Yesterday, I received an e-mail with a little video of Jamie Raskin announcing that his cancer is in total remission. Now that’s something to break out a little bubbly for! I also received a note (email) from Pat advising me she has out-of-town family visiting, so will probably not be around this weekend (so don’t worry.) I also received a grocery order. Most of it was there, But there was one substitution, and it was a bad one (which common sense should have prevented. So I had to file a refund claim for that. I got it all in and the shulder was still better after doing so than it was the previous day, so that’s a win. It’s still a work in progress, but I;m liking the progress.
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Politico – Biden’s hydrogen bombshell leaves Europe in the dust
Quote – European leaders have devoted tens of billions of dollars toward encouraging production of hydrogen, a clean-burning fuel that advocates say will create jobs and help fight climate change. But now, many of those jobs will be going to the United States instead. The clean energy subsidies that undergird President Joe Biden’s climate agenda have just prompted one Norwegian manufacturer to choose Michigan, not Europe, as the site of a nearly $500 million factory that will produce the equipment needed to extract hydrogen from water. And other European-based companies are being tempted to follow suit, people involved in the continent’s hydrogen efforts say — making the universe’s most abundant substance the latest focus of the transatlantic trade battle on green energy. Click through for details. I wish no ill to Europe, but I cannot help feeling that this is so cool.
National Public Radio – Researchers found a rare octopus nursery off the coast of Costa Rica
Quote – Scientists working off the coast of Costa Rica say they’ve discovered the world’s third known octopus nursery…. According to a press release, researchers witnessed Muusoctopus eggs hatch. They said it demonstrated that the area, known as the Dorado Outcrop, was hospitable to young octopuses… Scientists said the discovery also indicated that some deep-sea octopus species brood their eggs in low-temperature hydrothermal vents, such as the one where the nursery was discovered, where fluid heated in the Earth’s crust is released on the seafloor — like hot springs. Click through for story. I cannot bring myself to be surprised that octopuses have discoverd this and made use of it for child care – they are so doggoned smart! But I am charmed by it. The octopuses in this story are neither South Asian nor mimics, but I couldn’t resist the chart below.
Experts in autocracies have pointed out that it is, unfortunately, easy to slip into normalizing the tyrant, hence it is important to hang on to outrage. These incidents which seem to call for the efforts of the Greek Furies (Erinyes) to come and deal with them will, I hope, help with that. As a reminder, though no one really knows how many there were supposed to be, the three names we have are Alecto, Megaera, and Tisiphone. These roughly translate as “unceasing,” “grudging,” and “vengeful destruction.”
Between an effective antibody for all the CoViDs (SARS-2) – mentioned in a short take today – and the promises of gene therapy, it’s been an impressive week. Yes, I realize I’ve put up a lot of good news/potentially good news today – but hey, it’s Mother’s Day.
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Gene therapy helps combat some forms of blindness – and ongoing clinical trials are looking to extend these treatments to other diseases
New gene therapies are helping to treat certain forms of inherited blindness. GettyImages
An estimated 295 million people suffer from visual impairment globally. Around 43 million of those people are living with blindness. While not every form of blindness can be cured, recent scientific breakthroughs have uncovered new ways to treat some forms of inherited blindness through gene therapy.
Jean Bennett is a gene therapy expert and a professor emeritus of ophthalmology at the University of Pennsylvania. She and her laboratory developed the first gene therapy drug for a genetic disease to be approved in the U.S. The drug, Luxturna, treats patients with biallelic RPE65 mutation-associated retinal dystrophy, a rare genetic disorder that causes visual impairments and blindness in patients early in life.
In March, Bennett spoke at the 2023 Imagine Solutions Conference in Naples, Florida, about what gene therapy is, why it matters and the success she and her team have had helping the blind to see. The Conversation caught up with Bennett after the conference. Her edited answers are below.
Jean Bennett speaks at the 2023 Imagine Solutions Conference.
What is gene therapy and how does it work?
Gene therapy is a set of techniques that harness DNA or RNA to treat or prevent disease. Gene therapy treats disease in three primary ways: by substituting a disease-causing gene with a healthy new or modified copy of that gene; turning genes on or off; and injecting a new or modified gene into the body.
How has gene therapy changed how doctors treat genetic eye diseases and blindness?
