New Cystic Fibrosis Treatment a "Game-Changer" | SciShow News

[@ScarletMarie0007]

Both my siblings died from complications due to CF. My sister didn't live past 17 and spent half Her life in the hospital. She died 7 days after her birthday at home in 2004. She was so weak she had to be carried and helped in the bathroom, was on IV and feeding tube treatments and oxygen. My brother got to age 22 but his last two years were spent chronically ill and in pain. He died in the hospital before Halloween 2012 after having to be revived 4 times. CF is no joke and gets such little attention RIP loved ones. We will always miss you

[@BRUXXUS]

Sj B I’m so sorry for you and your family’s losses. That sounds really horrible. Hope this news means it won’t have to happen to more.

[@davestylehenry]

I born in 2004

[@ThirdEyeAngel]

Sorry for your loss. Two brothers I’ve known almost my whole life have been fighting CF and the younger brother passed away recently. They are two of the sweetest people i’ve ever met and I really hope the older brother can get this new treatment. I feel your pain my man.

[@PhantomQueenOne]

Wow, I'm so sorry 😥

[@ViviontheGo]

Do you have cf too?

[@sylvestersstudio9403]

MY MOM JUST SIGNED THE PAPERWORK TODAY IM GONNA BE TAKING THIS MEDICINE

[@r.ridderbusch7303]

Congratulations, Sylvester! :-D

[@tammyelizabeth5157]

Good luck on the insurance and funding. And I hope you see wonders in your life.

[@sylvestersstudio9403]

@@tammyelizabeth5157 thank you! My costs are cut because I was part of the trial for the predecessor, and disability covers the rest

[@ht21]

Omg congrats!! Grab those PFT percentage points back!!

[@sylvestersstudio9403]

@@ht21 hell yeah! FOV1 in the 80s here I come!!

[@Sciencerely]

As a human biologist and researcher I really believe that cystic fibrosis will also be one of the first diseases we will be able to cure through gene therapy (I covered this topic in one of my videos). As explained in this video, cystic fibrosis is caused by mutations in a single gene (CFTR, this makes it comparatively great to treat using gene therapy). Through gene therapy, we can introduce an intact form of CFTR DNA into lung cells, which is then integrated into the genome of the patient. And this has already been done to improve the lung function of cystic fibrosis patients (although the effect is relatively small so far)! Feel free to ask me anything about that topic (or stem cells, since this is my field of research)!

[@TheArchaos]

While nature may have given us this form with all of its flaws, I think it is high time we correct all these random chance disease more permanently. I know its a trip fraught with peril but for the betterment of all suffering mankind, its a risk worth taking.

[@balthiersgirl2658]

I honestly hope so

[@Joe-xp6un]

@@gregorymalchuk272 Lmao, of course thats the first actual question for this guy. I'll probably save you the trouble and tell you more than likely not, the tissue that makes up the foreskin is filled with incredibly intricate, tightly packed, and highly sensitive nerve endings, that just simply aren't feasible to recreate with our current technology, and will probably be a HUGE pain in the ass to make with jank experimental future technology. If we do ever get anything close to re-attaching a human foreskin, it'll probably just be a bit of flesh to make you feel better about having the tip of your wiener hacked off, it will probably be painful, pale, sad-looking, and not exactly functional, not unlike modern SRS. You're better off doing what people have been doing for the past 200 years or so trying to get their foreskin back: Harbor disdain for society's double standard regarding genital mutilation and get on with their life with slightly less of your wee-wee.

[@TitanUranusOfficial]

This is *why* such treatments are so expensive right now, not just for CF, but for other conditions. The drug companies have to recover their R&D and make a profit *now* because bio-hackers with laundry-room labs are already working on ways to fix these problems.

[@allhumansarejusthuman.5776]

Ok. Here's an interesting question for you. Can induced pluripotent steam cells be utilized to redice or eleminate the effects of aging without a cancer risk assuming dna and doner cells are harvested before the host ages too much (i.e. gains too many mutations)

[@SzornFox]

29yo CFer here. This has been life-changing... I'm now going on walks WITHOUT my oxygen tanks, it's amazing. We in the community are super excited about this, but there's so little attention to this outside of our groups... Thank you. Here's hoping the price comes down &/or it becomes accessible outside the US...

[@apextroll]

Congrates!! It likely will be fast tracked outside America and given free until it is covered by government healthcare or private plans. They are doing this for many new biologic medications like humera, nucala or fasenra.

