please just approve the drugs - we patients and pick and choose
In January 2025 my mom’s lung cancer spread to her brain. The drug with the best chance of helping her was approved in China but not here.
A doctor told me, almost as an aside, that if she were Chinese she could buy it at a pharmacy.
So I flew to Shanghai and came home with 26 boxes in my luggage. That was 19 months ago. My mom is still here, still working, and in her words has had an amazing three years.
The drug was approved in China in 2023. The FDA approved it in July 2025. It still is not for sale here. I know of at least 30 Americans who have made the same trip.
I have been scared to tell this story because I worried it could cost my mom access to the medicine keeping her alive. But the problem matters more than my silence. These drugs need to be developed and available here, and that means FDA reform.
Art Allen at KFF Health News told our story this week. It also ran in The Washington Post.
kffhealthnews.org/health-ind…
are doctors incorporating this yet? or is TME important and is missing?
A milestone we have worked toward for years.
ASCO has recognized Functional Precision Medicine as an emerging innovation in cancer care—testing a patient’s living tumor cells against therapies to help identify what may work.
This is the approach behind the PARIS® test.
A major step forward—but much remains to be done to achieve broad oncologist adoption, insurance coverage, and access for every patient who could benefit.
Please read, share, and help us move this field forward. connection.asco.org/do/asco-…
in 2026, using the same chemo like FOLFIRI that was approved 26 yrs ago is f8cking depressing
surely we must have something better after 26 freaking years
Wei retweeted
What if the next advance in BCMA CAR-T isn’t simply stronger T-cell activation—but more precisely controlled activation?
Anitocabtagene autoleucel (anito-cel) uses a synthetic d-domain BCMA binder (ddBCMA) and is being developed for heavily pretreated relapsed/refractory multiple myeloma.
In this phase 1 study, 38 patients received anito-cel after ≥3 prior lines of therapy or with triple-class refractory disease.
The clinical results were striking:
🔹 100% overall response rate
🔹 79% complete response rate
🔹 24-month PFS: 57%
🔹 Median PFS: 30.2 months
🔹 36-month OS: 65%
But the mechanistic story may be even more interesting.
At the recommended phase 2 dose, 94% developed grade 1–2 CRS, with no grade ≥3 CRS. ICANS occurred in 16%, predominantly grade 1–2, with one grade 3 event.
Investigators compared the ddBCMA binder with a dual-VHH binder corresponding to the published ciltacabtagene autoleucel sequence.
Despite similar cytotoxicity, the ddBCMA CAR-T cells demonstrated less cytokine release, without antigen-independent activation or off-target cytotoxicity.
The binder also showed a faster off-rate—raising an intriguing possibility that the kinetics of antigen engagement may influence how CAR-T cells balance activation, effector function, and toxicity.
That leads to a broader question in CAR-T engineering:
Is the goal simply to maximize CAR signaling—or to engineer the right signaling dynamics?
As BCMA CAR-T moves into earlier lines of myeloma therapy, optimizing the quality of T-cell activation may become just as important as maximizing its magnitude.
The next generation of CAR-T may be defined not only by what the cell targets, but how the receptor engages its target.
nejm.org/doi/full/10.1056/NE…
#MultipleMyeloma #CART #BCMA #CellTherapy #Immunotherapy #CancerResearch #TranslationalMedicine
Legend
Why are we not doing more of this
Admin toxicity is horrible, and admin errors are taking life’s
I had a very romantic idea of what oncologists actually do all day.
Ever since my Dad got cancer, I’ve been fascinated by oncology. He's fortunate to have a great onco
Soon after, I read Emperor of All Maladies & this image became almost mythical
Over the past year, I started spending a LOT of time shadowing oncos at community clinics & NCI giants.
Stuff I witness horrifies me.
My fantasy is now maybe ~30% true
Oncos are forced engage with entities that have perfected the art of treating them like sh*t.
Numbers are brutal:
▪️96% say prior auth delays treatment
▪️80% say it has caused disease progression
ACCC recently gave this a name: administrative toxicity
Welcome to modern day cancer care..
Put brilliant minds through 15 years of training.
Then surround them with mind-numbing work
- - - - -
Plot Source: @Jori_health
- - - - -
Wei retweeted
Don’t drink a daily OJ, please 🙏
A glass has 12-18g fructose equivalents, a most dangerous type of sugar that promotes:
* Liver lipogenesis and fatty liver
* Higher triglycerides and VLDL
* Hepatic insulin resistance
* Uric-acid production
* Visceral-fat accumulation
Wei retweeted
The biology is consistent. IL-15 and IL-12 are key to persistence of CAR-T cells. The next evolution. Solid tumors.
