Rock Center   |  June 07, 2013

Experimental drug saves babies, but FDA hurdle remains

Dr. Mark Puder of Boston Children’s Hospital developed a treatment with a drug made with fish oil called Omegaven that he’s been using to help reverse fatal liver disease in infants. Despite the drug’s effectiveness, it has yet to be approved by the Food and Drug Administration. Puder says that the FDA has told him they want the usual rigorous controlled study where half the patients get the experimental drug and the other half does not. Puder refuses to do the randomized drug trial because it would mean that some infants may die. Dr. Nancy Snyderman reports.

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This content comes from Closed Captioning that was broadcast along with this program.

>>> here tonight involves a family whose personal heartbreak turned to joy right before colliding head-on with public policy . this family's journey started with a desperately sick child and their determination to help him at any cost. along the way they have seen the very best of the american medical system, but they've also been caught in a kind of medical limbo, an experience that could have serious consequences for a lot of sick children. dr. nancy snyderman has our report.

>> reporter: when sam o'connor was born, he seemed like a picture perfect healthy boy, but just days later his parents, debra and jason, sensed something was wrong.

>> he didn't move a whole lot. he was constantly sick. i mean, he was throwing up quite a bit. he looked like a miserable unhappy little boy .

>> i took him to the clinic in the morning. the doctor got her first look at him. and said, i really don't want to scare you, but i want you to just put him back in the car and go straight to the hospital.

>> reporter: his doctors in milwaukee, wisconsin, soon discovered sam was born with a defect. his intestines did not work and he could not absorb food.

>> and so we would go through these three difs them feeding him and watching him be relatively stable to very quickly all of a sudden going very much downhill and start to dehydrate and his blood work would just be all over the place.

>> reporter: even worse, the very intravenous nutrition that was keeping him alive was also simultaneously destroying his liver. in essence, slowly killing him.

>> there would be nothing more they could do. we should take him home, make him comfortable and enjoy whatever time we would have. they said he would never live to be 2 years old.

>> reporter: the o'connors refused to give up and embarked on an exhaustive search to save their son. finally they found a doctor more than a thousand miles away with a promising drug. when he said come to boston --

>> his exact words were -- i would be happy to care for your family. how soon can you get here?

>> reporter: so the family took a leap of faith and enrolled sam in a research study on an experimental drug . how quickly did you start to see a difference?

>> i saw it right away.

>> i think right away.

>> for me it was more so the personality change because i mean, to have him start responding to me. and playing? it's just like he's actually a person again. you know, he has almost like his life started at that point because before it was just enduring it.

>> reporter: sam is one of the lucky ones . he was able to get the drug he needed, but that's not usually the case of the one in ten americans afflicted with rare or so-called orphan diseases .

>> just one of those lucky, very lucky things.

>> reporter: dr. mark puder at boston children's hospital saved the o'connors' son with a drug called omegaven . he developed the treatment using the drug after watching four children a year die of liver failure at his hospital. he tested the new treatment on babies that had been transferred to boston children's in late stages of liver disease .

>> their doctors gave up on them.

>> reporter: so they were going to die?

>> they were going to die.

>> reporter: or there was omegaven ?

>> that's it.

>> reporter: as dr. puder would soon find out, discovering a new drug treatment is one thing, but getting it approved by the federal drug administration is another. right now omegaven is still experimental.

>> correct.

>> reporter: are you concerned that while you are awaiting fda approval babies are dying?

>> oh, i have no doubt. he's almost 2 months old now.

>> reporter: when we visited dr. puder, he was treating four babies with omegaven , including this 2-month-old suffering from severe liver injury. so has the omegaven made a difference in him yet? can you tell?

>> the difference we see right now is that the bilirubin has gone down a little bit. it was as high as 39.

>> reporter: meanwhile, it has been five years since sam o'connor started omegaven , and he is thriving. the omegaven reversed his liver disease but did not fix his intestines, so sam does not eat food for nourishment.

>> sam , can you do it for me?

>> reporter: sam knows how to help with his feeding routine at bedtime. through a port in his chest, he receives an overnight infusion of omegaven providing all the nutrients he needs to live.

>> can you hold that up for me?

>> hey. i want that to be closed.

>> reporter: since dr. puder's discovery eight years ago he's treated 200 babies with omegaven with remarkable results. yet, despite these success stories, and no notable side effects , omegaven has not been approved by the fda . here's the sticking point -- dr. puder says the fda has told him it wants the usual rigorous controlled study where half the patients get the experimental drug and the other half does not. but doctor puder refuses to do that. but the classic study is a randomized blinded trial.

>> that's right. and that's absolutely right. the problem with the randomized controlled trial is to say take 50 babies with all that this liver disease and only treat half of them when i know what our results are and honestly telling a mother to do that for the science is to me unacceptable.

>> reporter: unsipable because it's bad science ?

>> it's beautiful science.

>> reporter: bad medicine .

>> bad medicine .

>> we approve drugs that work for which we have evidence that they work. we don't approve hope at the fda .

>> reporter: until two years ago, dr. timothy cote was the director of the fda 's division for drugs that treat rare or so-called orphan diseases . the fda would not talk to us because the agency is not allowed to discuss any drug's application for approval. but dr. cote did agree to explain the policies in general.

>> they want to see the real data. they don't want to hear your belief. they don't want to hear how you feel about the drugs. they want to see the facts.

>> reporter: sometimes randomized clinical trials don't make sense. when do we put common sense and science in tandem?

>> i think the common sense and science are in tandem already.

>> reporter: what's more, dr. cote says, there is a way to get access to unapproved drugs. it's called compassionate use . and requires doctors to get special approval from the fda on behalf of their patients.

>> that's exactly what compassionate use is for. is when you have a fatal disease and you have an opportunity and you think something might work. we're not having babies die because the fda won't let the drug go. okay?

>> reporter: compassionate use is how dr. puder and how other hospitals like texas children's are providing omegaven for sick babies , but doctors say the drug application process is difficult and wastes precious time .

>> the problem with this disease in children is it's so rapidly progressive that you may lose the time to be able to rescue them. so if their liver disease is bad at two months and then four months now, there's a point where there's a point of no return.

>> reporter: and even those children who get omegaven in time can run into problems continuing treatment because they can't always get the drug where they live. that's because it's illegal to ship a drug that has not been approved by the fda across state lines.

>> can you say omegaven ?

>> yes, omegaven .

>> reporter: so every eight weeks sam o'connor and his dad must make an 18-hour-long trip from wisconsin to boston for a checkup and to get their supply of the drug. it's an expense the family cannot afford, and they have been forced to borrow, fund-raise and rely on the kindness of their community. if you could talk to the fda tomorrow, what would you say to them?

>> why don't you approve it?

>> i mean, i would like some real answers on why this process takes so long.

>> reporter: in the meantime, dr. puder continues to submit his results to the fda hoping it will approve omegaven so that more children like sam can be saved.

>> you like that?

>> first of all, what a cute little kid. dr. nancy snyderman 's here with us. what does the future look like?

>> the future is a little muddled. it's possible that the fda will allow moem gaven to come to market because there's just no rational way to put these sort of expensive drug trials together. and there has been a precedence for that in the past. cancer drugs, hiv drug, but the fda insists it's here to make drugs delivery in the u.s. safe. we've seen disasters from the past including fen-phen and other medications you know about. thalidomide in the past.

>> right.

>> it's here to protect us, but we need drugs to speed up especially in case