A New Partner for the Push to the Summit

Published: August 19, 2020 ~ 1

By Sharon King

In a different version of our story, Taylor may have celebrated her 22^nd birthday with us today.

Jim and I have wondered so many times in recent weeks: What would our daughter be doing if Batten disease had not cut her life short on an early fall day in 2018? She may be a recent college graduate figuring out a new job or starting graduate school while the world reacts to a pandemic. She may have someone special in her life. She may be living at home for a while or making her own home. We talk about how much she would love her nephew, Jack. We smile when we remember her bubbly, magnetic personality. Our youngest child was, after all, an extrovert in a family of mostly introverts.

Oh, how we wonder …

In the beginning, Taylor’s life held much hope and promise. That all changed, of course, when a flaw in her CLN1 gene surfaced shortly before her eighth birthday in 2006. Batten disease sentenced her to blindness, seizures, cognitive and motor impairment, and an early death.

Shortly after Taylor’s diagnosis, we set out to change the curve. We were told it would be close to impossible to develop a treatment for an ultra-rare disease. We chose to believe otherwise. Because we had to try — for Taylor.

We funded a handful of research projects for about five years. Then, we met a young researcher named Steven Gray with a big idea: gene therapy as an approach to replace the flawed gene causing CLN1 disease, Taylor’s form of Batten disease. Dr. Gray’s intellect was apparent, his passion infectious. We believed in him. And in 2013, we funded him.

Friends, family, neighbors … so many people shared their time and treasure to help us scale the mountain. Somehow, we climbed many mountains. We did it together: When one group of climbers finished their part, the next group said, “We’ve got this,” and kept climbing higher.

Dr. Gray’s research data was strong, and a few years later, it was time for a new partner to step in. In 2016, the work was licensed to Abeona Therapeutics. Abeona further developed the program, receiving an Investigational New Drug (IND) allowance from the FDA in May 2019. This allowance clears the way for a clinical trial, but the climb stalled. Taylor was already gone by then, but we were still pushing, clawing, fighting to keep our promise to her and help families like ours.

This week, Taysha Gene Therapies — where our friend Dr. Gray is now chief scientific advisor — announced the acquisition of the CLN1 disease program from Abeona Therapeutics. Taysha will move forward the IND and begin the long-awaited clinical trial in the near future.

It was incredible news to begin Taylor’s birthday week, and I have no doubt that my daughter is up there, pulling for us, pulling strings, making magic happen.

Taysha President, CEO and Founder R.A. Session II said “Taysha is built on a powerful foundation: a combination of proven science, accomplished colleagues and an unrivaled alliance with a gene therapy powerhouse. Alongside Steven Gray, Ph.D., Berge Minassian, M.D., and our additional brilliant partners at the UT Southwestern Gene Therapy Program, we are seamlessly building an integrated engine for new cures.”

I’m overjoyed that Steve Gray is once again part of reaching the summit for a CLN1 disease program that began with hope, a dream and people who cared enough to believe with us. While it wasn’t in time for Taylor, I’m certain the timing of this week’s announcement is her birthday wish from afar.

Thanks for your support over the years. We haven’t reached the summit, but Taysha will be a strong and able leader to help us get there. The final leg is a journey worth making for children who have no treatment options.

Children like Taylor.

One Year Later: Notice Served

Published: May 21, 2020 ~ 1

By Sharon King

There is so much love in this photograph. A family wrapped around its youngest member as she celebrates her achievement in completing a second 5k — and improving her time. You might be thinking this isn’t such a big deal, but Taylor was blind and beginning to suffer additional effects of CLN1 disease (Batten disease). She was a winner that day, as she was so many other days in her short life.

Taylor’s efforts inspired her older sister to begin running. When Batten disease stole Taylor’s ability to run, Laura took the baton and ran for her. Laura is still running for Taylor today, even though Taylor left us 20 months ago.

Imagine the photograph without Taylor. There is such a hole in the middle of our family, and we’ll never be able to fill it.

You haven’t heard much from Taylor’s Tale in the past year. We’ve spent a lot of time trying to figure out life without Taylor. My daughter finished her race, but I’ll never say that Batten disease won. It’s still running, and Taylor’s family and so many others are now carrying the baton on her behalf. Batten disease only wins if we quit before we reach the finish line.

I searched “Tips for Winning a Race” and found the following suggestions from WikiHow:

Go slow at first, then pick up your pace during the end of the race.
Keep most of your energy until the last lap.
Winning a race is a lot about the training and preparation as well as how skilled you are but that’s not everything. Winning a race has to do with the confidence, competitiveness and mindset of the runner, swimmer, biker, etc.
Without the heart and the will to win one will never win any race.