What if the next breakthrough in solid-tumor CAR-T isn’t simply finding a better target—but building a T cell that can sustain itself after entering the tumor?
A new report in the New England Journal of Medicine describes a remarkable case: a 3-year-old boy with chemotherapy-resistant metastatic hepatoblastoma achieved complete tumor regression lasting at least 12 months after two infusions of glypican-3–specific CAR-T cells.
What made these CAR-T cells different?
They were engineered to coexpress interleukin-15 (IL-15) and interleukin-21 (IL-21).
These cytokines are intended to enhance the persistence, proliferation, and functional fitness of the infused T cells—potentially helping them overcome some of the barriers that have limited CAR-T activity in solid tumors.
And the clinical result is striking:
🧬 Glypican-3–targeted CAR-T
🔬 IL-15 + IL-21 coexpression
👦 3-year-old with chemotherapy-resistant metastatic hepatoblastoma
🎯 Complete regression
⏳ Response sustained ≥12 months
This is only a single patient report, so it is far too early to draw conclusions about efficacy across hepatoblastoma or other solid tumors.
But the concept is important.
For years, much of CAR-T engineering has focused on the receptor: What should the T cell recognize?
Increasingly, the question is becoming:
What does the T cell need to survive, expand, and remain functional once it reaches the tumor?
Cytokine-armored CAR-T cells represent one approach to that problem.
This case provides an intriguing glimpse of what may be possible when we engineer not only what CAR-T cells see, but also how they behave once they get there.
Solid tumors remain one of the biggest frontiers for cellular therapy.
And sometimes, a single patient can show the field what might be possible next.
nejm.org/doi/full/10.1056/NE…
cure within you
🆕👇🏽Unveiling FULL results of our #NEST🪺 Trial in MSS “cold”🧊colorectal cancer
💡Immunotherapy (BOT🤖/BAL) BEFORE surgery
💯% disease-free to date✅
NEST1:~32 months
NEST2:~24 months
5 years of work—now in @CCR_AACR🙌🏽
🚀#NEST3 NOW OPEN!
@OncoAlert
🔗 aacrjournals.org/clincancerr…
Please solve cancer
With AlphaFold we mapped the protein universe - now with AlphaGenome Atlas we’re charting the human genome. It can predict the impact of all 9 billion possible single-letter DNA variants, helping scientists better understand disease. Freely available for academic research: alphagenome.google/atlas
Wei retweeted
Excited to see early customers already using Astra on Azure! azure.microsoft.com/en-us/bl…
Wei retweeted
Life is short. Celebrate every day.
She didn’t need a grand ballroom, a cathedral, or a fairytale venue. All she needed was a quiet hospital room—and the only man whose blessing meant the world.
Knowing her father was losing his battle with terminal cancer and wouldn't make it to her wedding date, she brought the entire wedding to his bedside. Amid the hum of medical monitors and tears of pure devotion, she walked into that room in her white gown just so her dad could see his little girl become a bride.
He held on just long enough for this moment. Two days later, he took his final breath—not in defeat, but as the proudest, happiest father in the world, carrying the vision of his daughter’s joy into eternity.
A daughter’s ultimate act of love. A father’s most beautiful final memory. Rest in peace. 🕊️🤍
#UnconditionalLove #FatherDaughter #PureLove #EmotionalMoments
Wei retweeted
From a randomized trial, the advantage of a ketogenic diet vs Mediterranean or low fat plant diet for people with pre diabetes and fatty liver (hepatic steatosis)
cell.com/cell-metabolism/ful…
yipit
Replying to @TheRideshareGuy
.@TheRideshareGuy One of the reasons why Uber is so successful is because we obsessively measure our performance across multiple metrics. Category Position is at the top of that list - i.e. how we are faring vs competitors in a certain city or country, which we track on a weekly basis.
We use lots of different third party sources like anonymized credit card and email panels, as well as public reporting, and we obviously know our own performance with high fidelity.
So when we make a public statement about our category position, we have high confidence in the data. We hold an even higher bar for disclosures we make on our earnings calls.
By the way, here's an updated view from Yipit that should confirm this statement as well.
Wei retweeted
At midnight on February 6th, earlier this year, the doorbell to our apartment rang. The doorbell was followed by a pounding on the door. I answered the door and a security person in our building handed me his phone. It was my oldest daughter Eloise. She had found my 26-year-old daughter unconscious on the floor of her apartment and had called 911. The EMT team was already there, but they did not know what was wrong with Lucy or to which hospital they would take her.
