Hacker News Board Hacker News, browsed like a classic message board
Live data from Hacker News

Haemophilia A trial results 'mind-blowing'

bbc.com

Post reply on HN
Print view View original

121–130 of 132 posts

Re: Haemophilia A trial results 'mind-blowing'

#121

Post by DoreenMichele »

wahern wrote: post #106
DoreenMichele wrote: post #97

Earlier quoted context omitted.

So explain to me how 70% of the body's immune cells being in the gut is unrelated to how diseases are typically mediated. Pretty please.

The gut contains 70% of immune cells because your gut is actually the main point of contact between your body and the external environment. The surface area of your gut is somewhere from 20x to 100x that of your skin. One could just as well argue that because the immune system is so well developed and robust in the gut that it's the last place to look for dysfunction. Or one could argue that a 70% share of immune cel…

Thank you for taking my question seriously.

I don't agree, but I appreciate the expanded perspective.

Re: Haemophilia A trial results 'mind-blowing'

#122

Post by baldfat »

test6554 wrote: post #78
baldfat wrote: post #53

Earlier quoted context omitted.

Thankfully artificial pancreas are looking like a good option until we can get to curing type 1. Not only does it save the number of shots like pumps we have today, but it can measure the sugar level and keep the swings down to a minimum. http://www.jdrf.org/research/artificial-pancreas/

They seem good as long as the patient's skin is not allergic to the adhesive used to attach it to the body. I know someone who had an insulin pump that worked perfectly except after a day or two, they would get a rash on the skin and the area would swell up blocking the delivery of insulin. Nothing like all this great technology that could save your life that is foiled simply by the type of glue used by the manufactu…

They go under the skin (Most of the plans I have read)

Re: Haemophilia A trial results 'mind-blowing'

#123

Post by aaavl2821 »

fgimenez wrote: post #119
aaavl2821 wrote: post #67

Earlier quoted context omitted.

i agree, the hemophilia data looks really good, and avexis' spinal muscular atrophy product looks great as well. i dont know much about voyager but just looked at their press release for the study; looks like they are delivering a gene to increase dopamine production? do you know if this could be a disease altering therapy or just a "better" levodopa? its still pretty hard from what i can tell to deliver oligos to sp…

Yes, you could frame the Voyager treatment as "better levodopa". Though, the mechanism would feasibly mitigate neuro-degeneration and improve the terrible dose escalation of the drug. Also correct about general delivery difficulties. Hadn't heard of Biontech's method - almost sounds like voodoo by your description. I think there is interesting blocking and tackling happening on an organ-by-organ basis. E.g. GalNAc fo…

I dont understand the biology behind biontechs spleen targeting secret sauce (i dont think they did either, but it got them a patent on an otherwise standard lipid formulation). there is a really good nature paper from summer 2016 that outlines a lot of their preclinical dev; the CEO said he sort of regrets publishing that as a lot of competitors sprang up afterwards

out of curiousity, what's the exosome hype? ive heard a few strong life sci VCs mention it, but i talked to a ceo of an exosome company and didnt quite understand the specific therapeutic potential

Re: Haemophilia A trial results 'mind-blowing'

#124

Post by fgimenez »

aaavl2821 wrote: post #123
fgimenez wrote: post #119

Earlier quoted context omitted.

Yes, you could frame the Voyager treatment as "better levodopa". Though, the mechanism would feasibly mitigate neuro-degeneration and improve the terrible dose escalation of the drug. Also correct about general delivery difficulties. Hadn't heard of Biontech's method - almost sounds like voodoo by your description. I think there is interesting blocking and tackling happening on an organ-by-organ basis. E.g. GalNAc fo…

I dont understand the biology behind biontechs spleen targeting secret sauce (i dont think they did either, but it got them a patent on an otherwise standard lipid formulation). there is a really good nature paper from summer 2016 that outlines a lot of their preclinical dev; the CEO said he sort of regrets publishing that as a lot of competitors sprang up afterwards out of curiousity, what's the exosome hype? ive he…

They are a phenomenally cool drug delivery vehicle as you can get differential uptake based on their surface markers. Check this out: https://www.sciencedirect.com/science/article/pii/S221138351...

Happy to geek out more offline. Email in my profile.

Re: Haemophilia A trial results 'mind-blowing'

#125

Post by sorokod »

test6554 wrote: post #80
aaavl2821 wrote: post #75

Earlier quoted context omitted.

hate to be the bearer of bad news, but there are something like 400+ drugs that have prevented type 1 in mice and another hundred or so that have reversed it. none have worked in people the most common animal model for type 1, the NOD mouse model, is notoriously bad. studies have shown that loud music cures diabetes in these mice. mice raised in one lab get diabetes differently than mice raised in another. the immune…

Lab mice are truly living in a golden age of medicine!

The humans in the 15th century lived in the golden age of the plague.

Re: Haemophilia A trial results 'mind-blowing'

#126

Post by king07828 »

patall wrote: post #73
king07828 wrote: post #66

This is awesome. I have Crohn's and am looking forward to an eventual cure. One thing I don't understand is why big pharma and VCs seem to focus on rare diseases instead of a gene therapy for changing eye color or hair color. With eye/hair color gene treatments, patients could undergo the therapy multiple times (if desired) and the pool of patients is all humans (not just rare diseases sufferers). This allows for amo…

I am sorry for that but to be honest your comment suggests that you do not have tiniest idea of how hard medical research is. Not only are the topics you suggest much more complicated than rare diseases (i.e. we do not know the exact molecular process but they are certainly much more complex and in the case of eye color, entirely developmental, so changing the genetic background would not switch your eye color), but…

I am sorry, but to be honest your comment suggests that you do not have tiniest idea of how insulting the first sentence of your comment is. Instead of simply conveying the idea that things are expensive, the first sentence of your comment is styled as a personal attack, which does not add to the discussion and should not have been included. For example, an alternative first sentence, which conveys the point being made without being a personal attack would be "Medical research is hard."

