Editor ’ s Corner
Musing on Medical Marvels
I
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T WAS CIRCA 1999 . I can picture the patient vividly : A mustachioed , unfailingly polite – but exhausted – middle-aged man holding a white tissue to his bleeding nose . A Ugandan immigrant , he was dependent on platelet and red blood cell transfusions , and his myeloma progressed following conventional chemotherapy and autologous hematopoietic cell transplantation , as well as a boatload of alkylating agents and dexamethasone . His disease had left him largely bedbound .
Hallway conversations among the myeloma cognoscenti were alive that year with news that the great innovator Bart Barlogie , MD , had evidence of responses in patients with late-stage myeloma to , of all things , the notorious drug thalidomide . Younger in those days and more risk-tolerant , we found a wholesaler in the United Kingdom , sought permission from the regulators for emergency use , and gave thalidomide a try .
After receiving treatment , the patient picked himself up out of bed and walked . Some years later , he brought me a small stone carving from his native country depicting a person helping another off the ground – I keep it in my home office even today .
At the 2017 ASCO Annual Meeting , more medical miracles were on display as the tsunami of immuno-oncology and precision therapeutics engulfed the proceedings . There was news about designer drugs , activated T cells savaging solid tumors , and patients with refractory lymphoma and myeloma achieving complete remissions by treading the same cellular therapy paths carved by patients with childhood leukemia and chronic lymphocytic leukemia ( CLL ).
I sat at the back of the hall as Siddhartha Mukherjee , MD , DPhil , delivered his excellent guest lecture about the “ deep personalization ” of medicine during the meeting ’ s opening session , contemplating chimeric antigen receptor T cells and other medical marvels I have been honored to witness as a myeloma doctor , from thalidomide to lenalidomide , bortezomib to carfilzomib , and daratumumab to venetoclax . Outside my own expertise , I have seen ibrutinib change the treatment of CLL , as well as the about-face of chronic myeloid leukemia and hairy cell leukemia as a result of new therapies .
Listening to the address , I thought about a family friend and frequent tobacco user who developed an advanced squamous cell carcinoma of the lung and how genomics and checkpoint inhibitors have made heavy inroads in a disease I had always associated with a fairly rapid demise . I applauded heartily at the proven logic that a mutation affecting a novel TRK fusion protein responds to a matched small-molecule inhibitor 75 percent of the time . Most of all , I thought about this great energy vortex of scientists and clinicians , charitable foundations , government agencies , entrepreneurs , angel investors , venture capitalists , biotech and Big Pharma , diagnostic companies , contract research organizations , patients participating in trials , and – yes – the regulators who have worked together in networks of mutual urgency and shared visions to accelerate transformative advances in just 20 years that allowed a hematologist to witness multiple patients respond to a repurposed drug .
Reality bit deeply into my red apple of optimism later that day . It began insidiously with a tale , possibly apocryphal , from an employee of a medium-sized pharmaceutical company about a niece who earned an A + in her middle school biology class on an essay about why drug companies were bad .
Later , I logged into Twitter to see what colleagues were saying about this great generation of oncology ; one well-meaning group was having a tweetathon
Keith Stewart , MBChB , MBA , is Carlson and Nelson Endowed Director of the Center for Individualized Medicine and Vasek and Anna Maria Polak Professor of Cancer Research at Mayo Clinic in Scottsdale , Arizona .
saying “ they ” ( drug companies ) were conspiring to bankrupt and hoodwink patients . CAPS LOCK was employed to express with vehemence how terrible it was that a drug that extended remission by an astonishing 10 months cost more than the current standard . Compounding my dismay was the tone of colleagues on the same subject during a question-and-answer period at another session during ASCO ’ s annual meeting .
It all reminded me of Henry Kissinger ’ s adage : “ Academic politics are so vicious precisely because the stakes are so small .” But in hematology / oncology , they aren ’ t . The stakes are high and every member of the team , including our colleagues in industry , deserves recognition for the good that has been wrought .
It would be a hardened disciple of commercial purity who failed to acknowledge publicly that advancing inventions , supporting serendipitous drug-repurposing discoveries , conducting clinical trials that cost millions of dollars to run , and manufacturing and distributing life-saving therapies are almost certain not to happen without the incentive of return on investment . I am not defending high drug prices or markups . I understand the vexation of colleagues , I sense the urgency patients feel , I worry about the political winds of research funding , and I don ’ t like hyped claims , “ manageable toxicity ,” or dodgy statistics any more than the next person . But I do wish for fair and balanced coverage .
It seems to me that every member of the medical-marvels team deserves respect even while the uncertainty and missteps of transformational change are still fogging the conversation .
Keith Stewart , MBChB , MBA
6 ASH Clinical News August 2017