Clinical Trials 2024: 11 Studies That Have Researchers Excited

2024 promises to be a good year for biomedical research, with results from 11 clinical trials poised to shape the future of medicine. Last year, the prestigious scientific journal Nature did something unique. They asked top researchers which clinical trials they think will significantly impact medicine in 2024.

They published the results in December, revealing 11 trials to watch out for this year. From cancer and depression to patient triage and artificial intelligence, researchers worldwide are investigating a range of diseases, conditions, and technological advancements, with clinical research showing encouraging results.

Today, we're breaking down each one to explain what researchers are studying and why their work may have enormous implications for the future of medicine.

Table of Contents

1. AstraZeneca — Breast cancer
2. University of Oxford — Malaria
3. The Netherlands Cancer Institute — Melanoma
4. Verve Therapeutics — High Cholesterol
5. Vir Biotechnology — HIV
6. Skåne University Hospital — Parkinson's disease
7. NHS Trust Hospitals — Lung cancer
8. Maastricht University Medical Center — Emergency room triage
9. University of Glasgow — Child mental health
10. Erasmus Medical Center — Lung cancer
11. Human Development Research Foundation — Perinatal depression

1. Austrian Researchers Target Breast Cancer Cells With New Drug

• Phase: 3b/4
• Organization: The Medical University of Vienna / AstraZeneca
• Focus: Breast cancer
• Treatment: Trastuzumab deruxtecan (Enhertu)

Already in phase 3b/4, researchers in Vienna published their phase 2 results in the journal Nature. In the study, patients with breast cancer and brain metastases received trastuzumab deruxtecan treatment, a new antibody-drug conjugate (ADC). Think of the ADC as a "smart bomb" that only explodes when it reaches its target, delivering the chemotherapeutic agent directly to cancer cells. So far, results show a 73.3% intracranial response rate to treatment.

Significance

The unique ADC design can cross the blood-brain barrier, a significant obstacle with this type of treatment in the past.

With brain metastases a serious concern in HER2-positive breast cancer patients, the high intracranial activity of Enhertu treatment is extremely promising. It may soon become the preferred option for patients with this type of cancer.

2. UK Scientists Immunize African Children Against Malaria

• Phase: 3
• Organization: The University of Oxford
• Focus: Malaria
• Treatment: R21/Matrix-M

Oxford University researchers are testing a vaccine with a 78% efficacy rate in immunizing young children from malaria, a 23% increase from anything currently available. British researchers conducted this study in several African countries, including Burkina Faso, Kenya, Mali, and Tanzania. Over 4800 children between 5 and 17 months old received the new vaccine against malaria, which showed a 78% efficacy rate maintained for up to one year. The university published the study's phase 3 results in The Lancet this year.

Significance

The R21/Matrix-M uses a unique arrangement of tiny particles targeting a malaria parasite protein. Previous malaria treatments for this age group had an efficacy rate of 55%, making this new vaccine a groundbreaking improvement.

Considering that every year, over 600,000 die from Malaria, many of them African children, the significance of this landmark discovery speaks for itself.

3. New Melanoma Treatment Could Avoid Need for Surgery

• Phase: 3
• Organization: The Netherlands Cancer Institute
• Focus: Melanoma
• Treatments: Ipilimumab + nivolumab

The Netherlands Cancer Institute published phase 2 results of their melanoma study in Nature. The trial included 99 patients with stage III melanoma who had abnormal lesions or growths that had spread to the lymph nodes. Researchers measured the response to neoadjuvant ipilimumab and nivolumab, observing a 72% positive and 61% major reduction in the affected areas.

Significance

The study's future implications are tremendous. With such high rates of efficacy, patients with stage III melanoma may no longer need surgery to remove lesions.

4. Verve Therapeutics Looks to Deactivate High Cholesterol Gene Mutations

• Phase: 1b
• Organization: Verve Therapeutics
• Focus: Heterozygous familial hypercholesterolemia
• Treatments: VERVE-101 / VERVE-102

Heterozygous familial hypercholesterolemia is a genetic condition that causes high levels of 'bad' cholesterol (LDL) in people from a young age through a gene mutation called PCSK9. Last year, Verve Therapeutics presented its preliminary results at the American Heart Association Scientific Sessions. Early indications show how in vivo base-editing therapy can permanently deactivate the specific gene mutation related to the condition, which affects 1 in 300 people.

Significance

While the study is still in phase 1 trials, early results show a significant reduction in the blood PCSK9 protein. If these indications are correct, this nanoparticle-delivered mRNA encoding therapy could offer a one-time treatment to lower LDL cholesterol.

5. Phase 1 Trials for HIV Vaccine Begin in the US and South Africa

• Phase: 1
• Organization: Vir Biotechnology
• Focus: Human immunodeficiency virus (HIV)
• Treatment: VIR-1388

With support from the Bill and Melinda Gates Foundation, this San Francisco-based biotech company has teamed up with the National Institute of Allergy and Infectious Diseases (NIAID) to get phase 1 trials underway in the United States and South Africa. Vir Biotech is investigating the VIT-1388 vaccine for its efficacy in HIV prevention. It uses a unique carrier (cytomegalovirus or CMV vector) to activate the body's T cells, which provide long-lasting protection against HIV.

