Rare Leader: Molly White, CEO of the Myotonic Dystrophy Foundation

January 4, 2018

The Basics
Name: Molly White

Title: CEO

Organization: Myotonic Dystrophy Foundation

Disease focus: Myotonic dystrophy is a genetic, autosomal dominant disorder. It’s considered a rare disease. It been called “the most variable disease in nature.” It looks different in every person that has it, although there are common characteristics. Every cell in your body can be affected, which means every system in your body can be affected, from your heart to your pulmonary system and breathing, to your skeletal muscles, your smooth muscles, which would be all the muscles you use to swallow and in your digestive tract. It also affects various processes in the brain, including executive function and the ability to focus. It can have an impact on people’s emotional and psychological health. It’s multi-systemic.

Headquarters: San Francisco

How did you become involved in rare disease: I was contacted by an executive search firm. I had been on the opposite side of the table, in corporate funding. I had built and run foundation funding programs at a number of large companies, including Nike, The Gap, and Visa. The search firm that contacted me knew about my work with those programs, and knew that in addition to building initiatives focused on specific issues, including health and wellness, that I liked building things.
Previous career: I started out in the nonprofit sector and after two years working with organizations in SF was hired to run the foundation at The Gap, and start their community affairs program. I spent about 22 years doing corporate philanthropy and social responsibility work. The Myotonic Dystrophy Foundation was my first sojourn back into the nonprofit sector.

: B.A. in painting from the University of Montana, Missoula, and a masters in Art History from the University of Iowa.

The Organization
Organization’s mandate: Care and a cure for myotonic dystrophy: That’s not just our mission, but also our mandate. We are committed to helping people live their best quality of life now while we help accelerate the search for therapies and a cure.

Organization’s strategy: To do an assessment of the landscape and understand what the opportunities and barriers are, and what the most time-sensitive needs or opportunities are, and then build a multiyear strategy to deliver on the results of that assessment. On the care side, we’ve conducted a pretty significant assessment of self-care, family care, clinical care, caregiver care, and we’ve reached out to some other organizations to help pull that information together. We created the first-ever clinical care guidelines for this disease globally, working with an international group of about 70 clinicians to develop them through a consensus-based process. We’re now working to get them all published, translated and implemented in clinical settings around the world. That’s an incredibly important project because when people go see a doctor right now for this disease, unless that doctor has treated other patients and built an experience base in this disease with those patients, they have no idea what they are looking at, they have no idea what the disease course is, what the typical symptoms are, or what the current therapeutic approaches are for this disease. Getting consensus-driven guidelines out there that can be published and adopted and translated and in the hands of clinicians should help drive real change for clinicians and patients. It’s a dissemination process that we know we are going to be tackling for a while, but we are committed to getting them out.

We look from the clinical care arena to areas of self-care and family care and understand how we can support patients in accessing the best quality of life they can have now, how they can improve their quality of life, what things they can personally do, and how they can take part in the networking and community building we offer that can lessen some of the isolation that is a common part of living with this disease.

We do the same thing on the cure side. We did a huge assessment of the drug development pipeline from basic science in academic labs all the way through to the payer environment and access. We put together a $5 million, three-year initiative to accelerate drug development called MDF 3.0. We just wrapped the bulk of MDF 3.0 initiatives up last month. There are a few projects that are still ongoing. We were able to support the development of biomarkers, endpoints, to support clinical trial design, fund the development of a new mouse model, support creation of a library of cell lines and much more. There was an enormous amount of work we funded, facilitated, or supported through MDF 3.0 to optimize drug development for this disease.

When we talk to pharmaceutical companies and biotech companies, we bring a PowerPoint presentation that lays out all the initiatives we’ve executed. We typically hear that they have not run into an advocacy organization that has done this level of work, and they are pretty impressed by it. They often say that they wish they’d known about it earlier so that they could include it in presentations to their investors. MDF 3.0 has done a tremendous amount to help expand the number of industry organizations focused on drug development for this disease. When I joined MDF in 2012, there were two organizations truly focused on developing therapies for myotonic dystrophy. Now we are interacting with around 20, and we want to double that number. We’ll tackle this objective the same way from a strategic standpoint —assess the landscape; understand what the opportunity, barriers, and constraints are, what the most time sensitive pieces are and what is most urgent, and we’ll figure out an initiative to achieve that goal.

