INAUGURAL REPORT TO DONORS
MESSAGE FROM THE FOUNDERS It’s hard to believe it’s been nearly three years since we launched Firefly Fund. Because of your generosity and support, we’re making a difference in the lives of patients and families with rare disease — specifically Niemann-Pick Type C (NPC). Firefly Fund is committed to filling unmet needs in the NPC Community. We’ve identified and developed three core programs of focus for our work. First, our Patient Access Fund is providing valuable support to NPC Centers of Excellence that are treating NPC patients who otherwise would not be able to access necessary and specialized care. Second, we’re funding translational medical research for children with neurodegenerative disease. And third, we are making enormous and exciting strides in newborn screening. We are proud to present you with this inaugural report and hope you’re as proud as we are of the progress we’ve made. You are indeed making a meaningful impact in the lives of children everywhere living with NPC. As we prepared this report and looked through the names of donors listed in the back, we were humbled, of course, by your generosity and overcome with immense gratitude. When we began this journey, we felt a lot of despair and uncertainty. Today, we are surrounded by a glowing – and growing – light of so many supporters and friends. Today we’re certain we’re not alone, and we also know that no matter how hard we work, we cannot accomplish our goals without you. You are the true heroes of Firefly Fund’s story. As we set our sights on 2020 and all of the possibilities in the new decade that await us, we’re more committed than ever. With your help, we know Firefly Fund will exceed our goal of raising $1 million in 2020. With gratitude, Pam & Chris Andrews
OUR LEADERSHIP
Pam Andrews Co-Founder & Executive Director
Forbes D. Porter, M.D., Ph.D. Scientific Advisory Board Member
Marianne Dwight Board Member
Chris Andrews Co-Founder & Board Member
MariBen Ramsey Board President
Berry Crowley Jack Gullahorn Colin Hovinga, Board Member Pharm.D., M.S., F.C.C.P. Advisory Board Scientific Advisory Board Co-Chair
Ben Machielse Board Member
Gus S. Park, M.D. Board Member
Amy White, M.D. Board Member & Scientific Advisory Board Co-Chair
OUR MISSION Our mission is to fund and support the research and education necessary to accelerate a cure for rare neurodegenerative genetic diseases that affect children and currently have no cure.
KEY PROGRAMS
Newborn Screening Firefly Fund leads a multidisciplinary NPC newborn screening working group focused on submitting a successful application to the federal government’s Recommended Uniform Screening Panel. We convened the first-ever clinical roundtable of NPC and newborn screening experts to demonstrate the clinical benefits of early intervention for NPC patients. We are contributing to the design and launch of ScreenPlus, a pilot study to screen 250,000 newborns for NPC. The study will offer the first opportunity for newborns anywhere in the world to be screened for NPC. Beyond NPC, our newborn screening program contributes guidance to other rare disease groups as requested by providing the “roadmap” we’ve identified and pathways for adding additional diseases that meet relevant criteria to state and federal newborn screening lists.
Translational Medical Research As part of our commitment to you, our donors, to focus on areas of unmet need in the NPC Community, Firefly Fund focuses support on translational medical research, by testing potential therapies in NPC animal models, and moving compounds from the bench to the bedside as expediently as possible. Firefly Fund is providing support for exciting translational science at the following academic institutions and NPC Centers of Excellence: (listed alphabetically): • Einstein School of Medicine; The Walkley Lab • National Institutes of Health; The Porter Lab • National Institutes of Health; The Pavan Lab • University of Miami; The Liebl Lab • University of Pennsylvania; The Vite Lab • University of Texas at Austin; The Martin Lab • Washington University at St. Louis; The Ory Lab
Patient Access Fund Firefly Fund’s Patient Access Fund is working to strengthen existing infrastructure that supports NPC families and the burdens that come with a rare disease diagnosis. Firefly Fund’s Patient Access Fund removes barriers and improves access to life saving therapies necessary for NPC patients. Firefly Fund provides support to academic institutions and Centers of Excellence that are committed to providing care to NPC families regardless of their ability to pay: • Dell Children’s Medical Center • Rush University Medical Center • University of Alabama
“Firefly Fund is a patient & family driven organization which has mobilized the entire NPC community to support the goal of introducing newborn screening for Niemann-Pick Disease Type C. Early diagnosis & intervention offers the best hope to families afflicted by this disease.”
