There is no nice way to tell someone they've got a brain tumor, and with a child it’s unimaginable. In fact, brain tumors are the leading cause of solid tumor cancer death in children. Proteasome inhibitors are a recently discovered drug class that is effective in many types of cancer and have reduced side effects to normal cells. Dr. Almaliti aims to develop novel potent and selective proteasome inhibitors that will specifically kill brain cancer in children. This innovative approach should result in the discovery of new clinical leads for treating brain cancers in children.

This grant is funded by and named for Luke's Army Pediatric Cancer Research Fund. This Hero Fund was created in memory of Luke Ungerer who brought smiles and sunshine wherever he went with plenty to share with everyone. He battled a brain tumor with a positive spirit and inspired others with his courage in his short life. This fund intends to carry on Luke’s legacy of positivity with the hope that it will ripple across many lives for many years to come.

Cancer research allows scientists to modify specific immune cells to recognize and kill cancer. One type of immune cell is called the cytotoxic killer T cell. This T cell has a receptor (TCR) that is used to recognize a structure on the cancer cell's surface called a peptide-major histocompatibility molecules complex I (pMHC I). pMHC I complexes are diverse and are rarely shared amongst patients. This diversity prevents the use of a classic TCR across multiple patients to avoid tissue injury that known as graft versus host disease (GVHD). To bypass these limitations, Dr. Elsabbagh propose to develop T cells expressing a TCR that can target a protein called CD1d. Unlike MHC I, CD1d is not diverse and is well expressed on various childhood cancers including acute myeloid leukemia (AML), which has been known for high rates of treatment-related toxicity and disease recurrence. These modified cells will be pre-made and used universally in any AML or other children’s cancers that expresses CD1d. Dr. Elsabbagh and team will also attach a recent discovered enhancing protein called MyD88 to the created receptor to enhance their anticancer activity. They expect these modified T cells will be able to recognize and kill children AML cells.

Cancer research allows scientists to modify specific immune cells to recognize and kill cancer. One type of immune cell is called the cytotoxic killer T cell. This T cell has a receptor (TCR) that is used to recognize a structure on the cancer cell's surface called a peptide-major histocompatibility molecules complex I (pMHC I). pMHC I complexes are diverse and are rarely shared amongst patients. This diversity prevents the use of a classic TCR across multiple patients to avoid tissue injury that known as graft versus host disease (GVHD). To bypass these limitations, Dr. Elsabbagh propose to develop T cells expressing a TCR that can target a protein called CD1d. Unlike MHC I, CD1d is not diverse and is well expressed on various childhood cancers including acute myeloid leukemia (AML), which has been known for high rates of treatment-related toxicity and disease recurrence. These modified cells will be pre-made and used universally in any AML or other children’s cancers that expresses CD1d. Dr. Elsabbagh and team will also attach a recent discovered enhancing protein called MyD88 to the created receptor to enhance their anticancer activity. They expect these modified T cells will be able to recognize and kill children AML cells.

There is no nice way to tell someone they've got a brain tumor, and with a child it’s unimaginable. In fact, brain tumors are the leading cause of solid tumor cancer death in children. Proteasome inhibitors are a recently discovered drug class that is effective in many types of cancer and have reduced side effects to normal cells. Dr. Almaliti aims to develop novel potent and selective proteasome inhibitors that will specifically kill brain cancer in children. This innovative approach should result in the discovery of new clinical leads for treating brain cancers in children.

This grant is funded by and named for Luke's Army Pediatric Cancer Research Fund. This Hero Fund was created in memory of Luke Ungerer who brought smiles and sunshine wherever he went with plenty to share with everyone. He battled a brain tumor with a positive spirit and inspired others with his courage in his short life. This fund intends to carry on Luke’s legacy of positivity with the hope that it will ripple across many lives for many years to come.

In Africa, the majority of children who get cancer die from their disease. This happens in many cases because the patients do not get a correct diagnosis. Without a precise and correct diagnosis, these children cannot benefit from the newest curative treatments. To help prevent this from happening, Dr. Lutwama will develop and test a strategy to diagnose pediatric cancer correctly in a manner that is affordable, reliable, and within a shorter time frame in resource-limited settings.