A year ago today, I thought we were coming up on a significant mile-marker — not yet the finish line, but the glimpse of something worth cheering about. On May 21, 2019, Abeona Therapeutics made an announcement:

… cleared to begin a Phase 1/2 clinical trial evaluating its novel, one-time gene therapy ABO-202 for the treatment of CLN1 disease, following acceptance of its Investigational New Drug (IND) application by the U.S. Food and Drug Administration.
Abeona Therapeutics

In other words, they were cleared to begin a clinical trial. Yet one year later, we are still waiting on the trial to begin. Word to the wise — just because you see the goal over the horizon, doesn’t mean you can slow down.

I’m looking at you, Abeona. I’m watching. And I’m not watching idly.

… pick up your pace during the end of the race…keep most of your energy until the last lap …

So, I’m asking myself, what is the mindset of top athletes? I believe it is the realization that whatever adversity they face, they still have the ability to be successful.

Confidence…

Taylor faced so much adversity — more than her fair share, for sure. Meanwhile, she never complained. She found her way.

We can find our way through this difficulty, too, because we have the heart and the will to win …

I’m a mother with high expectations (I’ve been in training for 14 years, and I’ve earned the right). From here on out, I intend to be fierce. Relentless. Take-no-prisoners tough.

For her.

For all of the children and families living with CLN1 disease, now and in the future.

Notice served.

Our Dream is Coming True

Published: May 26, 2019 ~ 0

By Laura King Edwards

I’m thrilled to share news of an incredible milestone achieved last week: FDA clearance of an investigational new drug (IND) application for a novel, one-time gene therapy to treat CLN1 disease (a form of Batten disease). This gives Abeona Therapeutics the green light to begin a Phase 1/2 clinical trial evaluating the treatment in patients. You can read the entire press release on Abeona’s website here.

Though we’re proud of everything we’ve done for children and others like Taylor, this is why we founded Taylor’s Tale more than a decade ago: to spearhead and enable the development of a viable treatment for CLN1 disease. So many called it an impossible mission, but we believed enough to try, even after it became clear Taylor wouldn’t survive long enough to benefit from such an achievement. The photo above was taken in the lab of Dr. Steven Gray, our partner and friend who developed the technology, on the day we celebrated the first birthday of mice treated with his innovative gene therapy approach. It was a happy day and a sign of more good things to come.

We’re grateful for Steve as well as Ale Rozenberg, who played an instrumental role in moving Steve’s work forward, and the entire team at Abeona Therapeutics for all they’ve done to guide it to the clinical stage. Thanks also to you, our donors, friends and volunteers, for your gifts of love, money and time. Our dream is coming true, but we couldn’t have done it alone.

Though this is a significant win, our work is far from complete, and I hope you’ll stay on the journey with us. To learn how you can continue supporting Taylor’s Tale, send us a note.

Global Genes Summit Unites Rare Disease Voices

Published: September 28, 2016 ~ 0

By Judy Mayer

You might assume that a summit dedicated to rare disease would be a negative experience, perhaps even bordering on tragic. You would be wrong.

The Global Genes RARE Patient Advocacy Summit held in Huntington Beach, California, on September 24-25 illustrates the positive energy that defines the rare disease community. Some people showed up in wheelchairs, while others had masks across their mouths and noses. One teenage girl brought her service dog, and one woman brought her oxygen tank.

While rare diseases create a seemingly endless variety of challenges, the summit participants all share the determination to help others and to leave no stone unturned to improve the lives of rare disease patients. continue reading →

Why a “Miracle” in Ohio isn’t the End of this Story

Published: May 3, 2016 ~ 4

By Laura King Edwards

The tireless team at CureBatten is in the national news again, this time for funding the launch of a clinical trial at Nationwide Children’s Hospital in Columbus, Ohio.

My heart is full of hope for Charlotte and Gwenyth Gray, daughters of Hollywood film producer Gordon Gray and his wife, Kristen. I wish I could put into words how much I want this treatment to save their beautiful little girls, Charlotte and Gwenyth. I wish I could explain how much I want the work made possible by CureBatten to spark progress for other devastating neurological diseases, including the many forms of Batten disease that still do not have a treatment. continue reading →

Gene Therapy: The Future is Now

Published: October 22, 2015 ~ 0

By Laura King Edwards

Predicting the future is a messy game, but “Back to the Future Part II” got a lot of things right (while we don’t have “Jaws 19,” we have plenty of big-screen TVs).