“Pam & Chris Andrews are strong advocates & community leaders who serve as a resource for parents of newly diagnosed children. They work tirelessly to advance treatment options and awareness of NPC. Their efforts are helping to facilitate research toward treatments for NPC.”
“Firefly Fund’s advocacy on behalf of children with NPC and other rare, neurodegenerative diseases is inspiring and making a difference. Today, thanks to Firefly Fund, more children are able to access care at Centers of Excellence.”
-DR. MARC C. PATTERSON, THE MAYO CLINIC
-DR. FORBES D. PORTER, NATIONAL INSTITUTES OF HEALTH
-DR. ELIZABETH BERRY-KRAVIS, RUSH UNIVERSITY MEDICAL CENTER
BECAUSE OF YOU. . . A potential new and novel therapy is being investigated by a working group that includes the National Institutes of Health and academic partners, exploring the modulation of TMEM97/Sigma2 receptor for the treatment of NPC and possibly several other neurodegenerative diseases. Patients diagnosed with NPC have access to life-saving therapies regardless of ability to pay. Parents of newborn babies in the state of New York will have the option to have their newborn screened for NPC for the first time EVER in the history of this critical public health program.
FINANCIAL REPORT Firefly Fund has invested more than $884,000 to accelerate a cure for NiemannPick Disease Type C.
ALLOCATION OF FUNDS
12%
Programs
15%
Fundraising
73%
Administration
8% ALLOCATION OF PROGRAM FUNDS Translational Medical Research
45%
47%
Patient Access Fund Newborn Screening
THANK YOU DONORS CORPORATE SPONSORS 3Ten Agora Club USA Altrua HealthShare Amherst Holdings Amicus Therapeutics Blackridge Bloom Communications Boutique Educational Services ButterLutz Interiors Caddo Associates Capitol Anesthesiology Association Chameleon Biosciences, Inc. Champions Westlake Cowboy Auctioneer Creative Pickle LLC
Custom Golf PPL Cyclo Therapeutics, Inc. Daily Juice E-Scape Bio Evox Therapeutics FTI Consulting, Inc. HSSK, LLC LOVE Cycling Lumos Pharma, Inc. Mallinckrodt Pharmaceuticals Navigant Consulting, Inc. Network for Good (Facebook Fundraisers) Orphazyme Pluckers Wing Bar
Ride Indoor Cycling Stratus Properties Stride Bio Taylor Collective Solutions Texas Advocacy Project The Isidorus Chapter of Alpha Chi at Miami University Wanderlust Yoga West Bank Flower Market Your Cause LLC (Employer Match) MPD Custom Golf
FOUNDATIONS Amherst Foundation Carl C. Anderson, Sr. and Marie Jo Anderson Charitable Foundation Meyer Levy Charitable Trust NCI, Lending a Hand NPSuisse The Elisabeth Crook & Marc Lewis Foundation The Ralph & Eileen Swett Family Foundation
Just Keep Livin Foundation The First Foundation Together Strong - NPC Foundation
THE KOUIJAN FAMILY
THE MOORE FAMILY
“Without the generous support of our families and friends we wouldn’t be where we are today,” says Gail Kouijan, mom to Alec and Haley, of Chicago. Together we are stronger.”
“Thank you for giving our daughters the possibility that they have and for a brighter journey ahead,” says Julie Moore, mom to Brynne and Kendall, of California. Please know that we truly appreciate all that you do!”
