Blockbuster Anti-Cancer Technology
The science story with the biggest buzz recently was probably the successful treatment of three leukemia patients by scientists from the University of Pennsylvania.
While the leukemia research is important, I don’t think that many understand where it fits in the bigger picture of cancer therapy development. I believe, in fact, that there are a half-dozen more- promising therapies.
The anti-cancer treatment that caught the media’s attention is actually a gene therapy from the University of Pennsylvania. Scientists genetically modified three patients’ own cancer-fighting T-cells and gave them back.
For some time, we have been able to modify T-cells to specifically attack cancers. The breakthrough is that this new technology creates T-cells that are very successful at replicating.
While this is exciting, it is not an optimal therapy, for one simple reason: It is a procedure, not a drug.
Because the use of gene therapies using patients’ individual genes is extremely expensive, it is not the best “mass market” solution for treating cancers. To keep costs and delays down, we need off-the-shelf products.
Regenerative medicine is the exception to that rule, because it is the only way to turn the cellular clock of aging back to zero.
Fortunately, there are therapies in development that will be as effective as the U of Penn technology at killing cancers. They will not, however, require genetic engineering for each patient.
Off-the-Shelf Genetic Engineering
One company is developing a technology for attacking cancers that will employ off-the-shelf genetically engineered stem cells.
I spoke with them recently about this lead therapy, which is based on the use of genetically engineered stem cells. Originally, the company will use the government-approved embryonic cell line. Eventually, it is expected that induced pluripotent stem cell lines will be used, due to objections to embryonic cells.
The cells that they will use, by the way, are not capable of developing into embryos. In fact, they are potentiated endothelial cells that have been further modified through genetic engineering. The first drug candidate is a truly brilliant and, to me, surprising application of regenerative medicine.
Endothelial stem cells are the cells that repair the cardiovascular system. They are rare in adults, but are naturally attracted to the new blood supplies created by cancers via angiogenesis. These cells, if transfused into a patient with cancer, will hone in on and stick to cancer cells.
In time, they would be cleared from the body by the immune system because they are transplants. In reality, however, they won’t live long enough to be removed.
Using Stem Cells as Remote-Controlled Cancer Killers
These endothelial cells will have genes turned on using genetic engineering that create very specific enzymes. Once they have attached to the cancer cells, a prodrug will be administered to the body.
A prodrug is not an active drug itself. It is a substance that can be converted into one in the presence of certain enzymes. Since those enzymes exist only in the new endothelial cells that have congregated around the cancers, they convert the prodrug into a lethal cancer-killing drug at the site of the cancer. Once the payload is delivered, another genetically engineered switch is activated via a harmless drug that causes the endothelial cells to undergo apoptosis, cell suicide. They are then removed from the system.
I think the most interesting thing about this therapy is that it is systemic. That is, because the genetically engineered cells are attracted to cancers wherever they are in the body, even metastasized cancers can be targeted.
They are currently doing preclinical studies with transgenic mice. These are mice with human cancers.
This same technology can also be employed for use in diagnostics. Because markers can be attached to these cancer-seeking endothelial cells, it will be possible to use them to locate cancers before they become dangerous. Because different cancers have different markers, it becomes theoretically possible to identify the exact nature of a cancer and tailor therapies for maximum effectiveness.
It bears repeating that the cost of these diagnostics and therapies will be far less expensive and time-consuming than the U of Penn technology.
In times such as these, it’s easy to get discouraged. Don’t be. In five or ten years, maybe less, people will look back and marvel at the people who had the foresight to buy transformational companies like this one when the entire market seemed ready to jump off a building.
Yours for transformational profits,