"(theme music playing)" "This week on "Vice,"" "the search for a cure for ALS." "Robert Hebron:" "We're fighting the clock." "What I don't know is if there's enough time for my daughter." "How many people in the US are getting your drug?" "In the US, nobody." "The FDA is worried about safety." "Every day that passes, he's losing more of himself." "♪ ♪" "(train horn blows)" "(yelling)" "Stand up!" "Stand up!" "Don't shoot!" "In 2014, the Ice Bucket Challenge raised more than $220 million for research into ALS." "(people screaming)" "Shane:" "Now, it put this fatal neurological disease in the national spotlight for the first time since Lou Gehrig's famous diagnosis in 1939." "(echoing) Today is the day" "I consider myself the luckiest man on the face of the earth." "(crowd applauds)" "But for us here at "Vice,"" "this was already a very personal story." "♪ ♪" "My name is Angelina Fanous, and I work at "Vice."" ""The Worst Part of the Ice Bucket Challenge is the People Criticizing It."" "(people screaming, cheering)" "Critics of the Ice Bucket Challenge dismissed it as viral narcissism, where people were more interested in showing off than helping out." "I wrote the article because the campaign raised hundreds of millions of dollars and it brought attention to a cause that had been widely ignored for over 75 years." "But that wasn't the only reason I wrote it." "(people screaming, cheering)" "Fanous:" "I was just having trouble typing at first, turning on my blow dryer..." "you know, the little things, and I didn't think anything of it until one day I went to spray sun-tan lotion, and the sun-tan lotion slipped completely out of my hand." "And I remember looking down and thinking, "Oh, my god, this is what paralysis looks like."" "Within a week, the best-case scenario went from shoulder surgery to spinal surgery to something we can't treat." "And I didn't know what "something we can't treat" was." "Like, what is that?" "Like, would I just have, like, a really fucked up left hand for the rest of my life?" "Until my neurologist gave me a phone number to call for a follow up." "He's like, "You need to call this for a second opinion."" "So, the next day, I called, and the woman who answered said," ""Thank you for calling the ALS and Lou Gehrig's Disease Center at Columbia."" "And I dropped the phone." "And I just sat on my bed." "I cried." "(sniffles)" "People now know what ALS is, but most don't understand how quickly the disease destroys your body and how hard it is to find any kind of treatment." "So, I set out to do a non-traditional piece of journalism:" "Reporting from the patient's perspective and showing firsthand the challenges we face." "I started with one of the first commenters on my story:" "Robert Hebron and his daughter Beth." "Hi, my name is Beth Hebron." "I'm here doing the Ice Bucket Challenge." "(screams, gasps)" "(Beth speaking)" "(chuckles)" "(chuckles)" "You know, it started out with she needs help cutting her food." "Okay." "You get over it, you deal with it, you move on." "And then it's like, okay, she needs help unzippering her pants to go to the bathroom." "Okay." "Learn how to do it." "Deal with it, move on." "To lose that autonomy and your independence... nobody gives that up easily." "You hang on to that as long as you can." "(people cheering)" "(Beth speaking)" "(clears throat)" "I really believe this will be cured someday." "You know?" "What I don't know is if there's enough time for my daughter." "And I really, you know, kind of hope and pray there is." "But we're..." "we're fighting the clock." "Fanous:" "ALS researchers are fighting the clock too, racing to find an effective treatment." "To see what progress they've made," "I visited the world's first research center solely focused on this disease:" "The ALS Therapy Development Institute." "Dr. Steve Perrin gave me a candid explanation of what ALS patients are up against." "What happens when you get this disease?" "Unrelenting muscle loss from wherever you get your first symptoms to spreading across the body." "Dr. Perrin:" "The motor neurons that control your movements, the brain of the cell is up in your spinal cord." "It sends these processes down out of your spinal cord into your muscles to where it actually innervates the tissue." "When that nerve pulls back, the muscle no longer has any signaling from the brain, if you will, on how to move and your muscles progressively just atrophy throughout your entire body, and that's ultimately what ends up causing paralysis" "and eventually death." "Yet your central nervous system and your brain is, for the most part in most patients, unaffected, meaning you have full cognitive function." "So, you know exactly what's going on around you, and yet you have no ability to communicate, speak, or