The quest for solid tumor CAR-T
For all their success in hematological cancers, the track record of CAR-Ts against solid tumors has been disappointing (though see here for an exception). One key obsta…
A Chinese study uncovers a new use for an old drug
Bleeding in the small intestine is often due to vascular malformations and is difficult to diagnose and manage because 1) the area is hard to reach w…
Tirzepatide in pole position
As previously described in this blog, GLP-1 agonists initially designed to treat glycemia in Type-2 diabetes are emerging as powerful weight loss agents in obesity indepen…
In search of a diagnosis: deploying genomics at scale
A substantial number of children have a disease identified as “rare” without having any kind of causative diagnosis (autism is not a causative dia…
New molecular diagnostics, especially tests based on next-generation sequencing and gene-panels, could revolutionize how diseases are evaluated. But experience shows that insurers won’t cover these te…
Genomic applications and scalability
Two very different papers about applications of genomics, one for a very common clinical scenario, the other for an ultra-rare disease.
It is well known that while…
Is the lack of new (non-viral) antibiotics a market failure?
A lament about the lack of success in getting the biopharma industry to invest sustainably in the development of new antibiotics against h…
Huntington’s disease: light at the end of the tunnel?
The awful thing about Huntington’s is that in most cases, people know they are going progressively lose their mind at an early age, but there is …
Target assessment with genetic polymorphisms
Please bear with me for a long (but interesting!) story. Bempedoic acid (Esperion) is an inhibitor of ATP citrate lyase (ACLY), an enzyme in the cholestero…
Genetic trade secrets
In 2012, the US Supreme Court decided a case known as “Prometheus” establishing that therapeutic methods based on biomarkers were not patentable – this essentially blocked one of…
GWAS, Regeneron and Geisinger, and liver disease
Genome wide association studies (GWAS) look at broad populations for gene variants associated with a particular phenotype. Often, like in Type II diabe…
There is such a thing as too much hygiene
The prevalence of asthma in children has increased dramatically over the last few decades. Observational studies have shown that children in “dirty” environme…
Surgical volume and referral for surgery:
The impact of surgical volume on outcomes has been well documented, but is it top of mind with physicians referring patients to surgery? Readers of the Journa…
The rise, fall, and rebirth of the Chinese healthcare system
A fascinating account of the evolution of the Chinese healthcare system which almost seems to be an upside-down picture of the rest of the …
A promising agent for Crohn’s Disease, a miserable illness
Crohn’s is an inflammatory bowel disease that is notoriously unpredictable; flares can affect any part of the digestive tract and lead to gra…
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