1. New weight-loss drugs

Several new weight-loss drugs mimic a hormone called glucagon-like peptide-1 (GLP-1) to target areas of the brain that regulate appetite and food intake. These drugs, which include Novo Nordisk's Ozempic and Wegovy and Eli Lilly's Mounjaro, were initially developed to treat diabetes, but are now also being used to treat obesity.

In clinical trials, these drugs have helped some patients lose more than 15% of their initial weight, leading some experts to call them a "game-changer" for obesity treatments.

"With a worldwide surge in obesity rates, the drugs could enable safe, effective weight reduction without the need for invasive measures and possibly help reduce the adverse outcomes linked with obesity," writes Gerard Doherty, surgeon-in-chief at Brigham and Women's Hospital.

2. RSV vaccines

Respiratory syncytial virus (RSV) generally causes mild or no illness, but it can lead to severe symptoms in vulnerable populations, including very young children and the elderly. After decades of research, RSV vaccines may soon be available.

In May, FDA approved the first-ever vaccine for RSV, Arexy developed by GlaxoSmithKline. The vaccine was approved for use in adults aged 60 and older. Peter Marks, director of FDA's Center for Biologics Evaluation and Research, called the approval "an important public health achievement."

An FDA advisory committee also recommended the agency approve a separate RSV vaccine intended for pregnant women to protect infants from the virus. The vaccine, which was developed by Pfizer, had an 82% efficacy rate at protecting infants in their first three months and a 69% efficacy rate at six months.

3. The next generation of mRNA vaccines

During the pandemic, mRNA technology was used to develop lifesaving COVID-19 vaccines, which have been administered to millions of people worldwide. Now, companies are working to develop a new generation of mRNA vaccines for other diseases.

For example, Moderna is currently testing an mRNA vaccine for RSV. In a trial of around 37,000 people across 22 countries, the company found that its vaccine was 83.7% effective at preventing RSV in people ages 60 and older and 82.4% effective at preventing RSV with three or more symptoms.

In addition, several companies, including Moderna and BioNTech, are working on their own mRNA vaccines to treat certain types of cancer.

4. New ALS therapies

Amyotrophic lateral sclerosis (ALS) is relatively rare neurological disorder that destroys motor neurons in the spinal cord and brain, leading to weakened muscles, paralysis, and death. Currently, there is no cure for ALS, and relatively few treatments are available.

However, ALS patients recently gained new treatment options after FDA approved two new ALS drugs. In September 2022, FDA approved Amylyx Pharmaceuticals' Relyvrio, a pair of drugs that slows ALS progression by protecting nerve cells. In April, the agency also granted accelerated approval to Biogen's experimental gene-based therapy Qalsody.

According to James Berry, a neurologist and ALS clinical researcher at Massachusetts General Hospital, the approvals for these drugs "signal an important step forward" in ALS treatments.

5. LLMs in healthcare

Large language models (LLMs) are a form of artificial intelligence (AI) that are trained on massive datasets to recognize patterns and relationships among words. After being trained, LLMs can be used to recognize, summarize, translate, generate, and predict text.

"LLMs could have a significant impact in medicine by streamlining and supporting the work of physicians," writes Vesela Kovacheva, director of translational and clinical research at Brigham and Women's Hospital. LLMs could be beneficial in clinical decision support and administrative workflows.

6. Breakthroughs in gene-editing therapies

In vivo gene-editing therapies are now in early-stage clinical trials. The therapy uses a genome editing system called CRISPR-Cas9 to make double-stranded cuts in DNA and repair errors in the genetic code.

In a 2021 Phase 1 trial, the therapy helped treat a man with transthyretin amyloidosis (ATTR), a disease in which a misfolded protein forms abnormal clumps and damages organs and tissues. The treatment was just as effective as standard care for ATTR but much less intensive.

According to Roger Hajjar, director of Mass General Brigham's Gene & Cell Therapy Institute, if the therapy is proven to be safe and effective, it could "signify a landmark in the history of biopharmaceuticals."