Significance

With no currently approved vaccines for HIV, the unique CMV vector approach represents hope in the battle against the long-standing global health challenge.

6. Swedish Scientists Attack Parkinson's Disease With New Therapy

• Phase: 1
• Organization: Skåne University Hospital
• Focus: Parkinson's Disease (PD)
• Treatments: STEM-PD

Also currently in phase 1, researchers from the Skåne University Hospital in Sweden are investigating the viability of transplanting a new stem cell therapy into the affected brain areas for patients with Parkinson's. The pioneering therapy, called STEM-PD, inserts dopamine neurons derived from embryonic stem cells into the brain, aiming to repair the damage caused by PD.

Significance

The unique nature of clinical research with STEM-PD may lead to significant advancements in treating PD and offer a more permanent solution than current options. The trial's focus on safety and efficacy could make way for future interventions using stem cell-derived neurons.

7. NHS Investigates Whether AI Can Spot Lung Cancer in X-Rays

• Phase: Randomized trial
• Organization: University College London / Nottingham University Hospitals NHS Trust
• Focus: Lung cancer
• Treatments: qXR

This ongoing randomized trial from the UK's National Health Service uses artificial intelligence to look for early signs of lung cancer in chest X-rays. Using sophisticated AI algorithms that can identify potentially cancerous abnormalities, the research aims to speed up lung cancer diagnosis to improve the chances of early intervention.

Significance

AI could drastically reduce the time it takes for patients to get a CT scan after an X-ray. Besides being more cost-effective and requiring fewer hospital visits, the use of artificial intelligence could be a game-changer in early lung cancer detection, saving lives by reducing the gap between screening, diagnosis, and treatment.

8. Dutch Researchers Focus on Enhancing ER Triage

• Phase: Randomized trial
• Organization: Maastricht University Medical Center
• Focus: Emergency room admissions and triage
• Treatment: RISKINDEX

Among our list of clinical trials 2024, this might be the first of its kind. The study uses RISKINDEX, a machine learning (ML) technology that analyzes patient data in emergency rooms to predict a 31-day mortality rate. The trial's parameters, published in the Scandinavian Journal of Trauma Resuscitation and Emergency Medicine, discuss how a sophisticated AI model could analyze huge amounts of data to assess mortality risk and optimize decision-making in the ER.

Significance

With increasing patient numbers and a high cognitive load on healthcare professionals in emergency rooms, not only does machine learning technology present an opportunity to prevent ER oversights, but it will also pave the way for future research into the applications of ML in clinical settings.

9. Scottish Researchers Focus on Improving Child Mental Health

• Phase: Randomized trial
• Organization: The University of Glasgow
• Focus: Child mental health
• Treatment: New Orleans Intervention Model

This U.K.-based study deploys the New Orleans Intervention Model (NIM) to evaluate its effectiveness in improving the mental health of mistreated children in foster care. The NIM is a family-oriented model focusing on relationship dynamics to improve a child's mental health. The protocol for this large-scale study involving nearly 400 families was published in Trials.

Significance

The study is the first of its kind since previous child services models didn't include mental health professionals in the policy-making and judicial process. With this study, researchers aim to improve how we make decisions about mistreated children in the system.

10. Rotterdam Scientists Use CT Scans for Lung Cancer Detection

• Phase: Randomized trial
• Organization: Erasmus Medical Center
• Focus: Lung cancer
• Treatment: CT Scan

Nearly 16,000 current or ex-smokers participated in this randomized trial to determine if low-dose CT screening over time could reduce the rate of lung cancer mortality. With a 90% adherence rate, researchers discovered a mortality rate ratio of 0.76 in the screening group, representing a significant reduction in lung cancer mortality. The results from the study were published in The New England Journal of Medicine.

Significance

With lung cancer a leading cause of cancer death worldwide, any study showing promise in improving early detection becomes noteworthy as potentially life-saving.

11. Pakistani Investigators Look Into Perinatal Depression

• Phase: Randomized trial
• Organization: Human Development Research Foundation
• Focus: Perinatal depression
• Treatment: THP-TA

This study aims to address the high occurrences of perinatal depression (during pregnancy and up to one year after childbirth) in women from low and middle-income countries. 980 pregnant women with previous depressive episodes participated in the study, which uses technology-assisted cognitive behavioral therapy (CBT) techniques to alleviate depression. A non-profit research trust in Pakistan published the study protocols in Trials last year.

Significance

Because of limited access to traditional means of clinical support in certain populations, studies such as this represent a groundbreaking approach to dealing with mental health. The use of technology-assisted therapy drastically reduces the wait times and treatment gaps for perinatal depression and other mental disorders in rural and low-income communities.

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