I was 7 years old when Marty McFly and Doc Brown traveled to Oct. 21, 2015 to save McFly’s kids. I dreamed about flying cars and robots and wondered how Nintendo managed to fit so many pictures on a single 8-bit video game cartridge. continue reading →

Gene Therapy and a Birthday Party

Published: June 20, 2015 ~ 4

By Laura Edwards

Every time I visit Steve Gray and the University of North Carolina Gene Therapy Center, I want to run home and immediately write a blog post and email all of my media contacts and friends in the rare disease community and tell them what’s happening in a lab two hours from my hometown. I want to tell them Steve and his team are going to change the world.

But when I get home, I can’t find the right words, and instead I just scroll through my pictures and reread my notes and wonder how I can possibly feel so inspired knowing we’re too late to save my little sister. continue reading →

Some Place I Can’t Describe

Published: November 18, 2013 ~ 24

By Laura Edwards

After months of training, planning and anticipation, it arrived: Charlotte’s Thunder Road Marathon, and my planned attempt to run 13.1 miles blindfolded to honor my little sister, Taylor, and support the fight against Batten disease.

On Friday, my colleagues at a creative marketing communications agency threw a purple-drenched pep rally, complete with the theme song from “Rocky,” a gift to Taylor’s Tale and an appearance by my husband, John (who schemed with them to plan the surprise).

At the race expo, I traded hugs with my former colleagues at the healthcare organization sponsoring the race and runners wearing purple for Taylor’s Tale on race day.

Friday night, Dr. Steve Gray, a UNC Gene Therapy Center expert whose lab’s Batten disease research is co-funded by Taylor’s Tale, arrived in Charlotte for the race.

Finally, race day arrived. John, Steve, my mom and I picked up Andrew Swistak, my sighted guide, and arrived in uptown Charlotte before sunrise. I did an interview with News 14 Carolina and took a couple of photos for Society Magazine.

Andrew, Steve and I headed to the start line just as the morning’s first sunlight painted the tops of the skyscrapers. And at 7:15, I took one end of a green bungee cord, pulled down the blindfold bearing my sister’s name and ran into darkness.

We got off to a slow start for the first few miles due to the policeman driving the pace car and charged with keeping the early starters at bay. We even took a wrong turn at one point when the pace car couldn’t keep up with us and had to wait at a busy intersection for the light to change before we could cross. But Andrew and Steve took it all in stride; a few miles in, the course opened up for us, and we picked up the pace.

Auditory cues mean so much more, and are so much more acute, when you can’t see. I loved hearing the reactions of people lining the streets to cheer on runners. First, they cheered for us as they’d cheer for any runner they didn’t know. Then, they’d notice something different about us and go silent before crescendoing into a loud roar. It was incredible to experience, and it gave me an extra kick. Several times along the course, we passed people who knew me or knew our story. I didn’t recognize all of them, but along one quiet neighborhood street, my good friend, Amy, surprised us. I recognized her voice as soon as she called my name. So much of human emotion is expressed in the eyes, and a thick blindfold concealed mine, but I hope she knew how much it meant to me to hear a familiar voice at that very moment.

A few weeks ago, during my longest blindfolded training run with Andrew, I ran untethered for a short period. During the race on Saturday, Andrew cut me loose a few times. Around mile 10, I ran without my guide for what felt like an eternity. I never felt closer to Taylor than during that stretch. I imagined her next to me, healthy, her legs in sync with mine, her voice dancing on the wind, her eyes drinking in the earth.

Just a short time later, we approached the Taylor’s Tale cheer station near the final stretch. Once more, Andrew took the bungee, and I ran past a screaming, adoring crowd. Their voices melted the cramps in my legs and filled my heart with love. In front of the station, I made a 90-degree turn on Andrew’s spoken direction alone, and we headed to the finish line. As we did, 70 teenagers clad in purple tutus, pompoms, sparkle and glitter took off after us. And as I hurdled over the first timing mat, then the second, and Andrew pulled me to a stop, and I lifted my blindfold and let the light come pouring in, I melted in the arms of my mom, who stood waiting for me at the finish line, crying, and the kids surrounded us, closing us off from the outside world, and suddenly, even though I had a medal around my neck and a timing chip on my shoe, I wasn’t at a race any longer, and I didn’t care that I’d just run a half marathon blindfolded. I was somewhere else, some place I can’t describe or ever return to again except in my dreams.

I ran 13.1 miles in the dark, but I didn’t take a single step alone.

We built Taylor’s Tale from the ashes of a tragedy that tried to burn my family to the ground. And Batten disease is the saddest thing I’ve ever known.

But Taylor’s Tale is not a sad story. Taylor’s Tale is a story of love and hope. And as I ran the final steps of Thunder Road, flanked by living angels and guided only by Andrew’s voice and Taylor’s courage, I knew:

Batten disease may have cast a dark shadow on our world, but I was running to the light.

I believed.

And I felt free.

