Ana Cristancho

Peter Marks, MD, PhD Director, Center for Biologics Evaluation and Research U.S. Food and Drug Administration 10903 New Hampshire Avenue Silver Spring, MD 20903 March 28, 2025 Sara Brenner, MD, MPH Acting Commissioner of Food and Drugs U.S. Food and Drug Administration 10903 New Hampshire Avenue Silver Spring, MD 20903 Dear Dr. Brenner: It is with a heavy heart that I have decided to resign from FDA and retire from federal service as Director of the Center for Biologics Evaluation and Research effective April 5, 2025. I leave behind a staff of professionals who are undoubtedly the most devoted to protecting and promoting the public health of any group of people that I have encountered during my four decades working in the public and private sectors. I have always done my best to advocate for their well-being and I would ask that you do the same during this very difficult time during which their critical importance to the safety and security of our nation may be underappreciated. Over the past years I have been involved in enhancing the safety of our nation's blood supply, in advancing the field of cell and gene therapy, and in responding to public health emergencies. In the last of these, during the COVID-19 pandemic I had the privilege of watching the vision that I conceived for Operation Warp Speed in March 2020 in collaboration with Dr. Robert Kadlec become a reality under the leadership of HHS Secretary Azar and President Trump due to the unwavering commitment of public servants at FDA and elsewhere across the government. At FDA, the tireless efforts of staff across the agency resulted in remarkably expediting the development of vaccines against the virus, meeting the standards for quality, safety, and effectiveness expected by the American public. The vaccines undoubtedly markedly reduced morbidity and mortality from COVID-19 in the United States and elsewhere. Many of these same individuals applied learnings from the pandemic during a flawless response helping…

ongoing multistate measles outbreak that is particularly severe in Texas reminds us of what happens when confidence in well-established science underlying public health and well-being is undermined. Measles, which killed more than 100,000 unvaccinated children last year in Africa and Asia owing to pneumonitis and encephalitis caused by the virus, had been eliminated from our shores. The two-dose measles, mumps, rubella vaccine regimen (MMR) using over the past decades has a remarkably favorable benefit-risk profile. The MMR vaccine is 97% or more effective in preventing measles following the two-dose series, and its safety has been remarkably well studied. Though rarely followed by a single fever-related seizure, or very rarely by allergic reactions or blood clotting disorders, the vaccine very simply does not cause autism, nor is it associated with encephalitis or death. It does, however, protect against a potential devasting consequence of prior measles infection, subacute sclerosing panencephalitis (SSPE), which is an untreatable, relentlessly progressive neurologic disorder leading to death in about 1 in 10,000 individuals infected with measles. Undermining confidence in well-established vaccines that have met the high standards for quality, safety, and effectiveness that have been in place for decades at FDA is irresponsible, detrimental to public health, and a clear danger to our nation's health, safety. and security. In the years following the pandemic, at the Center for Biologics Evaluation and Research we have applied the same unwavering commitment to public health priorities to the development of cell and gene therapies to address both hereditary and acquired rare diseases. During my tenure as Center Director we have approved 22 gene therapies, including the first gene therapy ever to be approved in the United States. However, we know that we must do better to expedite the development of treatments for those individual suffering from any one of the thousan…

Peter Marks’s resignation letter. Everyone should read this.

3rd year student Maggie Cassidy (@margaretmcass) in the @AhrensNicklas and @AnaGCristancho labs with a new review article - Branched Chain Amino Acid Metabolism in Developmental Brain Injury: Putative Mechanisms and Therapeutic Potential

There are days in life that shake you.

I’m shattered 💔 to share that I just found out that the US Government terminated my 2024 NIH Director’s Early Independence Award (~$2 million), threatening my long-promised assistant professor job at Columbia University
& academic career... 1/🧵

If folks are so worried about syllables, just say “stroke.” We’ll see images and examine.

But at 3 am as the neuro res, did I wonder if folks thought the clot was driving a car and crashed into the vessel when they said “CVA”? Yes, I did. The secret chuckle was appreciated. But I’m peds neuro

By Arthur Caplan:
"many scientists and administrators now seem to think that the best response to the war on DEI is to keep their heads low and wait out four hard years."

"the only way forward is speaking truth to power."

TBCK-deficiency leads to compartment-specific mRNA and lysosomal trafficking defects in patient-derived neurons Monogenic pediatric neurodegenerative disorders can reveal fundamental cellular mechanisms that underlie selective neuronal vulnerability. TBCK-Encephaloneuronopathy (TBCKE) is a rare autosomal recess...

Standing up for science by sharing this labor of ❤️
out today!

What underlies selective neuronal vulnerability in rare 🧬 disorders when the gene is ubiquitously expressed?

TBCK patient derived neurons have axonal predominant lysosomal and mRNA trafficking defects, 👇🏽
www.biorxiv.org/content/10.1...

Crowd protesting, holding signs

Crowd of scientists in Philly, "Make America Smart Again"

"Make America Smart Again" Philadelphia, Pennsylvania (📷 @WesleyWilson) #3E #USprotests #March7Science #StandUpForScience #Mar7

From Philly 💕💪

This is horrific and just the beginning. They will not stop at Columbia…

Thousands gather across U.S. in Stand Up for Science events Scientists rally across the country in response to layoffs and funding cuts ordered by the Trump administration

Breaking news: Thousands of researchers and their supporters, including recently fired federal workers, have gathered across the U.S. in response to layoffs and funding cuts ordered by the Trump administration. scim.ag/41zlPv4

Standing up for science in Philly!

