Sickle-Cell Breakthrough Hailed; Affordability Push Comes Next

A panel of doctors lauded the recent approval of CASGEVY, a gene therapy for sickle-cell disease, but called for advocacy to make the treatment affordable, especially for people on Medicaid.

The panel of clinical experts discussed the breakthrough Thursday evening at Michelle’s House at Chapel and Orchard streets, the site of the Sickle Cell Disease Association of America’s Connecticut chapter. 

James Rawlings, the president of Michelle’s House, introduced the panel, which included MDs Biree Andamarian, Cece Calhoun and Lakshmanan Krishnamurti, and keynoter Farrah Gallard of Vertex Pharmaceuticals, the company that manufactures CASGEVY.

CASGEVY is a one-time therapy that works through the modification of the BCL11A gene. Utilizing the patient’s blood stem cells, the treatment does not require healthy cells from a donor. This eliminates the risk of graft-versus-host disease because there are no foreign cells involved for the host to have an immune reaction to.

Treatment with CASGEVY is a multi-step procedure that includes preparing for stem cell collection, collecting those cells, making the CASGEVY, chemotherapy, receiving the CASGEVY and recovery. In all, the process takes over a year — and costs $2.2 million.

Complications include fertility issues in both men and women, and patients can never donate blood, organs, tissue or cells after undergoing the treatment. 

Krishnamurti emphasized that the treatment can give someone with sickle-cell disease a sense of normalcy for the first time, enabling life as a full-functioning member of society. Panelist Frank Tavarez-Mora, a third-year medical student at Quinnipiac who suffers from sickle-cell disease himself, said that gene therapies show significant promise in revolutionizing treatment processes and improving patient outcomes.

“The last time I gave a presentation on sickle-cell disease was in the 90s,” said Krishnamurti, who works as the chief of pediatric hematology at Yale and joined the panel from Zoom. ​“The last slide said, ​‘One day we will have gene therapy.’” 

In December, that vision came to fruition when the federal Food and Drug Administration (FDA) approved two new sickle-cell gene therapies, CASGEVY and Lyfgenia, for patients 12 and older. Although FDA approval ensures treatments are eligible to be covered by Medicaid, implementation is a state-by-state decision.

Calhoun emphasized the importance of giving sickle-cell disease the same attention that other diseases are allotted. She said the disease has not been prioritized adequately because it disproportionately affects Black people in the United States.

Rawlings noted that the hefty $2.2 million pricetag is nowhere near accessible for most patients. 

Still, treatments like CASGEVY could reduce the lifetime costs of hospital visits, trips to the ER and blood transfusions that those with sickle-cell disease experience. Andamarian added that the treatment could be beneficial for loved ones who serve as caretakers and help those with sickle-cell disease enter the workforce.

For those reasons, she said that legislators must understand that treatment costs need to be covered by Medicaid and insurance providers at large. 

Calling the audience to action, Calhoun quoted Frederick Douglass, imploring the attendees to make noise and ​“to agitate.” 

For those in Connecticut with insurance that does not cover treatment, Calhoun suggested participating in a gene-therapy trial that Yale is currently conducting. Krishnamurti noted that Yale plans to offer the treatments as early as August of 2024, and certainly by December. He urged individuals seeking treatment to begin the process now, highlighting how long it can take.

“I’ve been at this for a very long time,” said Rawlings. ​“This is a very optimistic time for me. I’m all in with this ​‘hope’ thing.” Echoing that sentiment, Krishnamurti asserted that treatments for disenfranchised populations ​“ought to be covered.” 

The primary challenge, said Andamarian, is proving to officials in Connecticut that its Medicaid funds should cover the cost of these gene therapies inside and outside the state. Underscoring that treatment centers providing the gene therapies don’t yet exist in Connecticut, she said the state must be willing to cover the cost for individuals who seek treatment in neighboring states.