THANK YOU DONORS INDIVIDUAL DONORS Julie & Sam Adams Lawrence Lewis Aldridge Brooke Alexander James Alsup Carl Anderson Stacey Anderson Brian Andrews Pam & Chris Andrews Elizabeth W. Andrews Elena & Constance Andrews Jean & Mark Andrews Mario Dipietrantonio & William Scott Andrews Belle & Abby Andrews (Lemonade Stand) Beau Armstrong Alice Arrington Lobell Jane Atwood Robbie Ausley Cindy & Paul Ballard Andrew Bangs Eugenie Baranoff Karen Bartoletti
Becky Beaver Laura Hobby Beckworth & John Beckworth Rob Belina Heather Bellino Chris Bemben Linda Benge Jennifer Berbas Stephanie Birko Haferbier Kelly and Jason Black Jennifer & Darren Boerner Teresa Bornsen Sonal Bowness Dorothea Boyle Dillon & Matthew Braks Daniel Brannon Cody Braunn Willy Braunn Tiffany & Chris Britton Marla Broaddus Sara Brook Jo Ellen Brown Stephen Brown Susan Brown
Carlie & Thomas Burdett Barbara Burr Beth & Kevin Burrows Blair & Michael Burton Sue & Marty Butler George Butts Anne Byars Jenny & Jarod B. Carter Patty Chandler Amy & Matt Chapman Melody Chatelle Laura Cho Elizabeth Christian Diane E. & Richard L. Clark Patricia & Michael Clement Amy & Marcelo Conde Jeannette Cook Jennifer Cook Wanda & Edward Coultas Dudley Court & GA Purcell Kendall & David Courtney Leilani Cronin Hope & Mike Crowe Beryl “Berry” Crowley
(aka Grandberry) Deborah & Timothy F. Crowley Jim Crowley Leslie Crowley Lisa Connell-Crowley & James Smith Mary Crowley & Dennis Dillon Vickie & Matt Crowley Ali & Sean Crowley Karen & Terry Crowley Zarghun Dean Beverly Dale Jim and Kelly Daniell Charitable Fund of the San Antonio Area Foundation Wally DeRoeck Anne & Matthew Desloge Amy & Jeffery Dolce Ann D. & Osborne J. Dykes III Sara Dysart
THE RUTHVEN FAMILY
THE ALVEY FAMILY
“It has been such a relief for our family since Cody and Kayla started receiving treatments in Austin at Dell Children’s,” says Dena, mom to 19-year-old Kayla and 22-year-old Cody, of Elgin, Texas.“Traveling to the NIH every month was exhausting and caused longer recovery times for Cody and Kayla. Thank you for creating the Patient Access Fund to ease the burden of life with a rare disease.”
“Pam Andrews and her husband Chris were the first to reach out in our darkest hour,” says Garland Alvey, dad to 4-year-old Abby, of Virginia. “Firefly Fund is a beacon of light for families facing the unimaginable. They shine a light on the troubles ahead, give guidance, while also reminding us to face the storm with strength and perseverance knowing that we aren’t alone.”
THANK YOU DONORS INDIVIDUAL DONORS (CONTINUED) Susie Dudley & Salvatore Conti Virginia Cox Marianne Dwight & Ed Cisler Laura Elias Barbara Elias Barbara Ellis Meredith & Will Ensign Margareha Erden Valerie Fairchild Sharon Fenoglio & E. Dominic Megann Ferguson Debra Day Floyd Bill Ford Steven H. Frantz Jolynn Free Elizabeth & Hank Fromme Jennifer & Joseph Gatti Maya Gelsi Margaret Gosselink Laura & Morris Gottesman Royce Gourley & Ferdinand Pasion Patricia Gray Linda Griffith Susan Cope Griffith
Hope Grossman & Jonathan Jacoby Patti & Jack Gullahorn Molly Haile Kerri Hampton Peggy Hanna Debbie Harmon Paula & Orrin Harrison Susan & David Hart Ashley & Kirk Haun Paula & Daniel Herd Timothy J. Herman Jessica & Jeff Hershey Joan & David Hilgers Patricia & Corey Hoffpauir Charles Hornung Rives & Anthony Howard Gretchen Huddleston Meta Hunt Kathy Hutto Jack Ingram Leah Clelland Iochim Andrew Jackson & Diane Dwight Sidhya Jayakumar Amy Jensen Jennifer Johnson Kristin Johnson
Luci Baines Johnson Sally & Ralph C. Jones Shannon Jones Lynn & Jared Jordan Liz Judlin Kinkella Debbie and Steve Kaflowitz Karen Kahan & MariBen Ramsey Kimberly Katzburg & Jared Dunten Irina Katsnelson Trish & Hal Katz Rusty Kelly Allison Y. Kenney Kim Kibby Kelly Kirkland Janice Klekar Al Koehler Claire Korioth Lanora & George P. Lahtinen Daniel Lamont Diane Land & Steve Adler Emily Lange Elizabeth Lang-Miers Kristi & Charles Larkam III Joe Lea Lana Khazen Levy
Erin & Chris Lee Heather & Darren Lenton Lana Levy Linda Lieberman Marco Lima Adam Loewy Leslie & Andrew Lohan Susan Longley Jan & Chris Loughran Rachel Lowder Susan Lubin E. Clark Lutz Amy Lyle & Ashley Green Dusty & Travis Machen Leonza & Ben Machielse Nadine & Marc K. Mackin Susan MacLean Joseph Maldonado Elizabeth Malone Jessica Mangrum Meredith Marks Cari Marshall Fay & Steve Martin Chris Mattson Jennifer May & Steve Gelsi Linda K. May Melinda May Sue & Phillip Maxwell
THE FERGUSON FAMILY
THE MILLER-BRUMBELOE FAMILY
“A firefly produces but a small speck of light in the darkness,” says Meghann Ferguson, mom to 5-year-old Liam, of Virginia. “A whole community of fireflies can light up the night sky. That is what the work of Firefly Fund represents to our family. A whole community of parents, friends, researchers, and doctors working together to illuminate the darkness of rare disease.”
