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Sanjay Gupta MD

New FDA-Approved Pain Pill Contains More Medication than Vicodin; Brothers Have Rare Disease; Hookah: Just the Facts

Aired April 26, 2014 - 16:30   ET

THIS IS A RUSH TRANSCRIPT. THIS COPY MAY NOT BE IN ITS FINAL FORM AND MAY BE UPDATED.

DR. SANJAY GUPTA, CNN HOST: Welcome to SGMD.

Today, a tough choice. A family with two kids, both have a crippling disease. But only one can get a new experimental therapy.

Plus, a surprising new toxic trend that's now on the rise among college students.

But first, we know that opiates can be addictive. We know overdoses are becoming more common. And we also know that Zohydro is a powerful new pain killer has stirred up more than its fair share of controversy.

(BEGIN VIDEOTAPE)

GUPTA (voice-over): Dr. Jean-Marie Perrone has watched the number of deadly drug overdoses go up and up, and says that's why she voted against federal approval to sell Zohydro. It's a potent new pain killer.

DR. JEAN-MARIE PERRONE, MEDICAL TOXICOLOGY: We're in the middle of a prescription drug epidemic and we thought that that was related to an upswing in prescriptions for these opioid analgesics, like Oxycodone or long-acting Oxycodone.

GUPTA: In fact, roughly 20 percent of Americans have used prescription drugs recreationally. And of those, 14 percent became addicted.

Car crashes are no longer the number one reason people die accidentally in the United States. Nowadays, it's actually prescription drugs. That's because on any given day, people take more than the recommended dose. Mix and match or take medications not prescribed to them. Maybe take pills with alcohol and all of it can make for a deadly dose.

In fact, the most recent data shows 37,000 drug overdose deaths in one year, mostly accidental. About 21,000 involve prescription drugs. And of those, 75 percent were painkillers.

Zohydro is pure Hydrocodone. It has five times more than the strongest Vicodin. Not only that, it can be easily crushed and snorted. But let's not forget chronic pain is a huge issue, tens of millions suffer from it and some patient advocates say there is a place for a drug like this.

But Dr. Perrone disagrees.

PERRONE: As a physician, as a drug safety person, I think that this is not the choice that's going to help our population.

I mean, from my standpoint --

GUPTA: Perrone sat on an advisory panel that voted against Zohydro, 11-2. But despite that lopsided rejection, FDA officials gave the green light to put this drug on the market. It is a schedule 2 drug, meaning, it needs a written prescription. It also carries a strict warning label. In a statement, Zogenix, Zohydro's manufacturer, said, quote, "The fact is that Zohydro ER is a novel pain medicine that fills an important medical need."

(END VIDEOTAPE)

GUPTA: Joining me from FDA headquarters is commissioner, Dr. Margaret Hamburg.

Welcome to the program. Thanks for being here.

DR. MARGARET HAMBURG, FDA COMMISSIONER: Thank you.

GUPTA: You know, it's interesting, this whole issue, it seems to stir up a lot of controversy. Zohydro, the state of Massachusetts tried to ban it, 30-state attorney generals have asked you to reconsider this approval.

Your own advisory panel voted 11-2 against it.

What are we missing here? Why are those folks wrong on this issue?

HAMBURG: Well, I think this should not really be about a single drug. It really reflects the growing concerns about opiate addiction abuse, misuse and overdose, and the need to balance strong strategies to combat that public series health program with ensuring access to important pain medications for patients in need.

I think there are a lot of misperceptions about this particular drug, Zohydro. But I think what it really brings into focus is that we, as a nation, need to do a better job to combat the serious problem of opiate addiction.

I hope that we can really work together to focus on what are the key drivers of that problem and what are the meaningful, enduring solutions.

GUPTA: Did it surprise you that your advisory committee -- again, a very knowledgeable committee -- was so against this? Again, it wasn't equivocal. It was 11-2. HAMBURG: Well, that's an important question. I want to underscore that that advisory committee met at a point in time, indicating that they could understand the benefits for patients, but they were concerned about the overall safety in the context of the epidemic of opiate overdose addiction and abuse.

And what we did was after that advisory committee met, we actually put in place a number of really quite stringent safeguards and new labeling so that it's much more clear to the prescribing physician that this is a powerful opiate drug with addiction potential, that it's only to be used for severe pain that requires round-the-clock opiate treatment when other treatments aren't adequate, that they need to be very sure that this is the right drug for their patient and reminds them of the addiction potential and the need to monitor their patient.

