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The FDA, Juno Therapeutics, and the ethical imperative of transparency

BMJ 2016; 354 doi: (Published 15 August 2016) Cite this as: BMJ 2016;354:i4435
  1. Spencer Phillips Hey, research fellow1 2,
  2. Aaron S Kesselheim, associate professor of medicine1 2
  1. 1Program on Regulation, Therapeutics, and Law (PORTAL), division of pharmacoepidemiology and pharmacoeconomics, Brigham and Women’s Hospital, Boston, US
  2. 2Harvard Center for Bioethics, Harvard Medical School, Boston, US
  1. akesselheim{at}

Despite deaths, investigational new drugs are still protected trade secrets of the manufacturer

On July 7, Juno Therapeutics announced that the US Food and Drug Administration (FDA) had placed a clinical hold on the company’s phase 2 trial of its investigational immunotherapy, JCAR015, as a treatment for patients with relapsed or refractory B cell acute lymphoblastic leukaemia (ALL). The trial planned to enrol 90 ALL patients. However, at the time of the hold, only 20 patients had been enrolled, three of whom had died unexpectedly from cerebral oedema.1

The FDA lifted its hold three days later without further public comment, and Juno representatives explained that the three deaths were thought to be related to a mid-trial modification to the protocol. The original protocol included the administration of cyclophosphamide as a sensitising agent to enhance the efficacy of JCAR015. However, after the trial was underway, a second sensitising agent, fludarabine, was added on the basis of promising evidence from the company’s other immunotherapy studies. In its public statements, Juno attributed the deaths to the combination of fludarabine and JCAR015.2

Before a new drug can be tested on US patients, the sponsor …

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