In the past, many doctors did not think it necessary to identify the genetic basis of eye disease because treatment was not yet available. However, a few specialists, including me and my collaborators, identified these defects in our research, convinced that someday treatment would be made possible. Over time, we were able to create a treatment designed for individuals with particular gene defects that lead to congenital blindness.
Gene therapy treatments are now available in pharmacies and operating rooms all over the world.
Gene therapy is even being used to restore vision to people whose photoreceptors – the cells in the retina that respond to light – have completely degenerated. This approach uses optogenetic therapy, which aims to revive those degenerated photoreceptors by adding light-sensing molecules to cells, thereby drastically improving a person’s vision.
You created one of the first gene therapies approved in the US. What is the current state of the clinical use of gene therapy?
There are now many approved gene therapies in the U.S., but the majority are combined with cell therapies in which a cell is modified in a dish and then injected back into the patient.
Many forms of gene therapy are helping to treat blindness. GettyImages
The majority of those therapies target different forms of cancer, although there are several for devastating inherited diseases. The drug Skysona is a new injectable gene therapy medication that treats boys ages 4 to 17 with cerebral adrenoleukodystrophy, a genetic disease in which a buildup of very-long-chain fatty acids in the brain can lead to death.
The gene therapy that my team and I developed was the first FDA-approved project involving injection of a gene therapy directly into a person – in this case, into the retina. Only one other FDA-approved gene therapy is directly administered to the body – one that targets spinal muscular atrophy, a disease that causes progressive muscle weakness and eventually death. The drug, Zolgensma, is injected intravenously into babies and children diagnosed with the disease, allowing them to live as healthy, active children.
There are now more than two dozen FDA-approved cell and gene therapies, including CAR T-cell therapies – in which T cells, a type of immune system cells, are modified in the laboratory to better attack cancer cells in the body – and therapies for various blood diseases.
What are you currently working on that you’re most excited about?
I am very excited about some upcoming clinical trials that my team will soon initiate to target some other devastating blinding diseases. We will incorporate a new test of functional vision – how your eyes, brain and the visual pathways between them work together to help a person move in the world. This test utilizes a virtual reality game that is not only fun for the user but promises to provide an objective measure of the person’s functional vision. I hope that our virtual reality test will inform us of any potential benefits from the treatments and also serve as a useful outcome measure for other gene and cell therapy clinical trials involving vision.
What are the biggest challenges gene therapy faces?
The biggest challenges involve systemic diseases, or diseases affecting the entire body rather than a single organ or body part. For those diseases, super-high doses of gene therapy reagents must be delivered. Such diseases involve not only technical challenges – such as how to manufacture enormous amounts of gene therapy compounds without contaminating them – but also difficulties ensuring that the treatment targets diseased tissues without causing toxic immune side effects. That level of a problem does not exist with the eye, where relatively small doses are used and exposure to the rest of the body is limited.
Another challenge is how to address diseases in which the target gene is very large. Current approaches to delivering treatments into cells lack the capacity to hold large genes.
Cost remains a key issue in this effort – gene therapy drugs are enormously expensive. As drug manufacturers are able to refine this technique, gene therapy drugs may become more commonplace, causing their price to drop as a result.
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AMT, this news carries a lot of potential. Some of it will probably not work as expected and hoped. But even if only a few of the possibilities pan out, the results could be amazing. I’m thinking today of the pain mothers of children with some kind of genetic problem have had to suffer ocer the millennia, and hoping that may someday be a thing of the past.
Yesterday, I managed to get the weekly email out before sunset, and was able to slow down a little and saw a headline in the ProPublica newsletter of a follow-up to the story of the teens who don’t want to be sent back to their abusive father. Under pressure from public opinion and a couple of prosecutors, the judge has (at least temporarily) vacated his order, and the kids will be able to see the light of day while this is being hashed out. It’s not a total victory, but it is a cease-fire and allows them to breathe.
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Crooks and Liars – Jack Smith Subpoenas The Maids At Mar A Lago
Quote – That includes Mar-a-Lago staff like a housekeeper and restaurant servers, as well as Trump political aides like Margo Martin, who was a press assistant in the White House and then stayed with Trump as he relocated to Florida after leaving office. The prosecutors, led by special counsel Jack Smith, are “casting an extremely wide net—anyone and everyone who might have seen something,” an unnamed source told CNN. Click through for details. This takes me back to Agatha Christie and other detective novels featuring the British upper class. I don’t know why, but it appears to be unsurmountably difficult, not just in fiction, but IRL, for the wealthy to grasp that nothing is secret from the help.
