N of 1
The benefit 2013 was a marvel of mass enthusiastic support and the after effects are still reverberating through Ben Towne Center for Childhood Cancer Research! Ben Towne Foundation supporters have risen to the challenge throughout this past year and as a result of your generous contributions I am happy to say our research program can forge ahead– pedal to the metal. There is much to be done as we strive to create momentum and plan for a sustainable future of creating new more effective and less toxic therapies for cancers that afflict children. THANK YOU!
For those of you who attended the benefit or who have been keeping abreast of the Center’s progress, our story has recently become tangible — we have broached the divide from wanting to help children in their battle, to having a name and a face, that of Lynsie Conradi, who bravely agreed to participate as our first patient on our leukemia T cell therapy protocol. While we were cautious in our expectations of what the lowest dose of reprogrammed T cells might do to help against a leukemia that was unresponsive to powerful chemotherapy, once infused back in to Lynsie’s body, those leukemia killing T cells multiplied somewhere between 1,000-10,000 fold and slammed that leukemia into a remission – that state when even with our most sensitive testing no leukemia cells can be detected. That was a homerun for Lynsie at a time when she needed one the most! And though we today stand at an N of 1 (only one patient treated thus far), we have witnessed the power of the reprogrammed immune system in action. Without your fiscal contributions that allowed us to accelerate the timetable to opening this trial, Lynsie would not be with us today. What a return on your philanthropic investment in Ben Towne Foundation! Your generosity was also recently was leveraged in our successful bid for a $1 million Life Sciences Discovery Fund Opportunity Grant to support our leukemia trials in collaboration with our partners at FHCRC. 1 +1 = 10
We have our second patient’s T cells made and infusion will take place here shortly. Critically, because we executed flawlessly in Lynsie’s care through the protocol, the FDA has released us from the initial trial age restriction of enrolling patients 18-26yrs of age, and the trial can now accept children from 1yr to young adults up to 26 years of age. Just last week we received three referrals of children in (desperate) need of effective therapy for their chemotherapy resistant leukemia. It is clear the pace of our trial will increase dramatically now. As we move forward, the T cell dose will increase as long as safety is maintained, and, if patients consistently respond as Lynsie did, we may be in the position to close the trial early and move on to the important Phase II trial to prove statistically we have a powerful therapeutic effect.
The pieces are coming together for launching a second leukemia trial this November. This trial, called PLAT-02, will seek to help children who relapse with Acute Lymphoblastic Leukemia after a bone marrow transplant. It is very important that we try to help these children given that of the fatalities that occur due to refractory ALL in kids occur in this setting. Since relapsed ALL is the biggest contributor of pediatric cancer mortality and it happens most frequently after a BMT we would be remiss to not try making a difference in this setting. For most kids, a relapse after BMT is the end of the road…..the most powerful applications of radiation and chemotherapy have failed and children are often too frail for much more. For many children we treat on PLAT-02, the T cells will be their last exposure to therapy. What this could mean, if our T cell therapy puts these patients in to remission, and the remission sticks, is that T cell therapy is more effective than their previous BMT. Such evidence could justify replacing BMT with T cell therapy. A game changer!
In many ways leukemia is the low hanging fruit for T cell therapy — a relatively easy target. Dealing with solid tumors such as neuroblastoma, brain tumors, and sarcomas to name a few will be much more difficult, in large part because we do not yet know what targets on the tumor cells will be safe to go after, and, because within a solid tumor resides potent immunosuppressive factors our T cells will need to evade. Much of our research and development efforts at BTCCCR are focused on these issues. We are making progess, and our first neuroblastoma trial is about a year out from being ready to submit to the FDA. This trial will focus on helping children that relapse or do not get in to a remission in the first place. Annette and Dr. Park have been pushing this project forward at an intensive pace (I am trying to get out of their way!). Adam and Ali are making solid progress in their engineering of T cells for attacking medulloblastomas, ependymomas, and gliomas. We are on a two-year horizon to a clinical trial. Sarcoma research is in its infancy and we are actively searching for target antigen leads through our collaborative work within the SU2C Cancer Dream Team projects.
So you can see, we have big plans to help many children. Leadership at Seattle Children’s is inspired by what they are seeing and truly transformative ideas are being floated around for building a new cancer research facility with a state of the art high capacity bio factory to serve children not only in the Seattle area, Pacific Northwest and the entire West Coast, but also on a national/international scale. Seattle Children’s is thinking big and I am certain your enthusiasm and support is fueling that fire! Stay tuned as this story unfolds.
I do think we are at a transformative moment in medicine. These do not come along every day, every year, or even every decade. Imagine how it must have been to be a pediatrician in the hospitals back when the polio vaccine was rolled out, and the wards crammed full of iron lungs keeping paralyzed children alive simply emptied out. A terrible villain vanquished. There are a lot of kids in hospital beds at Seattle Children’s and around the world battling leukemia and other childhood cancers every day. And you are a participant in this unfolding transformative moment. Perhaps generations in the future, people will marvel at the actions taken by the generous supporters of Ben Towne Foundation who spear headed the end of childhood cancers (starting with acute lymphoblastic leukemia) as to what they were once was known for — a killer of children.
Stay strong BTF’ers!!! You are inspiring us to move faster and reach farther!!