[@AnnaAngeli2]

This is super exciting! I'm a 33 yo CFer. I haven't been able to start taking it yet. I'm so happy for you, especially being able to walk without your oxygen tanks. That's huge!

[@hayleysnider3735]

apextroll You’d think that, but many countries do not cover the older modulators. It’s honestly a crime against humanity in my opinion.

[@ht21]

Congrats!!! May your PFTs be high and steady!

[@malikmuhammedfaisalaamirpa2501]

i am father of CF daughter 13 year old she is very sick i am from pakistan we have not medican for CF can anyone help me to save on life

[@swordzofgodz]

My brother has CF, he’s doing amazing. He recently got a lung transplant, God bless Canada!

[@nicholasneyhart396]

Hope all is well.

[@Nancy-pc6sf]

Wow👏👏👏💕💕

[@malikmuhammedfaisalaamirpa2501]

i am father of CF daughter can you help me how your brother is covering

[@Emre.55]

311k a year just to be able to live like a regular human being... I don't even have the words to sum up my thoughts on this.

[@logmover123]

That's capitalism for ya.

[@DarkParagon]

I have many words to sum up my feelings on the people over pricing this; but I'd like this comment to not get deleted for profanity so I'll only say: Leeches.

[@vsolyomi]

That's an emergent technology cost. As it's refined it will hopefully get cheaper. Mass production is unlikely though, as there're not that many people.

[@saintchuck9857]

@@vsolyomi cheaper like insulin?

[@gypsysprite4824]

or you can move to any country that is not the USA...

[@CBlakeDavis]

Cystic Fibrosis patient here: I started the drug exactly a month ago and it completely changed my life. A few notes: It's pronounced "Try-KAFF-tuh", not "Try-COFF-tuh" Yes, this is a game changer. Some CF patients who were on the list for a double lung transplant have now been taken off the list because this drug made such an impact. My lung functions (i.e. air force, capacity, strength) increased by 9% within 5 days. My 2+ hours of treatments a day are MASSIVELY easier. My joints don't ache as much, my energy has increased tremendously. This is a MIRACLE drug.

[@phantomphan006]

The timing of this. We just had to get our son tested for CF this week. It was negative, but I'm glad to hear of the developing science around the disease.

[@dube7729]

Hey 90% is no laughing matter. If they came out with something for Ms that affected 90% of MS sufferers I would say wow

[@user-yn9mp4bt3q]

Ocrilauzumaub. Clinical trail is 90% effective so yes. My roomate has not had a single relapse in 5 years. It is now on the market approved. 0 symptoms 0 side effects 😎

[@mjb405]

There is a relatively new drug for MS that's been very effective for many people. It's called Ocrevus. I don't know how well it works for all types of MS but I know it's at least effective for relapsing remitting MS. The main downside is how damn expensive it is. It could cost me up to $65,000 a year if my insurance didn't cover it

[@user-yn9mp4bt3q]

Wow

[@user-yn9mp4bt3q]

@@mjb405 my roomate was in the clinical trials. It is truly amazing. We are receiving it free now because of the trial but the cost once the trial ends is scary.

[@user-yn9mp4bt3q]

@@mjb405 if you can afford it get it. It has been a life saver for us. No comparison to the old ineffective drugs.

[@davetoms1]

5:53 "Just really, really sad. And that resonates with me." The way Hank said "me" tore my heart out of my chest and kicked me in the nuts. I wanna give him a hug.

[@ambermiller3590]

One of my closest friends have CF, I’ve seen how they’ve struggled throughout the years. She’s doing great now! Playing sports in high teams and everything. This is so exciting for he r

[@malikmuhammedfaisalaamirpa2501]

can you pass her contact to get information

[@ross335]

If only Claire had made it long enough to see this :(

[@wcurty336]

This is actually sad

[@commonlowlife8085]

Whos clair?

[@Fellow_Traveller1985]

@@commonlowlife8085 I believe she was a youtuber with CF.

[@jacobmortimore]

Dude I legit came to comment this....😭

[@emmajo5443]

This is what I first thought :(

[@kirasinclair1061]

My sister-in-law has CF and is very excited about this! (As is the rest of the family!) Today is her birthday and she is 32.

[@92RKID]

Happy birthday for her! The best gift would be knowing she could get access to the med. Here's hoping she can soon!

[@ThrottleKitty]

Scientists: Here's a new game changing medical treatment Company: Cool, multiply the asking price by a thousand and sue any company working on an alternative.

