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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 »

Epigenetic Therapy to Overcome Checkpoint Adaptation in Rhabdomyosarcoma

Alveolar rhabdomyosarcoma (aRMS) is a cancer of the soft tissues. In many cases, the disease responds to chemotherapy, but in other patients our current treatment options fail and the cancer spreads throughout the body–a deadly outcome. Our team has identified a cancer-causing gene (also known as an oncogene) called Pax3:Foxo1 that causes current treatments to fail. Remarkably, Pax3:Foxo1 can silenced by entinostat, a drug recently granted FDA breakthrough designation for breast cancer.

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 »

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 »

Epigenetic Therapy to Overcome Checkpoint Adaptation in Rhabdomyosarcoma

Alveolar rhabdomyosarcoma (aRMS) is a cancer of the soft tissues. In many cases, the disease responds to chemotherapy, but in other patients our current treatment options fail and the cancer spreads throughout the body–a deadly outcome. Our team has identified a cancer-causing gene (also known as an oncogene) called Pax3:Foxo1 that causes current treatments to fail. Remarkably, Pax3:Foxo1 can silenced by entinostat, a drug recently granted FDA breakthrough designation for breast cancer.

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 »
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