The Foundation raises funds for research that will find new and innovative ways to treat neuroblastoma, and ultimately, find a cure for this disease. We want to create a World Class Neuroblastoma Research and Care Center. We know we can make this world so much better for these kids and families with the best doctors, research, treatment, love and support. No dollar amount is too small as we appreciate anything and everything. This is going to be a big job, but it is one we know we can do, with the help of all of you. Thank you for your donation.
Your donation to The Ronan Thompson Foundation funds the nation's top oncologists. The Foundation's fundraising assists these oncologists to perform ground-breaking clinical trials in the search for a cure for pediatric cancer.
The RTF partnered with Solving Kids' Cancer on an immunotherapy clinical trial using children's own immune cells that are re-engineered to become cancer-killing T-cells (T-cell CARs) targeting specific markers on neuroblastoma cells called GD2. A similar technology was recently first used in children with relapsed leukemia and has been a real breakthrough - over 50% complete responses in 1 1/2 years. We are hopeful that T-cells CARs for solid tumors like neuroblastoma will be just as successful as the early results in blood cancers. This is a first-in-children study with a goal to enroll 14 children and complete in early 2015.
This T-cell neuroblastoma trial recently opened at Texas Children's Cancer Center/Baylor School of Medicine, much to the excitement of families who desperately need innovative treatment options. We are also excited to have supported the development of this new cutting-edge therapy that introduces a new category of treatment that has the potential to be more effective and less toxic than chemotherapy, radiation and targeted drugs.
Ph I T-cell GD2 CAR for neuroblastoma (Baylor)
To date, 14 patients have been consented for procurement and 2 patients have been treated on Dose Level 1. We are currently within the 6 week waiting period prior required by the FDA prior to proceeding with Dose Level 2.
Our first patient continues to have detectable T-cell CARs within his peripheral blood samples. The number of cells was stable through week 4 and then decreased significantly at the week 6 evaluation. He had clinical improvement, but unfortunately had progression of disease on imaging. Our second patient is due for his disease re-evaluation in 2 weeks. The 3rd patient is set to enroll this week at the next dose level.
CHANGES TO THE STUDY
Based on data obtained from other T cell studies being conducted at CAGT, we plan to change our T cell manufacturing process from using IL-2 to another cytokine profile. The amendment to the FDA will be submitted on 2/3/14. In addition, we are considering the option of providing fresh cells (as compared to our current frozen product) for those in whom the timing may work.
ALK Monoclonal Antibody Development for neuroblastoma
The central aim in this project is to develop antibodies against the ALK extracellular region that can be used alone or in combination with small molecule ALK kinase inhibitors to block ALK as an oncogenic driver in neuroblastoma.
CHOP's studies in the first project period have yielded a range of antibodies on which to work for Antibody-Dependent Cell-Mediated Cytotoxicity (ADCC) applications, as well as avenues for combining agents to identify useful inhibitory approaches.
The inhibitory antibodies have not shown effects against mutated ALK to date – but the fact that binding is retained indicates that these would still be useful for ADCC. Meanwhile, CHOP has set up the pipeline in this first year of study to screen rapidly through additional antibodies for those that do have robust inhibitory effects on mutated ALK.
This year, the Ronan Thompson Foundation is partnering with Solving Kids' Cancer, to fund a clinical trial directed by Dr. Yael Mosse at Children's Hospital of Philadelphia (CHOP). Solving Kids' Cancer generously agreed to match the amount donated by the Ronan Thompson Foundation. We are very excited to be working with a internationally renowned organization on such an important co-venture.
The Foundation's goal for this clinical trial is: $50,000, for a total joint donation of $100,000. With your help, we plan on achieving this goal by the end of the year.
Thank you for your donation and helping make this goal a reality!