tv Sanjay Gupta MD CNN November 22, 2014 1:30pm-2:01pm PST
manufacturers to prompt users to change that default password automatically. if you're very paranoid just put some tape over that webcam. back to you. >> i like that idea, laurie. thanks some. cnn news room continues at the top of the hour. "sanjay gupta m.d." starts right now. there are giant pharmaceutical companies with thousands of employees who spend billions of dollars trying to crack the genetic codes and develop treatments for diseases. the couple you're about to meet? they're virtually on their own. for nearly four years the hempills have allowed me to follow along on a journey where the word "no" is not an option. ♪ twinkle twinkle little star how i wonder what you are ♪ >> as a dad of three daughters, i can tell you, it doesn't get much better than this. [ cheers ] >> a time you could never
recapture. in most families the kids grow up, onto bigger and better things. for addison and cassidy hempill and their parents, hugh and chris, life took a different turn. >> it was really frustrating. because it's difficult when you know something's wrong and then you can't really get an answer. >> this is a couple that hates to lose. chris was a basketball star at u.c. berkeley. hugh played hockey at the university of vermont. >> ultimately ended up in netscape, the the internet company, that's where i met chris. >> they were on the cutting edge of netscape when the company, the whole internet, was just getting off the ground. >> ways in the p.r. department. so i'd call media people and talk about the internet. they had no clue what the internet is, what e-mail is. >> in 1999 the young couple cashed out their stock options, got rich, and left to work for themselves. >> we decided to have kids.
and i really wanted to be a soccer mom and pta and all that. and just kind of take it down a notch. >> a few years later, the twins were born. for the first year and a half they seemed healthy. the first problems were subtle. >> we started noticing they started having problems with balance and coordination. and not seeing things kind of in front of them, like toys. >> and then it got worse. much worse. some of this is hard to watch. stumbles, lost words. the doctors were reassuring at first. less so as the months went by. >> this is easier. usually i'm listening when they're accessed. >> eventually, dr. caroline hastings, a specialist at children's hospital of oakland, in california, diagnosed the
twins with a rare genetic disorder. niemen pick type c. it has a nickname that says it all. childhood alzheimer's. >> is that a fair comparison? >> yes. it's how the kids look. they appear to be developing normally, develop speech, they walk, they talk, they have good quality lives, and then they start losing those abilities. >> niemen pick is caused by a faulty gene that helps the body process cholesterol. cholesterol is made and used by nearly every cell in the body. in a patient with niemen pick the cholesterol accumulates, especially in nerve cells, until it reaches levels that are toxic. >> it causes abnormal tis in movement and in thinking and swallowing. and children usually die in their mid-teens. >> and there is no known treatment. it is the worst news imaginable.
a generation ago, a diagnosis like this was a pretty lonely thing. but by the time the hempills got the news about ad addy and cassie there were support groups popping up and research groups found available with the touch of a key stroke. >> my computer wouldn't allow me to search. so i found this disc. >> chris poured through hundreds at a time, printing them out and sticking them into her big pink binder. in late 2007, chris read a paper from scientists in texas. they had a colony of mice with niemen pick and giving them a compound called cyclodextrin. the mice were living twice as long. people knew about this stuff but they didn't think it was medicine. it was basically filler for lab experiments. it's in consumer products, too. >> it's in chewing gum. it's in fa breeze.
it's in tide. >> in febrez it binds to odors and eliminates them. could it bind to cholesterol in the twins's bodies and eliminate them? what's more it seemed safe. it was sugar. that's it. just a form of sugar. >> i think this one is the one that's fine. >> but hugh and chris weren't truly convinced until they saw this, a remarkable video posted by dr. charles veet, a veterinary researcher at the university of pennsylvania. >> these cats essentially have cholesterol metabolism just like the twins. so they have the same kind of genetic defect that addy and cassie have just naturally. >> this video shows cats with a feline version of ni,men pick type c. the cats on the left are shaking and staggering. the cat on the right has been treated with the compound. >> what did you think it meant for your own children? >> it's very easy to translate that to your kids because you
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of the more than 6,000 known diseases they only have treatments for 500. for every 10,000 compounds you patent you can expect one success. that's just one drug on the market. after you've only got a few hundred potential customers, there's no way to get a return on your investment. for big pharmaceutical
companies, rare diseases look like a big black hole. for the hempills it's just another challenge to overcome. it was more than a year after the grim diagnosis, and the twins were getting worse. the family had no time to lose. >> for one doctor and her support staff and two parents, it's a daunting task. so we have effectively like a little tiny biotech or something running out of our kitchen. >> we spend a lot of time on our computers, making the right kinds of connections and trying to move things forward. >> they mix this with saline. >> the girls were four now. and hugh and chris were feeding them cyclodextrin, a special form of sugar. >> we tried the different products. you tried everything before you gave it to your children.