Civil Discourse – Courage
Quote – After all of the speculation over whether there might be some type of standoff at Mar-a-Lago if charges are filed against Trump, we get this quiet concession from the lawyers. It’s an early acknowledgment that Trump isn’t above the law in these anticipated proceedings. He will have to follow “normal procedures” just like anyone else who is charged with a crime. Of course, he will be doing it with a Secret Service agent at his side. The agent will presumably go through all of the booking procedures with him and accompany him in court. That’s a good reminder that we are in uncharted territory from here on out, but unprecedented doesn’t mean the procedural rules don’t apply to Trump. It’s a good sign that his lawyers have been forced to concede that before charges are even filed. Click through for full article. Of course I can’t possibly track the entire internet and every newspaper in the country – but, to my knowledge, Joyce Vance is the only attorney to publish a matter-of-fact description of what may be expected if and when. I think it’s worth sandwiching in.
The 19th News – The radio divide: How airplay reinforces the gender gap in country music
Quote – The year 2015 was especially emblematic, [Jada Watson,… the principal investigator of the SongData project,] noted, with Gary Overton, then the chief executive of Sony Nashville, saying, “If you’re not on country radio, you don’t exist.” After that came “Tomato-gate,” when radio consultant Keith Hill told an industry publication, “If you want to make ratings in country radio, take females out,” before going on to compare women artists in country to the tomatoes, not the lettuce, of the salad of country radio. These remarks were “pretty indicative of how the industry mainstream works,” Watson said of country radio in the years following the fallout surrounding The Chicks. “If you’re not a White, cisgendered, heterosexual, able-bodied male, you’re not on air and you’re not on the radio and so you don’t exist within that space. There are multiple barriers to access to [the country music industry] and the biggest one is radio.” Click through for analysis. I don’t hate country music, but am not a huge fan. I do, however, highly appreciate many of its performers, past and present, who understand what love of country means – and act accordingly. To name just a few, Woody Guthrie, Willie Nelson, Dolly Parton, Brad Paisley. It’s the fans who are shallow, not the genre.
Yesterday I received the email that my ballot has been received. Good news. There was other news also, consequential and inconsequential, but today I am just focusing on humor, because that also came in multiple emails, and I think we all could use some. That’s also why the FFT is just a wordplay, only marginally related to news. If you really want some har news, Letters from an American touches on the presser held by DOJ and also the letter to Biden from Congressional Progressives which weems to have been overinterpreted.
Psyche – Just when in history did men decide that women are not funny?
Quote – Allow me, an historian, to offer evidence about the modern origins of this myth, instead of theories about the supposed evolutionary advantage of bro jokes…. Perhaps the answer will come as no surprise: it was when men began to value humour highly that they decided women didn’t have it. Click through for story. Just offhand, my mind jumped first to “Much Ado About Nothing” (1598), specifically to Beatrice and Benedict, who are supposed to be a subplot, but whom audeiences have always considered the stars, and who “never meet but there’s a skirmish of wit between them.”
Yesterday, putting this post together, I realized I had picked two good news stories. I didn’t set out to do that. It was just, having read so much legal-judicial stuff, I was looking for something new and different, and these jumped out. Besides being good news, the two have something else in common – both hark back to the Obama administration in some way.
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The 19th – 53 years ago, the White House sought to end hunger. Now it’s trying again.
Quote – “This conference is engaging all sectors — the public sector, the private sector, community-based organizations — all around shifting the conversation from just getting food into people’s hands to also making sure that we get healthy food into people’s hands and that it is seen as a public health issue, rather than just an emergency food issue,” said Jason Wilson, vice president of marketing and development for the Partnership for a Healthier America, a nonprofit organization created in conjunction with former First Lady Michelle Obama’s “Let’s Move!” campaign. Click through for details. Fifty-three years ago most Republicans were human beings, and only a few were fascist monsters. Now, it’s the other way around. I widh the conference alll the luck in the world.