[@konway17]

Throttle Kitty that’s American healthcare for you

[@allhumansarejusthuman.5776]

You forgot. Get paid by the goverment (via grants) to develop the drug. And test it. Then multiply a reasonable profit by thousands..

[@DeandreSteven]

@@konway17* american pharma companies *

[@Soofkin]

They get a 7 year Monopoly on the medicine and after that every company can make that drug Just look at Teva who had a Monopoly on some eye nurve treatment Basically the rule is if you made the drug, you and only you can make it for 7 years... Good time to invest in stocks in that company

[@custos3249]

@@Soofkin Good thing people unable to afford it in those seven years won't die.

[@jackieXblue]

as a successfully treated sufferer of eosinophilic asthma, this made me incredibly happy for my fellow chronic illness warriors. We need to keep fighting, against our conditions and also for affordable access to medication!

[@HistoryNerd808]

It's such an awful disease, it's good to see that scientists have finally found a way to treat it

[@adammorgan1776]

Scishow, I have Cystic Fibrosis (34) and have been on Trikafta (Triple Therapy Drug, Elexacaftor, Ivacaftor & Texacaftor) and the results after just 4 weeks are absolutely amazing. I'm in the UK on managed access but hopefully soon it will be available for all eligible folk. This drug doesn't only improve the lungs but also other areas such as the gut, joints, appetite, general wellbeing and more. Cystic Fibrosis isn't only a lung condition but a multi-organ condition that affects almost all areas of the body. Trikafta does improve lung function, I went from 30% at the start to 57% 4 weeks later (normal range is 80-120%). Here's the thing though, lung function isn't the most important thing, it's a good indicator of where your lung health is but that's all it is, there are more important things like CRP levels (infection levels, normal range is 0-6) of which mine was sat around 50 and around 100 on jnfection requiring IVs. Lower CRP, less chest bleeds and less chest infections means less lung damage. I'm no longer needing to be listed for lung transplant. One thing Trikafta (and other such meds) can't do is repair the damage already caused but it does open up the airways in your lungs by helping to thin out and remove the sputum blocking the airways, I removed 0.5kgs of sputum in a week and over 250 plugs. This drug is a real game changer.

[@soulsearching7240]

The world misses you. I miss you. Thank you for blessing me with your precious ‘snippets’ in the short but dense time you spent your life here. Forever in my heart.

[@Town159]

I work in a respiratory lab in one of the biggest UK Adult CF Centres, and one of the few that did these trials. The improvements in FEV1 and Quality of a life have been immense for the handful of patients who are on the trial, the advancements in treatment over the past few years have been amazing!

[@bjs301]

This is great news concerning a horrible disease. When I was a little boy in the early 1960s my best friend had CF. I'd visit him, and his Mom had to spend what seemed liked hours pounding on his back to loosen the phlegm. I remember thinking she was torturing him, but my Mom explained it was the only treatment that helped. There were times he nearly suffocated, and he was the smallest kid in school. With his small size and his chronic cough, he was always getting bullied, at least through third or fourth grade. He somehow did make it through elementary school, but I moved away and later learned he had died. It will be great when kids don't have to suffer like my friend suffered.

[@waterunderthebridge7950]

The cause for the pancreatic maldigestions isn’t actually “thick mucus blocking the path”. In pancreatic tissue CFTR regulates secretion into the pancreatic ducts. Without balanced secretion the pancreatic enzymes don’t get into the intestines properly. That’s what causes the digestive symptoms (in very simplified terms).

[@Kendi-mm7hk]

Thanks for covering this! I have CF, and while the 311,000 sticker price is what shocks everyone, no one is paying that. Vertex does have incredible patient support and co-pay assistance, and most people are paying about $15 a month, or even less. With orphan diseases like this, a universal system doesn't work because there aren't enough patients to justify the cost. The UK just finally approved Orkambi, which has been out since 2015 in the US. I have my appointment monday, and I'm so excited to see what this drug does for me and the rest of the CF community!!

[@pcdeltalink036]

Yeah, currently with my insurance, copay assistance, etc. I’m paying nothing for it. It has helped my life so much!

[@emmatheteachie]

Amazing news, and a really clear explanation of how the drug works (& I learnt more about CF) - thanks!

[@Master_Therion]

$311,000 per year? Pharma companies makes me sick!

[@Reelix]

How much do you charge your employers to work for them?

