11-year-old becomes the first recipient of Novartis’s personalized CAR-T therapy

By Rajaneesh K Gopinath

The youngster from Watford was treated with the immunotherapy agent, Kymriah to counter a form of cancer that he has been battling for years.

Yuvan Thakkar became the first patient under the National Health Service (NHS) England, one of the four healthcare providers in the United Kingdom, to receive the Chimeric Antigen Receptor (CAR) T-cell therapy, Kymriah. He was diagnosed with Acute Lymphoblastic Leukemia (ALL) back in 2014 and has since undergone two rounds of chemotherapy and a bone marrow transplant. Usually in most cases (90%), such standard treatments substantially alleviate the disease but unfortunately his cancer relapsed. However, the timely arrival of personalized CAR-T cell therapy has given ample reasons for Yuvan and his family to be optimistic.

Kymriah – A brief history

In August 2017, the USFDA approved Novartis’s Kymriah (Tisagenleclecel) for the treatment of pediatric and young adult patients up to 25 years of age whose ALL relapsed after conventional treatments. The approval was based on the positive results from three key clinical trials and was the first FDA approved gene therapy. Last year it also received approval from the European Commission (EC). Kymriah was developed by Novartis in collaboration with University of Pennsylvania. The original cost of this personalized immunotherapy is £282,000 per patient, but all thanks to the deal between Novartis and NHS England last year, the price is now set at an affordable range.

CAR T-cell therapy

CAR T-cell therapy is a unique form of treatment wherein T-cells are obtained from the patient’s own blood and genetically modified to produce CAR T-cells. These modified T-cells are then infused in to the patient’s bloodstream where they destroy the tumor cells by specifically binding to cell surface proteins. Yuvan’s T-cells were extracted last November and were genetically modified at Rotterdam and Texas. Last week, Yuvan was infused with modified CAR-T cells at the Great Ormond Street Hospital in London thereby making him the first patient apart from the ones in clinical trials to undergo this ground-breaking therapy. Yuvan, a Lego enthusiast and an ardent cricket fan has plans to go see the Lego House in Denmark post his recovery. With that kind of mental toughness, he has become an inspiration for many.

References

1. https://www.bbc.com/news/health-47058069
2. https://www.gosh.nhs.uk/news/gosh-patient-first-receive-ground-breaking-new-cancer-therapy-nhs
3. http://www.drugtopics.com/clinical-news/tisagenlecleucel-kymriah-acute-lymphoblastic-leukemia
4. https://www.england.nhs.uk/2018/09/nhs-england-announces-groundbreaking-new-personalised-therapy-for-children-with-cancer/

Author

Rajaneesh K. Gopinath