Issue date: Nov 7, 1999

In this article:

By Jim Thornton

ever in history has there been a better time to get sick. From cancer and heart disease to arthritis and emotional anguish, we are blessed today with an unprecedented number of new medicines to fight the infirmities that would undo us. And the list of potent weapons grows weekly.

In 1999, American pharmaceutical companies invested a record $24 billion in drug research for 354 experimental cancer medicines; 104 for heart disease and stroke; 207 for diseases affecting children; and a slew of other formulations aspiring to fight everything from yeast infections to Alzheimer's disease.

These represent only the tip of the iceberg. Only one out of 10,000 compounds each year that undergo preliminary screening ever makes it to the marketplace. But dozens of new medicines that do prove safe and effective are likely to make a huge impact on the nation's health in the new millennium. Consider the following:

Thirty-nine treatments approved last year by the federal Food and Drug Administration now benefit 180 million patients with disorders that cost the health system $400 billion a year.

Life-threatening diseases such as AIDS and cancer, in many cases, have been transformed into treatable chronic conditions.

"Quality of life"-threatening conditions such as impotence and pathological shyness are yielding to medicinal intervention.

In their quest to find tomorrow's Viagras and Paxils, drug researchers have benefited enormously from an explosion in biomedical technology. "When Alexander Fleming noticed that penicillin mold growing in a petri dish was killing all the germs around it," says John Siegfried, senior medical officer for Pharmaceutical Research and Manufacturers of America, "it was a purely serendipitous discovery." Even after drug research was refined, accidental trial and error continued to rule for years.

Today, thanks to supercomputers and state-of-the-art imaging technologies, researchers can read everything from crannies in the outer "shell" of a virus to flaws in the genetic code. Armed with such knowledge, they can design drugs so specific they do only what is wanted, with mostly minimal side effects.

And research in one area can spill over into others. "The experience we gained using combined chemotherapy in cancer set the stage for the combination 'cocktail' approach now being used to treat HIV," Siegfried says. That could now aid in the treatment of auto-immune diseases. Says Siegfried, "There's a definite ripple effect."

Specific breakthroughs
Arthritis
Four drugs approved since 1998 are "stomach-friendly" anti-inflammatories sometimes called "super aspirins" -- Arava, Vioxx and Celebrex -- for the treatment of osteo- and rheumatoid arthritis, and Enbrel for rheumatoid arthritis. Vioxx also treats severe menstrual pain.

Cancer
Touted as a safer alternative to tamoxifen to treat breast cancer is an anti-osteoporosis drug called raloxifene. A study will compare the two drugs.

Herceptin neutralizes a specific protein overabundant in aggressive forms of breast cancer. While it can trigger serious side effects, it's free of two common problems from chemotherapy: hair loss and blood-cell suppression.

And at least 20 experimental drugs are being tested to prevent the growth of blood vessels tumors need to grow and spread. Two, angiostatin and endostatin, got much publicity after they proved effective at inhibiting tumor growth in mice by 99%.

Vaccines are in development to rally the immune system in patients suffering from malignant melanoma to colon cancer. One vaccine, in use in the Netherlands, may eventually come on the market in the United States.

Ironically, another promising cancer drug is one of the most reviled pharmaceuticals in history: thalidomide, which caused 10,000 "withered-limb" deformities in children whose mothers took the drug during pregnancy. Its potential use: inhibiting tumor growth and the occurrence of macular degeneration in AIDS wasting syndrome as well as ulcerative colitis, lupus and brain tumors.

Cardiovascular
Refludan, synthesized from leech saliva, is being used after surgery to fight clots.

A drug approved just last month for atrial fibrillation is Tikosyn, which blocks the outflow of potassium in only one channel of the heart.

Beta blockers are in a renaissance. A growing number of cardiologists now recommend that patients on a wide variety of cardiovascular medications also take beta blockers to boost their effectiveness.

Mental illness
The FDA approved Paxil for social phobia, a debilitating form of shyness, and Effexor XR for generalized anxiety disorder, a chronic "worrywart" disease historically difficult to treat.

Also approved: For schizophrenia, Zyprexa doesn't carry as many side effects as chlozaril.

Diabetes
About 14 million Americans suffer from Type 2 (adult-onset) diabetes. FDA-approved Avandia appears to provide Rezulin's benefits with much less risk of liver toxicity. Type 2 diabetics also can benefit from FDA-approved Glucophage, which lowers blood glucose in part by decreasing its production in the liver and absorption through the intestines. Glucophage has the added benefit of helping to foster weight loss in many patients.

In late clinical trials: an injectionless insulin for juvenile diabetes -- a nasal spray.

Glucose monitor GlucoWatch is pending FDA approval. The device uses tiny electrical currents to automatically open pores in the skin every 20 minutes and accurately measure glucose levels. It does this without the need for the repeated, painful "finger pricks" most patients detest.

Pain management
Two new delivery systems are available for a synthetic opiate called fentanyl to control extreme pain often experienced by cancer patients: Duragesic is a patch that steadily releases the drug over 72 hours to combat chronic discomfort. For so-called breakthrough pain, patients now can lick Actiq, left, a lozenge on a handle resembling a painkilling lollipop.

Infections
Researchers are working on a vaccine for otitis media ear infections in children. A nasal spray flu vaccine, FluMist, is expected to be available in a year. The FDA has approved Fomivirsen for a common secondary AIDS eye infection, cytomegalovirus (CMV), which can cause blindness.

Beyond pills
Not only drugs are quickly evolving: So are the ways they can be administered.

Soon it may be possible to "magnetize" a drug, then use an external magnet to direct it to the organ to be treated.

There's tremendous interest in monoclonal antibodies, which home in on molecular targets like microscopic bloodhounds; 13 are now approved.

As genetic engineers learn more about the causes of hemophilia, cystic fibrosis and other inherited diseases, it looks likely that special viruses will be used to "infect" human cells with the healthy replacement genes they need to thrive. Already, this is being experimentally injected into so-called P53 tumor-suppressing genes in cancers of the head, neck, lung and breast.