[@lauren8538]

Reelix are you arguing the researchers don’t get paid until the public starts paying for the drug??...

[@oblivionfire1365]

Coming from someone with CF and F508D I'm really hype to get on this. I've been hearing about its progress for over half a decade and I'm already on the Duel blend drug, doing so of the same work. The duel blend is less then half as effective as this one, and will be a huge step. For those wondering some predictions put the effectiveness as high as a 70% restoration in lung function, which is close to how CF carriers lungs behave. All in all, the numbers support this drug really being a game changer

[@malikmuhammedfaisalaamirpa2501]

please need help for one CF patient

[@hayleysnider3735]

My insurance initially denied this medication, currently appealing (and frustrated). I’m hoping they approve it early next week, and as soon as they do I’m overnighting it! Thank you Hank and scishow team for making this video, been a huge fan for years!

[@Trexrush1]

CFer here. Words cant express how excited I am to be on this. This is a lifesaving drug

[@estel-randir]

This made me tear up. I'm glad that this treatment finally happened.

[@verdatum]

90%? That's nearly unheard of, wow.

[@timmydirtyrat6015]

My uncle Michael died of CF in his teens many decades ago. I hope to god people will not have to struggle through what he had to struggle through.

[@AkaiAzul]

No worries. They’ll find a cheaper and slightly less effective alternative in 5 years and will be made available... To people outside the USA.

[@TheArchaos]

When you let legislation be run by money-crazed lunatics the government tend to end up with such problems. Land of the free it most certainly is, those with 99% of the money.

[@AkaiAzul]

LazyLife IFreak I’m disappointed that it’s happening today with Hepatitis C. We invented a cure, it’s crazy expensive, some one found a slightly less effective alternative 3 years later, it’s like 80% cheaper, and no plans to make it available to the USA because it’s “less endemic there.” That really shouldn’t matter, but here we are....

[@chelsey8737]

LITERALLY

[@mvmlego1212]

@@TheArchaos -- It depends on what you mean by "freedom". The U.S. isn't free from suffering--no society can be--but it _is_ free from oppression, at least more so than any major country now or in the past.

[@spindash64]

Gregory Malchuk You act like that’s an exclusively American practice. And like it’s somehow the most horrible thing anyone could ever do to someone.

[@SultaiSamurai]

I have cystic fibrosis & am 27. Hearing this news from the foundation was overwhelming I cried hearing this. For others who don’t have it it feels like concrete in your lungs & is so exhausting burning so much energy to keep the lungs going. I love medical science & wish those who’ve passed could hear this news

[@malikmuhammedfaisalaamirpa2501]

i am father of one CF daughter you are also suffering from this can you help me about treatment and which medicans are taking

[@vkrys1x]

@@malikmuhammedfaisalaamirpa2501 ask the doctor for the medicine trifikta

[@theoverseer393]

This is a great start. I hope they can become cheaper and be more affordable, and hope they find more treatments

[@ehco7573]

They'll only become cheaper when people prioritise humanity aver wealth. Which, when a lot of people think "greed is good", isn't gonna happen until we can rise up and hold people accountable for the suffering they cause, directly or indirectly.

[@diquedahlia1935]

This is amazing.. but my heart just broke for Claire :(

[@mojave7604]

This is great news! My good friend just had a double lung transplant! She’s still going strong.

[@FunkyGoodVibess]

My cousin has CF and this drug is a miracle for him.

[@soniakhan9472]

Just had a lecture on CF today thanks for the extra reading material

[@hilowize]

I’m glad you guys did a video that explains cf a bit! Finally have something good to show people when they ask

[@Pandora880]

Ive watched this show for a couple years now and im so happy to see them covering something cf related considering how rare it is it's like we're not seen but this makes it feel different ❤

[@jimbrewer498]

They've created a fantastic treatment but priced it out of reach of the people who need it most. Good going big pharma. I live in the northern most part of Idaho, my doctors are board certified in Canada and the US. I fill my scripts in Canada! Way less than half of what they cost just 30 miles south in the US!

[@WillAyeAmNot]

Dude, you're from Northern Idaho and have CF? Exactly the same as me, I just moved recently but I used to live in CDA

[@WillAyeAmNot]

Small world...

[@jimbrewer498]

@@WillAyeAmNot : Yeah, me too.

[@scalpingsnake]

I have Cystic Fibrosis so yay

[@tofusaid]

I’m just here to say Hank Green is a treasure

[@kemphoss-4791]

I second that

[@tofusaid]

Can I third it if I was also 1?

