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New Weill Cornell Precision Medicine Institute Plans to Offer Genomically Guided Treatment after CLIA Approval

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Through a newly created Institute for Precision Medicine, Weill Cornell Medical College and New York Presbyterian Hospital plan to begin offering targeted, individualized treatment informed by patients' genomes.

The institute first plans to guide treatment decisions for cancer patients using their genomic data, and then broaden the effort to those with common illnesses, such as cardiovascular disease and neurodegenerative disorders.

The new institute is currently awaiting regulatory approval from CLIA and New York State, according to its leader, Mark Rubin, a professor of pathology at Weill Cornell.

With that approval in hand, the center will begin using genome sequencing and other tools to inform treatment strategies for patients – first focusing on cancer, and then eventually broadening to other disease areas, he said.

While Rubin did not detail how the institute will recruit patients, he said the center plans to see cancer patients who can benefit from single-gene tests or other molecular diagnostics to inform treatment decisions, those with advanced diseases without treatment options, and patients who stop responding to standard treatments and could be redirected to other therapies.

"For some patients, there are very clear indications of whether they need a specific targeted therapy. Those are pretty straightforward," Rubin said.

"And then there is emerging data that sequencing, either exome or whole-genome, can provide insight on which treatments cancer patients might need that are not considered standard treatments," he said.

Insights from advanced sequencing technologies are also changing how researchers study patients, sometimes facilitating N-of-1 trials. "There are a few examples where treatments have been implemented and shown to be effective in a clinical trial of one," Rubin said, "where they are the only person on the trial because of their specific mutations."

He said the institute plans to be agnostic in terms of what technologies it uses for sequencing, but currently it relies on Illumina technology.

"We will have a number of different approaches, but the key is to do as best as possible in the clinical setting so that the results can be used in the management of patients," Rubin said.

According to Rubin, the institute aims to find the optimal ways to collect genomic data, analyze it, and store it. As the center gears up and sees larger numbers of patients, Rubin said it also plans to use data and samples it collects to support larger retrospective or prospective studies, for which the institute is considering partnering with the New York Genome Center.

But not all patients the institute sees may require large-scale genome sequencing, Rubin reiterated.

"It may turn out that the most efficient way to determine if someone has a certain mutation, like EGFR, is to run the single-gene test up front. That's not going to change for some types of disease," he noted. "So, what I see our role being is developing these more complex approaches, which could be whole-genome sequencing, or using multiplex panels of genes."

The institute will focus its efforts first on cancer patients because the development of genomically targeted therapies is relatively accelerated compared to other disease areas, so the potential to match a mutation in a cancer patient's genome to a potential treatment may be greater than for those with other illnesses. But according to Rubin, the institute does plan to expand to other populations, like cardiovascular conditions, neurodegenerative diseases, and possibly infectious diseases.

In addition, he said the Institute is also discussing how it might use prognostic genomic information to look at disease risk, with the potential to inform early interventional treatment decision making.

"Because we are a hospital that sees well patients being followed by their doctors, that's something we're contemplating as pilot," he said.

"We don’t have a plan in mind yet, but those types of studies are probably very important in specific disease entities, for patients at risk for a particular constitutional disease … or you could imagine we might screen large numbers of our patient population to look for risk factors that may not have been identified yet," he said.

Several commercial and academic groups have recently begun offering clinical cancer sequencing and other genomic analyses to potentially guide therapeutic decision making.

Foundation Medicine, for example, sells a test that sequences the exons of nearly 200 genes known to be mutated in solid tumors and provides a report informing doctors of actionable mutations.

Other firms, like Caris Life Sciences, provide reports to doctors and patients matching gene-expression or sequence data to potentially actionable therapies.

The University of Michigan and the International Genomics Consortium announced last fall that they were creating a non-profit company called Paradigm to provide a targeted-sequencing-based diagnostic service to guide personalized treatment for cancer and, eventually, other diseases (PGx Reporter 7/5/2012).

Though the new Weill Cornell institute plans to start fairly narrowly, Rubin's overall goals for the center may put it in a position to offer a potentially more comprehensive service than many of these other groups.

"The most challenging part right now is for us to understand that sequencing is just a test, something that may or may not be useful in itself. It's in working in the clinical setting that we are going to really define it," Rubin said.

The institute, in the early days of its operations, is still in a learning phase, but "expectations are high" for the effort to succeed, according to Rubin. "Our job is to live up to the promise [of sequencing] to help identify novel targets for patients who may not have any choices with respect to treatment," Rubin said. "And also to make discoveries that may be useful for a larger population."

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