What Drives Me

Published: May 2, 2013 ~ 0

By Laura Edwards

Tonight marked the fifth annual meeting of the board of directors of Taylor’s Tale. I slid behind the wheel of my car at 9 p.m. – almost three hours after I pulled into my parking spot and long after a faded sun dipped behind swollen, purple clouds outside the windows in the board room.

Four-plus years have passed since we became a public charity; six-plus years have passed since we declared war on Batten disease. The discussion at tonight’s meeting reflected the incredible progress that has been made since my mom placed a bulk order for copies of The Cure, Geeta Anand’s stunning account of how John Crowley raised $100 million in an effort to save his children from Pompe disease, and distributed them to a small battalion of hand-picked soldiers in a Charlotte living room in the fall of 2006.

Tonight, my mom sat at the head of a board room table to deliver her updates. Some of the faces around the table were the same; some of them were different.

One month ago, Mom attended the first Southeast Venture Philanthropy Summit in Chapel Hill. Other attendees included the Michael J. Fox Foundation, the Gates Foundation and sleek biotech companies of all shapes and sizes.

Three days ago, Mom and two other board members toured the University of North Carolina’s Gene Therapy Center Vector Core – the most advanced facility of its kind in the nation. Down the street, one of the nation’s top gene therapy experts, Dr. Steven Gray, is leading a two-year gene therapy study for two forms of Batten disease that is partially funded by Taylor’s Tale. If successful, the work could lead to a human clinical trial in just a few short years. And as much as we want this for Batten disease, it’s much bigger than that. If Dr. Gray gets this to work, it can treat a lot of people with all types of problems; the principles can be applied to many other diseases – from Parkinson’s disease to ALS (Lou Gehrig’s disease) to Alzheimer’s disease…I could go on. And not only that, but it will be a one-time, low-cost, minimally invasive treatment as opposed to life-long, expensive, potentially invasive treatments that – in many cases – address some of the symptoms but don’t treat the disease.

Seven years ago, I was learning the ropes of healthcare marketing and PR, coaching a girls’ soccer team, covering sports for the local paper and planning a wedding. I had a half-finished young adult novel and figured I’d get to it as soon as the honeymoon ended.

That all changed when I got the phone call.

Google “neuronal ceroid lipofuscinosis” and skim the search results. That’s how I first learned about our new world – and Taylor’s – on July 24, 2006, sitting at my desk at work, with my sobbing mother on the other end of the phone line.

The geneticist who diagnosed my sister said we shouldn’t bother with hope. My response from day one was “Screw that,” but fighting is easier said than done. It’s never been easy.

We’ve lost so much since that day.

But I’m proud of what we’ve achieved. Mom’s reports at tonight’s Taylor’s Tale board meeting embodied all that our team has accomplished and the astounding impact we stand to have.

Our fight began because of our love for one little girl. In those early days, we saw the love and the laughter and the courage that we so cherished about Taylor embodied in all of the children fighting Batten disease, and we fought for them too.

As we forged on, we learned more about the impact of rare disease: 30 million people in the United States – and 350 million worldwide. We realized that we could be doing more with the incredible scientific innovation we already have. We partnered with or endorsed organizations like the Global Genes Project and the EveryLife Foundation and went to Washington to lobby for all those fighting a rare disease.

And as we learned more and more about the wonders of gene therapy and the incredible people behind it, we realized that we could be part of something bigger than we ever imagined.

The possibilities of the immediate future and these next few years are boundless, and my mind races as I think about the impact we – the little group called Taylor’s Tale that my mom and I and a group of women who don’t like to take “no” for an answer founded in a mishmash circle of couches and ottomans and chairs over pimento cheese and egg salad sandwiches – could have – directly or indirectly – on millions of people.

That’s what drives my mind.

But in my heart, I’ll always be driven by my love for “T.”

We are the “They”

Published: June 20, 2012 ~ 0

By Laura Edwards

Today’s edition of the Boston Globe features a story about patients’ and patient advocates’ growing impact on drug development as pharmaceutical companies and the FDA respond to demands.

Every paragraph spoke to me. Every reference contained some connection to our own battle with Batten disease. Every word said, this is your story.

A Texas energy executive and a New York financial services executive, both fathers of sons with hemophilia, launched a biotechnology firm focused on a cure for the bleeding disorder because they grew frustrated with the lack of options for their children.

In 2008, 100 yards from my parents’ house, near the end of an hour-long walk fueled by passion for a treatment we could reach out and touch in our dreams but couldn’t fathom in real life, my mom and I made the decision to turn our steering committee with a couple of successful fundraisers, a website, a blog and a small group of dedicated volunteers into the non-profit organization known today as Taylor’s Tale, because we wanted more for children like my little sister.

The Michael J. Fox Foundation for Parkinson’s Research – mentioned a few paragraphs later in the Globe article – served as inspiration at a recent meeting of the board of directors of Taylor’s Tale – still fighting for kids like Taylor in 2012.

