Two recently published genetic studies have the potential to advance treatments for small cell lung cancer (SCLC). This aggressive form of lung cancer, described by the studies’ authors as in urgent need of new drug therapies, has seen no treatment advances in 30 years.

“In small cell lung cancer, which impacts about 30,000 to 40,000 people each year in the United States, there has been no therapeutic progress and very little research,” said lead author Afshin Dowlati, MD, Director of the Center for Cancer Drug Development at University Hospitals (UH) Case Medical Center Seidman Cancer Center in Cleveland, Ohio. “Additionally, there are no approved targeted therapies for the disease. These studies lay the foundation for future research aimed at finding important new treatments for this highly malignant cancer.”

A new mutation in SCLC may play a role in the disease’s development. The researchers found that the genetic mutation, called RET (rearranged during transfection), was linked to rapid cell growth. These findings were described in the Journal of Thoracic Oncology (2014; doi:10.1097/JTO.0000000000000234).


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The research team examined specimens in its database of metastatic SCLC tumors, which is one of the largest databases of its kind in the country. They found that the RET mutant protein was potentially linked to faster-growing cells that were sensitive to ponatinib and vandetanib, two new targeted drug therapies. Future clinical trials for patients with the RET mutation are planned to further validate the data.

“We were encouraged to find that these two cancer-fighting therapies are potentially effective at stopping cancer cell growth in certain small cell lung cancers,” said Dowlati, who is Professor of Medicine–Hematology/Oncology, Case Western Reserve University School of Medicine, and a member of the Case Comprehensive Cancer Center. “These findings have the potential to give cancer physicians a new tool to more effectively tailor treatments for patients.”

The other study, published in PLoS One (2014; doi:10.1371/journal.pone.0106784), outlines a novel approach to identify new drug therapies for SCLC based on tumors’ genomic profiles. The research team discovered new molecular targets for treating the disease by extracting data from SCLC tumors to examine drug sensitivity. Through this analysis, they outlined a promising new approach to predict which cancer-fighting drugs would be the most broadly effective at slowing tumor growth.

“This study enabled us to identify which drugs may be the most useful in which types of tumors,” said Dowlati. “Small cell lung cancer is one of the fastest growing cancers and these studies have yielded small but important therapeutic insights into this disease.”