“Chasing fireflies is a childhood memory many of us hold dear,” says Kayla Miller, mom to 8-yearold Kamryn, of Alabama. “The Firefly Fund helps our community chase childhood, and quality memories, for our children! We are appreciative for the efforts of all involved. NPC families face the unimaginable; yet, the Firefly Fund sparks hope for many childhood memories.”
THANK YOU DONORS INDIVIDUAL DONORS (CONTINUED) Brian McCall Kelly McClouth & Stephen Staha Sarah McHaney Seth McInteer Brianna & Justin McKinney Renee & Seth Meisel Elva Mendoza Leanne & Michael Messer Sally Metcalfe Carolyn Miles Garber Harriet Miers Helen Milby Gail & Neil E. Miller Jennifer Monday Goldman Ross Moody Kay & John Mooney Elisabeth Morrison Hope Morrison & Mike Crowe Michelle Mott Derek Naten Robert Neblett Karen & James Nelson Tricia Niederauer Betty Nowlin Dillon Nugent Nancy O’Brien Harriet O’Neill
Cathy O’Toole Peggy & John Odam Robert Ohyama Kathleen & Rufus Oliver III Catherine Orpett Mary Paver Jutta & Wolf Peddinghaus Melissa Perez Raquelle Peterson Jan & Gary Pickle Claire Pinkerton Christy and Turk Pipkin Jennifer Piskun Johnson Jennifer Polk Brenda Pollard Shawn & Jim Ponton Jennifer Purcell Karen & Gene Quandt Michael Racis Eve Radke Amreen Rajabali Jean & Dan Rather Steve Ravel Elizabeth & Gabe Raymond Jim Reeder Beverly Reeves Philip Reilly Michael J. Resnick (Resnick Family Charitable Fund)
Niki & Randy Reynolds Karen Riedel Robert Robinson Kelly and Bruce Robison Karen & Andrew Rohan Abigail Rosen Allison & Dean Rosen Marilyn Rosen Jason Rottinger Roque Ruggero Dena & Scott Ruthven James Sahn Monica & Marc Samuels Eddie Safady Carmela Saunders Scott Saunders Nancy Scanlan Nathan & KB Schatz Madeline Schell Sandra & Harris Schell Kitty Schild Randy & Stan Schlueter Adam Schramek Randi Eisner & William Schwartz Shelly Scott & Thomas A. Forbes Karen Kennedy Seidler & David Seidler
Diane Senterfitt Carolyn & Marc Seriff Anna Sheehan Dean Max Sherman Lynn Sherman Yvonne Shifrin Elizabeth Sibley Chandler & Kent Chandler Ricardo Silva Thomas Sims Holly & Michael Singletary Pam & Edward C. Small Martha Smiley Bea Ann Smith Kathryn Smith Shannon & Lamar Smith Rochelle Sohl Janet & Robert Stallard Kelli & Brian Stanford Kelly & Robert Steele Patricia & David Steinwedell Karen Stocks Chris Streiferd Laurie Swan Lisa Tauszig Amanda & Scott Taylor Emily & Eric Taylor Richard Taylor
THE BERNS FAMILY
THE MCGLOCKLIN FAMILY
“We are thankful every day for the generous donations which fund NPC research,” says Chris Berns, mom to 22-year-old Samantha, of Stillwater, Minnesota. “It has given our daughter a treatment for today, with long-term hope for the future.”
