Karen F. Murray, MD, Professor and Vice Chair of Clinical Affairs, Chief, Division of Gastroenterology and Hepatology, Department of Pediatrics, University of Washington School of Medicine, and Seattle Children’s Hospital talks about a drug that has already been approved for treatment of Hepatitis C in patients ages 12 and over, and is now in trial for use in children as young as three.
Interview conducted by Ivanhoe Broadcast News in October 2017.
I read that there are more numbers of kids being born with Hep C, how big of an inclusion are we talking about?
Dr. Murray: Yeah, Hepatitis C in children is not believed to be nearly as big a problem as it is in adults. Where adults have acquired the infection, usually through IV drug use historically, children who acquire the virus most commonly these days are born to a mother who is infected and they therefore are infected. The number of children who are acquiring the virus through that mechanism is actually going down as successful treatments are available for adults. But we still estimate that about 23,000-46,000 children in the United States are infected with the virus.
That’s a lot. Are there any symptoms?
Dr. Murray: Yes that is a lot. Most children are asymptomatic completely which is part of the problem, they have to be tested in order for us to know about it.
Talon though showed symptoms?
Dr. Murray: He actually was detected, as I recall early, right before his adoption, to be Hepatitis C positive. He was not symptomatic from the virus itself but he did have elevated liver labs.
What’s the problem then, if they are asymptomatic what is the long-term medical problem that they’re going to face?
Dr. Murray: Well the biggest problem with being asymptomatic is they’re not detected. For any disease for which there are symptoms people bring themselves to medical attention and they get the therapy that they need. But for Hepatitis C they may be asymptomatic and therefore remain undetected and therefore untreated for decades. And it is in that circumstance that their liver disease will potentially progress.
So Talon tried Interferon and it didn’t work for him.
Dr. Murray: It didn’t.
Then what happened? Tell us about the trial.
Dr. Murray: Talon was treated with Interferon and at the time that was all we had available and not only did it not work, but he developed significant side effects from the medication which were very common, which required us to stop the medication prematurely, which is in part probably why he did not respond. He then went many years without treatment and then when Harvoni became available for his age group, we were able to treat him with this. And it is a very different class of drug than Interferon. It’s provided orally, the side effects from the medication are minimal to none; the side effect profile is basically a placebo, it is very well tolerated and the efficacy of the treatment is truly astounding, ninety eight plus percent in his age group. And the treatment is only twelve weeks for the type of genotype that he has.
How is it working? The word cured is an amazing word that you don’t hear very often. How does it work?
Dr. Murray: It’s a direct-acting antiviral, so it has antiviral as well as immune modulary properties. It is clearing the virus within the first weeks of therapy, and even in studies in adults that have looked at the viral response within a week the amount of virus that is present in the blood stream drops dramatically. Certainly by a month most individuals have undetectable virus. And they were able to be treated within adults eight to twelve weeks typically and have the virus gone.
With results like that, it sounds like this could be a disease that goes away. Is that true?
Dr. Murray: It is actually true. We have every reason to believe that in individuals who remain sustained virologic responders, so those who remain negative for the virus six months after the end of treatment, the vast majority, and we’re still determining exactly how high that is but certainly in the high ninety percent, remain negative for the virus long-term as far as we can tell. Trials in pediatrics for long-term follow up are currently ongoing.
So even though the testing of this drug is finished, the clinical trial is over, now you’re just doing the monitoring?
Dr. Murray: Actually the answer to that question is both. The drug itself, Harvoni, is approved for adults, it is approved for children down to age twelve and the studies that led to that approval are complete. There are now studies ongoing enrolling children ages three to six and six to eleven. And those studies have not yet been published. It is expected that, assuming the results are as favorably as we think they’re going to be, that also will lead to approval. And then also in the older children who have already been treated long-term follow up is ongoing yes.
Check them every year that kind of thing?
Dr. Murray: Correct, exactly, for five years.
So Tal was ten, how did he get in?
Dr. Murray: Into the trial, he got in the six to eleven year age group.
I thought you said twelve.
Dr. Murray: Twelve up has been approved.
So he was actually part of the trial?
Dr. Murray: Correct he was part of the trial.
That’s pretty amazing it must feel pretty good to be part of something with such immense success.
Dr. Murray: It’s actually very rewarding to be able to bring treatments that are currently investigative to patients that have such a huge benefit to them as well as contribute to advancing the treatments that we have available for our population.
What’s in Harvoni, you said it’s a compound?
Dr. Murray: Yeah, it’s a combination of two direct acting antivirals, sofosbuvir and ledipasvir which are two different drugs. There is another drug combination sofosbuvir and ribavirin. Ribavirin is also an orally-administered in this case medication which has antiviral properties. It does not work alone but in combination with sofosbuvir can be very effective for some of the other Hepatitis C genotypes.
What’s next then just monitoring or do you move to a different strain of Hepatitis, what’s next?
Dr. Murray: Interesting question. We obviously we would like to be able to treat all children, all individuals with Hepatitis C and so we want to insure that it works in the youngest age ranges. I think the next thing will be how can we prevent this infection from being transmitted from mothers generally to children or to any adult to a child. And earlier detection of adults, getting folks who have limited resources get them the treatment that they need and be able to maybe even treat women during pregnancy to clear this virus.
END OF INTERVIEW
This information is intended for additional research purposes only. It is not to be used as a prescription or advice from Ivanhoe Broadcast News, Inc. or any medical professional interviewed. Ivanhoe Broadcast News, Inc. assumes no responsibility for the depth or accuracy of physician statements. Procedures or medicines apply to different people and medical factors; always consult your physician on medical matters.
If you would like more information, please contact:
Monica Langsted
206-987-1095
Anna Altars
Anna.Altavas@seattlechildrens.org
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