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Showing 1-20 of 284 results

Yael Mosse M.D.

Funded: 01-01-2025 through 12-31-2027
Funding Type: Research Grant
Institution Location: Philadelphia, PA
Institution: The Children's Hospital of Philadelphia affiliated with University of Pennsylvania

A child diagnosed with rhabdomyosarcoma (RMS) today is subjected to essentially the same therapy used fifty years ago. While Drs Mosse and Maris’s research programs have traditionally focused on neuroblastoma (NBL), both labs have recently extended their research programs to other pediatric solid tumors with high unmet need. Here they propose two parallel and complementary projects designed to create new immunotherapies for RMS, with Project 1 focused on the discovery of new immunoncology targets and cellular therapies, and Project 2 focused on the IND-enabling studies of a novel antibody-drug conjugate (ADC) directed against ALK for fusion positive RMS. This grant is supported by Alice's Arc US Inc. Alice's Arc operates in the UK and USA and is dedicated to funding research into finding a cure and less harsh treatments for rhabdomyosarcoma. The charity has created a global community of families, scientists and doctors coming together to help achieve this mission. Families are at the heart of the work and they can choose to create an Arc in their child/young person's name to represent their experience with rhabdomyosarcoma. This enables funds to be pooled for research, powerful advocacy and a long-term, sustainable platform to grow the charity and fund a pipeline of rhabdomyosarcoma research.

Roarke Kamber Ph.D.

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Funded: 12-01-2024 through 11-30-2026
Funding Type: Research Grant
Institution Location: San Francisco, CA
Institution: University of California, San Francisco affiliated with UCSF Benioff Children's Hospital

The recent development of therapies that stimulate the immune system to eliminate cancer has transformed treatment options for many patients. However, these therapies have generally been less successful in treating childhood cancers, in part because cancers in younger patients typically have acquired fewer of the genetic alterations that can be recognized by T cells, the immune cell type most commonly used for cancer treatment. This project aims to harness the cancer clearing functions of macrophages, a distinct immune cell type that can recognize and kill even those cancer cells that carry few genetic alterations. Dr. Kamber and colleagues will focus on identifying strategies that unleash macrophage anti-cancer functions in the context of Burkitt lymphoma, an aggressive form of lymphoma that is among the most common types of cancer in children. This grant is funded by and named for Jack's Pack - We Still Have His Back, a St. Baldrick's Hero Fund. Jack Klein was a ten year old who loved life, laughing and monkeys. During his illness, his community of family and friends near and far rallied around him under the moniker "Jack's Pack". Their slogan was "We have Jack's Back". After Jack succumbed to Burkitt's Lymphoma, his "pack" focused their energy and efforts to funding a cure...just as Jack would have wanted.

Fange Liu Ph.D.

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Funded: 07-01-2024 through 06-30-2026
Funding Type: Research Grant
Institution Location: Philadelphia, PA
Institution: University of Pennsylvania affiliated with The Children's Hospital of Philadelphia

In some types of cancer that affect children and teenagers, there are special proteins called 'fusion oncoproteins' that play a big role. These proteins are made when a gene called MLLT10 gets mixed up with other genes. These cancers are very dangerous and don't respond well to treatments available now. Dr. Lui and colleagues research is focused on the most prevalent MLLT10 fusion oncoproteins, common in a type of cancer called T-cell acute lymphoblastic leukemia (T-ALL) in kids and young adults. Findings show that this fusion protein makes groups of biomolecules called 'condensates,' which can mess up how cells read and use their genetic instructions. Dr. Lui believes that by studying these MLLT10 fusion oncoproteins in detail, they may learn how they change cells and find ways to stop them. Dr. Lui also believes if they can figure out how MLLT10 fusion oncoproteins work, it may also help to understand other similar fusion proteins. That knowledge could help develop better treatments for these kinds of cancer. This grant is funded by and named for Emily Beazley's Kures for Kids Fund, a St. Baldrick's Hero Fund. At the age of 8, Emily was diagnosed with Stage III T-cell lymphoblastic non-Hodgkin’s lymphoma and battled through three relapses. Her family prayed for a miracle but discovered Emily herself was the miracle, inspiring a community to come together to show love and change lives. She had a dream of starting a foundation to fund research and named it “Kures for Kids”. Today, Emily's family and friends carry on her dream and her mission in her memory.

