How Medical Research is Advancing Day-by-Day
Though drug development has been a topic of public conversation since the early days of COVID-19 vaccine research, it seems like a majority of publicized discussions have shifted from potential pandemic-pausers to weight-loss wonder drugs.
While this year’s discoveries may not be as flashy as a one-prescription-fits-all for shedding our winter weight gain, incredible leaps were made within the leagues of vaccinology, gene-editing, women’s health, Alzheimer’s Disease, cancer detection and many others.
So, as we enter this new era of budding opportunity, let’s take a moment to review some of the largest clinical research victories we may have skipped celebrating in 2023.
1. Sickle Cell Disease Treatment Using CRISPR Gene-Editing Technologies
Breakthrough: On December 8th, 2023, the FDA approved two separate gene therapies to treat patients suffering with sickle cell disease
Sickle cell disease affects nearly every organ in the body and is known to cause life-threatening events such as joint replacement, strokes and even heart attacks. Moreover, the victims of these lethal circumstances are often younger than 40 years old making this condition extremely challenging to navigate.
The two FDA approved technologies both utilize gene-editing to deliver essential blood components:
Lyfgenia is produced by Bluebird Bio and uses a piece of the sickle cell virus to deliver a gene for fetal hemoglobin production, which prevents blood cells from sticking together and constricting blood flow.
Casgevy is developed by Vertex Pharmaceuticals and employs CRISPR/Cas9 to genetically edit stem cells, causing them to produce more fetal hemoglobin.
Though it is important to note that these medications are not classified as cures for sickle cell disease, Medical Director of the Comprehensive Sickle Cell Disease Treatment Center of Massachusetts General Hospital Dr. Sharl Azar believes these treatments could lead to a longer life,
“The important thing to emphasize here is that these are not a cure for sickle cell disease. The patients will still have sickle cell disease on the other side of the intervention. The hope is that they will just have a milder form of the disease, where they can see a lot less pain and hopefully fewer complications and hopefully even a longer life."
2. First Drug Approved by FDA to Treat Alzheimer’s Disease
Breakthrough: On July 6th, 2023, the FDA granted traditional approval to Leqembi, the first drug shown to slow cognitive decline in early Alzheimer’s Disease patients
Alzheimer’s Disease currently affects over 6 million Americans and the number continues to grow.
Leqembi was given accelerated approval on January 6th, 2023, due to its successful early-trial results, and then was granted traditional approval on July 6th as the first medication to successfully target and mark amyloid beta plaques. Pinpointing and removing these plaques that build up during the early stages of Alzheimer’s disease is what is now known to slow the progression of the condition.
This discovery has given hope to millions of people across the globe who battle with this disease, and while it is not yet accessible to all, researchers believe this is just the beginning of Alzheimer’s research success.
3. mRNA Personalized Vaccines Being Used to Treat Pancreatic Cancer
Breakthrough: A study done by BioNTech revealed that the use of personalized mRNA vaccines alongside traditional cancer treatments stimulated T cells, which destroy cancer cells
Within this gene sequencing study, 16 patients were given a personalized mRNA vaccine that was split into 9 separate injection dates. Almost half of the study subjects experienced an immune response of triggered T cells.
During observational phases, it was found that the patients who had strong immune responses to their individual vaccine along with traditional treatment did not experience a return of cancer within a year and a half. Unfortunately, the trial patients who did not experience a strong immune response encountered a return of cancer within the year.
Though this treatment is still within its research and development phases, surgical oncologist Dr. Vinod Balachandran is exceptionally optimistic about what these vaccines mean for cancer treatment as a whole,
“It’s exciting to see that a personalized vaccine could enlist the immune system to fight pancreatic cancer - which urgently needs better treatments. It’s also motivating as we may be able to use such personalized vaccines to treat other deadly cancers.”
4. AI Technology Being Enlisted to Detect Cancer
Breakthrough: An AI technology called ‘Mia’ developed by the Imperial College London and Kheiron Medical Technologies detected 13% more early cancer cases during routine screenings typically done by humans
At this point, we have all heard about the ever-growing list of capabilities of artificial intelligence and machine learning. Whether you stand behind the AI movement or not, there is certainly something to be said about the so-called “Safety-net” these technologies can provide within the medical world.