Note: I ran the Thunder Road Half Marathon blindfolded not only to honor Taylor’s courage and raise awareness of rare diseases, but also to support Dr. Steve Gray’s gene therapy research 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 our fight to develop treatments for Batten disease and other genetic diseases, click here.

Thunder Road: The Details

Published: November 7, 2013 ~ 3

By Laura Edwards

I hatched the crazy idea to run a half marathon blindfolded for my little sister all the way back in the spring, and I ran blind for the first time five months ago. I’ve never spent this much time training for any race, and I can’t believe the big day is almost here. But in just nine days, I’ll put on a blindfold and run the most important 13.1 miles of my life to honor Taylor’s courage and support the fight against rare and genetic diseases. A lot of you will be part of the effort on race day – as a runner, cheerleader or supporter from afar. With some help from the Taylor’s Tale team and our friends at Run For Your Life, I’ve been tying up loose ends behind the scenes between training runs, media coordination and regular life stuff, like work and sleep. Check back throughout the next week for last-minute details, but the following should provide most of the information you need to be part of the effort on Nov. 16:

If you’re running:

You have until Sunday, Nov. 10 at 11:59:59 ET to register online here. If you miss the deadline, you’ll have to register at the race expo on Friday, Nov. 15. There is no race day registration. IMPORTANT: on the second page of online registration, under the option that says “Event Groups/Teams,” select “Taylor’s Tale” as your team. Note: if you already registered and did not select Taylor’s Tale, contact me using this form.
Thanks to the generosity of two donors, we will have the ability to provide Nike Dri-FIT shirts in Taylor’s Tale purple to the first 40 runners who register to run for Taylor’s Tale. If you have officially registered to run, please contact me using this form with your preferred shirt size (men’s sizes only) and the best way to get in touch with you on Friday, Nov. 15. We will establish a pickup location for the shirts. If you cannot coordinate a pickup or are not one of the first 40 runners, please try to wear something purple and help us “turn Charlotte purple” for Taylor on race day.
If you are running the half marathon, you are invited to join my guide (Andrew Swistak), Dr. Steven Gray of the UNC Gene Therapy Center and me. I will receive an early start time (7:15 a.m.) for safety; the official start time is 7:45. Our estimated pace is 9:00/mile, and we expect to be alone on the course for approximately eight miles before the leaders catch us. If you start early, you will not be eligible for awards.
If you are running the half marathon, the crowd at the official Taylor’s Tale cheer station will give you one last boost of energy on the final mile, near the corner of Martin Luther King Jr. Boulevard and S. McDowell Street. Keep your eyes and ears peeled for a bunch of people wearing purple and jangling cowbells!
Optional: remember why you run! Shirts will be screen printed with “Team Taylor’s Tale” and our website on the back. Save a few breaths to tell fellow runners and spectators about Taylor’s courage and our fight against rare diseases.

If you’re cheering:

Taylor’s Tale will have an official cheer station sponsored by Michelob Ultra. It will be located on mile 12 of the half marathon course near the corner of Martin Luther King Jr. Boulevard and S. McDowell Street. This is a prime location about one-third of a mile from the finish line. View a course map here.
Supporters will receive free Michelob Ultra and cowbells.
Arrive by 8:30 to get settled and ensure that you don’t miss Andrew, Steve and me as we run by en route to the finish line!
IMPORTANT: Runners will vote on the best cheer station, and the winning station will receive $1,000 to donate to charity! You can help us win $1,000 for Taylor’s Tale and the fight against Batten disease. To learn more about the contest, click here.

If you can’t make it to Thunder Road:

Running a race blindfolded to commemorate the five-year anniversary of Taylor’s first 5K is the best way I know to honor her determination in the face of the world’s most tragic disease. But being part of finding the treatment that saves people like Taylor would be the ultimate way to honor my sister and others like her. Even if you can’t make it to Thunder Road, you can be part of the fight to save lives.
I’m running Charlotte’s Thunder Road Half Marathon to support gene therapy research at the University of North Carolina at Chapel Hill (UNC). We announced funding for this important work on Rare Disease Day this year (Feb. 28) along with a coalition of other non-profit organizations, all founded by families fighting Batten disease. This work holds incredible promise for not only Batten disease, but many other devastating illnesses including Alzheimer’s disease, Parkinson’s disease and ALS (Lou Gehrig’s disease). If funding continues, we could have a clinical trial for kids like Taylor in just a few short years.
To make a gift and help keep the dream alive at UNC, click here. You can make a gift online or by mail. Taylor’s Tale is a 501(c)3 non-profit organization, and gifts are 100 percent tax-deductible.

That’s it for now, but stay tuned for last-minute details. I hope you can join us on the road!