Sickle cell disease is a rare but very common blood disorder amongst the black community. It is a genetic condition that not only affects the body but is developed in one’s red blood cells from the time they were born. It happens when a child receives two sickle cell genes (one from each parent).
Any and all major organs are ultimately affected by sickle cell disease. The liver, heart, kidneys, gallbladder, eyes, bones, and joints can all suffer damage from the abnormal function of the sickle cells and their inability to flow through the small blood vessels correctly.
To date, it affects 1 million to 3 million Americans with 8 to 10 percent of them being Black.
So, what is being done about it?
Bone Marrow Transplant
What many don’t know is that sickle cell disease can be cured in certain patients and is not a death sentence. A bone marrow transplant, which involves collecting healthy cells from a donor’s bone marrow and transferring them into a patient, can cure sickle cell disease. However, to qualify for the treatment, patients require a donor with fully matched tissue typing, which is often difficult to find. As a result, only about half of patients who qualify for bone marrow transplants actually receive one.
Umbilical Cord Blood
Furthermore, a new treatment, NiCord, developed by a clinical-stage biopharmaceutical company Gamida Cell has been granted breakthrough therapy designation by the U.S. Food and Drug Administration (FDA).
With NiCord, Gamida Cell is trying to make a difference for patients who can’t find matching donors. NiCord is an alternative to bone marrow transplants that instead uses cells collected from the blood of the umbilical cord. These cells are then cultured using a proprietary NAM platform technology, which enriches the cultured cells in highly functional stem and progenitor cells, as well as dendritic cells.
Gamida Cell believes that NiCord can provide a transplant option that is available to all patients in need, based on results from their clinical studies. Compared to unmanipulated umbilical cord blood, the therapy has improved time to neutrophil engraftment (incorporating the transplanted cells into the recipient tissue) from 21 days to 10 days; the number of patients achieving neutrophil engraftment (75% versus 18%); faster platelet engraftment (32 days vs. 46 days); and lower transplant-related mortality (19% vs. 39%).
Additionally, NiCord induced fewer infections and reduced the length of time spent in the hospital.
Gamida Cell is now waiting for positive feedback from the FDA and the European Medicines Agency (EMA) regarding the outline of the Phase 3 trial, after which it will soon begin assessing NiCord in patients with blood cancers.
How does it affect patients?
A bone marrow, stem cell or cord blood transplant is an expensive medical procedure. The cost will depend on:
As for umbilical cord blood treatment, although the costs are still unknown, studies have shown patients achieve recovery nearly twice as fast as those who received other transplants. This decreased recovery time should reduce the amount of time patients stay in the hospital, one of the largest costs involved with cord blood transplants. The reduction in recovery time could quite possibly make this method the most cost-effective option for hematopoietic stem cell transplantation.
For more information on the latest in sickle cell, visit our Health Conditions page on BlackDoctor.org.
Tia Muhammad, BS, is an award-winning freelance content & media creative, copywriter, blogger, digital designer, and marketing consultant. She owns the boutique content and digital media company, jackieGLDN|studio.
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