The next big thing in medicine could have been predicted 40 years ago. The baby boomers, who constitute almost 30% of the U.S. population, are getting older. As they age, the rates of heart disease, cancer, neurological decline, and other afflictions will soar. The pharmaceutical industry is well aware of this trend, and for years has focused its research on ways to ease the pain, if not cure it.
At least boomers are marching into the twilight years at a good moment. Huge advances over the past two decades in biotechnology, drug-discovery methods, and deciphering cellular behavior are finally coalescing to produce drugs and medical devices for conditions that had been virtually untreatable. This marks a big change from medical progress in the past, when the majority of new medicines merely offered marginal improvements over existing treatments, or alleviated symptoms of a disease rather than correcting the cause.
Over the next few years, promising new treatments for cancer, Alzheimer's, infectious disease, and cardiovascular conditions will break new ground. These treatments may not constitute cures, but for some patients they could turn once-deadly diseases into manageable conditions. Their design is based on a growing understanding of the biological underpinnings of a disease rather than the old hit-or-miss approach to drug development that required testing thousands of compounds until something finally worked.
A prime example is Eli Lilly & Co.'s (LLY ) new drug for sepsis, a deadly blood infection that is a leading cause of death in intensive-care units. Sepsis can quickly lead to blood clots, followed by multiple organ failure. It kills 30% to 50% of those affected--some 200,000 Americans a year--and it has been virtually untreatable. More than 20 experimental drugs for the disease failed in clinical trials. But Lilly finally broke the losing streak with Zovant, a bioengineered version of a protein that keeps blood from clotting. Investigators reported in February that, in a late-stage clinical trial, Zovant reduced the death rate from sepsis by 20%. Manufacturing problems have slowed its launch, but the Food & Drug Administration will likely approve it in October. "[Zovant] is going to make a huge difference to medicine," says Lehman Brothers Inc. analyst C. Anthony Butler.
Not all of the drugs in the pipeline will be as successful as Zovant, of course. Many may not even see the light of day. The Pharmaceutical Research & Manufacturers of America (PhRMA), an industry group, says it takes an average of 14.2 years to bring a drug from lab to market, and the rule of thumb is that only one out of five experimental drugs survives clinical trials and FDA scrutiny. Also, one of the biggest changes ever to hit medicine--the map of the human genome published earlier this year--won't have a significant impact on health for at least a decade.
Still, enough new drugs are nearing the market that an outline of the medical miracles to come can be traced. Here are some significant categories:
The Big C is the No. 2 killer in the U.S. after heart disease and will likely reach No. 1 as boomers age. That's because once people hit 59, they have a one-in-three chance of developing cancer, compared with 1-in-62 for people 39 and under. The pharmaceutical industry is aware of the need for better treatments: It spends more on cancer research and development--$5.3 billion last year--than on any other disease, and 402 oncology drugs are in clinical trials.
For its money, the industry is seeing a major shift in the treatment of cancer. Instead of trying to slash, burn, or poison tumors, researchers are refining therapies that take aim at the cellular mechanisms that cause tumors to grow uncontrollably. These drugs are designed to attack only diseased cells, and thus should have few or no side effects. They don't necessarily eradicate the cancer, but they aim to stop it from spreading. As a result, many doctors believe cancer will one day be a chronic, manageable disease, much like diabetes. "We can take a lesson here from the AIDS epidemic," says Dr. Harold Varmus, president of Memorial Sloan-Kettering Cancer Center in New York. "Keep the cancer from spreading, and you can keep the patient healthy."
Targeted therapies won a high profile in May with the rapid FDA approval of Novartis' Gleevec, which halts or delays the spread of a deadly form of leukemia. The drug blocks a protein that plays a key role in tumor division. Even a recent report that some of the sickest patients on Gleevec eventually relapsed hasn't dampened enthusiasm for the drug. "Gleevec should be able to work on a lot of solid tumors, so its application will go way beyond [leukemia]," says Dr. Faraz Naqvi, portfolio manager of Dresdner RCM Global Healthcare Fund. "I think Gleevec is coming out as a best-of-class drug."
There are plenty of other targeted therapies coming up. One promising group of drugs blocks the epidermal growth factor (EGF) that encourages rapid growth in more than 50% of tumors. ImClone Systems Inc. (IMCL ) is the closest to market in this class. It filed for FDA approval of IMC-C225 in June. AstraZeneca PLC (AZN ) and OSI Pharmaceuticals Inc. (OSIP ) are expected to file for approval of two other EGF drugs next year.
Here's where the demographics get really scary. About 10% of people age 65 and older and 50% of those over 85 develop this degenerative, incurable, and always fatal brain disease. Currently, about 4 million Americans suffer from Alzheimer's, but the Alzheimer's Assn. predicts that 14 million will succumb by the middle of this century unless a cure or preventive measure is found.
This is a particularly tough target, because the protein plaques and tangles that slowly destroy the brains of Alzheimer's patients have usually done so much damage by the time of diagnosis that salvage efforts would be futile. Most treatments in development aim to attack the disease in its very early stages--the goal being to prevent or delay the plaque from developing. One promising vaccine currently in early human trials is from Ireland's Elan Corp. (ELN ), in partnership with American Home Products Corp. In animal models, the vaccine caused a 70% reduction in plaque. Also being tested are the popular Cox-2 pain treatments, Celebrex and Vioxx, from Pharmacia Corp. (PHA ) and Merck (MRK ), respectively, and drugs from Eli Lilly and Bristol-Myers Squibb (BMY ) that block enzymes contributing to plaque formation.
Heart and blood-vessel diseases have been the No. 1 killer in the U.S. for 100 years and stroke is No. 3. But many doctors are optimistic. In July, the FDA indicated that it will approve the first new drug in a decade for acute congestive heart failure (CHF), which puts 1 million Americans in the hospital each year. Scios Inc.'s Natrecor is basically a replacement for nitroglycerin, but it works faster and is easy to administer. The same month, an FDA panel approved an implantable, pacemaker-like device from Medtronic Inc. (MDT ) called InSynch that could keep CHF patients from reaching the acute phase. CHF, which occurs when the heart beats erratically, is the end stage of many types of heart disease. It afflicts some 5 million Americans, and the cost of caring for them runs up to $40 billion a year. InSynch stabilizes the heart beat, allowing sufferers to lead a normal life.
Also promising is a drug-coated coronary stent from Johnson & Johnson (JNJ ) called Ravel, likely to be approved by the FDA this fall. Stents are used to hold open closed arteries, but scar tissue often ends up clogging the devices, necessitating further surgery. Ravel's drug coating keeps scar tissue from forming.
Scientific breakthroughs are unpredictable, and successful drug debuts only slightly less so. But both should materialize in the next few years to alleviate such ailments of aging as osteoporosis, impotence, eye disease, diabetes, and memory loss. In 10 years or so, you can expect lab-grown human tissue to replace worn-out body parts and drugs that can prevent a disease from occurring in the first place. Boomers might just go gently into that good night.
|Corrections and Clarifications In "Biotech for boomers" ("America's Future," Cover Story, Aug 20-27), the name of Eli Lilly & Co.'s drug for sepsis should have been Xigiris--changed from Zovant in June.|
By Catherine Arnst