From Fish to Pharmacy

Pharmaceutical testing by zebrafish molecular screening yields lendaldekar for leukemia

/ Author:  / Reviewed by: Robert Carlson, M.D

(RxWiki News) Genetic experiments have come a long way from growing peas. A new line of testing created a zebrafish with cells that can glow under certain conditions, which helps scientists figure out what drug is the best at killing cancer cells.

The research used the fish as a test subject for leukemia research, enhancing the immune system's T-cells to glow under a microscope when activated.

After administering the drug, researchers were quickly able to determine how effective the sample was at killing the cancerous cells just by looking at what was glowing. 

"Ask your oncologist about clinical trials available near you."

Part of a long-standing research project at the Huntsman Cancer Institute at the University of Utah, the laboratory showed that the molecule worked effectively in mice as well. The technique again performed beyond expectations in tumor samples from several forms of leukemia, including T-ALL, CML and B-ALL.

The laboratory analyzed 26,400 molecules, and the researchers decided that their best candidate for treating leukemias was the molecule which they have named Lendaldekar (LDK).

In short, Lendaldekar kills immature T-cells. In the human body, these developing cells shouldn't be in the bloodstream until they mature, because normally they're protected inside the small organ known as the thymus.

Instead, in some diseases such as leukemia T-cells rapidly multiply, spilling over into the blood and causing symptoms due to the lack of proper immune development. This activity causes damage by preventing other blood cells from developing normally.

"This is the first successful approach using zebrafish larvae to identify molecules with potency against leukemia from a small molecular library containing compounds with unknown activity," said Nikolaus Trede, M.D., one of the authors of the study.

"We are encouraged by LDK's path from a virtual unknown to a potential powerhouse against leukemia, and we are working on discovery of LDK's cellular target, which may ultimately help convert the compound into a drug that can be used in patients with leukemia."

In a two-week-long experiment described in the study, more than 60 percent of the zebrafish treated with LDK went into long-term remission of more than nine months, while others in the control group without LDK died from the cancer in less than 40 days.

Similar results were observed in follow-up laboratory experiments with mice, as well as experiments on human tumor samples.

Results were published in the journal of the American Society of Hematology, Blood.

Statements concerning any possible financial conflict of interest in the publication of this research were not made publicly available.

Reviewed by: 
Review Date: 
April 7, 2012
Last Updated:
November 8, 2012