Best Life: New cell therapy may provide solution to sickle cell disease

CLEVELAND, Ohio (Ivanhoe Newswire) – Sickle cell disease is a genetic blood disorder that impacts more than 100,000 Americans.

One in every 365 African American babies born today, will be born with this lifelong disease. One in 13 will carry the sickle cell trait.

It can cause extreme, debilitating pain throughout a person’s body.

Until now, there have been few approved therapies., but researchers are on the edge of a breakthrough.

Danielle Lee’s adventurous spirit shines through, but behind the smiles of this aspiring actress — unimaginable pain.

“I went from like screaming, crying, ‘please don’t let me die’ to ‘I just wanted the pain to be over.’ It is very sharp pain most of the time, to the point where holding my cell phone can hurt,” said Lee.

Lee was diagnosed before birth with sickle cell disease — abnormal hemoglobin makes red blood cells rigid and shaped like sickles. These cells die early, leading to a chronic shortage of healthy red blood cells which are essential for carrying oxygen throughout the body.

“The blood doesn’t go to where it should be. So, the oxygen doesn’t go to potential parts of the body,” said Rabi Hanna, MD Cleveland Clinic pediatric oncologist.

It causes severe pain in the bones, it can impact a patient’s heart, lungs, and eyes and even cause strokes. Dr. Hanna says medications can relieve some of the symptoms. Chemo also helps. Bone marrow transplants work when they’re not rejected.

Now, a one-time gene editing cell therapy modifies a patient’s own blood-forming stem cells to correct the mutation responsible for sickle cell disease.

“We attach the patient to apheresis machine that able to separate the stem cell from the red blood cells from the plasma,” siad Dr. Hanna.

Once the stem cells are collected and sent to the lab for gene editing, patients undergo chemo to destroy their remaining bone marrow, then the edited stem cells are infused back into their body.

“The cells, they can go to their bone marrow and they start to build their house that will produce a new white blood cells, a new red blood cells,” said Hanna.

Results show new white blood cells in patients at four weeks with no severe adverse effects. The patients have also been free of sickle cell disease pain attacks for an entire year. Danielle was one of the first in the CRISPR gene editing clinical trial.

“I have so much more energy, thank God,” said Danielle.

The average life of a sickle cell patient is in the mid-forties. Doctors hope the CRISPR gene editing technology will change that and allow patients to live a long, pain-free life.

The Ruby clinical trial aims to enroll 40 more patients ages 18 to 50 with severe sickle cell disease.

You can find out more here.

Contributors to this news report include: Marsha Lewis, Producer; Kirk Manson, Videographer; Roque Correa, Editor.

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