uncertainty there, but something to watch out for over the next couple of days. >> all right. jennifer, thank you so much. we'll see you back here at the top of the hour. first, a story about getting the worst news imaginable. finding out your child has a rare disease. >> 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 is virtually on their own. for nearly four years, the hemples 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 >> reporter: as a dad of three daughters, i can tell you it doesn't get much better than this. >> yea!

>> reporter: a time you can never recapture. in most families, the kids grow up on to bigger and better things. for addison and cassidy hemple and her parents, hugh and chris, life took a different turn. >> it was really frustrating because, you know, it's difficult when you know something's wrong, and then you can't really get an answer. >> reporter: this is a couple that hates to lose. chris was a basketball star at uc-berkeley. hugh played hockey at the university of vermont. >> ultimately ended up at netscape, the internet company, and that's where i met chris. >> reporter: they were on the cutting edge at netscape when the company, when the whole internet, was just getting off the ground. >> i was in the pr department, so i'd call media people and talk about the internet. and they had no clue what the internet is, what e-mail is. >> reporter: 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, you know, and just kind of take it down a notch. >> reporter: 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, you know, toys. >> reporter: 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. >> reporter: eventually dr. caroline hastings, a specialist at children's hospital of

oakland in california, diagnosed the twins with a rare genetic disorder, niemann pick type c. it's got a nickname that says it all. childhood alzheimer's. is that a fair comparison? >> i think that's a fair comparison. that's how the kids look. they appear to be growing and developing normally. they've developed speech this had they walk, they walk, they have good quality lives, and then they start losing those abilities. >> reporter: niemann pick is caused by a faulty gene that helps the body process cholesterol. you might not realize it, but cholesterol is made and used by nearly every cell in the body. in a patient with niemann pick, the cholesterol accumulates, especially in nerve cells until it reaches levels that are toxic. >> it causes abnormalities in movement and thinking and swallowing, and children usually die in their midteens. >> reporter: 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 hemples got the news about adddy and cassie, things were starting to change. there were patient support groups popping up everywhere, and there were also thousands of research papers, as chris found, available with the touch of a keystroke. >> my computer wouldn't allow me to search, so i found this disc. >> reporter: chris pored 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 it texas. they had a colony of mice with niemann pick, and they were giving them a compound. the mice were living nearly 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 febreze, it's in tide.

>> reporter: in febreze, it binds to molecules in the air that cause odor and eliminates them. could it find a cholesterol in the twins' bodies and do the same thing? 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, and then this is the one -- >> reporter: 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. >> reporter: this video shows cats with the feline version of niemann pick type c. the cats on the left are shaking and staggering. the cat on the right has been treated with cyclodextrin. what did you think it meant pore

your own children? >> it's very easy to translate that to your own kids because you see them suffering, too. it reinforced why we were doing this and the potential and even the risk that we needed to take. we were willing to do anything and take that risk. and you know, without risk, there's no reward. we miss out on the things that matter today. ♪ at axa, we offer advice and help you break down your insurance goals into small, manageable steps. because when you plan for tomorrow, it helps you live for today. can we help you take a small step? for advice, retirement, and life insurance, connect with axa. with a favorite book is nice. for advice, retirement, and life insurance, but i think women would rather curl up with their favorite man. but here's the thing: about half of men over 40 have some degree of erectile dysfunction. well, viagra helps guys with ed get and keep an erection.

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of the more than 6,000 known diseases, we only have treatments for about 500. and there's a rule of thumb for the companies who make new medicines. 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 hemples, it's just another challenge to overcome. ♪ >> reporter: it was more than a year after the grim diagnosis, and the twins were getting worse. the hemples 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, you know, making the right kinds of connections and just trying to move things forward. they mix this with saline. >> reporter: the girls were 4 now, and hugh and chris were feeding them cyclodextrin, a form of sugar.

