Editorial

Recent Support for Sarcoma Research

I’d like to report on what several organizations have done in the last few months to support sarcoma research. From my perspective, the entire sarcoma community is in their debt for what they have accomplished. They are excellent examples of why I often end my editorial with the phrase, “Together we are stronger and together our voices are louder.” I apologize for the length of this editorial, but as you will see, there is much to say.

Miles Alpern Levin Initiative for Rhabdomyosarcoma Research Commits $1 Million for Rhabdomyosarcoma Research

The newly established Miles Alpern Levin Initiative for Rhabdomyosarcoma Research is funding research into the development of new treatment options for patients with recurrent rhabdomyosarcoma (RMS). The Initiative has been funded through a $1 million commitment in memory of Miles Alpern Levin, thanks to the generosity of Dr. Nancy Alpern Levin and her parents, Robert and Marjorie Alpern. It will support the collaboration between Dr. Leonard H. Wexler of Memorial Sloan-Kettering Cancer Center's Department of Pediatrics and Dr. Neal Rosen of the Sloan-Kettering Institute (SKI).

RMS is a fast-growing tumor which accounts for about half of the soft tissue sarcomas in children. Sarcomas are cancers that develop from connective tissues in the body. RMS is a cancer made up of cells that normally develop into skeletal muscles. Any muscle in the body may be involved. For patients whose tumors recur or whose tumors have spread to other areas of the body, Memorial Sloan-Kettering is leading efforts for innovative treatment strategies, including novel combinations of chemotherapy agents, radiation therapy, and surgery.

Dr. Wexler is an internationally recognized expert on the treatment of rhabdomyosarcoma and Dr. Rosen is a premier translational researcher with special knowledge in the area of mTOR pathway inhibitors. By collaborating, these researchers are seeking to develop new, biologically targeted therapies for recurrent rhabdomyosarcoma. The most promising of these approaches is targeted therapy against the Type 1 Insulin Growth Factor Receptor (IGF-1R) and its downstream effector pathways. Preclinical research has shown that this agent is singularly effective against RMS, and plans for the drug's continued development would be to combine it with a mTOR pathway inhibitor, or with chemotherapy, or both, and to begin planning for clinical trials. This is brand-new territory in therapeutic drug discovery, and it is hoped that the collaboration between Drs. Rosen and Wexler will offer new treatment options to patients and generate considerable excitement in the pediatric sarcoma medical community.

The late Miles Alpern Levin, son of Dr. Nancy Levin and Mr. Jonathan Levin, and a grandson of the Alperns, inspired the family to make this philanthropic commitment though his own battle with rhabdomyosarcoma, to which he succumbed on August 19, 2007. Since he was first diagnosed with RMS in 2005, the 18-year-old wrote about his experiences as a cancer patient and published the pieces through a web blog. Mr. Levin reached thousands of online subscribers worldwide with thoughtful musings on life and hopeful messages such as "Keep Fighting, Stop Struggling."

ASCO Foundation Advanced Clinical Research Award (ACRA) in Sarcoma Established.

The Sarcoma Foundation of America announced that it is supporting a $450,000 Advanced Clinical Research Award (ACRA) through The ASCO Foundation. "We are especially proud of the ACRA grant, which was made possible through a partnership with the Capon Family," said Mark Thornton, M.D., Ph.D., President, Sarcoma Foundation of America. "We believe that by privately funding a long-term research grant of this magnitude, we can attract high caliber researchers who are interested in and capable of pursuing a significant sarcoma research project. It is by supporting the work of dedicated scientific researchers, we will eventually find a cure for sarcoma. It also demonstrates to those of us who are part of the sarcoma community that dedication of a small number of people united for a cause can truly make a difference!"

"Approximately 11,000 new cases of sarcoma will be diagnosed in the United States this year, and roughly 5,500 people will die of the disease. We need to find new and better ways to prevent, diagnose, treat and enhance the quality of life for people living with sarcoma. To that end, we are pleased to announce the Sarcoma Foundation of America and Capon Family will fund the first ASCO Foundation Advanced Clinical Research Award (ACRA) in sarcoma. This award is a vital component to the development of new therapies and progress against sarcoma," said Joseph S. Bailes, M.D., Chair, ASCO Foundation Board of Directors.

