"New" Disease

In the wards of Children's Medical Center in Boston last week, or making regular visits to the outpatient clinic, were 200 youngsters suffering from a mysterious disease with a forbidding name: cystic fibrosis of the pancreas. At Babies Hospital in Manhattan there were seven in beds and 80 outpatients; attending Los Angeles' Childrens Hospital were 150 known or suspected cases. Across the country are thousands of other victims, most of them probably unrecognized. For to most doctors, pancreatic fibrosis (also known as mucoviscidosis) is a "new" disease.

It was not until 1938 that Dr. Dorothy Hansine Andersen, a perceptive pathologist working at Columbia University and Babies Hospital, put together the symptoms she had seen in sick children and the physical changes she found in their organs after death. Thus, cystic fibrosis won medical recognition. It is marked by two chief sets of symptoms. One involves the lungs, which are blocked by a heavy viscid mucus, with frequent infections like pneumonia, and wheezy breathing or persistent, hacking cough. The other set of symptoms affects the pancreas, which fails to deliver the normal quota of enzymes to the digestive system, so that children eat voraciously but fail to gain weight, and have frequent, abnormal bowel movements.

Recessive Gene. After Dr. Andersen defined cystic fibrosis, doctors saw that previously they had been dismissing the lung symptoms as pneumonia, and confusing the intestinal upset with something else--celiac disease. Gradually, they learned that cystic fibrosis is by no means rare; by current estimates, one child in 600 is affected. It is inherited and may strike all the children in a susceptible family, or only one in ten, but the average is one in four.*

There is a chicken-and-egg argument as to what the root cause of the trouble is. Some doctors believe that it lies in the mucous glands, which secrete an unusually thick, viscid mucus. This clogs the lung airways and also blocks the tiny ducts in the pancreas, so that its enzymes (notably trypsin) do not get through to the digestive tract. Others think the pancreas itself is at fault, and that its failure to produce enough enzymes causes both the intestinal trouble and the stickiness of the mucus.

In the newborn, cystic fibrosis may reveal itself by the infant's inability to pass stools. Surgeons at Babies Hospital have devised a daring operation to open the intestine and flush it out with a trypsin solution. This technique is also being used in Boston under the guidance of Pediatrician Harry Shwachman, and in Los Angeles by Dr. Stephen Royce and his associates. It has prevented many deaths. After surgery, such a child will present the same problems as those whose symptoms develop a few months later in life.

Clearing the Chest. Until recently, the outlook was bleak indeed for all these youngsters. They were spindle-limbed and undernourished, no matter how much they ate. and they were always coming down with bronchitis or pneumonia. As doctors were learning to treat the digestive troubles and the lung disorders in turn, new drugs appeared to help them, and now the outlook for the little patients is more hopeful.

Dr. Royce, who has studied and worked with both Dr. Shwachman and Dr. Ander sen, puts it this way: "The main hurdle is clearing the chest. For that, we had sulfadiazine first, then penicillin, and each new antibiotic has offered a little more hope. The newest wrinkle is an aerosol with antibiotics, and we are working on plans for home treatment with aerosols three or four times a day, using a small air compressor and mask, which cost $75 to $100. In any case, it is an expensive disease: antibiotics for one patient average $40 a month. The parents of these kids are mostly broke."

All the antibiotics help to protect the child against lung infections, but aureomycin and terramycin seem to have the further power to stimulate growth. With a highprotein, low-fat diet, plenty of fruit (especially bananas for calories), lavish dollops of vitamin A, and preparations like pancreatin (intended to replace the missing enzymes), most patients win a new lease on life. Their bodies use fuel more efficiently, and they gain weight. It is a good sign when they begin to eat less, Dr. Shwachman notes in a pamphlet now being distributed to patients' parents.

Pancreatic fibrosis once 'seemed to be exclusively a disease of childhood because no known victim survived to adulthood.

Now, doctors are finding more cases of a milder type, and are successfully controlling the disease in patients well into their teens, and sometimes beyond. The main thing, researchers believe, is that doctors everywhere should suspect cystic fibrosis in the case of any young child who has a persistent cough. If fluid is collected from the duodenum and found to be low on enzymes, the doctors have found another case of this puzzling malady.

*In Los Angeles, seven-year-old triplets provide geneticists with valuable evidence: two are girls and identical twins, and have cystic fibrosis, while their brother (from a different ovum) is as healthy as any kid on the block.

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