From cancer to Alzheimer's: 9 promising medical developments and clinical trials to watch

An obesity drug has reached Phase III trials in children under 12 for the first time, Alzheimer's disease is being targeted even before memory problems appear, and new cancer drugs are increasingly selected not just by the organ where the tumor originated, but by its specific biological characteristics.

The end of August and beginning of September 2026 brought several important results and launches of large clinical trials—from cancer and cardiovascular diseases to COPD, kidney disease, liver disease, and osteoarthritis.

However, the word "breakthrough" should be used with caution here. In some cases, it is about positive Phase III results or regulatory approval already obtained. In others, it is only about the start of a study that still needs to show whether a promising idea works in patients.

1. Cancer: treatment is increasingly tied to the biology of a specific tumor

One of the most recent results emerged on September 8 in the treatment of small cell lung cancer—an especially aggressive form of the disease that often responds well to initial therapy but then quickly returns.

In the Phase III DeLLphi-305 study, the drug tarlatamab in combination with durvalumab improved overall survival compared to durvalumab alone.

The therapy was not used instead of standard initial treatment. Patients first received chemotherapy and immunotherapy, and the new combination was then used as maintenance treatment.

Tarlatamab is a bispecific drug: it simultaneously binds to the DLL3 target on the tumor cell and CD3 on T lymphocytes, helping the immune system get closer to the cancer cell and attack it.

The company has so far only reported the main Phase III results. Detailed data on exactly how much patient life expectancy has increased are yet to be seen.

In parallel, treatment of pancreatic cancer is changing.

In August, the US regulator FDA approved daraxonrasib for some adult patients with metastatic pancreatic adenocarcinoma after prior systemic treatment or in cases where multi-agent therapy is unsuitable.

Now researchers are going even further: in a new Phase III trial, a combination of targeted oral tablets will be directly compared with standard chemotherapy in previously untreated patients with tumors carrying the KRAS G12D mutation.

This reflects one of the key trends in modern oncology: the question is increasingly not just "what kind of cancer is this" but "what is the specific molecular vulnerability of this tumor".

2. Cardiovascular diseases: intensive treatment considered before the first heart attack

Usually, the most aggressive measures to prevent cardiovascular complications are associated with patients who have already had a heart attack or stroke.

The VESALIUS-CV study tests a different approach.

It included more than 12,000 people with high cardiovascular risk who had not yet experienced a heart attack or stroke.

Researchers added evolocumab, a PCSK9 inhibitor class drug, to standard cholesterol-lowering therapy.

According to an analysis presented at the end of August, evolocumab use was associated with an approximately 20 percent reduction in the risk of death in this group.

Follow-up lasted an average of 4.6 years, and the difference between groups began to become noticeable after about one and a half years.

This does not mean that the injectable drug is needed for every person with elevated cholesterol.

Study participants were in a significantly elevated risk group—for example, they had significant atherosclerosis or diabetes with additional risk factors.

The practical question posed by the study is different: should we wait for the first heart attack or stroke before maximally intensifying prevention in the most vulnerable patients.

3. COPD: biologic therapy may suit a broader range of patients

Chronic obstructive pulmonary disease remains one of the leading causes of death worldwide.

Even with modern inhaled therapy, some patients continue to experience exacerbations requiring antibiotics, steroids, or hospitalization.

In two Phase III trials—OBERON and TITANIA—the antibody tozorakimab reduced the frequency of moderate and severe exacerbations by approximately 30% compared to placebo.

The drug blocks the interleukin-33 signaling pathway—one of the mechanisms associated with airway inflammation.

Of particular interest is that the effect was observed not only in patients with high blood eosinophil levels.

This potentially distinguishes the drug from some existing biologic treatments for which a specific inflammatory profile plays a key role.

The FDA has already accepted the tozorakimab application for priority review.

However, the drug was studied as an addition to standard inhaled therapy, not as a replacement.

4. Semaglutide is being seriously studied in children as young as 6

One of the most debated results in September was a study of GLP-1 class drugs in young children.

In the Phase III STEP Young trial, participants were children aged from 6 to under 12 years with obesity.

One group received semaglutide once a week, and the other received placebo. Both the drug and control groups participated in a lifestyle intervention program: lower-calorie diet and increased physical activity.

After 68 weeks, 40.4% of children in the semaglutide group no longer met the obesity criterion used in the study. There were no such children in the placebo group.

The study met its primary endpoint—reduction in body mass index was significantly greater with semaglutide.

But the result simultaneously raises questions that cannot be resolved in just 68 weeks.

If therapy begins at age six, seven, or eight, doctors need to understand its impact on growth, nutrition, puberty, psychological state, and body weight after discontinuation.

Therefore, a positive Phase III result does not yet mean semaglutide becomes standard therapy for all children with obesity.

It shows something else: pharmaceutical treatment of obesity is now being investigated in an age group for which evidence was previously much scarcer.

5. Alzheimer's disease: treatment attempted before symptoms appear

Current Alzheimer's therapy is gradually shifting to earlier disease stages.

An international Phase III study called PrevenTRON is the next step.

It plans to enroll about 1,600 people aged 55 to 80 who have no memory or thinking problems but whose tests show signs of Alzheimer's pathology and a high risk of developing symptoms.

The experimental drug is trontinemab, an anti-amyloid antibody designed to improve penetration across the blood-brain barrier.

In September, the first center in the UK and Europe began offering participation in the program.

