The ETANTR (ETMR) Pilot Program: $170,000 annually

Embryonal tumors with abundant neuropil and true rosettes (ETANTR) is a rare category of brain tumors of children that generally occurs in the cerebrum (but not exclusively) and has a median survival of only 9-13 months. Due to the severe nature of this tumor and the scarcity of preclinical resources necessary to make drug screens possible, our consortium will start by developing cell lines and mouse models and then create shareable central resource(biorepository) for cell lines and mouse models.

The ASPS/Xp11 RCC/PEComa Cancers Consortium: $465,000

The mission of the ASPS/Xp11 RCC/PEComa/EHE Cancers Consortium is to catapult the discovery of treatment options for these cancers that share common molecular features (e.g., TFE3 gene fusions). Our goal is to advance novel drug and drug combinations to clinical trial for these cancers.

Leukemia Dynamics: $170,000 for one year

Gleevec inhibits the BCR-ABL oncogene in childhood and adult leukemias, but the drug is taken for years because “reserve cells” can lead to relapse if gleevec treatment is stopped too early. This pilot project explores why the gleevec-indifferent reserve cells may exist, based on unexpected preliminary data that leads to a radical new way to think of fusion oncogenes.

Epigenetic Therapy for Checkpoint

Epigenetic Therapy for Checkpoint Adaptation in Rhabdomyosarcoma: $120,000

In our studies, entinostat dramatically improved sensitivity to the rhabdomyosarcoma chemotherapeutics & radiation (see our publications in Genes & Development and Science Signaling). The Phase I study is underway (clinicaltrials.gov NCT02780804) … the race is on to generate the data enabling the Phase II clinical trial. But can we do better? We believe so by study of SMARCA4 and its inhibitors.

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).

The ETANTR (ETMR) Pilot Program: $170,000 annually

Embryonal tumors with abundant neuropil and true rosettes (ETANTR) is a rare category of brain tumors of children that generally occurs in the cerebrum (but not exclusively) and has a median survival of only 9-13 months. Due to the severe nature of this tumor and the scarcity of preclinical resources necessary to make drug screens possible, our consortium will start by developing cell lines and mouse models and then create shareable central resource(biorepository) for cell lines and mouse models.

The ASPS/Xp11 RCC/PEComa Cancers Consortium: $465,000

The mission of the ASPS/Xp11 RCC/PEComa/EHE Cancers Consortium is to catapult the discovery of treatment options for these cancers that share common molecular features (e.g., TFE3 gene fusions). Our goal is to advance novel drug and drug combinations to clinical trial for these cancers.

Leukemia Dynamics: $170,000 for one year

Gleevec inhibits the BCR-ABL oncogene in childhood and adult leukemias, but the drug is taken for years because “reserve cells” can lead to relapse if gleevec treatment is stopped too early. This pilot project explores why the gleevec-indifferent reserve cells may exist, based on unexpected preliminary data that leads to a radical new way to think of fusion oncogenes.

Epigenetic Therapy for Checkpoint

Epigenetic Therapy for Checkpoint Adaptation in Rhabdomyosarcoma: $120,000

In our studies, entinostat dramatically improved sensitivity to the rhabdomyosarcoma chemotherapeutics & radiation (see our publications in Genes & Development and Science Signaling). The Phase I study is underway (clinicaltrials.gov NCT02780804) … the race is on to generate the data enabling the Phase II clinical trial. But can we do better? We believe so by study of SMARCA4 and its inhibitors.

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).

Close Menu