Survival as a Service

“Medicine rests upon four pillars – philosophy, astronomy, alchemy, and ethics.” – Paracelsus

Nathan Vardi’s gripping book For Blood and Money: Billionaires, Biotech, and the Quest for a Blockbuster Drug tells the story of the discovery and commercialization of Ibrutinib, the breakthrough blood cancer drug. Originally synthesized by Celera Genomics as a tool compound for the study of an important enzyme in cancer cell survival and proliferation pathways (Bruton’s tyrosine kinase (BTK)), Ibrutinib was scooped up by the tiny firm Pharmacyclics for a relative pittance. Less than a decade later, pharmaceutical giant AbbVie outbid Johnson & Johnson to acquire Pharmacyclics for the incredible sum of $21 billion.

The driving force behind Ibrutinib’s meteoric rise was entrepreneur Robert Duggan, whose son had died of brain cancer at the age of 26. Despite having no prior experience in the field of oncology, a deeply motivated Duggan accumulated stock in Pharmacyclics with a sizeable personal investment, installed himself as CEO and Chair, and managed the company with what former employees would later characterize as ruthless efficiency.

Many of the early patients receiving Ibrutinib suffered from chronic blood cancers and had already received several rounds of toxic chemotherapy and other risky treatments. Some were near death and out of viable options. When such patients showed stunning results in just a matter of weeks with barely any negative side effects—some even walking out of the hospital feeling normal again—Duggan knew he had a winner on his hands. He would go on to move mountains to get the drug to market as swiftly as possible before selling the company. Duggan personally netted $3.5 billion in proceeds from the AbbVie transaction.

Multiple missions accomplished | Forbes

Although no form of blood cancer cracks the top ten in global incidence rates, hematological oncology receives outsized attention from scientists and investors alike. The nature of blood cancer is such that the field effectively represents the front line of combat in the war on cancer more broadly. Since blood cancer cells circulate in the bloodstream or accumulate in accessible tissues like lymph nodes, directly targeting them with medication is a far simpler task than reaching cells buried deep within solid tumors. Further, patients can be readily sampled for monitoring with minimal invasiveness—a few milliliters of blood drawn from a needle can lead to scores of sophisticated, rapid analyses. This also facilitates personalization of care, as the genetic profile of an individual’s cancer can be monitored through the disease’s progression. Perhaps most importantly, discoveries in blood cancer research often apply to other forms of cancer, making the size of the potential prize far greater than the narrow addressable market of the proving ground.

In the 15 years since Ibrutinib first exploded onto the scene, the pace of development in blood cancer research has reached dizzying heights. Several superior BTK inhibitors have been brought to market, including Acalabrutinib—the second drug profiled in Vardi’s book. New non-BTK attack vectors against blood cancers have also been opened, with many demonstrating equal or superior patient outcomes. Each time a new mechanism of action is proven, a swarm of researchers swoops in seeking to beat the drug that did the initial proving. Researchers have found that taking combinations of multiple drugs with orthogonal action modes can further improve some patient outcomes, vastly expanding the treatment menu available to the newly diagnosed.

American Society of Hematology conference | Pfizer

Given these unequivocally exciting developments, one might start to believe that a cure for many blood cancers is just around the corner, and perhaps for some solid tumor cancers shortly thereafter. After all, if these new wonder drugs work so well in series—knocking down cancer counts for years at a time before the next one is needed—surely hammering the cancer with four or five of them all at once might totally extinguish the disease? Unfortunately, the question of curing cancer can be a rather sticky one, replete with economic, scientific, regulatory, and ethical barriers. Let’s head to the front lines and explore each in order.

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