On the cusp of 2013, I’ve invited 11 of the greater Houston area’s top minds to write about something they believe, but cannot prove. A new entry in the 11 for ’13 series will be published each morning during the holidays.
Today’s mini essay comes from Dr. Todd Rosengart, chair of the Michael E. DeBakey Department of Surgery at Baylor College of Medicine at Baylor College of Medicine…
Although I can’t prove it, I am fairly certain that the next five years will witness cures for genetic diseases, the regeneration of healthy new organs from diseased ones, and even exciting new (gene therapy) treatments for cancer. I know, you’ve heard it before, but I really believe this time is different.
The field of gene therapy crossed a monumental threshold just this past month – after two decades of false starts – in the first commercial approval of a gene therapy “drug”. This approval by the European Union of a single dose treatment for a rare but lethal hereditary disorder portends a new era of like cures by gene transfer.
In our own laboratory and others around the world, we appear astonishingly to have been able to convert tissue scarred by a heart attack back into functioning heart muscle, by “simply” injecting three genes into the scar tissue. If fully validated, such cellular reprogramming could offer an important alternative to the transplants and devices now needed for advanced disease of the heart and other organs.
Finally, I believe real advances against cancer can be seen in rapidly growing understandings of the genetic basis of cancer, coupled with accelerating capabilities to decode individuals’ genetic maps (at facilities such as the Baylor College of Medicine Human Genome Sequencing Center here in Houston). Conceivably, new molecular therapies channeling this knowledge will soon bypass the naïveté of once toxic cancer treatments.
So, why this optimism after decades of languishing medical science? The answer is that medical innovation has not been languishing; it has been growing exponentially – doubling again and again until we are now benefiting from a critical mass of understanding. As “Moore’s Law” predicted the repeated doubling of computing power, even producing the voice recognition products unthinkable only a few years ago, a “Moore’s Law” of biotechnology will allow bona fide cures of once challenging diseases.
The hyperbolic trajectory of (cardiac) stem cell innovation is but one example of this pace. First mentioned as early as 1963 (by scientists at the Texas Medical Center), the first clinical trial involving cardiac stem cell treatment did not occur until almost forty years later. Less than a decade after that, the creation of stem cells was reported in 2006 (winning Yamanaka the Noble Prize only six years later), and only five years later came the first report of mature cell reprogramming (the biologic “alchemy” that is the basis of our heart muscle regeneration).
I think you get it – we’re learning and inventing biotechnology more and more quickly, each discovery building on the next. So, although I can’t prove it, I think we will see cures for many killer diseases sooner than we ever could have imagined.