In Africa, the majority of children who get cancer die from their disease. This happens in many cases because the patients do not get a correct diagnosis. Without a precise and correct diagnosis, these children cannot benefit from the newest curative treatments. To help prevent this from happening, Dr. Lutwama will develop and test a strategy to diagnose pediatric cancer correctly in a manner that is affordable, reliable, and within a shorter time frame in resource-limited settings.

Based on progress to date, Dr. Das was awarded a new grant in 2022 to fund an additional year of this International Scholar grant. When a cell divides, the DNA duplicates. However there may be errors in this process. Most are corrected by an in-built replication repair mechanism. If not corrected, this may lead to mutations. The repair mechanism itself may be faulty in some children with an inherited condition. They develop cancers in the brain, intestines and blood, with very high number of mutations. These cancers are difficult to diagnose and do not respond to standard chemotherapy and radiation. Dr. Das and colleagues have developed cutting edge yet inexpensive genomic tool, called 'signatures' which will help better diagnose this disease. The tool will also predict which children will benefit from a new, promising treatment known as immunotherapy. It will also help diagnose other family members before they develop cancers and initiate surveillance to improve their chances for survival. The condition is more prevalent in the developing world where the custom of marrying within one's community is prevalent. Hence validation of the utility of this tool and developing local capacity to use this will benefit large number of children and their families in underserved areas across the globe.

The 2022 portion of this grant is funded by and named for the Kai Slockers Pediatric Cancer Research Fund. Kai was diagnosed at 2½ with Atypical Teratoid Rhabdoid Tumor (ATRT), a rare and very aggressive brain cancer. Within two weeks of diagnosis, he passed away, a mere 3 months shy of his third birthday. When Kai took his last breath, the cloudy sky opened up with a bright ray of sun that streamed through the windows of his hospital room – the darkness of the disease was replaced with the light of hope and the peace of no more suffering. Whenever the sun is out, his family thinks of him, assured that his legacy of hope shines on. In his brief life, Kai shared his warmth, energy, goofy sense of humor, and caring heart with all those he met. This Hero Fund was created in his memory and will support research to help other kids with cancer have a better chance to fight and survive. It has a special focus on brain tumor research, specifically treatments that could minimize the harsh effects of brain tumor treatment. The Slockers family hopes to continue his legacy of light and hope through the funding of childhood cancer research.

A portion of this grant is generously supported by The Team Campbell Foundation. The Foundation was established in memory of Campbell Hoyt, who courageously battled anaplastic ependymoma, a rare cancer of the brain and spine, for five years before passing away in August of 2014 at the age of eight. Its mission is to improve the lives of families facing a childhood cancer diagnosis through raising awareness, funding research and providing psycho-social enrichment opportunities.

Based on progress to date, Dr. Das was awarded a new grant in 2022 to fund an additional year of this International Scholar grant. When a cell divides, the DNA duplicates. However there may be errors in this process. Most are corrected by an in-built replication repair mechanism. If not corrected, this may lead to mutations. The repair mechanism itself may be faulty in some children with an inherited condition. They develop cancers in the brain, intestines and blood, with very high number of mutations. These cancers are difficult to diagnose and do not respond to standard chemotherapy and radiation. Dr. Das and colleagues have developed cutting edge yet inexpensive genomic tool, called 'signatures' which will help better diagnose this disease. The tool will also predict which children will benefit from a new, promising treatment known as immunotherapy. It will also help diagnose other family members before they develop cancers and initiate surveillance to improve their chances for survival. The condition is more prevalent in the developing world where the custom of marrying within one's community is prevalent. Hence validation of the utility of this tool and developing local capacity to use this will benefit large number of children and their families in underserved areas across the globe.

The 2022 portion of this grant is funded by and named for the Kai Slockers Pediatric Cancer Research Fund. Kai was diagnosed at 2½ with Atypical Teratoid Rhabdoid Tumor (ATRT), a rare and very aggressive brain cancer. Within two weeks of diagnosis, he passed away, a mere 3 months shy of his third birthday. When Kai took his last breath, the cloudy sky opened up with a bright ray of sun that streamed through the windows of his hospital room – the darkness of the disease was replaced with the light of hope and the peace of no more suffering. Whenever the sun is out, his family thinks of him, assured that his legacy of hope shines on. In his brief life, Kai shared his warmth, energy, goofy sense of humor, and caring heart with all those he met. This Hero Fund was created in his memory and will support research to help other kids with cancer have a better chance to fight and survive. It has a special focus on brain tumor research, specifically treatments that could minimize the harsh effects of brain tumor treatment. The Slockers family hopes to continue his legacy of light and hope through the funding of childhood cancer research.

