Importantly, Geron did not get out of the stem cell business as most analysts have written. Instead, they decided to leverage their extensive years of research and knowledge about stem cells to develop a drug that would target the cancer stem cells that fuel tumorigenesis. Personally, I spent several years in a big pharmaceutical company screening drugs that target cancer stem cells. Every company developing cancer drugs knows that the Holy Grail is discovering a drug that can target the cancer stem cells. This is what Geron has developed.
Cancer Stem Cells and Imetelstat
Typically, cancer drug efficacy during development is measured by how much the drug can shrink the tumor or extend survival of the patient. However, after several months or years many cancers relapse. This is because the drug fails to target the cancer stem cells and only affects the bulk tumor. The idea of cancer stem cells has been around a long time and is well characterized in hematopoietic malignancies and some solid tumors like testicular cancer. For example, cancer stem cells in testicular tumors are extremely sensitive to the chemotherapeutic Cisplatin, making testicular cancer one of the most curable cancers with a 99% treatment success rate. It is well known that immortal stem cells rely on telomerase to stay alive and that it would be a perfect target for drug development. However, drug compounds developed failed to specifically target telomerase. To date, Geron's patented oligo design is the only drug that can permeate cells and specifically target telomerase effectively.
There is a lot of work going on trying to validate the CSC theory in the clinic and maybe Imetelstat will contribute to the proof of CSCs and how to defeat them. This still is not proven, but it brings into focus another aspect of imetelstat potential importance and value, even beyond what it may do for patients with MF. It is part of the cancer "Manhattan Project" into the final discovery of what makes cancer spread and recur.
If Imetelstat can stop cancer from metastasizing in the liver (which is what eventually kills people) we indeed DO have the holy grail and we shareholders will be in the Oprah/Bill Gates/Warren Buffet tax bracket.