Rare Disease Report

NIH Launches Initiative to Accelerate Curative Genetic Therapies for Sickle Cell Disease

SEPTEMBER 14, 2018
Krista Rossi
In an effort to accelerate a cure for sickle cell disease (SCD), the National Institutes of Health (NIH) has launched the Cure Sickle Cell Initiative to quicken the pace at which genetic-based therapies are developed. By utilizing the latest genetic discoveries and technological advances, the NIH aims to move these therapies to clinical trials within 5 to 10 years.
  
“The goal of the initiative is to build a platform for existing and future data that can be used by investigators to perform any analysis they wish,” Traci Mondoro, PhD, branch chief for the Translational Blood Science and Resources Branch in the Division of Blood Diseases and Resources at the National Heart, Lung, and Blood Institute (NHLBI), told Rare Disease Report®. “Any analysis done on behalf of the initiative will be used to assist the development of future clinical trials or to assist the US Food and Drug Administration in the approval/regulation of genetic-based therapies for SCD.”

Gene therapies that modify the patient’s own hematopoietic stem cells (HSCs) will be a focus of the Cure Sickle Cell Initiative, which is seeking to develop cures for a far broader group of individuals with the disease. Since these HSCs may possibly be modified and administered back to the patient via a bone marrow transplant, a cure may be viable for patients who cannot find a matched donor.
 
With its research partners, the initiative will create a national data warehouse of genetic therapies for sickle cell disease and conduct comparative analyses of therapeutic approaches to assess both clinical and cost effectiveness.

“The data will come from patient registries that currently exist and from ongoing/future clinical trials,” Dr. Mondoro added.

National networks will also be created to make learning and engagement with the research, clinical trials, and other activities happening across the country easier for patients and providers.

“The engagement of patients will be a cornerstone of this effort,” said the Initiative’s executive director, hematologist Edward J. Benz Jr., MD, president and CEO Emeritus of Dana-Farber Cancer Institute and the Emmes Corporation, in a recent statement. “Patients will work alongside researchers in developing and recruiting for clinical trials.”

In tandem with the NIH’s initiative, the NHLBI will aim to support the development of cell and genetic therapies resources, clinical trials, comparator analyses of different management strategies, data repositories and resources, and patient and advocate engagement activities related to curative therapies for this condition.

To kickstart the initiative’s research and engagement infrastructure, the NHLBI committed an additional $7 million. The NIH spends about $100 million on sickle cell disease research annually.

Basic research that spans decades in small-scale human studies and animal models on SCD have laid the foundation for potential, novel genetic approaches to cures, such as the genetic editing of bone marrow cells. The NHLBI program has been manufacturing cellular therapeutic products in collaboration with investigators. Such products include genetically-modified cells, which can safely be used in patients in clinical trials.
  
“There are ongoing early phase clinical trials in gene therapy—as opposed to gene editing—that are being performed to establish dosing and safety in small groups of patients,’ Dr. Mondoro said. “It is the goal of the initiative to assist phase 1 trials in moving forward and to assist therapies not yet in humans to meet the requirements to do so.”

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