[@kemphoss-4791]

@@tofusaid ill just fourth it then

[@okbamtheman]

I’m so glad my insurance covers trikafta. It is a life changer I used to be in and out of the hospital so much but now I rarely have to go in.

[@kca_randy]

Wow it could help most people with CF .That is awesome. My stepmom has ms her meds are $68k .That new drug is 4 or 5 times as much.Who could afford that ?

[@Call-me-Al]

People outside of USA in countries with universal healthcare? Please vote at every election, not just the presidential one, if you are an American.

[@johncitizen6791]

I am lucky to be in Australia as life saving meds are normally covered by the government. That's also why I pay my taxes.

[@hayleysnider3735]

Il Al actually it’s those folks who have the greatest issues with accessing Vertex medications because their countries won’t pay for them. People in the US generally don’t have an issue and do not pay that much after patient assistance.

[@kalebcrossley5279]

Thanks for bringing awareness to cystic fibrosis I lost a childhood friend this year she was a beautiful girl 18 years old and a beautiful singer xx

[@briesthoughts2261]

Great video! I love how you shared complex and detailed information to those not familiar with the topic. 👏👏👏👏

[@Sarakate601]

I have a friend who has TWO young kids with CF.. and knowing that the lifespan for CF is so short.. and that all these online personalities with it are dying I just.. looked at them like “it sucks looking at them and knowing they probably won’t live until they’re 30” but the I remembered I’d heard of a treatment that had come out just A BIT too late for the youtuber Clair to benefit from it.. so it’s AMAZING that kids today have a real shot.

[@free_spirit1]

Company: 311k/y Patients: i guess I'll die

[@abril561]

This makes me think of Claire Wineland :(

[@tamasv9825]

Me too 😔

[@juliakercsmar6587]

I only clicked to write this comment. I still miss her. I never felt a more profund intellectual bond with anyone. I can't imagine losing her as summon who new her like family members

[@tamasv9825]

@@juliakercsmar6587 nagyon imadtam a szemelyiseget

[@JoseRamirez-xv5ps]

Who?

[@tamasv9825]

@@JoseRamirez-xv5ps She was an amazing youtuber and she had a TED talk too and she died of CF look her up she is amazing

[@luuk341]

This is the stuff we should be seeing on news paper front pages! Great news!

[@mjducasse]

Started my meds tonight! So excited!

[@docsanti8971]

Very well explained! Thanks a lot

[@kelpengineer5303]

I had a dear friend with CF. She knew her time was limited, so she lived every moment to its maximum. I miss her...

[@Eddygeek18]

4:23 Only in the US, I can't believe how lucky i am to live the UK $10 per course of medication that's $120 a year per medication no matter what that medication is. And this is $300,000 a year which is just stupid.

[@Call-me-Al]

Yeah, in Sweden we have a payment ceiling for medicine, and for healthcare. Anything above those is free. Also, a lot of provinces provide free basic health care visits at this point. Taxes (in places with little corruption, sadly we aren't 100% free from it) is the cost of civilization. It makes life better for everyone because they don't have to fear their neighbours as much because their neighbours are going to be less sick and less desperate.

[@jlp6864]

its strange to read comments from americans who are happy to not have universal healthcare, like no one i know who has it would want to give it away for anything. the us "healthcare system" just seems barbaric to me.

[@beth8775]

@@jlp6864 As an American, I desperately want to see a universal system put in place here. So many people here have swallowed all the lies about how it's the only way to fund research, and how so many people die waiting for treatment (as if that doesn't happen here), and how there's no motivation to become a doctor if it won't make you so much $$ (as if new dr's here aren't drowning in student debt & that's not preventing students here from becoming dr's).

[@jlp6864]

@@beth8775 i hope the us will one day have one but i kinda doubt it tbh, what seems more realistic is maybe a few states having universal healthcare? and those arguments are sooo bad, if those people just looked at scandinavian countries they got universal healthcare, theyre rich countries AND many doctors move there to earn more money. but its not even an exclusive us type of thinking, whatever a country is used to will hardly change (fast).

[@injunsun]

I have this mutation, so thank you for the detailed account of how this works. I am a mere carrier but I am slightly affected. A nephew has heart defects that may pertain to this, as the deletion mutation can cause relatively minor organ defects (usually urogenital).

