Issue 26: In Utero, FDA, CMS, E.COLI, & May AI

Treating Genetic Disorders in Utero 

epic picture of a healthy baby

What a time to be alive! A couple in Germany was devastated when they found out their 2 year-old son had been diagnosed with hypohidrotic ectodermal dysplasia—an X-linked genetic disorder that prevents the ability to sweat and grow teeth normally. So when they were expecting twins, they consulted Dr. Holm Schneider, who had treated the same disorder in animals. By simply injecting the missing protein (ectodysplasin A) into the amniotic fluid, the offspring of the animals were born without the disorder. However, this had never been tried in humans, making the German couple the first to receive this treatment in utero. Miraculously, the twins have not exhibited any signs of the disease after birth. They are able to run and be active without becoming dehydrated whatsoever. Of course, doctors are unsure how long the effects of this treatment will last, but so far it has been working without any problems. This is groundbreaking news, as it may open up avenues for treating other genetic disorders in utero.

FDA Approves First Nonopioid treatment for Opioid Withdrawal

Okay, if you haven’t heard of the opioid crisis, you are living in underneath a (crack) rock. A new drug called Lucemyra (lofexidine hydrochloride) has shown to improve withdrawal symptoms when opioids are stopped abruptly. It just got approved by the FDA. The drug has been used in the UK for twenty years, so we are honestly super late to the therapy party. America is Great, but there is so much we can learn from other countries. Stay woke.

FDA approves first ever migraine treatment 

man with a migraine

For the first time EVER the FDA recently approved a drug to prevent migraines. The drug, Aimovig, is made by pharma giants Novartis and Amgen. As you can probably guess the drug is hella expensive costing upwards of $6,900/year. These pharma companies are gonna make bank given that there are 2.8 million Americans who suffer from debilitating headaches multiple times a year. The medication does not work in all patient groups but the good news is that it does not have any noted side effects in the short term. We will have to wait to see if there are any longer term side effects after patients take the drug over multiple years. Aimovig is an antibody which binds the protein CGRP which has been known to play a role in migraines since the 1980s. It's given via an injection every month. The previous treatments for migraines were all garbage so hopefully this one is as promising as it sounds.

CMS Thinking of Paying for New Gene Therapies for Cancer

Names like CAR-T and chimeric antigen receptor-T therapies have flooded the news these past couple of years. The promising and novel idea of conveniently stitching someone’s problematic cancer genes was panacea for some but concern for others. The FDA recently gave the green light to two  such therapies called Yescarta and Kymriah, respectively. It is now up to insurance companies to cover their payments. Recently, Center for Medicaid and Medicare Services (CMS) has been reportedly thinking about approving one treatment and is open to comments until June. This will potentially open the floodgates to hospitals throughout the US and give cancer patients a shot (pun intended) at treating their cancer. And of course $$$ for hospitals since these treatments may bill for close to $1.5 million in total length of stay.

E. Coli Under Control

let us eat lettuce 

Salad lovers rejoice! The recent E. Coli outbreak linked to Romaine lettuce is finally over. The outbreak, which affected 172 people over 32 states, is no longer a threat according to the CDC. The virulent strain found in the romaine lettuce we were all afraid to eat for the last few weeks was traced to Yuma, Arizona. According to the CDC, the last reported illness was May 2nd, but officials believe we are in the clear. In terms of the specifics of the outbreak, federal investigators are still searching for answers, but as long as we can go back to our normal lives, its alright. Just in case, officials say to check your egg cartons for any remnants of the recent salmonella outbreak in eggs, which affected 35 people across 9 states. According to the FDA, the recalled eggs have plant numbers of P-1065 and P-1359D, so if you still have any eggs with those numbers, toss them out!

Theresa May wants more AI in medicine

British Prime Minister Theresa May recently announced the NHS would be bank-rolling AI research in medicine to detect cancer and chronic diseases earlier. She has pledged millions of pounds in government funding. Btw 1 pound is $1.34 dollars for all the American homies reading this. It's estimated that by using AI for early cancer detection 22,000 lives could be saved per year starting in the mid 2030's.