Cook Children's Vital Role in Groundbreaking Hepatitis C Research
'The most rewarding thing I've done in the last 20 years. Hands down.'
Cook Children’s played a crucial role in the research of new drugs that could change the lives of teenagers with hepatitis C.
The Food and Drug Administration (FDA) has approved the use of Harvoni and Sovaldi in the treatment of young people between the ages of 12 and 17. The drugs have proven to be more than 95 percent effective in the treatment of hepatitis C for those who have used the drugs.
If left untreated over many years, hepatitis C causes cirrhosis of the liver, liver cancer and even death.
“This is the most rewarding thing I’ve done in the last 20 years. Hands down,” said Mary Suzanne Whitworth, M.D., medical director of Infectious Diseases, who came to Cook Children’s in 1995 and is the medical director of Infectious Diseases. “We have helped these kids. These children who had this infection will now, hopefully, be cured. Ten years ago, we did not think this could happen.”
Twenty years ago, because of the focus on adults taking injectable drugs, the diagnosis of hepatitis C in children was relatively new. Over the next five years, Dr. Whitworth and staff began to see more and more children diagnosed with the infection. Unfortunately, users of injectable drugs can pass the disease on to their children at birth.
It’s estimated that 23,000 to 46,000 children in the United States are infected with hepatitis C. But only about 20 percent of children get it from drug use. The only way to acquire hepatitis C as a child is at birth or through bloodborne transmission.
In the early 2000s, a treatment protocol with two different drugs to try and cure this infection was established.
Over the course of time, the success rate of this regimen was nearly 60 percent but it was associated with numerous side effects.
Then in March 2014, Cook Children’s began participating in the new study.
“I’ve never seen the kind of results to a drug as we’ve seen so far for this infection,” Dr. Whitworth said. “These children, if they are permanently cured, will now have a normal quality of life and be healthy. They can go to school and play sports. When you have cancer, you can brag to the world that you are cured. That’s not the case with hepatitis C because many of these kids don’t want others to know about their diagnosis. Their friends often don’t know. Fortunately, now they may never need to know.
Because the Infectious Diseases team was proactive, Cook Children’s enrolled two of the 10 teens in the world to participate in the initial trial. Later, they were able to enroll five more patients to fill in available slots.
The trend continued as the study moved to treating younger children, getting three out of the 10 worldwide slots for kids 6 to 11 years. In total, Cook Children’s had more than 15 kids participating in the trial, ranging from age 6 to 17. And so far, the results have been successful.
“The emotional part for the families is that their viral load is now undetectable," Dr. Whitworth said. "When families hear this, they just break down because they have carried the weight of this infection for so long. They have often been anxious since the child was born and now they don’t have to feel that way anymore.”