What we're up to
Active Research Projects
The “Alexa” Project
Alexa and children like her deserve a treatment regiment as aggressive as the cancer they are fighting. They are our inspirations – our heroes! The goal of The Alexa Project is to find new treatments to move towards clinical trials for all children affected with the neurofibromatosis-1 (NF1) associated brain tumors called juvenile pilocytic astrocytoma (JPA).
Big Data Analytics of a massive drug screen by Cell of Origin
We have partnered with a major pharmaceutical company to conduct an unprecedented screen of more than 640,000 compounds that, when combined, could offer new treatments for pediatric cancers that haven’t seen progress in decades such as: Rhabodmyosarcoma, Neuroblastoma, DIPG, ETMR, Wilms’ tumor and Osteosarcoma
The DIPG Tumor Cell Hotel
Fully funded by Storm the Heavens Foundation in memory of Philomena. To date $161,044 has been donated with another $161,044 of funding coming in 2019. Diffuse intrinsic pontine glioma (DIPG) is the greatest unmet need among childhood brain tumors, and this is in part because DIPG tumor cells weave themselves into the fabric of the pons
A Novel Treatment for Children with Anaplastic Wilms’ Tumor
We propose to study anaplasia in Wilms’ tumor at a preclinical level to discover why anaplastic disease tends to be resistant to standard therapies and to predict a rational drug combination which might be more effective and less toxic for these children.
Needle from the Haystack: Repurposed drug for Rhabdomyosarcoma
Already funded by a collective of partner family foundations! Soft tissue sarcomas are among the top 5 causes of death from childhood cancer. Until now, rhabdomyosarcoma (RMS) and non-rhabdomyosarcoma soft tissue sarcoma (NRSTS) have had dismal outcomes when unresectable, metastatic or progressive. To address this pressing & unmet clinical need, we have generated multiple genetically-engineered
REGN668 as an anti-metastasis agent for Pediatric Phase I/II Trials
Already funded by the National Cancer Institute (NIH) The major goal of this project is to test the hypothesis that IL4Rα blockade will prevent new soft tissue sarcoma engraftment, metastases and relapse by interfering with tumor cell – muscle stem cell interactions and thus represent an efficacious therapeutic strategy for children with rhabdomyosarcoma and other
Nuclear Receptor Tyrosine Kinases Mediating Chromatin Remodeling & Checkpoint Adaptation
Already funded by the Alex’s Lemonade Stand Foundation Alveolar rhabdomyosarcoma is a cancer of the soft tissues. This disease often responds to chemotherapy, but in many patients the available treatments fail –a deadly outcome. We identified how a cancer-causing fusion gene called Pax3:Foxo1 may lead to treatment failure: by turning on growth factor genes late
Osteosarcoma Checkpoint Adaptation (OCA) Pilot Project
Already funded by the Trey Foote Foundation This project funds a childhood cancer research scientist to explore how resistance to chemotherapy and radiation can be reversed. We believe that cell surface receptors go to the tumor cell’s nucleus in an unexpected way, and invoke genes that repair DNA damage. We believe that this can explain
Plexin-Semaphorin: A new signaling axis in Sarcomas
The Plexin-Semaphorin pathway is previously unexplored as a target in sarcomas but may play a central role in the progression of the soft tissue sarcoma, rhabdomyosarcoma.
The IL13RA2-HAS2 axis as therapeutic co-targets in DIPG
We hypothesize that HAS2 and IL13RA2 turn DIPG cells into “stem-like” cells that grow quickly and invade into the cancer-promoting environment caused by HAS2.
A Rapid Preclinical Drug Screening Assay in Quail Eggs
First to be applied to hepatoblastoma, this technology could speed discovery and testing of new drugs for many forms of childhood and adult cancer.
Ewing’s Sarcoma intra-vital drug testing
We have developed an inexpensive, time-saving replacement for mouse drug testing using quail eggs.
CureFast: creating a Legacy by accelerating Childhood Cancer Research
We propose to address gaps in basic and translational research by improving model systems for pediatric diseases. To this end, we have developed a Legacy Gift (research autopsy) program called the Cancer Registry for Familial and Sporadic Tumors (CUREfast) to enable parents of children with cancer to donate tumor tissue to the research community.
Macy’s & Addie’s & Avery’s & Dawson’s Hepatoblastoma Project: $120,000 annually
In the coming year, for which we are seeking funding, we will further validate volasertib-plus-irinotecan as a potential therapy for hepatoblastoma, continue DNA & RNA sequencing, and explore the epigenetic susceptibility of hepatoblastoma based upon the cell-of-origin.
Clear Cell Sarcoma BigData – Drug Discovery: $470,154 annually for 2 years
Clear Cell Sarcoma (CCS) is a very rare soft tissue of children and adults. The underlying clinical problem is that only surgery is curative: chemotherapy and radiation are ineffective. Relapse with metastases results in a significant unmet clinical need because surgery is not possible and effective targeted therapies have not yet been developed. To meet this clinical need by developing new therapies, the biology of CCS needs to be taken into consideration: not only is the EWSR1-ATF1 chimeric transcription factor/fusion gene present, but cooperating mutations may also be present.
