w-insights

Game changers - 11 Clinical trials that will shape medicine in 2024

Author: Pascal Hänggi, PhD

This edition of W-INSIGHTS is going to be a little more nerdy than usual. However, we share our views on this year's life sciences trends and how we can help you meet the challenges.

Clinical trials in 2024 to watch

With so many rollercoaster years since the start of the pandemic, it is impossible to predict exactly what the biomedical world will deliver this year. So, Nature Medicine, the world's leading multidisciplinary journal of biomedical research, asked top-notch researchers to name their top clinical trials (Table 1) for 2024 that are likely to have an outsized impact on medicine.

The range of innovations and new approaches is vast. From base editing, HIV vaccines and patient triage to artificial intelligence tools for diagnosing lung cancer. The common denominator is that these innovations present many new challenges for everyone working in healthcare. From digitalization, regulatory affairs, sales & marketing to physicians using the new approaches, these are game changers for all healthcare professionals. At Wirz & Partners, we stay one step ahead of the hurdles to be overcome and are committed to helping you and your team move forward.

 

Clinical trials to watch in 2024

Treatment Lead indication Organization Description Phase
Trastuzumab deruxtecan Breast cancer AstraZeneca Efficacy and safety of ADC in HER2-positive patients with
brain metastases
3b/4
R21/Matrix-M Malaria University of Oxford  Vaccine efficacy in children 5–36 months of age in four countries 3
Ipilimumab and nivolumab Melanoma The Netherlands Cancer Institute Neoadjuvant immunotherapy plus lymph-node dissection 3
VERVE-101 Heterozygous familial Verve Therapeutics In vivo base editing of PCSK9 1b
hyper-cholesterolemia
VIR-1388 HIV Vir Biotechnology Safety and immunogenicity of a CMV-based vaccine in adults 1
STEM-PD Parkinson’s disease Skåne University Hospital, Sweden Intraputamenal transplantation of human embryonic stem cell
derived dopaminergic cells
1
qXR Lung cancer Nottingham University Hospitals NHS Trust Class IIa CE-certified deep learning algorithm to analyze
chest X ray & CT
Randomized trial
RISKINDEX Emergency room admissions Maastricht University Medical Center Implementation of machine learning algorithm
in the emergency room
Randomized trial
New Orleans Intervention Model Child mental health University of Glasgow Effectiveness and cost-effectiveness of a service for
children 0–5 years of age in foster care
Randomized trial
CT scan Lung cancer Erasmus Medical Center Annual versus biennial CT scan screening of 26’000 people Randomized trial
THP-TA Perinatal depression Human Development Research Foundation Technology-assisted version of Thinking Healthy Program
delivered by peers
Randomized trial

Are we rewriting how we are? New therapeutics from CRISPR/Cas9 to ADC in the brain

We are only beginning to grasp the full implications of this extraordinary new breakthrough technology. The CRISPR-Cas9 complex is opening up completely new possibilities in cell therapy, immunotherapy and new areas of agriculture. It has already been used to save the life of a child with an incurable form of leukemia, and there is much, much more to come. There will be even more challenges for all healthcare professionals.

 

 

 

Base editing for hypercholesterolemia (VERVE-101)

  • Problem

Around 1 in every 300 people is born with heterozygous familial hypercholesterolemia , which makes it one of the most common inherited genetic conditions. It is characterized by extremely high LDL-C levels in the blood that, over time, cause plaque to build up in the arteries, resulting in reduced blood flow or blockage. This significantly increases an individual’s risk for heart attack and stroke.

  • Solution

Base editors can most simply be compared to pencils, in their ability to "erase" and rewrite a specific letter in a gene. Verve-101 is a gene editor to develop as single-course medicines to treat this cardiovascular disease. The muted PCSK9 gene should be permanently turn off with a modified Cas9 protein (CRISPR/Cas9 system) and reduce disease-driving LDL-C.

 

 

 

Stem cells for Parkinson’s disease (STEM-PD)

  • Problem

Most clinical trials for Parkinson's disease look at people with advanced disease. As a result, patients in much earlier stages of the disease, who may be more amenable to treatment, are often excluded. In the STEM-PD trial, dopaminergic neurons derived from human embryonic stem cells will be transplanted into the brains of patients with moderate Parkinson's disease.