Philly

And 🐑 🐖 🐇 🧫🪰🐟 models…but had to call out mice given the misinformation from last night’s address.

#NeoSky #NeuroSky #NIH

It’s going to be snowing ❄️ and chilly 🥶 here in Denver on Friday but we’ll be 🧬🧠🔬#StandingUpForScience at the capitol. Bring your gloves and goggles, friends! 👩🏽‍🔬🥽☃️ See you there! @cuanschutz.bsky.social @standupforscience.bsky.social

Hearing from the NIH clinical center:

"NIH has a freeze on all purchasing cards [as of yesterday]. No exceptions for most hospital supplies. Clinical center ran out of cups and now is having patients wash down their pills with water from tiny medicine cups."

This is very good advice for scientists who are now dipping their toes for the first time into organizing, or into speaking out.

Many things you try will not work. You just have to keep trying things.

The best resistance is positive persistence. 🧪

In this #rarediseaseday2025 we cannot remain silent. We must fight on the beach and the bench. Share. Tell your stories. Call your elected officials. Call your unelected officials. #fightonthebench

Yup. By Bill Bramhall

Fetal Neonatal Neurology fellowship for July 2026- Children’s Hospital of Philadelphia for child neurologists and neonatologists , in collaboration with the Div. Of Neonatology & the Center for Fetal Diagnosis & Treatment.
Applications open till March 10, 2025.
See link below:

Lovely day for a picnic to celebrate some good news with the best lab.

DOGE By The Numbers
theonion.com/doge-by...

The NIH’s new imposed standard indirect cost rate threatens life-saving brain & behavior research that benefits millions of Americans. SfN, with other scientific organizations,urge reversal of this policy & implore Congress to protect critical research infrastructure.

Full statement: bit.ly/4jVJnCL

New Fellowship Alert!!
Fetal Neonatal Neurology at Children’s Hospital of Philadelphia

www.chop.edu/division-neu...

@childneurosoc.bsky.social #fetalneonatalneurology #neonatalneurocriticalcare

ORDER GRANTING PLAINTIFF STATES’ EX PARTE EMERGENCY MOTION FOR TEMPORARY RESTRAINING ORDER ANGEL KELLEY, D.J. Plaintiff States’ Ex Parte Emergency Motion For Temporary Restraining Order [Dkt. 4] is GRANTED. Defendants and their officers, employees, servants, agents, appointees, and successors are hereby enjoined from taking any steps to implement, apply, or enforce the Rate Change Notice (NOT-OD-25- 068) within Plaintiff States until further order is issued by this Court. Counsel for Defendants shall file a status report with the Court within 24 hours of the entry of this Order, and at biweekly intervals thereafter, confirming the regular disbursement and obligation of federal financial assistance funds and reporting all steps that NIH, HHS and their officers, employees, servants, agents, appointees, and successors have taken to comply with the Court’s temporary restraining order.

COMMONWEALTH OF MASSACHUSETTS, ATTORNEY GENERAL DANA NESSEL ON BEHALF OF THE PEOPLE OF THE STATE OF MICHIGAN, STATE OF ILLINOIS, STATE OF ARIZONA, STATE OF CALIFORNIA, STATE OF CONNECTICUT, STATE OF COLORADO, STATE OF DELAWARE, STATE OF HAWAI’I, STATE OF MAINE, STATE OF MARYLAND, STATE OF MINNESOTA, STATE OF NEW JERSEY, STATE OF NEW YORK, STATE OF NEVADA, STATE OF NEW MEXICO, STATE OF NORTH CAROLINA, STATE OF OREGON, STATE OF RHODE ISLAND, STATE OF VERMONT, STATE OF WASHINGTON, and STATE OF WISCONSIN, plaintiffs.

BREAKING: Federal judge temporarily blocks NIH indirect costs rate cap issued Friday as applied to states that sued over the act this morning.

Judge Angel Kelley, a Biden appointee, ordered further briefing as well, and set a hearing on Feb 21 in the case. storage.courtlistener.com/recap/gov.us...

Apologies for this long post I put together to explain to non-scientists the impact of NIH cuts: Last night, an “atomic bomb” was dropped on the biomedical science community. We were informed that effective immediately, indirect costs on NIH grants (both current and future) would be fixed at 15%.

My occasional reminder to folks on tenure and promotion committees that there will be a cohort of people whose early careers were defined entirely by the pandemic and *waves vaguely* this nonsense, and it *will* impact their productivity.

It can't not.

Scientific advancements aside, this is economically irresponsible

Every $1 spent by NIH generates $2.46

For example, in 2023, $47B in NIH spending generated ~$93B

Halting NIH spending will LOSE the US a lot of money (and talent)

Science and writing, thinking and rethinking

Open Access
www.cell.com/matter/fullt...

Hi! Thanks for this! Former Gilliam!

More Than a Small Brain: The Importance of Studying Neural Function during Development The nervous system contains complex circuits comprising thousands of cell types and trillions of connections. Here, we discuss how the field of “developmental systems neuroscience” combines the molecu...

Looking for an escape over the holiday?
@meikeesther.bsky.social and I published a ViewPoint looking at the intersection of Developmental and Systems Neuroscience titled “More Than a Small Brain: The Importance of Studying Neural Function during Development”

www.jneurosci.org/content/44/4...