One of the more enjoyable parts of our work is that we get to interact with and support the work of so many truly brilliant and incredible people. Dr. Rebecca Gardner is one of them and she is the lead investigator for the Pediatric Leukemia Adoptive Therapy (PLAT)-01 and PLAT-02 trials. Many of you will remember her from this BTCCCR video, which gives me chills every time I watch it.
I have been thinking about Rebecca a lot these past weeks, as she holds a unique vantage point of being both a researcher and clinician, as well as the one doctor who has interacted with all of the PLAT-01 and PLAT-02 leukemia patients and families. So I thought perhaps it might be nice to have her answer a few question for us today.
1) As the lead investigator for PLAT-01 and PLAT-02, what was it like to present these incredible results at the American Society of Hematology Annual Meeting?
We all have grand ideas when we start something new, but it’s always followed by thoughts that this may not work the way we think it will. When we initially started PLAT-01 and PLAT-02, we had high hopes of being able to treat patients with Acute Lymphoblastic Leukemia whose disease has failed to respond to our standard therapies, and have them go in to remission. What we never could have imagined was how well our treatment would work. This past December I traveled to San Francisco to present our finding for PLAT-02 at the Annual Meetings of the American Society of Hematology. At the meeting, I was able to present that of the first 13 patients treated on PLAT-02, 11 of them went into remission. This is truly remarkable for this group of patient who had little to no other alternative options for treatment.
2) You are the one person who has interacted with all of these patients and their families. What has that experience been like? What does it feel like to tell a family that their child is in remission?
I always knew I was going to be a pediatrician, but it wasn’t until residency that I decided to become a pediatric oncologist. One of the things that were influential to my choice was meeting the families on the oncology floor at the hospital. There is something special in all of these families who are there with their kids, fighting cancer and it motivates you to keep looking for new and better ways to treat cancer. By far and away the best part of my job is when I get to walk in a room and tell a kid and their family that they are in remission. A little over half of all the kids who have been treated on the T cell trials have come from outside our region, and a few have even come from outside of the US. When families come for the study, they are hopeful but skeptical at the same time. These are kids who when they were first diagnosed, were given an 80-90% chance of a long term cure, yet they are here for the trial because those odds didn’t work for them. When T cell therapy works, it does its job in about 2-3 weeks, sometimes even faster. Recently we had a family whose child got T cells and those T cells went to work quickly, faster than we had ever seen them work. Seven days after the T cells were infused, the patient was in remission. I walked into the room of the patient and her parents and said, “Guess what? No Leukemia!” And the mom said, “What?!” When I told the family that the test showed 0% leukemia cells, they were in disbelief, noting that in their three plus year long battle with leukemia, they had never heard the number 0. When I saw them in clinic after a second test confirming that there were still 0% leukemia cells, you could just see the mom’s shoulders relax and she took a deep breath and for the first time in a long while, she felt hopeful.
3) What have been the most exciting and most challenging aspects of running a Phase One clinical trial?
The most challenging thing is that because we are still at the beginning of this exciting time, not everyone is eligible to participate. We get calls from all over from parents whose children have cancer and they are in desperate need of a therapy that will work. A lot of these kids have a cancer that is different from the leukemia that our trials are treating right now so unfortunately this trial wouldn’t work for him. And that is why the work at the BTCCCR is so important…all those people working in the lab, with the goal of making this therapy available to all kids with cancer.
The most exciting part is learning from each patient we treat with T cells. This means that we can continue to get better and better at using this therapy. The absolute best part of this trial is seeing that the T cells are going to work, and that the patient is responding to the therapy. It’s a feeling that never gets old and makes me feel like I have the best job in the world.
*Thank you so much Dr. Gardner for sharing with us today! We are grateful for your work and insight.