“Firefly Fund helps to give us hope for our daughter to have a healthy and bright future,” says Sara McGlocklin, mom to 4-year-old Marian, of California. “It is organizations like Firefly Fund, fighting for NPC patients, that are changing the lives of NPC patients today and in the future.”
THANK YOU DONORS INDIVIDUAL DONORS (CONTINUED) Tammy Templin Debbie & Joel Tendler Carra Thomas Kristie Tice Bobbi Topfer Sue & Terry Tottenham Marcia Trotter Nanci Tucker Dudley VanCourt Khara & Jesse C. Vincent James Wadlington Miranda Walker Dee Dee & Joe Bill Watkins Liz & Kirk Watson Lin & Andrew Weber Jennifer Wegmiller Teresa Werner Deborah West Thomas Meade West James Wester Chris Whitaker Amy White Stephanie & Bill Whitehurst Natalie & Ryan Wiggins Marilyn & Matthew Willson Gretchen L. Wilson
Seth Winick Matt Winkler Marc Winkelman Tomi Winstead Ewaen Woghiren Kristin Wollman Brooke Wood & Kristin Powers Katie & Brian Woodliff Ellen Woodward Roberta Wright Suzie & Eric Wright Mary G. Yancy Sandy & Dudley Youman Harriet Young The Thomas & Patricia Yoxall Family Charitable Fund of The Catholic Foundation Patricia Zellmer Stefani Zellmer & Chris Davis
THE QUANDT FAMILY
THE SCHRIVENER FAMILY
“Donations to Firefly Fund can alter the course of Niemann-Pick Disease,” says Karen Quandt, of Pocatello, Idaho, in memory of her son Ty. “Newborn screening for NPC will give families the opportunity to treat NPC disease before the disease progresses and will alter the course of this devastating disease.”
“Thank you to the donors of the Firefly Fund who are helping Emerson and many others with NPC live a full life,” says Destiny Schrivener, mom to 5-yearold Emerson, of Kansas City, MO. “Your donations give us hope for the future.”
UPDATE ON BELLE & ABBY Belle will be 10 years old on March 18, 2020, and we can barely believe it. As you know, she began steadily receiving an investigational therapy called Adrabetadex (aka VTS270) three years ago, after the onset of visible signs and symptoms of NPC. While she lost the ability to walk independently before she began receiving the medication, Belle remains stable in many other areas. In fact, some areas like swallowing, have improved! She still chokes when drinking thin liquids, but no longer chokes on food at every meal. Belle is a happy girl and a delight to be around. She plays a mean game of Crazy 8s and can hold a conversation with just about anyone. Abby, 5, is also on Adrabetadex (aka VTS270) and, to anyone who meets her on the street, appears completely neurotypical. Our expert clinicians however, have identified some developmental delays, which is a sign of NPC progression. Abby loves dance and gymnastics, loves knitting, and spending time with her friends. Her BFF is Lady, an 8-pound toy poodle. They are inseparable. So much so, we are considering training Lady to accompany Abby at her every-other-week hospital treatments. While we have reached a “new normal,” we harbor no illusions. The experimental treatment Belle and Abby receive merely slows the progression of NPC. This is why we must continue to fight NPC and to find additional therapies for this disease - Not just for our Belle and Abby, but for the entire NPC community.
HOW YOU CAN SHINE A LIGHT: DO N ATE Please visit www.fireflyfund.org or send checks to P.O. Box 92616, Austin, TX 78709 to donate. Your donations fund research efforts that will help us find a cure sooner, saving many children’s lives.
V OLU N TE E R It takes a village. Any amount of time you can donate could help immensely. Contact us today to learn more about how you can help.
SP RE AD TH E W OR D Sharing our story on social media and with friends helps us build awareness and support.
Visit www.fireflyfund.org for more information on how you can Join the Flight.
FIREFLYFUND.ORG P . O . B O X 9 26 16 A U S T I N , T X 78 709