In the Journals

Panelists: Advances in personalized medicine must be balanced with ‘clinical reality’

PHILADELPHIA — The rapid adoption and evolution of personalized cancer therapy has created several formidable challenges that must be overcome to maximize outcomes, according to oncology key opinion leaders.

For example, current policies may not ensure patient access to personalized treatments. Although genetic profiling and trials for targeted therapies are widely available in academic settings, access in the community setting — where a majority of patients with cancer are treated — is essential.

Further, despite the promise of modern regimens, much work remains to identify the underlying genetic causes of malignancies across patient subgroups.

“Personalized medicine will lead to major improvements in the practice of medicine,” said John Hoctor, vice president of government relations for the American Cancer Society’s Cancer Action Network. “However, it will be important to clearly identify obstacles and limitations along the way to successful implementation. With these challenges in mind, we are better able to work to develop and implement policies to achieve goals of maximally empowering patients with the most effective tools to improve their health outcomes.”

Hoctor and several other clinicians, researchers and health executives participated in a Cancer Action Network-hosted round table in November to explore the efficacy and availability of personalized medicine.

Panelists discussed the influence of genetics and genomics on cancer treatment; patient expectations of personalized medicine; and the accessibility of genetic testing and personalized treatment platforms outside academic centers.

Breakthroughs and setbacks

Although researchers and clinicians hope personalized medicine strategies increase the chance of treatment success among patients with cancer, the reality is personalized medicine is not a novel concept, according to William Kevin Kelly, DO, professor of medical oncology and urology and director of solid tumor oncology at Thomas Jefferson University.

William Kevin Kelly

“We have been doing precision medicine for decades,” Kelly said. “In the past, we have talked about predictive and prognostic markers — those are still important here. We talked about ALK mutations in lung cancer, which can pick up a portion of actionable patients.”

However, patients with known, targetable mutations may be in the minority.

“We have to remember that these are just a small fraction of patients — about 5%,” Kelly said. “That means there are 95% of patients we still do not have [personalized] treatments for. While we are focusing on the advancements, it is important to keep a check on the clinical reality.”

Still, personalized medicine represents a shift in how cancer is viewed and understood, as it once was considered a monolithic disease, said Wafik El-Deiry, MD, PhD, FACP, deputy cancer center director for translational research and co-program leader of molecular therapeutics at Fox Chase Cancer Center.

Wafik El-Deiry

“Historically, patients with a specific type of cancer have all received the same type of treatment,” El-Deiry said. “Moving forward, the world is changing. We are learning more about cancer and its heterogeneity. As such, we are developing treatments that [target] some cancers better than they do to others.”

El-Deiry — who specializes in the treatment of patients with gastrointestinal cancers — noted that personalized medicine techniques are becoming standard in many cancer subtypes.

“For treating colorectal cancer in 2015, we really must know what the KRAS status of a tumor is to be able to prescribe the best therapy,” he said. “For lung cancer, we need to know about epidermal growth factor receptor mutations and ALK mutations.”

David B. Roth, MD, PhD, chair of the department of pathology and laboratory medicine and director of precision medicine at University of Pennsylvania’s Perelman School of Medicine, discussed the value next-generation sequencing of actionable mutations offers from a practical, pathological perspective.

David B. Roth

“The average lung cancer sample we receive in the pathology lab is smaller than the tip of my pinkie finger,” Roth said. “From that, we are expected to do all sorts of testing by cutting little tiny tissue slices, which can take weeks. Once you have more than a handful of mutations to look for, it becomes a practical and logistical problem.”

Roth and colleagues at University of Pennsylvania developed a panel of actionable targets that could be useful for several cancer types. Additionally, they developed a panel applicable to hematologic malignancies.

“Our solid tumor panel has 47 genes,” Roth said. “We then developed the Penn Precision Panel, which has about 20 genes. We have already sequenced 5,000 patients, and we will very soon be using [these panels] on every single patient who comes through the door.”

Industry perspective

Availability of technology — through mass databases of genomic information and diagnostic tools — is vital to the implementation of personalized medicine.

Databases made available online can increase access to personalized medicine approaches in rural and community health centers, according to William A. Faucett, MS, LGC, director of policy and education at Geisinger Health System.

“We started building a biobank because we viewed personalized and precision medicine as the future of medicine,” Faucett said. “Our goal is to enroll 250,000 individuals, and we are currently approaching 90,000, with the goal of performing next-generation sequencing. We are adding about 1,000 exomes to the cloud per week.”

Personalized medicine eventually will be achievable across the board at the community medical level, Faucett said.

