In a precision medicine landmark, FDA approves first gene therapy

Kymriah offers a fundamentally new approach to treating acute lymphoblastic leukemia in children and young adults. Now IT departments gear up for a new era.
By Mike Miliard
01:05 PM

Calling the move "historic," the Food and Drug Administration announced approval August 30 for the first gene therapy in the U.S. 

It’s a gene therapy that hospital CIOs and IT shops, particularly those working on precision medicine initiatives, would be smart to watch since experts are already saying it is a fundamental turning point that holds promise to transform the practice of medicine. 

Here’s what healthcare executives and technologists should know. 

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New frontier in medical innovation

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 leukemia 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 customized 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 leukemia cells that have a specific antigen (called CD19) on the surface.

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

"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 Scott Gottlieb, MD. "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 for potentially life-threatening side effects, according to 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's also expensive, at roughly $475,000. That's 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 where the immunotherapy is administered, for as long as a month after treatment – leading to hefty travel and lodging costs.

What Kymriah means to IT shops

Beyond the financial implications, of course, there are significant IT hurdles to overcome, going forward, for widespread and efficient use of personalized 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, for instance, but not before the academic medical center 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 centralized 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 of 2017. "The other thing we did was ask, what are the high-priority applications that the school didn't have? “

Penn didn't have a common laboratory information management system, for instance, or a common sample management, sample inventory, sample tracking system. Wells said Penn also needed a data warehouse to aggregate all the required information and link it to clinical data so researchers could 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.

Peter Marks, MD, directory of the FDA’s Center 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." 

And one that IT departments are likely to support in the not-too-distant future. 

Twitter: @MikeMiliardHITN
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