Rare Disease Week on Capitol Hill

Published: March 5, 2017 ~ 0

By Laura King Edwards

I spent Rare Disease Week in Washington, getting to know other rare disease advocates while learning about legislative and regulatory issues and advocating for one in 10 Americans on Capitol Hill.

I had a great two days in our nation’s capital. But to really illustrate the meaning of those 48 hours, I have to start with the end of the story.

After landing at Reagan International Airport late Wednesday afternoon, Abeona Therapeutics Vice President of Patient Advocacy Michelle Berg waited for me at a bustling Starbucks.

Our meeting almost didn’t happen, but it was easily one of the most important on my trip to Washington. That’s because Abeona Therapeutics added an AAV-based CLN1 program to its clinical pipeline in September 2016. Those fancy acronyms simply mean Abeona took on the incredible work of my friend, Dr. Steve Gray, to make his novel gene therapy treatment available to patients with infantile Batten disease.

On Rare Disease Day four years ago, I introduced Steve Gray to a crowd of Taylor’s Tale supporters at a Charlotte hotel while my mom walked the halls on Capitol Hill. That morning, I told our friends Steve’s work would lead to a clinical trial for kids like Taylor in the not-too-distant future. In my heart I knew we were starting from square one, going to war against a monster disease on a shoestring budget.

But I hadn’t forgotten the 2011 night when Mom called me on the road driving home from a Batten disease workshop in Bethesda, Maryland. She couldn’t stop talking about meeting the junior investigator from the UNC lab just two hours from our home in Charlotte. I also hadn’t forgotten the 2012 day when Mom pulled together a group of funders for Steve’s work at a conference in London.

Even then, I knew deep down that we wouldn’t save my sister’s life. But I still wasn’t willing to accept an incurable disease without a fight. And I believed my own words at that Rare Disease Day breakfast, when I told a roomful of people we’d achieve our goal.

Abeona Therapeutics is committed to taking Steve’s revolutionary gene therapy treatment to patients who need it, but our work is far from over. At Taylor’s Tale, we understand that patient advocacy is more important than ever. And that’s where my time on the Hill – along with about 400 other advocates from all over the nation – was so crucial.

A couple of years ago, our friends at the Beyond Batten Disease Foundation produced a great video about the so-called Valley of Death that prevents many scientific discoveries from translating to meaningful treatments and cures for real people. Consider:

Each year, approximately $1 billion is invested into medical research in the United States.
That investment only produces about 20 to 25 FDA-approved medicines and therapies.

The truth is that most labs don’t have the resources to shepherd their work through the drug development pipeline – and regulatory roadblocks make the journey even more difficult. Just imagine: scientists like Steve Gray and others across the country develop potentially lifesaving treatments in their labs. They take meaningful treatments and cures to mice, sheep and pigs. They publish papers and earn recognition for themselves and their institutions. But often, their work doesn’t save a single human being. And people like Taylor continue to suffer and die.

That’s why, after an all-day legislative training session in Washington last week, I celebrated my birthday by advocating for the 30 million Americans with a rare disease in the halls of Congress. I attended meetings with Representative Joe Wilson, Senator Tim Scott and the office of Senator Lindsey Graham. I talked about the benefits of the OPEN ACT, bipartisan legislation that could double the number of rare disease treatments and should have been included in 2016’s landmark 21st Century Cures Act. And as I walked the halls and told our story, I couldn’t help but think that, in a time when our country is divided on so many levels, rare disease is one issue on which we should all be able to agree.

Taylor’s Tale is a story about love and hope. It’s a story about turning hope into reality. Sometimes I get dizzy thinking about all that has happened since my family and I shared our story publicly for the first time on a cold night 10 years ago. And it’s been hard watching my sister, who used to sing and dance, grow too sick to speak or walk.

But I can see the future, and it’s shining brightly.

If you have questions about Rare Disease Week, legislation relevant to rare disease patients or our work on their behalf, feel free to contact me.

Thanks to our friends at the EveryLife Foundation for Rare Diseases, which organizes Rare Disease Week on Capitol Hill and granted Taylor’s Tale a travel stipend that made it easier for me to attend and speak on behalf of millions of Americans in Washington.

Running for Taylor in 50 States: Alabama

Published: April 3, 2016 ~ 2

By Laura King Edwards

When I crossed the finish line of Charlotte’s Thunder Road Half Marathon blindfolded in November 2013, I knew the race would be a tough act to follow. But I didn’t intend to stop running for my sister, Taylor, and our fight against Batten disease and other rare diseases. That’s why I’m running a race in all 50 states – a feat not as rare as running 13.1 miles blind but one that I hope will help me spread our story far and wide.