I threw on some clothes and jumped in an Uber heading east toward Brooklyn. (Lucy lived alone in Williamsburg.) On the way, I learned that they were taking her to Elmhurst, a City trauma hospital in Queens. I arrived about five minutes after the ambulance to join Eloise, Lucy’s mom, and a friend, and waited to learn what was wrong.
After about 15 minutes, I asked a nurse where she was. I looked over his shoulder to his computer. Next to her name, it said “non-responsive.” I walked into the emergency room and wandered around looking for her until I found her unconscious on a gurney surrounded by several doctors and nurses.
By about 2:30am with the results from a CAT scan, Lucy’s doctors had determined that she had a massive brain hemorrhage and would need an emergency hemicraniectomy to release the pressure on her brain and remove the blood from the hemorrhage. I called our wonderful friend and family doctor Eddie Fisher and explained what was going on. He woke up Josh Bederson, Chairman of Neurosurgery at Mount Sinai, to find out more about Zach Hickman, the neurosurgeon on call that night. Dr. Bederson said Hickman was an excellent surgeon, which was comforting as we had no choice.
The surgery to save Lucy’s life began around 3:15am and finished around 5:30am. It was successful.
The following day, I joined Lucy in an ambulance while she was being transferred to Mount Sinai on Madison Avenue. Later that day, we determined from Lucy’s Oura ring that her hemorrhage had occurred around 9am, which meant that more than 19 hours had passed from the time of the hemorrhage to the completion of the surgery to release the pressure on her brain (I only wish @ouraring had an alert for this kind of a medical event. Imagine it could call a family member if the wearer doesn’t cancel the alert).
I later learned that the standard of care is not to do surgery to save a patient with a large brain bleed if more than five hours have passed since the hemorrhage. Even when the surgery is done, I was told that the likely outcome for the patient is a few months in a nursing home and death from pneumonia.
When I met Lucy’s doctors, I did my best to inspire them: “Let’s see what can be accomplished if we give her the best care possible and we invest unlimited resources to restore her to life.” And I promised that whatever we learned we would make available to everyone.
Dr. Chris Kellner, her neurosurgeon, and Dr. David Putrino, Director of Rehabilitation Innovation for the Mount Sinai Health System have led Lucy’s care team since that day. Words cannot describe the remarkable and compassionate care that she has received beginning with the EMT team and then from nurses, doctors, therapists, and the army of people who have worked to save her and return her to life. To this day, we have a daily Zoom where we discuss her progress and make adjustments to her care. While her care and oversight have been incredible, the learnings for the Mount Sinai team have also been elucidating and will assist in the care of many others.
Lucy began in a bad place. She was in a coma for several weeks and then awoke not being able to breathe on her own, unable to walk, see or speak.
Over the last six months, she has recovered her cognition – she understands everything including her circumstance – is able to walk a hundred or more steps at a time with assistance, is making progress with sounds, vowels and consonants and the beginnings of speech, but she remains unable to see.
Each day, she makes a little progress, and daily progress compounds. Every day I tell her that she just needs to make a little progress and it won’t be long before she is back.
We remain optimistic that Lucy will return to normal function. It will likely take years, but I believe it is only a matter of time, hard work, and technological progress, along with some, and perhaps a lot, of divine intervention.
Many people have been praying for Lucy and we are incredibly grateful for the prayers and remarkable support she has received. Lucy’s friends have been with her every day since the beginning, and their presence and friendship have saved her life and helped to rebuild and maintain her spirit. And on a very positive note, Lucy’s challenge has brought together our entire modern family who have all been incredibly devoted to her care and recovery.
Lucy's vision and other faculties may require some form of brain computer interface, work that is underway at Neuralink, Precision Neuroscience, Science, Synchron, Nudge, and other companies in the space.
If you are going to have a devastating brain injury, now is the best time in history for that to happen. We are living in a world when you can be confident that the blind will soon see again. We are going to do everything we can to help make that happen, including by assisting existing companies in the space.
With respect to our promise to make Lucy’s care available to others, we have made good progress. In May, a real estate colleague made me aware of a 93% vacant, brand new, 400,000 square foot Class A+ purpose-built biotech facility on West End Avenue between 65th and 66th Streets that missed the market and was available for sale. The Pershing Square Foundation acquired the building 60 days later.
We also put under contract an adjoining 130,000 square foot building at 320 West 66th Street that is currently being used by Saturday Night Live for studio space. The building has 35-foot ceilings with massive column-free spaces that can be converted into superb rehabilitation facilities. We will close on the SNL building in December.
