Finding experimental treatments for your disease is not as difficult to do as you might think. As a determined patient who takes charge of your own health care, effectively teaming with your physicians and other medical professionals, you might be able to find a phase I clinical trial at clinicaltrials.gov that involves a group of patients receiving an experimental treatment for your disease and a control group that is receiving a placebo (a medication that has no active ingredients).
Such trials are typically double-blinded. That means neither the participants nor those doing the study are aware of which patients are receiving the active medication and which are receiving the placebo. If your disease is life-threatening or in its final stages, you might be able to enter the clinical trial if you qualify. But what if you end up in the control group that receives the placebo? There is a 50 percent chance that even if you got accepted into the study, the treatment you received would be of no benefit whatsoever.
As a determined patient, you might want to talk to your doctor about receiving the experimental treatment. It may be possible for your doctor to get a hold of it and administer it to you, allowing you to receive the benefits if indeed the medication does turn out to be effective.