One of the most dreaded forms of cancer Leukaemia or blood cancer as it is commonly called has a new drug treatment that has raised hopes across the world. In an extensive research being carried out at the Children’s Cancer Hospital at The University of Texas M. D. Anderson Cancer Center, scientists have observed that a new kind of highly targeted treatment effectively treats acute leukaemia. In the animal models used by them, they observed that the growth of cancer cells was very effectively inhibited by not letting the cancerous cells being cleaned of their damaged proteins.
The researchers reported in Blood’s March online issue that the NPI-0052, the new proteasome inhibitor they were experimenting with, not only successfully kills leukemia cells, but also shows greater efficacy than its predecessor bortezomib when combined with other agents in animal models.
Proteasomes, according to these researchers, indirectly promotes cell growth by cleaning out the mutated or damaged proteins from within cells. This enhanced cell growth allows cancer cells to rapidly reproduce and proliferate. What proteasome do is by not allowing the mutated or damaged protens from being cleaned up effectively work as inhibitors blocking this process, resulting in cell death or apoptosis of the cancerous cells.
As far as proteasome inhibitors are concerned Bortezomib is the first and only one to have received the FDA-approval. Despite being highly effective for treating multiple myeloma and mantle cell lymphoma, in clinical trials against leukemia it has proven to be ineffective as a single agent. NPI-0052 varies from bortezomib in ways that scientists at M. D. Anderson hope will make NPI-0052 effective in a human clinical trial.
According to senior author Joya Chandra, Ph.D., assistant professor of pediatrics from the Children’s Cancer Hospital at M. D. Anderson “NPI-0052 targets the proteasome through different intermediaries and is more potent than bortezomib in leukemia cells and therefore we can use less of the drug to inhibit the proteasome”.
What is really remarkably amazing, and therefore promising is that when compared to bortezomib as a single agent, NPI-0052 inhibits the main enzymatic activity of the proteasome three times more effectively. As if this in itself were not enough NPI-0052 achieves four-fold greater synergistic effects than bortezomib when combined with a histone deacetylase (HDAC) inhibitor, another anti-cancer agent,.
Chandra’s group is the first group to be studying the effects of the drug in acute leukemia models and M. D. Anderson currently has a Phase I clinical trial led by principal investigator Razelle Kurzrock, M.D., to test NPI-0052 on adult patients with solid tumor malignancies and recurrent lymphoma.
A highly optimistic Chandra says “This drug, so far, has shown efficacy in animal models of leukemia, myeloma and colon cancer, and it has worked to kill multiple myeloma cells resistant to bortezomib,” he goes on to add that “as a result of our research, we’re looking at the feasibility of combining NPI-0052 with HDAC inhibitors in the future to treat leukemia”.
Now this does bring a completely novel drug to treat leukaemia close to reality which works on an equally novel method of making the cancerous cells kill themselves there by not allowing their growth simply by ensuring they are unable to clean themselves of the damaged and mutated proteins. Encouraging, isn’t it?
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