In the mid-1990s, Pat Furlong lost her two sons to Duchenne muscular dystrophy. Not long after their diagnosis, she borrowed $100,000 to finance research, posed as a doctor to get face time with Duchenne muscular dystrophy experts and pleaded her case with drug company execs.

Pat Furlong and my mom, Sharon, have a lot in common. They share the same vision. They have the same fearlessness. The same bulldog mentality. In the past several years, my mom has turned to Furlong more than once for advice.

One of the drug companies mentioned in the article, Genzyme, developed a drug ignited by a father who wanted desperately to save his children from Pompe disease – and would stop at nothing to succeed. The father is John Crowley; his story is chronicled by former Globe writer Geeta Anand in the book “The Cure” and, later, the movie “Extraordinary Measures.” At the first meeting of the steering committee that eventually became Taylor’s Tale, my mom gave a copy of “The Cure” to each of the women seated around the room. She gave us two assignments that day: to read the book, and to fight with her, no matter how tough the road might seem; because she believed, she hoped we could believe, too.

I posted a link to another interesting article – this from the Chicago Tribune – on our Facebook page last month. This story focused on parents of children with giant axonal neuropathy (GAN) who hired researchers to develop a treatment. Like Batten disease, GAN is a neurodegenerative disease – though it is far more rare (only 25-30 known cases worldwide, compared with at least 500 known cases of Batten disease in the United States alone). And yet these parents succeeded in raising enough money to put researchers on track for human clinical trial in the near future.

The Tribune article contained a quote from one of the GAN parents that has stayed with me since the moment I read it. In the six years since Taylor’s diagnosis, I’ve never heard anyone describe the existence of the close family member of someone with a rare disease so well:

“After Ethan was diagnosed, people would say to us, ‘Don’t worry, they’ll find a cure,'” Tkalec said. “And I’d say, ‘You don’t understand … there is no ‘they.’ We are the ‘they.'”

She’s right – we are the “they.” For as grateful as I am for the support we’ve received over the years – from building our non-profit organization to supporting it to offering friendship and, on rough days, a shoulder to cry on – I know that ultimately, this fight is ours to fight; that if we don’t fight, no one will. That the minute we stop fighting – the minute we stop believing – that’s when the mountain will become insurmountable.

To Believe

Published: January 30, 2012 ~ 10

By Laura Edwards

The following post is part of a blog hop organized by the R.A.R.E. Project to raise awareness of World Rare Disease Day (February 29).

On the morning of July 24, 2006, my parents sat stone-still in an office in Charlotte, NC as a genetics expert explained that their 7-year-old daughter, Taylor, had been born with something called infantile neuronal ceroid lipofuscinosis (INCL), a form of Batten disease. The doctor prescribed family trips, fun activities, etc. – anything to help make happy memories while there was still time. The disorder was fatal and had no cure.

An hour later, my brother, husband, and I answered my parents’ distress call and joined them at their house. While Taylor sat in her classroom at school, unaware of the tragically flawed single bead on the single gene out of the thousands of genes in her DNA, the rest of us sat on the floor of my parents’ bedroom, wrapped in each others’ arms, soaked in each others’ tears.

Another hour passed. The sun scaled the cloudless blue sky and baked the mid-summer Carolina landscape. We wordlessly walked to my SUV, drove to Taylor’s school, loaded her into the back seat and headed to her occupational therapist’s office. During her appointment, the five of us sat huddled in the car with the windows rolled down, unable to move. The July heat rose from the asphalt in shimmering waves. And then, without warning, the shock that had frozen our resolve melted away.

In the immediate aftermath of the diagnosis, we’d been unable to see past the doctor’s prescribed happy memories program. Unable to believe. Happy memories are nice. But for our family, they just wouldn’t cut it. That’s why we decided – as a team – to fight the monster that haunted Taylor’s DNA. “No cure” didn’t cut it, and we made no apologies for our demanding ways. And at that very moment – in that oven-like car – the spirit that eventually became a non-profit organization called Taylor’s Tale was born.

How many things do we take for granted today that were once considered impossible?

Ancient Egyptians wrote of “electric fish” almost 5,000 years before Benjamin Franklin attached a metal key to a damp kite string and flew the kite in a stormy sky. About 100 years later, Thomas Edison opened a lab in New Jersey, where he built the first incandescent electric lamp. The first telegraph was an iron wire. Today, we can have conversations with people on the other side of the globe. The first computer was the size of a warehouse. Now, smart phones are more powerful than those computers.

On December 14, 1903, Wilbur Wright flew an airplane over the sands of Kitty Hawk, NC for a whopping 3.5 seconds. The plane was constructed of wood and canvas and had a 12-horsepower engine. Sixty-six years later, men landed on the moon, and fighter jets flew across the sky at more than twice the speed of sound.

In the first half of the 20th century, frequent polio epidemics swept across the world, killing hundreds of thousands of people. But a vaccine developed in the 1950s reduced that number to around 1,000 per year. For much of human history, the elderly or weak had no answer for illnesses as simple as the common cold. But an accidental discovery in the 1940s – the antibiotic penicillin – has since saved millions of lives.

