A strong close to a banner year for progress against cancer: an opinionated take on NEJM highlights for December 2016

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Successful use of CAR-T therapy in a solid tumor

Chimeric Antigen Reception T-cells (CAR-T) are immune cells molecularly engineered to seek out and destroy cancer cells; the push to develop them into a scalable generally usable treatment is likely the most exciting challenge in cancer right now.  Successful CAR-T use has so far been generally confined to hematological tumors.  In a brief report, a group from City of Hope reports on the use of CAR-T in a patient who was dying from an advanced, aggressive form of brain cancer, which led to a regression of the tumors for several months. Beyond proper molecular targeting, key to success seems to have been direct delivery into cerebrospinal fluid in the areas affected by the tumor.  Regression of Glioblastoma after Chimeric Antigen Receptor T-Cell Therapy (free access)

 

A less complicated alternative to CAR-T?

Implementing CAR-T therapy it is technically challenging to scale, because it requires genetic re-engineering of human cells with a high degree of reliability, something that is not at all easy to achieve. For those cancers that already stimulate a significant cellular immune response, an alternative idea is to extract from a (surgically resected) tumor tissue block the T-cells, select those that are tumor reactive, and expand them in-vitro before reinjecting them in large numbers into the patient. In this case report, NCI scientists and clinicians describe performing the procedure in a patient with metastatic colorectal cancer, who went on the have dramatic regression of her metastases. An interesting twist to this therapy is that it is more akin to a bone marrow transplantation procedure than the administration of a new product. Given this, one wonders how the regulatory and IP frameworks around such an approach will eventually shape out.   T-Cell Transfer Therapy Targeting Mutant KRAS in Cancer; Drugging the Undruggable Ras — Immunotherapy to the Rescue? (subscriber access)

 

The future of health-product promotion

A fascinating perspective on how recent case-law, combined with cases currently winding their way through courts have the potential to overturn decades of established norms which constrain promotional activity for health products to labeled indications for which there is FDA-sanctioned evidence of safety and efficacy.  If this ends up happening, expect two things: (1) a much tighter health technology assessment process and utilization oversight by payers; (2) a lot more product-liability lawsuits. In this context, unfettered speech really is a two-edged sword for the health sector. Regulating Off-Label Promotion — A Critical Test (free access)

 

The growing importance of China

A short announcement that the Journal will be publishing Chinese translations of key articles; a rather unique instance of reversal from the overwhelming trend establishing English as the sole reference language of the sciences in the last decades. Making An Impact on Clinical Practice and Research in China (free 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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