Pearl Freier

*The deworming drug is fenbendazole not enbendazole. Sorry for the typo above

A 45 year-old man died after taking a deworming drug for animals. He had read about it having anti-cancer properties from social media sites—according to what his partner told the BBC. He bought the drug enbendazole from online supplier in a different country www.bbc.com/news/article... #medsky

Below is also a potential scenario at the NIH under HHS Secretary Kennedy—following the firing yesterday of the US jobs data statistician at BLS by POTUS. Below was likely meant to be a sarcastic post by a well-respected cardiologist but it's also a possibility at the new NIH/HHS #medsky

A potential resource 2 help patients whose prescription drug insurance isn't covering their drug—"Call the Inventors." A new AI & patient assistance chatbot by
@nplb.bsky.social for patients to look up patient support to ask about lower prices www.nopatientleftbehind.org/calltheinven... #medsky

Machine-learning driven strategies for adapting immunotherapy in metastatic NSCLC - Nature Communications The approval of first line immune checkpoint blockade (ICB) has improved outcomes for patients with metastatic non-small cell lung cancer (mNSCLC), however, whether patients would benefit more from IC...

Here's the link to the paper in Nature Communications: www.nature.com/articles/s41...

Machine-learning driven strategies 4 adapting immunotherapy in metastatic non-small cell lung cancer #NSCLC patients 2 choose betw immune checkpoint monotherapy & immunotherapy w/chemo combo 4 patient since guidance is ltd. Single-feature biomarkers i.e. PD-L1 are inadequate. Paper is below #medsky

Senators in Alabama, Louisiana, West Virginia, South Carolina, Maine, Kentucky, Alaska, Indiana, Kansas, & Pennsylvania also signed the letter. Universities & research centers that were supposed to receive NIH funding are at risk of losing thousands of jobs because of the freeze: #medsky #biosky

North Carolina senator & 13 of his colleagues sent a letter to Vought (Dir of White House Office of Management & Budget) to release the NIH funds immediately that Congress has already appropriated for research in cancer, heart disease, & rare disorders. www.tillis.senate.gov/2025/7/tilli... #medsky

PKU is rare disease phenylketonuria

PTC Therapeutics's oral drug for rare metabolic disease PKU was approved by the European Commission in June. The FDA was allowed to follow the EC's decision & approved Sephience yesterday to give US patients access too www.reuters.com/business/hea... #biosky #medsky

Project 2025 is gutting medical funding that helped Russell Vought's own kid Its architect's daughter has cystic fibrosis—and benefits from a "miracle drug" backed by an agency he's attacking.

Given his performance on @cnn.com, it’s time to tell this story again. Vought’s daughter is alive because of NIH. He’s destroying an institution that saved his daughter’s life. @mrjoncryer.bsky.social @jamellebouie.net @atrupar.com www.motherjones.com/politics/202...

This isn't biopharma or healthcare news-related but just sharing this anecdote from the new Billy Joel documentary. Billy: I once asked a great chef how he got to be so good. He said it's all in the recovery—how you correct your mistakes.

Here's Sean Khozin's blog post: From Breakthrough to Breakdown: The $900 Million FDA Rejection Letter www.insights.phyusionbio.com/p/from-break... #biosky #oncsky #melanoma

The collision between biological complexity and regulatory precision reached a defining moment today, when the FDA's rejection of Replimune's RP1 oncolytic virus therapy crystallized fundament... The collision between biological complexity and regulatory precision reached a defining moment today, when the FDA's rejection of Replimune's RP1 oncolytic virus therapy crystallized fundamental tensi...

For followers of FDA & oncology wondering why past & current FDA-ers are discussing Ph 3 melanoma drug on Linked In, below is a link to Sean Khozin's LI post in reply. He's an oncologist & ex clinical investigator at NCI (9 yrs) & ex FDA Senior Medical Officer (7yrs) www.linkedin.com/posts/seankh...