“Our vision is that, when you walk into the clinic, your genome will already be in the medical record,” he said.

Evaluation of the available tools — through a health technology assessment — may help clinicians sort through their many options.

“You cannot simply read the latest one or two journal articles,” said Vivian H. Coates, MBA, vice president of information services and health technology assessment at ECRI Institute, a nonprofit organization that researches approaches to improving patient care.

“We put together the entire body of evidence in order to assess its strength and quality and answer the specific key questions,” Coates said. “There are 36,000 genetic and genomic tests available out there, but many tests do not change patient management. We look for the evidence of analytic validity, clinical validity and evidence of clinical utility.”

Companion diagnostics are one such tool.

“Personalized medicine follows the science in an attempt to move away from the view of cancer treatment as a one-size-fits-all approach,” Vivian Pacheco, MBA, director of global marketing oncology companion diagnostics at Janssen Diagnostics, said during the round table. “Companion diagnostics allow us to help move in that direction.”

This has implications for how drugs are tested and approved.

“Biomarker diagnostics are needed — you need to find the specific tumor in order to target it,” Pacheco said. “You no longer have to work with the FDA only on how to develop a drug and bring it to market safely and with efficacy. You also have to develop a test alongside with it. Now, when a treatment comes to market, there will be a drug and a test, and clinical trials today reflect that.”

Looking forward

Despite the mounting excitement surrounding personalized medicine, lingering economic and ethical questions remain, Kelly said.

“There are disparities and ethical issues associated with genomic testing,” Kelly said. “What is your responsibility to notify a patient if you find a germline mutation? These tests can also be very expensive and very cumbersome. At Jefferson ... we do not test every patient. We try to test patients when we feel they arrive at the right time and can derive the most benefit.”

However, clinicians and industry professionals agreed that personalized medicine approaches will continue to grow in visibility and prominence in the fields of cancer treatment and research.

Further, the ACS will continue to encourage politicians to support funding that could make personalized medicine more widely available across the United States, Hoctor said.

“We are committed to encouraging our elected officials to do all that they can do to make personalized medicine available to all patients with cancer,” Hoctor said. – by Cameron Kelsall

Reference:

ACS Cancer Action Network Personalized Medicine Round Table; Nov. 3, 2015; Philadelphia.

Disclosure: HemOnc Today was unable to confirm the round table participants’ relevant financial disclosures at the time of reporting.

PHILADELPHIA — The rapid adoption and evolution of personalized cancer therapy has created several formidable challenges that must be overcome to maximize outcomes, according to oncology key opinion leaders.

For example, current policies may not ensure patient access to personalized treatments. Although genetic profiling and trials for targeted therapies are widely available in academic settings, access in the community setting — where a majority of patients with cancer are treated — is essential.

Further, despite the promise of modern regimens, much work remains to identify the underlying genetic causes of malignancies across patient subgroups.

“Personalized medicine will lead to major improvements in the practice of medicine,” said John Hoctor, vice president of government relations for the American Cancer Society’s Cancer Action Network. “However, it will be important to clearly identify obstacles and limitations along the way to successful implementation. With these challenges in mind, we are better able to work to develop and implement policies to achieve goals of maximally empowering patients with the most effective tools to improve their health outcomes.”

Hoctor and several other clinicians, researchers and health executives participated in a Cancer Action Network-hosted round table in November to explore the efficacy and availability of personalized medicine.

Panelists discussed the influence of genetics and genomics on cancer treatment; patient expectations of personalized medicine; and the accessibility of genetic testing and personalized treatment platforms outside academic centers.

Breakthroughs and setbacks

Although researchers and clinicians hope personalized medicine strategies increase the chance of treatment success among patients with cancer, the reality is personalized medicine is not a novel concept, according to William Kevin Kelly, DO, professor of medical oncology and urology and director of solid tumor oncology at Thomas Jefferson University.

William Kevin Kelly

“We have been doing precision medicine for decades,” Kelly said. “In the past, we have talked about predictive and prognostic markers — those are still important here. We talked about ALK mutations in lung cancer, which can pick up a portion of actionable patients.”

However, patients with known, targetable mutations may be in the minority.

“We have to remember that these are just a small fraction of patients — about 5%,” Kelly said. “That means there are 95% of patients we still do not have [personalized] treatments for. While we are focusing on the advancements, it is important to keep a check on the clinical reality.”

Still, personalized medicine represents a shift in how cancer is viewed and understood, as it once was considered a monolithic disease, said Wafik El-Deiry, MD, PhD, FACP, deputy cancer center director for translational research and co-program leader of molecular therapeutics at Fox Chase Cancer Center.

