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).

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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

Read More »

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

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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

Read More »

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

Read More »

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

Read More »

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.

Read More »
Clear Cell Sarcoma

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.

Read More »

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).

Read More »

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

Read More »

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

Read More »

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

Read More »

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

Read More »

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

Read More »

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.

Read More »
Clear Cell Sarcoma

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.

Read More »