For decades, the diagnosis of advanced lung cancer was, in practice, equivalent to a months-long sentence. Today, a pill taken once a day is changing that account. Recent research achieved an unprecedented result: more than half of patients with advanced lung cancer spent seven years without the disease progressing.
The research followed patients with advanced lung cancer using lorlatinib, a medication taken daily in tablet form. After seven years of follow-up, 55% of patients remained alive and without disease progression — an unprecedented result for an advanced disease.
Lorlatinib is not for every lung cancer patient. It works only in cases where the tumor is driven by a specific genetic change called ALK rearrangement. This change is present in around 3% to 7% of cases of the most common type of lung cancer.
Although it represents a small portion of cases, the advance draws attention because lung cancer continues to be the killer of men and women in the country, with 31,237 deaths per year, according to the National Cancer Institute (INCA).
One of them is Paula Piteri, who discovered the disease at age 38, with three children and dreams ahead of her. Today, she has been using lorlatinib for almost a year and claims that she has recovered something that the diagnosis seemed to have taken away: her perspective on the future.
But the enthusiasm for scientific data runs into a problem: the medicine is expensive, around R$37,000 per month for pills, and is not in the Unified Health System (SUS). Anyone who has access today has health plan coverage, got it through the SUS in court or paid for it.
A therapy tailored to a specific target
Lorlatinib is an example of what oncologists call targeted therapy: drugs designed to attack specific genetic changes that make a tumor grow.
This strategy represents an important change in the way cancer is treated. For decades, treatments were defined mainly by the affected organ — lung cancer received one protocol, breast cancer another. Today, genetic tests can identify which mutations are driving the growth of each tumor.
With this information, doctors can choose medications designed to block exactly the mechanism that keeps those cancer cells active. Lorlatinib is one of these cases: it is not indicated for all patients with lung cancer, but for a specific subtype of the disease that presents an alteration in the ALK gene.
In about 3% to 5% of non-small cell lung cancer cases, a rearrangement in the ALK gene occurs. This change causes the cell to start producing a defective protein that sends continuous signals for growth and multiplication, functioning as a permanently on switch.
Lorlatinib was developed to block this protein. By binding to the molecular target, the drug interrupts the signal that stimulates the proliferation of tumor cells.
The drug was designed to overcome two limitations observed in previous generations of ALK drugs: the resistance that the tumor develops over time and the difficulty in reaching the brain.
This last characteristic is especially important because brain metastases are among the most frequent complications of this type of advanced cancer. Between 25% and 40% of patients may develop brain tumors two years after diagnosis.
Lorlatinib can cross the blood-brain barrier — a natural protection system that prevents many substances from entering the brain and also hinders the action of several medications.
The drug is already approved and used in clinical practice. The new data presented now extends patient follow-up time and helps to understand how long disease control can be maintained.
And that's when the results impressed doctors: even after seven years of follow-up, more than half of the patients still had the disease under control.
For experts, the result changes the question when a patient is diagnosed: it is no longer how long the patient has, but what is my chance of living for many years with the disease under control?
According to oncologist Gustavo Schvartsman, from Hospital Sírio-Libanês, the observed gain is the result of a gradual evolution of these therapies.
"No one ever went more than five years without the disease progressing when there was only chemotherapy. With the first generation inhibitors, it was for 5% or 10% of patients; with the second generation, 20% to 30%; now we have reached 55%. The advance is very big and we see a huge step in the patient's daily life", he states.
A new chance for Paula
Paula Piteri was 38 years old when she discovered, in 2020, that a nodule in her lung was cancer. She says that she had recently become a mother and that she wanted to have plastic surgery. Leading a healthy life, without any history of smoking, she went for a routine exam when she discovered the disease.
In the first diagnosis, Paula did not undergo genetic sequencing. He removed the tumor and thought that, after the surgery, he would be free. However, in 2025 he received a new diagnosis: the cancer had returned.
“It was a shock, I am a mother of three children and this could change my life completely”, she says.
Today, she uses lorlatinib, provided by the health plan. The medicine is on the list of the National Supplementary Health Agency (ANS) and, therefore, plans are obliged to provide it.
"It's great to have perspective, to know that I have life to live. I want to be part of this and live for many years to come", he says.
What 7 years of data reveal and what we still need to know
Seven years of follow-up is a rare number in oncology, and those who research cancer recognize the weight of this. For oncologist Stephen Stefani, the data needs to be read within a historical trajectory: when there was only chemotherapy, no patient with this diagnosis reached five years without progression. The inhibitors of each generation expanded this horizon.
"The difference in effectiveness becomes very striking when you see this seven-year analysis and it is a relevant result for the patient", he assesses.
Matheus Henrique Dias, a biomedical specialist and molecular biology specialist who researches drugs for cancer treatment in the Netherlands, also says that the study has promising advances.
“For those who have this specific mutation, it will indeed be a big change,” he says.
But both raise points that the data does not yet answer. In the study, 45% of patients were unsuccessful and it remains to be understood why the therapy does not work for them.
Another point that Stephen and Matheus comment on is that there are side effects. In total, 52% of patients reported some effect on the central nervous system: cognitive changes, mood changes and, less frequently, more serious psychiatric impacts. In total, grade 3 or 4 adverse events — which are the most serious, with an impact on daily life — occurred in 77% of patients.
"We see that there are still some points that need to be considered. It is important that people live, but it is very important that they continue to be, to a large extent, themselves", says Dias.
Difficulty of access
Even before treatment, there is a less visible obstacle: molecular diagnosis. Identifying the ALK mutation requires genetic tests that, in many cases, are not available, not even with health plans.
"There is no point in carrying out the test in a system where the medicine will not be available. And there is no point in having the medicine if the patient did not get the correct diagnosis", summarizes Schvartsman.
And there is still the barrier to access to medicine. Today, according to the price list defined by the Ministry of Health, the medicine has a maximum price of R$37,000 for a box of 30 tablets.
Maintained for seven years, the period observed in the study, the treatment would exceed R$3.1 million per patient.
The medicine was debated at the National Commission for the Incorporation of Technologies into the Unified Health System (Conitec), but was not approved due to the high cost for the Unified Health System (SUS).
“The Medicines Committee considered that, despite the evidence presented and the benefits of the treatment, the cost-effectiveness ratio does not indicate efficiency for the SUS, considering that none of the interventions were below the cost-effectiveness limit”, pointed out the commission in the report.
g1 contacted the Ministry of Health to find out if there is a new plan for discussion about the incorporation, but received no response until publication.
