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Development and Exploration of the Feasibility of Using Locally Synthesized Small Molecule Inhibitors to Treat Human Acute Leukemia Cells

Sponsor
Taipei Medical University WanFang Hospital (Other)
Overall Status
Unknown status
CT.gov ID
NCT01142375
Collaborator
(none)
100
Enrollment

Study Details

Study Description

Brief Summary

Acute leukemias are commonly seen in elderly and have no effective therapy. In recent years, small molecule inhibitors have shown a tremendous promise in cancer treatment such as chronic myeloid leukemia and lung cancer. Thus, in this proposal, we intend to use our novel patented "indole" compound to conjugated with hydroxamic acid in histone deacetylases inhibitor, suberoylanilide hydroxamic acid and further modify its structure to generate novel small-molecule anticancer compounds. By screening acute leukemic cell lines, we will look for compounds that have shown potential for future drug development. In our preliminary studies, we have identified a novel compound, MPT0E001, to have marked growth inhibitory activity, induce apoptosis and downregulate c-Myc protein level. We intend to use MPT0E001 as a basis to develop novel compounds and test them in multiple leukemic cell lines and primary leukemia samples (Specific aim 1) and identify the mechanisms for downregulation of c-Myc and other pathways such as NF-kappaB and Akt (Specific aim 2). Once we have identified the lead compounds, we will use murine model to assess the acute toxicity profiles in different dose ranges and examine the effects of blood counts and vital organs. After toxicity study, we will test the lead compounds using in vivo xenogenic murine model using both cell lines and primary leukemia. Using this approach, we hope to develop novel compounds that are able to be used in future leukemic therapy.

Condition or Disease Intervention/Treatment Phase

    Study Design

    Study Type:
    Observational
    Anticipated Enrollment :
    100 participants
    Observational Model:
    Cohort
    Time Perspective:
    Prospective
    Official Title:
    Development and Exploration of the Feasibility of Using Locally Synthesized Small Molecule Inhibitors to Treat Human Acute Leukemia Cells
    Study Start Date :
    Apr 1, 2008

    Outcome Measures

    Primary Outcome Measures

      Eligibility Criteria

      Criteria

      Ages Eligible for Study:
      N/A and Older
      Sexes Eligible for Study:
      All
      Accepts Healthy Volunteers:
      No
      Inclusion Criteria:
      • Acute Myeloid Leukemia
      Exclusion Criteria:
      • Hematologic malignancies other than AML

      Contacts and Locations

      Locations

      No locations specified.

      Sponsors and Collaborators

      • Taipei Medical University WanFang Hospital

      Investigators

      • Principal Investigator: Hsin-Gjin Eugene Liu, Taipei Medical University WanFang Hospital

      Study Documents (Full-Text)

      None provided.

      More Information

      Publications

      None provided.
      Responsible Party:
      , ,
      ClinicalTrials.gov Identifier:
      NCT01142375
      Other Study ID Numbers:
      • Leukemia-001
      First Posted:
      Jun 11, 2010
      Last Update Posted:
      Jun 11, 2010
      Last Verified:
      Jun 1, 2010
      Keywords provided by , ,
      AML
      Additional relevant MeSH terms:
      Leukemia

      Study Results

      No Results Posted as of Jun 11, 2010