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Treatment of SCID Disease with Stem Cell Therapy


SCID is an abbreviation of Severe combined immunodeficiency. It is an inherited disease and is also known as Primary immunodeficiency disease and presents in infants. The 1weakened immune system is a basic presentation. The various symptoms of this disease are listed as follows:

  1. Extreme weakness
  2. Loss of Bodyweight
  3. Compromised immune system 2

Genetic alterations affect the function of T cells. B cells and NK cells may also be affected 3the major function of these cells is their aid provision in battling the viruses, bacteria, and Fungi that try to enter the body cells and infect it.4


  1. Complete medical History
  2. Physical examination
  3. Blood tests including complete blood cell count 5


ESCs and iPSCs:

The major component of adaptive immunity is T cells which protect its host again foreign pathogens. These cells are not produced directly in bone marrow, they are produced in the thymus gland.

In an Adult bone marrow, the Hematopoietic stem cells are functioning to provide all the necessary blood cells throughout life in order to maintain homeostasis. In this case of SCID, infants suffer from the intensively compromised immune system. It is necessary to come up with a solution to maintain this immune system and remove the deficiency of the Immune System. One of the methods is the production of HSCs. Hematopoietic stem cells are present in the bone marrow. Hematopoietic stem cell can be produced from Pluripotent stem cells.

These include two types of cells;

  • Embryonic stem cells
  • Inducible PSCs i.e. pluripotent stem cells.6

  • Brief description of the use of induced pluripotent stem cells to treat severe combined immunodeficiency diagnosis followed by drug screening, Disease modelling and Therapy.29


A constant effort is being established to avoid any more infant deaths due to the immunodeficiency diseases using the method of Stem Cell Therapy. The research was established in mice for experimentation of Embryonic Stem cells Therapy.

According to this research:

Mouse Embryonic Stem Cells were introduced into the mice blood. These cells are considered to differentiate into blood cells in vitro and they recapitulate yolk sac hematopoiesis. After this administration, the Embryonic Stem Cells derived B-Progenitors differentiated into marginal B cells and B-1 cells. In mice blood, B-2 cells were not produced. B-1 derived ESCs persisted in the recipient blood for more than 5 months. These newly introduced cells kept producing natural IgM antibodies in vivo. After this, Gene Profiling was done, which lead to the idea that there is a closed relationship between ESCs and YS derived B-1 progenitors. In the ESC culture, no hematopoiesis stem cells were detected. But long term engraftment suggested that Functional B-1 derived embryonic stem cells support the presence of HSC- independent B-1 growth.8

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Embryonic Stem cells and induced PSCs are both types of Pluripotent stem cells. Embryonic Stem Cells are derived from the inner cell mass of human blastocysts while iPSCs are produced in vitro. They share some common features which are listed as follows:

  1. Ability to differentiate indiscriminately into somatic cell types which also includes blood and immune cells.
  2. Indefinite Growth
  3. Maintenance of pluripotency.6

The major component of adaptive immunity is T cells which protect its host again foreign pathogens. These cells are not produced directly in bone marrow, they are produced in the thymus gland. The pluripotent stem cells have the potential to differentiate prominently into somatic cells. This achievement can be established only if the condition provided is suitable. Initial mesoderm induction followed by the specification to hematopoietic precursor cells. These procedures then proceed to the production of T cells which reduce the compromise in the immune system. The experiment done on mice are quite flattering but this method is still not being used in humans as mice and human blood are different.9 In the future, this technique may be used.

Assessment of cKit and CD41+ population is necessary to access the T cell differentiation process.10 evident markers are used to establish the data regarding the differentiation of these cells if the immunodeficiency is to be reduced.


SCID can be treated by Stem Cell Therapy using the Embryonic Stem Cells and induced pluripotent stem cells. In mice, this therapy is known to cause prominent effects. They produced Hematopoietic stem cells which cause the further production of blood cells and thus making improvements in the immune system of mice T cells and B Cells are a basic part of the immune system which can be produced in these therapies. Mice and Human blood are very different in this regards. In humans, this system of therapy is not being used still.

Adult Stem Cells:

A stem cell has the ability to divide and divide over again, this division produces more stem cells which descend into the production of more cells.11 there are different types of stem cells for each type of cell in the body. For Example: In blood, the Hemopoietic cells are used to produce the blood cells that include Red Blood cells, White blood cells and platelets.12 HSCs were obtained originally from Bone Marrow and the basic procedure used in this regard was Bone Marrow Transplantation. Nowadays, a new method is obtained, which extract cells from peripheral Blood or cord blood which is obtained from the placenta at Birth. The blood obtained from Placenta also called cord blood is an excellent source of Hemopoietic stem cells and thus very helpful in getting the immune system recovered.