>> yes. i mean, we do that all the time. i mean, most of the things that we can get over the counter, natural supplements, we do try it. i've tried their seizure medications, some of these medications. just to see what it feels like. >> the twins seem to improve. but it certainly wasn't dramatic. >> sometimes i'm just going through old videotape, and then i see and i can see the difference. but so it's just a constant reminder. >> there were reasons to hold out hope. the hempills had donated skin cells to the national institutes of health. >> this is our drug screening robot. >> in a special lab, a giant robot tested various compounds on the cells. >> put it in the automated microscope here which will tell you whether the drug or the chemical had any effect. >> they looked at 3,000 different drugs, at 15 different
concentrations. cyclodextrin was among just a handful of chemicals so though any effect. but according to animal research, to get the best chance of success you needed to inject it straight into the nervous system. the hempills would need signoff from the fda, the food and drug administration. the fda wanted safety data, and typically that could take years. but with cyclodextrin, that data already existed. the hempills just needed to get their hands on >> it once we realized that johnson & johnson had the safety data that we needed, then we contacted them. >> the problem was, johnson & johnson wouldn't hand it over. it was a trade secret. sharing data? that should have been the easy part. you can imagine the anger these parents now felt. chris let it out on her blog. >> all we need from them is to provide our doctors and the fda with more information from their files that could help save the lives of addy and cassie and
possibly 500 children around the world afflicted with niemann pick type c disease. i guess the world will soon find out how much johnson & johnson cares about kids who are dying. >> johnson & johnson heard the message loud and clear. the very next morning, they came through. just a few weeks after that, the fda gave the go-ahead. >> i still to this day vividly remember that first infusion and how scary it was. even though we knew fundamentally it was a safe thing. >> cassie is now getting the injection of cyclodextrin directly into her spinal fluid. not into the blood but directly into the spinal fluid. where it may have more of an impact. pretty good shot getting that needle right in there. >> i've done a few thousand. >> you've done a few thousand. >> over the next year, each girl had about two dozen infusions. >> it's possible that some
things might be reversible. >> and an amazing thing started to happen. the girls seem more attentive. they had fewer seizures. they'd lost a lot of their hearing. >> what are you mad about? >> new tests found it was better. remember, this is a disease that never gets better. >> i think it's the girls were not receiving the cyclodextrin that they would be in a much worse predicament. and it's even possible that they may not be with us at this time. >> but make no mistake. addy and cassie were still very sick. chris and hugh had been talking to researchers. and they now wanted to try a new method, a tiny pump implanted in the brain. it would deliver a steady stream of cyclodextrin. maybe the best hope to save the girls's lives. but the fda pushed back. their concern? it was too dangerous. >> when i received the call from the fda, i had one of those
heart-sinking moments. >> they don't want to get themselves in trouble because they let us do something that hurt our kids. but from our perspective, our kids are on a very well-understood, very rapid path to demise, anyway. >> it was about to be three years of back and zblort did people call you guys excessive risk take snrs. >> oh, yeah. >> who were these people? >> scientists. and for them it's not so much -- honestly, the parents in particular, lots of parents sort of said, oh, my god, i would never do that. i mean, they're monsters. they're experimenting on their children. my answer is, to each their own. >> in early 2013, the fda finally gave permission to implant the pump. the girls were nine years old. would it make a difference? laxyo down.
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april, 2013, the hempill family finally has permission to perform the surgery they hope will save their twin daughters. >> hi, i got your doggy, look! >> i'm shaving the hair just around the incision so it will be fairly unnoticable. they have nice thick hair. >> i think it's going to hit me like once they're really ready to go in.
>> are you being a good girl? yeah. okay, addy, i'll see you when you come out. >> it's going to be different. >> so how long have you been waiting for this day? >> oh, gosh. years now. >> at least. >> i just can't even believe. >> it and how are you feeling? >> just overwhelmed, really. i mean, some of these years i'm a little scared. but it's a lot of tears of joy, too, that we're finally going to get like a permanent solution. >> now it's in the hands of dr. peter sahn, implanting a small pump to deliver cyclodextrin straight to the brains of addy and cassie. it might be a new lease on life. it's also brain surgery with inherent risk and no guarantee of what it might do. >> this is the thing that they've been waiting for for
years. and it's officially in the operation is just being performed right now. but that reservoir through which they're going to put this medication? that's done. been a long road. >> it's been a very long road. it's hard to even remember the beginning. >> the operation, did it go just as you planned? >> the operation went perfectly. >> no problems. >> all was well. >> yea! >> but the feeling didn't last. >> chris, what happened? >> oh, geez. so after the operation, everything was going smoothly. we ended up getting the girls home. and it was kind of midday. and all of a sudden cassie started vomiting. they sent us over to the hospital. and they did a scan. and they could see that she had a massive brain bleed. it was just -- it was awful. the helicopter takes off. we're in tears, in shock. hop in the car and we just started racing 100 miles per hour down to oakland.
