Subject: (A Special Report): What's New Date: Published: 11/13/89 (339 lines) Source: Wall Street Journal. Copyright Dow Jones & Co. Inc. Technology (A Special Report): What's New --- Causes and Cures: Basic Discoveries Are Changing The Diagnosis and Treatment of Major Diseases ---- By Jerry E. Bishop THOSE OVER AGE 35 might remember the day in August 1963 when the nation was saddened by the death of President John F. Kennedy's infant son. The child, born 5 1/2 weeks prematurely, died of severe respiratory distress less than two days after birth; as often happens in premature births, the infant's lungs hadn't yet developed the lubricant-like film that coats the air sacs and prevents them from collapsing. Today, he might have lived. A quarter of a century after the president's son died, medical researchers are finally coming up with a way to prevent such tragic deaths, which occur at the rate of 5,000 a year. As many as six different versions of synthetic coatings for infants' lungs are just now emerging from research laboratories. That may not seem all that surprising; new medical treatments are announced almost daily. But the simultaneous development of six medications is more than just another medical advancement. It is also a dramatic illustration of how medical technology has changed over the past few years -- and how it will leave its mark in the 1990s. Until recently, medical research was largely a matter of discovery by trial and error. By contrast, the medical technology welling up out of today's laboratories is grounded in a new understanding of how the human body works and why it goes wrong. Basic discoveries in how the body controls the growth of cells, for instance, are opening a variety of treatments for cancer, the healing of wounds and the control of blood disorders. Unraveling the complexities of the human immune system is leading to advances against not only cancer but also arthritis, multiple sclerosis, diabetes, AIDS and innumerable lesser-known disorders. And basic breakthroughs into the mysteries of the genes are about to dramatically change the treatment of heart disease and may soon lead to cures or prevention of muscular dystrophy, cystic fibrosis, Alzheimer's disease, alcoholism and perhaps scores of other ailments that have defied medical science. Even the hardware of medicine is being built increasingly on foundations of basic biological and physiological knowledge. The new magnetic resonance imaging (MRI) scanners that deliver spectacularly detailed pictures of the interior of the human body are based on new knowledge of how atoms in living tissue behave under the influence of magnetic fields. Lasers are being brought to bear on problems ranging from near-sightedness to heart attacks because of new understandings of how living tissue reacts to the powerful light beams. Here is a brief look at how this explosion in basic knowledge is changing the diagnosis and treatment of some of the major diseases. Cancer The problem of detecting hidden patches of cancer cells is about to be solved by monoclonal antibodies. "Monoclonals" are man-made versions of the antibodies the human immune system launches against any foreign protein, such as a virus, that endangers the body. A monoclonal antibody can be designed that attaches itself only to one specific type of cancer cell and no other cell, normal or cancerous. Biotechnology companies have developed and are mass-producing a dozen different monoclonal antibodies. Several selectively seek out only colon-cancer cells while others are specific to ovarian, breast, lung or prostate cancers. For cancer detection, the companies have learned how to glue radioactive tracers to the monoclonals. Radiation detectors can reveal where the tagged monoclonals go in the body, thus revealing the location of patches of cancer cells too small to be seen by X-rays or MRI scans. Used before surgery, the monoclonals can guide the surgeon to hidden deposits of cancer cells that might otherwise be missed. After surgery or chemotherapy, they can reveal whether a second operation or round of chemotherapy is necessary, often obviating the need for exploratory surgery. At the same time, a score of therapeutic monoclonals, each of which selectively attacks and destroys a particular type of cancer, either are in clinical trial or are about to begin such human testing. Cancer patients also will benefit from the discovery of substances the body normally produces to trigger the multiplication of particular cells. "Colony stimulating factors" (CSFs), for example, stimulate the multiplication of colonies of bone-marrow cells. Destruction of the bone marrow is what limits the use of powerful anti-cancer drugs to less than curative doses. Cancer researchers now believe that the CSFs, by speeding the recovery of the bone marrow, will allow the use of higher, more effective doses of the anti-cancer chemicals. Other cell stimulators, called interleukins, are being used to activate white blood cells that normally attack cancers but that seem to be sluggish or quiescent in cancer patients. One widely publicized "killer cell" activator, interleukin-2, has produced remissions in a small percentage of advanced cancer patients who failed to respond to any other therapy. Heart Disease The new-found ability to locate and identify genes is uncovering an astonishingly large number and variety of inherited genetic defects that render people susceptible to heart attacks. The score of defects, or more, found so far all seem to alter, in one way or another, the