A step forward for kidney xenotransplantation
A report on the recent, well-publicized, temporary transplantation of kidneys from genetically modified pigs in two deceased individuals at NYU. The kidneys functioned well and there was no sign of acute rejection, but because these experiments took place over only 54 hours, this is not indicative of longer term compatibility of the transplant of kidneys from this particular strain of pigs. A question in my mind is why there have not been kidney xenotransplants in live patients yet – especially given a recent porcine heart transplant in a live patient at UMD and a good track record in kidney transplants into non-human primates. I suspect that part of the answer is that there are still clear ways in which to fine tune pig genetics for added compatibility: particularly where the coagulation and complement pathways are involved. Still, we are clearly getting very close to the first live human kidney xenotransplantations. Results of Two Cases of Pig-to-Human Kidney Xenotransplantation; Progress toward Pig-to-Human Xenotransplantation
Hope for IPF
IPF (Idiopathic Pulmonary Fibrosis) is the ALS of the lungs: an inexorable process that destroys healthy lung tissue leading to death in 2-4 years without a lung transplant. In the early 2010s, a couple drugs were approved for IPF (pirfenidone and nintedanib), but they only slow down progress of the disease. This is why preliminary results of a 12-week, phase 2 placebo-controlled study with the phosphodiesterase inhibitor BI 1015550 (Boerhinger Ingelheim) that appear to show a halt of progression are exciting. However, the analyses depend on Bayesian model or mixed model repeated measures, and when one looks at the primary data from the supplementary materials, the signal is dwarfed by the standard deviations, which for N in the double digits, leaves significant doubts on significance. BI is teeing up two large 1-year phase 3 trials which should allow a definitive finding – in about 3 years. Trial of a Preferential Phosphodiesterase 4B Inhibitor for Idiopathic Pulmonary Fibrosis
340B weirdness
The 340B program is a mechanism which requires pharma companies to provide drugs at a discount to participating organizations caring for low-income populations. These organizations get to charge full price to insurance, thus collecting a significant margin that supports indigent care (though, as this paper in the Journal attests, whether this is really the case is up for debate). Biopharma generally does not like this program, but in some situations, this might be flipped on its head. A Perspective article highlights how access to PrEP (Pre-Exposure Prophylaxis for HIV) has so far been very expensive, something that was supposed to change now that cheap generics have recently become available. Instead, hospital clinics are switching to much more expensive/non-generic PrEP drug combinations even though efficacious and safe generics exist; the 340B income from the high priced medication is just too attractive to pass up. Perverse Incentives — HIV Prevention and the 340B Drug Pricing Program
The disappointing state of low back pain understanding and management
Low Back Pain (LBP) is an under-appreciated but nevertheless huge cause of morbidity: and while it does not usually cause death, for many, it has been an entry point to opioid use with the consequences that we know. This is what makes a review of current evidence for the clinical management of LBP in the Journal so depressing – it’s simply so weak. Relative to cancer or other severe pathologies, we are a simply not investing the resources commensurate with the burden of disease from low back pain. Nonspecific Low Back Pain
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.