… Zebra petition filed with FDA Commissioner After three intense weeks, the MPN community delivered a petition with 526 signatures to FDA Commissioner Dr. Margaret Hamburg and a cover letter requesting a meeting to explore broad areas of agreement and means to strengthen safety and patient welfare in the clinical trial process through patient advocacy. Several eminent hematologists including Dr. Richard Silver, Dr. Srdan Verstovsek and Dr. Claire Harrison gave us their blessings along with the leadership of the MPN Research Foundation leadership. Now, if we can get Frank Underwood on our side, we’ve got it made.
Order your engineered T Cells from Amazon? The explosive growth of interest in CAR-T cancer drugs, genetically modified T cells a white blood cell that plays a major role in our immune system, got a further boost when Amazon’s founder Jeff Bezos plunked $20 million down to join other investors in Seattle biotech start-up Juno Therapeutics. A big fish in this pond is Novartis that has both bought up research firms working the CAR-T territory and is building a facility at the University of Pennsylvania to pursue research.
T cells are taken from patients and altered with genes designed to kill cancer cells (disabled H.I.V. is used since it’s knows the way into cells). These modified T celsl — now known as Chimeric Antigen Receptor cell — are then reintroduced into the patient and hopefully hone in a leukemic cells. Some early results are startling, producing rapid and complete remissions. And, since they’re living cells — unique to each patient — a single infusion can theoretically last a lifetime. The famous return policy of Amazon, however, doesn’t apply.
Steensma : What if Edison were a cancer researcher?
OK, it may not be one those game changing scientific insights but for those of us waiting for salvation at the dripping end of the drug pipeline a hard look at what’s gumming up the works may be equally important. A paper by David Steensma (Dana-Farber, Harvard Medical School) in the Journal of Clinical Oncology caught our eye last week. The newest member of MPNclinic, Steensma is also one hell of a writer. Along with co-author, MD Anderson’s Dr. Hagop Kantarjian, — remember him from the crusade to lower cancer drug prices? — Steensma makes a compelling case for streamlining the process that clogs development of new cancer drugs.
A substantial part of the increased complexity and cost is laid at the door of CRO’s, Contract Research Organizations, profit-making data management firms brought into existence by the load of regulatory auditing and documentation requirements surrounding current clionical trials. The cost part is dramatic: “As a result of the increasing regulatory requirements and the need for CRO involvement, the cost of research per patient on a phase IIIA oncology trial has ballooned from an average of $3,000 to $5,000 in the early 1990s to a range of $75,000 to $125,000 in 2013. In just 3 years (from 2008 to 2011), per-patient clinical trial costs increased by 70%, without reassurance that we are obtaining proportionately more quality for those research dollars.”
The complexity and delays inherent in launching a clinical trial are made graphic with the driest of statistics: “Within CTEP (the National Cancer Institute’s Cancer Therapy Evaluation Program), 296 distinct processes for phase III trial activation were identified, including at least 239 working steps, 52 major decision points, 20 processing loops, and 11 stopping points, with a median time from formal concept submission to trial activation of approximately 20 months (602 days) (emphasis added).
“If Edison were an American cancer investigator working in the early 21st century” write Steensma and Kantarjian, ” …His shirt might be soaked with sweat, but his productivity would likely be meager and his name unknown at the patent office. Today, the inspirational idealism of newly minted medical faculty members with a driving ambition to “cure cancer”—oncology’s Edisons—is being smothered under a mountain of red tape, their good intentions crushed by the logistical reality of what it actually takes to open and execute a clinical study.”
The article, “Impact of Cancer Research Bureaucracy on Innovation, Costs, and Patient Care” is well worth scanning for anyone interested in understanding the nature of clinical trial in the United States, today.
Take me back to the Contents
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