The study design is based on the fact that pathological changes in the brain can develop many years before a person notices memory problems.

If treatment at this stage can delay cognitive decline, the approach to the disease could change dramatically—from treating already-present symptoms to preventing their onset.

But "if" is the crucial word here.

It has not yet been proven that trontinemab can prevent dementia in symptom-free people. The answer to this question must come from the Phase III trial.

6. Kidney drug shows effect even in people without diabetes

Finerenone is already used for chronic kidney disease associated with type 2 diabetes.

The Phase III FIND-CKD trial tested whether this mechanism might also benefit patients without diabetes.

The study included 1,584 people with chronic kidney disease.

Over 32 months, kidney function declined more slowly in patients receiving finerenone than in the placebo group.

In addition, the drug reduced the risk of a combined kidney-cardiovascular outcome by 23%.

This is an important result, because chronic kidney disease is not always related to diabetes. One common cause of kidney damage is long-term high blood pressure.

However, the potential expanded benefit also means the need to closely monitor safety.

Elevated blood potassium levels were recorded in 17% of patients on finerenone versus 13.3% in the placebo group, although serious complications and discontinuation for this reason were much rarer.

7. In fatty liver disease, researchers move beyond biopsy to more important questions

Metabolic-associated steatohepatitis (MASH) can gradually lead to fibrosis, cirrhosis, liver failure, and liver cancer.

In recent years, drugs have emerged that can improve liver tissue on biopsy.

But now the bar for therapy is rising.

The global Phase III program PERFORMA is studying pemvidutide, an experimental drug that acts on both glucagon and GLP-1 receptors.

The trial plans to enroll about 1,800 patients with MASH and fibrosis stage F2 or F3, but without cirrhosis.

In the first stage, researchers will assess whether MASH resolves and fibrosis improves.

However, the program is designed to go far beyond one year.

Participants will be followed for about five years to determine whether the drug truly reduces the likelihood of cirrhosis, severe liver complications, transplant, or death.

This is an important shift for the whole field.

Improvement of a lab value or even biopsy is valuable, but what matters more to a patient is whether therapy reduces the risk of severe disease over a few years.

8. Gene therapy for knee osteoarthritis tested as a one-time injection

Osteoarthritis is rarely called a fatal disease, but it is one of the largest causes of chronic pain and limited mobility.

Two-year results from early investigation of experimental gene therapy PCRX-201 have drawn particular interest.

The drug is injected directly into the knee joint and uses a modified adenoviral vector to deliver a gene associated with the anti-inflammatory protein IL-1Ra.

The idea is to make tissues inside the joint continuously produce an anti-inflammatory signal after a single injection.

Phase I included only 72 people with moderate to severe knee osteoarthritis.

Among participants who continued follow-up, improvements in pain, stiffness, and function persisted for up to two years.

However, the result must be interpreted very cautiously.

By week 104, 33 of 72 participants had dropped out—almost 46%. The study itself was open-label and not designed to provide definitive proof of efficacy.

It also did not prove that the therapy restores damaged cartilage.

PCRX-201 is now being studied in a randomized Phase II trial of approximately 135 patients, where the drug will be compared against a control group.

9. Malaria and tuberculosis: not enough to show that a method works under ideal conditions

Infectious diseases remain a huge global problem, especially in resource-limited health systems.

For malaria, one of the most interesting questions now is no longer whether the R21/Matrix-M vaccine works in a clinical trial.

The vaccine is recommended by the World Health Organization and is already used in national programs.

Now the AVERT study must show how well protection lasts in a real-world health system.

The study plans to enroll about 20,000 children under five years in Burkina Faso and Uganda.

Researchers will compare the vaccination histories of children who presented with suspected malaria and tested positive with those whose tests were negative.

In real life, vaccination is more complex than in a controlled trial: doses may be given late, some children miss doses, transmission intensity changes, and mosquito nets and other preventive measures are used concurrently.

This is why real-world effectiveness may differ from initial trial results.

In tuberculosis, researchers are tackling another problem—long treatment with multiple drugs and the risk of bacterial drug resistance.

In 2026, a Phase II trial was registered in which new TB drugs—including TBAJ-587, BTZ-043, quabodepistat, and ganfeborole—will be combined in various regimens.

This does not yet mean a new short-course TB treatment is available.

The goal of early phases is to determine which combinations are sufficiently effective and safe to advance to larger studies.

What connects all these studies

These nine areas are very different, but together they reflect several broader shifts in modern medicine.

First, treatment is starting earlier. Cardiovascular risk is being actively lowered before the first heart attack, and Alzheimer's is being investigated and potentially treated before memory impairment appears.

Second, therapy is becoming more precise. In oncology, drug selection is increasingly driven by specific mutations and proteins in the tumor, and biologic therapy for chronic diseases targets specific inflammatory mechanisms.

Third, researchers must prove increasingly demanding things. It is no longer enough to show a change in a lab value, weight loss, or improved biopsy. What matters more is whether treatment prevents heart attacks, cirrhosis, loss of kidney function, disability, or death.

That is why not every headline-grabbing clinical trial result means a new standard treatment is available.

A Phase III start does not prove efficacy. Amyloid removal does not guarantee dementia prevention. Symptom improvement after experimental gene therapy does not mean joint restoration.

But it is these kinds of large trials that determine which of today's promising ideas will truly change patient care in a few years.

Read more about nine promising areas of clinical research in the ICHGCP overview.