The most commonly performed Hematopoietic stem cell Therapy is used in severe combined immunodeficiency. There are two problems in successful stem cell therapy. These two problems can be enlisted as follows:

The immune system of host:

One of the obstacles is that the host body or the recipient may react to the introduced cells. They may reject the implanted stem cells as the body has an innate system to react to foreign bodies which are known as Graft Rejection. So, the obstacle of graft rejection is terminated through the process of chemotherapy or Radiation therapy. These therapies confine the immune system of the host so that they do not render the grafted stem cells as foreign and begin to produce antibodies against them. Similarly, the situation worsens when defective or mutated HSCs and stem cells are unable to find a place for their establishment. This is known as “Engraftment Failure”. Chemotherapy is used to prevent this type of complication but chemotherapy is fatal itself as it has serious side-effects. These include bleeding problems due to low platelets, anemia and susceptible infections.13

The second obstacle most commonly observed is Graft vs. Host disease. In this case, the mature T cells or the T cells produced after division in the host, reject the body of the host and begin to attack their own body cells. In this case, to avoid complications, a right donor is chosen in which the mature donor who has the same Human Leukocyte Antigen (HLA) as the recipient is selected.14

Selecting a Donor:

The chance for a perfect match in siblings is 1 in 4. The donor must have the same Group of Human Leukocyte Antigen as the recipient so that there is no chance of failure or rejection of grafted stem cells. HLA antigens are specific for each of us. The exact structure of these HLA antigens is determined by a series of genes clustered on human chromosome no. 166. For the successful engraftment, it is necessary to understand and use the compatibility of HLA. It may be available in the siblings but in this modern day, it is an alternative to find a suitable and matched donor for transplantation through an International Computerised Program where individuals volunteer to provide their stem cells for transplantation. Sometimes parents are also used for Donor selection.15

In case, a perfect match is not available, one of the parents may be used as a donor. In this type, either parent has half of the same alleles as the recipient patient who is there, daughter or son. The problem in this donor selection is that the mature T cell of the haploidentical parent would not be able to recognize the HLA alleles that are very unique to the patient. This would cause Graft vs. host disease. This complication is overcome by removing the mature T- Lymphocytes from the bone marrow before introducing the stem cells into the patient.

The babies who are unable to find a matching donor are injected with T-Lymphocyte free stem cells. The selection for the best choice of the donor is accessed by the following factors:

  1. Type of immunodeficiency disease patient is suffering from
  2. A fraction of immune system left
  3. Degree of matching of potential donors
  4. Patient Age
  5. Type of stem cells available
  6. Degree of the sickness of the patient and the complications. 17

The procedure of Harvesting:

In the case of Bone Marrow Transplantation, the bone marrow is removed from the pelvic bone. Two teaspoons of bone marrow are drained through each puncture site. If more than this is taken, the sample of bone marrow becomes diluted. Such a diluted sample is suspected to contain mature T- cells and they may cause Graft versus host disease.

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Two teaspoons for every two pounds of the recipient body weight are usually taken. More than 101 puncture is required for adults but in the case of SCID, just a few punctures are sufficient due to the low weight of infants. The procedure is done under strong anaesthesia. All donors have prescribed pain medication for two to three days but they are allowed to leave the hospital overnight. The immune system of the donor is not compromised because marrow and HSCs regenerate themselves shortly. In case of extraction of Hemopoietic stem cells from peripheral blood, blood purification is done through a process called apheresis. Donor blood is collected from a donor and white cells are removed while the red cells are returned back through the opposite arm.18 to enrich the donor blood with maximum HSCs, subcutaneous injection of granulocyte-colony stimulating factor is injected in the days after which the blood is drained. This procedure moves the stem cells from Bone marrow to Peripheral Blood.


In the treatment of primary immunodeficiency diseases, the Hemopoietic stem cells transplant between HLA matched siblings has been in use since 1968. The first child to receive this therapy is well and healthy and even has children of his own. In the case of severe combined immunodeficiency disease, the risk of graft versus host disease is minimal.19 in this particular case, the perfect matching donor is found and used for transplant. The success rate, in this case, is as high as 90%. Almost 70-80% of the infants still survive in case the matching donor is not found. The chances of survival are very high if the patient is:

  1. Free from infections
  2. In Good Health
  3. Not suffering from any other pathology at the time of transplant.
  4. Doesn’t have lung damage.

In the case of SCID, the reconstitution of the number and function of the T-Lymphocyte is the rule. In some patients, normalization of antibodies production occurs. The reconstitution process is dependent on several Factors. These factors are enlisted as the use of chemotherapy and Radiotherapy and on the type of donor used for transplant.20

In some cases, reconstitution of antibodies production doesn’t take place. In these cases, the patient requires Ig replacement therapy so that the patient can be prevented from any sort of infection which is very common in case of SCID. Patients enjoy a Good quality of life even if the replacement therapy is required. The patients in case of SCID are infants, the transplantation of stem cells requires several protocols but the survival rate for SCID patients by stem cells therapy is very high and it is being used successfully in many areas of the world although the problem of donor matching still needs to be enhanced. Several Doctors are working free and several NGOs are striving to get rid of this disease.


