Dear friends and supporters of Prion Alliance:
As 2018 draws to a close, we are thrilled to share with you an update on our progress towards a drug for prion disease, and our vision for the road ahead.
This July, we announced publicly an effort we’ve been engaged in full force for the past few years: the development of an antisense oligonucleotide (ASO) drug for prion disease. While we’ve always been optimists in general, we can now say for the first time that there is a specific therapeutic strategy about which we are optimistic, and that we believe could yield the first effective drug for prion disease.
We have partnered with Ionis Pharmaceuticals, the company that recently developed an approved ASO drug for spinal muscular atrophy — the first disease-modifying drug for any neurodegenerative disease — and that has ASOs for several other neurological conditions currently in clinical trials. Their experience in this area has shown that ASO drugs can be safety delivered to a wide swathe of the human brain, remain stable for months, and can specifically reduce the amount of one targeted protein by ~40%. Building on Ionis’s success in this area and the inherent “building block” nature of ASOs — these drugs are made of the same A, C, G, and T bases that make up our DNA — we believe it should be possible to develop a drug that will reduce prion protein in the human brain and delay the onset of prion disease. To learn more about why we’re optimistic about this strategy, read Sonia’s announcement of the collaboration.
This summer, we spoke at CJD Foundation’s annual conference in Washington, D.C. to announce this project to the patient community. The response has been tremendous. We have been gratified to see that enrollment in PrionRegistry.org, the online research registry that we launched jointly with CJD Foundation and CJDISA last year, has steadily increased, with 166 individuals currently consented and enrolled. Our clinical research study at Massachusetts General Hospital has now seen its first 32 participants and is continuing to enroll. We continue to be blessed with an engaged, motivated patient community that wants a drug and is more than happy to participate in research in order to make it happen.
Meanwhile, we soon face a turning point in our own lives and careers: we recently received approval from our committees to defend our dissertations and receive our PhDs in May 2019! This is an exciting and welcome step forward after five years of intensive research. It’s also a daunting transition. ASOs could reach clinical trials in under five years, and we still have so much urgent work to do to make it possible — from mouse studies to biomarker studies to patient recruitment — that we cannot afford to break stride by changing jobs like most academics do every few years. Meanwhile, the Broad Institute, where we are based, continues to be an amazing scientific home that makes our unique journey possible. For both of these reasons, we want to stay where we are and continue our research just as we have been doing. Luckily, we have agreement in principle that we can keep our lab and our research positions at the Broad Institute after we graduate — but the federal fellowships that have supported our student stipends will soon run out, and we will soon be on the hook to find 100% of the funds to support our own research effort. While we are working hard on pursuing grants and other funding sources, we also know that donations will be an ever more important source of support for our work.
A donor has generously offered to match your contributions 1:1 up to a total of $10,000 through the end of the year. If you can, please make a tax-deductible donation to our effort to develop a drug for prion disease. You can donate online via PayPal, or mail a check to Prion Alliance, PO Box 391953, Cambridge, MA 02139. Thank you so much for your continued support.
Update Dec 10, 2018: a second donor has stepped up to match contributions from the $10,001 mark to $15,000. One week on, we’ve raised $8,170, so there is $6,830 of match left! Keep the donations coming! Thank you so much!
Sonia, Eric, and Daruka