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Howdy! It’s Meghana. At the moment, we’re studying in regards to the world’s costliest drug, plus we find out about immune senescence and a possible trigger for a kidney autoimmune illness.
The necessity-to-know this morning
- Akebia Therapeutics received FDA approval for Vafseo, a therapy for anemia as a result of continual kidney illness in sufferers on dialysis.
- Gilead Sciences licensed an experimental, IL-12-targeted most cancers drug from Xilo Therapeutics. The drug, known as XTX301, is designed to make immunologically “chilly” tumors extra susceptible to immunotherapies.
When sticker shock additionally appears price it
Final yr, the Institute for Medical and Financial Evaluation analyzed the newly accepted gene remedy Lenmeldy to find out what would possibly make for an economical value, and got here up with as much as $3.9 million. That’s not a far cry from the worth set by maker Orchard Therapeutics, which will likely be asking $4.25 million for the therapy, making it the world’s costliest drug.
Lenmeldy does enable some youngsters with the neurodegenerative illness metachromatic leukodystrophy to develop up and reside primarily regular lives. And that, based on David Rind, ICER’s chief medical officer, makes it well worth the hefty value.
“Right here’s one other drug that’s taking a bunch of children who could be useless and giving them a life,” he instructed STAT. “That’s price some huge cash. I imply, we pay loopy quantities of cash for medicine that do virtually nothing.”
Learn extra.
Reversing the immune system’s age (in mice)
As we age, our immune techniques inexplicably start to deteriorate. A concept has been that the bone marrow’s hematopoietic stem cells that morph into B cells and T cells start to get out of whack over time. A brand new Nature research exhibits how an age-associated imbalance in HSCs finally results in decline in immune perform. By restoring a youthful stability of HSC-derived immune cells with antibody injections, scientists had been in a position to reinvigorate the immune techniques of older mice.
If these findings can translate to human research, they might signify an avenue to enhance well being in ageing adults.
“Should you have a look at the ailments in aged folks — virtually all of the cancers and leukemias, inflammatory ailments, rheumatoid arthritis and so forth — I don’t know, however I anticipate that we’re going to be taught rather a lot about how this might assist,” the research’s senior creator instructed STAT.
Learn extra.
How a micro organism would possibly trigger a kidney autoimmune illness
Micro organism known as Akkermansia muciniphila can alter antibodies within the intestine microbiome so dramatically that immune cells can’t acknowledge them anymore — main the physique’s protection system to assault itself. It will probably take away the outer sugar coating IgA antibodies, which finally might play a task within the growth of an autoimmune illness within the kidneys known as IgA nephropathy, a brand new research in Science Translational Drugs says.
Nobody is aware of what causes IgA nephropathy, which causes kidney irritation, blood within the urine, and attainable kidney failure. There’s no treatment, and few remedies can be found. It’s comparatively widespread, with 1 in 10 kidney biopsies exhibiting indicators of the situation.
“We have to cease excited about this passive, oblique impact of the impact of the microbiota on human illness and autoimmunity,” the research’s lead creator instructed STAT. “And extra begin in search of the direct ways in which micro organism chilly be modifying molecules within the human physique.”
Learn extra.
Oprah’s questionable endorsement of GLP-1s
Oprah Winfrey is shifting the needle with the GLP-1 dialog — and which may be a trigger for concern, opines Lisa Shah, CMO of well being AI firm Twin Well being. Final week, Winfrey unequivocally endorsed medicine like Ozempic and Wegovy to deal with weight problems in what seemed like an informercial.
“Oprah discovered one thing that works for her, and she or he’s excited to share it with the world,” Shah writes. “Coincidentally, it additionally works for the pharmaceutical producers who, based on reviews, pay for lots of the medical consultants featured on her particular.”
Sufferers shouldn’t overlook that GLP-1s should not a panacea — and {that a} answer to metabolic illness would possibly take an individualized method, as an alternative of the blanket promise of the drug du jour.
Learn extra.
Extra reads
- A number of sclerosis has distinct subtypes, research finds, pointing to totally different remedies, STAT
- The Meals is Drugs motion originators are attempting to standardize medically tailor-made meals, STAT
- Moderna’s early vaccine pipeline underpinned by EpsteinBarr, norovirus and shingles prospects, FierceBiotech
- Novocure’s machine proven to sluggish most cancers development within the mind in trial, Reuters
