01/26/2026
Incredible update on some of our friends in the same drug trial. 💕
And through every step of this journey we hold Reed in our hearts. 💚
It’s been a while since I’ve shared an update on how some of the VWM research is going. As many of you know, we traveled with Reed out to Utah roughly two months before his passing. Receiving the email inviting us to screen for the trial was truly a dream come true—something we had worked toward for so many years. But God had other plans, and deep down, we knew it.
Reed’s VWM had progressed too far, and his body simply wouldn’t have been able to handle the travel. God knew that, and He gently guided us toward that decision, knowing we would need those final weeks to soak up every moment with our boy. While our path looked different than we had hoped, so many of our VWM friends are now on Fosigotifator and are doing incredibly well. I wanted to share this update so you know the fight isn’t over—and that we are continuing to move closer to ending this awful disease.
A little more about Fosigotifator (FGT): it is referred to as an “activator,” meaning it increases the activity of the EIF2B protein complex, helping brain cells better manage stress and function more normally. It works by activating the EIF2B gene complex—the exact location of the mutation that causes VWMD. Doctors describe it as essentially holding the complex together so it can function properly, even though it remains mutated. For most patients, there are no side effects, and it is taken easily once daily.
The image included in this post shows Ella’s MRI scans. The scan on the right is from April, and the scan on the left is from August 2025. The darker areas represent regrowth, while the lighter areas show where the brain was previously damaged or lost. In the newer scan, there are significantly fewer light/void areas and clear signs of improvement.
Of course, neither scan resembles a completely healthy brain. The black areas represent fluid that has filled spaces where myelin (white matter) has disappeared. An MRI is essentially a water-based image, and while it has limitations, it can still tell us a great deal. As best as doctors can tell, the brain is slowly remyelinating. If you know anything about neurodegenerative diseases or leukodystrophies, you understand how extraordinary this is. Remyelination does not happen on its own with VWMD—myelin is only lost over time.
The most compelling evidence, of course, is the functional improvement people are seeing. There’s no way to know what the ceiling will be—how much function may return or what abilities will look like long term—but we continue to pray and hope for ongoing progress each day.
Additionally, FGT appears to be doing an excellent job protecting against decline caused by fever and infection. Recently, one of our friends contracted COVID, ran a 104-degree fever, and experienced a severe seizure. While no one wants to test the limits of this medication, the doctor said it was the highest fever and most intense seizure he’s seen in a patient on FGT—and the child weathered it remarkably well. We’ve also heard of another little girl on the medication who fell and hit her head on a tile floor and is doing fine. All signs point to FGT doing its job in protecting these children.
All of the children in the trial are doing well, with most seeing similar results. Improvements appear to be more dramatic in younger children. This drug is currently on a special FDA fast-track approval process, and there is hope that the formal approval process could begin as early as this year.
Our Journey with Jax - Vanishing White Matter Disease
Jax has been on the trial drug since October. From his mama: He’s been experiencing episodes of flushing, stiffening, and crying—which is notable because he rarely cries. He also sometimes seems dizzy when sitting up, and his skin appears more fragile, resulting in additional pressure sores. While very few patients have experienced side effects, Jax and a few others who have seem to be further along in the disease process and on multiple medications. This has raised the possibility of medication interactions. Their neurologist is working to wean some medications to see if that helps.
The most important question, of course, is whether there are improvements—and his mama believes there are small but meaningful ones. His oxygen levels have been more stable, he may be moving his arms slightly more, and he appears to have better head control. At their neurology appointment, the doctor felt confident that Jax was tracking her with his eyes. They are hopeful that once the side effects are addressed, improvements will become clearer. They will travel back for another appointment in January, which will include a follow-up MRI.
A Miracle for Ella Rose
Ella has been on FGT since April 2025, and her progress truly feels miraculous. Before the trial, she was unable to use utensils due to tremors and coordination challenges. Recently, she fed herself and finished an entire cup of mac and cheese for the first time. Even more incredible, her appetite is stronger than ever—she has gained more weight since starting the trial than she has in years.
Her physical therapy walking tests show remarkable improvement:
6-Minute Walk Test
• Sept 23, 2024: 158 ft
• Sept 15, 2025: 246 ft
30-Second Walk Test (average of 3 trials)
• Apr 10, 2025: 16.1 ft
• Sept 15, 2025: 35.3 ft
Hunter
Hunter has been on Fosigotifator since November and is doing amazing. His mama recently shared that Hunter climbed onto the couch by himself. This may sound like a small thing—but it isn’t. For nearly a year, Hunter had lost the ability to do something that once came effortlessly: crawling up to sit with his family. Hunter will soon return to Boston for his three-month follow-up, which includes another MRI. Many families are seeing white matter regrowth at this stage, making this appointment especially significant and hopeful.
Jada Foundation
Jada and her family traveled all the way from South Africa to the U.S. and have been asked to stay for approximately eight months. The transition has not been easy, but her mama is doing an incredible job, and each day seems to be getting a little lighter. Jada has been on FGT for seven weeks and is thriving.
From her mama: Jada has a new laugh—a deep, joyful giggle that feels like it comes from somewhere new. She’s gained 4kg after years of fighting for every gram, which feels nothing short of miraculous. She looks healthy and radiant. Her speech has blossomed—new words, full sentences, and a voice we once feared we might never hear. Every word feels like a victory. Jada’s next appointment is at the end of February.
Archer’s Fight Against VWMD
Archer reminds me so much of Reed. He has been on FGT since last summer and has shown incredible progress. His parents report that his speech is returning, he can now use his left arm and hand to pick things up, and he eats at every mealtime while working on more challenging textures. He uses his stander for several hours a day, his vision has improved, and his brain lag has significantly decreased. More than anything, he is happier than he has been in a long time. Archer’s next MRI is scheduled for this summer.