move." "Is there even a viable treatment for it right now?" "There's no effective treatment or a cure today." "There's some on the horizons." "We're optimistic we're gonna get there." "Fanous:" "ALSTDI received only $4 out of the $220 million raised by the Ice Bucket Challenge, which they immediately channeled into the most promising drugs." "A big part of our research program at ALSTDI is pharmacology." "Um, so basically, that's trying to understand how therapeutics are working in a whole animal system." "We focus on mice." "And the way we use the animals is, number one, to try to understand the disease, but also, and maybe more importantly, we're trying to find treatments." "The first mouse that we're looking at, it has the gene in it that would cause the disease." "But it hasn't had onset yet." "So, you can see he's a little spunky." "Fanous:" "So... how far progressed is this mouse?" "This one is probably about three-quarters of the way through its disease progression." "It doesn't have a normal gate with its hind limbs anymore." "It starts to drag its toes a little bit." "And ultimately, it won't be able to use its hind limbs at all." "A lot of our effort is to test therapeutics to make this mouse basically have a better, longer life." "We've tested a lot of drugs... probably somewhere on the order of 300 different compounds." "We've had three show efficacy up until now, and those are our clinical trial candidates that we're moving forward right now." "Fanous:" "Clinical trials are how drugs are tested before they're released to the public." "And because there's currently no proven treatment for ALS, these trials have become the only chance for patients to access drugs that could help them." "But for patients like Matt Bollina, just getting into a trial is a battle in itself." "Bellina:" "You don't want me to push you?" "You want to do it all on your own?" "That's good." "Bellina:" "I really think of, like, the kids and my wife as the victims of the disease because, you know, they're ones that have to pick up the slack and change their lives." "(child speaks)" "Bellina:" "Buddy, you know I can't." ""Please, Dad." See, this is, like, the saddest thing in the world, isn't it?" "He's like, "Please, Dad." "Just come run with me."" " And I'm, like..." " (child 2 yells)" " Sorry, dude." " Aww." "You know?" "Doesn't that suck?" "Bellina:" "I was flying for the Navy." "I was flying Prowlers." "And then I started having symptoms and the Navy didn't know what it was, so they... they grounded me." "They said, "You can't fly anymore."" "Fanous:" "What was, like, the plan of action for treatment?" "Did you look at experimental stuff?" "I had already started about a year before that, taking stuff, 'cause I had a feeling that I had ALS." "So, I had been taking a bunch of experimental stuff already." "What about clinical trials?" "From the day I was diagnosed," "I had already had too many years worth of tests to make..." "I can't get into any 'cause I have two years since onset." "So, I can't get into a clinical trial." "Oh, wow." "So you were diagnosed, and you were, like..." "Immediately outside of the exclusion criteria." "It's... it's miserable because, like, you want to be part of the solution too." "Like, you want to be providing data to the scientists, but you can't do it." "It's ridiculous." "Like, I..." "I'm like, "I..." "I've been, you know, on and off" ""risking my life for 10 years." "Let me try some drugs that are gonna potentially help."" "I'll take the risk, and I'm..." "I'm not scared of it, you know?" "Fanous:" "For most rapidly fatal diseases, the standard drug approval process is a hotly-contested issue with many terminally-ill patients willing to accept the risks that come with an unapproved drug." "One of the most coveted trials is called NurOWN." "It slowed down the progression of the disease in 92% of the patients who received the full treatment." "The BrainStorm trial at Mayo accepted 16 patients." "We had almost 1,000 patients on the waiting list." "Fanous:" "Like many ALS patients," "I applied to this trial and didn't get in." "And push down." "Hard, hard, hard, hard, hard, hard..." "Fanous:" "But Kevin Haas was one of the very few who did." "When they called and said that I can have that spot, um, I just couldn't believe it, because I thought," ""How many tens of thousands of people are praying and hoping for the same chance?"" "Dr. Windebank:" "One of the important steps in the BrainStorm trial was that the cells come out of the bone marrow and then they're treated in a way that makes them protective for nerve cells." "They're kind of enhanced stem cells." "You might feel a little pressure in your abdomen now, okay?" "Dr. Windebank:" "He'll have a spinal tap, and that allows us to