tv America Tonight Al Jazeera February 17, 2016 9:30pm-10:01pm EST
ever, curing diseases almost thought to be incurable. and pushing the envelope further, taking on the toughest medical challenges, killers like ebola, zika, and other u undiagnosableth curable. "america tonight's" lisa stark got a look at the one place in america where the seemingly impossible might find reason for hope. >> this is building 10, a surprising generic name for a place that is anything but. the clinically center as the national institutes of health is the largest hospital devoted entirely to clinical research. for many it's the last stop when all else has failed. leaving some to call it the national institute of hope. it is where cutting edge treatments and even cures have been discovered. and where, for the last 10
years, dr. forbes porter has been doing research on a disease so rare there are only about 500 known cases. it is called neiman pick c, or npc. destroying many of the body's devices, the ability to eat even. >> the development of symptoms is slow so it's hard sometimes to pick out that these kids truly have something that's out of the range of normal. >> reporter: these are symptoms julia kane knows all too well. the 15-year-old was diagnosed with npc when she was just 11. >> how does it make you feel? what does it do to you? >> it bounds up cholesterol in the membranes that causes me to forget things a lot. and not memorize things like
other people memorize things. and it causes me to not walk right. like my walking is not stable. >> reporter: yulia's mom tanya knew something was wrong from the moment her daughter started walking. >> she was clumsy, she bruised really easily. when she was three or four, she was doing forward rolls across the floor, when she came upright her eyes would be rolled back in her head, but then they would come back down. >> her concerns would be brushed aside. >> when i took her to the pediatrician, it was like, a first time mother, nothing to worry about. and i wasn't a first time mother. >> and for years she sought answers. and when the tests came back normal the red flags were piling
up. >> when she started kindergarten she had a hard time coming up with the kids. in third grade, we would spend five or six hoou hours a night e page of homework. you had to walk her through every problem step by step and when you got it to the end, she wouldn't know how to do it. >> when tests were showing her i.q. was dropping, she was brought to a specialist. valleyed liver enzyme, which lairts led tledto the diagnosis. >> we were in a strange way excited to finally have a name for it. okay, now we know what it is. >> soon after they made their first trip to building 10 but there was no treatment. just data collection.
>> did you struggle with the idea of bringing your daughter all the way up here and going through all these tests and the poking and the prodding when it wasn't for a treatment, it was for data? >> yes. we have to. because no parent, no child, should have to go through what we just went through to get here. or have to be told, oh, i don't know what to tell you, you know, it's terminal. there's no treatment. that shouldn't come from a doctor's mouth. it shouldn't ever. >> so these parents come to you, they finally found you, they're looking for hope. i'm sure you want to give them hope. but i imagine you have a very difficult cross to bear here because you have to ground that in the reality of neiman-pick disease. >> the parents and the patients deserve to know what this disease is. it is a horrible disease.
so we owe it to them to be honest with them. at the same time, we don't want to crush hope. >> reporter: right now porter is leading an important clinical trial that julia and 13 other kids are part of using a drug called psyc psyclodextrin. >> is this going to be cure? >> i don't think the drug is going to be a cure, it has the potential to help. >> to slow down the symptoms? >> to slow down the symptoms, to buy time to work towards the cure. >> these are not therapies. these are experiments and i mean despite all of the pretrial data
and research that you have, it still boils down to you're doing experiments on kids. is that something that you struggle with? >> you're exactly right. they are experiments. do i struggle with it, no. because how else are we going to help these kids? my role as a clinical scientist is to do that's that experiment in a way that protects that child and gets the data to prove that this drug works or doesn't work. from my perspective, that's my role. role, is to conduct these experiments and to help the parents understand that. >> the kanes do understand that. so once a month they make the trek to florida to building 10 for three or four days of test,
treatment and hope. >> stopped, youth. >> do you notice the treatments helping? can you though any difference in her? >> my husband seems to, more than i. he seems to think that it's helped her balance. i would have to agree with him to an extent, but then there's days, too, when i think really? i don't see it today. (laughing). >> long term i know that it's slowing it down. >> they stay across the street at the children's inn a private nonprofit residential facility that provides a place for entire families whose children are being cared for at building 10. make a snow angel. >> it is a place to feel safe and at home free of charge. >> how important are the relationships with other kids
that you have here? because this is probably the only place where you go where you can talk to kids who are facing similar serious situations like you are. >> i don't know. >> do you have friends here? >> yeah. >> are they different than kinds of friends you might have back holm or at school? >> yes, because some of them have the same disease i have. and some kids have other, different stuff. and my friends at home don't have anything wrong with them that i know of. >> reporter: do you remember life before neiman pick? >> no. >> do you remember being able to run around or remember things easily or remember life different? >> if i looked at something or someone brought something up, i'd probably remember it. but just sitting here, i can't think of anything.