The “Alexa” Project
Alexa and children like her deserve a treatment regiment as aggressive as the cancer they are fighting. They are our inspirations – our heroes! The goal of The Alexa Project is to find new treatments to move towards clinical trials for all children affected with the neurofibromatosis-1 (NF1) associated brain tumors called juvenile pilocytic astrocytoma (JPA).
Big Data Analytics of a massive drug screen by Cell of Origin
We have partnered with a major pharmaceutical company to conduct an unprecedented screen of more than 640,000 compounds that, when combined, could offer new treatments for pediatric cancers that haven’t seen progress in decades such as: Rhabodmyosarcoma, Neuroblastoma, DIPG, ETMR, Wilms’ tumor and Osteosarcoma
The DIPG Tumor Cell Hotel
Fully funded by Storm the Heavens Foundation in memory of Philomena. To date $161,044 has been donated with another $161,044 of funding coming in 2019. Diffuse intrinsic pontine glioma (DIPG) is the greatest unmet need among childhood brain tumors, and this is in part because DIPG tumor cells weave themselves into the fabric of the pons
A Novel Treatment for Children with Anaplastic Wilms’ Tumor
We propose to study anaplasia in Wilms’ tumor at a preclinical level to discover why anaplastic disease tends to be resistant to standard therapies and to predict a rational drug combination which might be more effective and less toxic for these children.
Needle from the Haystack: Repurposed drug for Rhabdomyosarcoma
Already funded by a collective of partner family foundations! Soft tissue sarcomas are among the top 5 causes of death from childhood cancer. Until now, rhabdomyosarcoma (RMS) and non-rhabdomyosarcoma soft tissue sarcoma (NRSTS) have had dismal outcomes when unresectable, metastatic or progressive. To address this pressing & unmet clinical need, we have generated multiple genetically-engineered
REGN668 as an anti-metastasis agent for Pediatric Phase I/II Trials
Already funded by the National Cancer Institute (NIH) The major goal of this project is to test the hypothesis that IL4Rα blockade will prevent new soft tissue sarcoma engraftment, metastases and relapse by interfering with tumor cell – muscle stem cell interactions and thus represent an efficacious therapeutic strategy for children with rhabdomyosarcoma and other
Nuclear Receptor Tyrosine Kinases Mediating Chromatin Remodeling & Checkpoint Adaptation
Already funded by the Alex’s Lemonade Stand Foundation Alveolar rhabdomyosarcoma is a cancer of the soft tissues. This disease often responds to chemotherapy, but in many patients the available treatments fail –a deadly outcome. We identified how a cancer-causing fusion gene called Pax3:Foxo1 may lead to treatment failure: by turning on growth factor genes late
Osteosarcoma Checkpoint Adaptation (OCA) Pilot Project
Already funded by the Trey Foote Foundation This project funds a childhood cancer research scientist to explore how resistance to chemotherapy and radiation can be reversed. We believe that cell surface receptors go to the tumor cell’s nucleus in an unexpected way, and invoke genes that repair DNA damage. We believe that this can explain
Plexin-Semaphorin: A new signaling axis in Sarcomas
The Plexin-Semaphorin pathway is previously unexplored as a target in sarcomas but may play a central role in the progression of the soft tissue sarcoma, rhabdomyosarcoma.
The IL13RA2-HAS2 axis as therapeutic co-targets in DIPG
We hypothesize that HAS2 and IL13RA2 turn DIPG cells into “stem-like” cells that grow quickly and invade into the cancer-promoting environment caused by HAS2.
A Rapid Preclinical Drug Screening Assay in Quail Eggs
First to be applied to hepatoblastoma, this technology could speed discovery and testing of new drugs for many forms of childhood and adult cancer.
Ewing’s Sarcoma intra-vital drug testing
We have developed an inexpensive, time-saving replacement for mouse drug testing using quail eggs.
CureFast: creating a Legacy by accelerating Childhood Cancer Research
We propose to address gaps in basic and translational research by improving model systems for pediatric diseases. To this end, we have developed a Legacy Gift (research autopsy) program called the Cancer Registry for Familial and Sporadic Tumors (CUREfast) to enable parents of children with cancer to donate tumor tissue to the research community.
Macy’s & Addie’s & Avery’s & Dawson’s Hepatoblastoma Project: $120,000 annually
In the coming year, for which we are seeking funding, we will further validate volasertib-plus-irinotecan as a potential therapy for hepatoblastoma, continue DNA & RNA sequencing, and explore the epigenetic susceptibility of hepatoblastoma based upon the cell-of-origin.
Clear Cell Sarcoma BigData – Drug Discovery: $470,154 annually for 2 years
Clear Cell Sarcoma (CCS) is a very rare soft tissue of children and adults. The underlying clinical problem is that only surgery is curative: chemotherapy and radiation are ineffective. Relapse with metastases results in a significant unmet clinical need because surgery is not possible and effective targeted therapies have not yet been developed. To meet this clinical need by developing new therapies, the biology of CCS needs to be taken into consideration: not only is the EWSR1-ATF1 chimeric transcription factor/fusion gene present, but cooperating mutations may also be present.
