Rare Leader: Pam Crowley Andrews, Executive Director, Firefly Fund

The Basics
Name: Pam Crowley Andrews

Title: Co-founder and Executive Director

Organization: Firefly Fund

Social Media Links:

Disease focus: Niemann Pick Type C1 (NPC), which is an ultra-rare, progressive, and fatal neurovisceral disorder with highly heterogeneous systemic and neurological symptoms that cause unpredictable progressive deterioration of the central nervous system oftentimes referred to as Childhood Alzheimer’s. Caused by mutations in chromosome 18 in the NPC1 gene, the disease is inherited in an autosomal recessive manner, meaning that both parents must carry mutations in the same NPC1 gene and the child must inherit recessive genes from both parents to be affected by the disease. The incidence of NPC is estimated to be about 1 case per 100,000 live births.  There are currently about 2000 diagnosed cases of NPC worldwide.

Headquarters: Austin, Texas

How did you become involved in rare disease: I became involved in rare disease when both of my daughters were diagnosed with NPC in March of 2016.  My older daughter, Belle (now 9), was diagnosed with NPC after a long diagnostic odyssey that spanned many years and included countless appointments with doctors and specialists. Belle’s diagnosis was eventually made by genetic testing through whole exome sequencing. It was suggested immediately after Belle’s diagnosis that we test our younger daughter, who was 18 months old at the time, although we had absolutely no reason to believe that she also had NPC. As you might imagine, our pain more than doubled when genetic testing revealed that that our younger daughter, Abby (now 5), also tested positive for NPC. 

Previous career: The irony is not lost on me that my more than 25-year career has been largely focused in health care.  My first job out of college was in Washington, D.C. where I lived for a short stint.  I worked at The White House in the First Lady’s office during a time in our country when there was much debate about our nation’s public health system.  That was the beginning of what has been an exciting and satisfying career. However, I never dreamed that I would be on this side of healthcare—as a rare disease parent, personally affected by so many health policies that I’ve worked on.  After DC, I moved home to Texas and worked in the Texas Legislature for the Senate Committee on Health and Human Services and the House Committee on Public Health. I joined the private sector in 2003 as a consultant for a large international consulting firm in their government healthcare solutions practice. My career as a consultant evolved over time and I took on more of a leadership role within my firm in 2006 to focus on strategic initiatives and business development. That is what I was doing the day I got the call from the geneticist regarding the results of my daughter’s genetic testing. As I reflect back and think about that fateful day in March of 2016, I think I knew immediately that all of the work I had done in my life was now meant to be used as a foundation for saving my children. One door has led to another, which has led to another and while the 21-year-old graduate on her way to Washington, D.C. for her first job never dreamed of one day walking through a door to a life with an NPC child, I know beyond the shadow of a doubt that it’s my responsibility to put my experience to work for the NPC community. As you might imagine, that old cliché of “You never know where your journey in life will lead you” has never felt so true. Today I am the co-founder, along with my husband Chris Andrews, and executive director of the Firefly Fund.  As time allows, I continue as a consultant for various clients. Additionally, I serve as an appointed member to the Newborn Screening Advisory Committee at the Texas Department of State Health Services. 

Education: B.A. psychology and political sciences from Hollins University in Roanoke, Virginia

The Organization
Organization’s mission: Firefly Fund’s mission is to fund and support the research and education necessary to accelerate a cure for rare neurodegenerative genetic diseases that affect children and have no cure.

Organization’s strategy: As an organization with limited and precious resources coupled with wanting to make a meaningful impact in NPC, we’ve spent a good amount of time developing and implementing our strategy. Firefly Fund is committed to filling unmet needs in the NPC community and not duplicating good work that has already been done. We’ve identified three primary programs that are the core of everything we are currently doing. Within months of filing the paperwork to create Firefly, we learned there was another NPC patient-inspired organization, The Ara Parseghian Medical Research Fund, which has funded projects related to the basic science of NPC for the past 25 years. My husband and I were so grateful when we learned this and made a commitment to Cindy and Mike Parseghian after meeting them in June of 2016 that the Firefly Fund would focus on complimenting the incredible work they began more than two decades ago and continue to do today at APMRF. Firefly Fund has three core programs.

