Every year, advances in pharmaceutical research lead to newer and better medicines for treating, and sometimes preventing, human illness. From the most common to the rarest of diseases, patients and physicians have more treatment options than ever before.

“One of the most notable recent advances in pharmacotherapy is the development of targeted therapies for diseases,” says Brad Pusser, R.Ph., Pharmacy director at FirstHealth Moore Regional Hospital. “Today’s medicines are more specific for what they treat and work more effectively with fewer side effects.”

Pharmaceutical and biotechnology researchers are targeting some of the greatest areas of medical need. In 2005 alone, the FDA approved 28 new prescription medicines—three for cancer, three for type 1 and type 2 diabetes, five vaccines, and assorted treatments for blood disorders, rheumatoid arthritis, HIV and other medical conditions—according to “New Drug Approvals in 2005,” a report by the pharmaceutical Research and Manufacturers Association (PhRMA).

And that’s just the beginning.

PhRMA reports that medical researchers are studying more than 900 new medicines for diseases of aging, 241 medicines for neurological disorders, 399 potential cancer treatments, 146 medicines to combat heart disease and stroke, 197 treatment possibilities for mental illness and 185 new medicines for infectious diseases.

From 1995 to 2005, pharmaceutical companies introduced more than 160 new medicines for adults and children with rare diseases that affect 200,000 or fewer people. Narcolepsy, hypotension (low blood pressure), growth-hormone deficiencies, Crohn’s disease and Lou Gehrig’s disease are some of the rare diseases recently targeted by pharmaceutical researchers.

In the last decade particularly, Pusser has seen significant advances in therapies for cancer, cardiovascular disease and diabetes—three of the most debilitating and prevalent health concerns in North Carolina and nationwide.

Coronary heart disease
Coronary heart disease, which results from the gradual buildup of fatty plaques in the arteries that carry blood to the heart, is the most common type of heart disease and the leading cause of heart attacks in the United States. According to Pusser, new types of blood-thinning medications
(anticoagulants) and drugs that prevent blood clots from forming (antiplatelets) are benefiting people who need surgery to open narrowed arteries.

“These medications, when used in combination with stents, mesh tubes that prop open the artery and help restore blood flow, have reduced the number of open-heart surgeries to treat coronary heart disease,” he says.

Cardiologists often implant stents in coronary arteries after angioplasty, a nonsurgical procedure to clear narrowed arteries performed in the cardiac catheterization labs at FirstHealth’s HeartFirst Center.

Hypertension and hyperlipidemia
For people with high blood pressure and high cholesterol, two major risk factors for stroke and heart attack, pharmaceutical innovation has armed physicians with an array of effective medication and dosing choices. Doctors routinely prescribe medicines to lower blood pressure and low-density lipoprotein (LDL or “bad” cholesterol) in conjunction with lifestyle changes, or when a healthy diet and exercise alone have not sufficiently lowered cholesterol or blood pressure levels.

“One of the biggest breakthroughs of the decade has been statin medications for treatment of high cholesterol,” Pusser says. “I would not be surprised to see everybody taking one at some point in their lives.”

Statins work in many different ways to prevent the formation of cholesterol in the liver or the absorption of cholesterol into the bloodstream. In addition to reducing LDL cholesterol, statin therapy can over time lower triglycerides and raise HDL (“good”) cholesterol in the bloodstream.

According to Pusser, sometimes people on prescription therapy for chronic health conditions have problems following their treatment plan, because the medication may cause unpleasant side effects or be difficult or inconvenient to take.

“Patient compliance with medication therapy can be a problem if the side effects are worse than the disease, especially in people with high blood pressure, who often feel fine and do not have any symptoms,” he says. “For example, older blood pressure medications had a tendency to make you tired, which could discourage people from taking them as prescribed. That sedating effect has been removed from many of the newer therapies.”

Some of the older antihypertensives also required patients to take four to six pills over the course of one day. Pharmaceutical manufacturers have addressed that problem by introducing extended-release formulations, which usually are dosed at one pill once a day, and by developing different classes of medications that work in different ways to treat high blood pressure.

Doctors may prescribe diuretics, angiotensin-converting enzyme (ACE) inhibitors, angiotensin receptor blockers (ARBs), vasodilators, alpha-blockers, beta-blockers, calcium-channel blockers, or central alpha-agonists—either individually or in combination with another antihypertensive.

Stroke
Doctors at FirstHealth hospitals have successfully treated people with a breakthrough clot-busting medication called t-PA (tissue plasminogen activator.)

“Intravenous t-PA is the first FDAapproved therapy for acute ischemic stroke (a blockage in a blood vessel in the brain), but doctors have a brief window to deliver t-PA therapy, usually within the first three hours after stroke symptoms begin,” says Pusser. “Prompt evaluation and treatment of stroke symptoms can reduce the potential for disability and death.”

