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Niche Science And Targeted Medicines Vs. “Magic Bullets”

Maybe you read the other day in The New York Times that the pharmaceutical industry has a problem. Big blockbuster drugs like Lipitor are going off patent and the industry leaders don’t have new blockbusters showing promise to replace them. So the big companies search for little companies with new discoveries and they consider buying them. Industry observers think the days of $5 billion-a-year drugs to lower cholesterol or control diabetes may be past for awhile, and the companies will have smaller hits with new compounds for autoimmune conditions and cancer.

When I saw my oncologist for a checkup yesterday — the news was good — we chatted about the article and the trend toward “niche science.” We welcomed it. We didn’t think — from our perspective — the world needed yet another drug to lower cholesterol. We need unique products to fight illnesses that remain daunting, some where there are no effective drugs at all. For example, my daughter has suffered for years from what seems to be an autoimmune condition called eosinophilic gastroenteritis (EGID). Her stomach gets inflamed with her own eosinophil cells. They would normally be marshaled to fight a parasite in her GI tract but in this case, there’s nothing to attack. So the cells make trouble on the lining of the stomach and cause pain and scarring. Right now, there’s no “magic bullet” to turn off these cells. My hope is some pharma scientists will come up with something to fill this unmet need.

In the waiting room before I saw my doctor at the cancer center in Seattle I overheard a woman on the phone speaking about her husband’s new diagnosis of pancreatic cancer. I was sitting at a patient education computer station nearby. When she was finished I introduced myself and showed her some webpages to give her education and hope: pancan.org and our Patient Power programs about the disease. She was grateful. I did tell her — and she already knew — that there was no miracle drug for pancreatic cancer and that it was a usually-fatal condition. But that there were exceptions and, hopefully, her husband would be one. Of course, wouldn’t an effective medicine be best?

Last night I thought of another situation waiting for new pharma science: I was watching one of my favorite television shows: “House M.D.” You know the one where the world’s greatest diagnostician, Dr. Gregory House (Hugh Laurie) says the most inappropriate things. This season, though, we see a bit of a kinder, gentler side as his love has finally blossomed with his boss, chief medical center doctor Lisa Cuddy (Lisa Edelstein). Last night the writers gave us — and House — a major whammo: Dr. Cuddy has cancer. First it was a mass in her kidney and it turned out to be renal cell carcinoma. We’ve done programs on that so I know it pretty well. Then came news that was even worse: A scan showed the cancer had spread to several places in her lungs. The episode ended with House hearing that news and muttering “she’s dead.”

Oh, if only pharma would have a magic bullet for Dr. Lisa Cuddy, or the husband with pancreatic cancer, or my daughter. On next week’s “House M.D.” we’ll find out if some clinical trial might give hope for Dr. Cuddy. Certainly anyone with pancreatic cancer or EGID is on the lookout for the same.

Having recently had dinner with some scientists at a pharma company, I know they’re really working hard for a medical breakthrough. While investors might keep their fingers crossed for a pill that millions could take for years, I know many people where a targeted medicine for their less-common cancer or debilitating inflammation would be an answer to their prayers.

I welcome your comments.

Wishing you and your family the best of health,

Andrew

*This blog post was originally published at Andrew's Blog*


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