15 year LTFU burdens on patients include:
-Frequent visits, testing, requirements
-Travel burden and costs, often tied to specialized treating centers
-Ongoing disruption to work, family life, and daily routines
-Psychological burden of prolonged monitoring with diminishing clinical yield
NEW - Data-informed optimization of CAR T-cell therapy long-term follow-up
At >60K patients treated, CAR-T LTFU is due for a reset. AEs are infrequently reported >3 years post-infusion, so 5 years is more appropriate considering low risk, cost burden, burdens on patients jitc.bmj.com/content/14/4...
-CAR-T Applications in Autoimmune Diseases
-CAR-T for Fibrosis & Neurodegenerative Disorders
-In Vivo CAR-T
-Local Production & Global Patient Access
Join us for the CAR T Scientific Signature Series event May 9 in Dublin @isctglobal.bsky.social Annual Meeting www.isctglobal.org/annual-meeti...
Top 5 #FDA Takeways
1) NGS now the regulatory backbone for genome editing safety
2) FDA wants layered approach to off-target assessment
3) Low-frequency events matter, must be detectable
4) Patient genetics now part of risk assessment
5) Genome integrity no longer optional for DSB-based gene editors
🚨NEW FDA Draft Guidance Safety Assessment of Genome Editing in Human Gene Therapy Products Using Next-Generation Sequencing #FDA #Regulatory #geneediting #genetherapy
Top 5 Takeaways in reply
www.fda.gov/regulatory-i...
What the peptide craze reveals about Americans’ relationship with risk. Great piece from @sarah-todd.bsky.social www.statnews.com/2026/04/06/r... It's so odd how many people will not trust proven biomedical approaches but embrace and literally shoot up with unproven things like peptides
Celebrating innovation at @upenn.edu ft. @marcoruella.bsky.social, Saar Gill, Stephen Schuster (@penncancer.bsky.social), Carl June, @bllphd.bsky.social (@pennpathlabmed.bsky.social), Mark Engleka, Michael Yocum (PennPCI), Nicola Mason (Penn Vet) & Liang Feng (SEAS) tinyurl.com/mryfmk7u
NEW - Public Service Announcements linked to Ring That Bell to run on radio stations across the USA are designed to raise awareness about the song and its connection to cancer advocacy. We aim to inspire action and support for cancer initiatives. Listen at RingThatBellSong.com below music video
🚨Congratulations to Prof. Dr. Carl June & Prof. Dr. Michel Sadelain! 2025 laureates of the Broermann Medical Innovation Award for the development of the CAR-T cell #immunotherapy #cancer #CancerResearch pressreleasehub.pa.media/article/dr-c...
Our website is up for Ring That Bell" ringthatbellsong.com
Is Germline Editing Feasible and Should it be Permissible? I joined @urnov.bsky.social, @kiranmusunuru.bsky.social, ISCT President Miguel Forte & Chief Regulatory Officer Bambi Grilley to discuss #ethics #bioethics & technological hurdles of embryo editing www.isctglobal.org/telegrafthub...
My CNBC.com segment on implications for pediatric vaccine schedule from recent politics. 2003 saw ~5,000 cases of pertusis, in 2024 there were over 35,000, and for 2025, some models show 70,000+
We risk a return of communicable diseases in children.
www.youtube.com/watch?v=00cI...
youtu.be/px__ULMCnLM?...
How future cures begin as ideas in laboratories ft. Carl June, @bllphd.bsky.social (@pennpathlabmed.bsky.social), David Porter, Stephen Bagley, Jakub Svoboda (@penncancer.bsky.social) & Donald O'Rourke (@PennNSG) tinyurl.com/32hd7y2m
🚨Just published - Multiplex engineering using microRNA-mediated gene silencing in CAR T cells #celltherapy #genetherapy #immunotherapy www.frontiersin.org/journals/imm...
Announcing the website for the song Ring That Bell 🔔 cowritten w @magsmccarthy.bsky.social an emotional story of a cancer patient facing relapse and the fierce advocacy, support, and groundbreaking science that offer new hope. #cancer #cancerresearch #immunotherapy #hope ringthatbellsong.com
Nearly two dozen people developed food poisoning in Florida after consuming tainted raw milk from the same farm, state health department says (from @reportergoodman.bsky.social) www.cnn.com/2025/08/05/h...
🚨Coming soon! Multiplex engineering using microRNA-mediated gene silencing in CAR T cells. In a single lentiviral vector- multiplex silencing & expression of a CAR, better expansion & viability than gene editing by electroporation www.frontiersin.org/journals/imm...
More disease coming, unfortunately in kids. Adults making the bad decisions for them.
My new 2-minute 5-point short on why one shouldn't go to stem cell clinics or at least seriously weigh risks + low chance of benefit. Good little video for scientists and physicians too looking for clear explanation when people ask #stemcells #stemcell www.youtube.com/shorts/EX9iU...
Cutting NIH to “save” money is fundamentally flawed
-Modest annual savings pale compared to multi‑trillion‑dollar health, innovation, and productivity losses www.statnews.com/2025/07/28/n...
From Pink Sheet on continued departures from FDA’s biologics division and challenges backfilling openings. FDA biologics center reported 106 departures from Jan. 1 to June 30. These numbers don’t include the 100-150 who were part of the reduction-in-force
insights.citeline.com/pink-sheet/a...
FDA’s cancer review team overseeing breast and gynecological products has lost about 1/3 of its medical review staff and the malignant heme group will lose about 2/3 of its medical review staff—all to voluntary resignations clustered among seasoned reviewers
insights.citeline.com/pink-sheet/a...
There should be a cable channel fact checking all the cable news channels and their paid "pundits" in real time
If gifted narrative filmmaker Marwin Gansauge asks, "Can I make a movie about you?", what do you say? More than ever, science communication is desperately needed to convey the importance of research in developing new treatments for patients. Filmed in Vancouver & Dublin youtu.be/YrevpY-DNmw
Is Makary's FDA already heading toward a breaking point? ipscell.com/2025/07/is-m... #stemcells #stemcell #CRISPR what does it mean for the cell and gene therapy field?
How did the pandemic start?
I have avoided publicly commenting on this for 5 years. Here's my take as someone who has no stake at all in the game.
gidmk.substack.com/p/pandemic-r...
If real facts matter to you
Sign of the times.