Category: Uncategorized

Almost A Year Ago — We Said This Day Was Upon Us… Sickle Cell Is Cured, And FDA Approved — But At ~$2.2 Million Per Patient? Who Will / How To Pay?!

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We first covered this, just about 11 months ago — and it is excellent, ground-breaking news, for Vertex, CRISPR Therapeutics and blue bird bio. But it ultimately may not mean too terribly much — for people of limited means, worldwide, with the life-long disease.

Even so, this is a watershed moment, after centuries of early-deaths, and more recently, life-long disability — largely inside the communities of Black African origin, in the US, UK, Europe and elsewhere… in addition to Africa itself. This is truly remarkable news. We could see sickle-cell relegated to the dust-bin of history the way polio was.

The real problem though, lies — as with all genetic therapies — in the daunting cost to be “cured”: best estimates put the cost of cure, including a year or more out of commission… at over $2.2 million a million dollars per patient. In the US, that may make marginal sense, because the costs of a life-time dealing with the effects of (uncured) sickle-cell, greatly exceed the therapy’s likely costs.

But in the developing world, there is not likely to be any way to incent any provider to deliver it, unlike the very cheap COVID-19 vaccines, by comparison. So it is likely that sickle-cell will continue to be scourge, outside North America and Western Europe. Still — this is an amazing achievement for biotech / life sciences. Here’s the latest:

…The U.S. Food and Drug Administration (FDA) on Friday approved two gene therapies for sickle cell disease, making one of them the first treatment in the United States based on the Nobel Prize-winning CRISPR gene editing technology.

Casgevy, developed by partners Vertex Pharmaceuticals, and CRISPR Therapeutics, and bluebird bio’s Lyfgenia were approved for people aged 12 years and older….

It is, in fact… a functional… cure.

Now you know (and as long time readers recall, we covered Vertex closely, as the second legacy graphic implies — for a decade of Hep C cure market battles).

Be excellent to one another; for we — as a society — now face some very important choices: should a sickle cell sufferer (largely in communities of color) be any less advantaged, in the public health reimbursement scheme… than say a sufferer with solid organ tumors / cancers? I submit that we need to confront this, directly — as Keytruda rumbles ever onward, at ~$22 billion a year. Onward.

नमस्ते

The ISS Turns 25… A Quarter-Century Of International Space Science!

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This celebration will continue for a few weeks, but it is one for the books. An international cooperative venture — including cooperation between scientists and astronauts whose countries are increasingly hostile to one another (i.e., Russia… and just about the entire Western World) — is a reason to have hope, for the future of humanity.

If the science leads, there is little we as a global community cannot achieve. That is the central lesson of the ISS. This first module was Russian; the next was a NASA contribution… and in the decades since, Canada, the EU, Britain, India and others have provided immeasurable support, as well as… space science personnel / astronauts and staffers.

Space botany, human research, and bioprinting were among the recent — and worldwide broadcast — science endeavors demonstrated aboard this wonder of solar powered space engineering. Despite occasional hiccups, the station remains a vital laboratory in the sky, through a quarter century. Here is just a bit of the story, at NASA — with video below:

…Growing plants in space is critical as astronauts prepare for longer missions farther away from Earth. Space agriculture may help feed crews and provide a cleaner breathing environment aboard spacecraft and space habitats. Crews will have to be self-sustainable relying less on cargo missions packed with food, fuel, and supplies from Earth.

NASA astronauts Loral O’Hara and Andreas Mogensen from ESA (European Space Agency) took turns servicing the Advanced Plant Habitat replacing environmental control components on the research device. NASA Flight Engineer Jasmin Moghbeli assisted the duo reconfiguring the botany facility that has grown a variety of vegetables in the Kibo laboratory module….

O’Hara and Moghbeli then joined Flight Engineer Satoshi Furukawa from JAXA (Japan Aerospace Exploration Agency) for eye checks that are part of the CIPHER suite of 14 human research experiments….

Microgravity gives researchers the chance to learn how humans, machines, and materials react to months-long stays. It jumpstarts medical research, proves material strength, and generates new markets for items that can only be made in orbit….

This is a model, for those paying attention at home, and around the globe. Please emulate it. Onward.

नमस्ते

The Aging Hubble ‘Scope Will Safely Resume Star Gazing… Tomorrow: NASA

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This is indeed decidedly good news.

