Teen says he’s ‘doing great’ after undergoing experimental sickle cell treatment

(NEW YORK) — Sixteen-year-old Jonathan Lubin loves basketball, playing the drums and going to the gym.

But until recently, his parents worried he wouldn’t live to see the age of 40.

Lubin was born with sickle cell disease, a genetic illness that causes abnormal ‘C’-shaped red blood cells that clog blood flow, causing severe pain episodes and organ damage.

More than two years ago, Lubin became one of the youngest patients to sign up for a still-experimental CRISPR gene editing therapy.

The treatment, which he received at New York-Presbyterian/Columbia, required chemotherapy and involved a complex months-long process of harvesting his own stem cells, editing them and then reintroducing them into his body.

But today, Lubin is able to do everyday activities that used to be risky.

The teen said he has not had a pain crisis or hospitalization in over two years, a stark contrast from his childhood, which has been characterized by hospital visits every few months.

“It’s been about two years and I’m doing great,” Lubin told “Good Morning America.” “Overall, I’m just feeling way better.”

For Lubin and other clinical trial volunteers, it’s been a major transformation.

And researchers say that thanks to the bravery of these volunteers, the U.S. Food and Drug Administration recently approved two new gene-editing therapies for sickle cell disease, including the CRISPR-based therapy Lubin received.

The CRISPR-based gene therapy Lubin received is made by Vertex Pharmaceuticals, and developed in partnership with CRISPR Therapeutics. The FDA approved a second gene therapy made by bluebird bio.

Sickle cell disease is a genetic condition that affects approximately 100,000 Americans – primarily Black Americans with African ancestry, according to the Centers for Disease Control and Prevention.

Researchers estimate that roughly 20 to 25% of those with the disease are sick enough that they would be good candidates for the newly approved treatments, which were approved for people aged 12 and older.

Jimi Olaghere and Victoria Grey – both in their 30s – said they volunteered to have a chance to be better parents to their children. Both travelled out of state to be treated at Sarah Cannon Research Institute and HCA Healthcare’s TriStar Centennial Children’s Hospital in Nashville.

Prior to his treatment, Olaghere said he felt like he was “living in a nightmare” with frequent hospitalizations and pain crisis.

“Now I wake up, I get the kids ready for school,” Olaghere said. “It is complete night and day. It is a completely different life.”

But the sticker shock of both newly-approved treatments – both multi-million dollars – has raised questions about access.

Given the high cost of treating repeat pain crisis over the lifetime of a person with sickle cell disease, some insurance providers have opted to cover the new treatments.

And the Biden Administration has created a new access model for Medicaid patients, designed to lower the medication cost and improve access to gene therapy.

The program is set to begin in January 2025.