An All-Too Common Rare Disease

Sickle cell disease patients have been waiting a long time for a breakthrough; CSL Behring’s expert on what’s next for those who face pain, organ damage and shortened life spans.

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Sickle cell expert Dr. Greg Kato at his desk
CSL Behring Senior Director of Global Clinical Programs for Hematology Dr. Greg Kato

Dr. Greg Kato has seen it up close: sickle cell disease patients in physical pain and their family members suffering the emotional strain of caring for a sick loved one.

One patient’s father comes to mind, says Kato. He was a single dad in Baltimore, by the bedside of one daughter wondering if he would have to miss her sibling’s graduation day.

What exactly is sickle cell disease, so-named for way it curves red blood cells like sickles or crescent moons? Kato, who joined CSL Behring this year after 30-plus years of caring for children and adult sickle cell patients, calls it a “life-limiting disease with acute complications.” That means strokes even in 7-year-old children, he said. Sickle cell disease, which mostly affects minority populations, can be mild in childhood only to rev up in adolescence, disrupting life just as teens are ready to get on with their lives as adults.

“It’s unpredictable. Their disease goes up and down,” said Kato, who spent 13 years at Johns Hopkins, 11 at the National Institutes of Health and another six at the University of Pittsburgh.

The disease causes pain episodes, making it difficult for some patients to keep up with school when they’re young and work a full-time job when they’re older. Not having reliable transportation burdens many and that can prevent patients from getting to medical appointments, a cornerstone of managing a chronic illness. Over time, damage accumulates on the heart, lungs and brain, Kato said. A 35-year-old might need kidney dialysis.

“They become old before their time,” said Kato, CSL Behring’s Senior Director of Global Clinical Programs for Hematology.

With two clinical studies underway, CSL Behring would be a new entrant in the race to provide overdue relief in the form of potential new treatment options. Kato, most recently with the University of Pittsburgh’s Blood Science Center at the Vascular Medicine Institute, is advising his new colleagues to learn as much as they can about the disease and the patients who are living with it.

“Be open minded, listen and learn,” he said. “Be humble and listen, meet people where they are.”

There’s an urgent need for more treatment options and Kato, also a longtime researcher, wants to invest his energies in that direction. Though over 2,000 stem cell transplants have been done, it’s a risky treatment and is unlikely to be the answer for all patients, he said. An estimated 100,000 people live with sickle cell disease in the United States alone.

Sickle cell disease gets oversimplified as an illness whose twisted red blood cells get “stuck” in the body, Kato said. That explanation does not communicate the true complexity of what happens to the cells and the byproducts of those red blood cells as they quickly break down in the body, he said. Deeper knowledge about the disease is opening new avenues of innovation.

Assessing the scientific landscape, there’s reason for hope when you look at the last few years, which saw an acceleration in both research and demonstrable progress, he said. After the U.S. Food and Drug Administration approved the first treatment for sickle cell in the mid-‘90s, the next one wasn’t approved until 2017. But in 2019, a third treatment won approval and two weeks later, a fourth, he said.

At one time, few companies were interested in developing sickle cell treatments, Kato said, based on his time in Baltimore and Pittsburgh research centers. That’s changed.

“(Today), the sickle cell physicians can’t answer all the phone calls from interested biotechnology companies,” Kato said. “I am really excited to help lead the new CSL889 hemopexin program, I’m looking forward to helping to support the CSL200 sickle cell gene therapy program. We have entered a new era for sickle cell disease.”