Woman given experimental cancer treatment as a child breaks record for longest time in remission
A Texas woman who was treated with an experimental form of cancer therapy as a toddler has broken records and been in remission for nearly two decades.
In 2006 at the age of four, she was diagnosed with neuroblastoma, a rare type of cancer which develops in the neuroblasts in the adrenal glands.
These small organs sit on top of the kidneys and are responsible for releasing hormones that control digestion, blood pressure, breathing and heart rate.
When the cancer spread to her bones and risked becoming terminal, doctors referred her for highly experimental therapy trial to save her life.
The unidentified now-23-year-old woman was one of the earliest American patients to be given CAR-T therapy at Texas Children’s Hospital in Houston as part of a trial.
The treatment involves modifying patient’s immune cells in a lab to make them better at killing cancer cells.
Despite having a 38 percent chance of surviving, she’s cancer-free 19 years on and mother of two healthy children.
In 2006 at the mere age of four, the patient was diagnosed with neuroblastoma. (Stock image)
The doctors who treated her said: ‘She has never required any other therapy and is likely the longest-surviving patient with cancer who received CAR-T therapy.
‘Encouragingly, she has subsequently had two full-term pregnancies with normal infants.’
Professor Helen Heslop, who led the trial, said: ‘It’s nice to have such long-term follow-up and to see that even if it was a very early CAR T-cell – and there’s been a lot of work to make them better – we were still able to see a clinical remission that’s been sustained for this long, so that she’s grown up and is leading a normal life.’
CAR-T therapy is now widely available and used over 30,000 Americans with blood cancers including lymphomas, some forms of leukemia and, most recently, multiple myeloma.
The treatment is about 76 percent effective against blood cancers and was first approved in 2017 — with six versions now available.
But at the time, it was highly experimental.
The Texas woman was one of 19 children involved in the clinical trial that took place between 2004 and 2009 for neuroblastomas.
In a span of seven years from the time of their treatment, 12 out of 19 patients died due to their cancers coming back.
Six others are still alive and remain cancer – free. One patient stopped showing up for their annual check-ups.
An illustration of a T cell, blue, attacking a cancer cell, red. This underlines how CAR-T therapies work
CAR-T is currently only given to those who have undergone other cancer treatments such as surgery to remove tumors but have relapsed.
According to the National Cancer Insititute, the therapy has resulted in long-term survival in fewer than half of the patients treated over the years.
It is also known to cause serious side effects, including cytokine release syndrome, which can lead to organ failure and nervous system problems.
However, the Texas woman is the only person who had active solid tumors at the time, not received any other cancer treatment apart from CAR-T and has since remained cancer-free.
Sneha Ramakrishna, a paediatric oncologist and cancer researcher at Stanford University School of Medicine in California, who was not involved in the study said: ‘This provides me with a lot of hope.
‘We’re going to unlock CAR-T cells for people with solid tumours.’
The paper was published on February 17 in Nature Medicine, a peer-reviewed journal.