Emily Johnston M.D.

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Funded: 07-01-2024 through 06-30-2026
Funding Type: Research Grant
Institution Location: Birmingham, AL
Institution: University of Alabama at Birmingham affiliated with Children's of Alabama

It is known that children with cancer have higher rates of hospitalization, ICU admission, and death than children without cancer and COVID-19. Children with cancer and COVID-19 also frequently have changes in their chemotherapy. Yet, critical data is lacking regarding COVID-19 in children with cancer and guidelines about how to manage these vulnerable children. Dr. Johnston and collegaues will leverage the national registry of children with cancer and COVID with data on >2,400 children from >100 institutions to examine (1) how the clinical course of children with cancer and COVID-19 compares to earlier in the pandemic, (2) how the clinical course of COVID-19 in children with cancer is impacted by vaccination and antiviral therapy, and (3) physician and healthcare systems factors that influence COVID-19 management. Dr. Johnston will use that information, literature review, and expert discussion to inform an expert panel tasked with developing guidelines for management of COVID-19 in children with cancer.

Mark Rutherford Ph.D.

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Funded: 07-01-2024 through 06-30-2026
Funding Type: Research Grant
Institution Location: St. Louis, MO
Institution: Washington University in St. Louis affiliated with St. Louis Children's Hospital

Cancer patients take life-saving drugs that, unfortunately, can result in peripheral nerve damage. For example, many patients receiving cisplatin experience permanent hearing loss. There is one therapy that has been approved to mitigate cisplatin-induced hearing loss, however, the reduction in hearing loss is modest (< 30%) and this mitigating treatment is associated with poorer overall survival rates due to inhibition of cisplatin's cancer-fighting properties. Thus, it is approved for low-risk pediatric patients only. To develop a better alternative, Dr. Rutherford and colleagues are testing novel compounds they have developed at Washington University, which have shown to protect the ear from noise trauma. With hearing tests and with anatomical measurements of the cochlea, Dr. Rutherford will attempt to prevent hearing loss following cisplatin treatment in models. After this innovative project proves successful, subsequent model studies will determine if Dr. Rutherford's therapy inhibits cisplatin's cancer-fighting role.

Gary Kupfer M.D.

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Funded: 07-01-2024 through 06-30-2026
Funding Type: Research Grant
Institution Location: Washington, DC
Institution: Georgetown University affiliated with MedStar Georgetown University Hospital

The study of genetic disease of cancer predisposition has served as a model for understanding cancer in general. Fanconi anemia is a rare genetic disease of failed blood production and cancer proneness, including leukemia and head and neck cancer. The genes and encoded proteins participate in DNA repair. However, an examination of cancer databases of DNA sequence shows that Fanconi genes are mutated in up to 30% of all head and neck cancers in non-Fanconi patients. Dr. Kupfer and colleagues have studied one particular mutation that resides in the Fanconi FANCD2 gene that interrupts its protein binding to another important gene BLM, which also participates in DNA repair. This proposal will seek to study the normal function of the FANCD2-BLM interaction in the cell and the consequences of its disruption. Dr. Kupfer also seeks to identify ways disruption of the normal pathway will render cancers vulnerable to molecular targeting to improve therapeutics.

Benjamin Kann M.D.

Funded: 07-01-2024 through 06-30-2026
Funding Type: Research Grant
Institution Location: Boston, MA
Institution: Brigham and Women's Hospital, Inc.

Survivors of pediatric brain tumors have a high risk of medical problems that can negatively affect the quality of their lives. Particularly concerning are effects on brain development, including learning and emotional well-being, and metabolism, which can lead to obesity and muscle loss. There is an urgent need for tools that can better predict which children are most at risk so that they can be offered treatments to prevent these problems. Dr. Kann's and colleagues have developed medical imaging tools that use artificial intelligence on routine brain scans to track and predict 1) muscle weakness and malnutrition, and 2) brain development in children. Dr. Kahn and team will test these tools in large datasets from hospitals and clinical trials of pediatric brain tumor patients and survivors to predict the risk of these negative effects in each patient. The tools developed may be used in clinical trials to improve quality-of-life for childhood brain tumor survivors.

Carl Allen M.D., Ph.D.