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. >> reporter: the twins seem to improve. but it certainly wasn't dramatic. >> sometimes just going through old videotape, and i see and i can see the difference. so it's just a constant reminder. >> reporter: there were reasons to hold out hope. the hemples had donated skin cells to the national institutes of health. >> this is our drug-screening robot. >> reporter: in a special lab, a giant robot tested various compounds on the cells. >> and put it in the automated microscope here which will tell you whether the drug or the chemical had any effect. >> reporter: they looked at 3,000 different drugs at 15 different concentrations.

cyclodextrin was among just a handful of chemicals to show 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 hemples would need sign-off 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 hemples just needed to get their hands on it. >> once we realized that johnson & johnson had the safety data we needed, then we contacted them. >> reporter: 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. >> reporter: 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. >> reporter: cassie's now getting the injection 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 in there. >> i've done it a few thousand. >> reporter: you've done it a few thousand. over the next year, each girl had about two dozen infusions. >> it's possible that maybe some

things might be reversible. >> reporter: and an amazing thing started to happen. the girls seemed more attentive. they had fewer seizures. they had lost a lot of their hearing. >> oh, what are you mad about? >> reporter: new tests found it was better. remember, this is a disease that never gets better. >> i think if 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. >> reporter: but make no mistake, addi 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' 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-thinking 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 very, very well understood, very rapid path to demise anyway. >> reporter: it was about to be three years of back-and-forth. did people call you guys excessive risk takers? >> oh, yeah. >> reporter: who are these people? people online? >> scientists. and for them, it's not -- i mean, 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, you know, to each their own. >> reporter: in early 2013, the fda finally gave permission to implant the pump. the girls were 9 years old. would it make a difference? ♪

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april 2013. the hemple 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 noticeable and they have nice, thick hair. >> i think it's going to hit me

once they are really ready to go in. >> are you being a good girl? yeah. see you when you come out. >> it's going to be different. >> for how long have you been waiting for this day? >> gosh, years now. i just can't even believe it. >> and how are you feeling? >> just overwhelmed, really. i mean, some of these tears i'm a little scared but it's a lot of tears of joy, too, that we're finally going to get a permanent solution. >> now it's in the hands of dr. peter sun, implanting a small pump to deliver cyclodextrin straight to the brain. it might be a new lease on life. it's also brain surgery with an inherent risk and no guarantee of what it might do.

>> this is the thing that they have been waiting for for years and it's officially in. the operation has just been performed right now. that reservoir for which they are going to put this medication is done. it's 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 is well. >> yay. >> but the feeling didn't last. >> 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 starting 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 awful. the helicopter takes off. we're in tears, in shock, hop in the car and we just started

racing 100 miles an hour down to oakland. >> so it does make you do some soul searching, was that the right decision? i can say for sure 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 addie has done better. >> how are the girls going? >> i would go out on a limb and say that they are doing better than they were two years ago. >> they look bright. >> yes. it's a brightness. 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 would never really look at us. they would be all over the place. now they hold the gaze. >> here comes our grandpa. >> after two injections of cyclodextrin in their spine, they were almost deaf and their

hearing is almost normal. neurologically, that just doesn't happen. >> over the last four years that i've been following the hemple 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 hemples have another dream. it's that some day, somehow they will find a way to talk with the girls. >> goldfish. >> would you like to have some goldfish? good choice. >> 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 there's this technology that's 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 a click of a mouse. >> what i really want to get to is where they can tell us what

they are feeling and where they are hurting because right now we have no way of knowing and so when she starts bawling, it's really, really scary and daunting because we don't know what to do. i mean, it's like, how do you know what to do? >> but i also know, for all they have accomplished, getting to this point isn't enough. and so the hemples are asking, what's next? one path they settled on is deeply controversial. medical marijuana. >> almost done. >> chris and hugh say cannibis oil has decreased the girls' seizure. and it's without the thc, the chemical that gets you high. they were able to get their medical marijuana license to sell in the state of marijuana. >> this is it?

>> this is a little overwhelming. >> we're in the cannibis business in order to have medicine for our children first and foremost but what we quickly discovered is there are a hundred other families that either can't get or can't afford the medicine. >> here we go. >> 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! >> 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 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 we're sitting here on a sugar compound that's essentially nontoxic that's in febreze and it's going into children's brains now and could be the lifesaver that we're looking for. >> in 2012, the national institutes of health watched a trial of cyclodextrin in children with neiman pick, very similar to what the hemples tried. i'm always struck by what a few committed people can do to change the world. in this case, the parents' determination to help a loved one. the human factor is brought to you by cancer treatment centers of america, care that never quits.

good morning. good to see you on this sunday. i'm christi paul. >> and i'm victor blackwell. we begin this morning in ferguson, missouri, where residents are anxiously awaiting the grand jury to reconvene tomorrow. they could decide whether the officer who killed unarmed michael brown could be indicted but it could extend to the january 7th deadline. 12 members of the grand jury are having to sit through so