7. A non-hormonal menopause treatment

As women transition to menopause, they may experience a wide range of symptoms, including hot flashes, night sweats, sleeplessness, mood changes, and more. Although hormone therapy is considered an effective treatment, not all women can or want to take it.

In May, FDA approved the first nonhormonal drug to treat hot flashes in menopausal women. The drug, which is made by Astellas and will be sold under the brand name Veozah, provides women with a "safe and effective treatment option," according to Janet Maynard, director of FDA's Office of Rare Diseases, Pediatrics, Urologic, and Reproductive Medicine.

8. A new era of precision oncology

Bifunctional cancer therapies consist of two parts. One identifies tumor cells while the second kills the cancer cells while limiting damage to healthy cells and tissues.

One approach to this therapy involves using attaching tumor-homing molecules to radionuclides to destroy tumor cells. Another approach uses antibodies combined with chemotherapy drugs.

"Together, these therapies are improving outcomes for cancer patients and defining a new era of precision oncology," writes David Ting, associate clinical director for innovation at Mass General Cancer Center.

9. AI tools to detect lung cancer risk

Recently, researchers developed an AI-based tool that can predict whether a patient will develop lung cancer within six years. The tool uses images from low-dose CT scans to make its predictions about patients' lung cancer risks.

According to Florian Fintelmann, a radiologist and physician-scientist at Massachusetts General Hospital, the tool aims to close gaps in lung cancer screenings for both smokers and non-smokers, particularly as rates of lung cancer among non-smokers rise.

10. New vision therapies to protect retinal cells

Several different conditions can cause neurons in the retina to degenerate and die, which leads to impaired vision and eventually blindness. To help patients preserve their vision, researchers are developing different neuroprotective therapies to protect retinal cells from injury and death.

One potential therapy is an inhibitor against the fast receptor, which has been implicated in several retinal diseases including macular degeneration, glaucoma, and optic neuropathies. Phase 1 trials of the therapy have shown promise, and other trials are underway.

11. Diagnostic blood tests for Alzheimer's disease

Currently, several companies are working to develop simple and cost-effective blood tests that can diagnose Alzheimer's and other neurogenerative diseases years before symptoms develop. So far, three tests by C2N Diagnostics, Quest Diagnostics, and Quanterix are available on the market.

These blood tests detect tiny amounts of abnormal protein in the brain to determine whether a patient has Alzheimer's. In addition, early data suggests that these tests have an accuracy similar to spinal taps or specialized PET scans.

Overall, these tests "could rapidly accelerate the development of effective treatments for these diseases" and lower "the future public health burden of Alzheimer's disease, which is projected to skyrocket by 2050," writes Steven Arnold, director of the Alzheimer's Clinical & Translational Research Unit at Massachusetts General Hospital.

12. Treatments to slow the progression of type 1 diabetes

Type 1 diabetes is a chronic illness that occurs when the immune system attacks insulin-producing cells in the pancreas, leading to a life-long dependence on exogenous insulin. However, while insulin replacement is an effective treatment, it's not a cure for the disease.

Last November, FDA approved the first treatment designed to delay the onset of insulin-dependent Type 1 diabetes. The treatment, which was developed by ProventionBio and Sanofi, is a monoclonal antibody that delays the onset of type 1 diabetes in children and young adults who are at risk of the disease.

"The drug's potential to delay clinical diagnosis of type 1 diabetes may provide patients with months to years without the burdens of disease," said John Sharretts, director of the division of diabetes, lipid disorders, and obesity in FDA's Center for Drug Evaluation and Research. (Mass General Brigham press release, 6/14; World Medical Innovation Forum release, accessed 6/15)

This watchlist covers 7 disruptive therapeutics and diagnostics. All included clinical innovations require clinicians, life science leaders, and payers to utilize appropriate and consistent frameworks for thinking about the evidence and data needs related to adoption and implementation. Download now  to find out what healthcare leaders need to know about the evolving therapeutics landscape.