2020 Gift of Hope
Our 2020 Gift of Hope further supports Dr. Rutka’s research lab for the current clinical trial proposal that makes use of an experimental drug delivery system for DIPG developed by the Rutka Lab that uses MRI guided focused ultrasound to temporarily open the blood brain barrier specifically within the tumour and not the normal parts of the brain. This temporary opening provides a therapeutic window to delivery chemotherapies precisely developed to target the patient’s tumour. An extensive series of preclinical experiments are currently underway to optimize the procedure for use in patients.
We have dedicated our 2020 Gift of Hope to Isabelle Borkowski who lost her battle with DIPG on December 16th, 2020. Our thoughts are with her family during this difficult time.
Fundraising options are quite limited due to COVID 19 and social distancing requirements. We hope to actively fundraise again in the future.
2019 Gift of Hope
We are pleased to announce that $20,000 has been granted to support Dr. Rutka’s research project – Identification and characterization of immunotherapeutic targets in diffuse intrinsic pontine gliomas and $10,000 has been granted to support this clinical trial – A safety and feasibility study to evaluate Blood-Brain Barrier(BBB) Disruption Using Transcranial MRI-Guided Focused Ultrasound with Intravenous Ultrasound Contrast Agents and Chemotherapy in the patients with Diffuse Intrinsic Pontine Gliomas.
We will be given the opportunity to meet with Dr. Rutka & Dr. Ramaswamy later this year to get an update on their progress with their research.
As written by SickKids, the research that our Gifts of Hope will support.
Identification and characterization of immunotherapeutic targets in diffuse intrinsic pontine gliomas.
James Rutka Lab
Diffuse intrinsic pontine glioma (DIPG) is a devasting brain tumour that primarily occurs in young children with an overall survival of 8-12 months and a less than 1% survival at 5 years. Despite over 10,000 published chemotherapy studies, none have shown significant improvement in patients. Therefore, it is vital to explore novel therapeutic avenues and targets for DIPGs.
To accomplish this, the Rutka Lab has already demonstrated enhanced delivery of chemotherapy drugs in preclinical models using MRI guided focused ultrasound to temporarily open the protective blood-brain-barrier specifically within the tumour without involving nearby normal tissue. Excitingly, this work has moved into the development of a Phase 1 clinical trial (currently in REB review) to test focused ultrasound safety in patients with DIPG.
In addition, the Rutka lab is exploring the utility of immunotherapy in DIPG, the focus of this proposal. Immunotherapy is a type of treatment that utilizes the body’s immune system in order to fight cancer. One of the most important cells in the immune system are the T cells, which directly kill cancer cells in the body by recognizing that it is an unhealthy cell. However, cancers can often evade recognition and consequently grow and spread. CAR T-cells have taken the spotlight in immunotherapy, because they are engineered to recognize specific cancer related proteins, which effectively recognize and kill cancer cells.
The Rutka lab has recently identified a validated protein target, PRAME, which is specific to DIPGs and is not found anywhere else in the brain. As a result, PRAME can be targeted using immunotherapy with virtually no harm to the rest of the body. Firstly, we are characterizing PRAME to identify its functional role, and importantly, investigate it as a CAR-T immunotherapeutic target in pre-clinical trials. We anticipate the results of this study will inform new and innovative clinical trials aimed to increase the outcome of children suffering from these complex and fatal tumours.