Reading the review paper on NET research that I summarized here yesterday, I was struck by how many of the drugs under study have already been approved for other cancers and diseases. It is something I’ve known for a long time, but reading the full listings yesterday really brought it home to me what it means to be an orphan disease.

What an orphan gets

Like human orphans, orphan disease patients too often end up with the hand-me-downs and the leftovers. Even octreotide, the NET standby, was developed as a way to help chemo-therapy patients tolerate the side-effects of their treatments. That it happened also to have an impact on NET carcinoid syndrome symptoms and slowed the progression of that disease was little more than a happy accident that boosted the drug maker’s bottom line for the drug.

But from a drug company’s point of view there is no real profit in developing drugs whose initial purpose is to deal with NETs. Drugs for NET are not a multi-billion dollar market–at least not yet.

And neither drug companies nor the federal government have much interest in funding research on anything they are not virtually certain will work before the first experiment they pay for is run. For a drug company, an experiment that does not lead to a profit fairly quickly is not one they are overly interested in funding.

Government, too

The federal government’s interest in nearly certain success comes from a concern over being seen as spending taxpayer dollars foolishly. NBC’s Fleecing of America stories sometimes bother me because they can be overly critical of government spending on pure science. Senators, congressmen, and reporters all seem to want every dollar to have an immediate return. Having hung around with science and engineering folk for most of my life, I know that an immediate concrete return on research and development spending does not always happen.

Without events like the Pan-Mass Challenge, the WEEI/NESN Jimmy Fund Radio-Telethon, and the Jimmy Fund Marathon Walk the money to do the fundamental medical research neither drug companies nor governments will fund would not happen. The lines of NET cells now growing in vitro would not exist without that money. The progress made this year toward an animal model for NET would not exist without that money. The NET patient genome study currently underway would not exist without that money. Our knowledge of which receptors to target would not exist without that money.

We can do better

The Dana-Farber Cancer Institute is like every other cancer research center in the country. They all have events like the Marathon Walk to provide them with the money they need to do the research that has to be done before government or private enterprise will get involved.

Our Marathon Walk team is designed to generate that seed money from which a strong Program in Neuroendocrine and Carcinoid Tumors can grow. But we need your help, both as donors and as walkers if we are going to get NET out of the orphanage.