The Childhood Cancer Blog

Jay Scott: Zebrafish, MicroRNA and ALSF...oh my!

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You hear us saying all the time how the funds you help us raise are supporting innovative research projects searching for a cure – and they truly are! Just in the last week we received notice that two projects that we funded have been published in prestigious medical journals demonstrating their significance not just to us – but to the medical community as a whole. Right up there with the clinical findings is the feedback we receive from the grantees about the impact that your support makes possible. Check it out…

“Without ALSF's support we would not even be close to this discovery. Now because of your funds we are publishing the findings in Blood. The technician I was able to hire is absolutely fabulous and continues to make very nice progress on target identification.” - Nikolaus Trede, MD, PhD

Nikolaus Trede, MD, PhD, a researcher at the Huntsman Cancer Institute was awarded ALSF funding looking at a new treatment for T-cell acute lymphocytic leukemia (T-ALL), a particularly hard to treat type of childhood cancer. Dr. Trede proposed to test the treatment in zebrafish. Why zebrafish? Well, little known fact to us non-scientists, but apparently zebrafish’s genomes and immune systems are similar in many ways to those of humans and they can easily mimic human cancers. The targeted treatment that the research team tested, Lenaldekar (LDK), was successful in treating the zebrafish with T-ALL and even showed promise in slowing the disease in mice. This is even more exciting because since LDK targets the cancer cells (not the healthy cells as with existing chemotherapy treatments) the treatment is significantly less toxic. You can read more about Dr. Trede’s study that was published in last week’s issue of Blood (which is also posted on our website).

“This work would not be possible without the support of ALSF! I am so grateful to you guys!” – Rami I. Aqeilan, PhD

Rami I. Aqeilan, PhD of The Ohio State University also received funding from ALSF for a project that was looking at osteosarcoma, the most common type of bone cancer and a leading cause of cancer death in adolescents. Dr. Aqeilan and his team identified (for the first time) a MicroRNA signature reflecting the development of the disease. What does this mean? Well, the researchers found that the level of expression of specific MicroRNAs directly correlated to whether a tumor developed into metastatic disease or was more likely to be responsive to therapy. I like to think of it as uncovering a recipe of sorts. You need certain amounts of specific ingredients to end up with a cookie. Too much of one thing and too little of another and it won’t all come together. Of course, in this case – that cookie is an osteosarcoma tumor, but you get my gist. By identifying this MicroRNA “recipe”, the researchers can better predict who might benefit most from specific treatments. In addition, their findings may help lead to more targeted treatments so desperately needed for kids battling the disease. You can read more about this study in the April 2012 issue of Cancer Research (also posted on our website).

So, yes – your support and efforts on behalf of ALSF are making a significant and meaningful difference in the lives of children with cancer. Thank you, as always, for helping to make sure zebrafish and MicroRNA are a part of our vocabulary!

-Jay Scott, Alex’s Dad