We are also acquiring an adjoining vacant lot with additional air rights. With just the existing zoning rights, we can add a 150,000 square feet for a total of 680,000 square feet, a lab footprint larger than Rockefeller University, and that’s without including the potential for an upzoning that would allow for substantially more buildable area on the site’s 3.4 acres with spectacular views of the Hudson.
Our goal is to build the world’s greatest brain research, rehabilitation, recovery, human optimization, and longevity institute. We have named it The Ackman Oxman Institute or the AOI for lack of a better name, but also to reinforce the point that Neri and I and our family are all-in on the mission.
The AOI will be patient-centric. It will not be an academic research institute that produces lots of papers, a Nobel Prize winner or two, but little if any results for patients. We will be laser-focused on cures, treatments, devices, rehabilitation and exercise equipment, and targeted and basic research with a goal of massively accelerating the time from idea to innovation to production to helping a patient.
While the AOI will be a non-profit, it will have highly commercial instincts. The AOI will have its own venture funding and will work to develop innovations to create companies that we will seed, assist, and spinout to ensure technologies, treatments, techniques, and drugs get to patients as promptly as possible.
On one 3.4 acre campus in what is still the greatest city in the world, we will do neurosurgery, neuroscience, rehabilitation, nutrition, BCI and device development, human trials, hyperbaric oxygen treatments, and life extension programs, and we will mandate and incentivize collaboration among the teams with no silos, politics, bureaucracy, or any other constraint that is inconsistent with the mission.
Mount Sinai will be an important partner and deservedly so, but it won’t be our only hospital or medical school partner as we don’t believe any institution has a monopoly on the best ideas or the best talent. We don’t believe in exclusive relationships because that is not in the best interest of patients.
Five years ago, we considered launching a brain institute inspired by Neri’s mom who sadly died from Alzheimer’s. We couldn’t make the math work as the real estate was too expensive and we believed it would be too difficult to recruit the best talent from universities to our effort.
Since then, the real estate became available at a 70% discount, universities became a much less attractive place to work due to politics outweighing meritocracy, protests that disrupt learning, the curse of antisemitism, and a decline in funding. Fortunately, during the same time, I made sufficient personal economic progress to make the AOI possible.
The advance of AI in the last few years will also enable us to greatly accelerate our mission. AI still has a lot to learn about human intelligence and the brain, and the AOI should be at the forefront of the interplay between the brain and AI.
Today, I am making a public filing disclosing a gift from Neri and me of ~$400 million or 10,000,000 shares of Pershing Square Inc. (PS) to the AOI. It is very early days for Pershing Square so these shares are intended to anchor the long-term work of the AOI as the shares compound over time while generating what we expect will be a growing stream of quarterly dividends to fund the Institute.
We will also be announcing an additional gift of similar and potentially greater size which won’t be in the form of Pershing Square stock to provide the AOI with the short- and intermediate-term runway necessary to enable it to achieve its goal of becoming a self-sustaining institute, which reinvests all of its revenues, royalties, and the economic rewards of company formation to advance the fields of brain health and human longevity.
Neri and I have chosen to anchor the funding of the AOI to maintain vision alignment and limit the need for the organization to focus on fundraising. We expect the AOI to be the best-resourced brain, rehab, recovery, and longevity institute in the world.
We are grateful to have been able to form a board which includes Dean Kamen (our generation’s Thomas Edison), George Yancopoulus (CEO of Regeneron), James Rothman (Nobel Laureate), Bernardo Sabatini (neuroscientist), Chris Kellner (neurosurgeon), Olivia Flatto (CEO Pershing Square Foundation), Neri Oxman, and myself.
We have recently identified a CEO who we expect to announce by October along with other key hires, and are beginning searches for a Chief Scientific Officer, a Chief AI/Technology Officer, a Chief Operating Officer, a Chief Financial Officer, and other key leadership roles.
If you find what we are building compelling and want to be part of the leadership team that creates and builds the AOI from a standing start, please send an email to: [email protected] with a short note as to why you believe you can help. Please include your best three ideas for the AOI along with a summary of your background and your most important accomplishments.
We promise strict confidentiality to those expressing interest in working with us.
We have learned from Lucy that the brain can recover from even catastrophic injury. There is so much more work to be done as the mind is a terrible thing to waste.
For details from my Pershing Square SEC filing see:
sec.gov/Archives/edgar/data/…



























