ASCO Annual Report 2017: Immunotherapy 2.0 Advance of the Year

Eileen Koutnik-Fotopoulos

June 2017, Vol 8, No 3 - Value in Oncology


Advances in immunotherapy are benefiting increasing numbers of patients living with cancer. Since 2011, the FDA approved 15 immunotherapies in oncology, including 5 immunotherapy drugs in 2016. This surge of progress using cancer immunotherapy has led the American Society of Clinical Oncology (ASCO) to name “Immunotherapy 2.0” as its cancer advance of the year for a second year in a row.

The 2017 annual report of ASCO highlights the key progress achieved in patient care and emerging trends in oncology (Burstein HJ, et al. J Clin Oncol. 2017;35:1341-1367).

Immunotherapy 2.0 Key

Immunotherapy 2.0 refers to the next phase of immunotherapy, which is focused on identifying patients most likely to benefit from these new treatments, looking for mechanisms of drug resistance, and reducing toxicities. In 2016, immunotherapy research showed that checkpoint inhibitors are especially effective in tumors associated with genetic mutations and those with high levels of the PD-1 or PD ligand 1 (PD-L1) protein.

“Over the last year, there has been a wave of new successes with immunotherapy. Research has proven this approach can be effective against a wide range of hard-to-treat advanced cancers previously considered intractable. Researchers are now working to identify biologic markers that can help increase the effectiveness of treatment and determine who is most likely to benefit from immunotherapy,” stated ASCO President Daniel F. Hayes, MD, FASCO, FACP, in his introduction to the report.

The biggest advances in immunotherapy in recent years have been in immune checkpoint inhibitors. In the past year, the FDA approved immune checkpoint inhibitors for 5 new tumor types, including lung cancer, head and neck cancer, kidney cancer, bladder cancer, and Hodgkin lymphoma. For example, the FDA’s approval of atezolizumab (Tecentriq) in 2016 marked the first new treatment for bladder cancer in more than 3 decades, and the first PD-L1 checkpoint inhibitor to be approved by the FDA. The first 2 checkpoint inhibitors approved, nivolumab (Opdivo) and pembrolizumab (Keytruda), are PD-1 rather than PD-L1 inhibitors.

Checkpoint inhibitors have revolutionized the treatment of patients with non–small-cell lung cancer (NSCLC). In 2016, results from a large clinical trial comparing pembrolizumab versus chemotherapy in patients with PD-L1–positive advanced NSCLC were instrumental in establishing pembrolizumab as the new standard of care for this patient population, and underlined the importance of PD-L1 testing to reap the most benefit from treatment.

Advances in Personalized Medicine

Another key trend that is driving progress against cancer is precision medicine. This approach includes treatments directed at individual molecular targets, genomic testing, and new ways to combine traditional cancer treatments.

In 2016, targeting molecules important in the growth of certain cancers resulted in new targeted therapies for advanced cancers of the lung, breast, and kidney, as well as several hard-to-treat forms of blood cancer, such as acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL).

For example, the recently FDA-approved midostaurin (Rydapt) targets the FLT3 mutation in patients with AML, and was shown to prolong survival, in combination with chemotherapy, compared with chemotherapy alone.

Liquid biopsies may also help personalize cancer therapy by providing information about genetic changes in the tumor as cancers grow and spread. This information can be used to stop a treatment that is becoming resistant to therapy or to switch to another treatment that is tailored to a different mutation as it appears.

Another advantage of a liquid biopsy is its potential to provide a snapshot of the full landscape of genetic changes in the tumor. The cobas EGFR Mutation Test v2 to detect the EGFR T790M mutation associated with NSCLC helped to identify patients who would be eligible for osimertinib (Tagrisso), which was approved in 2016.

Also in 2016, the cobas EGFR Mutation Test v2 became the first “liquid biopsy” to be approved by the FDA to identify patients with exon 19 deletions or exon 21 substitution in the EGFR gene who may benefit from erlotinib (Tarceva). Researchers are also proposing the use of liquid biopsies to predict recurrence in patients with stage II colon cancer.

Patient-Centered Care

Patient-centered care is another important advancement in the cancer community. The report highlights 2 key ASCO initiatives in 2016, including CancerLinQ, which facilitates the use of big data to improve the quality of cancer care rapidly, and ASCO’s first clinical trial, Targeted Agent and Profiling Utilization Registry (TAPUR). TAPUR is designed to evaluate FDA-approved targeted cancer drugs and collect data on clinical outcomes in addition to the drugs’ approved indications.

New tools to help bridge the gaps between physicians and patients are under development, such as an Internet-based tool for self-monitoring symptoms that immediately alerts the care team when patients report a worsening symptom. Another effort is education and navigation programs for underserved populations.

In addition, the report reviews clinical research, providing long-awaited answers about outcomes for patients with early prostate cancer related to active surveillance, surgery, and radiation. A study of 1643 men with localized prostate cancer and a median follow-up of 10 years showed no significant differences in mortality among the 3 approaches. This finding will help inform treatment discussions between physicians and patients.

Genetic Testing and Vaccination

A growing understanding of cancer biology has led to the identification of genetic mutations that may increase the risk for different cancers, including ovarian cancer and pancreatic cancer.

For example, in addition to mutations in the BRCA1 and BRCA2 genes, a recent study showed that women with the RAD51C and RAD51D mutations have an increased risk for ovarian cancer.

Genetic testing is becoming an important component of cancer risk assessment, diagnosis, and treatment, and should be incorporated into clinical practice. Some insurance companies are making it difficult for oncology practices to integrate genetic testing, but “ASCO opposes any policy that introduces an unnecessary barrier to the appropriate use of genetic testing services or has the potential to negatively affect patient care,” stated Dr Burstein and colleagues.

In 2016, ASCO issued a policy statement recommending the use of human papillomavirus (HPV) vaccine, to prevent cervical and other HPV-related cancers. ASCO urges oncologists to advocate actively for HPV vaccination.