Funding strategy: It’s tough for this disease. In a lot of other diseases, you have an affected family member, and then parents or other family members who are not affected and fully in the world of work, and therefore able to commit to supporting organizations and efforts targeting education and therapies. The MDF community includes many families where there are three generations of family members that are all living with the disease. We have a disproportionate number of people who are out of the world of work due to their disabilities and have financial constraints as a result. I did an assessment recently of our database and found that four out of five people who contribute to the foundation are people not living with the disease or have the disease in their families, but instead are friends of affected individuals. That’s pretty unusual.

At the same time, we’ve gone from an operating budget of around $500,000 to an operating budget of around $2 million in the last five years, in order to execute all of the work we just discussed. It’s a challenge to raise those funds, but that’s what we need in order to be able to drive the advocacy, and the research, and the care related programs. We have to continue to identify people who are interested in engaging in the disease and are in a position to help. Everyone who is capable of being a donor is incredibly precious to us.

What’s changing at your organization in the next year: We have a few team members that will be retiring, or going on to medical school, so we’ll have some exciting new faces joining the team. Having just finished MDF 3.0, which was the $5 million drug development initiative completed in 2017, we’re going to framing up the next major piece of work to accelerate therapy development. As I noted earlier, one of the things we’re interested in at the foundation is doubling the pipeline. If we went from two to 20 from 2012 to 2017, can we get to 40? Can we have 40 pharmaceutical and biotech companies heavily focused on developing therapies and a cure for this disease? That’s the moonshot we’d like to get tackle next.

The question is going to be, what are the things we need to do to make that happen. We’ve had a number of conversations with industry and academia about that. We’re going to present some initial approaches at the strategic planning offsite that occurs every January, and then figure out how we can finalize and fund them, because they’re likely to be very expensive. The MDF 3.0 initiative helped get a lot of infrastructure into the disease arena to make it far less risky for pharma and biotech to consider as a potential target than it was five years ago. The question now is, what else do drug developers need to commit to DM and what else can we do to remove any final barriers?

Management Style
Management philosophy: My ideal is to hire great people who know what they are doing and support them in doing great work. I’m not interested in micro-managing people. If there’s an opportunity to help someone achieve their professional goals, I’m super excited about that, and often what that ends up meaning is that at some point it’s time to move on. I’m a big proponent of helping people identify what that is, and helping them work toward it in their current position. What I don’t like to do is breathe down people’s necks and check on everything that they are doing. We’re a very small organization and everyone pretty much owns their vertical. I’m your wing person. I’m here to support you and partner with you. If we get people who are junior, it’s a problem if I need to do their job with them, because there’s not enough bandwidth in the organization for that. We look for team members who are self-starters, self-motivating, and seasoned, or willing to get there.

Guiding principles for running an effective organization: One thing I’ll say about working at places like Nike is that the bar for quality is so high. As a result, the bar for execution around here is pretty high. In every piece of output we generate, whether it’s an internal agenda for a meeting down to the publications we produce and the programs we launch, we are extremely committed to high quality work on every single thing. It’s just the way we operate. People who don’t take that same approach tend to run into trouble here. Because we turn out so much work, everyone operates just about in the red, and it can be a a grind. There is a lot of work that gets pumped through this pipeline every day and we expect it to be high quality. It takes a special type of person to operate that way.

Best way to keep your organization relevant: We have to be the partner that people want, whether they are on the industry side, or on the care side and working with our families and effected individuals. We make sure we are incredibly responsive and sophisticated about the needs in the space and are committed to addressing them better and faster than anyone else.

Why people like working for you: People who like working here tend to be people who like to be in their lane, own their work, and be in charge of it. They get supported 150 percent, but they don’t have a lot of oversight and day-to-day management. I meet with everybody once a week at a one-on-one, and we have a whole staff meeting every week. Otherwise team members call me if they need me.

We are urgent around here about what we are doing. We know we’re on the cusp of seeing the first therapies come to market. We know we have started work on a potential cure. We know there’s work in the pipeline that is just accelerating pretty quickly, and people outside of the space are saying that myotonic dystrophy is a hot disease. It’s an incredible moment to be working in myotonic dystrophy. For people committed to really good work who want to have free rein to do it, it’s a good place.