body's normal processing of fats and cholesterol. The aberrations lead to a buildup of the cholesterol-rich deposits in the coronary arteries that, in turn, eventually leads to a heart attack. Scientists are trying to discover how widespread each defect is in the population. They hope to introduce new blood tests that could tell an individual whether he or she has inherited a genetic susceptibility to coronary heart disease. The genetic blood tests ultimately may replace the present cholesterol blood test that misses many people with a genetic predisposition to coronary heart disease because their total blood-cholesterol level appears normal. Genetic tests would allow heart specialists to tailor their diet and drug prescriptions to the individual rather than spend months in trying each therapy to see which, if any, works best in each patient. Heart surgeons are developing new "Roto-Rooters" to clear out coronary arteries already clogged with deposits. At present, such heart patients have either coronary bypass surgery or balloon angioplasty, in which inflating a tiny balloon in the artery squeezes open a passage for blood flow. A new device, using a rotating, cylinder-shaped blade, shaves the deposits away; it's being tested to see whether it's better than the balloon idea. Trials are about to begin to see whether the new "excimer" laser can etch the deposits away with its invisible beam. The excimer laser is so delicate it can etch designs on a butterfly's wings; it should avoid the danger that hampers other lasers in artery unclogging -- that of accidentally burning a hole in the artery. Arthritis New knowledge of what causes inflammation of tissues is opening potential new therapies for rheumatoid arthritis and other inflammatory disorders. A new class of drugs appears to prevent the tissues from making the substances (called leukotrienes) that cause inflammation. These new drugs work differently from cortisone, aspirin and the "non-steroid anti-inflammatory drugs" like Motrin or ibuprofen that are the current mainstays of treating inflammatory disorders. Several leukotriene blockers are being tested against arthritis, psoriasis and inflammatory bowel disorders like Crohn's disease and ulcerative colitis. An initial, short-term test with one experimental leukotriene blocker, made by Pfizer Inc., produced favorable changes in body chemistry of 800 rheumatoid arthritis patients. Sufferers of the inflammatory bowel disorders also may soon benefit from an old drug. Methotrexate, a 40-year-old anti-cancer drug now being used for arthritis, produced improvements in 16 of 21 patients with either Crohn's disease or ulcerative colitis in an early test in Seattle. In the future, there's the chance that a monoclonal antibody, similar to those developed for cancer, might be wielded against inflammatory diseases. Biotechnology companies are developing monoclonal antibodies that would knock out the white blood cells that are mistakenly attacking and inflaming the body's own tissues. Such an auto-immune attack on the collagen of the joints is believed to cause the inflammation of rheumatoid arthritis. Multiple Sclerosis The progressive crippling of multiple sclerosis (MS) is the result of the immune system's steadily -- and mistakenly -- destroying the outer covering of the nerves. High doses of radiation that dampen the immune system appear to retard the progress of the disease in some patients treated at a Newark, N. J., center. A half-dozen medical centers have begun testing the idea of treating MS by separating the watery part of the blood -- the plasma -- and replacing it with plasma substitutes. This temporarily gets rid of the antibodies and other toxic immune elements that attack the nerves. Initial results indicate this plasma exchange, combined with immune-suppression drugs, can mitigate the relapse of the disease that periodically afflicts certain MS patients. A similar but more exotic idea is being tried at the University of Pennsylvania. It is believed that malfunctioning of the white blood cells known as T cells is why the nerves are being attacked in MS. So in the experiment, MS patients are given a drug that makes the T cells sensitive to ultraviolet light. A pint of blood is removed, and the part containing the T cells is exposed to ultraviolet light. The T cells are so changed by the light that when they are returned to the body, the immune system regards them as "foreign" cells and destroys them. It's a way to selectively knock out the troublesome T cells without dampening the entire immune system as drugs and radiation do. Enlarged Prostate Urologists are testing non-surgical treatments for aging males' most common health problem: an enlarged prostate gland that interferes with urination. The usual treatment for this problem is a 20-minute operation in which a catheter is used to surgically reopen a channel for the urethra. Now some urologists and radiologists are borrowing an idea from heart surgeons: They use a catheter to insert a tiny balloon through the urethra into the enlarged prostate. The balloon is inflated for several minutes, reopening the channel. Advocates say the procedure is easier and cheaper than conventional surgery. Another experiment involves temporarily inserting a tiny microwave probe through the urethra into the prostate. The microwaves heat the prostate. In one test, hourlong treatments semiweekly seemed to improve 20 of 21 men. At least two drugs are being tested to see whether they can reduce the size of enlarged prostates without