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

NCT ID: NCT01142375

Last Updated: 2010-06-11

Basic Information

• Recruitment Status: UNKNOWN
• Total Enrollment: 100 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2008-04-30

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.

Conditions

Acute Myeloid Leukemia

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Eligibility Criteria

Inclusion Criteria

• Acute Myeloid Leukemia

Exclusion Criteria

• Hematologic malignancies other than AML

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Taipei Medical University WanFang Hospital

OTHER

Sponsor Role: lead

Responsible Party

Taipei Medical University WanFang Hospital

Principal Investigators

Hsin-Gjin Eugene Liu

Role: PRINCIPAL_INVESTIGATOR

Taipei Medical University WanFang Hospital

Other Identifiers

Leukemia-001

Identifier Type: -

Identifier Source: org_study_id