Last month I ran the Auburn Running Festival Half Marathon, marking state number 12 in my quest for 50. continue reading →

What I’d Buy if I Won Powerball

Published: January 14, 2016 ~ 0

By Laura King Edwards

Tonight marks the drawing for the record-breaking, $1.5 billion Powerball lottery.

I don’t normally play the lottery, but when some of my coworkers decided to buy tickets together, I thought, why not? So I threw my $2 into the pool.

And that got me thinking: what would I buy if I won Powerball? continue reading →

2013: The Memories

Published: December 29, 2013 ~ 2

By Laura Edwards

As the sun sets on 2013, our seventh full year fighting the war against Batten disease, I know in my heart that in many ways, the story has just begun.

On Jan. 4, we remembered my Grandma Kathryn, an angel in life who earned her wings on Christmas Day 2012. My grandmother, who loved her grandchildren more than she loved her own life, urged us to fight the demon from the moment we got Taylor’s diagnosis in the summer of 2006. She had no way of knowing that she, too, had a tragic neurodegenerative disease lurking inside of her. There was nothing she wanted more than an answer for the disease that dared to steal her granddaughter’s life. I fight for Taylor, but I fight for Grandma Kathryn, too.

On Feb. 28, World Rare Disease Day, Taylor’s Tale announced that we had joined an international coalition to fund gene therapy research at the University of North Carolina. Dr. Steve Gray and his mentor, Dr. Jude Samulski, are not the only talented scientists working on Batten disease. But I believe in them because of 1) what I’ve learned about the science of Batten disease in seven-plus years, 2) what I’ve been told by their respected peers and 3) something in my heart that I can’t describe. And I believe in my heart that if we can continue to fund this project, we’ll have a clinical trial for kids like Taylor in a few short years.

For the fifth consecutive year, music students and teachers held a playathon for Taylor’s Tale in Raleigh, N.C. Their efforts raised thousands of dollars and a great deal of awareness for our fight against Batten disease. I love these kids and their teachers. This event is special.

In April, Taylor’s former classmates at The Fletcher School held their second cardio craze fundraiser with local celebrity Andre Hairston. They turned a school gym into a sea of love and hope.

On June 1, I announced my plan to run the Thunder Road Half Marathon blindfolded to honor Taylor and support the fight against rare diseases.

Eleven days later, Mom spoke at the National Institutes of Health (NIH) on behalf of Hannah’s Hope Fund. Mom and Lori Sames, founder of Hannah’s Hope, delivered amazing speeches, and Hannah’s Hope gained approval from the Recombinant DNA Advisory Committee (RAC) in its quest to begin a human clinical trial for another childhood neurodegenerative disease called GAN.

This year, Taylor’s Tale also welcomed new board members. Our first- and second-year board members have made an incredible impact on our fight against rare diseases in 2013.

During the five and a half months that I trained to run 13.1 miles blindfolded, I learned more about my sister’s dark world, and myself, than I could have ever imagined. And race day, Nov. 16, is an experience I’ll never forget. No words of mine can describe what happened at the Thunder Road Half Marathon for the fight against rare diseases.

In many ways, 2013 has been the most successful year for Taylor’s Tale since my mom and I founded it with a small but determined group of volunteers in a Charlotte living room seven years ago. We connected with members of Congress and developed contacts with other key individuals. We partnered with a leading patient advocacy organization called the Global Genes Project and were adopted by a wonderful, local philanthropic organization called Playing for Others. We’ve raised more money in other years, but we’ve never extended our reach in such a big way, or had this much excitement surrounding a funded project, or harnessed the power of a great story the way we did with the run…until now.

But while 2013 has been an incredible year for Taylor’s Tale, it has been a difficult year for Taylor in every way. When we founded Taylor’s Tale, my sister was an animated, spunky, running, playing, talking, singing 8-year-old with most of her vision. But Batten disease is a demon. Taylor has a beautiful singing voice, but she can no longer talk. Her two 5K finishes inspired my blindfolded half marathon, but she can no longer walk without assistance, and her wheelchair is on order. My sister taught herself to read before kindergarten and learned Braille after she went blind, but she can’t read or write anymore. My sister can’t use a fork and spoon, because Batten disease stole her fine motor coordination. I can see the sadness in her eyes, even though her eyes can’t see me.

Batten disease has won every battle.

But it will NOT win this war.

