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

NCT01142375 | Unknown

• 1 other identifier: Leukemia-001
• Study Type: observational
• Target: 100
• Locations: 0 countries
• Sites: N/A

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

Keywords

AML

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Acute Leukemia

You may qualify if:

• Acute Myeloid Leukemia

You may not qualify if:

• Hematologic malignancies other than AML

Sponsors & Collaborators

• Taipei Medical University WanFang Hospital: lead

Biospecimen

Retention: SAMPLES WITHOUT DNA

Bone Marrow

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Hsin-Gjin Eugene Liu

  Taipei Medical University WanFang Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: June 9, 2010
• First Posted: June 11, 2010
• Study Start: April 1, 2008
• Last Updated: June 11, 2010

Record last verified: 2010-06