Our first program is focused on funding medical research related to translational science for NPC.  Specifically, what that means for us is that we are focused on moving compounds and potential therapies from academic labs into the clinic as quickly as possible. Our second program is newborn screening for NPC.  We’ve convened a national multi-disciplinary working group that is representative of various community and industry NPC stakeholders who all share a common vision: to add NPC to state and federal newborn screening lists. We recently announced the launch of a pilot study of newborn screening for NPC where we will screen approximately 200,000 newborns.  When this historic program launches in a few months, it will be the first time any newborn born anywhere in the world will be screened for NPC. Through this study, we plan to collect the evidence necessary to support what NPC expert clinicians already believe— that early diagnosis and intervention for NPC patients improves health outcomes.  This study will also give us an opportunity to learn more about how NPC can fit into our existing public health infrastructure. Please stay tuned for more details on this development at www.firefly.fund.

Our third program is the development of a Patient Access Fund.  All too often, my husband and I get calls from families across the country, and frankly all over the world, because they can’t access the care they need for their loved ones with NPC. Thanks to the generous support of our partners and donors, Firefly’s Patient Access Fund is supporting three different NPC centers of excellence with grants that allow these families to travel and get the care they need.

Firefly’s mission is inclusive of all rare neurodegenerative genetic diseases that affect children and have no cure.  Our long-term strategy has always been to use our learnings from our initial work for NPC, and where our work can be translated across other rare neurodegenerative genetic diseases, we will be prepared to focus on those diseases as appropriate. 

Funding strategy:  Our funding strategy is aligned with the organizational strategy of the Firefly Fund.  We are raising money for the three core programs that I outlined earlier. We believe these three programs are areas of unmet need in the NPC community and that is what we’ve communicated to our donors. It gives our donors great comfort to know that we are committed to not duplicating good work that is already being done in the NPC community and that we are committed to making a meaningful impact for this horrible rare disease by focusing on areas of unmet need. Our donors have entrusted the Firefly Fund with an enormous responsibility and we take great pride in the work we are doing.  It is meaningful and important work.

What’s changing at your organization in the next year: We are in our second year of existence. We celebrated our second anniversary on World Rare Disease Day, February 28,2019. Strategically we will continue to build upon our three core programs. As I mentioned earlier, we will be launching a first-of-it’s-kind pilot study of newborn screening for NPC this fall. The study will last for five years and will screen approximately 200,000 newborns.  Due to the historic nature of this study, we will be learning new and critical information about the incidence and prevalence of NPC. We will stay busy. One thing that you will hear me talk a lot about is moving a disease that is unheard of, undiagnosed, and generally unrecognized into our existing public health infrastructure. Firefly Fund’s three core programs are designed to assist and accelerate our ability to move NPC into the public health system. This is the focus of what we are doing this year, and next year, and the year after that-every year until we’ve moved the mountain.

Management Style
Management philosophy: We can accomplish more together than we can alone.

Guiding principles for running an effective organization: I try to remind myself that this is a marathon and not a sprint. There is urgency to what we are doing and my inclination is to work very hard and fast.  We’ve done a lot of important work for an organization that is just two years old. And I know that we have a very long way to go.  There is a lot of work to do and we need to pace ourselves. Work hard.  Be kind. Don’t forget to breathe. 

Best way to keep your organization relevant: I believe that if we focus on what’s in the best interest of the NPC children/patients, there is no way we can become irrelevant. And what we learn from NPC we will translate to other rare neurodegenerative genetic diseases that affect children and have no cure.  I guess if we accomplish that goal, then we will no longer be relevant.  If I live to see that day, it will be an amazing day.   

Why do people like working with you: I don’t know if people would say they like working with me. I think a lot of people would say that they appreciate my work and I know for sure that anyone I’ve ever worked with or for will tell you that I work very hard and that I have a strong work ethic. In general, I think people know that they can count on me to get the job done. But I can also be very demanding, and I believe that all of the details are important. I work hard and expect the same of people I work with.  There are days when I can be very enthusiastic and passionate about this work, which of course is inspired by my daughters, Belle and Abby. This work is deeply personal for me. In fact, this isn’t really work because this is my life. 

