Make sure they study those tumors in my brain because if those tumors can help some kid some day not die from cancer like I am, I’d like that –
It’s hard to have cancer.
– Carson Leslie
- Cancer is the #1 cause of death by disease in children
- Brain cancer is the deadliest
- Medulloblastoma is the most prevalent
- Current treatment for medulloblastoma is EXCEEDINGLY HARSH
- Current treatment for medulloblastoma is DECADES OLD
Our recent analysis by PricewaterhouseCoopers revealed, NO organization has focused efforts on medulloblastoma… UNTIL NOW!
1. Invest in novel medullo research
2. Collaborate with likeminded organizations
3. Coalesce the medullo community
Current Medulloblastoma Collaborative Projects
American Society of Clinical Oncology (ASCO) Young Investigator Award in medullo.
ASCO is the world’s premier oncology organization, with over 45,000 members.
“The joining of forces between our organizations represents our synergistic commitment to advancing research in medulloblastoma and ensuring a cadre of investigators focused on this important area.” – Gerald McDougall, ASCO/Conquer Cancer board member, Board Chairman CLF/#cureMedullo
Award Term: 8/1/2019-7/31/2020
Anthony P.Y. Liu, MBBS, MMedSc
St. Jude Children’s Research Hospital
“Cerebrospinal fluid derived circulating tumor DNA as an actionable biomarker in medulloblastoma”
Research Progress Summary Report – March 2020
Medulloblastoma is the most common malignant tumor of the brain in young children. Treatment consists of surgical resection, radiotherapy and chemotherapy. Unfortunately, approximately one-third of patients still succumb to their disease. Despite recent research that offered insight into the genetic alterations present in medulloblastoma, a sensitive tumor marker for confirmation of treatment response and determination of microscopic residual disease is lacking. Liquid biopsy is the study of tumor-derived material in body fluids, this includes tumor-derived DNA fragments, or cell-free DNA. Cell-free DNA analysis has been explored in adult-onset brain tumors, but such approach has not been systemically adopted for children with medulloblastoma.
Supported by the Conquer Cancer – #cureMEdullo powered by Carson Leslie Foundation Young Investigator Award.
We initiated a study to evaluate the clinical utility of profiling cell-free DNA from cerebrospinal fluid in children with medulloblastoma.
Using cerebrospinal fluid collected during routine disease evaluation from children with medulloblastoma enrolled on two multi-institutional clinical trial led by St. Jude Children’s Research Hospital, we developed an experimental pipeline to allow comprehensive analysis of cell-free DNA present at low quantities.
Our interim result confirms the capability of our proposed method to capture tumor-derived cell-free DNA in cerebrospinal fluids.
The detection rate of tumor-derived cell-free DNA correlates with disease extent, reflects response to treatment conventionally determined by magnetic resonance imaging, and allows early detection of relapse.
Furthermore, cell-free DNA analysis provides an alternative strategy for analysis of disease at progression without the need of repeated surgical procedures.
Overall, findings support the use of cell-free DNA analysis as a novel tumor marker for better risk-stratification and treatment personalization. Our ongoing project will capture data from an expanded cohort of children with medulloblastoma and offer further evidence for integration of cell-free DNA profiling into the next generations of medulloblastoma protocols.
Anthony P.Y. Liu, MBBS, MMedSc, received his medical degree from the University of Hong Kong in 2009. He completed a residency in pediatrics and training in pediatric hematology/oncology at Queen Mary Hospital, Hong Kong, as well as a Master’s degree in genetic counseling. A keen clinician-scientist with an interest in pediatric central nervous system tumors, Dr Liu has been a Pediatric Neuro-Oncology Fellow at St. Jude Children’s Research Hospital, Memphis, TN, since 2017. His ongoing research is focused on biomarker development, circulating tumor material and molecular characterization of pediatric brain tumors, including medulloblastoma, pineoblastoma, and other embryonal tumors.
Stingray Therapeutics is introducing an immune-therapy in medulloblastoma.
#cureMedullo is investing deeply and directing funds into this novel immune-therapy that the Director of #cureMedullo believes is ‘vastly differentiated from typical biotech investments’.
“I am especially motivated as I returned from the largest annual global scientific conference on immune system-focused therapies in cancer, the Society for Immunotherapy of Cancer conference, in Washington, DC. During this conference, the timeliness and uniqueness of the opportunity involving Stingray’s novel, immune-therapy became readily apparent. The strength and uniqueness of the program, its accelerated path based on medulloblastoma, the successful track record of Stingray’s researchers and their established position in the CPRIT framework, we believe this program is vastly differentiated from more typical biotech investments.” – Walter Capone, Director of #cureMedullo strategic plan
Addressing childhood cancer is a top priority for CPRIT with 12 percent of their portfolio going to pediatric cancer research, which is proportionately three times more than the national rate. Earlier this month in Dallas, the Carson Leslie Foundation and CPRIT gathered the best minds in pediatric brain cancer research to chart ways forward for increasing collaboration. Presenters and attendees represented research institutions from around the state, discussing targeted therapies, immunotherapy, the opportunities presented through CPRIT-funded pediatric core facilities, and how best to address survivorship.
“Carson Leslie, a Dallas native, died of medulloblastoma at the age of 17 in 2010. His family has established The Carson Leslie Foundation to raise funds for pediatric brain cancer research. Further, one of Carson’s last wishes was that his brain be used to enhance understanding of his disease. CPRIT is honored to collaborate with the Carson Leslie Foundation in providing partial funding for this award and in overseeing its administration.”
“Treatment for childhood medulloblastoma, with a few exceptions, has changed very little of the past two decades. This needs to change. We need to find new, less toxic cures for all children with medulloblastoma.” – Dr. Daniel Bowers
Daniel Bowers, M.D.
- Associate Professor, Pediatric Hematology and Oncology at UTSW
- Medical Director, Pediatric Neuro-Oncology at Children’s Health
- Medical Director, After the Cancer Experience for childhood cancer survivors
- Well known nationally for his understanding of side effects of childhood brain tumor survivor
Our goal is to find a cure for medulloblastoma and we need your support to create a cancer-free future.
Let us know how you can make an impact!