The Greatest Race

Published: December 22, 2013 ~ 0

By Laura Edwards

Thanks to my friends at Run For Your Life, who hooked me up with a free pair of purple Saucony Triumphs and some other swag for putting together one of the biggest – and most awesome – teams at Charlotte’s Thunder Road Marathon. I also snagged a pair of limited edition, stereo-Bluetooth earphones from yurbuds.

running gear

I don’t think anyone’s ever made a shoe quite like the Brooks Glycerin (the only shoe I’ve worn in a race for three-plus years), but I’m willing to branch out in the name of purple and variety. And the earphones are just cool.

I’m already filling my race calendar for the coming year, and though I’ll never be able to match the epic journey of my blindfolded run for my sister at last month’s Thunder Road Half Marathon, I couldn’t be more excited about running for Taylor and the fight against rare diseases in 2014.

Less than a week after I kick off my sixth year of running for Taylor with the Charlotte 10 Miler, Taylor’s Tale and other organizations from more than 70 countries will recognize Rare Disease Day. On Feb. 28, 2014, the seventh annual Rare Disease Day will provide a platform for patients, patient representatives and others to raise awareness about rare diseases and the huge impact they have on patients’ lives. Since its founding in 2008, Rare Disease Day has contributed to the development of national plans and policies in many countries, including the United States. Last year, Taylor’s Tale sent two board members, including my mom, Sharon King, to Washington to attend sessions, visit with legislators and advocate on behalf of the 30 million Americans who suffer from a rare disease.

As my mom and her travel buddy, Debbie Carney, forged relationships with key decision makers and gained valuable knowledge, the rest of the Taylor’s Tale team joined with Dr. Steve Gray of the UNC Gene Therapy Center to announce co-funding for a two-year research project that, if successful, could lead to a clinical trial for children with two forms of Batten disease. Ten months later, Dr. Gray’s work is on track, and our team is focused on securing additional funding to help move the project past the first two years and toward our goal of a treatment.

My husband and I are hosting Christmas this week. This weekend, we did some December “spring cleaning” to get our house in shape for the holidays. I pulled all of my race medals down from the plastic hook on the office closet door, where I’d thrown them up in a haphazard fashion. I counted seven from 2013:

As I spread them out on the carpet, I relived each race, from a rain-soaked Charlotte 10 Miler in February to a rain-soaked Huntersville Half Marathon last weekend. I realized I set a new personal record (PR) in every race except the one I ran blindfolded. And I felt Taylor’s absence at every single one. As I sat alone on the floor and ran my fingertips over those medals, feeling the raised details of each one as a blind person would, I thought about how much my sister has declined this year.

I got faster in 2013 – a lot faster. I owe it to a good friend who ran my first race with me and helped me – a born sprinter broken by soccer – believe I could be a distance runner; to the shoes that were made for my balky ankles and feet; and to the doctor who convinced me that lower mileage and cross training might actually make me better on race day. But more than anything, I owe it to my sister, who gives me wings when my lungs burn and my body wants to quit. More than anyone else, my sister, who can no longer walk without assistance, taught me how to fly.

I have big plans for 2014, both on and off the race course. I intend to keep setting PRs. But at the end of the day, my medals are just worthless chunks of metal.

Dr. Gray and others are racing to save kids like Taylor and the millions of people fighting a rare disease.

Theirs is the greatest race of all.

The Finish Line

Published: November 14, 2013 ~ 4

By Laura Edwards

In less than 36 hours, I’ll cross the finish line of Charlotte’s Thunder Road Half Marathon, completing the biggest road race of my life. I’ll take off my blindfold and let the light come pouring in.

We won’t have an answer for kids like Taylor by Saturday afternoon. But I hope that my run, and the runs of all 50-plus people who will put on a purple shirt for Team Taylor’s Tale at Thunder Road, will help us get closer to the finish line in the race that really matters.

Thanks to all those who will help us turn Thunder Road purple for Taylor on race day.

Thanks to all of our supporters who will rock the cheer station at the final stretch and give ALL runners the boost they need to get to the finish.

Thanks to Dr. Steve Gray for dedicating his life to finding treatments that could save people like my sister. He has the talent and the passion to lead us into the light.

Thanks to my guide, Andrew, who helped me find my way in the dark.

Thanks to my family, who always believed in me.

Thanks to my sister, whose bravery inspires me every moment of every day.

It IS possible to find beauty in the midst of a tragedy. Focus on what’s good, appreciate the support of others, work hard, keep your eye on the finish line, and never, EVER stop believing.

I will run the Thunder Road Half Marathon blindfolded to support gene therapy co-funded by Taylor’s Tale at the University of North Carolina Gene Therapy Center. Donations to this cause are 100 percent tax-deductible. To support my run and our fight to develop treatments for Batten disease and other genetic diseases, click here.

