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.
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.
Inducible Caspase 9 Suicide Gene To Improve The Safety Of Donor T Cell Addback After Haploidentical Stem Cell Transplantation
Funder
National Health and Medical Research Council
Funding Amount
$566,232.00
Summary
In bone marrow transplantation, donor immune cells are important for fighting cancer cells and infections but can also attack healthy tissues, causing graft-versus-host disease (GVHD). We will use gene technology to insert a safety switch called inducible capase 9 (iCasp9) into the donor immune cells which will make them susceptible to an otherwise non-toxic chemical. This will make it safer to boost the recipients’ immunity because these cells can be rapidly eliminated if GVHD occurs.
A Phase 1 Clinical Trial Of A Human Chimeric Anti-Activated DC Antibody To Prevent AGVHD In High Risk Allo HSCT.
Funder
National Health and Medical Research Council
Funding Amount
$670,736.00
Summary
Bone Marrow transplants provide life saving therapy for leukaemias, lymphomas and other life threatening blood disorders. One of the major life threatening complications is acute graft versus host disease (AGVHD) in which the doner immune system damages the patient's skin, liver and gut, amongst other tissues. Dendritic cells initiate and direct immune responses. We have shown that dendritic cells are central to the initiation of AGVHD and have shown that a marker called CMRF-44 is expressed on ....Bone Marrow transplants provide life saving therapy for leukaemias, lymphomas and other life threatening blood disorders. One of the major life threatening complications is acute graft versus host disease (AGVHD) in which the doner immune system damages the patient's skin, liver and gut, amongst other tissues. Dendritic cells initiate and direct immune responses. We have shown that dendritic cells are central to the initiation of AGVHD and have shown that a marker called CMRF-44 is expressed on activated dendritic cells before AGVHD emerges. We have developed potential new therapeutic antibodies that target activated dendritic cells and shown that they are effective in preclinical studies. This project will further validate these antibodies, then test their safety and their ability to prevent AGVHD in patients. The trial will also test whether they have the expected additional beneficial effect of preserving protective anti-viral and anti leukaemic immune responses.Read moreRead less