Maybe it's because we're drawn to human interest stories during the holidays, but many tend to stand out this time of year and stay with us.
That's certainly the way I feel about the story of little Tatiana, the newborn daughter of Elise and Bruce Saiaana of Seattle. Upon her birth four months ago, Tatiana was in immediate critical condition from inhaling fluids, which caused her lungs to collapse. She was given little chance of surviving the ambulance ride to the nearby hospital, which provided the only treatment that might save her — placing her on an extracorporeal membrane oxygenation, or ECMO, machine. Doctors told her parents that because the infant's lungs were not fully developed, she might not be able to survive if removed from the machine. They were told to prepare themselves for losing their baby.
"She was still fighting, so we had to fight for her, too," her mom told a local TV reporter.
Her doctors were equally committed to doing all they could for the child, but there were no traditional treatments to which they could turn. In a last-ditch effort to save her, they were able to obtain a waiver to use a drug known as perflubron. Perflubron was tested briefly in the 1990s, but testing was abandoned because the drug was not so effective as researchers had hoped it would be. Though it was never submitted for Food and Drug Administration approval, it is available in Canada and Europe and has become well-known for its properties as an oxygen-rich liquid that fills and expands the lungs and then quickly evaporates.
Doctors started treatment with perflubron and soon began to see improvements. Tatiana started breathing on her own. This now healthy, beautiful baby girl was soon on her way home for the holidays as the best Christmas gift a family could ever ask for.
"If it wasn't for that drug, then there would be nothing left to do for her," said her mom, adding that she and her husband hope that Tatiana's story inspires efforts to get this drug approved in the U.S. for children like their daughter.
It is a story not unlike the one I shared last week about Deanna Tedone-Gage and her father's search for a treatment for ALS. It is further testament to the need for medical science and pharmacology to begin to rethink the current approach to advancing the treatment and cure of illness.
During the past two decades, the pharmaceutical industry in particular has focused almost exclusively on an automated, high-tech approach to discovering drugs derived from synthetic compounds and has shunned traditional trial-and-error chemistry and natural compounds.
Modern pharmaceuticals are supposed to represent the practical payoff of basic high-tech research, yet for every billion dollars invested in research and development since 1950, the number of new drugs approved has continued to fall by one-half every nine years. As to the successes, an estimated 70 percent of new drugs approved by the FDA in 2013 were specialty drugs, defined as being used by no more than 1 percent of the population. New treatments for broader diseases, such as diabetes, continue to elude modern approaches.
The cost of discovering a promising new compound is roughly 100 times more than it was in 1950, and new compounds take at least three times as long to be approved. No wonder promising drugs such as perflubron are abandoned before they are fully tested and approved. Given the increasing difficulty of winning approval and the spiraling costs involved in the process, it's not surprising that some companies have responded by abandoning research.
Meanwhile, fundraising has rarely been more difficult.
Still, donations continue flowing to the ALS Association from its "Ice Bucket Challenge." The 2014 haul includes a surge of year-end checks totaling $32 million. This contribution spike effectively tripled the national association's research budget. Worldwide, $220 million was raised, according to an NBC News report.
This fundraising phenomenon has left medical charities that raise funds for diseases affecting far higher numbers of people scrambling to find ways to duplicate the ALS Association's massive success. But this may not even be possible for the ALS Association to repeat. No one knows exactly why it worked. It seems to have had a little serendipity going — a little like what so characterized many important new drug breakthroughs following old-fashioned trial-and-error methods, which continue to produce a success rate that trumps those of new-age approaches.
Write to Chuck Norris ([email protected]) with your questions about health and fitness. Follow Chuck Norris through his official social media sites, on Twitter @chucknorris and Facebook's "Official Chuck Norris Page." He blogs at http://chucknorrisnews.blogspot.com. To find out more about Chuck Norris and read features by other Creators Syndicate writers and cartoonists, visit the Creators Syndicate Web page at www.creators.com.