Back To Top

[Lisa Jarvis] Hard part of sickle cell breakthrough

The approval of Casgevy, Vertex Pharmaceuticals Inc. and Crispr Therapeutics Inc.’s gene therapy for sickle cell disease, is a transformative moment in medicine. Not only is it the first Crispr-based drug to reach the market -- it’s a potentially life-altering advance for a patient population that has been for too long ignored and underserved by the medical establishment. Lyfgenia, a second gene therapy from Bluebird Bio that uses different technology, was also given the Food and Drug Administration’s green light.

Now comes the hard work of treating patients.

That’s no easy task. It starts with ensuring the one-time potentially curative treatment is made available to the patients who would benefit from it most -- and doing so in an equitable way. But, as I’ve explained in the past, the clunky US health care system has no good mechanism for paying for what is certain to be a wildly expensive drug. That could put it out of reach for many, at least in the early stages of its launch.

And the health care system needs to build the infrastructure to support patients throughout treatment and beyond. That means not only helping patients navigate the process of receiving the highly-personalized drug -- a monthslong ordeal that includes a lengthy hospital stay -- but ensuring people continue to be supported in the months and years after treatment.

Several issues can’t fall off the map. One is the effect on a patient’s fertility. To prepare their bodies for the infusion of edited cells, patients must undergo several days of harsh chemotherapy, a process that can destroy eggs and potentially permanently damage sperm. “The biggest hesitation for a lot of our families is the infertility,” says Benjamin Watkins, director of stem cell transplant and cellular therapies at Children's Hospital New Orleans. Often, when describing the gene therapy process to families, the moment the topic of fertility comes up, “the conversation is over,” he says.

Although insurance companies typically cover fertility preservation (which involves freezing and storing eggs, sperm or reproductive tissue) for cancer patients, that benefit is not typically covered for people with sickle cell disease. Given the steep cost -- egg retrieval starts at around $20,000 -- that needs to change. The need is particularly acute for the roughly two-thirds of sickle cell patients insured by Medicaid. One bill pending in Massachusetts would open the door to that coverage and, if passed, could be a template for other states to follow.

The fertility question ties into another critical need for people who receive gene therapy: survivorship support. That means offering ongoing medical treatment to ensure any potential short- and long-term side effects are monitored.

Some of the challenge comes from understanding a body no longer tethered to a chronic disease. That could mean learning how to distinguish between garden-variety pain, residual pain related to organ damage from the disease, and an actual sickle cell crisis.

And many patients will need further mental-health support to deal with the profound emotional experience of being functionally cured. Coming to terms with the new normal, no matter how badly it was wanted, is a process. As Jimi Olaghere, who received the Vertex drug as part of a clinical trial, noted recently in a poignant op-ed in the MIT Technology Review, “Some people become enmeshed with their chronic disease. In many ways, your disease becomes part of your identity and way of life.”

“So many of our patients have been told they’ll die by their 30s or 40s,” says Sharl Azar, medical director of Massachusetts General Hospital’s Comprehensive Sickle Cell Disease Treatment Center. With gene therapy offering them a different future, they must now imagine a life well into their 50s and 60s or beyond. And they will need support as they learn to “balance the new responsibility that comes from being able to live a more ‘normal’ life,” Azar says. That could mean potentially having their first steady job or finishing school.

The handful of sickle cell centers that will initially offer Casgevy and Lyfgenia seem prepared to provide the kind of long-term psycho-social support that patients will need. That component can’t be lost if these treatments start to be administered by doctors without the same deep understanding of the disease.

It's also fair to worry about society’s commitment to a patient population that has been sidelined for so long. After the excitement over the medical breakthrough fades, will the support still be there for these patients in a decade or two? Not everyone will be eligible for or even want gene therapy, and there needs to be continued funding to ensure everyone is supported, no matter the severity of their disease or their chosen treatment course.

The arrival of this Crispr-based gene therapy for sickle cell disease could be a profound moment in medicine. Let’s ensure everything is done right to ensure it takes its rightful place in history.

Lisa Jarvis

Lisa Jarvis is a Bloomberg Opinion columnist covering biotech, health care and the pharmaceutical industry. -- Ed.

(Tribune Content Agency)



By Korea Herald (khnews@heraldcorp.com)
MOST POPULAR
LATEST NEWS
subscribe
소아쌤