National Leiomyosarcoma Foundation sponsors LMS Research

The National Leiomyosarcoma Foundation of Maryland, New York gave a $70,000 grant to a research team at the Bassett Research Institute to study the cancer's response to melatonin. "We're going to do the first experiments in a way just to see if melatonin has some activity in inhibiting cancer," said Dr. Blask. "we will be able to use the results of the study, If it turns out to be positive, to justify a larger study that would actually involve treating people who have LMS."

Ed Hoag of the National Leiomyosarcoma Foundation, said that the foundation's work is in memory of his late wife, who died of LMS. "She wanted the awareness out there," Hoag said. "That this disease is there, and we need to do something with it." "It's giving everyone that's been diagnosed with this disease another chance," said Lori Hoag of the Foundation. "A sense of hope to think that there's another researcher out there willing to fight for our cause."

Cure Alveolar Soft Part Sarcoma International Augments Research Project Funding

iCureASPS (Cure Alveolar Soft Part Sarcoma International) adds over $34,000 in additional support to existing and new ASPS research projects at the Dana Farber Cancer Institute: (1) the GVAX clinical trial; (2) the creation of an Alveolar Soft Part Sarcoma tumor transplant in mice; and (3) the Identification of Kinase Inhibitors that target ASPS. For more information, visit the iCureASPS website.

The Chordoma Research Foundation and the Chordoma Foundation

The Chordoma Research Foundation (CRF) was started by Johnny Nelson, and his parents Bruce and Lynette of Clifton New Jersey, after Johnny was diagnosed with a clival chordoma in 2004, during his freshman year of college at the University of Maryland. Through CRF, Johnny and his parents raised over $50,000 with the goal of funding chordoma research projects and the creation of a chordoma BioBank.

Immediately after the Chordoma Foundation was granted 501(c)(3) tax-exempt status in October 2007, the Nelson’s generously directed all CRF funds to support the work of the Chordoma Foundation. Additionally, the Nelson’s have personally pledged $75,000 to match all funds raised by the Chordoma Foundation before Dec 31, 2007, essentially doubling the impact of every donation. These substantial contributions go a long way towards the $500,000 that the Chordoma Foundation hopes to raise by April 2008 in order to establish a BioBank, develop cell lines, create animal models, and host the Second International Chordoma Research Workshop. These two organizations shared a common mission and we are glad to be able to join forces in our search for a cure. The Nelsons have now closed CRF and have become actively involved with the Chordoma Foundation.

Desmoid Tumor Research Foundation Announces Its 2007 Grant Awards

Matt van de Rijn, MD at Stanford University: ROR2, a potential novel therapeutic target in desmoid tumors. This is a new grant for a three-year project studying Receptor tyrosine (RTK's) which are proteins that are expressed on the surface of cells and are known to play an important role in cellular differentiation and tumor growth. ROR2 is a RTK which is expressed at high levels in a significant subset of desmoid tumors.
David E. Joyner, PhD et al, Huntsman Institute, University of Utah: Do Factors Mediate Desmoid Tumor Invasiveness and Drug Sensitivity? This is the second year of a two-year study. Desmoid tumors can grow rapidly and be highly invasive or they can remain static for many years. These investigators are studying whether the proteins EGF (epidermal growth factor) and TGF-alpha (transforming growth factor) influence the invasive nature of desmoids and if either factor determines desmoid drug sensitivity.
Benjamin A.Alman, MD et al, Hospital for Sick Children: Identifying the Desmoid Initiating Cell. This is the second year of a three-year study. A small portion of stem cells in a given tissue give rise to all of the cells that make up that tissue. These cells help regenerate and maintain the tissue over its lifespan. These stem cells have been identified in a variety of tumor types and are called tumor initiating cells (TIC). These investigators will be testing the hypothesis that desmoid tumors contain a subpopulation of TIC and that desmoid tumors are derived from mesenchymal stem cells (MSC) that have a mutation leading to elevated levels of beta catenin, a protein thought to be instrumental in the development of familial adenomatous polyposis along with a mutation in the APC gene.
Dina Lev, M.D. et al, MD Anderson Cancer Center, University of Texas: Molecular Determinants of Desmoid Tumor Development and Progression. This is the second year of a three-year study. These investigators will be studying three molecular factors believed to be important in desmoid tumors: beta catenin, TCF-3 (a transcription factor) and ER-beta (an estrogen receptor).

I am sure there are many more examples that I am not aware of, but these are the ones that I received notes about during the past few months. I found them inspirational examples for us to follow.

Best,

Bruce

Bruce D. Shriver, PhD

Editor-in-Chief, ESUN