Throughout two pilot phases, Mia detected 24 cancer cases and recalled 70 additional women to be tested for other common cancers.
According to Imperial College London Machine Learning Professor, Dr. Ben Glocker, Mia has not only already exceeded international expectations, it will continue to provide uncharted benefits within cancer detection,
“Seeing firsthand that the use of AI could substantially reduce the rate of missed cancers in breast screening is massive, and a major boost for our mission to transform cancer care with AI technology.”
Altogether, while this technology is barely out of its pilot phase, its contributions toward early stage cancer detection landed it on the list of some of the biggest medical breakthroughs of 2023.
5. Gene Therapy Approved for Children with Duchenne Muscle Dystrophy
Breakthrough: In June, 2023, the FDA approved the first ever gene therapy for children ages 4-5 battling with Duchenne Muscle Dystrophy
Called Elevidys, this therapy is designed to deliver a gene that leads to the production of dystrophin, a protein that is present throughout typically developing muscles.
Much like Leqembi, this drug was granted accelerated approval for some patients and is being carefully observed before traditional approval. While it is not yet on the wide-scale market, experts in both research and muscle dystrophy are optimistic about the clinical benefits of Elevidys.
6. New RSV Vaccines Have Been Released to Target High-Risk Groups
Breakthrough: The FDA recently approved two RSV vaccines for patients over 60 years old, a vaccine for pregnant mothers and two monoclonal shots for babies younger than 8 months
The FDA released two vaccines approved for people aged 60 years or older by the names of Arexy and Abrysvo. Both of these vaccines are recombinant protein vaccines, meaning that they cause the body to produce RSV antibodies, and the only marketable difference between the two is that Arexy contains an immune-enhancing adjuvant while Abrysvo does not. The CDC does not recommend one vaccine over the other at this time.
Also released this year was an FDA approved maternal RSV vaccine that pregnant women can get during their third trimester, in between weeks 32 and 36 of pregnancy. This vaccine aims to pass antibodies from mother to child within the womb and provides protection from RSV throughout the first 6 months of life.
Lastly, the FDA approved two monoclonal antibody shots for babies under 8 months old. While these shots are not the same as a vaccine, they are laboratory-made proteins designed to mirror the body’s pathogen-fighting abilities.
Though a lot of our attention has turned to other viruses such as COVID-19 and the flu, Chief Innovation Officer of Boston Children’s Hospital Dr. john Brownstein hopes these renewed vaccines will cast some spotlight on the dangers of RSV,
“It still has significant public health consequences for those that are most vulnerable, and so now having a high efficacy vaccine is really a tremendous breakthrough for the last year and I think will have significant public health benefit for years to come.”
7. Over-The-Counter Birth Control
Breakthrough: In July of 2023, the FDA approved the first ever over-the-counter contraceptive, called Opill, and slated it to go on shelves early 2024
Throughout the progression of women’s health, the history of gaining accessibility to contraceptives has been a storied one. From the very first marketed birth control, known as Enovid, to contraceptive options now, there have always been challenges in accessibility.
Now, with the semi-recent approval of the progestin-only Opill, contraceptive convenience has never been closer for women. While it is not yet available, the brand has announced it will be coming to retailers nationwide in the near future.
8. Identifying the Cause of Morning Sickness
Breakthrough: A team of researchers from the United States, United Kingdom and Sri Lanka discovered a link between sensitivity to GDF15 hormone and morning sickness severity
Researchers have finally discovered the reasoning behind symptoms of nausea and vomiting experienced by 80% of pregnant women worldwide: A sensitivity to a hormone called GDF15, which is produced by a fetus within the placenta during gestation.
Recent studies show that women who presented severe symptoms of morning sickness were the most sensitive to the GDF15 hormone, and researchers have begun working with this information to create long-term solutions.
Ultimately, the medical breakthroughs of 2023 have dubbed this past year as one of massive progression among clinical treatments and technologies. While our industry will always have room for improvement, it is our pledge as modern researchers to strive tirelessly to lessen the gap between known and unknown.
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