Only rarely does an experienced editor get a spine tingle from a new paper. For the first time ever, today, I predict that a Nobel Prize for medicine will be awarded to J. Martin Brown, DPhil, Oxford, a professor at Stanford University School of Medicine.
Professor Brown and his colleagues have discovered and reported a fundamentally new approach to the treatment of solid tumors, beginning with the devastating glioblastoma multiforme.
Here is how it goes: Tumors need blood in order to grow. Powerful radiation can kill many cancer cells. It also kills the cancer’s blood vessels. How then do any surviving cancer cells regrow after radiation if they have no blood supply? Where do the nutrient blood vessels come from?
Professor Brown and colleagues hypothesized that circulating bone marrow derived cells recreated endothelium, and thus vasculogenesis (not angiogenesis), thereby providing the needed blood for the cancer to recur.
A drug called plerixafor (AMD3100 or Mozobil) was designated an orphan drug by FDA in 2003 for stem cell transplants and further in 2009 for use in bone marrow transplantation.
The investigators took laboratory mice bearing glioblastoma multiforme xenograft tumors and administered therapeutic irradiation.
After irradiation, they treated the mice with plerixafor to test whether, by blocking the bone marrow derived cells from becoming endothelial cells, they could prevent them from providing blood nourishment to the irradiated cancer.
Recurrence of the mouse glioblastomas was completely inhibited throughout the study period, and absent by post-mortem histology.
It’s a long way from mice to humans. But this drug, plerixafor, is already approved for human use. It is said to be benign.
Glioblastoma kills several thousand Americans each year, all after “standard of care” has run out of plausible options. Not to mention the countless others of the half million Americans who die from cancer each year, many beginning as solid malignant tumors.
Plerixafor should now be tried on humans, after fully informed consent, beginning as Clinical Trials with an N of 1.
And the results, whatever they are, should be shared promptly with the broad medical and cancer communities.
Don’t worry about “off-label use”; much of oncology therapy is “off label”. Worry most about your patients.
George Lundberg is a MedPage Today Editor-at-Large and former editor of the Journal of the American Medical Association.