A recent news story echoes many others over the past few months. In this one, researchers in New York genetically modified mesenchymal stem cells so that they could be grown endlessly in bioreactors. This story sounds like the plot line of the Will Smith movie “Legend” where a famous cancer researcher uses gene therapy to cure cancer-all patients are cured. However, after the cure, a funny thing happens, they start turning into photophobic, genetically modified humans that eat the rest of us. Smith spends the better part of the movie running around trying to find the few normal humans that are left, trying to find a cure, and hunting down the photophobic creatures. The Canadian story makes the point that these new cells have been created because it’s simply too inconvenient to take fresh stem cells from a patient every time you need to cure something. The story also explains that the University is working with the inventor to patent the discovery.
These genetically altered cells brings up the wisdom and utility of an entire new area of stem cell research, what I call “Frankencells”, also known as Induced Pluripotent Stem Cells or IPS cells. This shift in stem cell research began a few years ago. At the time, the animal and early pre-clinical and clinical research was showing that adult stem cells could do some pretty miraculous things. The research still shows that promise. However, all of a sudden somebody discovered that it was possible to insert genes into normal skin cells and turn them into embryonic like stem cells. While this was very innovative and a truly interesting scientific discovery, the obvious question was why? Despite the obvious questions, this discovery was hailed by the media with the same fervor as the invention of Penicillin. The researchers were honest, what they had done was incredibly dangerous until proven otherwise, meaning creating normal somatic cells that can artificially grow forever was likely a really bad idea. The unintended consequences scale was through the roof, especially when it came to creating cancer. As more researchers came up with the own Frankencells, it seemed like the media had been captured with this new way of avoiding the controversial issue of embryonic stem cells. However, the media seem to miss that adult stem cell researchers had already published more than 20,000 papers showing that none of this was needed, simply using the patient’s own cells or transplanting them from one patient to another could accomplish all the same things with likely a much better safety profile.
So why the focus on Frankencells when they seem to be about as useful to us right now as udders on a bull? This past year has seen major attacks on patents for stem cells and genes, with the USPTO and the courts calling them body parts that aren’t novel. Novelty is the currency of patents and the biopharma industry, it’s needed for a valid patent to issue. By creating cells that don’t exist in nature, these researchers are guaranteeing novelty and a patent. This provides business barriers to entry for pharma and in this case, the ability to grow cells in bioreactors allows them to manufactured like an antibiotic or a new statin drug. While the patient’s own stem cells are much safer by default and will likely will work just as well based on the published research, they likely can’t be patented and certainly can never be mass distributed. While these Frankencell discoveries are important science, they seem to be stealing the show from the more important clinical discoveries in autologous adult stem cells, ones that are much less likely to produce complications are ready for clinical translation now.
In summary, basic science is important and one day what I’m now calling Frakencells will become standard of care and likely produce great clinical results surpassing what adult autologous or allogeneic cells can accomplish. However, the pursuit of these cells now as therapy seems driven solely by patents, business models, and profit, not by medical need. If we are ever going to see true healthcare reform, we will need to have a medical therapy discovery system that puts clinical need first and profit second. This will require an extensive overhaul of our health system so that treatments get to patients faster, with or without a patent and a valid business model.