Hopefully last week you saw the exciting announcement – that the first clinical trial application from the Ben Towne Center for Childhood Cancer Research using reprogrammed T cells for the treatment of children with neuroblastoma was authorized by the FDA for patient enrollment! This trial, called ENCIT (Engineered Neuroblastoma Cellular Immunotherapy) -01, is a Phase I first-in-child trial for children with relapsed or treatment unresponsive neuroblastoma.
Dr. Julie Park, along with Dr. Annette Kunkele, has been instrumental in getting us to this stage, so we thought it might be nice to have her answer a few questions for us! Dr. Park was Ben’s oncologist (many of you will remember her from our benefit 2011) and holds a unique perspective as someone who is both a researcher and clinician. She will also be serving as the lead investigator for ENCIT-01.
1) Tell us about ENCIT-01. As a clinician and researcher, what does it feel like when you receive FDA approval for a new trial?
This is a clinical trial that will study whether we can collect special immune fighting cells (T cells) from patients with neuroblastoma, modify them in the laboratory to recognize neuroblastoma cells and then study the safety of giving those back to patients. The trial will enroll those patients with neuroblastoma whose cancer has either not responded well to initial therapy or has recurred following completion of therapy. Getting FDA authorization and now local IRB regulatory approval to enroll patients on the trial is incredibly exciting.
At the same time, I and our clinical research team at Seattle Children’s Hospital and the Ben Towne Center for Childhood Cancer Research recognize the hard work ahead, but we are uniformly eager to start enrolling patients. I have been caring for patients with neuroblastoma for nearly 20 years and have been involved in many other clinical trials, including earlier T cell trials. While we have made advances, we desperately need new therapies for this dreadful disease. I am so fortunate and excited to be able to bring such novel therapy to our patients.
2) When will kids begin enrolling in the ENCIT trial? How do you choose who gets to enroll? Where will the kids come from?
We are now approved by our Seattle Children’s Hospital IRB to begin patient enrollment. We are finalizing last details for implementation of the trial and expect that we will be able to enroll and collect T cells from our first patient by December 2014. Patients are eligible to enroll if they have high-risk neuroblastoma that has not responded adequately to initial therapy or has recurred during or following completion of therapy. Patients must also meet requirements for good function of their heart, liver and kidneys. Children from anywhere nationally and internationally are eligible to enroll.
3) Neuroblastoma is a very different beast than leukemia. What are the obstacles the T Cells face in trying to destroy neuroblastoma cells? How is this trial different and or the same as the other two that are happening at the BTCCCR?
The ENCIT trial is very similar to the ongoing leukemia trials except that it will target L1CAM molecule on neuroblastoma cells instead of CD19 that is present on acute lymphocytic leukemia cells. The process by which we will collect blood cells from the patients and manufacture the cells is identical to PLAT-02.
We hope that the T cells that are put back into the patient’s body will travel through the blood stream and attack neuroblastoma cancer cells (note from btf: as seen in this laboratory video). However, we anticipate the solid tumors may not respond as easily to T cell therapy when compared to the successes we have seen with leukemia. Our trial will be using T cells that are better equipped to activate themselves and kill tumor cells when infused into the body than T cells used in earlier trials for neuroblastoma. Despite these advancements, we know that solid tumors like neuroblastoma develop a local tumor environment that can depress the activity of T cells or even prevent them from getting access to the tumor. We will be studying the effects of T cells closely and are working with investigators nationally to better understand these barriers.
Thank you so much Dr. Park for taking time out of your busy schedule to tell us more about ENCIT! We are grateful to you, Dr. Kunkele and the team at the Ben Towne Center for all your efforts!
And our immense appreciation to Ben Towne Foundation donors – who by supporting the preclinical trial efforts helped make this milestone possible!