Lynne Meryl Mofenson

As the number of children with AIDS increased dramatically in the United States and around the world during the late 1980s, the depressing fact was that little could be done to prevent infants from getting HIV, the virus that causes the terrible disease.

Fast forward more than two decades to November 2011 when Secretary of State Hillary Clinton pronounced that creating an AIDS-free generation worldwide, one in which no children are born with the HIV infection, is not only possible, but a U.S. policy priority.

Among those who played a pivotal role in curtailing the deadly epidemic among children is Dr. Lynne Mofenson, a physician at the National Institutes of Health (NIH) who helped design and conduct a seminal pediatric AIDS clinical trial, and has since dedicated her career to conducting additional research and influencing national policy in the field.

“Lynne has been the preeminent scientific leader in the prevention of AIDS in children in the world,” said Dr. R.J. Simonds, a vice president at the Elizabeth Glaser Pediatric AIDS Foundation. Many people have contributed to AIDS research, but Simonds said Mofenson’s work provided clear direction and speed.

“She moved the field along uniquely because of her command of scientific issues,” he said. “Her credibility and knowledge base are unparalleled.”

Mofenson, a pediatric infectious disease specialist, arrived at NIH in 1989, and two years later became involved in starting a bold and controversial clinical study that used the only available anti-AIDS drug, zidovudine, or AZT, as a prevention strategy for the children of HIV-infected pregnant women.

No one had thought of using this drug as a way to stop mothers from passing the disease to their babies, and many couldn’t quite believe its efficacy, said Ambassador Eric Goosby, the U.S. Global AIDS Coordinator. “Frankly, we were blown away,” he said.

To Mofenson, launching the controversial study was a “no brainer.”

“It was a horrible disease, killing mothers and babies. We had to do something,” she said.

The seminal clinical trial demonstrated a two-thirds reduction in the risk of HIV transmission from mother to child, down from 25 percent to 8 percent. It turned the tide on pediatric AIDS.

This clinical trial was the beginning of a long-term collaboration among researchers—called the Pediatric AIDS Clinical Trials Group—that conducted a series of successful studies to identify and optimize strategies to block mother-to-child transmission of HIV, the primary way children become infected.

After the initial trial was pronounced a success in 1994, Mofenson stacked her plate with the additional role of policy development. Within six months, the U.S. Public Health Service Task Force that she chaired formulated and released national recommendations on pediatric AIDS prevention.

She worked with multiple agencies to implement these recommendations, including getting the Centers for Disease Control and Prevention to recommend universal HIV testing for all pregnant women, the Food and Drug Administration to approve use of AZT in pregnant women, and Medicaid to ensure that health insurance covered use of the drug.

The landmark policy had a rapid and dramatic impact. By 1996, 80 percent of HIV-infected pregnant women in the United States were receiving zidovudine, and the number of HIV infections in children dropped from more than 1,600 per year to less than 500. Today, the number of HIV cases in U.S. children has dropped to less than 100 a year.

“I have no doubt that her scientific acumen to design critical research and translate findings into global clinical practice, her passion to share and collaborate and her hard work and tenacity over the years have uniquely and substantially contributed to preventing AIDS in thousands of children in the U.S. and around the world,” said Goosby.

Mofenson now serves as a leader in research and policy on the world stage, expanding the clinical trials network to developing countries in Africa and elsewhere to fight pediatric AIDS.