How Healthcare Brew readers are innovating around sickle cell disease treatments

As New York City leaders considered legislation to establish a professional education and genetic screening programs for sickle cell disease, Healthcare Brew asked readers to share other ways their cities, hospitals, or companies are finding ways to treat the condition, which disproportionately affects Black people in the US.

Here’s how Healthcare Brew readers from Westchester Medical Center and KovaDx responded:

Westchester Medical Center (WMCHealth): The Sickle Cell Disease program at New York-based WMCHealth’s Maria Fareri Children’s Hospital screens newborns for the painful condition that affects red blood cells and offers cutting-edge therapies—including stem cell transplants—to infants, children, and young adults.

With less than 20% of children eligible for the traditional cell transplant model, which relies on siblings who are perfect tissue matches, WMCHealth experts (and researchers at other facilities) have experimented with transplanting modified, partially matching cells from biological parents. The therapy, used as part of a clinical trial, has helped cure more than 90% of young patients, said Mitchell Cairo, Maria Fareri’s chief of pediatric hematology, oncology, and stem cell transplantation and director of the Childhood and Adolescent Cancer and Blood Diseases Center.

It’s not the only clinical trial underway to experiment with potential sickle cell cures, Cairo said. But “what makes this special is that [potentially] everybody has a donor.”

Cario said that having biological parents as donors is meaningful because children inherit the genes from them. “That, along with the high success rate, makes this important,” he added.

More than 40 patients thus far have participated in the trial, which Cairo said WMCHealth is conducting in partnership with the University of California Los Angeles Medical College and the Milwaukee-based Medical College of Wisconsin. Cairo said about three-quarters of the patients have been treated in New York. The trial is open only to patients aged one to 35.

The therapy isn’t cheap: The cost for such a stem cell transplant can vary between an average of $250,000 to $500,000 over a six-month period, Cairo said, though he added that it’s often covered by Medicaid, Medicare, and other insurers.

And the outcomes, he argued, are worth it.

“This is a disease in which [biological] parents spend their entire time worrying every minute of the day whether they’re going to get a call from school—or from their child—and they’re going to be in the emergency room. It’s the kind of disease where the child can’t do anything that their peers are doing; they’re in pain most of the time,” he said. “Then, all of a sudden, they are completely normal. It is one of the most exhilarating experiences to watch the transformation: to see a child or a family go from what was no hope to the world is waiting for them.”

KovaDx: The medical device startup is developing red blood cell imaging tools (for homes and hospitals) that can screen and monitor sickle cell patient blood health and biomarkers to predict and prevent sickle cell-related pain, company co-founder and chief technology officer Tim Adamson told Healthcare Brew. He said KovaDx is also looking to partner with pharmaceutical companies that are developing sickle cell therapeutics.

“If a sickle cell [patient] had our device in their home, they could be running this blood test maybe once a week or a few times a week, and based on their blood health, if it starts decreasing over the course of a few tests, then they can be warned or maybe their healthcare team could be warned that they’re likely to have a sickling crisis soon,” he said. “They could take some interventions and avoid it.”