[@DCexpat]

@~4:50 Hank says that whether those with CF will get the drug depends most on insurance companies. Well, kinda, but It's much more accurate to call out the drug manufacturer, who clearly has the most direct control over its own prices.

[@pcdeltalink036]

I’ve been on it for about a year now and it has helped me so much!

[@KateAtNight]

Stephi Lee is a youtuber who has documented some of her journey with CF and she started taking this, it changed everything for her.

[@hhjk377]

Drug makers: we've found a new game-changing drug for CF. CF patients: Wow we may be able to live longer now that- Drug makers: It's 311,000 bucks cause reasons. CF patients: LOL nvm.

[@boredom_gaming_3405]

It’s the people that sell the product rather then the people who make it xd

[@REIDAE]

the "reasons" is the costs of r and d, running a company and salaries. A certain profit margin also must be made in order to satisfy stakeholders investing money into the company but also to grow the company so it can keep developing new drugs. If no money can be made then the no one would be trying to create the cure/treatment in the first place.

[@hape3862]

@@REIDAE Wrong! Most research is done at state universities with tax payer money. Corporations don't bother with r&d as much as you think.

[@REIDAE]

@@hape3862 Search up the actual company (vertex pharmaceuticals incorporated) before opening your mouth. And while youre at it, also go research how much of the national budget goes into pharmaceutical r and d and compare it with the yearly expenses on pharmaceutical company financial statements while also taking into consideration the number of these companies that operate in the US alone.

[@AdamSmith-gs2dv]

@@boredom_gaming_3405 Well the drug company has to pay back researchers, clinical trials, the long bureaucratic FDA approval process, and make up for price controls implemented by Socialized medicine countries. There is a reason most new drugs come from the US, it's the only nation to make a profit from developing drugs everywhere else the government steals that profit in the name of "social welfare"

[@RichardEricCollins]

I've been on kaftrio for 18 months now. Changed my life. I was getting bad, very bad. Start of end. All other meds had stopped working. Within a few months I was well again. I am better now than I was 20 years ago. It is a wonder drug. Has been amazing and changed life.❤

[@veronicadavanzo2064]

This is fantastic news! Excited for everyone who gets to try this out, and hoping that the company will get some competition to help with the price.

[@Trexrush1]

Took my first dose today! Im so excited

[@katerow9019]

They just announce they’re going to fast track this being covered in New Zealand!! When I was born, my mum was told I wouldn’t live past my teens… this is something my parents could have never imagined happening, I’m so excited!!

[@ImMiggy]

Make a video about Tripple E Disease starting to spread around the U.S

[@baboon500]

I learned recently in my pulmonology class about cystic fibrosis and how to treat it. I only knew ivacaftor, lumacaftor and tezacaftor. This is exciting.

[@incognitobye9031]

One of my best friends has cf. I’ve been friends with her for 12 ish years. Last year she started trikafta. 2 months ago when she went to her doctor they said that her sweat levels (how they determine if a child has cf) were below the diagnosis level. She ~essentially~ is cured (using “cured” lightly)

[@sebastianelytron8450]

Not only do I have Cancer, MS, Parkinson's Disease, Cystic Fibrosis, and Alzheimer’s; I went to the Doctor recently and was told that I have _another_ oddly named disease: Hypochondria.

[@zain4019]

Sebastian Elytron I’m really glad for that last word:)

[@jdenmark1287]

That's funny.....but guess what even hypochondriacs die of something, even the illnesses they think they have

[@BRUXXUS]

Well played.

[@sdfkjgh]

4:43 Depending on insurance companies, and the U.S. Gov't's ability to regulate them? We're screwed!

[@BudgetBeautyBabe]

I just started this drug! Thank you for doing this video, it can be difficult to explain how it works when people ask about it.

[@oldaccount6152]

I wish claire wineland had made it long enough to watch this. RIP beautiful soul, we miss you dearly

[@BRUXXUS]

Wait, I didn’t know we had the medical tech to do protein re-folding?! That’s amazing, and probably partly why it’s so expensive. With tech like that, would a cure for prion infection be possible?

[@vsolyomi]

And as those are even rarer iirc it would probably be even more expensive.

[@Jason-bd5iq]

But how would they make money from that. How many people die from prions a year? Also it can’t really be called an infection if prions aren’t alive.

[@_Thoughtful_Aquarius_]

@@Jason-bd5iq , viruses cause infections, and viruses aren't really alive, at least that's my understanding.