Mentor: I’m fortunate to come from a family of strong and independent women. I’ve always worked for and around very intelligent and dynamic women. I probably have different mentors for different areas of my life. My NPC mentor is Cindy Parseghian, without question. She’s a fellow NPC mom. Three of her four children had and lost their battle with NPC. Cindy continues the work she began nearly 25 years ago with the APMRF despite the unimaginable pain that she carries with her. And she is an example to me that you can get through life’s most difficult circumstances and continue living with grace, courage, and joy. Cindy and her husband, Mike Parseghian, have literally paved the way for the NPC community.  I can’t bear to think of where we would be without them. 

On the Job
What inspires you: My daughters– Belle and Abby

What makes you hopeful: My husband always reminds me that nothing great has ever come from “hoping” that something will happen or “hoping” that something will change.  Essentially, he says to me often that hoping means doing nothing and expecting an outcome. We believe strongly in the work that the Firefly Fund is doing and we believe that this work will move NPC in to a new public health paradigm. The only thing that we have control over is our own actions. I can’t control the fact that both of my daughters have NPC and it is a devastating reality that we live with everyday.  But I can control what I choose to do about it. It gives me great comfort to know that the work we are doing at Firefly Fund will ensure that babies yet to be born with an NPC diagnosis in their future will have better health outcomes because of the work we are doing.   

Best organization decision: To make newborn screening for NPC a priority for the NPC community and to hold firm in my belief that this work needs to be advanced by the patient community rather than a particular industry stakeholder. And also to recruit my dear and long-time friend, former colleague from D.C., and fellow Texan, Allison May Rosen of 3D Communications to be my co-pilot for this important work. It has been a great pleasure to reconnect with Allison on this work that is so deeply personal and to know that she has quite literally been there for me and with me since the very very beginning of my career.  It’s a full-circle experience and sometimes it seems as if 26 years hasn’t really passed by since we last worked together. 

Hardest lesson learned: Not everyone will work with the same urgency that I do. And, that’s okay.

Toughest organization decision: Every decision we make is vitally important because of the nature of what we are dealing with and what we are doing. 

Biggest missed opportunity: We are a very young organization and I’m not sure that I believe we’ve had a missed opportunity, yet.  However, there is always an opportunity to raise more money. 

Like best about the job: I don’t really think of what I do everyday as a job.  I’m just living my life; Firefly Fund is just an extension of my life.  The work we are doing at Firefly Fund is the most important work I will do in my lifetime.  Being a mom to Belle and Abby, an advocate for NPC, and the work we are doing at the Firefly Fund  –  I believe this is my life’s work.

Like least about the job: The politics involved in navigating our healthcare system. Healthcare is a $1 trillion business in the United States. There are only 100 people diagnosed with NPC in the United States. It’s easy to get ignored as an ultra rare disease group. We need to unite and identify our patient population and we need to collaborate globally and succinctly if we want to create meaningful impact for NPC.  I think we are headed in the right direction.

Pet peeve: When people say to me, “if anyone can handle this, it’s you.” 

First choice for a new career: Aesthetician. I’m a skin care junkie and love all the products. 

Personal Taste
Most influential book: Orphan: The Quest to Save Children with Rare Genetic Disorders, by my friend and someone I have tremendous respect for, Dr. Philip Reilly.  Shortly after our daughters were diagnosed with NPC, Dr. Reilly spent some time counseling Chris and me, and he was the first person to encourage us to focus on newborn screening for the NPC community. He wisely explained to us, from the perspective of a venture capitalist, that if Firefly Fund focused on ensuring that NPC patients can be identified at birth, then what will happen as a result is that NPC patients will have plenty of treatments in no time. He explained that newborn screening for NPC would accelerate the drug development process because we would be helping “them” to help us. Of course, whatever we can do to accelerate the process so that NPC patients and families can have treatments available is, obviously, a priority for us.

Favorite movie: I need to watch more movies. My husband loves movies and wishes I would watch more movies. I can’t even remember the last movie I saw. I’m too busy with the girls or working. 

Favorite music: I am from Austin, Texas and we are the live music capital of the world. We have been spoiled with really good live music accessible to everyone in this city for a long time. If I were to pick a favorite genre of music, it would be country music (much to my husband’s horror). I’m a sixth-generation Texan, after all. Let’s just say it’s in my blood. It’s genetic. 

Favorite food: Tex-Mex. I love a good taco.

Guilty pleasure: Getting my hair and nails done with my two favorite girls, Belle and Abby.

Favorite way to spend free time: With my husband and daughters at our home in Austin swimming in our backyard and making dinner on the grill. And the dogs are with us, too.