GUPTA: You know, the headline, Dr. Hamburg, a lot of people hear is that pain killer abuse is a big problem. You just alluded to this. One of the stats that's always staggering is that 80 percent, roughly, of the world's pain medications are consumed in the United States. The concern that you hear is that could Zohydro, making Zohydro available, could it make the problem worse? What do you think? How do you answer that concern?

HAMBURG: Well, I think we need to deal with the appropriate prescribing of opiates and we need to find better treatments than opiates for chronic pain. Actually, FDA is working very actively with the research community and with product developers to try to find non- opiate treatments for pain that are effective.

GUPTA: One of the strategies, talking about opiates overdose and some of these horrifying statistics, is tweaking the formula so the pills that can't be crushed, tamper resistant, abuse deterrent. There was a letter, an article --

HAMBURG: Right.

GUPTA: -- in "New England Journal of Medicine" saying that it does -- it can be helpful, the abuse of OxyContin pre-tamper resistant went down after it was made tamper resistant. So, is it -- should we have made a Zohydro tamper resistant version first before releasing it?

HAMBURG: Well, sadly, at the present time, the abuse deterrent technology is very much in its infancy and we are encouraging the research community and companies to develop new, better abuse deterrent technologies. At the present time, there's only one drug out of about 30 of the extended release, long-acting opiates and there is none in the immediate release category that has demonstrated abuse deterrent formulation.

So, we need to keep pushing on abuse deterrent technology and we're encouraging it and working with sponsors. And that's something that I hope we'll have down the road.

GUPTA: Dr. Hamburg, I appreciate you being on the program. This is a very important issue for us as well. We've been reporting on this idea that, you know, accidental overdoses from these opiates are one of the leading causes of death in this country and, yes, this is a fixable problem. Glad to hear that you're on the case.

HAMBURG: We are.

GUPTA: Up next, another tough call. Two brothers with a devastating illness, only one is being allowed to try a new experimental therapy.

(COMMERCIAL BREAK)

GUPTA: Imagine having two sons with a fatal disease. One gets experimental therapy and thrives. The other doesn't and has to sit by and just watch. It's tough to imagine. Certainly as a father, I'm having a hard time doing that. But for the family you're about to meet, it is their reality.

(BEGIN VIDEOTAPE)

GUPTA (voice-over): Fifteen-year-old Austin Leclaire has always wanted to be part of the high school band, but day-to-day, his mom Jenn says it's becoming harder and harder to play.

JENN MCNARY, MOM: Austin's arm function is getting worse. I can see him struggling to lift his drum sticks.

GUPTA: Austin has a severe form of muscular dystrophy known as Duchenne. Muscular dystrophy or MD is the disease that Jerry Lewis has been raising money for, for the past 60 years.

(on camera): Have you noticed anything over the last couple of years that's become more challenging?

AUSTIN LECLAIRE, PATIENT: It's been harder to get in my bed and position myself.

GUPTA (voice-over): Now, having one son with Duchenne would be hard enough for any mom. Jenn has two -- Austin and his younger brother, Max. Boys with Duchenne first lose the ability to walk around the age of 10. Most don't live past their 20s.

But 12-year-old Max seems to defy these odds, he plays soccer, he rides a bike, and Jen believes it's due to an experimental drug called eteplirsen. Max began weekly infusion treatments of the drug 2 1/2 years ago. He was part of a small clinical trial of 12 boys.

Austin didn't qualify because he was already in a wheelchair.

(on camera): How much of a difference has it had in their lives?

MCNARY: I mean, I think you're literally looking right now at the mother of two children with Duchenne, one who is going to be the first child to survive the disease and one who is going to have the last child to die from it.

GUPTA (voice-over): Since the trial started all 12 boys have seen their symptoms start to stabilize, and there don't seem to be many side effects. But the FDA still has not approved the drug. They say they want more evidence.

(on camera): What the FDA says, look, we're just not sure yet. This is part of why we exist, is to be sure that it is safe. Yes, it's been a two-year trial. But we've got to follow these kids for more time. We're not sure how effective it is because, again, it's too small.

MCNARY: We have said that we will accept the possible risk that this drug isn't as effective as we think it is five years down the line or some kind of side effect develops. But in the meantime, while they are not sure and while they are collecting more data, our kids are dying.