Cancer
While a cancer “cure” still eludes medical researchers, they have made tremendous strides in developing newer and more effective medicines to combat breast, lung, colorectal and other solid-tumor cancers, as well as leukemias, lymphomas and other malignant diseases.

“Newer generations of anticancer agents are more disease specific, more targeted and more patient friendly,” says Ellen Willard, M.D., a board certified hematologist/ oncologist at Pinehurst Medical Clinic and medical director of FirstHealth Hospice & Palliative Care. “We’re seeing more oral medications and more immunotherapy, or biological therapy, as opposed to straight chemotherapy.”

As the name suggests, immunotherapy uses the body’s own immune system to fight cancer cells. Medical researchers have developed different types of immunotherapy agents that work differently inside the body.

“One of the most recent advances in breast cancer treatment has been the introduction of Herceptin (trastuzumab), a type of immunotherapy known as a monoclonal antibody,” Dr. Willard says. “Herceptin works by itself or in conjunction with chemotherapy. It’s been shown to decrease a woman’s risk of recurrent breast cancer and is effective in treatment of advanced breast cancer. But it’s only appropriate for patients with a certain type of very aggressive breast cancer.”

For people with colorectal cancer that has spread beyond the primary tumor site, Dr. Willard often prescribes another monoclonal antibody, Avastin (bevacizumab), in combination with chemotherapy. This type of immunotherapy is designed to slow or stop tumor growth by affecting the blood vessels that tumors need to grow and survive.

In addition to targeted therapies, new oral chemotherapies and supportive-care medications are improving the quality of care for people who are fighting cancer.

“Most chemotherapy is by infusion, but pharmaceutical manufacturers have begun to introduce tablet forms,” says Pusser. “These are much more convenient for patients, because they don’t have to sit for a six-hour infusion.”

Supportive-care medicines help patients manage side effects of cancer treatment, such as nausea, vomiting, fatigue and anemia. Dr. Willard considers them a “very important part of a patient’s overall treatment plan.”

A newly emerging area of cancer care involves the development of cancer vaccines. In June 2006, the FDA approved the first and only vaccine to prevent cervical cancer and precancerous lesions caused by the human papillomavirus (HPV.) The vaccine is approved for use in 9- to 26-year-old girls and women, and the acting FDA commissioner called it “a significant advance in the protection of women’s health.”

“Cancer is not just one disease,” Dr. Willard says. “I have seen many life-saving and life-enhancing developments in therapy, but we have a long way to go. There are so many more therapeutic possibilities researchers have yet to discover.”

Diabetes
Advances in pharmacotherapy are making a difference for the millions of Americans who are living with diabetes.

“We’ve seen a lot of new medicines in the last couple of decades for both type 1 and type 2 diabetes,” says Jason Joyce, Pharm. D., Pharmacy director at FirstHealth Richmond Memorial Hospital. “Before that, insulin and sulfonylureas were the basic lines of defense. Now, we have many more classes and combinations of diabetes medications available.”

“New diabetes medicines are very specific,” says Lisa Cutrell, Pharm.D., a pharmacist and certified diabetes educator at Richmond Memorial Hospital. “We now know there are different complexities in diabetes, and it’s become common for physicians to prescribe multiple medications that work in different ways to help patients control their blood sugar.”

Insulin remains a standard of diabetes care, but doctors today have more choices when developing treatment plans for their patients. Insulin now is available in rapidacting, short-acting, long-acting and intermediate-acting formulations, which may be used alone or in combination, depending on a patient’s specific needs. Pharmaceutical researchers also are innovating needle-free ways to deliver insulin doses. New options include a powdered, inhalable form of insulin and pre-filled insulin pens.

People with diabetes face a greater risk of developing cardiovascular disease, and many individuals take medications to lower their blood pressure and cholesterol along with their diabetes medicine.

“With healthy lifestyle changes such as diet and physical activity, patients can delay or prevent many of the long-term complications of the disease,” says Benny Morse, Pharm.D., a pharmacist at Richmond Memorial Hospital. “Other preventive measures, such as statin therapy, can help reduce cardiovascular risk factors in this patient population.”

“There is no ‘magic pill’ for diabetes,” says Joyce. “Medication is only part of the picture; without lifestyle changes, you won’t get to the point where you need to be.”

Targeted patient types
In the decades since oral contraceptives revolutionized women’s health care, clinical researchers have continued to develop and introduce targeted medicines for specific patient types. In addition to advances
in treatment for breast and gynecological cancers, pharmaceutical companies are meeting women’s health care needs with medicines for urinary incontinence, menopausal symptoms, osteoporosis, irritable bowel syndrome and other diseases that affect women more frequently than men.

According to PhRMA, more than 300 medicines are under development for diseases of major concern to women, including 41 for breast cancer, 33 for ovarian cancer, 41 for diabetes, 36 for arthritis, 23 for depression, 21 for Alzheimer’s disease and 13 for multiple sclerosis.

