2022.03.06 Decentralized Science
Reminded once again of the unfortunate penetrance of profit-seeking behavior in healthcare that may unknowingly impede the development of revolutionary therapies, simply due to their limited consumer base.
Having dedicated the past three-ish years to studying resistance mechanisms in leukemia, the story of Gleevec (imatinib) pushes me to consider the possibility of decentralized research financing.
If corporate approval is not required to advance scientific pursuit in areas overlooked by economies of scale, perhaps patients with rarer conditions can be cured without needing their unique afflictions to affect a threshold proportion of the general populace.
In my opinion, yield-bearing assets are crucial financial tools that could transition this idea to reality, and one of the reasons why I am an avid proponent of decentralized finance.
“For Novartis, it was the exquisite specificity of CGP57148 (Gleevec) that was precisely its fatal undoing. Developing CGP57148 into a clinical drug for human use would involve further testing—animal studies and clinical trials that would cost $100 to $200 million. CML (chronic myeloid leukemia) afflicts a few thousand patients every year in America. The prospect of spending millions on a molecule to benefit thousands gave Novartis cold feet…
Novartis had a plethora of predictable excuses: “The drug… would never work, would be too toxic, would never make any money.”… In early 1998, Novartis finally relented. It would synthesize and release a few grams of CGP57148, just about enough to run a trial on about a hundred patients…To Novartis, CGP57148, the product of its most ambitious drug-discovery program to date, was already a failure…
By June 1999, with many of the original patients still in deep remissions, Gleevec was evidently a success. This success continues; Gleevec has become the standard of care for patients with CML. Oncologists now use the phrases “pre-Gleevec era” and “post-Gleevec era” when discussing this once-fatal disease.
Hagop Kantarjian, the leukemia physician at the MD Anderson Cancer Center in Texas, recently summarized the impact of the drug on CML: “Before the year 2000, when we saw patients with chronic myeloid leukemia, we told them that they had a very bad disease, that their course was fatal, their prognosis was poor with a median survival of maybe three to six years, frontline therapy was allogeneic transplant…and there was no second-line treatment… Today when I see a patient with CML, I tell them that the disease is an indolent leukemia with an excellent prognosis, that they will usually live their functional life span provided they take an oral medicine, Gleevec, for the rest of their lives.”
The Emperor of All Maladies: A Biography of Cancer (p.436-439)
Siddhartha Mukherjee