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Funded: 07-01-2024 through 06-30-2026
Funding Type: Research Grant
Institution Location: Houston, TX
Institution: Baylor College of Medicine affiliated with Vannie E. Cook Jr. Children's Cancer and Hematology Clinic, Texas Children's Hospital

The ultimate goal of this project is to define the safest, most effective therapies for children and young adults with Langerhans cell histiocytosis (LCH), which aligns with St. Baldrick's mission to find cures for childhood cancers and give survivors long and healthy lives. LCH is a blood cancer most common in children that creates destructive inflammatory lesions that can be fatal. LCH is caused by mutations activating the MAPK growth pathway in developing blood cells. Current front-line therapy fails to cure over 50% of patients with disseminated disease, and safe and effective options for subsequent therapy is not known. High-dose chemotherapy can be effective, but is toxic. MAPK inhibitor therapy alone does not appear to be durable based on early trials. Dr. Allen and colleagues hypothesize that MAPK inhibition will make cells more sensitive to chemotherapy. Dr. Allen will therefore test safety and efficacy of a new approach of combining chemotherapy with targeted MAPK inhibitor therapy.

Anusha Preethi Ganesan M.D., Ph.D.

Funded: 07-01-2024 through 06-30-2026
Funding Type: Research Grant
Institution Location: San Diego, CA
Institution: University of California, San Diego affiliated with Rady Children's Hospital San Diego

Medulloblastoma (MB) is an aggressive childhood brain cancer that is fatal in 40% of patients. MB shows abnormal activation of growth pathways in tumor cells which help them to grow. Dr. Ganesan's studies show that there are T cells (immune cells) within MB and they have potential to kill tumor cells. However, immunotherapy that boosts the activity of these T cells have not been successful and it is not known why. The goal of this project is to understand why immunotherapy has not worked in MB and whether immunosuppressive myeloid cells contribute to this restraint. Dr. Ganesan and colleagues will also study if combined treatment that inhibits the growth pathways (targeted therapy) and stimulates the T cells/immune system (immunotherapy) may together lead to greater tumor killing in MB mouse models. To test if combined therapy would work in humans, Dr. Ganesan will coculture a 3D version of patient’s brain tumor with their own T cells expanded from their tumor. If effective, these studies may lead to new treatments for MB.

Jun Qi Ph.D.

Funded: 07-01-2024 through 06-30-2026
Funding Type: Research Grant
Institution Location: Boston, MA
Institution: Dana-Farber Cancer Institute affiliated with Boston Children's Hospital, Harvard Medical School

Despite remarkable improvements in treatment for children with some types of cancer, pediatric brain tumors remain an area that desperately require more effective and low toxic therapy solutions. Dr. Jun Qi has formed a multi-disciplinary team to identify novel targets for pediatric brain tumors and develop new strategies to suppress the targets for patient treatment. Using a chemical strategy, Dr. Qi and his team aim to disrupt the functions of these targets to effectively inhibit brain tumor cell growth and block tumor progression in the models that resemble the real disease. The study focuses on improving on-target effect and, more importantly, on getting these potential drug candidates into the brain. The proposed study will translate from bench to bedside for patient care and result in a novel therapeutic strategy with significant improvements in survival and reduced morbidity for pediatric brain tumor patients to fulfill the mission of St. Baldrick’s Foundation.

Poul Sorensen M.D.

Funded: 05-01-2024 through 04-30-2026
Funding Type: Research Grant
Institution Location: Vancouver, BC
Institution: The University of British Columbia affiliated with British Columbia Children's Hospital, British Columbia Cancer Agency

Ewing sarcoma (EwS), the 2nd most common childhood bone sarcoma, is an aggressive tumour that primarily affects children, adolescents, and young adults. When EwS tumor cells spread to other parts of the body, known as metastasis, survival is drastically diminished to only 15-20%, which has not changed for decades. Immunotherapy empowers a patient’s own immune system to attack cancer, which has tremendous promise as an alternative to chemotherapies that are often toxic, especially to a growing child. Dr. Sorensen and his team recently identified a protein that is highly expressed on the surface of EwS cells, while showing only minimal to absent expression in normal tissues, nominating IL1RAP as a very promising therapeutic target. With their collaborators at the University of Pittsburgh, they have identified specific antibodies binding to IL1RAP and have engineered these antibodies to be conjugated to a drug that kills EwS cells potently. In this project, they will perform the extensive validation of these compounds to enable the design of early clinical trials for the treatment of EwS. This Better Ewing Sarcoma Therapies (BEST) grant is supported by a unique partnership of funders through the St. Baldrick’s Foundation: D-Feet Cancer, The Faris Foundation, The Shohet Family Fund for Ewing Sarcoma Research, an anonymous donor, and the family and friends of Martha Riedel.

