Mia Cara Ferreira, a six-year old South African girl who has been battling Acute Lymphoblastic Leukaemia (ALL) since 2015 and unexpectedly relapsed in May 2017, will be the first person from the African continent to receive CAR T-cell therapy.
Unlike conventional chemotherapy, CAR T-cell therapy 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.
The process involves the modification of T-cells (known as immune cells), after which they 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 of Philadelphia (CHOP) implemented a trial phase in August 2011, which saw an 83% remission rate within three months of treating the 68 children who participated in the study. Moreover, the US Food and Drug Administration (FDA) approved the first CAR T-cell drug (Kymriah™) in August this year, which means that the benefits of its use outweigh the potential risks associated with its use. Currently the treatment is only available in the US.
Commenting on the promise that CAR T-cell therapy brings, Mia Cara’s father, José Ferreira, said: “CAR T-cell therapy offers an alternative solution to the extremely risky process of receiving chemotherapy, a bone marrow transplant and radiation that Mia Cara would have to endure otherwise.”
“She is only six years old and we are not willing to risk the long-lasting side effects of these therapies, which could include brain damage. Our wish as parents is to give our child the best chance of survival and to try minimise the risks and the side effects, especially since she relapsed after receiving intensive treatment for more than 21 months,” continued Ferreira.
Since her recent relapse, Mia Cara has been accepted as part of the on-going CAR T-cell therapy programme at CHOP. She 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 CHOP, which is scheduled for 2 January 2018.
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 R10 million over the next two years. This excludes the laboratory costs which are covered as part of the trial and amounts to $454,000 (approximately R5.8 million).
Unfortunately, the family’s medical aid won’t cover these costs and they have embarked on various fundraising campaigns, via the Hope for Mia Cara Campaign, to raise the funds to help Mia Cara undergo this life-saving treatment. To date, the foundation has received R3.3 million. A further R6.4 million is necessary to reach the target, depending on the exchange rate.
“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,” concluded Ferreira.
The Ferreira family acknowledges that there are likely hundreds of thousands of people on the African continent that are in desperate need of this treatment, and feel strongly that other parents, family members and friends of cancer patients deserve to share the hope that they now have for their daughter’s recovery. They are in process of registering a non-profit for this purpose and have already started discussions with various institutions. They, together with friends, hope to get a handful of celebrities involved to up the drive for this campaign, and further aim to lobby one of the few pharmaceutical companies developing this treatment to bring the trial to South Africa.