Sarcoma is a terrible but rare group of diseases that disproportionately affects our children and adolescents.
Although chemotherapy has greatly improved prognosis for patients with rhabdomyosarcoma, Ewing sarcoma, and osteosarcoma, for the majority of patients with other subtypes of sarcoma, prognosis is quite dismal, especially after metastasis is detected.
Fortunately, a small group of scientists and clinicians across the United States are dedicated to finding better treatments for patients with this disease. Thanks to persistent scientific and clinical research, the last decade introduced several new chemotherapeutic agents that can improve the outlook of patients with metastatic disease. However, there is still so much more to do.
Sarcoma Cancer research does not receive adequate federal or private funding. By raising awareness and providing financial support to brilliant and dedicated doctors, new research and clinical trials can be accomplished.
We are a 501(c)(3) charitable organization. We strive to make a difference, and help to find a cure.
July 19, 2023
Dana-Farber Cancer Institute
Dr. Kimberly Stegmaier
April 12, 2022
Integrative Therapies Program
Mass General Cancer Center
Ewing's Sarcoma Research
Dr. Timothy Chan
Cold Spring Harbor Laboratory
Dr. Christopher Vakoc
$15,000 to Mass General Hospital
Dr. Edwin Choy
$5,000 to Caring For a Cure
$15,000 to Cold Spring Harbor Laboratory Sarcoma Research Program,
Dr. Chris Vakoc
$10,000 to Mass General Cancer Center,
Dr. Edwin Choy
$10,000 to Cleveland Clinic,
Ewing Sarcoma Research,
Dr. Jacob Scott
July 15, 2023
July is Sarcoma Cancer Awareness Month
We are proud to announce the donation of $10,000 to Dr. Kimberly Stegmaier at Dana-Farber Cancer Institute, Boston, Ma. The Stegmaier lab, is leading the way to help find a better way to treat and cure Sarcoma cancer.
The Stegmaier laboratory has a focus on Ewing sarcoma, the second most common childhood cancer involving the bone. While much progress has been made in treating patients with localized Ewing sarcoma over the last two decades, little progress has been made for patients with metastatic or recurrent tumors. Stegmaier laboratory seeks to identify new targets/drugs for patients with high-risk disease and to understand the molecular origins of this childhood solid tumor.
Ewing sarcoma is driven by a cancer-promoting protein known as a fusion oncoprotein. Part of one chromosome abnormally fuses with another chromosome resulting in the production of an abnormal protein. In the case of Ewing sarcoma, the most common fusion oncoprotein is known as EWS-FLI1. Stegmaier lab has also determined that Ewing sarcoma cells need a precise amount of EWS-FLI1. Too much or too little of the protein, the so-called Goldilocks phenomenon, is damaging to Ewing sarcoma cells. One major effort in our laboratory is to better understand the function of the EWS-FLI1 protein and working closely with chemistry collaborators to identify drugs that inhibit or degrade EWS-FLI1 or drugs that increase EWS-FLI1 to a toxic level in the cancer cells.
A second major strategy used by our laboratory is to identify other Achilles heels in this disease. The lab is using a state-of-the-art genome-editing tool called CRISPR that allows us to knock out each gene in the genome and to assess the impact on Ewing sarcoma and other cancer cells. With the Broad Institute, they have screened a large collection of Ewing sarcoma cell line models, systematically knocking out each gene in the genome, one by one, to identify those necessary for Ewing sarcoma cell survival. The laboratory has identified several exciting candidate genes and aims now to better understand why Ewing sarcoma cells depend on those genes for survival and to identify chemicals to inhibit these potential drug targets.
Finally, a third strategy has been to systematically screen Ewing sarcoma cells against collections of drugs and to compare the response of Ewing sarcoma cells to that of hundreds of other cancer cells. Here, they have identified a unique collection of drugs that are highly active in Ewing sarcoma compared to these other cancers. The Stegmaier Lab is now hard at work to understand why Ewing sarcoma cells are so exquisitely sensitive to these chemicals and to determine whether any of these drugs can be translated to the clinic.
Amazing work Dr. Stegmaier!
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