Last week, NASA put the ‘scope into safe mode, while it evaluated a gyro- issue. The ‘scope will now resume imaging — using the three remaining good gyroscopes. Excellent — and honestly… surprising — news:

…NASA plans to restore the agency’s Hubble Space Telescope to science operations Friday, Dec. 8, following a series of tests to gain insight into the gyro performance that caused the spacecraft to pause science operations last week.

After analyzing the data, the team has determined science operations can resume under three-gyro control. Based on the performance observed during the tests, the team has decided to operate the gyros in a higher-precision mode during science observations. Hubble’s instruments and the observatory itself remain stable and in good health….

That is — absolutely — an unexpected holiday gift. I had pretty much guessed that this would be end of mission, for the now nearly 34 year old prodigy. Some times, the cold of space — along with constant radiation blasts, over three decades… end the circuits’ ability to function. But that day is not… today. Grin.

नमस्ते

In Which Hinderaker Continues To Try (To Appear To) Change His Stripes. But That Tiger Is Far Too Long In The Tooth, Now.

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John thinks he’s found a winning wedge issue: paint all who are not hard right GOP as… wait for it!… Jew-haters.

Uh-huh.

John apparently simply missed the memo that… many, many Jews are very liberal — perhaps most of them (having seen what authoritarian / totalitarians like Hinderaker can do, if not opposed — by clever but open minds). And I daresay, even more of them self-describe as middle of the road Democrats (though my experience is they are on the left side of that road).

Sure — there also are quite a few who are hard right (at least as to internal Israeli affairs — “Bibi believers“)… and most of those folks tend to have the highest overall net worth, in the world-wide Jewish community.

So, obviously — Hinderaker sees a potential NEW donor group (he’s never had deep donations from Jewish factions) ones that might save his silly, bitter and now struggling… blog.

So that is — at bottom — all there is to see, here.

Cheers.

Texas Hellscape Update — Feckless TX GOP AG Paxton, On Civil “Bounty Hunter” Cases — If Doctors Do Provide Women’s Health Services…

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This is… deplorable.

After the order I mentioned earlier today, GOP MAGA AG Ken Paxton issued a statement to threaten abortion providers, under other (likely unconstitutional) laws Texas has passed. It purports to enable a bounty hunter law, to allow private citizens to report on, and go after any abortion provider or anyone who enables the same. [Sheesh. Hellscape meets Stazi indeed.]

Here is that late-breaking ghouls’ report:

…Within hours, Attorney General Ken Paxton issued a warning of his own. “The Temporary Restraining Order (“TRO”) granted by the Travis County district judge purporting to allow an abortion to proceed will not insulate hospitals, doctors, or anyone else from civil and criminal liability for violating Texas’ abortion laws,” he said in a statement.

“This includes first degree felony prosecutions.”

Paxton acknowledged that Cox’s OB/GYN, Dr. Damla Karsan, was shielded by the order, the TRO “does not enjoin actions brought by private citizens” — a blatant threat to people who seek or provide abortions….

What a completely churlish pig. Out.

नमस्ते

[U] Finally Got Around To Reading The Dec. 1 DC Opinion That (Unsurprisingly) Holds Tangerine Doesn’t Hold An Absolute Immunity Card.

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Updated @ 9:40 PM EST: Tangerine is trying to delay his trial (again!) on these felonies, by taking an immediate appeal of the below ruling — he says nothing else can be decided until he reaches the Supremes on immunity. Balderdash.

The able Judge Chutkan has set a very short fuse tonight, on his appeal, thus:

. . .MINUTE ORDER as to DONALD J. TRUMP

Upon consideration of Defendant’s [178] Motion for Order Regarding Automatic Stay of Proceedings Pending Appeal, it is hereby ORDERED that the government shall file any opposition to Defendant’s Motion by 5:00 p.m. on Sunday, December 10, 2023, and that Defendant shall file any Reply by 5:00 p.m. on Tuesday, December 12, 2023. Signed by Judge Tanya S. Chutkan on 12/7/2023. . . .

End update.

USDC Judge Chutkan has written a very fine opinion here, in 48 pages.

It is spare in its prose — and elegantly courtly… harkening back to an era when federal judicial opinions were scathingly lethal… largely as a result of the civility and aplomb with which they eviscerated felonious actors.