Sujatha Venkataraman Ph.D.

Funded: 10-01-2023 through 09-30-2025
Funding Type: Research Grant
Institution Location: Denver, CO
Institution: University of Colorado affiliated with Children's Hospital Colorado

Chimeric Antigen Receptor (CAR)-T cells have been clinically effective in patients with leukemias and lymphomas. Dr. Venkataraman’s goal is to bring similar success in treating a fatal brain tumor in children called DIPG (Diffuse Intrinsic Pontine Glioma). A major obstacle in treating brain tumors with CAR-T cell therapy is a lack of antigens which are tumor specific, or which are absent on normal vital tissues that can lead to off-target toxicities. To overcome this risk, Dr. Venkataraman and colleagues have successfully generated and tested the functionality of a novel “logic-gated” CAR-T cells targeting two distinct antigens, CD99 AND B7H3 that are highly expressed on DIPG but present singly on certain normal cells. This gated “AND” CAR-Ts will have full-activation against DIPG cells having both the antigens while sparing the single antigen expressing normal cells and will now investigate the safety, preclinical efficacy of these CAR-T cells against DIPG and evaluate its translational relevance to DIPG patients.

Mitchell Cairo M.D. 

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Funded: 10-01-2023 through 09-30-2026
Funding Type: Research Grant
Institution Location: Valhalla, NY
Institution: New York Medical College affiliated with Maria Fareri Children's Hospital at Westchester Medical Center

Children, adolescents and young adults with recurrent or refractory Osteosarcoma have a very poor prognosis, with a dismal 6mo overall survival of less than 5%. Presumably, this poor prognosis is in large part secondary to the development of resistance to chemotherapy and radiation. More recent studies employing therapies that release and activate the patients’ immune cells, called T-cells, and even targeted T-cells have not improved this poor prognosis. Dr. Cairo proposes to investigate novel and innovative methods of combinatorial immunotherapy to circumvent known mechanisms of resistance. Together with colleagues, he proposes to investigate at the bench (in the laboratory) and in models with osteosarcoma alternative methods of combination immunotherapy including natural killer cells (NK) that we have been engineered in the laboratory to also circumvent mechanisms of resistance and to additionally express a single or dual target that are present on the osteosarcoma cells. They further plan to investigate the efficacy of adding other immunotherapies to enhance the function and persistence of these targeted NK cells with antibodies, and two different NK activating cytokines. They will also investigate the optimal combination of this immunotherapy in children, adolescents and young adults with recurrent or refractory osteosarcoma to determine the safety and efficacy of this approach. Finally, Dr. Cario and team will determine what are the genetic and immune mechanisms of resistance after these novel combinatorial immunotherapy approaches utilizing state-of-the-art laboratory techniques. The goal of this grant is to develop novel combinatorial immunotherapy that will significantly increase the overall survival in children and adolescents with poor risk osteosarcoma. To make a significant impact for kids fighting osteosarcoma, five funders have banded together with St. Baldrick’s to support this grant – The Helping Osteosarcoma Patients Everywhere (HOPE) Super grant supported by Battle Osteosarcoma, the Faris Foundation, the Zach Sobiech Osteosarcoma Fund of Children’s Cancer Research Fund, the Children’s Cancer Fund NY (supporting the Maria Fareri Children’s Hospital and New York Medical College) and Nationwide Children’s Hospital.

Iannis Aifantis Ph.D.

