Stem Cell Therapies

Stem Cell Therapy

The severe combined immunodeficiency disease also referred to as SCID, is an immunodeficiency syndrome. It’s an uncommon disease that is characterized by the impairment of the development of the T cells and B cells. They occur as a result of genetic mutations. This disease leads to the defective response of the humoral immune system, as a result of the improper B lymphocyte activation. The two types of the immune system are usually affected; that is, both the cellular and the humoral component of the immune system. Severely combined immunodeficiency syndrome is the most prevalent form of primary immunodeficiencies. There have been more than 9 gene mutations linked to this disease. In addition, people affected by this immunodeficiency disease are usually predisposed to be affected by microorganisms such as bacterial, viral or fungal diseases. They do often present with a disease such as diarrhea, pulmonary diseases, and inability to thrive. The prognosis of the disease is usually bad. Babies affected by the disease tend to die within a year if they’re not treated. The cause of their death is usually recurrent infections. The most effective treatment for the disease is hematopoietic stem cell transplantation. There is usually a stem cell supplier for doctors, also, stem cells can be gotten from a healthy donor.

What Are The Symptoms Of Severe Combined Immunodeficiency Disease?

The most common presentation of this disease and other primary immunodeficiency diseases is the susceptibility of affected people to catch infections. They regularly have recurrent, and infections that usually prove difficult to treat, as compared to someone with a healthy immune system. In addition, they are also predisposed to diseases that someone with a normal functioning immune system wouldn’t have. They are known as opportunistic diseases. Below are some of the common signs and symptoms of this diseases;

  1. Pulmonary infections: Persons affected by the severe combined immunodeficiency disease tend to have regular and recurrent lung diseases such as recurrent pneumonia and so on.
  2. Hematological diseases: Patients with severely combined immunodeficiency disorders are also prone to having some blood disorders. Examples of these disorders include thrombocytopenia, anemia and white blood cell disorders.
  3. Gastrointestinal problems: They also present with some digestive problems such as loss of appetite, cramping, constipation, nausea, and vomiting.
  4. They also tend to have a delay in their overall growth and development.
  5. Patients affected by the severely combined immunodeficiency syndrome usually do have autoimmune diseases such as lupus erythematosus disease, erythematous disease and so on.

What Are The Causes Of Severe Combined Immunodeficiency Disease?

The Severe combined immunodeficiency disease is a genetic disease. It occurs as a result of the mutation of some genes. This disease can be inherited; which implies that it can be passed down from parents to their children.

What Are the Risk Factors For The Severe Combined Immunodeficiency Disease?

The most important risk factor for the disease is a family history of the disorder. Parents that have a history of the severe combined immunodeficiency disease in their family should endeavor to undergo several tests to confirm if their fetus would have their disease or not, even before the baby is born. There are some screening tests that could easily check for the presence of the disease even in fetuses.

What Are The Complications Of The Severe Combined Immunodeficiency?

There are many complications that could occur as a result of the severe combined immunodeficiency disease. Some of them are;

  1. Autoimmune disorders: the Autoimmune disease is a disorder in which the immune system of an individual fails to recognize the body tissues as its own, therefore leading to an attack on the tissue. These are usually serious diseases, that could cause a lot of damages if not well managed. Examples of autoimmune diseases are systemic lupus erythematosus, rheumatoid arthritis and so on. Autoimmune disorders are prevalent in people affected by the severe combined immunodeficiency because of gene mutations, and also as a result of the inability of the immune system to fight diseases.
  2. Increased risk of having cancer: The weakened immune system leads to the inability of the immune system in fighting against the development of abnormal cells and tissues in the body. This uncontrolled growth of cells and tissues leading to the development of tumors, which sometimes can be malignant.
  3. Recurrent infections: This is the most common complication of the severe combined immunodeficiency disease. They are prone to frequent and recurrent infections because of the weakened cellular and humoral components of the immune system.
  4. Delayed growth and physical development: Children affected by this disease tend to have a reduced and slowed growth. They tend to achieve the developmental milestones much later than usual, especially when compared to people with a healthy immune system.
  5. Diseases of the organ systems of the body: People affected by this disease also tend to have complications in the various organ systems of the body. For example, patients tend to have pulmonary infections, heart diseases, digestive problems such as constipation, nausea, vomiting, diarrhea and so on.

How Can This Disease Be Prevented?

The severe combined immunodeficiency disease is a genetic disease, therefore, nothing can be done to prevent it. However, a lot can be done to prevent affected people from having infections and complications. Some of the practices that could be done are;

  1. Maintaining a good
  2. Eating a balanced diet
  3. Avoiding stress
  4. Vaccinations- to prevent affected people from disease such as measles, chickenpox and so on.
  5. Being physically active.

How Is This Disease Currently Treated?

The focus of the treatment is usually to prevent and manage the complications of the disease. Also, the immune system of the patient is boosted.

  1. Medications such as antibiotics are given to the patient to treat infections. Also, prophylactic drugs are also given to prevent infections.
  2. The immune system is boosted by administering immune therapy. Gamma interferon therapy is usually done. They are given to s=induce the immune system cells into production

Stem Cell Therapy And Severe Combined Immunodeficiency

Stem cell transplantations are the only cure for this disease. Healthy stem cells are harvested and transplanted from an individual with the normal immune system, to the person affected by the disease. The old stem cells of the patient would usually be destroyed through chemotherapy and radiotherapy before the new stem cells are transplanted.


Gene therapy offers severe combined immunodeficiency hope. (2012). Springer Healthcare News, 1(1).

Gene therapy offers severely combined immunodeficiency hope. (2012). Springer Healthcare News, 1(1).

Hönig, M., Schulz, A. and Friedrich, W. (2011). Hematopoietic Stem Cell Transplantation for Severe Combined Immunodeficiency. Klinische Pädiatrie, 223(06), pp.320-325.

Ozdemir, O. (2016). Neonatal Screening Test for Severe Combined Immunodeficiency of Primary Immunodeficiency Diseases: TREC Assay and Its Limitations. MOJ Immunology, 3(6).