Wonkette -Schools Go Solar, Save Millions On Energy, Upgrade Classrooms, Pay Teachers More — Yes, In The USA
Quote – It’s been another crazy exasperating week, so we bet you could do with some really cool news about public schools that are switching to clean solar energy, saving millions of dollars in some cases, and using the savings to improve the schools and even their communities. This isn’t a proposal in some position paper about how we might create a wonderful clean energy future, either — it’s been going on for a while now, as the New York Times recently reported [There is a free gift link in the article but it’s too long to reproduce here]. The story notes that one in 10 US public and private schools in the US was using solar energy by early 2022, according to a report from clean energy nonprofit Generation 180 — twice as many as in 2015. That’s one more benefit of the rapid decline in the cost of solar panels in the last couple decades. Hey, if you’d like to say “Thanks, Obama,” you certainly could, since clean energy investments in the 2009 American Recovery and Reinvestment Act helped jumpstart the widespread adoption of renewable energy and the resulting reductions in solar energy costs. Click through for story – stories actually. It’s happening in states you would not ecpect. Amazing.
Yesterday, I basically tried to keep things slow and calm. It was a pill-organizing weekend, and everything runs out at different times, so there is literally never an occasion when I don’t have to run to another room for a new bottle of something OTC or order a prescription renewal – I generally do that in advance for the following fortnight so I can at least get the bottles filled and capped without interruption (otherwise this klutz would be knocking them over and using choice language.)
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The 19th – Magnolia Mother’s Trust marks a history-making three cycles of paying Black mothers $1,000 a month
Quote – The Magnolia Mother’s Trust is now the longest-running guaranteed income program in the United States. But the program, which gives Black mothers in Jackson, Mississippi, $1,000 a month for one year, no strings attached, was never meant to last forever. “I don’t think that’s the systems change we need,” said Aisha Nyandoro, CEO of Springboard Opportunities, which runs Magnolia Mother’s Trust and provides programs and services to families that live in federally subsidized affordable housing. Instead, the goal of the trust is to model what could be, in a more just and fair society. CLick through for story. If Republians really wanted a world with more happy and healthy people, this is what they would be doing (which of coursethey don’t.) Likewise, if they really wanted a world of people who would fall easily into categories and be content with that, they should be emulating Brave New World – the ohly dystopia I’m aware of in which the characters aren’t aware that it’s a dystopia. But Republicans don’t want that either. They aren’t happy unless someone is getting hurt.
Vice – Scientists Achieve the Impossible, Safely Destroy Toxic ‘Forever Chemicals’
Quote – In a new paper published [this month] in the journal Science, a team of researchers have uncovered a new way to dispose of a class of these chemicals under comparatively mild conditions, including ambient pressure and temperatures as low as 176 degrees Fahrenheit. William Dichtel is a lead author on the paper and a professor of chemistry at Northwestern University. He said in a press conference about the work on Tuesday that one of the exciting benefits of this discovery is that the reaction leaves no damaging products in its wake. “We were pleased to find a relatively low temperature, low energy input method where the one specific portion of these molecules falls off and sets off a cascade of reactions that ultimately breaks these PFAS compounds down to relatively benign products including fluoride ions… that are in many cases found in nature already and do not pose serious health concerns.” Click through for background and more info. Nobody saw this coming – even the scientists were pleasantly surprised. And good news is always welcome.
Yesterday, I set up my meds for the next two weeks, because I hadn’t done it Saturday. Oh, well, that’s why I keep a couple of extras filled – so I can miss a day withut actually missing a day. And nthat was pretty much all I did. the weather is what my Aunt Mary used to call “enervating.” (Yes, love of words runs in my family – both sides.) And I want to save as much energy as I can for the hearings which I understand are scheduled for Tuesday and Thursday.
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Crooks and Liars – SNEAKS: PA GOPers Advance Anti-Abortion Amendment Last [Thursday] Night
Quote – The first step happened last night, but the long-term plan is to get an anti-abortion constitutional amendment on the ballot next year…. In PA, the process is they need to pass through both chambers twice, in consecutive legislative sessions, then they hit the ballot. If Rs time the anti-abortion one correctly, it will hit the ballot in an off year, low turnout election with least amount of voter participation Click through for story. Yes, this is one state, but it could happen elsewhere.
