Six-year-old Mia Cara Ferreira from Somerset West, who has been battling Acute Lymphoblastic Leukaemia (ALL) since 2015, will be the first person from Africa to receive the treatment following her unexpected relapse in May this year. Since her diagnosis, Mia Cara has had an intensive treatment of chemotherapy, which has been unsuccessful so far.
The little girl will now be part of the multimillion-rand chimeric antigen receptor (CAR) T-cell therapy, which is offered as part of a clinical trial at the Children’s Hospital of Philadelphia (Chop).
Now only available in the US, CAR T-cell therapy involves the modification of T-cells (immune cells). Unlike conventional chemotherapy, it is a gene-editing treatment by which the patient’s own cells are harvested and re-engineered to act as “killing machines” within the body. After editing, these cells are reintroduced to the body to attack and destroy cancer cells.
The treatment has been referred to as one of the most exciting developments in oncology and childhood leukaemia in decades, and has yielded particularly good results with regards to the treatment of ALL.
The children's hospital implemented a trial phase of this treatment in August 2011, which saw an 83% remission rate within three months of treating the 68 children who participated in the study.
This year two CAR T-cell therapies were approved by the Food and Drug Administration, one for the treatment of children with ALL and the other for adults with advanced lymphomas.
Nevertheless, researchers caution that, in many respects, it’s still early days for CAR T cells and other forms of adoptive cell transfer, including questions about whether they will ever be effective against solid tumours like breast and colorectal cancer.
Mia Cara’s parents, who have so far fund-raised R4million of the R10m needed for the therapy, researched available treatment options and then applied to be part of the US clinical trial, after which she was accepted.
This form of treatment stands in stark contrast to Mia Cara’s preceding reality: to receive another round of intensive chemotherapy, radiation and a bone marrow transplant in South Africa. A suitable bone marrow donor would still need to be found a - a process that can be very lengthy often taking months and sometimes years.
Commenting on the promise of CAR T-cell therapy, Mia Cara’s father, José Ferreira, said such therapy would offer an alternative to the extremely risky process of receiving chemotherapy, a bone marrow transplant or radiation.
“She is only 6 years old and we are not willing to risk the long-lasting side effects of these therapies.
"Our wish as parents is to give our child the best chance of survival and to try to minimise the risks and the side effects, especially since she relapsed after receiving intensive treatment for more than 21 months,” he said.
Mia Cara, who will soon be travelling to the US with her mother, grandmother and her little sister, Sofia, will start the treatment from January 2.
Mia Cara recently underwent the initial cell harvesting process. These cells are being modified in a laboratory and will be kept frozen until she is ready to start the next phase of treatment at the Chop in January. Doctors are hopeful that, after being infused back into Mia Cara’s bloodstream, the re-engineered cells will continue to multiply and will potentially last for months - even years - preventing any further relapses and ridding her body of the cancer altogether.
CAR T-cell therapy is, however, still highly unaffordable, due to the specialised nature of the procedure - and will cost the Ferreira family over R10m over the next two years. This excludes the laboratory costs which are covered as part of the trial and amount to $454 000 (about R5.8m).
After the family’s medical aid turned down the application to fund the treatment, the family started to fund-raise through different initiatives, including crowdfunding campaigns.
“Considering the shocking 4% recovery rate for those South Africans who undergo certain cancer treatment without a bone marrow donor, it is imperative that South Africa gains access to alternative solutions, such as CAR T-cell therapy," said Ferreira.
"It is our sincerest hope that this treatment will ultimately change the future for so many other South African children who are in dire need of alternative treatment for ALL. It would make the world of difference if this treatment became available in our own country and across Africa."