Can More Investigations on Miracle Patients be The Answer To Better Cancer Treatment

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During clinical trials for experimental cancer drugs, some patients simply respond better than others. A tiny fraction of patients sees dramatic results, responding so well to treatment that they survive forms of cancers that quickly kill their counterparts. Perakslis, who heads up pharmaceutical giant Takeda’s oncology data science program and is a visiting faculty member in the Department of Biomedical Informatics at Harvard Medical School, argues that their genes could hold clues that cancer researchers have been missing.

In a recent editorial in the journal Science Translational Medicine, the former FDA chief information officer and kidney cancer survivor joins a growing chorus of voices calling for more research on exceptional responders. If scientists sequence and study the genomes of these patients, Perakslis argues, they could uncover potential cures that may work for traditional patients, too.

“We as humans may or may not be smart enough to find it, but there’s a reason.” A hunt for that reason could reveal new information about different cancers, he argues – as in one case in which a patient with metastatic bladder cancer saw dramatic results with an experimental drug that was abandoned after an unsuccessful trial.

Analysis of the patient’s genome eventually identified a previously unknown biomarker found in some other bladder cancers. Given that over 8 million people die of cancer each year worldwide, Perakslis says it’s time to establish a national exceptional responder network among clinical trial participants that offers free genome sequencing, registers all patients who do unexpectedly well during clinical trials, and makes their data available to qualified researchers.

If the kind of broad-scale initiative open to patients with the rarest diseases were available to cancer patients, too, he argues, unlocking their secrets could become easier and cheaper – and reveal critical clues faster. He participates in the National Cancer Institute’s Exceptional Responders Initiative, a project aimed at understanding the molecular underpinnings of those whose cancers respond to all different kinds of treatment.

Because of the insights yielded by exceptional responders’ genes, Zenklusen says, researchers now realize that some cancers share molecular signatures despite the anatomical location of the tumors themselves. Patients who have demonstrated an exceptional response to any modality – experimental or no – are welcome, but must fit strict criteria before molecular characterization takes place.

“Unfortunately, when we find something that is interesting, we tend to get very excited. Cancer is a horrible disease. But sometimes we communicate the information a little too enthusiastically.”

He worries that leaping toward a national registry before it’s been firmly established that exceptional responders really have smoking guns tucked into their genome could mislead the public into thinking that personalized, precision cures for cancer are just around the corner. For him, the challenge is data density – which makes it even more urgent to collect as much data from exceptional responders as possible.

“[There are] so many cancers, subtypes, unique patients, potential treatments,” he says.

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