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Funded: 09-01-2023 through 08-31-2025
Funding Type: Research Grant
Institution Location: New York, NY
Institution: New York University School of Medicine affiliated with NYU Langone Medical Center

CAR-T cell immunotherapies, treatments that use T cells constructed to recognize tumors and kill them, revolutionized how doctors treat children with B cell leukemia (B-ALL). These killer T cells recognize a specific protein expressed on the surface of the leukemic cells. Unfortunately, leukemia frequently relapses and often finds ways to "switch off" the expression of this protein, making T cells unable to track and kill them. This notion is called "antigen escape," as the tumor finds a way to escape the immune treatment. Dr. Aifantis plans to identify ways to avoid antigen escape by boosting the expression of the surface recognition protein. The study aims to validate such mechanisms in an organism using CAR-T cell models and sequencing patient cells. At the same time, Dr. Aifantis will design screens that will help identify surface antigen-specific regulators, so researchers can one day create combinatorial protocols using CAR-T cells and targeting specific antigen surface expression regulators.

Jennifer Kalish M.D., Ph.D.

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Funded: 07-01-2023 through 06-30-2025
Funding Type: Research Grant
Institution Location: Philadelphia, PA
Institution: The Children's Hospital of Philadelphia affiliated with University of Pennsylvania

Beckwith-Wiedemann Syndrome (BWS) is a cancer predisposition syndrome and patients with BWS have a significantly increased risk of developing hepatoblastomas. The same genetic changes on chromosome 11 that cause BWS are found in 40% of hepatoblastomas. Dr. Kalish has previously shown that noncancerous liver and HB tissue from patients with BWS have distinct molecular signatures, suggesting the events that set up patients with BWS for HB are due to these changes on chromosome 11. Using the largest BWS collection of tissues worldwide, Dr. Kalish and colleagues will study the specific features of BWS and nonBWS liver cells and HB cells to determine how the changes of chromosome 11 lead to HB. Cell models derived from liver tissue will be used to test how these changes cause the transition from normal liver to HB. This work is a critical step in developing targeted therapies for patients with HB.

Stella Davies MBBS, Ph.D.

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Funded: 07-01-2023 through 06-30-2025
Funding Type: Research Grant
Institution Location: Cincinnati, OH
Institution: Cincinnati Children's Hospital Medical Center affiliated with University of Cincinnati College of Medicine

Children receiving bone marrow transplant can have serious complication such as bloodstream infections and graft versus host disease and some children die of these complications. Alteration of the bacteria in the gut by treatments including antibiotics is an important cause of these complications. In a previous study Dr. Davies and colleagues have tested the use of human milk to help keep gut bacteria healthy in very young children and found that this treatment worked. They are now studying a purified sugar from human milk, 2-FL that can be given easily as a medicine. Dr. Davies will also test a novel rapid urine test and a blood test to assess health of the gut bacteria during the study. Current tests require a stool sample and can take a long time. This trial will generate the data needed to perform a large-scale multi-center randomized clinical trial that will best prove how well this treatment works. This grant is generously supported by the Rays of Hope Hero Fund which honors the memory of Rayanna Marrero. She was a happy 3-year-old when she was diagnosed with Acute Lymphoblastic Leukemia (ALL). She successfully battled ALL, but a treatment induced secondary cancer claimed her life at age eight. Rayanna had an amazing attitude and loved life. She, like so many kids facing childhood cancer, did not allow it to define who she was. This Hero Fund aspires to give hope to kids fighting cancer through research.

Eric Raabe Ph.D., M.D.

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Funded: 07-01-2023 through 06-30-2024
Funding Type: Research Grant
Institution Location: Baltimore, MD
Institution: Johns Hopkins University School of Medicine affiliated with Johns Hopkins Children's Center

Atypical teratoid/rhabdoid tumors (AT/RT) are the most common malignant brain tumors of infancy. Standard therapies lead to severe toxicities and poor overall survival. Dr. Raabe aims to identify novel therapies to reduce toxicities and improve survival. Dr. Raabe and colleagues found that cancer cells rely on activation of the integrative stress response (ISR) to maintain cell equilibrium and survival. However, if the ISR is activated too intensely or for too long, cells undergo apoptosis and die. Paxalisib and gemcitabine are medications that induce considerable cell stress, further activating the integrative stress response, and extending survival in models of AT/RT. Dr. Raabe is investigating how these medications act together to target cell stress pathways and their impact on survival in models of AT/RT. The findings will translate directly through the International Pacific Pediatric Neuro-Oncology Consortium (PNOC) into a new clinical trial treating children with relapsed or refractory AT/RT. This grant is named for Hannah’s Heroes, a St. Baldrick’s Hero Fund created in honor of Hannah Meeson and pays tribute to her fight by raising awareness and funding for all childhood cancers because kids like Hannah “are worth fighting for.”

