Biologics—a new class of drugs derived from living organisms—have great potential for treating brain and other diseases. They interfere with the way a disease causes damage, rather than treating the disease’s consequences, and can be individually tailored to the person taking the drug. Because biologics are created using DNA technology, rather than a uniform chemical reaction with a predictable outcome as are traditional drugs, their risks are significant and often unpredictable. The economic rewards for the biotechnology companies that develop these drugs are high, but so are the costs. The authors ask how patients, physicians, industry, and government can successfully balance these risks and rewards.
Living organisms, ranging from human cells to bacteria, are being used to produce new kinds of drugs that are providing promising treatments for some frustrating medical conditions. These drugs, called “biologics,” rely on the insertion of a DNA sequence into living cells or organisms; the cells then grow and produce a large, often complex, protein using their own natural machinery. Biologics differ in important ways from the more traditional common forms of drugs, which are smaller, simpler molecules produced from carefully sequenced chemical reactions between inorganic (nonliving) materials.
As scientific “bluebloods,” with their origins in research that earned multiple Nobel Prizes, biologics are particularly exciting to clinical researchers because they hold promise for diseases that have had few effective treatments. Already, they have become significant for several immunological, inflammatory, and neurological diseases, and the market for them has risen into the tens of billions of dollars. Despite their promise, however, the development process has also revealed serious (and unexpected) adverse effects, suggesting that caution is warranted.
Biologics for Multiple Sclerosis
The development of biologics to treat multiple sclerosis is illustrative of both the rewards and the risks of these new drugs. Treatments for disorders affecting the brain represent, so far, only a small portion of biologics developed, but biologics for multiple sclerosis have fundamentally changed clinical treatment of the disease.
Multiple sclerosis is a chronic, recurrent, inflammatory (and presumably autoimmune) disorder that results in injury Biologics are particularly exciting to clinical researchers because they hold promise for diseases that have had few effective treatments. of the myelin coating of nerve cells in the brain and the central nervous system. When myelin is damaged or destroyed, the ability of nerves to conduct impulses to and from the brain is disrupted, resulting in highly variable symptoms that may include cognitive changes, difficulty in walking or balance, vision problems, pain, fatigue, and bladder or bowel dysfunction. Multiple sclerosis affects both men and women, often in the prime of their lives, and is one of the leading causes of disability among young women.
Before the first biologic was developed to treat multiple sclerosis, only symptomatic treatment was available, usually anti-inflammatory medications such as steroids. The development of the biologic interferon beta-1b (Betaseron), which was approved in 1993, opened a new class of “immunomodulatory” treatment that could be taken on a regular basis to prevent relapses, slow disease progression, and potentially alter the course of the disease. Other interferons for multiple sclerosis followed, and the market for biologics for multiple sclerosis is now more than $4 billion per year.