Rosenberg's "Gene Therapist, Heal Thyself" 2000

From Biol557

Current revision as of 19:50, 7 March 2010

• As a giant physician in medicine once reminded his students, "if what you're doing isn't working, try something else."
• And so it is with gene therapy; over a decade of promise and clinical trials has not resulted in a single unequivocal case of therapeutic efficacy by the year 2000.
• The authors express their concern over repeated promises of a "golden age" of gene therapy even in the light of the Gelsinger tradgedy--the death of a gene therapy patient.
• It is improper for scientists to insert themselves into the "culture and discord" of the field (that is, the politics, the rhetoric) because it can lead to improper decisions that are bad for patients, for science, and for society.
• The authors even go so far as to compare scientists who "insert themselves into the culture and discord" to viruses!
• Bone marrow transplants and chemotherapies for cancers have become important therapeutics and their development came at the price of some lives, too.
  • However, the scientists who developed these therapeutics maintained wide support of their science by promising little and delivering much--the opposite of gene therapy, thus far.
• We must have self-critical scientists to run these studies.
  • They must be scientists who will ask of themselves whether they or their family members would join their study.
  • They must honestly asses the benefit-to-cost ratio.
  • They must attend to good communication with study participants, proper timeliness, proper safety, honest assessment of result legitimacy, and proper discussion / disclosure of untoward results.
    • Untoward: "unfavourable, adverse, or disadvantageous; Unruly, troublesome; Unseemly, improper" ref
  • The authors call the members of the American Society for Gene Therapy to "pledge themselves to sound, disciplined science in the public interest and eschew uncritical winner-take-all gambles."
• The authors call for higher legislation of conflict-of-interest issues which seem to be common in gene-therapy trials.
  • They mention both personal conflict-of-interest (i.e. an investigator has stock in a company that is helping to fund their clinical trial) and institutional conflict-of-interest (i.e. a university is invested in a company that is running clinical trials through investigators at the university).
• Perhaps, if gene therapy scientists heed the old dictum (think "if what you're doing isn't working, try something else"), we can bring gene therapy to fruition and save some lives.