The Associated Press - Monday, December 02, 1996 11:51:00 PM.
Lauran Neergaard, Associated Press Writer

Since none of the three new and potent medicines revolutionizing AIDS care is yet approved for child use, pediatricians and parents have begun struggling on their own to determine safe doses -- fearing that otherwise the children will die waiting as drug companies study the question.

"I looked over to my daughter and thought, `How could I sit here and try to save my life and not my daughter's?'" Johnson, of Baltimore, angrily told government AIDS experts last week. "We are not going to let our children die without a fight."

Under a pediatrician's care, Johnson's 9-year-old now is one of just a handful of children nationwide taking one of the new drugs. So far, she is doing well. "I want other children to have this chance," Johnson said.

Drug makers say they're working hard to get the new drugs, called protease inhibitors, to children. They have studies planned for early 1997 on everything from liquid formulas to drug "sprinkles" that parents would mix into applesauce.

The drug companies say children spit out earlier liquid formulas because they were too bitter. And the companies had problems getting the right drug absorption.

Still, "in hindsight, perhaps we should have moved forward to get some experimental data" sooner, said Dr. Miklos Salgo of Hoffman LaRoche, maker of the first protease inhibitor, saquinavir.

The issue doesn't just touch AIDS. Eighty percent of prescription drugs are sold with no information on how safe or effective they might be for children.

A little more than 10,000 of the nation's half a million AIDS cases have been in children and teen-agers. Some 3,156 children under 13 and 1,452 teens are still alive and in need of medicine compared with tens of thousands of adults.

But doctors say it's unethical to ignore children just because there are fewer victims.

"AIDS kills children just like it kills adults," said Dr. Nancy Hutton of Johns Hopkins University's Children's Center. She wants drug makers to test new AIDS medicine in children as soon they test adults, changing decades of scientific practice.

Of the nine AIDS drugs sold, four of the oldest are approved for children.

But the new protease inhibitors are so effective for adults that pediatricians want to use them in children. They just don't know how. The Pediatric AIDS Foundation surveyed over 950 child patients and found only 74 taking proteases.

"I had parents who said, `Well, I'll just give my child some of mine,'" Hutton recalled.

That's dangerous, because the wrong dose can cause drug resistance. So Hutton furiously sought early data from drug makers to calculate her own doses of ritonavir, the only liquid protease sold, for six very ill children, including Johnson's 9-year-old daughter.

A few months later, all six children are doing well, although Hutton warns that she doesn't know how long the effect will last or what is the best dose.

Of the three proteases:

--Merck & Co. began child testing indinavir in July 1995, hoping to seek Food and Drug Administration approval for children and adults simultaneously. But one formula didn't dissolve properly in children's stomachs. Adult-sized capsules did fight HIV in children's blood, but not as much as they do in adults nor for nearly as long. Children probably metabolize the drug too fast, theorizes Merck's Dr. Paul Deutsch, who is hunting a better child's dose.

--Ritonavir was created as a liquid for children, but Abbott Laboratories says it believes in ensuring a drug is safe in adults before testing children, something it says couldn't be done in the three years between ritonavir's discovery and its sale. A study in 46 children unveiled last summer suggests 500-800 milligrams a day is safe and may fight the virus; confirmatory studies begin by spring.

--Not only did Roche's liquid saquinavir taste bitter, it didn't dissolve into blood properly. So Roche created tiny "sprinkles" of saquinavir that children would swallow in applesauce, providing just mild bitterness if they crunched the bits. Testing begins by spring.

The FDA's outside scientific advisers issued a stern warning to drug makers last week not to seek approval for any more AIDS drugs without at least preliminary data on children.

But the FDA says the only way to enforce that is to refuse to approve new AIDS drugs without child data -- unfair to desperate adults also awaiting the medicines.

FDA pediatrician Sam Maldonado says angry parents have more clout. Their activism, she says, "creates more pressure than we can."

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