GUPTA: Frustrated with the process, parents like Jenn have become vocal advocates, pushing for this drug's approval. It's become a contest of the emotional stories of these children and parents versus the very drug approval process itself.

MCNARY: I want to know that they understand what children go through in a clinical trial.

GUPTA: Finding therapies for rare or orphan diseases like Duchenne can be difficult because of the small number of patients and the extreme progression of the disease. By definition, rare diseases have fewer than 2,000 cases in the country. In the case of Duchenne, there are just 15,000.

MCNARY: Rare disease is such that if we don't get our children in clinical trials and get these drug out quickly, there's no hope for these kids.

GUPTA (on camera): If you were able to get this medication, what do you think it would do for you?

LECLAIRE: I think it would help me be able to do -- be able to do the things I like and for it to be easier for me to do them, like play the drums.

(END VIDEOTAPE)

GUPTA: And me now from Washington is Jenn McNary. You just met her in the piece, Austin and Max's mother. The fate of this drug has been in limbo for quite some time. But just this week, the FDA did lay out a road map for the company, Sarepta, to conduct more trials, to get the drug possibly to more children and potentially lead to its approval if the results pan out.

Jenn, you and I had a long conversation about this. The new trial is not going to launch for a few months. When it does, first thing is first, will Austin be able to get the drug, do you think?

MCNARY: You know, the FDA has allowed for a flexible trial design with no placebo, and it will include non-ambulant children. So, in theory, Austin fits. But again, we don't get special treatment. He will be in line with all the other boys that could possibly benefit from this drug. GUPTA: You know, I had a chance to talk to Austin. You know, he's a smart kid. We talked about a lot of different things. Have you told him this most recent news? Does he know he may be a candidate?

MCNARY: Oh, yes. So, the morning we got the news, it was 6:55 and actually, we were kind of thinking we were going to get some news because just the timing was right.

And so, you know, Christine, my boss, Christine McSherry (ph), were at the house, waiting for the announcement to come out, waiting for the press release and Austin was woken up to the news that the FDA was moving forward and that he should be considered for a trial, and if all goes well -- you know, they could be dosing really, you know, by September. I would say at the latest. I'm hopeful it will be sooner. I feel like the FDA has said that they can move forward as soon as possible.

So, as soon as they have drug I really hope that they're beginning to recruit and dose these children.

GUPTA: I would have loved to have seen the expression on his face. I'm sure there was a big smile and he appreciated that.

Jenn, thank you for spending so much time with us. I think it's really an important story to tell. Appreciate you being on the program.

MCNARY: Thank you so much.

GUPTA: And up next, a once common danger was almost wiped out, but now is on the comeback trail. It's dangerous stuff. It can kill and many doctors don't even recognize it.

(COMMERCIAL BREAK)

GUPTA: Up until the 1960s, there were more than half a million people catching measles in the United States every year, but times change thankfully. Thanks to vaccinations, hardly anyone my age or younger has even seen a case of measles.

But, you know, you can't take this for granted. CDC just put out a warning about a new outbreak.

CNN's Stephanie Elam has the story from Los Angeles.

(BEGIN VIDEOTAPE)

DR. ERIC G. HANDLER, ORANGE COUNTY HEALTH CARE AGENCY: This is a highly infectious disease.

STEPHANIE ELAM, CNN CORRESPONDENT (voice-over): It may start with a fever or a cough, but a splotchy red rash is its signature.

HANDLER: You're infectious four days before you even develop symptoms. So, you may not know you're sick.

ELAM: Many people in the United States have never seen measles since it was all but eradicated a decade ago.

HANDLER: I have been practicing for over 30 years. I've seen it. I know what to look for. But the new physicians haven't seen it.

So, part of the campaign that public health has been doing so to make sure they know what to look for.

ELAM: In California, the number of confirmed cases has risen in the last couple of years. So far this year, 58. In Orange County alone, the number of cases has skyrocketed.

HANDLER: We've had 22 cases here Orange County. That's a lot more than we normally have. We've had over the past five years, no more than one or two cases a year.

ELAM: Why the sudden outbreak? The reason is the growing number of parents refusing to vaccinate their children. Last year in California, there was a 15 percent increase of people opting out of immunizations; in Orange County, a 30 percent increase.

