The Valerie Fund News

Statement from The Valerie Fund on FDA Approval of Sickle Cell Disease Gene Therapies

The Valerie Fund is elated at the announcement of the FDA approval of the new gene therapies for sickle cell disease. We are filled with hope at the impact access to this treatment will have on the lives of the thousands of children treated through the eight Valerie Fund Children’s Centers in NJ, NY, and Metro Philadelphia.

The FDA approval of two cell-based gene therapies, with Casgevy utilizing gene editing CRISPR technology, to provide treatment for the pain crises that plague sickle cell sufferers is a game-changing therapeutic development. Halting the pain crises benefits the patient’s quality of life, as well as their quantity of life. Pain crises arise from blockages in blood vessels, as the sickled red blood cells become stuck, impeding oxygen delivery. These new therapies will provide relief from pain, as well as prevent organ damage that can lead to potentially life-threatening disabilities or early death.

“This is indeed, great news for our patients with sickle cell disease. Traditionally, we were only able to treat… now we have such novel transformative therapies available for them and we are optimistic for the outcomes,” said Dr. Shalu Narang, Valerie Fund Children’s Center Pediatric Hematologist-Oncologist with Children’s Hospital of New Jersey at Newark Beth Israel Medical Center. The Valerie Fund Children’s Center at Newark Beth Israel Medical Center treats more patients with sickle cell anemia than any other hospital in New Jersey.

We would also like to recognize former Valerie Fund board member Krista McKerracher, who led the hemoglobinopathy research and development activities at CRISPR Therapeutics, investigating the use of CRISPR Cas-9 technology. Her dedicated efforts have led to this potentially curative therapy for sickle cell disease and β Thalassemia. From current Valerie Fund Board member Dominic DiBari, “Congratulations on this truly extraordinary achievement for mankind and making a life-changing difference in sickle cell patients.”   

All of us at The Valerie Fund are excited and hopeful for a better future where children aren’t in pain or dying from sickle cell disease. “This will help the thousands of children that come through the Valerie Fund Children’s Centers, reducing their time spent in treatment, or in the emergency room in a pain crisis. They’ll be able to live a normal life instead of one marked by the experiences they’ve missed while in a pain crisis,” Barry Kirschner, Executive Director of The Valerie Fund.

We wait with bated breath for when these therapies will be available to The Valerie Fund patients in New Jersey, New York City, Long Island, and metropolitan Philadelphia and look forward to re-defining how we at The Valerie Fund support the pediatric sickle cell patients treated at our eight centers.