Healthy Skepticism Library item: 24

Warning: This library includes all items relevant to health product marketing that we are aware of regardless of quality. Often we do not agree with all or part of the contents.

Publication type: news

Hale V.
Creating More Paths to Hope
Newsweek 2004 Dec 6

Full text:

They were good friends of ours, and they were desperate.
Their 13-year-old daughter had been diagnosed with a deadly form of cancer called Ewing’s sarcoma. Her doctors had little to offer, and the cancer was spreading fast. Was there anything, they begged us, that we could do to help? My husband is a physician, and I had worked for years in the Food and Drug Administration and in the biotech industry. With the help of a of a former FDA colleague, we immediately began searching for experimental compounds that might offer even a glimmer of hope.

And we found them. In fact, two substances had shown initial promise in fighting the cancer yet both were sitting idle on laboratory shelves. No clinical trials were planned. No one was working to develop the compounds into drugs. The cancer, though deadly and potentially curable, was too rare to create an attractive market for new treatments.
Drugmakers had better investments to pursue.

Shocking? Of course. But the harsh truth is that dozens, perhaps hundreds, of potentially life-saving compounds sit on university or drug-company shelves because there isn’t a profitable market for them.
The story is usually the same. A dedicated pharmaceutical scientist, testing compounds in the lab, finds something that looks promising. It works in cell cultures. Preliminary animal studies look good. But research isn’t development, and development costs a lot of money. If a new drug isn’t likely to generate big profits, chances are it will never leave the laboratory. Sometimes the target is a rare condition like Ewing’s sarcoma, which strikes only a few thousand Americans a year. But it can also be a disabling or deadly disease that strikes millions of people people unlucky enough to live in an impoverished country.

It’s easy to blame drug companies, and they’ve had their fair share of criticism. But let’s face it: they’re in the business of making money.
Their shareholders demand it. Why would a profit-driven company spend millions developing treatments for a parasitic infection found only in Africa when it can make billions on new drugs for baldness or erectile dysfunction? Clearly we have to find other ways of bringing needed drugs to market.

This year we’ve just completed the third phase of clinical trials for our first drug, paramomycin, which targets visceral leishmaniasis. Few Americans have ever heard of the disease, but it afflicts 1.5 million people worldwide and kills some 200,000 every year. We expect the new drug to be approved by fall 2005. And if all goes well, we’ll soon begin testing a drug to treat Chagas’ disease, a scourge that affects people in Central and South America. So far we’ve focused on medicines that can save the most lives or ease the most suffering, especially in places where the cost of prescription drugs is beyond reach. But there are still plenty of compounds worth developing for rare diseases like the cancer that took the life of our friends’ daughter.

As scientists and companies hear about our efforts, many are calling to say, “Hey, we’ve got something you may be interested in.” And many of them are volunteering their time and expertise. That’s been enormously gratifying, of course, but not surprising. Most pharmaceutical researchers got into the business because they wanted to ease suffering and save lives. Systems may be flawed, but most people want to do the right thing. All they need all any of us need is to create a path.