Top medical breakthroughs of 2024
Key Takeaways
Industry Insider
“Earlier this year, a breakthrough study—PURPOSE 1 in women in South Africa and Uganda—gave new hope to ending the HIV epidemic.” - Anoop Raman, MD, MBA
“One game-changing breakthrough in 2024 was the emergence of hemolysis detection in whole blood on blood gas instruments with the GEM 7000 hemolysis detection,” - Chris W. Farnsworth, PhD,
This year brought many pivotal moments in healthcare, with groundbreaking medical advances that overshadow the AI buzz in terms of their potential impact. These advances are helping to bring forth, protect, save, and extend lives in ways that are close to miracles, potentially altering the life trajectory of countless patients and reshaping the practice of medicine.
Long-acting injectable medications to end the HIV epidemic
HIV continues to be a global health burden and epidemic despite substantial progress in patient-related outcomes, such as 69% fewer deaths in 2023 compared to the peak in 2004, —according to the World Health Organization (WHO). Nevertheless, in 2023 alone, 76,000 children and 560,000 adults lost their lives due to HIV-related causes.[]
On the bright side, said Anoop Raman, MD, MBA, Chief Medical Officer of Complex Care at AbsoluteCare, to MDLinx, “Earlier this year, a breakthrough study—PURPOSE 1 in women in South Africa and Uganda—gave new hope to ending the HIV epidemic.”[] In the study, “a twice-a-year subcutaneous injection [lenacapavir, developed by Gilead Sciences] demonstrated 100% prevention of contracting HIV,” he said.
There are still over 1.3 million people who have newly contracted HIV worldwide, he adds.[] “This would give us the ability to prescribe twice-yearly injections to general or at-risk populations abroad or in the US that could prevent HIV—with significantly higher adherence and efficacy compared to daily oral PREP treatment.” Further trials are now underway in the US, which Dr. Raman is excited about.
Dr. Raman also mentions observing a move towards long-acting injectables (LAIs) like lenacapavir in the last decade, and even more so in the previous year for serious mental illness, substance use disorder, obesity, diabetes, and HIV care.
He describes this move as life-changing: “One of the joys of being an HIV provider this past year has been transitioning people from a daily oral regimen for their HIV to bimonthly injections of cabotegravir and rilpivirine. I have had patients come back to our office giving hugs and in tears of joy, feeling they are no longer shackled to taking a medication that reminds them of their HIV positivity daily, or fearing someone may find their medication bottles and have their HIV status revealed.”
Dr. Raman believes “more medications will move towards implants or LAIs, drastically improving adherence and patient outcomes. Because ultimately, the medications we prescribe only work when our patients take them.”
The advent of in-line hemolysis detection on a blood gas analyzer
Research suggests that up to 70% of lab result errors come from preanalytical errors, including those from hemolyzed blood samples.[] For this reason, lab professionals and manufacturers have been prioritizing identifying and addressing these preanalytical errors to improve the accuracy and reliability of clinical lab results.
“One game-changing breakthrough in 2024 was the emergence of hemolysis detection in whole blood on blood gas instruments with the GEM 7000 hemolysis detection,” according to Chris W. Farnsworth, PhD, associate professor of pathology and immunology at Washington University School of Medicine. He is also Medical Director of Clinical Chemistry and of Point-of-Care testing at Barnes Jewish Hospital.
“Blood gas syringes are just as likely to be hemolyzed as a specimen in a typical collection tube, but we in the lab often don’t place them under the same scrutiny with regard to the potential analytical impact of hemolysis,” he says. “This poses a huge threat to patient safety, and hospitals like mine have limited where we place point of care testing.”
Dr. Farnsworth believes that the advent of in-line hemolysis detection will revolutionize how blood gas testing is performed. “This breakthrough has allowed us to place blood gas testing at the point of care, particularly in areas with high hemolysis rates like the emergency department,” he says. “Specific to our ED, this has facilitated more rapid treatment in patients that are in diabetic ketoacidosis, requiring potassium measured prior to insulin administration. Another place this has impacted us has been in pre-procedural areas such as the cath lab, facilitating more rapid treatment where we historically relied on in-lab testing,” he concludes.