inject the stem cells into the fluid around his spinal cord." "Sir, are you doing okay?" "Yes." "Dr. Windebank:" "And when you have an injury anywhere in your body, those cells will move out of the bone marrow and they go to the area that was injured and they aid the healing process." "Now, we're taking these healing cells and we're putting them into the nervous system." "So, we're putting them into a place where they don't usually go." "I don't know, it's kind of, like, surreal." "I remember sending my paperwork to get into this trial." "(sighs)" "I don't feel like crying on camera today, so I think we should probably cut." "Fanous:" "Due to the limited scope of these trials, many ALS patients, like Wissam Mejed, are left with no other option than to go abroad to seek treatment." "(Wissam speaking)" "Bureaucracy and... and all that is getting in the way right now, and the FDA is worried about safety." "I mean, the side effect of ALS is death." "It's not right." "I mean, every day that passes, he's losing more of himself." "And that goes for every ALS patient." "Yeah, he's willing to take the risk, which, I mean, as far as safety goes..." "Yeah." "Yeah." "Yeah." "If there were options here in America, we'd prefer to do it here, but... there are no options right now." "Fanous:" "Wissam was forced to leave the country." "His family and friends raised $30,000 to send him to Lebanon for an experimental stem cell treatment that works in a similar way as NurOWN." "We're in Beirut and we are on our way to see Wissam the day before his big stem-cell procedure." "Fanous:" "What are you expectations for tomorrow?" "No, that's my goal too." "You can kind of see it in my left hand." "Also in my right hand I've atrophied a little bit." "No, I know." "(crying) I know." "Fanous:" "We followed Wissam as he prepared for treatment." "How are you doing, buddy?" "Hey, how are you?" "I'm good." "How are you feeling?" "Fanous:" "Oh, yeah." "Hopefully they find a cure soon before anything happen." "I love him so much." "I don't want to lose him." "Fanous:" "I wanted to understand why patients like Wissam who are severely disabled are forced to travel halfway across the world just to access treatment." "So, I met with Dr. Janet Woodcock, the director of the FDA's" "Center for Drug Evaluation and Research." "For a disease like ALS, where a drug can work for one person and not necessarily the other because no two ALS patients are alike, what's the harm in just releasing a drug if it's safe?" "First of all, most drugs have liabilities." "If FDA would just release a lot of ineffective drugs out there, we would never find out... we'd never find out which ones are harmful, we'd never find out which ones are beneficial." "We know this from long and bitter experience." "But if a patient is willing to take the risk?" "I spoke to a Navy jet pilot who has the disease, and he's willing to sign off on it." "I'm willing to sign off on it." "Again, what is the harm of releasing a drug that we're willing to take the risk for?" "Well, we don't want to stand between people trying investigational drugs." "Okay?" "So, there's a..." "large access programs that FDA runs where we agree that people who wish to try investigational products can." "Sign on to that." "We agree with that philosophy." "Fanous:" "The FDA program is called Expanded Access, and it allows patients to try unapproved drugs." "But in the months it often takes to complete the paperwork," "ALS patients can lose their ability to walk, speak, or breathe on their own." "Exhausted by the process, patients often give up." "And as we learned from Rich Casey, the CEO of a company that developed a highly promising drug known as NP001, even innovative drug companies need revenue to keep their research going." "But in the US, drug companies are not allowed to charge for unapproved drugs, and that leaves American patients out in the cold." "Fanous:" "How many people in the US are getting your drug through compassionate use and how many people are getting it in Europe?" "In the US, nobody." "And the reason it's available there and not here is that their laws allow us to be reimbursed for our costs." "'Cause we obviously don't have the financial resources to just give it away." "And in Europe, we can get reimbursed for our costs." "Here in the US, you can't." "It's against the law to sell an... an unapproved drug." "How did it feel when ALS patients who responded to your drug came to you and asked you for the drug?" "It's... it's a horrible, horrible feeling." "I mean, you feel like less than human." "Because these..." "these people were doing well." "And of course I wanted to give it to 'em, but there was no way." "We... we had no money at that time." "Um, there was questions about whether we