With all our talk about finding new therapies for people it’s sometimes easy to lose track of the kind of very basic research that it sometimes takes to develop those therapies. And by very basic what I’m talking about in this case is yeast.
Yes, yeast are a long way from being human. And yes, they don’t exactly suffer the recognizable symptoms of a condition like Parkinson’s disease. But what they do is divide quickly and produce a lot of protein that scientists in a lab can then study.
Take the protein alpha-synuclein, which builds up and forms clumps in the brains of people with Parkinson’s disease. Scientists can’t just poke around carrying out experiments in a person’s brain, so instead a group at Whitehead Institute for Biomedical Research turned to yeast designed to over-produce that same protein.
It turns out yeast don’t like excess alpha-synuclein clumps any more than humans do. The cells turned sickly when they cranked out the extra protein.
The scientists could then expose these cells to different compounds to find ones that restored the yeast to health. They found one such compound, which they then tested in tiny roundworms and then in rats, which also produced excess alpha-synuclein. In each case, the compound helped the animals clear out the excess protein. They reported this findings in the journal Science.
But what about humans? The group took skin samples from people with Parkinson’s disease and created reprogrammed stem cells out of those samples. When those stem cells matured into brain cells, they showed symptoms of Parkinson’s disease. The compound they’d found in the lowly yeast improved symptoms in those cells.
The scientists stress that their compound is far from being a drug. They have a lot of work to do showing that it–or a compound with similar functionality–might be safe to test in people. But the scientists might not even be this close if they hadn’t started the search in the lowly yeast. A story in the Boston Globe quoted Vikram Khurana, who was one of the study’s authors:
“It was a real surprise. We would have had no clue about this target if we hadn’t had yeast genetics to show us the way.”
Amy Adams
The Stem Cellar 
Watch This Mother Fight To Get Her Kids A Medicine She Thinks Could Save Their Lives
McNary is among a group of patient advocates who are publicly lobbying the Food and Drug Administration to approve the drug based on that 12-patient clinical trial after the medicine’s maker, Sarepta Therapeutics SRPT -6.64%, applies early next year. She’s had meetings with top officials at the FDA. The argument is this: the FDA could grant what’s known as accelerated approval, a type of approval that has a built-in ability to withdraw a drug if bigger trials don’t confirm it is effective, based on the ability of eteplirsen to boost levels of dystrophin, the protein that is missing in the muscles of kids with Duchenne.
This is a risky strategy. Another, apparently similar, drug from competitor Prosensa did not show any efficacy in a much larger study. Results from small trials often aren’t replicated when larger groups of patients are studied. In the past, patients have demanded access to drugs for cancer, for Parkinson’s and for other diseases that ended up not working when larger trials were done. But it is possible, especially given the chorus of mothers like McNary, that the FDA will allow eteplirsen onto the market early. Either way, she is proof of the way that patients are starting to change the way that medicines are developed with the power of their voices. And the video below is only four minutes long.
http://www.forbes.com/sites/matthewherper/2013/10/23/watch-this-mother-fight-to-get-her-kids-a-medicine-she-thinks-could-save-their-lives/
Yes it might be a risky strategy but most of the clinical trials that had been conducted are considered to be risky for some reasons.