In a move it has described as "historic”, the Food and Drug Administration has announced the approval of the first gene therapy in the United States, with experts describing it as a fundamental turning point that could transform the practice of medicine.  

Kymriah, the brand name under which Norvatis is marketing the drug tisagenlecleucel, offers a new treatment option that will first be explored for use in cases of acute lymphoblastic leukaemia in kids and young adults.

The treatment, a genetically-modified autologous T-cell immunotherapy, was approved by FDA for use in patients aged 25 or younger who demonstrate certain characteristics of cancer.

Doses of Kymriah are customised using an individual patient’s own T-cells, which are genetically modified to include a new gene that has a specific protein — known as a chimeric antigen receptor, or CAR — that directs the T-cells to target and kill leukaemia cells that have a specific antigen called CD19 on the surface.

The FDA says the safety and efficacy of Kymriah were demonstrated in one multi-centre clinical trial of 63 paediatric and young adult patients with relapsed or refractory B-cell precursor ALL. The overall remission rate within three months of treatment was 83 per cent.

“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” said FDA Commissioner Dr Scott Gottlieb.

“New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses.”

But the immunotherapy carries a big risk of potentially life-threatening side effects, according to the FDA. It could cause cytokine release syndrome, a systemic response to the proliferation of CAR T-cells causing high fever and flu-like symptoms, as well as certain dangerous neurological symptoms.

It is also expensive, at roughly US$475,000, in addition to traditional chemotherapy, which is administered before the Kymriah with the aim of improving the treatment’s success rate.

And because the side effects are potentially so serious, patients will be required to stay within two hours of the 32 hospitals in which the immunotherapy is administered, for as long as a month after treatment, leading to hefty travel and accommodation costs.

Beyond the financial implications there are significant IT hurdles to overcome for widespread and efficient use of personalised medicine.

From data warehousing to EHR design to interoperability, ongoing technology challenges will test IT departments as immunotherapy and other tailored treatments come to the fore.

The research on Kymriah was pioneered at Penn Medicine, but not before the academic medical centre had pursued an extensive retooling of its infrastructure to meet the demands of precision medicine.

“One of the first things we did was to say, ‘Look, we’re not going to get down this road to precision medicine if we don’t have centralised support and a holistic view of IT within the school’,” Brian Wells, Penn Medicine's then associate vice president of health technology and academic computing, said ahead of the HIMSS and Healthcare IT News Precision Medicine Summit in June.

“The other thing we did was ask, what are the high priority applications that the school didn’t have?”

Penn didn’t have common laboratory information management, sample management, sample inventory or sample tracking systems. Wells said Penn also needed a data warehouse to aggregate all the required information and link it to clinical data to allow researchers to access it more easily. 

Despite the challenges inherent in such a novel treatment, researchers and FDA officials said the promise of this new cellular therapy is very real.

Dr Peter Marks, director of the FDA’s Centre for Biologics Evaluation and Research, described Kymirah as a first-of-its-kind treatment for serious disease.

“Not only does Kymriah provide these patients with a new treatment option where very limited options existed,” Marks said, “but a treatment option that has shown promising remission and survival rates in clinical trials.”




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