Mount Sinai researchers receive NIH grant to compare new treatment options for sickle cell disease

The Mount Sinai Health System has received a $12,180,625 grant from the National Heart, Lung and Blood Institute to compare new treatment options for sickle cell disease and determine which work best for specific patients.

Sickle cell traditionally has been a neglected disease, but it benefited from a flurry of innovation over the last decade and there are now three new medications approved for the disease."

Jeffrey Glassberg, MD, Director of the Mount Sinai Sickle Cell Program

"While this is welcome news, clinicians now have a new challenge. No studies have compared the drugs to each other or looked at their use in combination, so there's very little information to help decide which of the new drugs are best for which patients."

The study, known as REAL (Registry Expansion Analyses to Learn) Answers, is a collaboration among 10 sickle cell centers across the United States that will implement a novel observational study approach called target trial emulation. This approach enables researchers to gain, within five years, knowledge that is equivalent to dozens of randomized trials.

"We hope to accomplish a century worth of research in a single grant cycle," Dr. Glassberg says. "Patients need answers now."

The study will enroll and collect data from 1,200 sickle cell patients using a structure similar to that of a clinical trial, including blood draws, tracking hospital admissions, and documenting drug administration and adverse events. The researchers will also explore genetic predictors of response to medications, so that clinicians will be able to provide more personalized treatment plans based on each patient's genetic makeup as well as their other unique characteristics.

"Hydroxyurea, the only drug available to treat sickle cell disease until 2017, was initially approved for people who had severe pain crises," Dr. Glassberg explains. "We found out 20 years after its approval that the drug actually improves organ function and prolongs life, but we had only been giving it to people in pain, which is not everyone. I don't want that to happen again with these new drugs. We're going to investigate all of them, individually and in combination, so we can give the right drug to the right patient immediately, and don't repeat our past mistakes."

The study will be led by Dr. Glassberg in collaboration with co-Principal Investigator Abdullah Kutlar, MD, of the Medical College of Georgia. Additional partners in the project include Duke University, Medical University of South Carolina, St. Jude Children's Research Hospital, University of Illinois at Chicago, Washington University in St. Louis, University of California San Francisco, Research Triangle International, and Tufts University.