Progress Report 2012

Alan Rosenthal Research Fund | Memorial Sloan-Kettering Cancer Center

Solitary fibrous tumor (SFT) and Hemangiopericytoma (HPC), once considered separate entities, were recently merged into one disease type based on their identical appearance and characteristics. This cancer may develop virtually anywhere in the body, and fifteen to twenty percent of patients progress with either local recurrence or distant metastases. What drives this malignancy to spread is not well-understood, as no recurrent genetic defects have been identified to date. As a result, we have no effective therapies to target the cancer's fingerprint -- and unfortunately, long-term prognosis for patients with advanced disease remains poor.

Thanks to a generous gift from the Alan Rosenthal Fund for Research in Sarcoma, we studied the genetic expression of twenty-three SFT banked tumor samples from various sites throughout the body. Next, we conducted a detailed pilot study on six matched tumor/normal samples selected from the initial twenty-three. We discovered five critical points:

  1. SFT showed a distinct genetic signature when compared to all other sarcomas. All SFTs uniformly overexpressed three FGFR1, FGF2, and DDR1. This is extremely promising, as it identifies potential targets for therapeutic intervention - i.e., SFT and HPC patients could be given a cocktail of specific enzyme blockers.
  2. SFT showed universal over-expression of IGF2, a growth-promoting hormone.
  3. SFT expressed high levels of TEM-1/Endosialin, which is a newly-discovered marker for tumor angiogenesis -- or blood vessel growth. These vessels act as the lifeline for cancer's growth and spread.
  4. No genomic subgroups emerged when looking at tumors from different anatomic sites. For example, historically meningeal tumors were considered different from soft tissue sites. Our data demonstrated that the genetic signatures of the tumors are the same, regardless of location, and that observed differences are based only on tumor grade and stage.
  5. In studying the six matched pairs of SFT tumors, at least thirty potentially relevant mutations were detected.

Looking ahead, our next steps are to:

  1. search more extensively for genetic drivers of this disease;
  2. validate the mutations detected during our pilot study; and
  3. screen candidate genes in a larger sample size of SFT patients. In particular, a TEM-1 inhibitor is now in phase I clinical trial in solid tumors, and has shown response in a patient with metastatic SFT. We would like to test this drug against fifty SFT tumor samples to see if this positive response is repeated.

Ultimately, our research team's mission is to pinpoint the drivers of SFT and HPC, so that new, more effective, least-invasive therapies can bring patients better length and quality of life. This work is highest priority: few therapeutic options exist, and those available offer little in the way of sustained remission. Philanthropy has been vital to our efforts: the support of the Alan Rosenthal Fund for Research in Sarcoma has allowed us the freedom to move quickly on leads, and to test promising theories as they arise. We remain grateful for your kind support.


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.
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