would survive as a company." "It's a really heart-wrenching, you know, discussion with these patients." "Um, and then of course, you know, most of them end up dying, so it's very..." "it's tragic, you know?" "Fanous:" "MIT economist Dr. Andrew Lo thinks the real issue here is that the FDA's current approval system treats all diseases the same." "So, he developed a complex formula that judges risk on a disease-by-disease basis." "You're saying that not all diseases should be treated equally." "ALS is a good example." "Patients might be willing to take a bigger risk of getting a drug that may not work if there is some chance that that drug might work." "Whereas somebody who is taking acne medication may not be willing to take any kind of a chance for adverse consequences." "So, the idea is to use this mathematical framework that we developed and to be able to come up with a single number that says," ""Here, this is the threshold you ought to use" ""for approving drugs for this disease," ""whereas for this other disease that's maybe not as serious, you can use a different threshold, a more conservative threshold."" "Fanous:" "Lowering that threshold would not only increase access for patients, but also incentivize pharmaceutical companies to invest in these treatments." "Dr. Lo:" "Drug companies, when they think about investing in a particular drug development program, they've got to factor in how much they're going to put into it versus how much they can get out of it." "And if there's a lot of uncertainty about whether or not they can get a drug approved, they may not invest dollars initially." "If on the other hand, we change the standards for these terrible illnesses, then drug companies are willing to invest more, we might get cures sooner rather than later." "Fanous:" "If this theory is correct, the adoption of the technique would lead to faster approvals and more trials... which would open up the possibility of treating or even curing a whole host of diseases." "That first effective treatment for ALS is going to be the biggest game-changer we've ever seen." "And ultimately because ALS does look like other progressive neurological diseases..." "Alzheimer's, Parkinson's, Huntington's... some of these drugs will also work there." "So, the investment that was made to do this hard work in ALS is also going to reap benefits in other patients with significant unmet needs as well." "Fanous:" "Without re-evaluating how we approve drugs for diseases with no viable treatment, the only option patients have is how to spend their remaining days." "(computer voice)" "(chuckles)" "Fanous:" "Eric Valor is paralyzed from the neck down." "A machine pumps oxygen into his lungs and eye tracking technology is his only means of communicating." "I need to feed him." "Ooh." "Because he doesn't burp sometimes, when we open the tube, it burps for him." "Fanous:" "Three times a day," "Eric receives his meals through a feeding tube, which is inserted directly into his stomach." "Eric's mom:" "Look at his arms." "It's just literally skin and bones." "Same with the legs, and so we have to fight to keep weight on him." "Fanous:" "Still, Eric is in constant danger." "(Eric triggers alarm)" "When he triggers this alarm by scrunching his cheek, it means he's choking on his own saliva or mucus." "When Eric needs to be suctioned, it's immediate because he can choke to death." "Oh, my god." "Because you have to remember, he can't cough and he can't sneeze, he can't, uh, hiccup, he can't burp." "That's why the caregivers are so close." "Eric has to have 24/7 care." "He cannot be left alone at all." "Not even for a minute." "Fanous:" "This isn't just Eric." "This is a decision all ALS patients will eventually have to make." "And this disease is so aggressive, in the short time since we filmed with Beth, she too is faced with the decision of whether or not to get a tracheotomy, lose what's left of her natural speech," "and indefinitely breathe through a ventilator." "(inhales)" "Doctor:" "Push it out." "Keep going, keep going, keep going." "Okay." "(breathing harshly)" "What are you thinking?" "What's going on in your brain?" "Fanous:" "What I learned from doing this story is that the FDA's one-size-fits-all system doesn't work for neurodegenerative diseases like ALS." "We need wider and better access to clinical trials and experimental medicine here in the United States." "(blows horn)" "The Ice Bucket Challenge has proved people want to find a solution." "And we want to be part of this process too." "Every 90 minutes, someone with ALS dies and another is diagnosed." "The FDA needs to understand that this is urgent and it needs to act." "ALS slowly tortures its victims, and all we're asking for is the fundamental right to save our own lives."