About 30 years ago, children infected with HIV were expected to live only a few short years. Substantial advances in understanding the inner workings of the virus and developing effective drug therapies have since extended the life expectancy of those infected. Children with HIV can now expect to live — and live well — far into adulthood. And just as importantly, these new therapies, when given to HIV-infected pregnant women, can prevent almost all newborn infections.

Understanding the complex steps involved during infection with HIV to develop methods of preventing infection is only part of the HIV research agenda. Another, equally important, factor involves research into the most effective and manageable care for those already infected with the virus.

The improvement in the quality and length of life in those with HIV can be attributed to a remarkable collaborative global research effort, of which Children’s Hospital is proud to be a contributing member.

Richard Rutstein, MD, medical director of the Hospital’s Special Immunology Service, has cared for HIV-infected children for 25 years, the last 20 at Children’s Hospital. He leads numerous clinical research studies looking at new antiretroviral agents, the effects of the drug regimen on patients, and the impact of HIV-related illnesses on the patients’ future health and long-term survival.

The success of the worldwide AIDS research initiative is evident in the dramatic decrease in perinatal HIV infection. Dr. Rutstein and his colleagues reported that among families followed at the Special Immunology Family Care Center, the rate of transmission of HIV from mother to infant dropped from 20 percent to less than 1 percent when the women were treated with a three-drug regimen. Their findings led to the present perinatal HIV treatment programs.

“HIV perinatal prevention programs are by far the most cost-effective and efficacious perinatal programs to date,” says Dr. Rutstein. “All pregnant women should be offered HIV testing early in pregnancy, and again in the third trimester.”

Today, Dr. Rutstein and his colleagues are part of national multisite research networks funded by the National Institutes of Health, the Centers for Disease Control and Prevention, and the Agency for Healthcare Research and Quality. These multisite networks of pediatric/adolescent and pediatric/adult HIV treatment centers follow segments of the HIV population to provide extensive longitudinal data on the clinical outcomes of those infected. This research will continue to guide investigators on the path for future translational research efforts that can vastly impact and enhance the health and longevity of infected individuals.

Much of the clinical research conducted by Dr. Rutstein and his team as part of these multisite efforts focuses on new drug treatment strategies and safety monitoring studies. Dr. Rutstein, Carol Vincent, PhD, and the IMPAACT and PHAS teams and Special Immunology team, as well as Jim Vagnoni, MSW, and Mary Tanney, RN, MSN, CRNP, MPH, and the adolescent teams lead by Christine Ambrose, MSW, LSW, and Marne Castillo, MEd, PhD, are together leading the Hospital’s clinical endeavors in HIV and AIDS prevention and treatment.

HIV clinical research at Children’s Hospital continues to flourish today despite several global challenges. Challenges at the heart of this research involve providing less costly and less toxic therapies while ensuring long-term survival through adherence to an increasingly arduous and difficult-to-follow anti-retroviral drug regimen.

Another challenge for investigators and healthcare providers is to ensure access to affordable HIV medicines to the populations hardest hit by the virus.

“The number of infected children and teens in the United States represents a small percentage of infected cases worldwide,” says Dr. Rutstein. “We need to maintain our focus in finding ways to bring therapies to those populations where HIV has the greatest impact and where efficient and affordable access to healthcare is suboptimal.”