Last night I came home from an eight-hour shift, sorting bills, when he walked in.
âI need $300.ââšâNo.ââšâYou have money.ââšâI have money for the mortgage.â
I told him starting next month he contributes or he doesnât live here. He said I sounded like his father. Then: âIf you tell me no again, I swear youâre going to regret giving birth to me.â
For anyone who would like to hear Mark Carneyâs outstanding Davos speech in full here it is. This is what true global leadership looks like.
Canada should be immensely proud today, because they are leading the fight back when others dare not.
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An inspiring scientific story that shows what is on the line in our political debates.
In a world-first medical breakthrough, a baby named KJ Muldoon recently became the first person ever treated with a completely personalized CRISPR gene-editing therapy crafted just for him.
The baby had been diagnosed with a genetic condition called CPS1 deficiency, which meant that KJ couldnât break down ammonia in his body, which is a byproduct of protein metabolism. This meant doctors had to restrict the amount of protein he could have, limiting the intake to just enough to grow. This meant he was malnourished, and still at a high risk of brain damage or death.
But in an unprecedented race against time, scientists and doctors in Philadelphia rewrote what was thought possible in medicine.
A team of scientists developed a one-of-a-kind CRISPR-based treatment tailored to KJ's unique DNA.
Most gene therapies take years to develop, often aiming at common conditions affecting many patients. But this time, scientists condensed the process into six months, building a therapy that exists only for one child.
The treatment used lipid nanoparticles to deliver three doses of CRISPR components directly to KJ's liver. The goal was to correct the genetic error that inhibited his ability to metabolize ammonia.
When KJ received his first shot, he was at the 7th percentile of weight for his age. The doctors had to tell the parents that they had no idea what his reaction would be, or if it would even work. But everything seems to have worked out well. Now KJ is at the 40th percentile of body weight. He may still need a liver transplant, but the prognosis is hopeful.
We could celebrate this as a great human achievement, and leave it at that. But I write about politics and public policy, and we should not fail to learn from this story.
Unfortunately, we have an administration that is at war with nearly everything that made this innovation possible: immigration, federal funding of science, big pharma, and, in the form of MAHA, even medical science itself.
Let's start with immigration. Here are the names of the doctors and scientists mentioned in the NYT coverage of the story
Kiran Musunuru
Rebecca Ahrens-Nicklas
Fyodor Urnov
Sadik Kassim
David Liu
Of five names, only one is old stock American.
Another scientist mentioned is Peter Marks, although he didn't work on this research. Marks wrote an editorial accompanying the research paper, arguing that the treatment used "eventually could also be used for more common genetic disorders like sickle cell disease, cystic fibrosis, Huntingtonâs disease and muscular dystrophy."
Dr Marks oversaw gene-therapy regulation at the FDA before leaving over RFK's war against vaccines.
The scientists who accomplished this feat rave about the system that made it possible.
The result âis a triumph for the American peoplesâ investment in biomedical research,â Dr. Urnov said.
The researchers emphasized the role government funding played in the development.
The work, they said, began decades ago with federal funding for basic research on bacterial immune systems. That led eventually, with more federal support, to the discovery of CRISPR. Federal investment in sequencing the human genome made it possible to identify KJâs mutation. U.S. funding supported Dr. Liuâs lab and its editing discovery. A federal program to study gene editing supported Dr. Musunuruâs research. Going along in parallel was federally funded work that led to an understanding of KJâs disease.
âI donât think this could have happened in any country other than the U.S.,â Dr. Urnov said.
Those who worked on saving KJ were proud, Dr. Urnov said.
âWe all said to each other, âThis is the most significant thing we have ever done.ââ
Remember, these debates are not theoretical. It matters that we have a free market system in which corporations can make money, an immigration system in which the most qualified people in the world can come here and work on the most important questions facing humanity, and a regulatory regime in which real scientists rather than conspiracy theorists and cranks have power. All of this is being threatened today.
Scientists at MIT developed a form of magnesium called Magnesium L-Threonate that boosts brain levels quickly & efficiently. It is the only magnesium that can cross the blood-brain barrier.
A study of adults with cognitive impairment reversed brain aging by nine years.
Very proud of our study published in @NEJM. 100% clinical complete response with dostarlimab alone in mismatch repair-deficient locally advanced #RectalCancer. No radiation or surgery! @ASCO#ASCO22@MSKCancerCenter https://t.co/sZypoHBtj7