FDA-approved gene therapies bring hope for sickle cell patients, but high costs pose challenges

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Saturday, January 20, 2024
Gene therapies effective for sickle cell but high cost poses challenge
Two newly approved gene-editing treatments for sickle cell disease can be life-changing for patients, but the multi-million-dollar price tag is posing a challenge for hospitals.

LOS ANGELES (KABC) -- It's been more than a month since the FDA approved two milestone gene-editing treatments for sickle cell disease, but lining up patients for these therapies will be a challenge as hospitals navigate the high price tag. But researchers say these new therapies can be life-changing for many patients.

Sickle cell disease has plagued 15-year-old Langston Bennett of Los Angeles with debilitating pain crises.

"Like sharp pain, like a throbbing sharp pain in the chest sometimes," he said.

Sickle cell disease affects the African American community disproportionately. Roughly 2,000 babies are born with it every year. The cost to health care is staggering.

"About $3 billion per year. And that doesn't count the lost opportunities that patients and families with sickle cell struggle with," said Dr. Leo Wang with City of Hope Children's Cancer Center. Wang and his colleagues are the only ones west of Texas offering new gene-modifying therapies to treat sickle cell.

Lyfgenia uses a piece of a virus to produce healthy fetal hemoglobin genes. The other, Casgevy, genetically edits stem cells to do the same. Casgevy is a therapy based on CRISPR, which stands for "clustered regularly interspaced short palindromic repeats."

"These are both therapies that require taking the blood stem cells out of a patient and then fixing them in the lab and then giving them back to the patient," Wang said.

He said a bone marrow transplant, which Bennett just underwent, is a cruder form of gene therapy in which a patient's blood forming system is replaced with a healthy one. In comparison, the new procedures are more precise and no donor is needed.

"This would be an autologous cure, an autologous therapy," Wang said.

The earliest patients treated on these therapies are still fewer than five years out from treatment. So researchers don't fully know the long-term side effects, yet the initial results are impressive.

"The patients who have gotten this far look like they've been cured, but we will have to see," Wang said.

One of the biggest barriers is the cost which is about $2 to 3 million. With FDA approval, the hope is that insurance will cover it

"Paying for this out-of-pocket is going to be impossible, and we need insurers, including government insurance like Medi-Cal, to cover it," he said.

With new gene therapies being developed at a furious pace, Bennett said he hopes everyone who needs it will have access.

"I know a lot of people besides me that have sickle cell, especially in my community, especially South Central," Bennett said.

He's grateful to be starting 2024 sickle cell free.

MORE: SoCal center treats young sickle cell patients from birth

At Kaiser Permanente's Regional Pediatric Sickle Cell and Hemoglobinopathy Center in Downey, early intervention takes on a new meaning.