[@anarmyofroombas8880]

We are working on drugs to treat Lewy Body Dementia, a disease that is very similar to prion diseases. By definition prion diseases are transmittable, so we havent tackled that problem yet.

[@Jason-bd5iq]

Touché Yeah, there’s a debate as to whether viruses are alive, many biologists say “no” because they can’t reproduce without a host cell.

[@ivovinicius5529]

thank you sci show, you guys are always a source of great learning entertainment and, as i am a medical student in brazil, that will be useful (if the drug becomes cheaper), at least i hope thanks again

[@cutepiku]

Meghan died from CF a few years back at the age of 30, leaving behind her husband and 3 kids. I'd known her since I was born because she was my sister's best friend even as children. CF has always been an important topic to my family because of Meg, a fighter who was told she wouldn't live passed 10. Great to see advancements in treatment. I wish she could have lived to see this. But it's comforting to know that others won't have to suffer the way she did.

[@FlaggedVideoGuy]

I wish the US would regulate the pharma industry. The high cost is likely due to the massive research spending required to fund clinical trials and drug development combined with the small patient population. Its no small task to get something like this to market and could cost a company 1 billion dollars in failed attempts and research ideas. Regardless, allowing any price to be set is careless and downright heartbreaking for patients who could benefit from this medication. Government price negotiations need to be apart of drug approval process in the states as it is for most of the world. A proper health economic model investigated by non-profit agencies or government bodies would regulate this price effectively. There does need to be some incentive for companies to even fund the development of these medications but it should have its limits.

[@AnimilesYT]

"It's not as creepy as it seems. It's just really really sad" Describes my love life spot on.

[@sdfkjgh]

4:23 77% of $311,000 is still $239, 470, so... Pretty sure there's still more price-gouging.

[@jasonscst]

The good news, with all new drugs, is they dramatically go down in price over time as R&D is covered and then new methods/mass production occurs.

[@beth8775]

Tell that to diabetic patients that need insulin.

[@estelsil]

Hooray for Mary Frey!

[@DeathrashWhiplash]

My wife has CF and my boy has a mild form of CF and any children we have will be 50% CF or not, it is a huge deal to me seeing advancements in CF treatments and potential cures.

[@clm652]

INCREDIBLE! So happy for those it helps!

[@Nihilnovus]

Thumbnail looks like my next new tattoo

[@okastbloart8395]

tragoedia veritas and thumbnail

[@FieryRedDonkeyOfHell]

Ok

[@sylvestersstudio9403]

Hell yeah I want a tribute tattoo of cystic fibrosis too

[@sylvestersstudio9403]

@Tom from Myspace you can take inspiration from a cool drawing without copying it completely, I mean cystic fibrosis is a huge part of a lot of people's lives. If it means something to them I don't see why you can't let them have that

[@mikebizz1263]

@Tom from Myspace Seeing that you are somewhat convinced that this is a little kid that wrote this comment, do you think you really needed to go on with a whole paragraph using big boy words and meanings, or do you just like reading what you wrote..?

[@danielleg.1084]

Mary Frey is in this drug!!! It’s doing her wonders!!!!!

[@marie-helenemartel7147]

Coming from a Québec region where there is a high number of people affected by the disease ( we have also 4 others genetic diseases which are very rare elsewhere) I am glad here people won't have to deal with ridiculously expensive meds prices like in the U.S.

[@ancientruins2856]

I think my father is in need for these new drugs & treatment plus future careful studies shall help us better.

[@rj6110]

very exciting, hopefully it can be made accessible

[@carlhopkinson]

Wow, sounds like a very sophisticated approach.

[@undisclosedidentity9893]

I have CF and thank god doctors and scientists are working to treat and cure it rather than promote it as an identity and lifestyle like they do with some other illnesses

[@useful-dustbin3899]

When i run in cold morning i have cystic fibrosis is it normal can i slove it without medicines?

[@ramsdawg]

I saw the thumbnail and thought man, how is that going to help?

[@TwlightDutch]

I was literally at a CF research day talk when the notification of this video popped up. I am now positive that scishow is stalking me.

[@RaeWakefield]

I really like this upload.

[@sierram3141]

The American health care system is sickening. How much are these companies profiting off the sickest of the sick? You could easily make a healthy profit off a drug that is required to be taken for the rest of someone’s life without pricing it at over $300 000 a year. This is greed at its finest.

