Value Propositions – October 2014

October 2014, Vol 5 , No 8 - Value Propositions


Medicare Begins Coverage of Only FDA-Approved Noninvasive Stool DNA Test for Colorectal Cancer
Medicare now provides coverage for the only FDA-approved noninvasive stool DNA screening test for colorectal cancer (CRC), after the Centers for Medicare & Medicaid Services (CMS) issued its final National Coverage Determination in October. Cologuard is an easy-to-use test that can be self-administered at home. This test has demonstrated a 92% accuracy rate for detecting CRC in people at an average risk, with 87% specificity.

Exact Sciences, the maker of the test, announced that the coverage will go into effect immediately, although the company does not have information about the exact dollar amount that Medicare will cover for the test. This decision by CMS constitutes the first final coverage determination by the agency for a medical device that was approved by the FDA and is a part of the FDA and CMS parallel. Cologuard is the first noninvasive test for CRC that analyzes stool-based DNA as well as blood biomarkers to detect cancer.

“Colorectal cancer is often considered the most preventable, yet least prevented cancer due to the lack of patient compliance with screening. CMS’s decision to cover Cologuard will provide Medicare’s 50 million patients with access to this accurate screening option,” said Kevin Conroy, Exact Sciences President, CEO, and Chairman.

CRC is treatable when detected early, but it is often diagnosed at a late stage, when a cure is no longer feasible. CRC remains the second-leading cause of cancer-related death in the United States.

Exact Sciences Corp Press Release; October 9, 2014




NCI Launches Study of Exceptional Responders to Cancer Therapies: A New Phase in Precision Medicine?
The National Cancer Institute (NCI), a part of the National Institutes of Health, has launched the Exceptional Responders Initiative, a study that will investigate the molecular factors in patients with cancer whose tumors demonstrate exceptional responses to drug therapies. Investigators will examine tumor specimens from patients in clinical trials whose tumor demonstrated an exceptional response compared with other patients in that trial, or other patients who achieved an exceptional and unexpected response to a noninvestigational therapy.

The reasons why certain patients with cancer demonstrate unexpected and prolonged remissions after standard therapy remain unclear. The goal is to identify potential molecular markers that will predict positive responses to certain therapies, which will then allow oncologists to better select the most appropriate treatment regimens for individual patients.

Tumor tissue and clinical data will be collected from NCI-based and other clinical trials for patients with exceptional responses. Other samples will be collected from standard cancer care settings, such as community practice, and from clinical trials funded by pharmaceutical companies or other sources. Letters are being sent to cancer centers and other oncology practice settings nationwide, asking them to assist in this effort and provide appropriate information for patients demonstrating exceptional responses.

“The feasibility of this approach is supported by reports in the literature of relevant mutations in tumor specimens from patients who experienced an exceptional response to a drug in a clinical trial, even though that drug failed to meet the trial’s endpoint for clinical benefit,” said Louis Staudt, MD, PhD, Director of NCI’s Center for Cancer Genomics, and a co-leader of this study.

“The ability to identify molecular markers that are able to predict a clinical response in these subsets of patients will provide us with the tools to further advance our ability to conduct studies consistent with the principles of precision medicine,” noted Barbara A. Conley, MD, Division of Cancer Treatment and Diagnosis, NCI, and a co-leader of the study.

The ultimate goal is for oncologists to be able to identify patients who may respond to agents with the same or similar mechanism of action, and to build on the data generated by this study that will provide clinicians access to the full genomic data of 100 cases of exceptional responders to identify patterns in drug response.

NCI Press Release; September 24, 2014




New Collaboration on Novel Immunotherapy Vaccine May Revolutionize Lung Cancer Treatment
The drugmaker Boehringer Ingelheim (BI) and CureVac, a leader in mRNA (messenger ribonucleic acid)-based drug development, announce an exclusive collaboration for the continuing development of CureVac’s CV9202 vaccine, an immunotherapy vaccine that has shown promising results in early development for the treatment of lung cancer. BI is launching clinical trials for the vaccine in at least 2 settings—(1) in combination with afatinib (Gilotrif) in patients with advanced or metastatic EGFR-mutated non–small-cell lung cancer (NSCLC) and (2) in combination with radiotherapy in patients with unresectable stage III NSCLC.

This investigational vaccine represents a novel approach to cancer treatment by optimizing the mRNA to mobilize the patient’s own immune system to fight the tumor with a specific immune response. CV9202 is a combination of mRNA molecules coding for 6 antigens overexpressed in lung cancer, and is designed to induce an immune response against the tumor.

According to BI, this new agreement reflects the company’s focus on novel treatments with high therapeutic value for patients. “In our collaboration with CureVac, we will investigate combining existing treatments with the approach of sustained activation of the immune system,” said Professor Klaus Dugi, Chief Medical Officer at BI.

Ingmar Hoerr, Co-founder and CEO of CureVac GmbH, noted, “Cancer immunotherapy represents one of the biggest innovations in cancer treatment of recent times, and we are delighted to now be working with Boehringer Ingelheim.”

PRNewswire, September 18, 2014




Mechanism of Action of Low-Dose Aspirin in Reducing Cancer Mortality Identified
New data presented at the 13th Annual AACR International Conference on Frontiers in Cancer Prevention Research identified the mechanism of action by which low-dose aspirin reduces the risk for cancer metastasis. That mechanism is believed to be the inhibition of COX-1 and COX-2 pathways. The realization that the COX-2 pathway is involved in aspirin’s cancer prevention activity is new; previous studies have only found a link to the COX-1 pathway.

“Our findings may explain how even a very low dose of aspirin taken daily can produce such a substantial reduction in cancer deaths and metastasis,” said Pierre Massion, MD, Professor of Medicine and Cancer Biology, Vanderbilt University School of Medicine, Nashville, TN.

“We found that the potency of low-dose aspirin in inhibiting COX-2 in the tumor cells is as great as or greater than its potency as an inhibitor of COX-1 in the platelet,” Dr Massion said. “This indicates that at a cellular level, aspirin is not selective for the platelet, but could also block COX-2 in cancers.”

Dr Massion and colleagues found that in addition to blocking platelet function by inhibiting the COX-1 pathway, low-dose aspirin also reduced levels of the prometastatic molecule prostaglandin E2 (PGE2) through inhibition of the COX-2 pathway.

“Thus, the finding that it [aspirin] blocked PGE2 production is likely surprising to most investigators in the field,” Dr Massion said.

Previous studies have shown that taking aspirin for ?5 years reduces the incidence of all cancers by 38%. “This benefit is seen even at the low doses of aspirin required to inhibit platelet aggregation via inhibiting the COX-1 pathway, a finding consistent with the known participation of platelets in the metastatic process,” he noted.

The investigators found that (1) the doses of aspirin required to inhibit COX-2–dependent PGE2 production were the same or lower than those needed to inhibit COX-1–dependent platelet aggregation; (2) aspirin inhibited PGE2 production by 45% and reduced the levels of a metabolite of PGE2, prostacyclin, by 37%; and (3) by blocking platelet COX-1, aspirin inhibits platelet adherence to the cancer cells to prevent metastasis, suggesting that these 2 mechanisms work together to reduce cancer mortality risk.

American Association for Cancer Research; September 29, 2014