We refresh a prior analysis of two cohorts of oncology development programs: one with first-in-human (FIH) in 2012, and the other with FIH in 2017H2. We use a Kaplan-Meier approach to track whether pr…
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…
Summary
After challenging conditions in 2022, deal-driven biotech spending appears to have picked up in 2023. To quantify this trend we have systematically reviewed deals announced during this period …
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…
PDF here
Summary
The idea of a single blood-test to detect many cancers is attractive, but is it practical? Can an MCED enhance, or even replace, current screening tests?
This paper delves into these …
Download a PDF of this article here.
Hematologic cancers account for ~10% of annual new cancer diagnoses and continue to have some of the poorest overall outcomes, particularly for older adults.[1] Ho…
An unusual article on road safety
Motor vehicle accidents are a major share of morbidity and mortality in the young, and as such prevention has long been considered a medically adjacent area (e.g. wit…
Is Parkinson’s α-synuclein going to be as elusive a target as β-amyloid has been for Alzheimer’s?
Alpha-synuclein aggregates are characteristic of Parkinson’s disease and genetic variants of this prot…
PDF: Trends in oncolytic virus therapeutics: then and now
The idea of using pathogens to treat disease is not new; ranging from phage therapy to malariotherapy (resulting in the only Nobel in medicine…
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…
A cell therapy success against a solid tumor
In CAR-T therapies, T-cells are transformed to express an antibody on their surface that allows them to home in on cancer cells and effect killing. This ap…
The opioid crisis and patient abandonment
A perspective from a February issue that I originally missed highlighting the plight of patients who have been on a long-term opioid regimen for chronic pain.…
There’s a major disruption looming in how the site-of-care for infusions is managed which will have repercussions for plan competition, delivery system economics, PE hunting for healthcare opportunity…
Cancer and mutational complexity
Probably the biggest news in cancer treatment this year is the approval of sotorasib (Lumkras, Amgen) which received accelerated approval in lung cancer with mutated K…
We can cure Hep C but not immunize against it
Sovaldi and other drugs have made curing chronic Hepatitis C a routine, if expensive, proposition. Still, given how common Hep C infection is, and how it …
Small but real progress in ALS:
Despite enormous public attention and significant effort, ALS remains a disease for which the development of new therapies has been challenging. Animal models showed po…
PDF: An ADC renaissance: a resurgence of antibody drug conjugates for cancer
Summary:
Antibody-drug conjugates (ADCs) have seen a record number of approvals in 2020. This comes after a first era of i…
Remdesivir works… but not enough to change the public health perspective
The eagerly awaited results of the remdesivir NIH trial are out, and it’s solid but not smashing, although this is a partial re…
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…
TAVR for all?
TAVR has become the standard of care for patients who need an aortic value replacement but are at high/medium surgical risk. But what about those at low-risk? Two studies answer that qu…
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…
Targeted conjugated cancer therapeutics – so few, but may be more soon
The idea that one could combine the precision of a targeted biologic with the potency of a traditional cytotoxic to demolish a tu…
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…
Peanut medicine that won’t cost peanuts
Allergy to peanuts is a major issue and though prevention is now possible in infants there is a huge population for whom actual survival is connected to vigilan…
Transthyretin heats up
Last month saw a couple landmark papers about the use of parenteral RNA drugs (from Alnylam and Ionis) in hereditary transthyretin amyloidosis with a focus mainly on mitigating …
It’s hard to quit (and E-cigs don’t help)
A large trial (6000 participants) comparing free cessation support, e-cigarettes, and a $600 cash incentive for sustained abstinence shows that none of these …
Are we nearing an asymptote with implantable cardiac pumps?
Severe heart failure is common, and spare hearts for transplant are rare, which has led to the development of implantable mechanical alterna…
A-fib in heart failure – time to be aggressive
Over the last 15 years, there has been a growing body of evidence for the effectiveness of catheter ablation to treat atrial fibrillation (a-fib), a cond…
Playing chess against cancer
Tumors are not intelligent, but, because they have escaped mutational control, they constantly probe for mutations that will allow them to escape chemotherapeutic suppress…
Successes in gene therapy for hemophilia B and A
Hemophilia A and B are X-linked genetic diseases which prevents the formation of functional coagulant factors VII and IX respectively and cause a prope…
Taking stock: two decades of progress in heart failure:
Here comes a clever study using existing clinical trial data to assess progress in standard of care over time for heart failure. For each trial,…
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 …
“My name is T-Cell…, James T-Cell”
Immune T-cells are licensed to kill other cells through a quick molecular kiss of death, and as such are potentially powerful allies in controlling a tumor. For obvi…
I am not sure how many docs continue to do this, but I still read the actual hard copy of my NEJM, and that means I flip past ad pages with smiling grandfathers playing with grandchildren thanks to su…
Continued progress in multiple myeloma
About 25,000 patients are diagnosed with multiple myeloma yearly in the US. Despite being initially treatable, typically this disease is ultimately lethal. Follo…
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…
The recent World Medical Innovation Forum on Cancer convened by Partners Healthcare in Boston was attended by leaders in oncology from around the world including top: clinicians, bench scientists, pol…
A new focus on the diagnostic reliability of clinicians
Two perspectives highlighting the recent report from the National Academy of Medicine (formerly IOM) entitled “Improving Diagnosis in Health Car…
Systems biology finally gets real: an unexpected use for a diabetes drug
Chronic Myelogenous Leukemia (CML) has been the poster child first for a disease with a precise genetic cause (the Philadelphia…
What are annual physicals for?
Annual physicals are costly (~$10 billion annually) and have never been shown to improve outcomes, but people value them. In this dichotomy lies a lot of the inner tensi…
Promise for systemic amyloidosis (and beyond?)
Systemic amyloidosis is an uncommon disease in which abnormal cells (typically antibody-producing B cells) produce large amounts of protein that deposit …
The emergence of a new approach to drug development in cancer
Cancers are classified by the organ or tissue from which they arise, but as our molecular understanding increases, another level of categ…
Palbociclib – first to target cyclin dependent kinases – breast cancer
As all biology majors know, cyclin dependent kinases are critical elements controlling the machinery of cell proliferation. They…
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 …
Our selection from a month with relatively few exciting articles - perhaps this long Boston winter has us all down.
Precious metals and health plan buying:
The implementation of the ACA has placed …
A vaccine for dengue finally nears the market
Dengue is a mosquito transmitted viral infection that is often severe and occasionally fatal, and that has been identified as a growing public health thre…
Summary
Allegheny Health Network (AHN), the major delivery system in Pittsburgh owned by Highmark, and Johns Hopkins Medicine have signed a MOU to create an affiliation between Allegheny and the John…
Summary
The case for an oncology ACO can be compelling but CMS rules for ACOs within fee-for-service (FFS) make value difficult to demonstrate
A new physician-hospital-payer partnership in Florida …
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