Some MPNs, MDS, and other cancers are "orphan" cancers. In the U.S., the Orphan Drug Act refers to treatments for diseases that affects fewer than about 1 in 1,500 people. The act was designed to encourage research and development of treatments for orphan diseases. The thinking behind the act is that the market for an orphan drug treatment is so small that companies wouldn't bother with the research and development needed to produce a treatment. The Orphan Drug Act has stimulated more treatment options, but it has not done much to keep the cost of newer treatments affordable.

The American Hematology Society (ASH) has a big conference every December where pharmaceutical companies and blood cancer researchers and clinicians meet to discuss all types of cancer. If you look at their reports (Dr Mesa at Mayo used to give updates on MPN news from ASH), you will see lots of discussion on orphan blood cancers.

One problem that makes treatment for rare blood cancers is diagnosis. GPs often see abnormal blood counts but aren't sure what they're looking at. Mine went undiagnosed for probably about 5 years because symptoms seemed more age related and platelets were only in 500s. In addition, the tools to diagnose the problem sometimes need improvement. Before genetic mutations could be determined, thrombocythemia was diagnosed by process of eliminating which diseases it wasn't. Now diagnosis is quicker and more precise.

I realize none of this addresses the frustration patients feel. But it may help to know that there is lots of movement behind the scenes, doctors do care, but diagnosis and treatment methods take a long time to come to fruition and to become affordable.

Hang in there, everyone! If you have a rare cancer, you might ask your doc if he can forward your patient info to a research center. Collecting data pools with as many patients as possible helps researchers spot patterns and pursue new lines of research.