Ground-breaking Hepatitis C treatment research at Cook Children's

Mary Suzanne Whitworth, M.D., describes hepatitis C as a slowly ticking time bomb for children who have been diagnosed with the infection. If left untreated over many years, it can cause cirrhosis of the liver, liver cancer and even death.

Facing these devastating complications, parents are desperately seeking a treatment for their children. Imagine the relief of being part of a life-changing study using an experimental combination of ledipasvir and sofosbuvir, Harvoni, that has resulted in undetectable levels of the virus in these children.

“This is the most rewarding thing I’ve done in the last 20 years. Hands down,” said Dr. Whitworth, who came to Cook Children’s in 1995 and is the medical director of Infectious Diseases at Cook Children’s. “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.”

The research comes at a crucial time. The Centers for Disease Control and Prevention released data last week that states that hepatitis C related deaths reached an all-time high in 2014, more than doubling from 2010. More people have died (2,194 cases reported in 2014) from hepatitis C than 60 other infectious diseases including HIV, pneumococcal disease and tuberculosis.

The CDC says the increase in deaths is frustrating because of the advancement in medications to treat the disease. The increase in deaths includes lack of testing and diagnosis, especially for those using injectable drugs.

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.

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 a new study. There is great hope that this study will be permanently successful for all of these children.

Because our 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.

“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. “The emotional part for the families is that their viral load is now undetectable. 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.”