A portion of this grant is generously supported by The Team Campbell Foundation. The Foundation was established in memory of Campbell Hoyt, who courageously battled anaplastic ependymoma, a rare cancer of the brain and spine, for five years before passing away in August of 2014 at the age of eight. Its mission is to improve the lives of families facing a childhood cancer diagnosis through raising awareness, funding research and providing psycho-social enrichment opportunities.

Osteosarcoma is the most common primary bone tumor in childhood. The survival rate remains dismal, mainly due to ineffective therapeutic approaches for the relapsed/metastatic patients. One major obstacle of treating osteosarcoma is lack of suitable preclinical models. Dr. He's studies have established the first cultured osteosarcoma tissue model (an organoid). Dr. He aims to establish the first biobank of osteosarcoma organoids from patients as an open resource for the field, and utilize this organoid biobank to evaluate a novel class of therapeutics targeting key signaling pathways in osteosarcoma cells. This study will provide a powerful platform for predicting clinical treatment responses and developing new therapeutics for treating osteosarcoma.

Osteosarcoma is the most common primary bone tumor in childhood. The survival rate remains dismal, mainly due to ineffective therapeutic approaches for the relapsed/metastatic patients. One major obstacle of treating osteosarcoma is lack of suitable preclinical models. Dr. He's studies have established the first cultured osteosarcoma tissue model (an organoid). Dr. He aims to establish the first biobank of osteosarcoma organoids from patients as an open resource for the field, and utilize this organoid biobank to evaluate a novel class of therapeutics targeting key signaling pathways in osteosarcoma cells. This study will provide a powerful platform for predicting clinical treatment responses and developing new therapeutics for treating osteosarcoma.

In the U.S., children with a blood cancer called Hodgkin lymphoma (HL) are usually treated successfully. Some of these children will suffer health problems several years later because of the treatment they received. Because of this, doctors use powerful imaging tools to identify patients who are likely to do well or not. Those who are likely to do well require less treatment and those who are less likely to do well can receive more treatment. But in low-income countries like Malawi, these tools are unavailable, and the children there often receive treatment that may be unnecessary. Scientists have found unique abnormalities in adults with HL that can tell us who is less likely to do well. Here, Dr. Ozuah is testing whether these abnormalities are present in children and could be used to decide how best to treat children with HL in low-middle income countries

In the U.S., children with a blood cancer called Hodgkin lymphoma (HL) are usually treated successfully. Some of these children will suffer health problems several years later because of the treatment they received. Because of this, doctors use powerful imaging tools to identify patients who are likely to do well or not. Those who are likely to do well require less treatment and those who are less likely to do well can receive more treatment. But in low-income countries like Malawi, these tools are unavailable, and the children there often receive treatment that may be unnecessary. Scientists have found unique abnormalities in adults with HL that can tell us who is less likely to do well. Here, Dr. Ozuah is testing whether these abnormalities are present in children and could be used to decide how best to treat children with HL in low-middle income countries

Based on progress to date, Dr. Mulama was awarded a new grant in 2020 to fund an additional year of this Scholar grant. Kaposi sarcoma-associated herpesvirus is a virus that causes cancer known as Kaposi sarcoma, which is very common in HIV+ children, especially in Africa and sometimes in individuals who get an organ transplant. Dr. Mulama is designing and testing a vaccine that prevents and treats the viral infection, as well as antibodies to detect infection in people. He will also test the vaccine so that one day it can be used as a treatment to prevent Kaposi sarcoma-associated herpesvirus infection and Kaposi sarcoma in more than 40,000 patients worldwide each year.

Based on progress to date, Dr. Mulama was awarded a new grant in 2020 to fund an additional year of this Scholar grant. Kaposi sarcoma-associated herpesvirus is a virus that causes cancer known as Kaposi sarcoma, which is very common in HIV+ children, especially in Africa and sometimes in individuals who get an organ transplant. Dr. Mulama is designing and testing a vaccine that prevents and treats the viral infection, as well as antibodies to detect infection in people. He will also test the vaccine so that one day it can be used as a treatment to prevent Kaposi sarcoma-associated herpesvirus infection and Kaposi sarcoma in more than 40,000 patients worldwide each year.

