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SirT6 1 point

If political news has you down as of late - just remember news from science is 🔥 right now.

Earlier this week, pembrolizumab halved the odds of dying in a first line non-small cell lung cancer trial.

And now today, gene therapy offers a hope for a cure to one of the world’s worst blood diseases. Good video summary of this study here.

A couple things to watch for with this particular study:

  • The drug effectively “cured” (I’m still not used to using that word in these indications) the mild form of the disease. Patients with more severe mutations still required some transfusions. Can the protocol changes the company wants to make move these patients to a cure?

  • Can we build an infrastructure in parts of the world where these mutations are most common (poor countries in Africa, for instance) that enables the drug to be delivered?

  • Cost? No doubt this will be expensive. Can our insurance/healthcare system adjust to paying high prices for curative therapies?

dirtmaguirk1688 2 points

Was this an academic study or done by biomarin, sgmo, once?

SirT6 1 point

This was a BioMarin trial.

Also, if you are interested in gene therapy, see the newest links in this sub - Blubird bio just published an impressive gene therapy paper for Beta Thalassemia.

The least six months have been an incredible ride for the gene therapy field.

SirT6 1 point

This video is based on the results of the New England Journal of Medicine article, AAV5–Factor VIII Gene Transfer in Severe Hemophilia A .

SirT6 2 points

If political news has you down as of late - just remember news from science is 🔥 right now.

Earlier this week, pembrolizumab halved the odds of dying in a first line non-small cell lung cancer trial.

And now today, gene therapy offers a hope for a cure to one of the world’s worst blood diseases. Good video summary of this study here.

A couple things to watch for with this particular study:

  • The drug effectively “cured” (I’m still not used to using that word in these indications) the mild form of the disease. Patients with more severe mutations still required some transfusions. Can the protocol changes the company wants to make move these patients to a cure?

  • Can we build an infrastructure in parts of the world where these mutations are most common (poor countries in Africa, for instance) that enables the drug to be delivered?

  • Cost? No doubt this will be expensive. Can our insurance/healthcare system adjust to paying high prices for curative therapies?

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SirT6 2 points

Read the article and report back: https://www.nature.com/articles/s41588-018-0102-3

Tl;dr: there is a well known association between EBV and risk for these autoimmune diseases; here the researchers provide additional evidence that this relationship may be causal.

peptidyl 5 points

Isn’t this pretty established? Every medical textbook I’ve seen implies EBV genome is present in Hodgkin Lymphoma, Nasopharyngeal carcinoma, and primary CNS lymphoma to name a few.

SirT6 3 points

Plenty of more work to be done understanding the contribution to oncogenesis, participation in tumor maintenance and whether there are any therapeutic interventions which may make sense for EBV+ tumors (just to name a few of the low-hanging fruits).

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SirT6 commented on a post in r/science
SirT6 2 points

Interesting work - I'll be excited to give it a close read later.

That said, I think the field is much more interested in resistance to anti-PD1 therapy than it is anti-CTLA4 therapy. The existing clinical anti-CTLA4 ab is just more toxic and less potent than the anti-PD(L)1 class.

SirT6 commented on a post in r/science
SirT6 11 points

Not provided in the abstract (and I don’t want to pay for the full article), but:

1) How are government services being quantified? Would something like defense spending count?

2) How do they address the obvious correlation != causation argument? Why isn’t it that religious people are less motivated to want big government or any of the dozen various other possible ways to explain the observed corrrelation?

SirT6 commented on a post in r/sciences
SirT6 2 points

Bacteriophages are an interesting case. Lots of hype when they were first discovered. The whole field deflated a fair bit when antibiotics hit the scene. Some new interest now that antibiotic resistance is becoming a bigger concern.

For a sense of where the FDA stands with regards to this therapeutic modality, they held a workshop last summer with some good insights. Transcript here (PDF).

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SirT6 1 point

Adcom briefing documents are “draft” reports in the sense that the opinions of the reviewers may change on the basis of the advisory committee meeting that the report serves as a jumping off point for.

We’ll never see a more detailed take by the FDA on a drug than an Adcom document. Everything contained in the report is legit (though may be contended by the drug sponsor or committee) and usually revised by multiple FDA scientists.

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SirT6 1 point

Rather than a paucity of information it all looks like a thorough and pretty convincing 'little to none' for the side effects and adverse events they looked for.

That's why I love Adcom briefing documents. They provide an unique opportunity to peer behind the curtain of a developmental drug.

I'm still trying to wrap my head around how this sub can have 18.5 million subscribers and 1500 mods but little more than a handful of comments for the most part.

Short answer: reddit is complicated. More subscribers isn't always a good thing.

That said, if you are interested in this type of content then take a look at r/sciences - a subreddit I just started to try to 'modernize' the way science is discussed on reddit.

Cheers!

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It's the terror of knowing what this world is about

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I make drugs for the treatment of cancer. Life science and biotech are my passions. Opinions are my own.

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