[@bearcatben4762]

Its not even avalible in canada, its better that it be expensive and available than it just not be available

[@nicholasheimann4629]

Crisper enhanced, for safe insertion site at the correct locus, safe viral vector CFTR gene therapy. It should be easy to aerosolize it into the lungs, use a different viral vector I.V. for the pancreas.

[@AnnaAngeli2]

It's actually delta, not del, at the end of the CFTR mutation's name. Also, this has been such big news in the CF Community. Like Hank, said there are still people who won't be able to take the medication, because their genetic mutations aren't treated by Trikafta. However, it is awesome, and I'm hoping that others with CF will soon be able to take CFTR modulators that do treat their mutations.

[@briantylers5784]

Just started trikafta 2 weeks ago, my lung funtion has already improved from 24% to 40% , although that might not seem like a lot, it sure feels like it! I can breathe so much better, and no junk in my lungs, i'm very greatful.

[@athrunzala798]

Very nice. I was diagnosed with atypical cf a few years ago when I started getting pancreatitis out of nowhere. I still get flare ups on occasion, but my hospital visits are much less frequent

[@VEE3RDEYE]

Stephi Lee needs this

[@WintrBorn]

My mom lost a childhood friend when the girl was in her late teens or early 20s. Back in the 60s, survival rates were pretty abysmal. Every one of these steps is huge.

[@evilferris]

This is wonderful news!

[@mvmlego1212]

Would somebody with some knowledge of business or pharmaceuticals mind explaining why they price the drug so high? I know they need to recoup research costs, but it seems to me that the _more effective_ way to do that is by reducing the price to increase their customer base. The whole deal with pharmaceuticals is that research is expensive, but production is cheap, right? So, if they cut their price by 50% (for example), then they only cut their profit per dose by ~50%. At the same time, it seems it would more than double their customer base (by more-than-doubling the number of people who could afford their product), which would be an overall increase in profit. What gives? P.S. In case this wasn't clear from the way I worded my comment: responses along the lines of "caPiTaLIsm Is eViL" are _not_ answers to my question. I want to know why the current price is optimal from a _business_ standpoint.

[@therealmrtwonuts]

Well in the US at least. Drugs that are developed fall under a patent, and that patent expires after a decade or two. While the company owns that patent, they have exclusive rights to production. This incentives pharmaceutical companies to specifically develop drugs which fall under these catagories: 1. drug is 'life saving' 2. drug is deemed crucial to live a normal life 3. drug will be needed repeatedly 4. drug is going to be prescribed by doctors This allows for that pharmaceutical company to not only recoup the costs of making the drug, but maximize their return on that research investment (since they can decide whatever price they'd like). Hence why these sorts of drugs are what's researched. If it actually cured the individual, it wouldn't be profitable. If you want to see how this sort of business model can have horrible effects on society, i highly recommend you research the prices and patent holders of life saving drugs such as epinephrine, and how out of hand the pharmaceutical market has become. TLDR: Pharma companies are in the business of developing drugs which people need to live, and gain a monopoly on that drug due to patents and can charge whatever they want.

[@beth8775]

It is impossible to rationally discuss this without discussing the corporate greed fueling it. Go look up for yourself just how much pharma corps spend on research vs CEO bonuses or advertising & sales agents.

[@mvmlego1212]

@@therealmrtwonuts -- Thank you for the level-headed answer, but I think you missed the point. I understand that, due to patents, pharmaceutical companies can charge whatever they want without losing customers to other companies. They cannot, however, charge whatever they want without losing customers, period. Some people won't buy the product at all, because they simply can't afford it. By reducing the price of their drug, the company would increase the number of customers who could afford the drug. For a drug that is priced so high that less than .01% of families could afford it, it seems like reducing the price would increase the number of doses sold by a larger factor than it would reduce the profit-per-dose. The overall effect would be an increase in profit.

[@mvmlego1212]

@@beth8775 -- You missed the point by a large margin. There's no need to discuss corporate greed, since I'm already assuming that they're greedy--i.e. that they'll act in such a way that maximizes their profit. What I want to know is how pricing >99.9% of their potential customers out of the market maximizes their profit.

[@AdamSmith-gs2dv]

1. Research is really expensive and involves a lot of failure 2. Finding participates for a clinical trial is expensive and takes a long time 3. The FDA approval process is expensive, long, and beraucratic. 4. Price controls in socialized medicine nation's make selling the drug there unprofitable so companies make that up by over charging us.

[@807pranavghandade8]

It Increases effectiveness in humid countries by 0.05 and also it has higher infectivity than lethality.