(on camera): Is there any reason to support this idea that vaccinations could be dangerous to children?

HANDLER: Absolutely not. There are serious consequences to having -- getting the measles.

ELAM (voice-over): So here, they are trying to spread the word faster than the virus that vaccinations work.

HANDLER: The two immunizations, the MMR that you get, is almost 95 to 98 percent protective.

ELAM: There haven't been any new cases here in three weeks and more people are getting their shots. Maria Guadarno (ph) tells me she brought her children in for their immunizations two days after hearing about the outbreak.

HANDLER: Education can overcome these perceptions.

ELAM: Another reason for the outbreak is our shrinking world. According to the Centers for Disease Control, 93 percent of the people diagnosed with measles in California contracted it abroad or from someone who just returned from overseas.

HANDLER: Measles is pretty prevalent in other countries. So when you go and visit, you need to make sure that you're immunized.

ELAM (on camera): Dr. Handler says that the majority of the people that are opting out of vaccinations are in the more affluent parts of Orange County. But he's hopeful through education that their minds will be changed.

Stephanie Elam, CNN, Orange County, California.

(END VIDEOTAPE) GUPTA: You know, I want to take a moment and just drive home a very important point about vaccinations. Maybe this will make the case for you -- take a look at this map and pay careful attention to where the measles cases are around the world. This is not hard to see.

In parts of the world where vaccination rates are lower, the number of cases can be higher, exponentially so. In U.K., for example, where vaccination rates have dropped a bit lower that they've had here, they had several recent outbreak.

Maybe this all makes sense t you, but it's important to point out that when you get a vaccine, you're not just protecting yourself. You're protecting others around you. So, even a small drop in vaccination rates can lead to a large rise in cases.

You know, a lot of doctors have never even seen a case of measles. It's worth pointing out that the main symptoms are going to be fever, cough, conjunctivitis, but also this very distinctive rash. It's what people remember.

But severe complications can be infections in the lungs and the brain and that can be fatal. So, you've got to make sure that your kids are vaccinated. And also, anyone who was born after 1956, if you did get vaccinated, when you see your doctor, should ask them to make sure you're still protected.

Still ahead, a double edge sword for college students -- they're smoking fewer cigarettes. That's the good news. But they pick up a new trend that could be even more toxic.

(COMMERCIAL BREAK)

GUPTA: This week, the FDA proposed its long-awaited regulations on e- cigarettes. I want to remind you here. This is what one of those e- cigarettes looks like. You can tell it has a battery pack over here. This is the nicotine vapor over here. Once it's connected together, you take a puff that activates the battery, and you get a nicotine vapor.

The FDA is saying, look, we want companies to disclose ingredients, we want to keep them out of vending machines, we want to put on warnings and keep them away from teenagers.

There's also something else as part of this whole thing that caught my eye, the rules go further. It would cover other tobacco products as well with, including hookahs, which -- this may surprise -- but 40 percent of college kids used a hookah last year alone.

(BEGIN VIDEOTAPE)

GUPTA (voice-over): Many college students believe it's safer than cigarettes, and unaware that hookah smoke can be just as toxic.

This is a traditional hookah. The basin is filled with water, the tobacco heated using charcoal, producing smoke that moves up a chamber and is inhaled through a hose. You might hear that the water filters out the cancer-causing toxins. Don't believe that, that's not true. In fact, hookah smokers are exposed to more carbon monoxide and smoke than cigarette smokers.

In fact, here's a number you won't forget -- in a 60-minute hookah session, smokers are exposed to 100 to 200 times the volume of smoke inhaled from a single cigarette. Users don't often realize the volume of smoke they're inhaling because the water cools the smoke, making it less irritating to the lungs but no less deadly.

(END VIDEOTAPE)

GUPTA: Now, it's worth pointing out that the warnings labels proposed by the FDA would flag the risk from hookah smoke as well as e- cigarettes. Nicotine in just about any form is addictive and it's a vicious cycle to break. Remember, tobacco is one of the most difficult habits out there, even more so than heroin or cocaine.

Now, before we go, I want to tell you about this neat project I'm really excited about. It's an interactive that we just launched in our Web site, 10 ideas that are revolutionizing health care. That's the idea, from the operating table to the kitchen table. To learn more about this, you can head to CNN.com/healingthefuture and check it out there.

Time, though, now to get you back into the "CNN NEWSROOM" with Don Lemon.