Alexander Kabanov Ph.D, D.Sci.

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Funded: 07-01-2023 through 06-30-2025
Funding Type: Research Grant
Institution Location: Chapel Hill, NC
Institution: University of North Carolina at Chapel Hill affiliated with UNC Children's Hospital

Dr. Kabanov and colleagues propose an entirely new way to treat medulloblastoma, the most common malignant pediatric brain tumor. Current treatment requires radiation followed by a year of chemotherapy, fails almost half the patients, and can leave survivors with lifelong brain injury. Tiny extracellular vesicles called exosomes produced by a type of immune cell called macrophages were discovered to travel from the bloodstream into brain tumors. Dr. Kabanov will load exosomes with an agonist of Toll-like receptor to reprogram medulloblastoma tumor associated myeloid cells and enhance their tumoricidal properties. If successful, the therapy will improve medulloblastoma treatment by replacing the current radiation and chemotherapy with the one that is less toxic and more effective. The second year of this grant is funded by and named for the Miracles for Michael Fund, a St. Baldrick's Hero Fund created in memory of Michael Orbany who was diagnosed with medulloblastoma when he was 6 years old. After completing initial treatment, his cancer relapsed within a year and he passed away at the age of nine. Michael had unwavering faith and perseverance, wanting most of all to make others happy. This fund honors his tremendous strength to never ever give up. The first year of this grant is funded by and named for the Strong & Courageous Hero Fund, established in honor of Jonah. It celebrates his survivorship from medulloblastoma and his goofy, loving, inclusive personality. This fund was inspired by Jonah’s desire to help other kids with cancer and supports research of brain tumors and the multitude of challenges facing survivors post treatment.

Carl Allen M.D., Ph.D.

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Funded: 07-01-2023 through 06-30-2025
Funding Type: Research Grant
Institution Location: Houston, TX
Institution: Baylor College of Medicine affiliated with Vannie E. Cook Jr. Children's Cancer and Hematology Clinic, Texas Children's Hospital

Pediatric Burkitt Lymphoma (BL) arises from errors during immune (B cell) development. Treatment failure is associated with dismal outcomes, and many pediatric BL survivors will suffer long-term toxicities from therapy, highlighting the need to explore opportunities to identify patients who may be cured with less intense therapies. Little is currently known about the biology of pediatric BL and clinical implications of specific mutations. Therefore, systematic analysis of tissue from children treated on clinical trials represents a unique opportunity to gain insights from valuable specimens to inform biologic risk-based chemotherapy and identify potential targeted therapeutic strategies. Dr. Allen will characterize intrinsic and acquired genetic factors that underlie pathogenesis and predict response to therapy in patients with pediatric BL who have completed treatment on COG clinical trials. This grant is funded by and named for Jack's Pack - We Still Have His Back, a St. Baldrick's Hero Fund. Jack Klein was a ten year old who loved life, laughing and monkeys. During his illness, his community of family and friends near and far rallied around him under the moniker "Jack's Pack". Their slogan was "We have Jack's Back". After Jack succumbed to Burkitt's Lymphoma, his "pack" focused their energy and efforts to funding a cure...just as Jack would have wanted.

Ling Li Ph.D.

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Funded: 07-01-2023 through 06-30-2025
Funding Type: Research Grant
Institution Location: Duarte, CA
Institution: Beckman Research Institute of the City of Hope

Each year, approximately 1000 Americans aged 20 years or younger are diagnosed with acute myeloid leukemia (AML). Currently, even the most effective targeted drug BCL2 inhibitor-venetoclax (VEN) cannot eradicate all leukemia cells. The remaining cells cause disease recurrence and result in a very low overall survival rate for AML patients. In preliminary studies, Dr. Li found that targeting an enzyme called ADSS2 promotes pediatric AML cells sensitivity to VEN induced mitochondrial apoptosis, resulting in a synthetic lethality. This study will ask how ADSS2 preserves AML cells fitness and test the effectiveness of a first-in-class ADSS2 inhibitor combined with VEN or other BCL2 family protein MCL1 inhibitor in models of AML. If successful, this could lead to a clinical trial with potential impact for childhood AML patients.