Wafik El-Deiry

“Historically, patients with a specific type of cancer have all received the same type of treatment,” El-Deiry said. “Moving forward, the world is changing. We are learning more about cancer and its heterogeneity. As such, we are developing treatments that [target] some cancers better than they do to others.”

El-Deiry — who specializes in the treatment of patients with gastrointestinal cancers — noted that personalized medicine techniques are becoming standard in many cancer subtypes.

“For treating colorectal cancer in 2015, we really must know what the KRAS status of a tumor is to be able to prescribe the best therapy,” he said. “For lung cancer, we need to know about epidermal growth factor receptor mutations and ALK mutations.”

David B. Roth, MD, PhD, chair of the department of pathology and laboratory medicine and director of precision medicine at University of Pennsylvania’s Perelman School of Medicine, discussed the value next-generation sequencing of actionable mutations offers from a practical, pathological perspective.

David B. Roth

“The average lung cancer sample we receive in the pathology lab is smaller than the tip of my pinkie finger,” Roth said. “From that, we are expected to do all sorts of testing by cutting little tiny tissue slices, which can take weeks. Once you have more than a handful of mutations to look for, it becomes a practical and logistical problem.”

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Roth and colleagues at University of Pennsylvania developed a panel of actionable targets that could be useful for several cancer types. Additionally, they developed a panel applicable to hematologic malignancies.

“Our solid tumor panel has 47 genes,” Roth said. “We then developed the Penn Precision Panel, which has about 20 genes. We have already sequenced 5,000 patients, and we will very soon be using [these panels] on every single patient who comes through the door.”

Industry perspective

Availability of technology — through mass databases of genomic information and diagnostic tools — is vital to the implementation of personalized medicine.

Databases made available online can increase access to personalized medicine approaches in rural and community health centers, according to William A. Faucett, MS, LGC, director of policy and education at Geisinger Health System.

“We started building a biobank because we viewed personalized and precision medicine as the future of medicine,” Faucett said. “Our goal is to enroll 250,000 individuals, and we are currently approaching 90,000, with the goal of performing next-generation sequencing. We are adding about 1,000 exomes to the cloud per week.”

Personalized medicine eventually will be achievable across the board at the community medical level, Faucett said.

“Our vision is that, when you walk into the clinic, your genome will already be in the medical record,” he said.

Evaluation of the available tools — through a health technology assessment — may help clinicians sort through their many options.

“You cannot simply read the latest one or two journal articles,” said Vivian H. Coates, MBA, vice president of information services and health technology assessment at ECRI Institute, a nonprofit organization that researches approaches to improving patient care.

“We put together the entire body of evidence in order to assess its strength and quality and answer the specific key questions,” Coates said. “There are 36,000 genetic and genomic tests available out there, but many tests do not change patient management. We look for the evidence of analytic validity, clinical validity and evidence of clinical utility.”

Companion diagnostics are one such tool.

“Personalized medicine follows the science in an attempt to move away from the view of cancer treatment as a one-size-fits-all approach,” Vivian Pacheco, MBA, director of global marketing oncology companion diagnostics at Janssen Diagnostics, said during the round table. “Companion diagnostics allow us to help move in that direction.”

This has implications for how drugs are tested and approved.

“Biomarker diagnostics are needed — you need to find the specific tumor in order to target it,” Pacheco said. “You no longer have to work with the FDA only on how to develop a drug and bring it to market safely and with efficacy. You also have to develop a test alongside with it. Now, when a treatment comes to market, there will be a drug and a test, and clinical trials today reflect that.”

Looking forward

Despite the mounting excitement surrounding personalized medicine, lingering economic and ethical questions remain, Kelly said.

“There are disparities and ethical issues associated with genomic testing,” Kelly said. “What is your responsibility to notify a patient if you find a germline mutation? These tests can also be very expensive and very cumbersome. At Jefferson ... we do not test every patient. We try to test patients when we feel they arrive at the right time and can derive the most benefit.”

However, clinicians and industry professionals agreed that personalized medicine approaches will continue to grow in visibility and prominence in the fields of cancer treatment and research.

Further, the ACS will continue to encourage politicians to support funding that could make personalized medicine more widely available across the United States, Hoctor said.

“We are committed to encouraging our elected officials to do all that they can do to make personalized medicine available to all patients with cancer,” Hoctor said. – by Cameron Kelsall

Reference:

ACS Cancer Action Network Personalized Medicine Round Table; Nov. 3, 2015; Philadelphia.

Disclosure: HemOnc Today was unable to confirm the round table participants’ relevant financial disclosures at the time of reporting.