How are treaments for tough diseases improved?

Occasionally someone askes me “When will they find a cure for leukemia?”
Of course this is a tremendously naive question that ignores the  tremendous improvements in treatments that have developed in the last  20 years.   I usually answer the question by trying to explain that  cancer has to be fought on hundreds of different fronts and that while  many battles have been won, the war is certainly a long way from being  over.
My answer on this type of question always felt a little bit hollow.   What exactly was a battle in this context?   Why are the survival  statistics for certain types of cancer so much better now than they  were 20 years ago?
Today, I read a short article in the health section of Slate.com  that provided a great explanation on how the treatment of juvenile  leukemia was improved from a 20% to 80% survival rate between the early  70’s and late 90’s.   From my reading across a wide variety of cancer  related subjects, I think this short article provides a good paradigm  for how health treatments are improved for just about every tough  chronic disease.
Here’s the article- http://www.slate.com/id/2193294/  (also pasted below)

Old Drugs, New Tricks Why big health advances rarely involve new medicines.
By Darshak Sanghavi
Posted Tuesday, June 10, 2008, at 1:36 PM ET

Leukaemic cells

Leukaemic cellsBetween the early 1970s and the late 1990s, the  long-term survival rate of children with leukemia skyrocketed from less  than 20 percent to around 80 percent. Over this relatively short  period, many children presumed to be dying instead ended up living. As  remarkable as the surge is the reason for it. Dr. Steve Sallan, the  chief of staff at the Dana-Farber Cancer Institute in Boston, recently  told me that not a single newly discovered drug was involved. Nobody  invented some magical genetic therapy either. So what changed?
Too often, medical advances get advertised as the work of  swashbuckling doctors and patients who take big risks against big odds  and seize miraculous results with new treatments taken straight from  the lab. That narrative is misleading. As with pediatric leukemia, the  reality often is far less dramatic but no less impressive, and therein  lie critical lessons for patients with many chronic, tough-to-treat  diseases like asthma, attention-deficit disorder, and obesity.
The leukemia doctors saved lives simply by refining the use of  old-school drugs like doxorubicin and asparaginase. Over the course of  almost a dozen clinical trials, they painstakingly varied the doses of these older drugs, evaluated the benefit of continuing  chemotherapy in some kids who appeared to be in remission, and tested  the benefit of injecting drugs directly into the spinal column. The  doctors gradually learned what drug combinations, doses, and sites of  injection worked best. And they kept at it. With each small innovation,  survival rates crept forward a bit—a few percent here and there every  couple of years—and over decades those persistent baby steps added up  to a giant leap.

Today, we’re far more likely to hear exaggerated tales of  breakthrough new drugs, aggressively marketed and hyped. But it’s the  leukemia story that’s the historical norm. Back in the early 20th century, for example—decades before the discovery of antibiotics—tuberculosis mortality fell almost 70 percent (subscription required) due largely to careful studies of nutrition and hygiene. From 1980 to 2000, death from heart disease plummeted an astonishing 50 percent,  almost entirely from the use of existing medicines and surgical  treatments. These were gradually tweaked, like leukemia therapy, in  response to scores of incremental studies. During the past 30 years,  mortality from diabetes in men also has decreased by half, largely due to improved use of flu vaccines, smoking reduction, and possibly aspirin use—but not a new blockbuster drug.
Of course, new drugs can sometimes change everything. Example: Genentech’s novel angiogenesis inhibitor Lucentis, which restored vision in patients (subscription required) with macular degeneration. But such successes  are incredibly rare and even in cases like Lucentis, often unforeseen.  (James Watson, the co-discoverer of DNA, imprudently predicted in 1998 that angiogenesis inhibitors might “cure cancer in two years”  and said nothing about their use for treating eye disease.) And in  truth, we don’t have that many new drugs to call on anyway. Last year,  for example, the U.S. Food and Drug Administration approved only 19 entirely new drugs, many of which treat pretty rare diseases or offer little benefit over older medications.
If the greatest medical advances depend mostly on small but  consistent improvements in the use of old drugs, why do certain  specialties (such as psychiatry) fall behind others (such as  cardiology) in producing major results, like a 50 percent  population-wide improvement? The difference isn’t related to a lack of  drug choices. A psychiatrist now has a bewildering array of medications  to treat, say, attention-deficit disorder or depression, just as a  cardiologist can choose from dozens of anti-hypertensive pills. And the  influence of pharma companies is roughly equivalent in both specialties.
The real problem for lagging specialties is that they possess  numerous poorly studied, often recently approved drugs instead of a  small core arsenal of older drugs that are well-understood and so can  be dosed systematically. As the experience with leukemia shows, that’s  exactly the wrong way to cure disease. Successful specialties are  anchored by centralized, rigorous professional organizations that  served, over decades, as clearinghouses for study after study aimed at  calibrating therapy. Thus, cardiologists depended on the Framingham  Heart Study and the scientific committees of the American Heart  Association, pediatric oncologists have the Children’s Oncology Group,  and children’s lung specialists have the Cystic Fibrosis Foundation.  These specialties don’t pin all their hopes on new miracle cures;  instead, they do the grunt work of incremental clinical trials with the  pills they have. And as a result, they save many lives.
That doesn’t mean duplicating these successes is easy. Just as no automaker has successfully copied Toyota’s ingrained kaizen culture (which The New Yorker’s James Surowiecki likens to a hard-to-follow “regular, sustained diet”), the incremental doggedness of certain medical subspecialties resists imitation. But the lagging subfields should try.
Doctors in these fields first should take a long, hard look at their  priorities. Lagging fields are often the scene of paralyzing turf  battles between various institutions over clinical trials. By contrast,  the more successful specialties have overcome such pettiness and forged  nationwide partnerships to churn out study after study. The successful  specialties also encourage studies that choose incremental goals over  the big score.
Old Drugs, New Tricks Why big health advances rarely involve new medicines.

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