Adult stem cells have the capability to divide into several other stem cells and differentiate into other cells. Bone Marrow Transplant is also used to obtain stem cells so that they can be introduced in patients with immunodeficiency. In the case of SCID, only a bone marrow transplantation is required for introducing into a patient. Hemopoietic stem cells are obtained from Peripheral Blood. Red Blood Cells are removed from this peripheral blood while white blood cells are introduced into the patients. Graft versus Host disease and host rejection are most commonly seen a problem in this matter. To avoid any complications, chemotherapy and Radiotherapy are used before therapy. After making sure that the Donor and Recipient cells match, the Stem Cells are harvested and they produce good results in infants with SCID. The success rate of stem cell therapy is 90% in the case of matched donor and host. In case, where donor match is not found, T cells progenitors cells are removed from graft and then introduced into the host. The success rate in this situation is 60-80%. The chances of survival of SCID patients increase if they are healthy, not suffering from any infection at the time of transplant and are a match of Donors.

Cancer Stem Cells:

28Discussion between the uses of cancer stem cells appeared in the early 19th century. The cancer stem Cells are specific stem cancer cells that have specific characteristics that are associated with normal stem cells.21 specifically, they have the ability to give rise to all cell types that are found in a particular cancer sample.

Cell sorts that area unit found in an exceedingly explicit cancer sample.

30One theory was conducted for the detection of the proliferation of Cancer stem cells in the SCID mouse in that theory cancer stem cells were delivered intravenously or subcutaneously through Xenotransplantation technique.

Figure.2: Method to study the proliferation of Cancer stem cells in the SCID mouse.

According to the figure, it was clearly depicted that there was a generation of heterogeneous secondary tumour which was analogous to that of the primary tumour and finally there was the existence of CSCs in the cell suspension. 31However, there was still confusion that after grafting some of the cell lines were able to produce secondary tumours while some of them weren’t and hence fro the research it was concluded that SCID mouse xenotransplantation was not sufficient to provide the existence of CSCs.

The failure of traditional therapies, Chemotherapy, and Radiotherapy, most commonly used is usually attributed to these cancer stem cells. Sources of cancer stem cells may be different. They may arise from differentiated cells, restricted progenitor cell or even normal adipose-derived stromal cells. The normal stem cells are the most common target of mutants and carcinogens, as these cells already possess active self-renewal pathways.22 the first cancer stem cell was obtained from human’s inpatient of acute myeloid leukaemia. After this extraction of cancer stem cell, the therapeutic value of cancer stem cells was recognized, these cells are now being used to arrange for methods of targeted therapy. These target therapies may be used in the future to treat cancer cells. Nowadays, some of the techniques are being used for cancer marking by using the already obtained cancer stem cells.23
Cancer Stem Cells are pathogenic, or rightfully carcinogens. SCID treatment cannot use such cells as it will lead to the formation of cancer cells and with the already compromised immune system due to immunodeficiency, it will lead to a worsened situation of the patient. So, it will be ridiculous to use cancer stem cells in the treatment of severe combined immunodeficiency.

The cancer stem cells were first obtained from Acute Myeloid Leukaemia. These cancer stem cells are known to cause cancer prognosis inpatient. Cancer formation begins from these cancer cells. These cancer stem cells are known to have features of normal stem cells i.e. they produce White Blood Cells, Red Blood Cells and platelets. These cells cause cancer, so they cannot be used in the treatment of SCID by introducing the cancer stem cells into the infant with immunodeficiency. So, they have no importance in this case of SCID.

Clinical Applications of Stem cell Therapy:

The various clinical applications of Stem Cell Therapy can be enlisted as given below:

  1. Embryonic stem cells can be used to greatly increase their numbers. These embryonic stem cells have the ability to repair tissues. These cells May even be immuno-privileged.24
  2. The stem cells have a great clinical value in the Case of patients who are suffering from leukaemia. The bone marrow transplant is advised in these cases to provide normal white blood cells.
  3. Stem Cell Therapy is used in various diseases to acquire better immunity and functional capabilities.25
  4. In infants, who suffer from severe combined immunodeficiency, this stem cell therapy is the best available treatment.
  5. In the case of Acquired Immunodeficiency Disease i.e. AIDS, this therapy is sometimes used to a better quality of life in patients infected with HIV.
  6. Stem Cells of different parts have their capabilities to produce particular cells, so these stem cells may be used to produce the specific cells required.26

Commercial Applications of Stem Cell Therapy:

Commercial applications of Stem Cell Therapy can be used in various ways:

The stem cells are not only being used and analyzed in laboratories but also in several commercial companies in various countries of Europe. The main focus of these commercial centres is the production of regenerative medicine. Cell therapies have gained considerable importance in commercial Centres with the aim of maintaining, improving and restoring the structural and functional capabilities of human tissues or organs. They may also be used in repair and compensation of damaged organs and tissues caused by Trauma or disease.27


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