Progress Report 2017

Alan Rosenthal Research Fund | Changing the Lives of People with a Rare Sarcoma

Soft tissue sarcomas are relatively rare tumors that arise in the fat, muscles, nerves, tendons, or blood or lymph vessels. They are often curable with surgery. But for people with tumors that cannot be surgically removed or come back after surgery, there are few treatment options, because sarcomas typically do not respond well to traditional chemotherapy. Moreover, they are a complex and highly diverse group of cancers: There are more than 80 different sarcoma subtypes, each with its own treatment strategy and challenges.

Targeted therapies, which work by taking advantage of genetic alterations in tumor cells, hold promise for bringing sarcomas under control. The challenge is to identify the molecular changes driving the development and growth of each subtype, and to identify or create anticancer drugs that work by targeting those alterations. Memorial Sloan Kettering Cancer Center (MSK) is a leader in this area, with a database that combines tumor samples from patients with clinical information such as their age at diagnosis and details about their tumors and treatments.

Solitary fibrous tumors (SFT) are soft tissue sarcomas that develop in the blood vessels. Once more commonly known as hemangiopericytomas, these tumors represent only 3 percent of the soft tissue sarcomas diagnosed at MSK. They are an area of research focus for Aimee Crago, MD, PhD, a surgeon who specializes in the care of people with soft tissue sarcomas. The goal of her studies is to analyze the roles of specific genetic mutations in SFT development and growth. Through the creation of new laboratory models, she and her team hope to use this information to identify novel markers to predict a patient’s outcome and find new targets for therapy that could improve patient care.

Evaluating SFT Mutations

Dr. Crago and her colleagues are developing systems to analyze a mutation called NAB2-STAT6 that is involved in SFT. It is a “translocation”—a genetic error that happens when two normal genes trade places, and neither works properly. They are using MSK-IMPACT™—a technology invented here that detects genetic mutations in cancers, from the most common to the rarest—to find other genetic errors involved in SFT. MSK is a recognized leader in precision oncology: identifying genetic mutations that cause an individual’s cancer, and matching the patient with the most appropriate drugs that target these mutations.

MSK-IMPACT testing showed that genes called TERT promoter and TP53 and the PI3 kinase pathway are involved in 42 percent, 20 percent, and 14 percent of SFT cases, respectively, and warrant further study as potential therapeutic targets. SFT patients with the TERT promoter also have poorer outcomes.

Looking Ahead

Soft tissue sarcomas are rare tumors, and SFTs are even rarer. It takes a team like Dr. Crago’s and resources such as those at MSK to devote the time and energy necessary to learn more about these challenging cancers and to amass knowledge that can lead to meaningful changes in patient outcomes. The results of Dr. Crago’s investigations can be used to identify new drugs for SFTs and to predict which patients may benefit most from specific treatments. Kindly consider supporting this endeavor, which has extraordinary potential for improving the futures of patients with SFT and other sarcomas not only at MSK, but around the world.


Please join us in support of this important research, as you will be providing the means for Dr.Mary Louise Keohan and her team to conduct pioneering cancer research.

You can contribute by clicking here or by calling 646-227-2992

To be sure your gift goes directly to Hemangiopericytoma research, make certain that you address your gift to the Alan Rosenthal Research Fund.
All gifts regardless of size really do make a difference. MSK is a tax exempt organization........a 501(c)(3)