Based on progress to date, Dr. Lian was awarded new grants in 2018 and 2019 to fund additional years of this International Scholar grant. Based on progress to date, Dr. Lian was awarded a new grant in 2018 to fund an additional year of this International Scholar grant. MYCN-driven neuroblastoma accounts for about 30% of neuroblastomas and is associated with an extremely poor prognosis. Casein Kinase 2 (CK2) is an enzyme that is currently in clinical trials to treat multiple cancers. However, its efficacy on MYCN-driven neuroblastoma remains unknown. Dr. Lian's research aims to test if CK2 inhibition can serve as a new strategy to treat MYCN-driven neuroblastoma.

A portion of this grant was named for The Amanda Rozman Pediatric Cancer Research Fund, a St. Baldrick's Hero Fund created in memory of Amanda Rozman and honors her courageous battle with neuroblastoma by funding promising new treatments and clinical trials in the area of translational research.

Based on progress to date, Dr. Lian was awarded a new grant in 2018 to fund an additional year of this International Scholar grant. MYCN-driven neuroblastoma accounts for about 30% of neuroblastomas and is associated with an extremely poor prognosis. Casein Kinase 2 (CK2) is an enzyme that is currently in clinical trials to treat multiple cancers. However, its efficacy on MYCN-driven neuroblastoma remains unknown. Dr. Lian's research aims to test if CK2 inhibition can serve as a new strategy to treat MYCN-driven neuroblastoma.

A portion of this grant was named for The Amanda Rozman Pediatric Cancer Research Fund, a St. Baldrick's Hero Fund created in memory of Amanda Rozman and honors her courageous battle with neuroblastoma by funding promising new treatments and clinical trials in the area of translational research.

Based on progress to date, Dr. Wasswa was awarded new grants in 2018 and 2019 to fund additional years of this International Scholar grant. Whereas more than 80% of children with leukemia and lymphoma in the United States are cured with chemotherapy, in Africa a diagnosis with one of these diseases is an outright death sentence. To enable adaptation of chemotherapy protocols from the U.S. to treating children in Africa, Dr. Wasswa is studying the prevalent types of leukemia and lymphoma in children in Malawi and how their genetic code may affect response to chemotherapy.

Based on progress to date, Dr. Wasswa was awarded a new grant in 2018 to fund an additional year of this International Scholar grant. Whereas more than 80% of children with leukemia and lymphoma in the United States are cured with chemotherapy, in Africa a diagnosis with one of these diseases is an outright death sentence. To enable adaptation of chemotherapy protocols from the U.S. to treating children in Africa, Dr. Wasswa is studying the prevalent types of leukemia and lymphoma in children in Malawi and how their genetic code may affect response to chemotherapy.

Based on progress to date, Dr. Zhang was awarded new grants in 2017 and 2018 to fund additional years of this International Scholar award. Though cure rates have improved dramatically, geographic inequities in childhood acute lymphoblastic leukemia (ALL) therapy remain evident between developed vs. low-middle income countries. In China, about 7,700 newly-diagnosed children with ALL every year receive therapy thanks to the coverage provided by a special national health insurance scheme established in 2010. With the drastic increase in access to clinical care, the challenge is now shifting from remedying the inability to pay for ALL therapy to delivering better therapy and improving outcome. Dr. Zhang is working to bring improved therapy to China. This represents an unprecedented opportunity for translational research of childhood ALL in China, with potential impacts for a large number of patients.

Based on progress to date, Dr. Zhang was awarded new grants in 2017 and 2018 to fund additional years of this International Scholar award. Though cure rates have improved dramatically, geographic inequities in childhood acute lymphoblastic leukemia (ALL) therapy remain evident between developed vs. low-middle income countries. In China, about 7,700 newly-diagnosed children with ALL every year receive therapy thanks to the coverage provided by a special national health insurance scheme established in 2010. With the drastic increase in access to clinical care, the challenge is now shifting from remedying the inability to pay for ALL therapy to delivering better therapy and improving outcome. Dr. Zhang is working to bring improved therapy to China. This represents an unprecedented opportunity for translational research of childhood ALL in China, with potential impacts for a large number of patients.