Rare cancers. Actually they’re called orphans. There are more than 6,000 rare diseases. One in 2,000 will be diagnosed in their lifetime with a condition so rare, there’s no financial incentive to research for a cure. Orphan cancer’s drug development lags behindMy husband was that 1 in 2,000. He was a healthy man who came down with a toothache. It was a squamous cell carcinoma of the maxillary sinus. But, how do we inspire interest in a condition that might strike only every few years?
Steve Kirsch, self made billionaire and Silicon Valley Entrepreneur is inspired the hard way. He was diagnosed with a rare blood cancer. Life expectancy for a man his age is around five to ten years. He’s hoping, as are his three school age kids and his wife, to buy more time by funding research. Only 1500 people a year are diagnosed with Waldenstrom Macroglobulinemia. The cost of developing a successful drug is estimated at $1 billion dollars. No drug company, with its investors in mind, would bother with Steve Kirsch’s condition. Yet, consider this: Of the more than 1.5 million people who will be diagnosed with cancer this year, about a third will be diagnosed with the more common cancers: prostate, breast, colon. The rest–the vast, ironic majority, will be individually diagnosed with cancers like Steve’s or my husband, Will’s. What a powerful constituency they would be. If only they could be heard.
Loading ...
3 responses so far ↓
1 JayMoney // Oct 22, 2007 at 11:26 pm
At What Cost?
The rare cancers (orphans) are going to forever be overlooked. The low numbers of those affected with a particular rare form will be put on the back burner. The cost coupled with the very low numbers compared to the most common forms of cancer will always leave these small number of rare forms on the back shelf.
How do you inspire interest? I think that people closest to those who are affected or have passed have to set up to the table, to create foundations and make a platform where you can discuss these forms of cancer and also discuss possible treatments and hopefully solutions to these diseases. As always in America you are not heard unless you “act a fool” or until some major national figure is afflicted with some rare form of cancer. Then the national spotlight will be turned on and the funding and possible drug makers will jump on board to help with R&D.
One day this county will wake up and stop saying it’s all about me, or it’s all about I, when that happens the word community will take on it’s true meaning “COMMUNITY”.
2 Ron Romeis // Oct 25, 2007 at 10:19 pm
Not all of us are nameless or hopeless. The disease that Mr. Kirsch has developed, Waldenstrom’s Macroglobulinemia (”WM”), is the continuing focus of the International Waldenstrom’s Macroglobulinemia Foundation (”IWMF”) www.iwmf.com, which focuses its contributed resources on research and educatiion. My late wife had breast cancer. I have WM. Cancer survivorship is not a question of diverting resources among cancers, but of recognizing the value of all human life and increasing funding for all cancers’ Cure.
3 Dr. Bill Lloyd // Nov 4, 2007 at 10:13 am
You have all made great points. Here’s something else to consider.
Unlike complex, multifactoral conditions like breast cancer or diabetes, the remarkable narrow spectrum of rare/orphan diseases is that researchers are able to focus on one specific defect responsible for the condition.
The childhood eye tumor Retinoblastoma is a great example. There are only 400 cases every year in America. Why bother? Research in this rare tumor traced the cancer’s origin to a single defect in Chromosome 13. It became the first known tumor suppressor gene, the first human tumor gene to be successfully cloned, and revolutionized our understanding of cancer genetics. Not bad for an ‘orphan’!
The payoff? These advances have been successfully applied to research involving far more ubiquitous conditions like breast cancer, lung cancer, and diabetes.
Forward-thinking decisionmakers can appreciate the genuine value that emerges from diligent research applied to rare/orphan conditions. We just need to keep reminding them!
Leave a Comment