In 1998, two siblings were diagnosed with a rare, genetic disorder called Pompe disease. Doctors told their parents the children likely had just months to live. It wasn’t just that they hadn’t caught the disease early enough. The disease was so rare, in fact, that no scientist had even bothered to explore possible treatments for it.

Those parents were John and Aileen Crowley. Their story became The Cure, an award-winning book that later inspired the movie Extraordinary Measures. Before the story made its way to the big screen, though, it made its way to my mom, Sharon King, who shared it with a group of about 10 women – who, on a sunny day in late 2006, each took a copy of The Cure and enlisted in my mom’s newly created army with one mission: to fight Batten disease.

Years after doctors thought they’d be gone, the Crowleys’ kids, Megan and Patrick, are still fighting. And so are we.

As a matter of fact, I’ve never once looked back or considered quitting since the day my family made that promise to each other in a hot car or the day just a few months later when my mom – our fearless leader – sounded her battle cry over a crate of hardcover copies of The Cure. And while I know we have an uphill battle in our quest to save my sister Taylor’s life and make the world a better place for all those who suffer from a rare disease, I still believe.

Because to truly believe is to understand the difference between impossible and undiscovered.

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World Rare Disease Day is February 29 – thirty days from today. Thirty is a significant number because:

Thirty million Americans have a rare disease – that’s more than the total number of people living with cancer WORLDWIDE (28 million according to the Livestrong Foundation)!
Thirty percent of children with a rare disease will die by their fifth birthday.

To hear more shocking statistics and blog hop to read more stories of people fighting rare diseases, click here.

Search for the Light

Published: October 14, 2011 ~ 2

By Laura Edwards

Autumn has arrived in Charlotte. The tops of the trees outside my office window are a fiery red, and the nights are cool.

Five autumns ago, my mom went on Amazon.com and ordered hardcover copies of Pulitzer winner Geeta Anand’s The Cure. They arrived in a box one late afternoon as the first crimson and gold leaves settled on my dad’s emerald green lawn; she must have ordered 20 copies.

The Cure, which last year became a movie called Extraordinary Measures, tells the story of John Crowley’s fight against Pompe disease, a severe neuromuscular disorder that affects his children, Megan and Patrick. At the time of the diagnosis, Pompe had no cure – and no treatment. But Crowley and his wife, Aileen, refused to accept the death sentence Pompe prescribed for Megan and Patrick.

John Crowley relentlessly fought for his children’s right to live. He quit his job, invested everything he had in a biotech start-up and went on a worldwide hunt for the scientist who could save Megan and Patrick’s lives. Along the way, some questioned his ethics. Always, doubters marveled that he could risk everything for two children destined to die young.

For much of the journey, no one believed in Crowley but Crowley. But after a long, hard-fought effort that encountered many setbacks, his children received enzyme replacement therapy – and bucked every dire prediction.

Several days after that box of books arrived on my mom’s doorstep five autumns ago, a small group of women, myself included, huddled around her in a Myers Park living room. Between us sat the box. I listened and watched as my mom declared war on Batten disease and urged the rest of us to join her on the battlefield. My mom’s voice cracked as she described our opponent, but her resolve never wavered.

Taylor and Mom

After my mom finished speaking, I took a copy of The Cure for myself and gave one to each of the other women. And that very moment, Taylor’s Tale was born.

This week, two of the women in the room that historic day – my mom and Taylor’s Tale’s new advocacy chair, Callie Alley, flew to Washington, DC for the first annual US Conference on Rare Diseases and Orphan Products. The highlights of their visit included the opportunity to hear John Crowley speak in person. The story, though no longer new or foreign to those of us who have been fighting all this time, still inspires us. And my mom still inspires me.

My mom is battle-worn. But she is not battle-weary. A little more than two months into her new role as president of Taylor’s Tale, and five years and three months into a parent’s worst nightmare, she is still the same fearless leader our team – and the entire rare disease community – needs.

Thank you, John Crowley, for setting the bar for families who aren’t willing to settle for “no cure.”

Thank you, Mom, for showing us the way as we march onward in our search for the light.

Extraordinary Measures

Published: January 17, 2010 ~ 0

By Laura Edwards

Shortly after Taylor was diagnosed with Batten disease in the summer of ’06, my mom bought a box full of copies of The Cure, the bestselling book about John Crowley and his fight to save his children from a fatal disease. She gave copies of the book to friends and asked them to fight with her. And from that simple act, Taylor’s Tale was born.

Now, the Crowleys’ amazing story will be told on the big screen. Extraordinary Measures, starring Harrison Ford, Brendan Fraser and Keri Russell, opens on Friday, January 22. If you’ve supported Taylor’s Tale and/or followed our story over the past three-and-a-half years, please consider going to see the movie with friends and using it to help share the story of our own journey and fight for a cure for Batten disease.

You can also help support Batten disease research by visiting the movie’s website and casting a vote in support of a video posted by another family who has two children fighting the disease. Click here to visit the Inspirational Quilt and type ‘noah’ in the search box to watch the video. You can vote every day! Thanks in advance for supporting this special family and the quest they share with Taylor’s Tale and our family to find a cure for Batten disease.