>> so it does make you do some soul searching about was that the right decision? i can say for sure that i wouldn't even today make a different decision. >> another operation saved cassie's life. but even now she's still paralyzed on her left side. her sister addy has done better. >> hugh, how are the girls doing? >> i would go out on a limb and say they're doing even better than they were two years ago. >> they look bright to me. >> yeah. it's a brightness. and that's before in their eyes you just couldn't see that. and for us, it's eye contact. our kids would always look around us. they'd never really look at us. they'd be all over the place. it's like now they hold the gaze. >> here comes our grandpa. >> after two injections into their spine of the cyclodextrin, the twins were almost deaf. and their hearing went almost back to normal. and that's unheard of from a progressive neurological disease. that just doesn't happen.
>> you can see that it actually says new drug. >> over the last four years that i've been following the hempill family, i've seen it over and over. find a solution. try it. make it more widely available. they even helped set up a business to market cyclodextrin. >> what are you going to choose, cassie? >> the hempills have another dream. it's that someday, somehow, they will find a way to talk with the the girls. >> goldfish. >> would you like to have some goldfish? good choice. >> we like goldfish. >> this just might be a step in that direction. >> and since the girls can't talk they can just use their eyes. so they stare into the screen. and this technology is able to pick up where their eyes are looking. so once they focus in on a picture, it kind of makes that selection. so you think about it like the click of a mouse. >> what i really want to get to is where they can tell us what they're feeling, and where they're hurting.
because right now we have no way of knowing. so when she starts bawling, it's really really scary and daunting. because we don't know what to do. it's like how do you know what to do? >> but i also know for all they've accomplished, getting to this point isn't enough. and so the hempills are asking, what's next? one path they've settled on is deeply controversial. medical marijuana. >> almost done. >> chris and hugh say cannibis oil has cut down the number of seizures that the girls suffer. >> good job. >> and as with most families i've met, the hempills use a strain without thc, the chemical that gets you high. just recently, they were awarded licenses to grow, process and sell medical marijuana in the state of nevada. >> wow. here you go. >> this is it, huh? >> yeah. it's a little overwhelming. >> check out this planned growing site. >> we're in the cannibis
business in order to have medicine for our children first and foremost. but we quickly discovered there's another 100 families who either can't get or can't afford the medicine. >> here we go. whoa! >> whoa! >> when your kids have been near death for almost a decade, well, it's impossible to describe what that does to the parents. to chris and to hugh. >> good job! >> you did it! >> but they know the work they've done has also paved the way for children all over the world. brazil, the netherlands, spain, japan, and so many countries parents are infusing their children with cyclodextrin. no small thanks to this mom and dad. >> we really could have the first drug in history that's stopped a progressive neurological disease in animals. so we don't know how that's going to play out in the patients. but i mean, when you think about that, there's just nothing for
alzheimer's, als, parkinson's. and there's been millions of dollars poured into these diseases. and yet here we are sitting on a sugar compound that's essentially nontoxic that's in febreze. and like it's going into children's brains now and could be the life saver that we're looking for. >> in 2012, the national institutes of health launched a trial of cyclodextrin in children with niemann pick, very similar to what the hempills tried. so far now the safety looks good enough that they are planning for a bigger study. i'm really struck by what a few committed people can do to change the world. in this case, a parent's determination to help a loved one. >> "the human factor" is brought to you by cancer treatment cents of america. care that never quits. you used to sleep like a champ.
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hempel. good evening. you're the cnn news room. i'm poppy harlow thrive evening in new york. we have new developments in ferguson, missouri. a grand jury wants more time to consider whether to indict officer darren wilson in the fatal shooting of an unarmed black teenager, michael brown. the 12-person panel will reconvene monday at the earliest, we are told. and in ferguson, some businesses are boarded up, people are on edge awaiting this decision. thousands of demonstrators are poised to react to whatever the grand jury decision is. now they must wait at least a few more days. just hours ago, michael brown's father handed out thanksgiving