INTRODUCTION

    Because ASPS is such a rare disease, even when the point comes that promising agents come along, it is going to be difficult to perform ASPS specific clinical trials.  But we will cross that bridge when we come to it.

    In the meantime, we need to be on the lookout for any sign that any agent that is currently being developed for cancer might have activity against ASPS.

    If such a drug comes along, it will no doubt not be in clinical development for ASPS patients.  It will most likely be in development for breast, colon or some other type of cancer, and may "happen" to work in ASPS.

    If this ever becomes the case (and such a breakthrough could happen any time, given the breathtaking pace of recent new drugs against cancer (for example, Herceptin, Gleevec and Erbitux), we in the ASPS community must be ready to act!

EXPANDED ACCESS

    The U.S. Food and Drug Administration has developed new regulations that are making it easier for individual patients to get access to promising drugs that have not yet reached the market.  In addition, there are patient lead advocacy groups such as the Abigail Alliance for Better Access to Developmental Drugs and the Cancer Action Now Foundation, who are fighting for our rights to have access to these promising drugs.

    EXPANDED ACCESS GOALS OF THE ALLIANCE AGAINST ALVEOLAR SOFT PART SARCOMA

    At such time that a drug under development is found to have promising activity in ASPS, or whether there is a theoretical chance that an agent would show promise in ASPS, the following steps are necessary:

• Identify an oncologist willing to sponsor a "Single Patient Investigational New Drug (IND) Application" with the FDA.
• Make contact with the pharmaceutical company or biotechnology company involved with the development of the drug.  Here are a couple of websites to get you started: Pharmacy.org and Cato.com.
• Work with the oncologist to create a brief protocol about how the drug will be used in ASPS (probably the same dose and regimen that is showing promise in the main cancer type for which the drug is being developed).  A lot of information about this process can be found at:  www.fda.gov/cder/cancer/singleind.htm.
• Work with the drug company to establish a mechanism to supply the drug to your oncologist.
• Work closely with both the oncologist and the drug company to cooperate with any information and tests they may need to provide you with the promising drug.