Regarding the substance of your remarks, if 10,000 people have a rare disease then the 100 million dollars it takes to develop that therapy can only be amortized over that small pool, which translates to $10,000 per person, assuming that every person pays for the therapy.

If 3 billion people (a rough estimate of everyone connected to the internet) is the market size for hair/eye color treatment and the 10 billion dollars of R&D gets amortized over that full-size, then the cost is $3.33 per person. If only 10% of people want to change colors, and only 10% of those people are willing to partake in the therapy, then the cost becomes $333.33 per person to amortize the R&D expense, which is in the same order of magnitude as contacts or a salon hair treatment.

Re: Haemophilia A trial results 'mind-blowing'

#127

Post by king07828 »

aaavl2821 wrote: post #74
king07828 wrote: post #66

This is awesome. I have Crohn's and am looking forward to an eventual cure. One thing I don't understand is why big pharma and VCs seem to focus on rare diseases instead of a gene therapy for changing eye color or hair color. With eye/hair color gene treatments, patients could undergo the therapy multiple times (if desired) and the pool of patients is all humans (not just rare diseases sufferers). This allows for amo…

the cost of manufacturing a gene therapy would probably exceed what most people are willing to pay for changing eye or hair color. why use gene therapy when colored contacts or hair dye work, and much more easily reversible in case you dont like it? also, it is incredibly expensive to get a gene therapy through fda trials. there hasnt really been a "true" gene therapy approved in the us yet (excepting car-t), and the…

>the cost of manufacturing ...

>it is incredibly expensive ...

These may be outweighed by the potential market size of 3 billion people (a rough estimate of everyone connected to the internet). If R&D and manufacturing have a total cost of 10 billion dollars, then this reduces to $3.33 per person of the potential market size.

>cant dose these drugs more than once

This may be a showstopper for now. At a certain point, the technology should improve to allow multiple treatments so that a single individual could have red hair for a year, blonde hair for a year, black hair for a year, ...

The cool thing about growing blonde hair instead of dying hair blonde, is that nothing more needs to be done and no further treatments need to be had (unlike dyes). This does assume that the individual has enough patience for the growth process

Re: Haemophilia A trial results 'mind-blowing'

#128

Post by king07828 »

Kalium wrote: post #68
king07828 wrote: post #66

This is awesome. I have Crohn's and am looking forward to an eventual cure. One thing I don't understand is why big pharma and VCs seem to focus on rare diseases instead of a gene therapy for changing eye color or hair color. With eye/hair color gene treatments, patients could undergo the therapy multiple times (if desired) and the pool of patients is all humans (not just rare diseases sufferers). This allows for amo…

I can think of a couple of reasons. First, they think of themselves as companies that address diseases. They are concerned with the ill, rather than people seeking cosmetic changes. Second, I can see some potential for major backlash. Literally going into the business of designer babies? Ethically tricky. Doing it only for the rich, because it'll be incredibly expensive at first? "Tricky" doesn't begin to cover it. P…

>they think of themselves as companies that address diseases. They are concerned with the ill, rather than people seeking cosmetic changes

Agreed. The joke being that black hair (or blonde hair or red hair) is a disease that can be "cured" by a different hair color :)

>backlash ... [against] designer babies

Agreed that this is an issue, especially if the treatment were marketed at unborn children. It may be better to focus on cosmetic treatments for adults to try and avoid this.

I expect this technology will eventually become ubiquitous in our lifetimes, with the only question being the time frame for which it will come to pass

Re: Haemophilia A trial results 'mind-blowing'

#129

Post by king07828 »

frgtpsswrdlame wrote: post #70
king07828 wrote: post #66

This is awesome. I have Crohn's and am looking forward to an eventual cure. One thing I don't understand is why big pharma and VCs seem to focus on rare diseases instead of a gene therapy for changing eye color or hair color. With eye/hair color gene treatments, patients could undergo the therapy multiple times (if desired) and the pool of patients is all humans (not just rare diseases sufferers). This allows for amo…

>I have Crohn's and am looking forward to an eventual cure. Check this out, it has a lot of interesting information: http://www.crohnsmapvaccine.com Could be interesting although obviously still in phase 1.

This is awesome!

from the link:

>Historically, MAP in humans has been difficult to study as it cannot be seen under an ordinary microscope and is very difficult to grow. Testing for MAP by the presence of its DNA (using PCR) has found MAP in up to 92% of Crohn’s patients but until now no-one has developed a test to show MAP in-situ in the tissues of people with Crohn’s disease.

It is fascinating that there is a virus that evades regular diagnostic techniques (i.e., using a microscope). That the virus can be found using genetic testing speaks to the power of genetic technology and how revolutionary the true understanding of genetics will be

Re: Haemophilia A trial results 'mind-blowing'

#130

Post by king07828 »

pishpash wrote: post #69
king07828 wrote: post #66

This is awesome. I have Crohn's and am looking forward to an eventual cure. One thing I don't understand is why big pharma and VCs seem to focus on rare diseases instead of a gene therapy for changing eye color or hair color. With eye/hair color gene treatments, patients could undergo the therapy multiple times (if desired) and the pool of patients is all humans (not just rare diseases sufferers). This allows for amo…

How much can you charge for cosmetic changes that can be easily done by less expensive means? I'm sure they've done the math.

>cosmetic changes that can be easily done by less expensive means

The less expensive means (contacts, hair dyes) are temporary and require maintenance. The benefits of a genetic treatment is that it is permanent (no cleaning of contacts, no subsequent salon visits), but should also be reversible

Post reply on HN