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| Gene therapy isn't some far-off conceptual technology. It has already been saving people quite literally from their death beds for years now. Emily Whitehead, pictured above, was dying from leukemia when Penn State University doctors saved her life with gene therapy. Doctors re-engineered her T-cells to seek and destroy the cancer that was killing her. |
It is hard to imagine how this could happen.
Let’s assume that all the technical obstacles that now plague gene therapy [1] are resolved. In other words, that we learn how to deliver genes efficiently to the appropriate tissues without any side effects. Gene therapy would still be appropriate for only a tiny fraction of human diseases.
This is because few diseases have a simple cause-effect relationship with a definable set of genes and their variants. Take heart disease, for example. It is the number-one cause of death worldwide. Family history is a strong risk factor for heart disease. But the risk added by any one gene that is linked to heart disease tends to be miniscule, despite the hype that attends their discovery. This headline, from a credible news source is a good example [Strong DNA link found to heart attacks].
Sounds like a big deal, right?. But read down the article and you find that people who carry two copies of this gene have a risk of heart attack that increases from 10% to 14%. Not so impressive.
To people with no knowledge of what gene therapy is or what current clinical trials underway are already achieving, this might seem like very disappointing news for those who hope for a much-needed and profound shift of the current pharmaceutical paradigm.
In reality, however, this is not how progress in gene therapy works. Not at all.
Real breakthroughs in gene therapy already saving human lives, do not revolve around finding the genes responsible for making one susceptible to a certain condition like cancer or heart failure, they revolve around changing genetic factors that make combating such conditions easier for the body.
For example, gene therapy already used (University of Pennsylvania) to successfully and fully cure leukemia involves re-engineering a patient's t-cells so that they can recognize and eliminate the deadly cancer sure to otherwise kill them. Dozens of patients have already undergone such therapy with the majority of them going into full and permanent remission, something pharmaceuticals had failed to do, thus qualifying the patients to partake in the clinical trial in the first place.
Likewise, contrary to the Forbes-Quora article, patients suffering from heart failure are not treated by identifying and eliminating whatever genetic characteristics may have made them susceptible to heart failure in the first place, but instead work by delivering new genetic instructions to heart muscle cells (American Heart Association) on how to properly repair themselves stronger and better than ever before.
Whether it is ignorance or an oblique attempt to tamp down enthusiasm in a transformative emerging technology that jeopardizes an influential and well-entrenched industry, one thing is for certain — gene therapy will push forward curing diseases and regenerating defective or aging biology while also taking the profits out of healthcare so profoundly, it will be unlike any advance in healthcare in human history.
It is essential that people research and inform themselves regarding this emerging technology, because for whatever reason, Forbes has categorically failed to inform the public. First, people must work to move essential and ultimately more effective and cheaper cures into the hands of those who need them, and second, by being informed we inoculate ourselves from those who seek to use public ignorance to price-gouge when literal cures for heart disease and cancer are finally widely available.
Follow ProgressTH.org on Facebook here or on Twitter here.
In reality, however, this is not how progress in gene therapy works. Not at all.
Real breakthroughs in gene therapy already saving human lives, do not revolve around finding the genes responsible for making one susceptible to a certain condition like cancer or heart failure, they revolve around changing genetic factors that make combating such conditions easier for the body.
For example, gene therapy already used (University of Pennsylvania) to successfully and fully cure leukemia involves re-engineering a patient's t-cells so that they can recognize and eliminate the deadly cancer sure to otherwise kill them. Dozens of patients have already undergone such therapy with the majority of them going into full and permanent remission, something pharmaceuticals had failed to do, thus qualifying the patients to partake in the clinical trial in the first place.
Likewise, contrary to the Forbes-Quora article, patients suffering from heart failure are not treated by identifying and eliminating whatever genetic characteristics may have made them susceptible to heart failure in the first place, but instead work by delivering new genetic instructions to heart muscle cells (American Heart Association) on how to properly repair themselves stronger and better than ever before.
Whether it is ignorance or an oblique attempt to tamp down enthusiasm in a transformative emerging technology that jeopardizes an influential and well-entrenched industry, one thing is for certain — gene therapy will push forward curing diseases and regenerating defective or aging biology while also taking the profits out of healthcare so profoundly, it will be unlike any advance in healthcare in human history.
It is essential that people research and inform themselves regarding this emerging technology, because for whatever reason, Forbes has categorically failed to inform the public. First, people must work to move essential and ultimately more effective and cheaper cures into the hands of those who need them, and second, by being informed we inoculate ourselves from those who seek to use public ignorance to price-gouge when literal cures for heart disease and cancer are finally widely available.
Follow ProgressTH.org on Facebook here or on Twitter here.
