Early Results Suggest New EGFR Targeted Therapy Shrinks Worsening Lung Cancers with Fewer Side Effects

ASCO Annual Meeting
May 14, 2014

In a recent phase I clinical trial, about 50% of patients receiving a new targeted therapy for worsening non-small cell lung cancer (NSCLC) had the cancer shrink. Targeted therapy is a treatment that targets the cancer’s specific genes, proteins, or the tissue environment that contributes to cancer growth and survival. Specifically, this new targeted therapy, AZD9291, targets changes or mutations to the epidermal growth factor receptor (EGFR).

In a healthy cell, EGFR allows cells to grow and divide as needed. However, some cancer cells have a mutation that results in too many receptors, allowing the cancer to grow uncontrollably. About 10% to 15% of white patients and about 40% of Asian patients with NSCLC have changes to EGFR. In the United States, this means about 18,000 people diagnosed with lung cancer each year have an EGFR mutation. Other EGFR targeted therapies are available for lung cancer, but for most patients with EGFR mutations, eventually these drugs stop working, often within 10 to 14 months.

Unlike other drugs that target EGFR, this study showed that AZD9291 worked well for patients who developed the most common mutation, T790M, that causes other EGFR targeted therapies to stop working. Currently, the only treatment option for NSCLC with this type of mutation is a combination of two different EGFR targeted therapies that causes severe side effects.

The 199 patients who participated in this study had NSCLC with EGFR mutations that continued to grow or spread after receiving one or more of the currently available EGFR targeted therapies. As part of this study, they received different doses of AZD9291.

Overall, the cancer shrank for 51% of patients participating in the study. Patients receiving each of the different doses experienced some level of tumor shrinkage, even those with cancer that had spread to the brain. For the 89 patients in the study with the T790M mutation, 64% had the cancer shrink or stop growing, compared with 23% of those without this mutation.

AZD9291 also appears to cause fewer side effects than other drugs that target EGFR because it specifically targets the changed EGFR in the tumor, whereas other EGFR therapies target both the changed EGFR in the tumor and the normal EGFR in the skin and other organs, causing side effects such as severe and uncomfortable rashes.

What this means for patients

“There is currently no standard treatment for patients whose lung cancer worsens after initial treatment with an EGFR targeted therapy,” said lead study author Pasi A. Jänne, MD, PhD, a professor of medicine at Dana Farber Cancer Institute and Harvard Medical School in Boston, MA. “Although it is still a bit early, our study suggests that AZD9291 may offer an effective new therapy option for these patients, without the skin side effects we typically see with existing EGFR therapies.” AZD9291 is still being studied and is not currently available outside of a clinical trial. Talk with your doctor about clinical trials as an option when making treatment decisions.

Dr. Jänne was a recipient of a Conquer Cancer Foundation of ASCO Young Investigator Award in 2001.

Questions to Ask Your Doctor

  • What type of lung cancer do I have? What does this mean?
  • Does the cancer have EGFR or other mutations that contribute to cancer growth?
  • What are my treatment options?
  • If the cancer has worsened, what other treatment options are available?
  • What clinical trials are available to me?
  • What treatment option do you recommend? Why?

More Information

Guide to Lung Cancer

Targeted Treatments

Explaining Cancer Genome Research

EGFR Testing for Advanced NSCLC

Skin Reactions to Targeted Therapies