Stem Cell Transplant: The Sickle Cell Cure
- Pharm. Onyehalu Jennifer
- Dec 13, 2025
- 6 min read
For decades, sickle cell disease has been managed, not cured. Pain crises, frequent hospital visits, blood transfusions, organ damage, and a shortened quality of life have been accepted as “normal” for people living with the condition.
Today, stem cell transplant, also called bone marrow transplant, and gene therapy are the only two proven cures for sickle cell disease.
Stem cell therapy is not experimental, nor is it a miracle claim.
It is a real medical procedure that has been done successfully for years in different parts of the world and is now being done in Nigeria, including at Lagos University Teaching Hospital (LUTH).
This article explains what it truly involves.
What Is Sickle Cell Disease, Briefly
Sickle cell disease is a genetic blood disorder where the body produces abnormal hemoglobin. This causes red blood cells to become hard, sticky, and sickle-shaped instead of round and flexible.
As a result:
Blood flow is blocked
Pain crises occur
Organs become damaged over time
early mortality, especially in individuals without access to care
Current treatments manage symptoms. They do not remove the root cause.
What Is a Stem Cell (Bone Marrow) Transplant
A stem cell transplant replaces the patient’s faulty blood-producing cells with healthy stem cells from a donor.
These healthy stem cells settle in the bone marrow and begin producing normal red blood cells, not sickled ones.
When the transplant is successful, the body permanently stops making sickled cells.
That is why it is called a cure.
How This Discovery Started
The first successful bone marrow transplant for sickle cell disease was done in 1982 and reported in 1984. It was done in a child who also had another blood condition that required a transplant.
After the procedure, doctors noticed something remarkable. The child no longer had sickle cell disease.
This discovery changed everything.
Over the next decades:
More transplants were done
Protocols were refined
Survival rates improved
Risks reduced with better care
By the 1990s and early 2000s, bone marrow transplant became an accepted curative option for selected sickle cell patients, especially children.
How the Transplant Works (Step by Step)
1. Patient Evaluation
Doctors first assess:
Severity of sickle cell disease
Organ function
Overall health
Age
Ability to tolerate transplant
Not everyone qualifies.
The best donor is usually a fully matched sibling. This gives the highest chance of success and lowest risk of complications.
If no matched sibling exists, some centers use:
Half-matched family donors
Carefully selected alternative donors
3. Stem Cells Extraction
There are two sources here:
Bone marrow: Bone marrow stem cells are harvested under general anesthetic by removing 0.5 to 1.5 liters of marrow from the iliac crest (pelvic bone) using a needle inserted at multiple points. Due to the procedure's complexity, hospitalization, and risks like pain and infection, stem cells are more commonly taken from the blood now.
Blood: The donor is first injected subcutaneously with the growth factor GCSF for four to five days to mobilize stem cells from the bone marrow into the bloodstream. Once sufficient stem cells are circulating, blood is drawn from one arm and passed through an apheresis machine, which centrifuges and separates the stem cells before returning the remaining blood components to the other arm.
The patient receives chemotherapy (and sometimes radiation) to:
Destroy the faulty bone marrow
Suppress the immune system
Make space for healthy stem cells
This stage is critical and closely monitored.
5. Stem Cell Infusion
Healthy stem cells from the donor are infused through a vein, similar to a blood transfusion.
There is no surgery involved.
6. Engraftment and Recovery
The new stem cells travel to the bone marrow and begin producing healthy blood cells.
This process takes:
Days to weeks for engraftment
Months for immune recovery
Patients remain under close medical supervision.
Who Can Benefit Most
Stem cell transplant works best in:
Children and young adults
Patients with severe complications
Patients without advanced organ damage
Patients with a suitable donor
Early intervention gives better outcomes.
How Successful Is It
When a fully matched sibling donor is used:
Cure rates are very high
Most patients stop having pain crises
Blood transfusions are no longer needed
Life expectancy improves significantly
Success depends on:
Experience of the transplant center
Patient health
Quality of post-transplant care
The Risks You Must Know
This is not a simple procedure.
Possible risks include:
Serious infections
Graft-versus-host disease (donor cells attacking the body)
Organ toxicity
Transplant-related death
These risks are why the procedure is done only in specialized centers.
Cost of Stem Cells (Bone Marrow) Transplant
Stem cell transplant for sickle cell disease is a life-saving but expensive procedure. The cost varies widely depending on the country, hospital, donor type, and level of care required.
In countries like the United States, the United Kingdom, and parts of Europe, the total cost of a stem cell transplant for sickle cell disease can range from $100,000 to over $200,000.
In Nigeria, stem cell transplants are significantly cheaper than abroad but still expensive for most families. Current estimates place the cost at approximately ₦60 million to ₦90 million.
Stem Cell Transplant in Nigeria
Stem cell transplant for sickle cell disease is now being done locally.
A major milestone is the establishment of a bone marrow transplant program at Lagos University Teaching Hospital (LUTH), making curative treatment more accessible within the country.
This reduces:
Medical tourism
Cost burden on families
Delay in treatment and care
It also shows that advanced care is possible locally with proper investment and expertise.
What a Stem Cell (Bone Marrow) Transplant Means at the Gene Level
Sickle cell disease is caused by a mutation in the HBB gene, which controls how hemoglobin is made.
SS means the person inherited two sickle genes
AS means the person inherited one normal gene and one sickle gene
This mutation exists in every cell of the body because it is genetic.
What the Transplant Actually Changes
A stem cell (bone marrow) transplant does NOT change the person’s DNA in all their cells.
Instead, it replaces only the blood-forming stem cells in the bone marrow.
So after a successful transplant:
The bone marrow now contains stem cells from the donor
Those donor stem cells produce normal red blood cells
The blood behaves like AA blood, not SS
Pain crises stop
Sickle complications stop
👉 Functionally, the person is cured of sickle cell disease.
The Key Point Many People Miss
The transplant does not change reproductive cells (eggs or sperm).
That means:
The person’s blood genotype changes
Their original genetic makeup remains
So what is their genetic status after transplant?
This is why we say:
The transplant cures the disease, not the inherited gene.
Can a Cured SS Person Have an SS Child With an AS Partner?
YES. They can.
Because:
The cured person still produces SS sperm or eggs
The AS partner produces A or S
Genetic combinations:
S (from cured SS parent) + S (from AS partner) → SS child
S + A → AS child
So the risk of having a child with sickle cell disease still exists.
This Is a Very Common Misunderstanding
Many people assume:
“Since the person is cured, they cannot pass sickle cell again.”
This is not true with stem cell transplants.
The cure affects blood production, not hereditary DNA.
Current stem cell transplants do not alter inheritance risk.
What Should Such Couples Do?
Seek genetic counseling
Do prenatal testing if pregnant
Consider pre-implantation genetic diagnosis (PGD) with IVF if available
Make informed reproductive decisions
This distinction is critical and often misunderstood.
Final Message
Stem cell transplant is not for everyone, but for the right patient, it can mean a life without sickle cell disease.
It is a serious decision that requires:
Proper counseling
Honest discussion of risks
Experienced medical teams
Strong family support
But it represents something sickle cell patients have waited a long time to hear.
A cure is possible.
Stem cell transplant cures sickle cell disease
It does not erase the sickle gene
A cured SS person can still have an SS child
Marriage decisions should still consider genotype
With all this said, ask that genotype question on that first date.
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