When you or someone you care about has a disease, the first thought is of a cure. That’s human nature.
But the image of a biological researcher having an “Aha” breakthrough in a laboratory that creates a cure is just part of a plot for a movie you might enjoy with some popcorn.
In reality, cures are the cumulative result of hundreds, even thousands, of small steps forward carried out by hundreds or thousands of researchers over a long period of time.
In a pilot study, researchers analyzed recent approvals of two new drugs, one for melanoma (skin cancer) and one for cystic fibrosis, that are “sufficiently novel and important to be reasonably characterized as ‘cures.’” These drugs were used to test an analytical model that calculates relationships between scientific discoveries and cures. It puts numbers on the time and effort.
Take the development of the drug approved for melanoma, ipilimumab, which may have already saved the life of someone you know. To arrive at a drug that sends the disease into remission, 7,067 different scientists with 5,666 institutional and departmental affiliations were involved over the span of 104 years of research.
When crunched by the researchers’ metrics model, the numbers for the cystic fibrosis drug ivacaftor are similar: 2,857 different scientists from 2,516 different places, with discoveries spanning 59 years of research.
One purpose of the model researchers developed is to increase your understanding of how discoveries actually lead to cures, which they say is “limited.”
That’s not your fault. They add that research conducted to clarify the “principles of biological processes may appear to non-scientists as esoteric and irrelevant to public expectations.”
These well-meaning but opaque explanations don’t feed public support for biomedical research, which has dwindled over the last decade, mostly because funding to the National Institutes of Health (NIH) has been cut dramatically.
Lead author R. Sanders Williams, MD, says researchers wanted to develop an unbiased statistical approach to try and understand what’s necessary to find a cure, rather than resort to “anecdotal” stories.
To do that, they created a set of scientific assumptions, challenged those, and lay the result “out there” for you and the rest of the public to see. That was accomplished with computer algorithms.
“Nobody is gaming this, no one is using judgment or any biases,” says Williams, president of Gladstone Institutes. “The point is to better know how to get the cures we don’t have by learning from the past to inform the future.”
“Cures don't come from some genius having a single idea,” he says. “It simply doesn't happen that way. It requires the interweaving of multiple threads of science, from many diverse places and often, it happens in an unexpected way.”
The commentary, he adds, is pointed toward both the public and the government because the NIH needs far more funding to speed up progress, and needs to be separated from the political football that has become national healthcare.
Faster cures require a “broad base” of science, as the computer analysis found, “and only a healthy NIH can provide that,” Sanders says.
The last point you should take away from the historical analysis of cures: not all scientists are created equal. The study found there are influential contributors along the timeline to a cure who contribute the most.
These “elite performers,” for example, comprised 15 scientists from 7 institutions who published 433 articles spanning 46 years to arrive at the drug that cures melanoma in some cases.
For the drug that corrects a loss of function in cystic fibrosis patients, the metrics say the main contributors were 33 scientists and 7 institutions, which resulted in 355 articles spanning 47 years.
“As shown by our analysis, new treatments depend upon a broad base of scientific knowledge plus special contributions from a few exceptional scientists,” Williams says.
“The ultimate goal of this work is to find ways to accelerate progress towards future cures for cruel diseases that remain unsolved: Alzheimer's disease, Parkinson's disease, heart failure, deadly viruses, diabetes, many cancers, and others.”
February 03, 2016
Christopher Nystuen, MD, MBA