Update on Gene Therapy Plans (And the Year Ahead)

Last week we had a teleconference meeting with Dr. Kohn from UCLA, the head of the gene therapy trials.

It was an incredibly encouraging meeting.

Felix is number fourteen out of fifteen children in the trial. Of the twelve who have already gone through it, eleven are doing really well. (They’re still trying to figure out what’s behind the one child’s persisting problems.) It is so heartening to hear the doctors confidently talk about how they’ll continue to follow Felix into adulthood. Adulthood! My baby who wasn’t supposed to survive past infancy, alive and well in twenty years! What a thrilling thought.

Everyone feels very confident that this will work, that Felix will be cured.

The only disappointing news we got is that our gene therapy date is going to be pushed back a week. This is only to give the lab more time between patients. We’re now scheduled for July 6. In the grand scheme of things, a week is nothing; but we’re so eager to move on to the next phase! We were quite bummed out to hear it.

Dr. Kohn also gave us a bit of a timeline for the upcoming year. If all goes well, it should go something like this:

July 6-13: In Los Angeles for Gene Therapy

I plan on explaining gene therapy in depth yet, but for now: it’s essentially a bone marrow transplant using Felix’s own bone marrow, that has been modified to include the missing enzyme. (Update: gene therapy explained here.)

While at UCLA he will have his bone marrow harvested, will undergo chemo, and then have the corrected bone marrow cells infused back into his bloodstream. He’ll be monitored for a couple of days before returning to Canada. The whole thing takes about a week. (We’re taking a commercial flight both ways. We’ll be asking for your prayers that it all happens safely!)

Mid-July to Mid-August: In Hospital in Canada

Following gene therapy, Felix needs to be closely monitored for about a month to see what his cells do and to watch for infection. This can be done close to home — the results just need to be sent to UCLA. We’re not sure yet whether that will happen in London, where we already spent five months, or in Toronto, which is four hours away from our home.

It takes about two weeks for his platelets and blood cells to drop from the chemo, at which point he will be extremely susceptible to infection again — even more so than now. Which is why we’ll be staying in the hospital. It takes about two more weeks for his cells to go back up again and begin to stabilize. During this time he will continue to get the enzyme injections that he gets now.

Mid-August to Christmas: Strict Isolation at Home

If all goes well, we will return home after a month in the hospital and continue living in isolation as we are now. During these 4-6 months, we are waiting for the corrected cells to multiply and take over, enabling him to rebuild his immune system. We will stop the enzyme injections, with the expectation that his corrected cells will be able to make the enzyme on their own.

We might continue to have Lydia live with my parents since Felix will still be extremely immune-deficient for the first few months. So it’s likely that she will be living away from home for almost a full year. (This also breaks my heart.)

By Christmas/New Year’s, hopefully Felix can begin the process of being released from isolation. At that point we should know whether treatment was effective. We’d still have to avoid crowds or daycare (not that we would send him anyway), but perhaps he could take the occasional outing or see a few visitors.

And KISSES!! I’m counting on Christmas kisses!!!

* * *

So if all goes well, that’s the year ahead of us.

And here is a video of our sweet Felix, just as a reminder that he’s a real person and not just a medical diagnosis. (Sometimes I need that reminder when I hear about other kids going through medical procedures.)

This is how he wakes up every day — singing and celebrating life. He is SUCH A HAPPY GUY.

Thanks again for your continued prayers and support!