It looks like an FDA employee at CBER has publicly confirmed on Linked In in response to past FDA employee Sean Khozin's post that Prasad intervened to prevent Replimune's Phase melanoma drug from getting approved cc'ing @dereklowe.bsky.social @dshaywitz.bsky.social @erictopol.bsky.social #biosky ⬇️

FDA's Chief of Oncology Branch 1 at CBER wrote on Linked In re: Phase 3 drug rejection in response to Sean Khozin's
post: "the BLA clinical team thought the applicant had provided adequate evidence to support contribution of effect of the RP1 to nivolumab but leadership did not agree." #biosky

A record number of measles cases, whooping cough cases, and flu-related deaths in the US & HHS Secretary Kennedy also recently got FDA to block most Americans from getting a Covid vaccine in the fall if they want one. Why hasn't Kennedy been fired as HHS Secretary yet? #medsky

FDA’s cancer review teams lost ~1/3 of the medical review staff that oversee products 4 breast & gynecological cancers & malignant heme group may lose ~2/3 of its medical review staff h/t ex FDA Commish ('17-'19) & @sarahkarlin-smith.bsky.social insights.citeline.com/pink-sheet/a... #medsky #biosky

As revenge against everybody who sent him mean tweets during COVID, Jay Bhattacharya gets the honor of being the NIH Director who willfully killed American biomedical science

US FDA Cancer Reviewers Heading For The Exits, Potentially Impacting Review Timelines US FDA's cancer drugs division is feeling the effect of drug reviewer departures, despite efforts to keep them at the agency.

FDA’s cancer review teams lost ~1/3 of the medical review staff that oversee products for breast & gynecological cancers & malignant heme group may lose about 2/3 of its medical review staff h/t ex FDA Commish ('17-'19) & @sarahkarlin-smith.bsky.social insights.citeline.com/pink-sheet/a... #medsky

US FDA Cancer Reviewers Heading For The Exits, Potentially Impacting Review Timelines US FDA's cancer drugs division is feeling the effect of drug reviewer departures, despite efforts to keep them at the agency.

One of FDA's Oncology Division has lost what is essentially the equivalent of a full drug review team. It's malignant hematology review teams are expected to have a staff less than half the intended size as more staff depart this August.
insights.citeline.com/pink-sheet/a...

Here's the link to Ness Birmingham's op-ed in The Hill thehill.com/opinion/heal... #biosky

Here's the link to Ness Birmingham's op-ed in The Hill thehill.com/opinion/heal... #biosky

Ness Birmingham, ex Intellia Tx CEO, said that clear regulatory rules at FDA for personalized therapeutics would "unlock investment, while reimbursement frameworks will ensure payers can cover these treatments responsibly. Absent this, safe & effective therapies may never reach patients." #biosky

In order to turn personalized therapies for rare diseases (like baby KJ) into a new standard of care, Birmingham said FDA should establish a dedicated oversight group within FDA to review personalized therapeutics, set transparent safety & ethics standards & ensure rigorous tracking of outcomes.

Former Intellia Tx CEO now operating partner at @khoslaventures.bsky.social Ness Birmingham published an op-ed in @thehill.com calling for the FDA to have a dedicated framework that clarifies the rules, requirements & incentives including a new pathway for bespoke therapeutics & n-of-1 drugs #biosky

Assuming the NDA was filed around the same time, we might get to see a comparison in agency response rates. Or how understaffed and behind the FDA is right now...

All from a global Phase3 with the most sites/country in the US and most profits likely to come from the US. But great for SMM patients!

NCCN now recommends Daratumumab as category 1 option for high risk Smoldering Myeloma h/t @vincentrk.bsky.social #medsky #oncsky #biosky

It's unusual for the FDA to be behind the European Commission on a drug approval like this & prev a patient population w/ltd options. Were too many people laid off at the FDA or are there other problems? ODAC gave a positive vote in favor of approval h/t @vincentrk.bsky.social
& NCCN 1/n #medsky

Here's an NEJM article from December about the Phase 3 AQUILA trial: www.nejm.org/doi/full/10....