that i did when i was little. >> most parents with their kids they're looking to next month, next year, five years, ten years down the road. is that something you do? do you allow yourself to look to the future with julia? >> my husband does. my husband has dreams and we talk about them. and i try to do that. but it's very difficult. it's very difficult. last year she was a freshman in high school and i remember sitting at that first meeting you know and they're talking about all the wonderful things that are going to be happening over the next four years. and i remember sitting there in the back crying. because i didn't want to look four years. i didn't know what the next four years brought. >> julia says she doesn't worry about the future. for now, she's looking forward to her upcoming 16th birthday and her first prom, hosted by the tim tebow foundation for children with special needs.
>> is it hard sometimes to stay positive? >> yes. >> but you're determined? >> yes. >> so give me the next five year for julia, what do you see? >> a grown up, and a pretty grown up. and i'm going to have a bunch of adopted kids. >> why? >> because i like kids. >> why adopted? >> because i don't think i'd be able to have any that -- with this disease. >> those are going to be some pretty lucky kids. >> uh-huh. >> "america tonight's" lisa stark is here wit lisa fletcherh us. the expenses for treatments like this must be enormous. >> outrageous.
tanya julia's mother tells us every month her expenses exceed $52,000 a month. >> a month? >> a month. >> is that covered? >> the family does have to pay odeductible, but thankfully after that it is covered. >> this is not going to be a cure they understand that but what is the prognosis for helping her? >> it's not good. kids with npc typically don't live beyond their 20s. we have met some younger than julia and not doing as well. the fact that she and 13 others are in this trial, it slows down symptoms, by time, buy time to create other drugs, in concert with this drug they're using now or maybe independently to halt the disease. >> and other children as well. it also seems like it was awfully difficult to get to the
point where the family could get any kind of treatment for her. >> ten years, her mom knew something was wrong and couldn't get told anything other than, don't worry about it it's normal. it was a mother's intuition. they found some small things, all i've got to go on, send united states. they sent her and they examined julia's liver and that's what ultimately led them to a diagnosis of npc. she went to mayo and then to nih where she's participating this the clinical trial. >> "america tonight"'"america ta fletcher. thank you. we follow up with the cost much a cure that could make the difference between life and death at least for those who can afford it. and hot on "america tonight's"
it's here is another way the health e system can be put out of reach for those who may truly need it. americans pay more for prescription drugs than any other nation. there are gross questions about why, sit the hand of the marketplace or something more nefarious at work? "america tonight's" michael okwu
with a big pharma insider telling it like it is. >> martin screlly may be the most honest around. >> capitalist city, capitalist rules. >> but he also one of the most reviled. the backlash started in september when his former company, turing pharmaceuticals, raised the price of daramrim from 13.50 to an astonishing $750, a 5500% harkup basically overnight. it's true for any ceo of a for-profit country. company. >> no one wants to say it, not to minimize them or go half or
70% but to go to 100%. >> while it may have been frankly appalling, the price like wasn't illegal. >> it is legal is the bottom line. >> pilar baum is the ceo of infamous pharmaceuticals. baum took mairchts inside their" inside their laboratories. >> to the outsider, it looks like they've got a product and they basically stick label on it that's the most expensive price they can come up with. >> right. most companies will try get as much money for a drug as they possibly can. >> in response to touring's price like, pharmaceuticals took the country by surprise, releasing daramri to the market for $1 a pill. >> why $1 a pill? >> we are dealing with people in pain suffering that really need these medicines, we hear
directly from them. >> is touring a bully in a playground? >> i think they're taking advantage of a monopoly and that's not the way that pharmaceuticals should behave. >> but they're not the only one to blame. >> touring is not the biggest offender. >> the touring representative presented an opportunity for another approach, one that baum says is ethical and prudent. >> how do you reconcile that you're a businessman, trying the make a profit, and you have a moral obligation? >> we don't do with any of our drugs what you see touring do. we don't do this as a company, it's against our value proposition. particularly with these lifesaving medicines, i have to tell you, i think there are many, many pharmaceutical companies though, that are
empathetic but i just haven't seen that as a part of the discourse with martin shecrelli or a lot of these other companies. >> but infamous also has its critics. >> there are critics who say your pricing of daraprim is a publicity stunt. >> anybody who says that needs to get in the car, drive over to a hospital, and meet a patient that's suffering from one of these diseases. these people can't afford these drugs. this is not a joke for them. >> what's your most expensive drug? i mean i have to assume they're not all a buck a pill. >> we have drugs that are you know quite a bit more than adollar a pill. they do get, you know, we have drugs that our patients spend several thousand dollars a month for. nothing that is $80,000.
