Happy Women’s day. 💜On this important day, let us appreciate accomplishments, raise awareness of disparities, and give our support to women.
In that spirit lets take a look at, Marie Skłodowska-Curie: “l’exception scientifique”. As a scientist, I’ve heard her name many times, but have only come to appreciate her incredible determination and commitment recently. Marie had to move to France in 1891 because she was not allowed higher education in her native country, Poland. At the time STEM was very heavily male dominated: have a look at the picture of the 1927 Solvay conference [picture below]: 29 attendees, 1 woman.
Despite all this Marie was the first women to receive a Nobel price and first person to receive two Nobel prizes in two different disciplines (Chemistry and Physics). With only 65 Nobels to women to date, women are still very much a minority = 6.7% of the total pool of laureates. That being said, there are signals of improvement, a great example being the recent awards to Doudna and Charpentier, who won the prize in 2020 for their discovery CRISPR/Cas9 (!)
Now, I’m starting from my family at the youngest age possible - 8 months: I bought a book for my daughter “Little People, Big Dreams: Marie Curie” [link]… and she’s already heard the story like 20 times 😃
BlueRock Tx Ph I clinical trial for Parkinson’s disease continues to show positive trends at 18 months
BlueRock / Bayer’s cell therapy, Bemdaneprocel, continues to show signals of efficacy 18 months in, continues to show survival + engraftment of transplanted cell, and a relatively clean safety profile.
Using the Hauser Diary, which categorizes patients as being in the “ON” state when their symptoms are well controlled and in the “OFF” state when they experience a worsening of their symptoms, participants in the high dose cohort at 18 mo showed a mean increase of 2.7 hrs in time spent in “ON” vs baseline. Inversely, time spent in the “OFF” state decreased by 2.7 hours. The effects seem to be dose dependent and participants in the low dose cohort showed relatively less pronounced improvements. Importantly, F-DOPA signal continued to increase after stopping immune suppression therapy at 12 mo (per protocol).
A phase II study for further clinical testing of bemdaneprocel is planned to begin enrolling patients later this year.
Source: Bayer website
Quick take news
Novartis reports data from Phase IIIb trial of SMA therapy (SMART), affirming the safety and efficacy of Zolgensma for treating SMA [source]
Historically Zolgensma trials enrolled patients <8.5 kg with concerns about safety issues in older, given weight dependent dosing; in the new trial, patients between weight of 8.5 and 21 kg were enrolled, and most were either on Spinraza or Evrysdi. Nothing unexpected in the context of AAV gene therapy: All patients had >1 treatment associated side effects, generally elevated liver enzymes (a sign of liver damage) and/or reduced platelets
Regenxbio revealed new data from an early trial of a gene therapy for Duchenne muscular dystrophy that suggests it may work for older patients with the muscle-wasting disease [source]
City of Hope-developed Chimeric Antigen Receptor (CAR) T Cell Therapy Shows Clinical Activity in Patients With Aggressive Brain Tumors in a Phase 1 Trial [source]
Solid Biosciences Announces Licensing Agreement with Armatus Bio for the Use of AAV-SLB101, a Proprietary, Muscle-Targeted Capsid, in the Development of an RNAi Therapy to treat FSHD [source]
bit.bio Announces First Project Within Multi-Year Collaboration Agreement with The Michael J. Fox Foundation (MJFF) to Generate Human Cell Products for Research and Drug Discovery in Parkinson’s disease [source]
CAR-T cell therapy trial tracker - key themes from this week
5 new CAR-T studies posted on clinical trials.gov this week
2 x GPRC5D CAR-Ts in MM: on from BMS - BMS-986393 [source] and one from Guangdong hospital [source]
3 x CD19 CAR-T in autoimmune: (1) Kyverna in Adult Dermatomyositis [source], (2) Shanghai Ming Ju Biotechnology in SLE [source] and (3) Sana’s SC291 in lupus nephritis, extrarenal systemic lupus erythematosus, or anti-neutrophil cytoplasmic antibody-associated vasculitis [source]
Side note: what happens when you get vaccinated 217 times?
Freshly published in the Lancet… a report of a 62-year-old male hypervaccinated individual from Magdeburg, Germany, who deliberately and for private reasons received 217 vaccinations against SARS-CoV-2 within a period of 29 months. Some interesting facts:
Throughout the entire hypervaccination schedule he did not report any vaccination-related side effects
The man does not seem to have contracted SARS-CoV-2 infection = success
Importantly, while the case study suggests the individual did not get COVID, the final sentence and conclusion of the paper is clear: “We do not endorse hypervaccination as a strategy to enhance adaptive immunity”, i.e., Kids dont do this at home (!)
Source: the Lancet
*Any views and opinions expressed herein are those of the author (Marco Sabatini) and do not necessarily reflect those of his employer
Have I missed anything? Is there something you would like to hear more about? let me know !