surgery. One drug, from Merck & Co., blocks the formation of testosterone, the male sex hormone that causes prostate enlargement. An enzyme inhibitor from Abbott Laboratories also is showing some promise in reducing prostate size in early tests. Transplants The problem of obtaining human livers for transplanting to dying patients, particularly children, is being eased. Patients dying of liver diseases have had to wait for a donor to die before a transplantable liver could be obtained. This has been a major problem for children who have to wait until another child dies to obtain a liver small enough to be transplanted. The liver, however, is a regenerating organ, and it can be divided into several pieces, each of which will regenerate into a full-size, functioning liver. So surgeons are beginning to use each liver from a dead donor for transplants to two patients. Moreover, the half or quarter of an adult liver is small enough to be placed in a child where it will function and grow with the child. It also makes it possible for a living parent to donate a segment of his or her liver for transplanting to a small son or daughter. Meanwhile, a new chemical solution developed at the University of Wisconsin is increasing the length of time that livers can be preserved outside the body to 11 to 12 hours from four to five hours, thereby increasing the chances of getting a dead donor's organ to a needy recipient. Prenatal Diagnosis Earlier and quicker diagnosis of defects in the fetus are in the offing for pregnant women. The nwe chorionic villus sampling (CVS), in which cells are obtained from the developing placenta, is proving easier and almost as good in detecting defective fetuses as amniocentesis, which uses amniotic fluid. The simpler CVS procedure can be performed at seven to 10 weeks of pregnancy -- instead of 16 to 18 weeks for amniocentesis -- when abortion, if chosen, is easier. Added to the CVS advance is another PCR (for polymerase chain reaction) that can help provide a diagnosis ofa genetic disorder such as cystic fibrosis or muscular dystrophy within a couple of hours instead of two weeks. The PCR is a technique for taking DNA from only a few fetal cells and rapidly copying it until there is enough to detect a defective gene. It also can be used to tell a woman, on a "diagnosis while you wait" basis, whether she is a carrier of an inherited diseases. Surgery The discovery of a multitude of "growth factors" used by the human body is leading to faster healing ofsurgical incisions. Several of the tissue growth factors are being produced in quantity by genetic engineering. One such factor -- applied to skin wounds where small patches had been removed for grafting to other parts of the body -- caused the wounds to heal 20% faster than untreated wounds nearby. Scarless wound healing after surgery -- a kind of Holy Grail of plastic surgery -- may someday be possible. Surgeons who have been operating on fetuses to correct anatomical defects before birth have noticed there aren't any scars on the child at birth. Researchers are now trying to find out how fetal wound healing differs from "normal" wound healing. Infant Respirator Distress The prevention of collapsing lungs in premature babies is advancing rapidly. In recent months, Burroughs Wellcome Co. has been distributing a sterile, man-made powder to hospitals around the country. When reconstituted with sterile water at the crib-side, the powder produces a white, foamy liquid. Administered through a ventilator tube to the lungs of a premature infant in respiratory distress, the foamy liquid forms the soapy film that is missing from the tiny lungs. Tests on more than 2,000 premature infants show that the synthetic surfactant, as it's called, cut the number of deaths from respiratory distress by a third. Meanwhile, two other lung surfactants, one being developed by Abbott Laboratories and another by ONY Inc., a small, university-based company in Buffalo, are nearing the market. And, according to Physician's Weekly, a news publication for doctors, at least three other synthetic surfactants are in the development stage. --- Ms. Chase reports on science from The Wall Street Journal's San Francisco bureau. --- What's Hot The fastest-growing procedures, estimated by the Wilkerson Group and Healthcare Knowledge Systems Outpatient procedures, done in ambulatory surgery centers: Average cost to patient ESWL (lithotripsy fro breaking up kidney stones) $10,000 Fiberoptic bronchoscopy of lung (examines the lung through a fiber optic tube 350 Diagnostic procedures on fetus and amnion 1,000 MRI (magnetic resonance image) 850 Endoscopy of large bowel 500 Mechanical ventilation (mechanical breather) 300-600* PTCA (coronary angioplasty) 4,000 Cardiac catherterization (left heart) and coronary arteriography (usually done at the same time) 3,000 Inpatient hospital procedures: Average cost to patient Phototherapy (use of ultraviolet light in treatment of skin conditions and jaundice in infants) $140 MRI 850 ESWL 10,000 PTCA 4,000 Insertion of inflatable penile prosthesis 4,000 Laser Angioplasty (treats blood vessel blockage) 8,000 Fiberoptic bronchoscopy of lung 350 Diagnostic procedures on fetus and amnion 1,000 Mechanical ventilation 300-600* Temporary pacemaker 5,000 *Cost of maintenance and personnel [This article is made available here by Dow Jones Co. for the personal and non-commercial use of callers to this bbs, in the hope that it will be of some help to those who are suffering from the disease and others who are seeking to help them.]