  • Solution

It is the first time human embryonic stem cell therapy has been tested in Parkinson's disease, and people with early disease stage are targeted who are most likely to respond.

 

 

 

ADC for brain metastasis (Trastuzumab deruxtecan)

  • Problem

Brain metastases are a major problem in advanced breast cancer, affecting about half of HER2-positive patients, but there is only one approved treatment. The DESTINY-Breast study12 is evaluating the efficacy and safety of trastuzumab deruxtecan (Enhertu), an antibody-drug conjugate (ADC) that targets HER2.

  • Solution

A growing body of data suggests that some ADCs, despite their large size, may have intercranial activity and target brain metastases.

 

 

 

Immunotherapy for melanoma (Ipilimumab and nivolumab)

  • Problem

Stage 3 melanoma means that the cancer has spread from the skin to the lymph nodes and the current standard is adjuvant anti-PD-1 therapy, which improves recurrence-free survival, but a considerable proportion of patients have a relapse in the years after therapeutic lymph-node dissection, and no overall survival benefit has been found so far. Patients participating in this trial will receive neoadjuvant ipilimumab plus nivolumab for the stage 3 melanoma.

  • Solution

The hope is to see an overall survival benefit that could change the way stage III melanoma is treated.

 

Hurdles on the way to the solution and how to overcome them

These important breakthroughs are an ideal example of what scientists can achieve when they work together. At Wirz & Partner, we have shown that we understand what these extraordinary new breakthrough therapies mean for the life sciences sector. Regulatory affairs (ADC), quality assurance (e.g. stem cell therapy), supply chain management, sales and marketing (CRISPR/Cas9) are more important than ever, but many organizations lack the skills and new requirements for the new technologies.

Wirz & Partners Life Sciences Sector leverages our unique global reach and proven expertise to identify, recruit and manage the ideal candidates and leaders to keep your organization ahead of the curve and overcome any bottlenecks. We have the ability to attract and retain the talent required for your specific needs.

 

 

AI can do more than wiring cover letters – Digitalization, big data, and AI in life sciences

An AI revolution is brewing in medicine. What will it look like? Emerging generalist models could overcome some of the limitations of first-generation machine learning tools for clinical use.

 

 

 

CT screening for lung cancer (CT scan)

  • Problem

Lung cancer is one of the most common and serious forms of cancer. More than 4’000 people in Switzerland are diagnosed with this deadly disease each year. Two large randomized controlled trials have shown that lung cancer screening with CT can reduce mortality. However, implementation of lung cancer screening is likely to be limited, slow and of variable quality, partly because initial CT scans show no signs of cancer in nine out of ten people.

  • Solution

If the implementation of lung cancer screening can be optimized, it is expected that many people will quickly benefit from this high-quality screening technology and healthcare costs will be reduced.

 

 

 

App therapy for perinatal depression (THP-TA)

  • Problem

Many parts of the world face challenges in providing adequate and effective mental health care, but the lack of trained professionals is particularly acute in low- and middle-income countries. An app has been developed that enables a peer (a woman from the same community with no previous health care experience) to deliver a cognitive therapy-based intervention to women in their second or third trimester of pregnancy who are experiencing major depression.

  • Solution

The trial will compare the app with the standard face-to-face version of the therapy delivered by community health workers. It is hoped that the results of this trial will stimulate further innovation in this important area for a common mental disorder worldwide.

 

 

 

AI for early lung cancer diagnosis (qXR)

  • Problem

Early detection of lung cancer saves lives. Although nearly three quarters of lung cancers are diagnosed late, earlier diagnosis at any stage allows for better and more effective treatment. Artificial intelligence (AI) should be applied to chest x-rays as soon as they are taken to reduce the time to CT and the time to diagnosis from 63 days to 32 days.

  • Solution

The hypothesis that AI can detect lung cancer earlier and reduce the time to diagnosis by as much as 50% will lead to an immediate change in the standard of care to include AI.

 

 

 

Machine learning for patient triage (RISKINDEX)

  • Problem

Identifying which patients in the emergency department (ED) are at high or low risk shortly after admission could help guide patient care decisions. Several clinical risk scores and triage systems have been developed to stratify patients, but they often underperform in clinical practice. This study will use an AI model to predict 31-day mortality for patients presenting to the ED.

