The study authors concluded that although “single-patient anecdotes are often dismissed as failing to provide meaningful clinical evidence, this example illustrates the potential for such cases to inform future clinical development of drugs in molecularly defined populations.”

For the Exceptional Responders Initiative, investigators are asked to propose cases without identifiers. Dr Conley and colleagues then use internal and literature reviews to determine whether the case is of an “exceptional responder,” defined as a patient who has received a treatment in which less than 10% of all patients (either in a clinical trial or historical experience, in the context of the tumor type) had a complete response or partial response of at least 6 months, either a complete or partial response with duration of at least 6 months as defined by RECIST, or a complete or partial response at least 3 times longer than median duration of response, based on the available literature.

Continue Reading

If the case is accepted, clinicians are invited to enter details into Medidata Rave and to submit tissue samples, which are sent to the Biospecimen Core Resource at Nationwide Children’s Hospital in Columbus, Ohio, for nucleic acid extraction.

If sufficient amounts of nucleic acid are available, they are sent to the Human Genome Sequencing Center at Baylor College of Medicine in Houston, Texas, to undergo whole genome sequencing, messenger ribonucleic acid (RNA) and micro RNA sequencing, promoter methylation analysis, and single nucleotide polymorphism analysis.

“My hope is that we can find something that might down the road be predictive of a response, even for standard chemotherapy,” Dr Conley said. “Naturally, we would love to find something that would predict you wouldn’t respond, too, so that we wouldn’t give people treatments that wouldn’t help them, but we will tackle this one method at a time.”

Investigators with patients whom they believe to be “exceptional responders” to treatment are urged to contact the Exceptional Responders Initiative as soon as possible for potential inclusion in the study.


  1. Exceptional responders initiative: questions and answers. National Cancer Institute website. Accessed May 2017.
  2. Iyer G, Hanrahan AJ, Milowsky MI, et al. Genome sequencing identifies a basis for everolimus sensitivity. Science. 2012;338(6104):221.