Outside the Huddle: 10/9/22
By Jared Dashevsky, MD
1) How Covid-19 Has Evaded the Immune System
A recent study out of the University of Pennsylvania gives us a new explanation as to how the SARS-CoV-2 virus evades the human immune system. DNA in our cells wraps around proteins called histones, which make it more compact, preventing cells from transcribing those DNA segments into mRNA and, therefore, preventing translation into protein. The researchers found that SARS-Cov-2 contains a protein called Orf8 that prevents cells from removing these histones, thus preventing our immune system from reading and responding to the viral DNA. Check out a summary version of the study here (paywall) if you want a more detailed explanation, or take my word that we now have a deeper understanding of how the virus has evolved.
2) FDA Approves Amylyx’s ALS Drug, Priced at $158,000 per Year
The FDA approved Amylyx’s ALS drug, which is the first treatment to significantly slow disease progression and functional decline. The drug’s list price will be $158,000 annually, although patients won’t pay that amount. Last month, I wrote about FDA advisors voting in favor of approving Amylyx’s AMX0035, an ALS drug with modest benefits for a disease with severely limited treatment options. They had previously voted against approval but changed their minds after seeing more anecdotes, analyses and a promise to withdraw the drug if the ongoing larger clinical trial were to fail. In some ways, this story is similar to the controversial Aduhelm approval, but AMX0035 reportedly has a much lower risk of harm to patients than Aduhelm did. If the ongoing trial shows success, this will be a much-needed glimmer of hope for those affected by such a devastating disease, but a lot is riding on that success.
3) Mark Cuban’s Cost Plus Drugs on Track for Profitable 2023
Cost Plus Drugs, which is less than a year old, is now on track to be profitable by 2023. While it’s rare for new companies—especially those in healthcare—to turn a profit so quickly, I’m not surprised about Cost Plus Drugs, given its 10% week-over-week growth. The company also plans to open its manufacturing plant next month. Mark Cuban’s disruptive online pharmacy is successfully removing the middlemen (insurance plans and pharmacy benefit managers) from the equation to lower the cost of drugs. For some, those savings can reach upwards of 99% compared to traditional pharmacies. Overall, the road ahead looks quite bright for Mark Cuban’s pharmacy, and I hope they can expand their offerings to help even more patients in the coming years. I’ve already referred some patients I took care of in the hospital to Cost Plus Drugs.
4) Hundreds of Research Investigations Join Astronauts on SpaceX Rocket
SpaceX launched its 8th crewed mission and 5th commercial flight, aptly named Crew-5. The astronauts on this mission are carrying hundreds of experiments to the International Space Station to test the safety of space travel since space travel is anticipated to become much more common as civilian and private astronaut spaceflight grows. In particular, this mission includes a study from the Los Alamos national lab looking at how the gut microbiome changes in space. Good news for those of us stuck down here on earth. Some studies might benefit us too, such as one from LambdaVision looking at constructing artificial retinas in microgravity to treat degenerative eye diseases. The space station has always played host to tons of scientific research, and SpaceX’s increased access to the ISS will likely speed up progress.
5) Biohaven Officially Launches as New Pfizer Spinout Company
Biohaven officially launched as an independent company under Pfizer following Pfizer’s $11.6 billion acquisition, which finally closed. Biohaven focuses on neurological and neuropsychiatric diseases like migraines and has several anti-migraine drugs in the pipeline as well as already FDA-approved anti-migraine drugs. The company’s most popular anti-migraine drug is Rimegepant (Nurtec), a calcitonin gene-related peptide (CGRP)-targeting migraine medications. I covered Pfizer’s acquisition of Biohaven and its valuable anti-migraine medications here.