- Dubbed a ‘living drug’ by scientists, the treatment is known as CAR-T therapy
- It works by removing key fighting cells called T-cells from the immune system
- A cancer-fighting gene is placed in them before being given back to the patient
- Sufferers of advanced forms of blood cancer are expected to live for 6 months
- But the study found that slightly more than half were still alive nine months on
A groundbreaking gene therapy could treat cancer patients by manipulating their immune system has ‘cured’ more than a third of patients, scientists claim.
After being given the treatment, 36 percent of advanced blood cancer patients showed no sign of the disease.
Slightly more than half were still alive nine months on, despite sufferers only being expected to live for six months before succumbing to the disease.
And 82 percent of the patients had their cancer shrink by at least half at some point in the study, the researchers found.
After being given the treatment, more than one third of blood cancer patients showed no sign of the disease (scans of a 62-year-old patient three months after undergoing the treatment, cancer in black)
Dubbed a ‘living drug’ by experts, the treatment, called CAR-T therapy, removes key fighting cells called T-cells from the immune system.
Scientists then insert a gene that directly targets cancer into them, before giving them back to the patient through a drip.
Despite cancerous cells being well-equipped to evade detection from the immune system, the treatment helps the body battle the disease.
‘The numbers are fantastic,’ said Dr Fred Locke, a blood cancer expert at Moffitt Cancer Center in Tampa who co-led the study.
‘These are heavily treated patients who have no other options.’
Kite Pharma, a US-based pharmaceutical company, released the results from the first six months of its trial. However, the findings have yet to be reviewed by other experts.
The experimental gene therapy, called CAR-T cell, turns a patient’s own blood cells into cancer killers (pictured: scientists in the laboratory working on the CAR-T therapy)
WHAT IS GENE THERAPY?
Gene therapy is an experimental technique that uses genes to treat or prevent disease.
In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.
Researchers are testing several approaches to gene therapy, including:
- Replacing a mutated gene that causes disease with a healthy copy of the gene.
- Inactivating, or ‘knocking out,’ a mutated gene that is functioning improperly.
- Introducing a new gene into the body to help fight a disease.
Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), the technique remains risky and is still under study to make sure that it will be safe and effective.
Gene therapy is currently only being tested for the treatment of diseases that have no other cures.
Patients in the study had one of three types of non-Hodgkin lymphoma, a blood cancer, and had failed all other treatments.
Dimas Padilla, 43, was one of the 101 patients who underwent the experimental treatment.
He was told his cancer was slowly worsening, chemotherapy was no longer working and there was no match to enable a second try at a stem cell transplant.
But after undergoing the CAR-T therapy in August, he saw his tumours ‘shrink like ice cubes’ and is now in complete remission.
‘They were able to save my life,’ he said.
However, there are still concerns that the treatment has significant side effects and could even be deadly.
During the trial, two people died from the therapy – not their cancer – after their immune systems were placed into a state of over-drive.
And 13 per cent developed a dangerous condition where the immune system overreacts in fighting the cancer.
While roughly a third of patients developed anaemia or other blood-related issues, according to the researchers.
Nearly one third also reported neurological problems such as sleepiness, confusion, tremor or difficulty speaking, but these typically lasted just a few days.
Full results will be presented at the American Association for Cancer Research conference in April and the company plans to seek approval from European regulators later this year.
Company officials would not say what the treatment might cost, but other types of immune system therapies have been very expensive.