What we're up to

Ongoing 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

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The Functional Genomics of Wilms’ Tumor

Approximately 500 new cases of Wilms’ tumor are diagnosed each year in North America alone, making Wilms’ tumor the most common kidney cancer discovered in children. While the majority of these patients are cured, children with the anaplastic subtype of the disease do not respond well to current therapies, and a strong need therefore exists to develop more promising treatment options for these children.

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 »

Be a Dog’s Best Friend, Help a Child

To make it better for children & young adults with osteosarcoma, cc-TDI is exploring the types of non-chemotherapy drugs that re-program osteosarcoma tumor cells to stop growing – or cease to exist. This is a 2-step project: first we define which classes of drugs have the desired activity as single agents – then we find the right 2-drug combination to move into clinical trials.

Read More »

Next Steps in the Rapid Preclinical Development of a Targeted Therapy Combination for Diffuse Intrinsic Pontine Glioma (DIPG)

Remarkably, this is the first time that a group of basic and translational scientists and physicians from institutions throughout North America and Europe have come together as a consortium to focus on a bench-to-bedside approach to rationally target therapy for children with DIPG. Our Scientific Director, Charles Keller, MD, was responsible as co-Chair of the Children’s Oncology Group brain tumor new drugs committee (CNS-DVL) for building this coalition and coordinates activities among the 13 institutions worldwide.

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 Functional Genomics of Wilms’ Tumor

Approximately 500 new cases of Wilms’ tumor are diagnosed each year in North America alone, making Wilms’ tumor the most common kidney cancer discovered in children. While the majority of these patients are cured, children with the anaplastic subtype of the disease do not respond well to current therapies, and a strong need therefore exists to develop more promising treatment options for these children.

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 »

Be a Dog’s Best Friend, Help a Child

To make it better for children & young adults with osteosarcoma, cc-TDI is exploring the types of non-chemotherapy drugs that re-program osteosarcoma tumor cells to stop growing – or cease to exist. This is a 2-step project: first we define which classes of drugs have the desired activity as single agents – then we find the right 2-drug combination to move into clinical trials.

Read More »

Next Steps in the Rapid Preclinical Development of a Targeted Therapy Combination for Diffuse Intrinsic Pontine Glioma (DIPG)

Remarkably, this is the first time that a group of basic and translational scientists and physicians from institutions throughout North America and Europe have come together as a consortium to focus on a bench-to-bedside approach to rationally target therapy for children with DIPG. Our Scientific Director, Charles Keller, MD, was responsible as co-Chair of the Children’s Oncology Group brain tumor new drugs committee (CNS-DVL) for building this coalition and coordinates activities among the 13 institutions worldwide.

Read More »
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