In men’s health, new therapies are relieving symptoms of benign prostatic hyperplasia (BPH), an enlargement of the prostate that causes discomfort and urination difficulties, and other urologic conditions such as erectile dysfunction. For both men and women, gender-specific research continues in the areas of mental health, pain management, cardiovascular disease, cancer and other therapeutic categories.

Pharmaceutical researchers have also taken aim at diseases that affect older adults and children. According to PhRMA, 158 new medicines are in testing or awaiting FDA approval for asthma, cancer, infectious diseases, juvenile rheumatoid arthritis, cystic fibrosis, diabetes, epilepsy, attention-deficit/hyperactivity disorder, and other ailments that afflict children and adolescents.

Drugs targeting Alzheimer’s disease, stroke, Parkinson’s disease, macular degeneration, osteoarthritis, chronic obstructive pulmonary disease (COPD), neuropathic pain and sleep disorders are just some of the 900-plus medications in development for aging-related afflictions.

Tobacco cessation
We all know the high price of tobacco use and, in particular, cigarette smoking: increased risk for stroke, high blood pressure, heart disease, lung cancer and pulmonary disorders such as emphysema. Many of these health risks can be dramatically reduced or even reversed by breaking the body’s dependence on nicotine.

According to the Tobacco Control Branch of the North Carolina Department of Health and Human Services, “After one year of (an individual’s) quitting cigarettes, the excess risk of heart disease caused by smoking is reduced by about one-half. After 10 years, the risk of lung disease for former smokers is less than one-half that of a continuing smoker. In five to 15 years, the risk of stroke for former smokers returns to the level of those who have never smoked.”

For many who are trying to live without lighting up, nicotine-replacement therapy (NRT) is a helpful and effective tool, says Dianne Homan, R.N., of the FirstQuit tobacco-cessation program, a part of FirstHealth Community Health Services. “Nicotine-replacement patches, gums and lozenges are available over the counter, and the Nicotrol inhaler and Nicotrol NS nasal spray are available with a doctor’s prescription,” she says.

The patch works by delivering a steady baseline dose of nicotine, which satisfies nicotine receptors in the brain. Many experts recommend combining different NRT methods, but this is best done under supervision, as in the FirstQuit program.

“Anyone who is addicted to nicotine has receptors in their brain waiting to be filled,” Homan says. “The receptors can be satisfied by nicotine in any form; but if you smoke or chew too much tobacco or use too much NRT, you may experience nicotine toxicity—shakiness, nausea, watery mouth and headache.”

“The great thing about NRT is that it gives you time to manage the necessary behavioral change and
the psychological addiction without having to deal with marked withdrawal symptoms associated with
the chemical addiction,” she adds. “On NRT, you may have subtle versions of withdrawal symptoms, but
that is what fine-tuning and self-moderation of nicotine levels is all about—to relieve withdrawal symptoms by satisfying the nicotine receptors in the brain.”

Along with NRT, doctors sometimes prescribe the antidepressant tablet bupropion for people who are trying to quit smoking. “Using NRT in combination with bupropion works well for some individuals by satisfying the body’s nicotine cravings,” says Homan. “The bupropion helps lessen anxiety while the individual goes through the quitting process.

Overall, medication use doubles the quit rates, Homan says, but medicines are only one tool in the three-part process used in the FirstQuit program.

“Medications only address the chemical dependency,” she says. “Equally important are behavior and cognitive change. We help people to identify their triggers for smoking and to formulate plans to manage those triggers when they arise. Cognitive change, or the way you think about tobacco use, comes about with education, identification with a new nonsmoking lifestyle and getting out of denial about the unhealthy effects of smoking.”

Bacterial infections
At FirstHealth Montgomery Memorial Hospital, a critical access hospital, advances in antibiotic therapy are making a positive impact on the quality of care, according to Director of Pharmacy John Walker, R.Ph.

“Many new oral and intravenous (IV) antibiotics have been introduced in recent years,” he says. “These medications play a big role in improving outcomes for patients with pneumonia, which is one of our top admission diagnoses.”

One of the newest classes of antibiotics, quinolones, is available in once-daily tablet and liquid forms, the latter of which is a convenient dosing option for people who have difficulty swallowing tablets.

Walker supports efforts by pharmaceutical manufacturers to introduce more convenient drug-delivery systems and longer-acting formulations of their medicines.

“Medications that once were taken two to three times a day or more now are available in daily or even weekly doses,” he says. “We’re also seeing many new products in the form of topical preparations such as transdermal patches. In my mind, developing longer- acting drug forms of many medications presently being used that can be administered safely and promote patient compliance should be a huge focus of manufacturers. This would be a great benefit to our rapidly aging population, which takes multiple medications to stay healthy and has an increased susceptibility to side effects, drug interactions and overdoses.”