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Cancer Genomics Experts Hope Precision Medicine Effort Will Raise Awareness, Improve Clinical Trials

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NEW YORK (GenomeWeb) – Although President Obama's recently announced precision medicine initiative needs to be approved by Congress, and details of specific projects were scarce, a number of experts in the cancer genomics field are considering the positive impact it might have on research and patient care.

After noting the precision medicine initiative in his State of the Union Address earlier in January, last week the president announced the launch of the $215 million effort.

The initiative's near-term goal is to focus on cancer, and it included $70 million for the National Cancer Institute to spur research into the genetic basis of cancer by expanding genetically based clinical trials, studying cancer biology, and establishing a national "cancer knowledge network" to generate and share knowledge.

In an editorial published the same day in The New England Journal of Medicine, National Institutes of Health Director Francis Collins and NCI Director Harold Varmus wrote that "oncology is the clear choice for enhancing the near-term impact of precision medicine." Significant advances have already been made in identifying cancer driver genes, specific genomic signatures, and using next-generation sequencing-based tumor profiling to guide treatment, they noted.

But going forward, they said, the cancer-focused part of the initiative should address obstacles that have been encountered in precision oncology: "unexplained drug resistance, genomic heterogeneity of tumors, insufficient means for monitoring responses and tumor recurrence, and limited knowledge about the use of drug combinations."

Raise awareness

One of the most immediate impacts, which does not rely on Congress approving funding, is that the President's announcement will raise national awareness about cancer genomics.

"It is serving to put a lens on precision medicine," Gianrico Farrugia, medical director at the Mayo Clinic Center for Individualized Medicine, told GenomeWeb.

While the funding itself will not be the "sole answer" and will also require public/private partnerships, the fact that the president mentioned precision medicine in a State of the Union address will help "energize the field," Farrugia said. "It's a major step forward."

Jeff Boyd, senior vice president of molecular medicine at the Fox Chase Cancer Center and executive director of its Cancer Genome Institute, said that particularly because the precision medicine initiative was such an "out of the box" item, he thinks people will pay attention, especially cancer patients.

"I think it will lead them to ask more questions to their physicians," he said. "And to the extent that patients become more educated about precision medicine in cancer and ask about clinical trials, it could help get more patients on clinical trials, which is extraordinarily important."

Victor Velculescu, co-director of cancer biology at Johns Hopkins University, likened the inclusion of precision medicine in the State of the Union to President John F. Kennedy's initial declaration of wanting to put a man on the moon. Similar to how that inspired people to go into engineering and science, he thinks the precision medicine initiative will inspire people to go into genomics and bioinformatics.

Clinical trials

Beyond raising awareness, the initiative could have more tangible impacts as well. Included in the initiative was a specific mention of clinical trials within the cancer genomics space, an incredibly important facet toward bringing the benefits of cancer genomics research to patients, said Boyd.

"The only way this field will move forward in a substantive way in the near term is a more refined program for clinical trials," he said. Such clinical trials involve genetically profiling individuals, treating them with a drug that targets their genetic profile, and comparing to the standard of care. Such trials, often called basket trials, can be smaller and therefore less expensive, and involve partnering with pharmaceutical companies.

For instance, the NCI-MATCH trial is profiling patients with a targeted NGS panel to randomize them into one of many different phase II trials. NCI has also launched a study called Molecular Profiling based Assignment of Cancer Therapeutics (M-PACT), in which researchers will sequence the tumors of hundreds of patients for 391 different mutations in 20 genes known to impact response to various targeted therapies.

Such trials are "important for the cancer space because they indicate a willingness to look at different ways to approve drugs," Farrugia said, "which would be a significant step forward."  While the research field is already moving beyond defining cancer by its tissue of origin, most drugs are still approved for tissue-specific cancers, rather than the specific genomic profile of a tumor, which can hinder drug access to patients that might benefit.

Arul Chinnaiyan, director of the University of Michigan Center for Translational Pathology, said that the move toward these types of basket trials like NCI-MATCH, is a good area of focus.

"Definitely the field is moving in that direction," he said. And the precision medicine initiative will help "fuel the move in that direction." Increasingly, researchers are finding that "binning patients based on mutations and pathways, rather than the tissue site of origin," makes sense, he added. The "one size fits all approach is not going to work."

Such clinical trials could also "help shed greater insight on the mechanisms that define being a good responder or not" to particular drugs, said Harry Ostrer, professor of pathology at the Albert Einstein College of Medicine. Also, "identifying a drug that's not only going to be effective but is not toxic is important."

Finally, a third hope is that focusing on clinical trials will generate evidence that makes the case to third-party payors for reimbursement. There is already anecdotal evidence suggesting that genomic tumor profiling makes a difference clinically and could be cost effective, Chinnaiyan said, but the hope is that the initiative will "push the field forward to really substantiate" not only the clinical evidence but cost-effectiveness evidence. The cost of doing sequencing-based analyses is relatively small compared to putting a patient on some of the available biological therapies, and if the sequencing-based analyses help avoid ineffective, expensive drugs, then it is worth it, he said.

"We need to show that what we do is valuable to the patient, physician, and the payor," Farrugia said. "And if this accelerates our ability to show value, it will affect reimbursement."

Gaining more broad reimbursement decisions will also help expand access to genomic-based cancer tests.

"The big impediment right now in terms of availability of testing is reimbursement," Fox Chase's Boyd said. "The future is highly dependent on CMS and the private third-party payors to make their conclusions about the value and efficacy of these tests and begin reimbursing at a reasonable level."

Patient care

In terms of the impacts of the precision medicine initiative on actual cancer patient care, those are harder to quantify, especially since the initiative did not include specific details on the types of projects on which it would focus.

Chinnaiyan said that he would like to see an emphasis on using genomic technologies to analyze actual patient samples, as opposed to archival tissue, which he said would help speed the transition from research to clinic. In addition, he said "the real promise is in bringing these approaches earlier in the development of cancer so we have a better chance of treating the disease."

Currently, genomic tumor profiling often focuses on patients with advanced cancer, Chinaiyan said. In those cases, "the tumor has evolved quite significantly and it is difficult to develop a precision medicine approach because it has developed so many resistance mechanisms."

Johns Hopkins' Velculescu agreed that for precision medicine in oncology to truly have an impact on patient care, genomics would need to be integrated earlier. "My dream scenario is precision early diagnostics," he said, "where we have a test in the home, that you can buy off the shelf" for early detection of specific lesions.

Healthy skepticism

Despite the potential promise that precision medicine holds in the field of oncology, the researchers all noted that the level of funding indicated by the president was not enormous and was contingent on being approved by Congress.

"My concerns are not in the specific areas they are targeting; they are right on the mark. It's a matter of the details — how they will get there and how will they do it with this amount of capital," Boyd said. In addition, he said, it is not clear whether this would be a one-time investment, or whether the initiative indicates long-term ongoing funding.

"The funding isn't huge," Ostrer noted. "And needs to be looked at in terms of the overall effort supported by the federal government."

Chinnaiyan agreed that the amount was "not incredibly large," but said it does "suggest a commitment to precision medicine and will maybe foster additional efforts in the field."

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