Unlocking A Medical Mystrey: Laron Syndrome

Rare form of genetic mutation found in dwarfism also prevents cancer and diabetes.
3:00 | 12/19/13

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Transcript for Unlocking A Medical Mystrey: Laron Syndrome
you on a journey to unlock new hope in a medical mystery. We travel 3,000 miles and find a community of people with dwarfism, little people living in remote villages and something about them could hold the secret to preventing cancer for everyone. What is it? Abc's john quinones takes us there to find out. Reporter: Meet freddie, maria and lucas. They're a kind of medical wonder, little people with a rare kind of dwarfism. The same genetic mutation that keeps them small also prevents them from getting cancer and diabetes. How old are you? You're 15? Reporter: Lucas stands less than three feet tall, his sister five years younger is nearly twice his height. What is life like being your size? Reporter: Life can be difficult, he says, always having to ask for help, but he says he's gotten used to it. Worldwide only about 300 people are known to have this rare genetic disorder and fully one-third of them live here in the remote mountain side villages of southern ecuador this syndrome is so prevalent here. These doctors have been studying them for more than 20 years hoping to harness the secrets of their genes into life saving medication. Now they're developing a pill that mimics the syndrome. The key human growth hormone which fuels the growth of cancer cells. Doctors hope that by blocking its receptors they can significantly reduce the on set of cancer and kill the cancer cells when they form. That's what happens naturally in little people like yolanda. You could hold the key to preventing cancer and diabetes. Yes, she says. It will make me very happy if those diseases go away. Reporter: We met 13-year-old maria who, like the others, continues to offer herself up for research. If my daughter can help save other people, her mother says, then we'll do it with pleasure. These little people of ecuador willing to give the world a huge gift. What a gift that would be, a pill that might prevent cancer and diabetes. Researchers have made progress using the mutation to block cancer I mice and now they're hoping and working on developing that pill so it might affect humans. This is real scientific research going on tonight? It's a huge deal. As we speak, some of the world's top scientists are conducting six clinical trials in holland, italy, the mayo clinic. They're not only studying the potential effects of this medication but a star vacation diet that mimics this syndrome. High hopes. I love the woman's reaction when you said do you know you could be the key to preventing cancer in the world. It was a lovely expression. Thank you so much, john, for the journey.

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