Mount Sinai gets $12M NIH grant for study to personalize treatments

Observational study will track comprehensive data on 1,200 SCD patients

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by Mary Chapman |

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Two people each hold the side of a giant check, flanked by balloons and amid confetti.

A $12.2-million grant awarded to the New York-based Mount Sinai Health System will enable a nationwide observational study to help better personalize sickle cell disease (SCD) treatments.

“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 Mount Sinai’s sickle cell program, said in a press release.

“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,” Glassberg said.

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NIH awards $1.9M to project to help SCD children stick to treatment

REAL Answers study is collaboration of 10 sickle cell disease centers in US

The National Institutes of Health-funded study, called Registry Expansion Analyses to Learn (REAL) Answers, is a collaboration of 10 sickle cell centers across the U.S. It will employ a novel approach called target trial emulation that’s expected to allow scientists to gain knowledge within five years that’s equal to dozens of randomized clinical trials, according to Mount Sinai.

“We hope to accomplish a century worth of research in a single grant cycle,” said Glassberg, who will co-lead the study. “Patients need answers now.”

The study will seek to enroll and gather data from 1,200 SCD patients and use a structure similar to that of a conventional clinical trial, in that it will track hospitalizations, blood draws, and treatment-related adverse events. In addition, researchers will study genetic predictors of treatment response, with the overarching goal of enabling clinicians to provide more personalized treatment plans based on each patient’s genetic background and other unique characteristics.

We hope to accomplish a century worth of research in a single grant cycle. Patients need answers now.

While Glassberg acknowledged the proven benefits of the pioneering sickle cell treatment hydroxyurea — which was federally approved in 1998 to help reduce the frequency of vaso-occlusive crises — he said the therapy has not been optimally prescribed over the years. The aim of the new observational study is to avoid that with newer treatments.

“Hydroxyurea, the only drug available to treat sickle cell disease until 2017, was initially approved for people who had severe pain crises,” Glassberg said. “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’s co-principal investigator is Abdullah Kutlar, MD, of the Medical College of Georgia at Augusta University, with project partners including Duke University, St. Jude’s Children’s Research Hospital, Washington University in St. Louis, University of California San Francisco, and Tufts University.