Blood transfusion is a vital part of medical practice. Patients expect that this treatment will be given only where it will help them, while the community expects that the money spent on transfusion is being spent wisely. The proposed program of work addresses critical deficits in understanding in transfusion medicine. It will improve the evidence base to inform clinical decision-making, provide strategies which ensure this evidence is translated promptly into clinical practice, and extend this ....Blood transfusion is a vital part of medical practice. Patients expect that this treatment will be given only where it will help them, while the community expects that the money spent on transfusion is being spent wisely. The proposed program of work addresses critical deficits in understanding in transfusion medicine. It will improve the evidence base to inform clinical decision-making, provide strategies which ensure this evidence is translated promptly into clinical practice, and extend this evidence base to inform clinical policy and planning in Australia.Read moreRead less
A Phase I Study Of Autologous CD19 Specific Chimeric Antigen Receptor T-cells For Therapy Of Relapsed And Refractory B-cell Leukaemia And Lymphoma (The Auto-CAR19 Trial).
Funder
National Health and Medical Research Council
Funding Amount
$584,666.00
Summary
Most people with leukaemia and lymphoma who relapse early after chemotherapy die of their disease. Inserting special genes into immune cells can enable them to kill leukaemia and lymphoma and has led to dramatic cures, but the cost of the viral vectors used to make these cells is prohibitively expensive. We will make leukaemia and lymphoma specific immune cells from patients using an inexpensive non-viral system, then administer the immune cells to patients to assess their safety and efficacy.
A Phase I Study Of PiggyBac CD19 Specific Chimeric Antigen Receptor T-cells For Therapy Of Persistent And Relapsed B-cell Leukaemia And Lymphoma Post Allogeneic Stem Cell Transplantation (The CARTELL Study).
Funder
National Health and Medical Research Council
Funding Amount
$357,590.00
Summary
Most people with relapsed leukaemia and lymphoma after bone marrow transplant die of their disease. Inserting special genes into immune cells can enable them to kill leukaemia and lymphoma and has led to dramatic cures, but there is little experience in bone marrow transplant patients. We will make leukaemia and lymphoma specific immune cells from normal bone marrow transplant donors, then administer the immune cells to transplant patients to assess their safety and effectiveness.
Proof-of-concept Studies For A Novel Anti-thrombotic Agent
Funder
National Health and Medical Research Council
Funding Amount
$632,352.00
Summary
Blood clots cause most heart attacks and strokes, and platelets are the blood cells that form these clots. Drugs that block platelet function, such as aspirin, are used to prevent heart attack and stroke but are frequently ineffective. Here, we will develop a new drug that prevents platelet incorporation into blood clots, that will be suitable for the prevention of heart attack and stroke in humans, and that may improve on existing therapies.
A Clinical Trial Of Partially HLA-matched Unrelated Donor Microtransplantation For Prevention Of Relapse In Patients With Acute Myeloid Leukaemia Ineligible For Standard Haemopoietic Stem Cell Transplantation
Funder
National Health and Medical Research Council
Funding Amount
$154,828.00
Summary
Acute myeloid leukaemia has a poor prognosis in patients unable to undergo bone marrow transplant, in particular in the elderly. No proven therapy improves their poor outcome. There is an urgent need to identify clinically applicable, non-toxic therapies for this group of patients. We will perform a clinical trial of "microtransplantation" using unrelated stem cell donors in combination with chemotherapy to try to reduce the relapse rate in these patients without the toxic effects of standard st ....Acute myeloid leukaemia has a poor prognosis in patients unable to undergo bone marrow transplant, in particular in the elderly. No proven therapy improves their poor outcome. There is an urgent need to identify clinically applicable, non-toxic therapies for this group of patients. We will perform a clinical trial of "microtransplantation" using unrelated stem cell donors in combination with chemotherapy to try to reduce the relapse rate in these patients without the toxic effects of standard stem cell transplantation.Read moreRead less
Harnessing RNA Interference In Gene Therapy Vectors For ?-thalassaemia
Funder
National Health and Medical Research Council
Funding Amount
$719,188.00
Summary
There is an urgent need to develop safe and effective treatments for ?-thalassaemia. We anticipate that ?-globin-specific RNAi sequences will synergise with ?-globin transgene expression to achieve balanced ?-/?-globin ratio in a clinical setting. Given that one of the major issues with current gene therapy vectors is achieving high levels of expression, we believe this will be a more effective gene therapy strategy than ?-globin transgene expression alone.
Targeting Disease-initiating Cells In Myeloproliferative Neoplasms
Funder
National Health and Medical Research Council
Funding Amount
$477,170.00
Summary
The myeloproliferative neoplasms (MPN) are a related group of blood disorders. Despite the advent of targeted therapies, patients have significant ongoing morbidity, mortality and financial cost. A key reason underlying the persistence of disease is the presence of a stem cell pool that is resistant to targeted therapy. Clinical data has suggested that interferon may target these disease stem cells. We propose to use in vivo, validated disease models to investigate the role of interferon in MPN.
The Evolution Of Acute Myeloid Leukaemia By In Situ Transformation Of Haematopoietic Stem Cells
Funder
National Health and Medical Research Council
Funding Amount
$646,966.00
Summary
Acute myeloid leukaemia (AML) is a devastating form of blood cancer that can affect people of any age. The survival of patients with AML is poor and this is because the disease usually comes back after chemotherapy (this is called relapse). Fewer than half of all patients with AML can be cured. We have recently developed a new, and improved, model of AML in the lab, which we will use to test an exciting new treatment for patients with AML.