Acute Myeloid Leukemia Research Studies at Texas Children’s Cancer and Hematology Center - $150,000

Acute Myeloid Leukemia Research 

Acute myeloid leukemia (AML) is a specific type of blood cancer that affects children of all ages. It makes up 20-25% of all blood cancers in children and remains challenging to cure. Current treatments rely on harsh chemotherapy that can make children susceptible to infections and other complications, such as heart failure, requiring them to stay in the hospital for months at a time. Despite this intense therapy, over 40% of children with AML will die of their disease and 50% will relapse, meaning their cancer comes back after initially being cured. Our research focuses on understanding how certain cancer cells escape cell death from chemotherapy, as this is a primary cause of relapse.

 

 

Natural Killer Cell Trials at MD Anderson Children’s Cancer Hospital - $105,000

Brain Tumor Trial

Phase I clinical trial testing the safety and feasibility of using autologous expanded natural killer(NK) cells (a subset of a child’s white blood cells from their own immune system that have been collected and multiplied) and infusing them into the fourth ventricle of the brain in children with relapsed fourth ventricle brain tumors.  A further step of the trial is evaluating the dose levels of therapy. NK cells have the innate ability to seek and destroy cancer cells.  This trial includes the FDA required validation of the protocol and facility to manufacture these NK cells and employing highly trained personnel necessary to make NK cells in accordance with national safety standards. This trial incorporates the emerging concept of local delivery of therapy by infusing the NK cells directly into the fourth ventricle of pediatric patients using a surgically implanted ventricle catheter.

 

Solid Tumor Trial

Clinical trial for children with very aggressive solid tumors, primarily sarcomas that are resistant to standard chemotherapy and radiation, to explore “ready to use” natural killer (NK) cells provided from blood bank donors and expanded using an FDA approved manufacturing technique to expand these NK cells.  NK cells offer the hope that an immune approach will change the course of treatment for these very aggressive tumors and this project explores the use of NK cells of donors rather than the patient’s own NK cells.

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Sarcoma Research Studies at Texas Children’s Cancer Center - $405,000

Osteosarcoma Studies

Studies performed in test tubes and on mice to understand the molecular events that are regulated by microRNA miR-214 which is thought to increase the tumor forming capability and metastatic spread of osteosarcoma, a bone sarcoma predominantly seen in children and young adults with overall 5-year survival rates which have remained largely unchanged for the past 30 years.  If the mechanism can be understood by which miR-214 affects tumor formation and progression, miR-214 may be identified as a new target for which therapies may be developed to improve survival rates.

 

Sarcoma Research Studies and Trials at MD Anderson Children’s Cancer Hospital - $230,000

Sarcoma Research Studies

HER 4 Study

Study using a new agent to target and inhibit HER 4 which allows osteosarcoma to metastasize to the lungs.  If pulmonary metastasis can be prevented, then the hope is that the survival of children with osteosarcoma may be increased from 65 percent to over 85 percent.

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Hydroxycholroquine Study

Study based on pre-clinical studies of adding hydroxychloroquine to existing chemotherapy to inhibit autophagy which is the process that allows cells to survive chemotherapy.

 

Correlative Studies

Study evaluating blood samples from patients enrolled in sarcoma trials using a perfected method of detecting circulating sarcoma cells in the blood for evidence of early relapse so that new therapy may be initiated and be impactful.  Feasibility has been shown to use this same method to isolate tumor cells from the blood to study biologic changes in cancer cells during treatment to help unravel the biologic processes responsible for relapse.

 

Sarcoma Trials

New approaches to overcome the mechanisms that allow sarcoma cells to metastasize to the lungs.

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Inhalation Aerosolized Therapy

Trial of aerosolized chemotherapy to test dose levels for inhaled interleukin-2 (IL-2, a protein that regulates the activities of white blood cells) in children with osteosarcoma. Administering inhaled drugs in a clinical trial setting requires careful monitoring of the effects of the therapy on lung function.  Funding of this trial includes providing devices that send information digitally from patient homes as part of trial observations.  Results from the completed trial are currently being analyzed to determine how to best use such therapies in the future.  

 

Sarcoma/Brain/Spinal Cord Research Studies and Trials and Pediatric Supportive Care at MD Anderson Children’s Cancer Hospital - $285,000

Drug Delivery Microdevice

The rarity and diversity of sarcoma tumors make treating the disease difficult. Thanks to a partnership between MD Anderson and the Massachusetts Institute of Technology, researchers can now use an implantable microdevice — 4 mm in diameter — to test up to 18 therapies at once and measure their effects directly at a tumor site. The team works with pathologists to learn results in just 24 hours! This approach is moving the field in a more personalized direction for the treatment of pediatric sarcoma and allowing clinical researchers to evaluate potential drug candidates much faster than before.

 

Treating Pediatric Brain and Spinal Cancers with Stem Cell Transplantation

We need new therapeutic approaches to improve outcomes for young patients facing brain and spinal cord cancers, and a swelling tide of oncologists believe stem cell transplantation holds undiscovered promise. An MD Anderson Children’s Cancer Hospital team is combining high-dose chemotherapy with stem cell transplantation to reduce tumor burden while a “replacement” immune system prevents its return. The team is also examining the role vascular permeability plays in treatment effectiveness.

 

Pediatric Supportive Care Research

While many researchers at MD Anderson Children’s Cancer Hospital focus on specific anti-cancer drug interactions, others focuses on ways to supplement that approach holistically. New studies are looking at ways to better a patient’s cancer experience — either by improving clinical outcomes or minimizing distress and suffering — through a commitment to energy-balance principles. This refers to the effects of diet, physical activity and genetics on a patient’s disease and overall health.