  • Solution

If the AI model is validated, its impact could include identifying patients who will deteriorate despite clinical judgement.

 

Hurdles on the way to the solution and how to overcome them

Artificial Intelligence (AI) has the potential to transform healthcare and disrupt the medical field in significant ways. It has shown remarkable progress in tasks such as diagnosis, data analysis and precision medicine, and is already being used in areas ranging from patient triage to cancer detection. However, it is difficult to predict how doctors will react to being second-guessed by an AI model.

The demand for top talent who understand the importance of AI & Big Data is a strong motivation for Wirz & Partners to help your organization through the transformation and implementation process. As such, we were one of the first national headhunting firms to establish an AI & Big Data & Analytics practice within retained and executive search. Recognizing this as one of the key strategic concerns for large companies and organizations, we have taken steps to recruit and analyze key metrics in data, distribution and visualization, and use our experts to manage these systems and resources. And at Wirz & Partners, we not only recruit AI, BI and Big Data seniors, our own processes are fully digitized and we benefit from the extensive use of AI and BI. We use and leverage AI and semantic search algorithms, among other cutting-edge technologies, in our executive search process.

 

 

The lost world - New approaches for old problems and neglected diseases

The Covid-19 pandemic continues to challenge global supply chains with shortages, delays and disruptions; manufacturers and technology companies around the world are struggling to get the parts and supplies they need. It has also shown what can be achieved in a short period of time (Covid-19 vaccine) so that neglected and 'forgotten' diseases can be targeted with new technologies.

 

 

 

T cell vaccine for HIV (VIR-1388)

  • Problem

1.3 million people acquired HIV in 2022. 630’000 people and 84’000 children died from HIV-related causes globally. The study is designed to evaluate the safety of a vaccine (VIR-1388) to prevent HIV infection. HIV-negative adults in good general health will be enrolled. VIR-1388 is a cytomegalovirus vector vaccine that induces potent, unique and durable T-cell responses that can potentially prevent HIV acquisition.

  • Solution

The study will determine if the vaccine is safe and sufficiently immunogenic for a HIV vaccine.

 

 

 

Long-term efficacy of R21 malaria vaccine (R21/Matrix-M)

  • Problem

A major problem with malaria vaccines, and one reason it has taken more than 100 years to develop a vaccine, is that an exceptionally high antibody response is needed for the vaccine to work. Forty vaccines with the same circumsporozoite protein antigen have entered the clinic, and only two have shown any meaningful efficacy (RTS,S and R21).

  • Solution

They believe R21/Matrix-M will show better efficacy than RTS,S, especially later in the trial, because R21 uses a nanoparticle with a much higher density of antigen on the surface. This is why vaccines such as the human papillomavirus vaccine last a lifetime.

 

 

 

Intervention model for infant mental health (New Orleans Intervention Model)

  • Problem

There are currently nearly 100’000 children in care in the UK, many of whom have behavioral problems and face an uncertain future. Abused and neglected children in particular are the most vulnerable groups in any society. They are at much higher risk of suicide, mental and physical health problems. Better approaches (intensive assessment and treatment) are needed to help these children, such as the New Orleans Intervention Model.

  • Solution

This first-ever randomized controlled trial (BeST?) will assess the effectiveness and cost-effectiveness of the New Orleans Infant Mental Health Intervention Model compared to usual social work services for children aged 0-5 in foster care. The model could radically change the way such children are supported worldwide.

 

Hurdles on the way to the solution and how to overcome them

Neglected diseases are so called because they are virtually absent from the global health agenda. Even today, they have very limited resources and are almost ignored by global funding agencies. However, the Covid-19 pandemic has shown what can be achieved in a very short time when people work as a team. In neglected diseases in particular, sales and marketing will be key, as not every treatment will be a blockbuster, given the limited financial resources of the majority of patients.

Our Sales & Marketing Executive Search practice is unique. It is based on our expertise and in-depth understanding of what it takes to be a successful sales and marketing executive. For our clients, we identify the best in class professionals to fill the role of Sales Officer and Chief Marketing Officer. As experienced headhunters in this field, we place great emphasis on the excellent track record of all potential Chief Marketing and Sales Officers we present to our clients.

 

https://www.nature.com/articles/s41591-023-02699-5#citeas

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