It’s no small thing

8-7-2017
It’s no small thing – Sheba develops groundbreaking new research techniques using mice

Sheba’s far-reaching research capacities are pushing the boundaries of medical knowledge on a daily basis. From the commonplace to the most rare medical conditions, Sheba’s researchers are working to better save lives with new treatments. Techniques developed in one research study often fuel innovations in other areas.

In the search for a better understanding into Angiomyolipoma, a form of amniotic kidney tumor, Sheba researchers created a model of the tumor in mice mimicking the human version of the tumor allowing them to conduct more detailed analysis of the tumor than ever before. This breakthrough has potential to open the development of many new drugs that previously could not be tested on animals.

Led by Prof. Benjamin Dekel, of the Pediatric Stem Cell Research Institute and the Director of the Division of Pediatric Nephrology at the Edmond and Lily Safra Children’s Hospital at Sheba, this groundbreaking study replicated the amniotic kidney tumor in mice. This unusual form of tumor is made up of three types of tissue: veins, smooth muscle, and fat, typically appearing in patients with the genetic disease Tuberous Sclerosis Complex (TSC).

Using the mouse model, the team was then able to identify the protein PPARG as a key protein in the formation and growth of the human. Indeed, when the tumor was treated with PPARG inhibitors, the tumor’s growth dramatically halted and significantly lost the ability to grow once they were injected back into the mice.

Though researchers from around the globe have set their sights on unmasking this tumor, as, until now they were unable to conduct animal testing, much remained unknown about the cells from which the growth is conceived, and indeed about the mechanisms that cause it to appear and grow. What medications had been developed to treat this tumor (inhibitors of the mTOR path) are only effective in some of the patients, underscoring the urgent need for new treatments.

Thanks to Prof. Dekel and his team, a significant step has been taken towards the development of effective treatments for this rare form of cancer. It remains to be seen how many other life-saving treatments will come into being as a result of their groundbreaking research technique.

 






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