Join the Taylor’s Tale team and help us turn Thunder Road purple for Taylor! Online registration is closed, but you can still register at the race expo on Friday, Nov. 15. Wear purple and run for us to help raise awareness on race day. If you’d rather cheer, click here for details about the official Taylor’s Tale cheer station on the course! Contact me with any Thunder Road-related questions.

The Reason

Published: November 10, 2013 ~ 4

By Laura Edwards

The Thunder Road Half Marathon is less than a week from today. When I closed my eyes and took my first steps as a blind runner on a middle school track on June 5, I only hoped that I would cross the finish line standing on Nov. 16. But now, with 15 blind runs under my belt, including a 10-mile run just seven minutes shy of my sighted personal record for that distance, I feel confident that Andrew and I will run a great race for Taylor and the millions of people fighting a rare disease.

With Thunder Road just days away, my attention has shifted away from training for the race to considering last-minute logistics, such as:

We have more than 40 people running for Taylor’s Tale, giving us one of the largest teams at Charlotte’s largest road race; somehow, we have to get purple Nike Dri-FIT shirts to our runners between Tuesday (when they come back from the printer) and Friday.
We’ve received local, statewide and even national media interest in our story; juggling interviews, especially for TV, with a full-time job can be like trying to put together a jigsaw puzzle with a couple of missing pieces.
The race begins and ends in uptown Charlotte; this morning, I squinted over my cup of decaf coffee at the parking map posted on the race website and tried to find the corner near the finish line where our cheer station will be located.

As much as I want our supporters to have convenient parking, I’m most concerned about my dad, who will be with Taylor on the morning of the race. I’ve often dreamed about what I will do when Andrew and I cross that finish line. I can’t even begin to imagine how I will feel.

I called my first post about this race “Run to the Light.” After 13.1 miles in the dark on Saturday, I’ll take off my blindfold; I hope my little sister is the first person I see. But last night, for the first time, I grew concerned about finding parking close enough to the finish line that my sister can make it there.

In the five months since Andrew and I began training for Thunder Road, Taylor has slipped deeper into the dark chasm of Batten disease. She struggles to walk, even with a walker. She suffers from myoclonic jerks. Batten disease has silenced a once beautiful singing voice. I don’t remember the last time my sister talked to me. I wish I’d known it was the last time. I would have savored it, or recorded it, or made a note of the date.

Five years ago, my sister ran her own triumphant race at Thunder Road. She ran tethered by a bungee cord to a sighted guide, just as I will do on Nov. 16. She stumbled and fell a few times, but she pulled herself to her feet, brushed herself off and said she could keep going. And she RAN across that finish line.

But that was five years ago. I know a lot about Batten disease. I may have majored in English, but I can describe the science of Batten disease in cold, technical terms. And I know this to be true:

My sister is dying.

I talked with a writer at a national magazine for a possible story yesterday morning. She asked me if I believe that this run, or the efforts of Taylor’s Tale, can save my sister.

I believe in Dr. Steve Gray, who will run alongside Andrew and me on Saturday. I believe that Steve and the team at the UNC Gene Therapy Center can save the lives of kids like Taylor. But as much as I believe in Steve and a handful of other talented scientists around the world working on Batten disease, I don’t know the answer to that writer’s question.

horseback riding

I do know this, though:

There will ALWAYS be another Taylor if we do nothing. Children and families shouldn’t have to endure a tragic disease with no known cure. And I believe we WILL beat Batten disease.

For me, Taylor’s courage as a runner will always live on as a symbol of her never-give-up attitude in her fight against Batten disease. Blindness kicked her and knocked her down when she ran that 5K at Thunder Road in 2008, but she pulled herself up and kept going. I won’t give up in MY fight on behalf of people like her until the day we cross the ultimate finish line.

Taylor didn’t stop running until her body gave out on her…and neither will I.

I will run the Thunder Road Half Marathon blindfolded to support gene therapy co-funded by Taylor’s Tale at the University of North Carolina Gene Therapy Center. Donations to this cause are 100 percent tax-deductible. To support my run and our fight to develop treatments for Batten disease and other genetic diseases, click here.

Join the Taylor’s Tale team and help us turn Thunder Road purple for Taylor! Click here to register for the marathon, half marathon or 5K by TONIGHT at 11:59 p.m. ET. On the second page of registration, under “Event Groups/Teams,” select “Taylor’s Tale” from the list under “Choose an Existing Group.” If you miss this online registration deadline, you can also register at the race expo on Friday, Nov. 15. Wear purple and run for us to help raise awareness on race day. If you’d rather cheer, click here for details about the official Taylor’s Tale cheer station on the course! Contact me with any Thunder Road-related questions.