we're not one of those companies. >> while baum believes in ethical pricing he stops short of calling for more regulation. should there be some more oversight? >> i don't think that price fixing price caps are necessary. i think companies should be free to price things how they wish. they have something like touring wants to charge $750 for a medicine that day before might have been $13 a pill they have every right to do that. and we just want to have the right to price ours at 99 cents. >> martin screlli touring's form are ceo was arrested and charged with securities fraud. while the charges aren't related to the now infamous price like, the saga could still serve as a cautionary tale for other industry leaders, if not, it is consumers who will continue to pay the price. michael okwu, al jazeera,
so now we've heard the explanation from an industry insider and what he's doing to try the keep the price of drugs within reach but we also know the very sophisticated medical treatments available today can come with some very high price tags and the sickest patients, the ones grit tonesdesperate to be savede the subject of price gouging. one that can now be a killer can
now be stopped in its tracks at least for patients who can afford the price of a cure. >> i had a hip replacement in '05 so they had to do preliminary blood work, dental work blood work blah blah pla, that's wheblah,that's where it c positive. >> at first she didn't recognize how severely her health had been compromised. >> the big fear is your liver? >> my big fear is my liver. because cirrhosis, that's it. and i'm not ready to die. >> and how are you? >> i'm good. >> undertreatment for other unrelated health are props, she asked her doctor about a new hep c drug she saw on tv? >> he says it's the latest thing an everyone is using it and it can treat you between 8 and 12
weeks. >> and it's a cure. >> it's a cure. no remission bit, it's a cure. >> harvoni and orelated drug from the same drug maker, have been proven to be more effective, in cure rates in three months of treatment or less. but the cost of the cure is nearly $100,000, per patient. >> it's very expensive. so even if there is a cut rate discount, it's still very expensive. >> jay costman is her doctor. like most of costman's patients, dundin's health care is covered by state funded medicaid. >> we treat predominantly all inner city low income either insured through public
assistance or uninsured taxpayers. >> that means other patients or tamp strapped patients are left to fit the bill. delaying or make it difficult for hep c medical patients to get their treatments. her application was rejected four times. >> denied completely because harvoni is not necessary because you do not meet the hepatitis c criteria. >> translation, the provider says they won't pay for her medical miracle drug until her disease gets worse. frequently reject help c patients are aren't deemed sick enough. to a patient like dara, it's a creug cruel ancruel and possible threatening rejection.
it is a pragmatic and necessary decision for the courts. matt sale's organization represents the nation's medicaid directors. >> if we were to simply open up the doors and say yes, everyone who is suffering from this disease at any stage at any condition, everyone gets it, we would be spending as much on that one drug as we would on every other single drug in the program combined. >> you are going to bankrupt the system. >> we bankrupt the system. >> even though systems are required to, resisted giving states much of a deal. >> they didn't really want to play because they didn't have to. >> the drug maker gilead, didn't want to are have an interview
but the lifetime cost of treating the disease. but with drug costs already as much as 15% of state medicaid budgets, and more miracle drugs being ineventualitied, analysts say hep c drugs could be just the first test of how far america's health care budget could be stretched and whose cure society is willing to pay for. dara, who has no history of iv drug use, is proof that insurers don't care whether she lives or dies. >> i think this is what drug companies want to you do, get discouraged. >> but she's not going to give up. she finally won coverage for her drugs. now after eight weeks of treatment she reports no signs of hep c. she does face another test next
month just to be sure but sphargs. so far, so good. you can talk to us on twitter or facebook and come back, we'll have more of "america tonight" tomorrow. >> ankara explosion. >> the purpose of this attack is to intimidate our country and to inflict fear into our citizens mind. >> world leaders president erdogan promises retaliation. military buildup? >> there is every evidence, every day that there has been an increase in militarization at a point in time or another.