Questions and answers from the first fireside chat with president Alana Newhouse
Hi, everyone! Below is a Q&A made up of questions from this past Sunday’s fireside chat as well as those that have come in over the transom in the past week. Redundant questions have been combined. Many thanks to everyone who came to this first event, and looking forward to seeing everyone next month! In the meantime, please feel free to send any questions to alana.newhouse@cureangelman.org.
--Alana
Q: What's the cadence of the newsletter and what content will be included?
A: Weekly on Tuesdays, with 3 main pillars of information--science, organization, and community updates. We’d love feedback from the community on what you want to see here. The newsletter has been active for six weeks now so if you’re not getting it, make sure to check your spam or sign up through our website.
Q: What is the difference between you and John? How are your jobs different?
A: John is the chairperson of the board of directors, which is the body tasked with the strategic vision of FAST. I am tasked with communicating that vision to the staff and making sure they have effective plans for manifesting it in reality, and with ensuring that everyone’s work is properly communicated with all of our various stakeholders—resulting in a best-in-class foundation, resourced and positioned to get us closer to a future filled with meaningful treatments for everyone living with AS.
Q: Will you continue to hold the gala in different cities and the same weekend moving forward?
A: People had asked us to try out different cities to create geographical diversity, which felt right, and I think we will continue to do that. As for the date, we don’t have set plans currently, but I’ll update everyone when I know more.
Q: What is the difference between the SAB, FAC, and FAST Board of Directors?
A: The FAST Board of Directors is a group of Angelman parents who drive the vision for the organization. As I explained in my letter, until now this has been a working board—meaning this group of people were responsible for both the strategic planning (deciding which scientific programs were worth investing in, building vital ancillary programs like the biomarker consortium, identifying pharmaceutical companies that would make responsible and effective partners on AS, and so on) as well as the practical tasks that manifested this strategy in reality--all while holding down often demanding full-time jobs outside of this. One of the changes underway this year is that the board will be making the now-overdue shift into a strategic board, with a staff of skilled professionals on board to do the practical work. They have always been, and remain, volunteers. There are many examples of rare disease advocacy organizations that are managed by a full time professional staff.
The Scientific Advisory Board (SAB) is a group of scientists who, also on a volunteer basis, help identify cutting-edge or compelling research opportunities, review grant proposals, and advise any science-focused staff. FAST Advisory Council (FAC) a group of people who have loved ones with AS (also volunteers) with professional skill sets necessary to advance priority programs and initiatives, like supporting government policy as it relates to state and national awareness of the needs of those living with Angelman syndrome, editing and writing scientific content for newsletters and blogs, supporting the biomarker and outcome measure consortium, and creating an army of representative families to ensure we stay aware of the needs of everyone in our worldwide community.
Q: I see value in both FAST and ASF, do I have to pick sides?
A: Absolutely not! I haven’t. I personally think the ASF is incredible at connecting families to the programs and resources they need for education, safety, quality of life, and more, and I know it’s a view shared inside FAST.
Q: Why doesn’t the board answer questions on Facebook and keep the community up-to-date there, as it always did under prior leadership?
A: Facebook offers so many advantages, not least of which is the ability to connect with a global community. But the imperatives of my job as shepherd of FAST’s messaging process are accuracy and sensitivity—both of which are challenged by these platforms. The speed at which Facebook operates actively encourages mistakes of fact, miswordings, and pile-ons, none of which advances our goal of creating an informed and confident community.
Moreover, a significant swath of our community is not even on Facebook! Given all of this, the primary channels for messaging vital information are going to be the ones used by everyone in the FAST community—our newsletter and our website. But everything posted there will then also be filtered through social channels, to ensure that we reach as many people as possible. And during each fireside chat, I will publicly answer questions emailed in that seem relevant for the wider community. (Oh, and on that note: If you do NOT want your question aired publicly, please note that when you email it in. It’s usually pretty clear which questions are meant to be private, but—as ever—better safe than sorry.)
Q: Why is FAST saying they still need $200m to advance more programs? If FAST has so much money now, why are they still asking the community to fundraise?
A: One of the insights that drives FAST’s work is the notion that successful treatments are never discovered on their own; the more efforts underway in a given field—and the more diverse—the more likely it would be to find truly effective therapeutics, and eventually one that could be considered a cure. So we cannot stop at the first or the second idea when it’s our job to chase down every possible option, and to filter out the absolute best ones for everyone around the world living with AS—regardless of genotype, and regardless of age.
Lucky for us, there are currently ten promising methods to treat AS in the pipeline, including the program acquired this summer by Ultragenyx. The cost to take one drug through proof of concept, IND enabling, Phase 1/2 clinical trials, phase 3 clinical trials and approvals can be well over $100 million! To take the full basket of our current promising programs just far enough along before we can give them to trusted partners will, we estimate, be $200 million.
Q: Are there any plans in the works to make the summit and gala more affordable?
A: There are, and there should be more. After COVID, travel and service industry prices increased exponentially, not just in the US but around the world. FAST has worked hard to obtain the best prices for the Science Summit & Gala. We have also offered more scholarship opportunities than ever before to families in need, as well as discounted early bird specials on room rates and the parent ticket giveaway. That said, we are certainly taking this financial question into account as we research options for next year’s summit and gala. If you have thoughts or suggestions, tell us!
Q: If FAST is growing and you are adding on paid staff, will that slow down the road to a cure if you have people on payroll now?
A: It should do the opposite, which is why all other successful rare disease organizations have staffs— like Cystic Fibrosis Foundation, CureSMA, Parent Project Muscular Dystrophy, and Rett Syndrome Research Trust, and more. And not only because professionalizing will make it more likely that we can attract the sort of investors and pharmaceutical partners who can help push treatments through the pipeline. In general, having skilled and experienced staff makes everything move more efficiently, and hence faster. I hope you’ll see what I mean as I begin introducing the community to our team in the monthly fireside chats.