Desmoids are benign but aggressively invasive tumors. No treatment is 100% successful and there is no known cure. Until most recently there has been very little research done in the United States on Desmoids, and there was not even a foundation or organization to support such research or even to help those diagnosed with Desmoids. Current treatment is surgery (often removing massive amounts of tissue and a new Desmoid will often recur in the scar tissue), chemotheraphy, or amputation. Desmoids strike all ages and genders and patients do sometimes die from complications of these tumors.

A petition is being sent to legislators advocating that a FDA guidance (a special document which helps open up new avenues in clinical research) be instilled for sarcoma research.

More information follows. Please consider writing the FDA and your legislators.

Forwarded from the Desmoid Tumor Research Foundation (sample letters follow, including one for use by patients):

I am asking each of you to write to your congressional representatives to ask for their support of a Citizen Petition that is important to all patients with rare diseases like desmoid tumors.

Here’s the background: It is difficult for researchers to get FDA approval of clinical trials in rare cancers because of requirements that studies must have hundreds of patients, in addition to other limitations. The FDA has been very inconsistent and unpredictable in the approval process for rare cancers. For example, a recent drug for osteosarcoma was denied approval despite demonstrating efficacy without toxicity.

Under its current authority, the FDA can issue a Guidance Document that would clarify the pathway that a pharmaceutical or biotech sponsor would follow to gain approval for a product for use in a rare disease population. An FDA guidance document would help facilitate research in rare sarcomas, including desmoid tumors.

A Citizen Petition requesting the issuance of a guidance document for rare cancers research was submitted to the FDA in October 2007. We need to ask our congressional representatives to write to the FDA in support of this Citizen Petition.

(For your information, letters of support of the Petition have already been sent to the FDA by Congressman Israel, Towns, Kennedy, Cooper and Congresswoman Valezquez.)

Please send this sample letter below to your congressmen and senators to help move important research forward! You can find their addresses here. Thank you!

Best wishes,
Marlene
(Director of the Desmoid Tumor Foundation Research)

Commissioner Andrew C. von Eschenbach, M.D.Food and Drug Administration
5600 Fishers Lane
Rockville, MD 20857

Dear Dr. von Eschenbach:

I am writing in strong support of the Citizen Petition submitted by The Alliance Against Alveolar Soft Part Sarcoma (“TAAASPS”) and the Sarcoma Foundation of America (“SFA”) (FDA docket number 2007P-0420). The TAAASPS and SFA Citizen Petition requests that FDA issue a guidance document for the accelerated approval of drugs and biologics that are intended to treat rare cancers. I believe the Citizen Petition merits the Agency’s careful consideration and FDA should move forward to issue the requested guidance document.

The Citizen Petition asks the Agency to provide guidelines for the approval of any drug or biological product that is intended to treat a rare cancer, but for which large size studies may not be feasible due to the rarity of the cancer or ethical constraints in conducting such studies. As the Citizen Petition points out, rare cancers affect a small percentage of the U.S. population. Because of the rarity of the cancer, clinical trials, especially those requiring the sponsor to demonstrate a survival benefit, are often difficult to conduct and complete. However, under the Agency’s current approval process, such studies are usually necessary to the approval of the product. As a result, drug companies typically avoid the risk of development, and many people affected by rare cancers are left without a viable therapeutic treatment option. By providing a guidance document concerning the realistic pathway to approval of treatments for rare cancers, FDA can pave the way for companies to continue to develop and seek approval of products intended to treat rare cancers, and to ensure that these products reach patients who desperately need them.

I urge the Agency to act on the Citizen Petition’s request and provide needed guidelines for the approval of drug and biologics intended to treat rare cancers. These guidelines can help preserve and promote public health by making products available to persons suffering from rare cancers.

SAMPLE LETTER FOR PATIENTS:
Sample letter to send to your Congressman / Senators:

Dear ___________:

I am a patient with a rare sarcoma called desmoid tumor. There are few patients with this disease. This makes clinical research difficult with the present restrictions such as the requirement of procuring a large population of patients before a clinical trial can be approved.

We need your help to encourage the FDA to facilitate research in rare cancers! I am writing you to ask you to please send the following letter to the FDA in support of the Citizen Petition submitted to the FDA in October 2007.

Thank you,
[your name and address here]