I didn’t forget about you last month! It just seemed sensible to focus on ICAS exams (see vlog). So, I decided to postpone and combine the November and December newsletters. Thank you for your patience! 💐
Nov/Dec on Blue Steens
Paying for patient expertise
Scientists use the unique insights patients provide to inform their research. Working with all stakeholders early in the R&D process can help make it more productive and lower development costs. Despite a consensus that patients and patient advocates should be compensated for their contribution, it is unclear how.
Who is afraid of … granny?
After coming across an article talking about the 'grey tsunami', I couldn’t help but to utter some thoughts about ageism in this quick, spontaneous blog post. By the way, who comes up with such terms, and why do we reinforce them? What has happened to our intergenerational communication and appreciation?
As a Man Thinketh
Whenever you are torn by doubt or pain, pick up James Allen’s ‘As a Man Thinketh’. A quiet, poetic little book to reinvigorate a resilient mindset.
Will bonding curves be researchers’ best friends?
Continuous token bonding curves, formerly referred to as curation markets, describe a blockchain application with interesting qualities for academic researchers of any scientific specialism. Can they fix academic publishing?
The first 5 ICAS exams are over. It was a mixed bag despite solid preparation. Let’s see what the results will be.
Some more study tips have been added to the playlist.
Next month on Blue Steens
We will explore
... economic suggestions to incentivise the development and commercialisation of antibiotics.
Beyond Blue Steens
Autologous cell-based therapy
Novartis dropping internal efforts on CAR T in solid tumors - Novartis will focus its CAR-T strategy on blood cancer and step back from solid tumours. CAR-T are used in an immunotherapy that manipulates the patient’s own T cells to fight cancer. One major obstacle in solid tumours is their immunosuppressive environment (tumour defence strategy), which prevents the protective activity of these T cells.
On that note - Danish researchers develop new type of immunotherapy - A nanoparticle-based immunotherapy developed at Aarhus University harbours the hope to be effective against all types of cancer. It inactivates an immunosuppressive gatekeeper in the tumour vicinity to unleash the natural tumour-killing immune response. Besides the therapeutic promise, I find the approach fascinating because I used to research monocytes/macrophages, key actors in this strategy.
First patient dosed with Chiesi’s Holoclar - The first patient outside a clinical trial received stem cell-based NHS treatment indicated for chemical eye burns. The therapy involves transplantation of a tissue-engineered cellular layer derived from the patient’s own eye cells.
This kind of tech is not available for complex organs yet. However, progress has been made in performing allografts (transplanting donated organs) using hearts after cardiac/circulatory death - First donation after circulatory death heart transplant performed in the U.S. - Successful interventions have been performed in the UK, Australia and now also USA.
Pot, bugs & FDA
NICE backs first cannabis-based drugs, both from GW Pharma - This is the first time that NHS England makes plant-derived cannabis-based drugs available to patients. The indicated diseases are epilepsy and multiple sclerosis. The drugs’ non-psychoactive main active ingredient, cannabidiol (CBD), has been available as oil in high-street shops for a wee while now. It is interesting to see licensed CBD products finally passing NICE assessment. Disclaimer: I worked as a laboratory researcher in a joint project between GW Pharma and the University of Aberdeen a few years ago.
FDA approves new antibacterial drug to treat complicated urinary tract infections as part of ongoing efforts to address antimicrobial resistance - New antibiotics hitting the market is always great news, as some say we’re in a crisis on that front with antimicrobial resistance having outpaced drug development. Blue Steens’ January article will elaborate on this.
FDA Approving Drugs at Breakneck Speed, Raising Alarm - The FDA has been approving drugs in critical areas, such as rare diseases, in high numbers and on tight schedules over the last years. Granted, new technologies mean that we finally have (potential) therapies for previously untreatable conditions. Whilst accelerated review speeds up patient access, consumer advocates question safety and efficacy of fast-tracked drugs. Underneath this lie complex questions around risk and stakeholder priorities.
Speaking of rare diseases and the need for speed - Low enrollment stymies completion of rare disease clinical trials - “Overall, almost 11,800 patients were enrolled in trials that were completed but were not published after four years.”
Righteye wins breakthrough device designation for Parkinson’s test - The device tracks a patient’s eye movements as part of the Parkinson diagnosis. The hope is that this non-invasive technique will enable early and more accurate identification of the disease thanks to its AI and machine learning capabilities. It is unclear when it will be commercialised.
Deep learning with nuke med quantifies prostate gland uptake, predicts cancer survival - A trained and validated deep-learning algorithm for analysing prostate PET/CT scans that may be used for prostate cancer prognosis. Apparently at R&D stage.
Mainstay Medical receives Australian approval for neurostimulation implant - The device is used to treat lower back pain and had already been approved and used in the UK. The implant connects to the spine to stimulate muscles involved in counteracting the pain mechanism.
A silicon chip that mimics the brain’s neurons could help fight paralysis - The implant communicates like artificial neurons and could be used to replace or support nerve cells impaired by injury or disease; suggested indications are heart failure and Alzheimer’s.
Samsung execs face jail for destroying evidence in Biogen JV fraud probe - After breaking accounting rules by inflating the joint venture value (by $3.9 billion before IPO in 2016) and hiding or destroying data and documents, 3 Samsung BioLogics execs face prison sentences of up to 2 years.
Let’s not end on a bad note…
2019 pharma/biotech Year in Review by AtlasVenture (works well at 1.5x speed if you want to save time):
Innovation nation: keeping Scotland at the forefront of science and technology research - Showcasing this out of local pride for my chosen home. 🏴
“In fact, 77 per cent of Scottish research was classified as either world leading or internationally excellent in the most recent Research Excellence Framework exercise and 85.9 per cent was judged to have an outstanding or very considerable impact on the economy, society and culture.”
R. Willis, 1847, The Works of William Harvey, M.D., Letter IX [p. 616] - Below is a wonderfully open-minded quote taken from a letter by the renowned physician William Harvey. It was written in 1657 in response to another physician asking for assistance with an unusual case. I’d like to extend its meaning to any rare or less common medical condition beyond the definition of disease; for example gender incongruence. In its widest sense, Harvey’s thoughtful statement teaches us that the unusual helps explain the usual; that we can learn from the differences between us.
“Nature is nowhere accustomed more openly to display her secret mysteries than in cases where she shows traces of her workings apart from the beaten path; nor is there any better way to advance the proper practice of medicine than to give our minds to the discovery of the usual law of nature, by the careful investigation of cases of rarer forms of disease.”
Enjoy the festive break, “Guten Rutsch” and happy New Year!