Dr. Farnsworth hopes this innovation will “drive other vendors towards solutions that can detect hemolysis in whole blood and continue improving patient care.”
FDA approval of two gene therapies, Casgevy and Lyfgenia, for the treatment of sickle cell disease
Approximately 100,000 people are living with sickle cell disease (SCD) in the US. That’s about 0.03% of Americans, says Dr. Raman. “Patients with sickle cell disease (SCD) go to the hospital about 1000% more often than the general population due to severe and debilitating pain crises.”
According to Dr. Raman, one significant medical leap was that the FDA approved two gene therapies, Casgevy and Lyfgenia, for treating SCD in December 2023.[] These medications are the first cell-based gene therapies for patients 12 years and older. “These two new gene therapies do not simply offer a way to decrease pain but to cure the disease for patients,” Dr. Raman notes.
“The Centers for Medicare & Medicaid Services (CMS) is testing a new payment scheme to help ensure that patients with sickle cell disease can access these costly gene therapies,” he says. He is hopeful that all patients with SCD who are candidates for treatment will have access to this cure.
Multiple births from one ovarian stimulation and retrieval cycle
WHO statistics show that roughly 1 in 6 people worldwide have infertility problems.[] These can be a source of psychological distress—including stress, anger, low self-esteem, depression, and anxiety—as well as reduced quality of life and financial issues.[]
“Many families and individuals assume that when they begin an egg retrieval cycle, as part of the in vitro fertilization (IVF) process for treating fertility problems, it will only lead to a single healthy birth, which can be disheartening for families conscious of the time and resources needed for multiple cycles to build their family,” says Dr. Thomas Molinaro, Chief Medical Officer, IVI RMA North America. “These cycles can be costly, and 29 states still do not require private insurance to cover fertility services. Even if a patient does have insurance coverage, their insurer may not cover multiple ovarian stimulation cycles,” he adds.
“An IVI RMA team of researchers from Boston IVF presented a groundbreaking study []that challenges the traditional “one and done” assumption in IVF,” says Dr. Molinaro. “The study demonstrated that it’s possible to achieve two live births, and sometimes more, from just one ovarian stimulation cycle.”
According to Dr. Molinaro, “This research revolutionizes possibilities for the roughly 400,000 cycles performed annually and individuals looking to build their families.”
In the near future, Dr. Molinaro expects to see the most exciting innovations in fertility treatments and reproductive medicine from the increased and thoughtful use of AI. For example, “AI can help analyze images of embryos and select the best embryo using data-driven decision-making.” It can also assist with patient communication and help determine a patient’s best course of treatment, ultimately shortening the time to pregnancy, he says.
Precision treatment of brain cancer through focused ultrasound
A fascinating development in neurosurgery is the precision treatment of brain cancer with focused ultrasounds, says Gary Schwartzbauer, MD, PhD, neurosurgeon at the University of Maryland Medical Center and associate professor at the University of Maryland School of Medicine.
Focused ultrasound (FUS) is a novel technique that allows for improved, safe, and precise drug delivery to the brain by temporarily opening the blood-brain barrier (BBB).[] The BBB is a protective membrane that keeps harmful foreign substances out. However, this barrier also makes it difficult for medicines, like chemotherapy drugs, to reach their target. As a result, these drugs are less effective and may cause worse side effects when they build up in other organs and tissues instead of reaching the brain.[][]
“At the University of Maryland Medical Center, we pioneered the use of focused ultrasound to penetrate the brain’s blood barrier to deliver chemotherapy directly to brain tumors,” says Dr. Schwartzbauer. “We’re now involved in research that uses nanotechnology to also deliver chemotherapy directly to brain tumors. Both of these technologies are poised to transform brain repair for a multitude of diseases.”
Dr. Schwartzbauer also cites the role of AI. “AI is making waves in how we plan and perform surgeries; we’re actually at the forefront of research that studies the use of AI-powered augmented and virtual reality for surgical planning, anatomy education, and even to predict surgical outcomes.”