Between the very common and the very rare – An opinionated take on NEJM highlights for October 2017

Between the very common and the very rare – An opinionated take on NEJM highlights for October 2017


Another tool for prevention in cardiovascular disease

Taking aspirin daily has been standard of care for cardiovascular disease for decades, but attempts to demonstrate the additive usefulness of other agents to prevent clot formation have not been successful. Now things have changed: in a large study of patients with established cardiovascular disease, those who took rivaroxaban (Xarelto, Bayer) daily on top of aspirin did markedly better than with aspirin alone, although with increased risk of bleeding (mostly gastrointestinal, but very rarely fatal or disabling).  Interestingly, the number of major cardiovascular events avoided by the combination (~0.7 event / 100 treated patients / year) appears to me to be similar to what is observed with a PCSK9 drug, and given that rivaroxaban is less than half the cost, it’s a bargain!  Rivaroxaban with or without Aspirin in Stable Cardiovascular Disease; An Important Step for Thrombocardiology (subscriber access)


Change is afoot for rare congenital diseases

Providing chronic external supplementation of a missing enzyme has been a successful approach for a significant number of rare congenital diseases – it has also led to tremendous economic value creation for some biopharma companies.  Now, as the technology for reengineering stem cells becomes mature (e.g. as seen in CAR-T therapies for cancer), the paradigm appears poised to change from one of chronic treatment to definitive therapy through direct insertion of the missing gene in harvested autologous stem cells which are then reinfused and produce the missing enzyme endogenously. Leading the charge are diseases for which there is no option for external enzyme supplementation and for which allogeneic bone marrow transplantation has been validated as an (imperfect) approach. An example is Cerebral Adrenoleukodystrophy for which a study of 17 children who received cells reengineered by a Bluebird Bio protocol has led to results that are much better than the transplantation alternative (which anyway depends on the availability of a matched donor). It is only the beginning though, and a number of biopharma companies whose business depends on chronic therapies of rare and ultra-rare diseases will need to figure out a world where cures are actually possible, and eventually, we hope, common.  Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy; Optimizing Treatment for Cerebral Adrenoleukodystrophy in the Era of Gene Therapy (subscriber access)


The New England Journal of Medicine is a premier weekly medical journal covering many topics of interest to the health sector. In this monthly series we offer an opinionated perspective on selected highlights that might be of interest to our clients and others.

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