You can help support DSRCT research by purchasing wristbands and stressrelievers through DSRCT Fundraising.
Conducting your own Research
- The Genetic and Rare Diseases Information Center can be reached at 888-205-2311, 888-205-3223 (TTY), or by e-mail GARDinfo@nih.gov.
- National Library of Medicine Gateway - Search numerous journals and articles for information about DSRCT.
- Entrez Pubmed Search for DSRCT - May need to update search
- The Online Mendelian Inheritance in Man (OMIM) - Database contains genetics resources that discuss Desmoplastic small round cell tumor
- 161 Fort Washington AVE
- New York, NY 10032
- Phone: 212-305-1168
- Researching a link between testosterone and DSRCT.
- Phone: 215-662-7910
- University of Pennsylvania Abramson Cancer Center
- Leading a study on DSRCT patients and bonemarrow transplants, to see if they are beneficial or not in fighting this disease. Also doing research with another doctor at the National Cancer Institute for Allo-Transplants.
Institutes and Foundations
- 1315 St. Joseph Parkway,
- Suite 1818
- Houston, Texas 77002
- Phone 713-659-1336
- FAX 713-659-1503
- DSRCT patients are encouraged to send samples of their tumors to the Stehlin Foundation to be a part of the nude mouse program. This research is free of charge for DSRCT patients.
- Tumor samples are implanted onto the backs of nude mice in the hopes that the cells will graft. Experiments are than carried out to determine which anti cancer drugs work best against the individuals cancer. The idea is that if the anit cancer drug works with the 'nude mouse' it will work on the human being too.
Letter from Stehlin:
Sent: Thursday, April 05, 2007 4:11 PM
Subject: RE: DSRCT News
Here is brief update of the DSRCT research.
We have one moderately slow growing DSRCT well established in the nude mouse model, and another one that is extremely slow growing. The speed at which these tumors grow is very important, as it has a strong influence on the rate at which we can perform studies and get results. As with most other human cancers that we have growing in the nude mouse model, the rate at which these cancers grow is relative to the growth rate in the patient. The good news from a clinical perspective is that DSRCT, compared to many other types of cancers, are relatively slow growing. But this means that they are also slow growing in the nude mouse. With some cancers, we may be able to grow enough cells in 3 to 4 months to initiate an experiment, and then in 8 to 12 weeks start analyzing the results. With the slow growing DSRCT, it has taken over a year to grow enough material to initiate our first experiment. I give you this background again just so you are aware of what we are up against.
We have now performed two studies, and we are very excited about the results. In one of the experiments we used a metronomic treatment approach. In this approach, the goal is not necessarily to eradicate the cancer, but to keep it in check. If effective, the low dose chemotherapy keeps the microvasculature from being established, and therefore restricts the nourishment that the cancer cells receive. The advantage to this type of therapy is that since the dose of chemotherapy is very low, it has no or little toxicity.
In the other experiment, we used our newest and most promising CPT derivative. We have been developing CPT compounds since 1989, and we think these have tremendous potential. CPT comes from a common tree, camptotheca acuminate. Our latest derivative shows not only to be a very effective anti-cancer agent, but of tremendous importance, as yet we have not been able to detect ANY toxicity with its’ use. In treating the DSRCT tumor in this second study with what we consider a moderately low dose, we achieved a near complete eradication of the tumor in less than three months of treatment. This dose could easily be increased by 4 or 5 times (or more) without any toxicity if necessary to achieve total eradication.
Here is my ‘disclaimer’. Yes, we are very excited about both of these studies. But these are only two studies. We need to reproduce these results, and test other patients’ tumors. As far as pushing the CPT derivative, we are on course to petition the FDA to start clinical trials with this derivative the first part of 2008. This is provided that everything continues to go according to our schedule. The filing for a clinical trial is an enormous financial undertaking and requires a vast amount of research data. We started this process almost a year ago. Please know that we are doing everything we can with the resources we have to push the DSRCT project.
- B. NIDDK, National Institutes of Health
- Sean Bong Lee, Ph.D.
- Genetics of Development and Disease Branch
- Building 10, Room 9N313
- Bethesda, MD 20892
- Email: SeanL@intra.niddk.nih.gov
- B.S., SUNY at Buffalo, 1989
- Ph.D., SUNY Health Science Center at Brooklyn, 1994
Research Statement (focus of research is predominately Kidney related, not DSRCT related. But DSRCT could benefit indirectly.)
The primary interest of this laboratory is to understand how perturbations during normal development process can lead to cancer. In particular, Wilms' tumor, a childhood kidney cancer, serves as a paradigm for studying development and cancer in our laboratory . . . . .
- Current treatment for DSRCT is minimally effective and the pathways that are responsible for generating the tumor are largely undefined. To identify the mechanisms of tumorigenesis in DSRCT, we are attempting to generate a mouse model of DSRCT by expressing the EWS/WT1 gene product in mouse embryonic stem cells. The mouse model, if successful, will then be used to dissect molecular pathways that are responsible for the formation of DSRCT, which can lead to the development of new therapeutics against DSRCT-specific molecular targets. - From Intramural Research conducted at The National Institute of Diabetes and Digestive and Kidney Diseases