So too, here. Trump has been… filleted, quite nicely. He will lose on his specious mid-process appeals, as well. This is solid black letter law… devastatingly delivered largely because of how free from any hint of exaggeration it is. [In this regard, you may want to directly compare and contrast the below — with any USDC opinion we’ve discussed out of Texas, in the last two years.] Here is the whole PDF — and a bit, for spice and flavor of it:

…Those concerns do not carry the same weight in the context of a former President’s federal criminal prosecution. First, the Supreme Court has largely rejected similar claims of a “chilling effect” from the possibility of future criminal proceedings. During the Watergate prosecution, President Nixon argued that if recordings of his conversations were subject to criminal subpoena, the Presidential decision-making process would be compromised because his staff would be less candid. Nixon, 418 U.S. at 705–06. The Court disagreed, stating that it “cannot conclude that advisers will be moved to temper the candor of their remarks by the infrequent occasions of disclosure because of the possibility that such conversations will be called for in the context of a criminal prosecution.” Id. at 712….

Indeed, the possibility of future criminal liability might encourage the kind of sober reflection that would reinforce rather than defeat important constitutional values. If the specter of subsequent prosecution encourages a sitting President to reconsider before deciding to act with criminal intent, that is a benefit, not a defect. “Where an official could be expected to know that certain conduct would violate statutory or constitutional rights, he should be made to hesitate.” Harlow v. Fitzgerald, 457 U.S. 800, 819 (1982). Consequently, to the extent that there are any cognizable “chilling effects” on Presidential decision-making from the prospect of criminal liability, they raise far lesser concerns than those discussed in the civil context of Fitzgerald. Every President will face difficult decisions; whether to intentionally commit a federal crime should not be one of them….

Yup. He’s soon to be… a convicted felon — in at least two separate jurisdictions, around the east coast.

Of The Hellscape That Is Texas State Law, Post Dobbs… Damn.

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This is the story of a married woman in Texas — a mom of two, already — who wishes to have another child, but the current zygote she’s carrying has a rare genetic defect — one that almost always results in a still-birth (all based on actual life science findings). This in turn means she would endure astonishing agony (both emotional and physical), should she be forced to carry to term (as Texas’s unconstitutional law… now plainly states). Her doctor (understandably) fears being jailed in Texas — if he does what is considered medically needed by all responsible MDs nationwide, here.

I know: you may say… but she can fly to Colorado, and solve it.

No… instead, she and her husband have courageously brought a very public declaratory judgment action in Texas, likely in part to show the world… what a fascists’ state Texas has become. Texas believes it has the right to jail doctors… for protecting the life of a mother. Here is the NBC News story, at noon-time today:

…A Texas judge on Thursday granted an emergency order allowing a pregnant woman whose fetus has a fatal diagnosis to get an abortion in the state.

Late last month, Kate Cox, a 31-year-old Dallas-area mother of two who is about 20 weeks pregnant, found out that her developing fetus has trisomy 18, a rare chromosomal disorder likely to cause stillbirth or the death of the baby shortly after he or she is born. . . .

Judge Maya Guerra Gamble quickly granted the requested order, which also allows Cox’s doctor to perform the abortion without fear of prosecution.

“The idea that Miss Cox wants desperately to be a parent, and this law might actually cause her to lose that ability is shocking and would be a genuine miscarriage of justice,” the judge said.

Johnathan Stone, an attorney with the Texas attorney general’s office who represented the state in the hearing, argued that Cox and her husband had not sufficiently demonstrated that they would suffer “immediate and irreparable injury” without an abortion.

“The only party that’s going to suffer an immediate and irreparable harm in this case” if the judge grants the requested order, Stone continued, “is the state….”

Now that is some Orwellian nonsense, Mr. Stone — you now claim that Texas owns a property right, in a particular woman’s uterus? You’ll have to answer to your maker for that, I guess… “THE STATE”?! Damn.

नमस्ते

Bill Otis Agrees With Scott Johnson: Tangerine Will Be The GOP Nominee. But Both Are Hilariously Wrong, After That.

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Look. I really don’t care. They are… delusional, as to the ultimate outcome, though. The both of them.

Of course Tangerine leads in every poll they cite. [But all those polls were wrong, in 2016 (missed HRC negative votes — will be Trump’s negatives in 2024); and they were wrong about 2020 (again underestimated Trump negative votes) and 2022 — well, that was a… tidal wave for real democracy — and a woman’s right to control her own uterus.]

That same effect… is magnified for 2024.

Democrats are playing the low information GOP pundits perfectly: let them think Tangerine has a cake walk.

Keep the dark horse Haley off the podium. And then… no matter whom the Democrats nominate, the GOP is over. Done.

And Johnson is right about this much: Trump will be a felony convict — perhaps even serving a sentence — when election day rolls around.

Charming — oh you feckless GOP regulars.

Out, grinning.