Helen's T Cells Are Collected: An Update on Her Treatment

We couldn’t go to sleep tonight without sharing how Helen’s day went.

Today, Helen underwent apheresis, the procedure to collect the T cells needed for her experimental treatment. The team hoped to collect 3 billion T cells. They collected 4.66 billion!

It went as well as we could have hoped. Helen was an absolute warrior. She showed bravery and strength, as she always does. She’s our hero, and we are so proud of her. We hope she understands us when we tell her that.

The support surrounding her today was extraordinary. St. Hubert Catholic School put away the uniforms and showed up in pink. Teachers at Jonathan Elementary, Helen’s new school, wore pink. Arctic Wolf went pink. Researchers visited Helen, Grandpa spent the day with Helen, and her new teacher stopped by to surprise her. We received prayers, texts, pictures, dinner, and flowers, and came home to the sweetest note from Allison, our nanny. Steph and I felt every bit of it. Thank you.

How We Got Here

We haven’t shared much about what led to today or what comes next. The truth is that there are so many unknowns, and sometimes it feels safer to stay quiet until something is certain. But you have helped bring Helen to this moment, and you deserve to know where things stand.

In June, after months of work with the FDA, Helen’s team received approval for an N-of-1 trial: an experimental treatment designed for her. It took tremendous work by scientists, physicians, advisors, and supporters to get here.

On Monday, Helen had a temporary central line placed. Today, her blood passed through an apheresis machine, which collected T cells and returned the rest of her blood to her body. The procedure took about five hours. Afterward, Helen was sedated again and the temporary central line was removed.

What Happens Next

Her collected cells are in transit to Case Western Reserve University in Cleveland. There, the team will engineer Helen's T cells using a drug product developed by Kommodo Therapeutics to produce TPP1, the enzyme Helen’s body lacks. The hope is that the cells can act as tiny “micropharmacies,” making TPP1 that other cells can use. Over the next several weeks, the cells will be grown, frozen, and put through safety testing. If everything proceeds as planned, Helen will receive them by infusion in late October or early November.

This has worked in the lab, but it has never been tried in a person. We don’t yet know how long the cells will last, how much enzyme they will produce in Helen, or how widely it will reach the places she needs it. We won’t know until we try. So we will try.

What We Hope For

We don’t often let ourselves imagine what success looks like. The emotional risk feels too great. But we pray this treatment can slow or stop what Helen is losing every day. If it doesn’t work as intended, we’ll learn from it, work with the scientists on what needs to change, and keep going. If it does work, we’ll do everything we can to help bring that possibility to other children with CLN2 Batten disease. And we’ll keep asking the next question: how do we help these children regain what this disease has taken?

Many of the messages we received today had a common theme, saying we should be proud of what we’ve accomplished. We're grateful beyond words for our supporters, our board, and the scientists and physicians who brought us here. Eighteen months ago, we would have given anything to reach this day.

But Helen can no longer talk. She needs our hands to help her walk. She has a feeding tube because eating and drinking alone can no longer sustain her. That makes it hard for us to celebrate a milestone without also feeling the urgency of everything still ahead.

Today, we're grateful. We're proud of Helen. And we're more determined than ever to keep going for her and for every family living with this disease.

Thank You

Thank you for standing with us, for believing in this work, and for helping make this possible. We felt your love all day.

Love,

Dan & Steph Born

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