Potential Sickle Cell breakthrough

Published: Sep. 11, 2019 at 10:35 PM CDT
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BIRMINGHAM, Ala. (WBRC) - Lynndrick Holmes underwent a gene therapy treatment at the National Institutes of Health in Washington, D.C. that has left him “sickle cell free”.

Doctors say it takes about five years without complication to declare him “cured” of sickle cell. But as he prepared breakfast for his family at home in Mobile recently, he remains mindful of how the disease used to disrupt his life at any given moment.

“I took my kid to Six Flags and (in the past) I probably would have went into a sickle cell crisis along the way” says Holmes. “And all the money I spent on hotels and tickets would have been to waste because I would have been stuck in a hospital”.

A sickle cell crisis happens when red blood cells become sickle shaped, clogging blood vessels, starving organs of oxygen and creating intense pain.

But at NIH in March, Holmes completed a treatment that involved taking stem cells from his bone marrow; fixing the gene that causes his cells to sickle; and reinserting that gene using the H-I-V virus, minus the parts of the virus that cause infection. That last part of the process takes place after patients undergo chemotherapy to prepare for the introduction of the new cells.

The result for Holmes and his family is freedom from the pain and disruption of sickle cell.

“Now he has the time to actually live for the first time in his life” says Holmes’ wife of three years, Dominique.

For Lynndrick, who is going to school to become a behavior analyst, being sickle cell free also ushers in a period of rediscovery. “Now I have to rebuild myself and figure out what am I actually capable of because I never got the chance to figure out who am I and what I can really do. Sickle cell was always a hinderance”

According to the NIH the trial has about 50 slots, most of which are already taken. Two of those patients will undergo the therapy at UAB.

Dr. Julie Kanter, who runs the university’s Adult Sickle Cell Clinic reminds us that sickle cell patients who have a family member who is a genetic match, can already be cured of the disease through a similar gene replacement process. She also has several other treatments that can help people live with the disease.

“I’m incredibly excited about this therapy as an option for people” she says.

To learn more about treatments available at UAB, go to www.uab.edu/medicine/sicklecell.

For more on the National Institutes of Health gene therapy clinical trial and sickle cell disease studies at the NIH, contact the Patient Recruitment Center at (800) 411-1222 or by emailing prpl@cc.nih.gov.

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