Mentor: In this job, the chairman Jeremy Kelly said to me, “You need to look at what Pat Furlong at Parent Patient Muscular Dystrophy is doing. She came from being a mom of two affected sons and an RN, to someone who built and runs one of the most effective patient advocacy groups in the world. She’s fearless, smart, and incredibly creative. When I first got here, I found out where their conference was, and I flew down and met her. PPMD has done a lot of groundbreaking work developing guidelines for the FDA, and around a lot of different areas of the patient advocacy space. When we look at who’s doing really good work, where there have been real opportunities to gain traction, we often look to PPMD. I wouldn’t say Pat is a mentor—she wouldn’t have the bandwidth— but they are an organization we look toward with real respect.

On the Job
What inspires you: You can’t do this job and not fall in love with this community. We have some of the most patient, caring, inspiring people in this community who live with some, as one of my board members would say, really “shitty” conditions. And they do it with a lot of grace, and they do it with a lot of humor, and they never give up.

What makes you hopeful: This disease is really lighting up. There’s no other way to look at it. Anyone you talk to in the drug development space will tell you that myotonic dystrophy is really taking off. Considering the fact that U of Rochester turned it off in a mouse model in 2006, and Ionis Pharmaceuticals, which brought the first drug to a clinical trial, first learned about myotonic dystrophy in ’08, it is amazing that their first drug was in the clinic in 2014. That’s a pretty vertical acceleration of learning about a disease, figuring out how your assets can target it, and developing potential chemistries and getting them tested. That’s an incredibly fast turnaround.

We’re getting a lot of cold calls from pharmaceutical companies and biotech companies that are interested in pursuing DM. They want to know about the disease. They want to know about the regulatory environment. They want some support from us to do a variety of things. We believe myotonic dystrophy is going to tip in the next couple of years, and then we’ll really start to see some things change. I want to be around for that.

Best organization decision: I would say that one of the best things we’ve done make the decision to hire a chief science officer. We brought in Sharon Hesterlee, who had been at PPMD running their research platform, and at MDA, who has a Ph.D., and who did a phenomenal job pulling together what became MDF 3.0.  There’s only so much you can do without some strong science credibility and information.

Hardest lesson learned: A lot of the work that we do here is spade work. It’s one patient, one city, one organization at a time. We work with clinical trial sites, clinical network sites that still forget to let their patients know about us and the resources we offer families. Building visibility and capacity around this disease out in the clinical care world and research world is tough. You just have to be dogged.

Toughest organization decision: Sometimes people get involved in this disease and they don’t know what they don’t know. If they want to make a contribution, sometimes the amount of money they want to donate and what we need to do to get it are too difficult or off-strategy to justify the work. The Gates Foundation has said you can spend $1 billion and not have any impact whatsoever. You have to understand where the leverage points are.

We have worked with donors who just want to start throwing money around because they are motivated, or they are affected, or there is something else driving them. They don’t necessarily stop to work with us and do all of the research regarding where they can get the most impact out of that donation. There’s only so much you can tell people. They have to come to that on their own. There have been some tricky situations we have had to negotiate as a result.

Biggest missed opportunity: One thing we have not been doing very well is talking about what we are doing, even with our own board at times. We haven’t been doing a good enough job of letting our donors know about everything we are doing. We brought in a new communications team member this year, who is going to help us change the game on that. People can’t get excited about you and support you if they don’t know what you are doing. It’s incumbent on us to do a better job of telling our story and the story of the disease a little more effectively and broadly.

Like best about the job: Working with the researchers and working with the community. The whole drug development space is fascinating. The regulatory environment is fascinating. The fact that we can have an impact on that is an exciting opportunity for us. And the community. You can’t walk away from an interaction with our community members and not feel motivated and committed to being  there when we get the first effective therapy into the market, and people can start to feel better.

Like least about the job: I like all the work I do here, even the mundane parts like processing checks. What I don’t like about this is that I just don’t have enough time to do it all. I would say I could use about 30 more hours a week here.

peeve: Not everybody is working as fast as we are. It gets frustrating when it takes forever to get some people to get stuff done. We wish they would hurry up.

First choice for a new career: If I thought I could have any impact, after I retired I would go into interior design.

Personal Taste
Most influential book: I don’t know about influential, but a couple of my favorites are The Shipping News by Annie Proulx and Middlesex by Jeffrey Eugenides

Favorite movie: I don’t see enough movies to have a favorite

Favorite music: My family is musical. My grandfather taught at the Eastman School of Music in Rochester, New York. My dad was a musician of sorts. I like alternative and indie rock, I like classical and jazz. Pretty much everything except some country.

Favorite food: Mediterranean

Guilty pleasure: Salted chocolate caramels

Favorite way to spend free time: Long hikes with my dog.

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