for our quest for a cure, and we’re writing to let you know what we’ve been up to and what’s on the agenda for the year ahead.
First, one piece of time-sensitive news. An anonymous donor has generously offered to match your donations 1:1, up to $10,000, through the end of the year. So if you’re willing and able, visit prionalliance.org/donate to make a contribution via PayPal, or send a check to:
Prion Alliance PO Box 391953 Cambridge, MA 02139
(If you’ve made a donation in the past couple of weeks, don’t worry – all gifts received since Thanksgiving will be counted towards the match. Also, thank you!)
Our quest to cure prion disease turns five years old today, we have been humbled and inspired by the amount of support and interest that our journey has generated.
This January, our study on the lifetime risk of different genetic mutations in prion disease came out, and has already led to a dramatic change in prognosis for some people. In February, Sonia was invited to speak on a panel with President Obama at the White House Precision Medicine Summit. Over the course of the year we’ve been featured in the Boston Globe, CBS Evening News, and NPR. But most importantly, back in the lab, we are finally living the dream that we set out to achieve five years ago: we are working every day on developing therapeutics for prion disease. The intellectual support we’ve received at the Broad Institute is unsurpassed, and we are now actively conducting high-throughput screens by several different methods in an effort to discover small molecules effective against prion disease.
Even as our work on drug discovery has moved forward, we’ve also come to realize that finding a therapeutic molecule is only, at best, half the battle. In order for a therapy to reach clinical trials and eventually get approved, we need biomarkers – things we can track in a living patient to tell whether a drug is having its intended effect. We’ve therefore launched an effort to collect and analyze cerebrospinal fluid (CSF) from hundreds of individuals, and we have been blessed by the generosity of patients who have donated their CSF for research, and of scientific collaborators who have shared these samples with us.
And there’s one other wrinkle. The way that clinical trials have traditionally been done, a drug must show a direct clinical benefit in order to be approved. In prion disease, that’s a big problem, because by the time patients develop symptoms and get diagnosed, their disease is often very advanced. Many neurons have been irreversibly lost, and some patients have little quality of life left to preserve. Animal experiments have shown, time and again, that some antiprion therapies are ineffective after symptom onset but can increase survival time by 2- to 4-fold if administered before symptoms or, particularly, before prion infection begins. It’s likely that in order to truly benefit patients, a drug will need to be given to healthy presymptomatic patients – people like Sonia. But because these people are not sick, it’s impossible to show a direct clinical benefit. Instead, we believe that biomarkers must become the basis not only for monitoring whether a drug has is intended effect, but also for deciding whether a drug should merit approval. This fall, Sonia was featured in MIT Technology Review’s Innovators Under 35, and she explained why clinical trial reform is so important for bringing prion disease to heel.
Looking forward to the year ahead, we’ll be stepping up our efforts both to discover a drug on one hand, and to pave the road to clinical trials and approval on the other hand. Your donation will go to support all of these efforts. And if you’re looking for other ways to support us, remember you can shop for us on Amazon Smile, and buy some swag from the Prion Alliance Zazzle store.
Thank you all so much for your love and support along this journey – we couldn’t do it without you.
Eric & Sonia