TAS1553 for Myelodysplastic-Myeloproliferative Diseases
A Study of TAS1553 in Subjects With Relapsed or Refractory Acute Myeloid Leukemia (AML) and Other Myeloid Neoplasms
See full descriptionAbout the trial for Myelodysplastic-Myeloproliferative Diseases
Treatment Groups
This trial involves 2 different treatments. TAS1553 is the primary treatment being studied. Participants will be divided into 2 treatment groups. There is no placebo group. The treatments being tested are in Phase 1 and are in the first stage of evaluation with people.
Eligibility
This trial is for patients born any sex aged 18 and older. You must have received newly diagnosed for Myelodysplastic-Myeloproliferative Diseases or one of the other 8 conditions listed above. There are 10 eligibility criteria to participate in this trial as listed below.
Approximate Timelines
Measurement Requirements
This trial is evaluating whether TAS1553 will improve 4 primary outcomes and 12 secondary outcomes in patients with Myelodysplastic-Myeloproliferative Diseases. Measurement will happen over the course of At specific timepoints from predose up to Day 2 of Cycle 2 (28 days per cycle).
Patient Q & A Section
What are common treatments for myelodysplastic-myeloproliferative diseases?
There are a small number of well characterised agents specifically used against myelodysplastic syndromes (MDS) and myeloproliferative syndromes (MPS) as well as non-specific agents with anti-inflammatory and immunomodulating properties (cyclophosphamide, vincristine, and hydroxycarbamide). Overall, a combination of these therapies is common.
How many people get myelodysplastic-myeloproliferative diseases a year in the United States?
About 15,000 people in the United States are diagnosed with an MDS/MPD a year, a high burden, but most suffer silently, receiving only secondary care.
What are the signs of myelodysplastic-myeloproliferative diseases?
Patients with peripheral pancytopenia are at increased risk of MDS. The prognosis of myelodysplastic-myeloproliferative disease is also poor, as they have a life expectancy of less than one year.
What causes myelodysplastic-myeloproliferative diseases?
The exact cause of myelodysplastic-myeloproterative diseases is unknown. Exposure to multiple environmental stimuli is likely to influence the development of this disease. Most cases of myelodysplastic-myelopproterative disease are genetically inherited.
What is myelodysplastic-myeloproliferative diseases?
MMPD and MDS are both disorders that result from clonal mutations in the hematopoietic progenitor cells. Myelodysplastic syndromes are characterised by a low hemoglobin, low pancytopenia and normal blood cell count. On the contrary, with MDS patients, there are an increased numbers of immature neutrophil and monocyte count along with increased number of mature neutrophils in the peripheral blood. Results from a recent clinical trial support the possibility if hematopoietic stem cell mutation is responsible for the monocyte counts elevation in the blood.
Can myelodysplastic-myeloproliferative diseases be cured?
To the best of the authors' knowledge, this is the first publication in the international literature to comprehensively cover the current advances in the treatment of MM/MPD. The reported data may provide a platform for further research.
What is the latest research for myelodysplastic-myeloproliferative diseases?
For patients in whom no treatment has been found, a multi-institutional collaborative effort is starting to identify prognostic markers and risk-stratifying agents. Additionally, it remains to be determined whether and in what context the use of the myelodysplastic-myeloproliferative classification classification, an international panel of pathologists has recommended for its clinical utility.
Does myelodysplastic-myeloproliferative diseases run in families?
Although MDS runs in families and a small percentage of all MDS patients in some regions have a family history of MDS, little is known about the underlying genetic mechanisms for the development of MDS in MDS families.
What is the primary cause of myelodysplastic-myeloproliferative diseases?
MDS seem to appear as a result of a complex interaction of genetic and physiological risks. The mechanism of MDS is not completely clear. The two main theories are aging and chronic autoimmune processes. There is no single theory explaining the formation of MDS unequivocally.
Who should consider clinical trials for myelodysplastic-myeloproliferative diseases?
Clinical trials for MDS and/or leukemias are feasible, providing results for patients and physicians. The patients' preference is the only important predictor for deciding whether to participate.
How does tas1553 work?
Results from a recent clinical trial is first to show that tas1553 directly binds to DNA and downregulates a subset of NF-κB target genes and is the first report on the molecular mechanism of tas1553. Because tas1553 does not affect B lymphocyte development, these effects occur mostly at the BMP signalling pathway and not NF-κB. Future work will determine whether tas1553's anti-cancer activity (at least in part) results from its effects on B cell differentiation (at least in part).
What is tas1553?
Thus we could see tas1553 as a linker among the different functional domains with potential applications. We are still missing an understanding or hypothesis regarding to what functions the Tas1563 protein plays in the cell.