Gray Family

01.10.2024

In November 2016, as my six-year-old Mila lay weak in the hospital from a battery of tests, I raced through photos, articles and scientific papers online to find the answer to what was happening to my daughter. I had combed the internet for years with no luck, but just then, in the dark of the night with Mila in the hospital bed beside me, I came across a video of Charlotte Gray.

Like Mila, she was born at the end of 2010, had thick silky brown hair, big brown eyes and a mischievous laugh. Like Mila, I watched as she zipped around on her scooter, dove into the pool and smiled wide for the camera. Like Mila, she had taken a mysterious turn and began a rapid decline. And then she and her little sister Gwenyth were diagnosed with the fatal condition, Batten disease.

It was on the video when she tripped, holding her mother's hand, that made my heart stop. As she fell in an unusually rigid way, it was like watching Mila. The interview with her parents that followed drove the pain even deeper. "Most children don't live beyond the age of 12", Charlotte's mom Kirsten said. "Children lose their eyesight and become blind. Then they can't talk or walk, and they're basically bed-ridden. They're given a g-tube because they can't feed themselves because they lose their ability to eat. And then they die.", she continued, tears running down her face as she fought to speak.

Just days after watching the video, Mila too was diagnosed with the same devastating disease. Both girls were destined to die. 

Charlotte's parents raced against time to stop their girls' disease and started one of the very first gene therapies to the brain. Because of their help and friendship, I was able to fight for Mila. Both girls were fortunate enough to be among the few who received treatment. But too many dominoes had fallen by then. They would become the pioneers who are helping those who follow. 

A few weeks ago, Charlotte turned 13. She is the age Mila would have been today.

01.12.2024

Gwenyth Gray is 10 1/2 years old. She smiles as her mother gently, but firmly supports her growing body as she takes slow steps toward the living room where her younger brothers await.

Gwen was just 3 when she received one of the first ever gene therapies to the brain which her parents fought day and night to start, raising the funds and putting together the scientists and doctors to pull it off. Some symptoms had already begun, but the hope was it would be in time for Gwen.

Today, Gwen is in a place far better than where she should be given her disease. Fourteen other children with Batten CLN6 were part of her trial which a company took over in order to commercialize it and make it accessible to many more. But the regulatory pathway and financial system weren't designed for biotechs pushing treatments for the small populations of rare disease. And so this medicine landed on a shelf for years, and with it the hope for the next child diagnosed.

For genetic treatments that exist today, we are still learning the most effective dose, route of administration and age to stop each disease in time. In these early years, it is the children like Gwen, her sister Charlotte and Mila who will teach us. It is only with their treatments that we will learn and improve.

01.16.2024

Mila was my first child. Like other new mothers, my time and energy moved away from taking care of myself to taking care of my baby. I nursed her, I bathed her, I rocked her to sleep. Then she grew and became more independent. She could walk and run and navigate the house on her own. She spoke and told me when she was hungry, tired or sick. She could feed herself, sit on the toilet, climb into her bed and fall asleep alone. But then, around four years old, she started to regress.

Just as I returned to holding Mila's hand again when she walked, cutting up her food into small pieces again and sitting her in a stroller to get through the grocery store, Kristen Gray was doing the same for her first child, Charlotte. Neither of us understood why our daughters were going in reverse. There was no taking care of ourselves. Our girls depended on us more and more by the month. 

Our answers came when both of our young daughters were diagnosed with Batten disease and we were told that all they learned they would soon forget.

As months and years passed, our relationship with our girls returned to their infancy where we held them in our arms and carried them around our homes, we changed their diapers now in sizes too large to find in stores, and we wiped the saliva from their mouths as they could no longer lift their arms to do it themselves.

02.06.2024

When Mila was diagnosed with a disease I was told was impossible to survive, I immediately wanted another child. But I stuffed that yearning into a box, closed it and pushed it to the back of a dark closet so I could fight for Mila. It was out of sight, but never gone. Toward the end of Mila's life, I reached back into the closet, opened the box and pulled out the longing, burning stronger than ever. Perhaps I wanted another child to fill the hole in my heart that was coming. Perhaps to ensure that Azlan wouldn't be left alone in this world, carrying the pain of what he had lived through. Perhaps to try to bring Mila back.

For better or worse, life as a single mom did not allow me to live my dream. But for parents like me, this decision often comes at an age and time that make having another child seemingly impossible.

Like me, Kristen Gray and her husband navigated this difficult path while at the same time fighting for Charlotte and Gwenyth, their only two children, both with a disease no child had ever survived. When the girls were 6 and 8 years old, they finally had a healthy little boy named Cal, and a year later another, Gavin. 

A new life of immense joy with the backdrop of immense sadness. 

Another unspoken fight in the lives of families like ours.

02.09.2024

In the last few years of Mila's life, when she woke up in the morning, she would lie in bed and wait until I came in to carry her downstairs. I put my hands on her, hugged her, sang to her so she never felt alone. But I was plagued by the possibility that she felt trapped, confused and scared.

Unlike Mila, Gwenyth Gray still has her vision and occasionally climbs out of bed and crawls down the hallway. She wakes up in the morning and opens her eyes to sunlight and the room around her. Gwen received one of the first gene therapies to the brain when she was just 3 years old, and 4 years later began taking a promising off-label medication. She is able to watch cartoons, use her hands to feed herself and interact with her little brothers. She smiles and she laughs.

In these early years of learning from genetic therapies, those fortunate enough to access a treatment are often searching for other complementary approaches. For even the luckiest of rare disease parents where hope still exists, the fight never seems to end.

02.16.2024

At the end of Mila’s life, when she could no longer remove a fly from her nose and her brain was losing volume, I was forced to think what Mila would want. A decision no parent should ever have to make.

Like me, Kristen Gray finds herself in the impossible place of having to think for her daughter Charlotte, who has lost nearly all of her abilities and can no longer tell her mother how she feels.

Sleeping with her one-year-old son on the sofa just outside Char’s room, Kristen jumps up and rushes to her daughter’s side numerous times every night to suction the saliva from her mouth and ease her back to sleep.

Each day, despite little to no sleep, Kristen and her husband get back up on their feet and face a full day of school drop-offs and pick-ups, therapies, doctors appointments, and jobs. They do it because they must.

Some mornings, there is a brief quiet moment to reflect, but just like that it’s gone and it’s time to do it all over again.