Transplant stories

The leukaemias are a group of cancers of the blood and bone marrow. Broadly speaking, these diseases can be classified as acute or chronic. Some leukaemias, particularly in the acute category, can progresses rapidly and aggressively and thus requires prompt treatment, whereas others are slower to progress.

The leukaemias are classified according to the cell type and chromosomal problems associated with the cancer.

Leukaemia incidence is strongly related to age, with the highest incidence rates being in older men and women. In the UK, around 9,000 people are diagnosed each year with leukaemia. (3)

Multiple myeloma is a cancer which affects plasma cells which are a type of white blood cell of the immune system. The disease arises in the bone marrow where plasma cells are generated and results in abnormal cells being produced. Healthy plasma cells help fight infections by making a range of proteins called antibodies which can attack and destroy bacteria and viruses. Myeloma develops when there is a molecular abnormality in the plasma cells which changes their DNA and causes them to make abnormal proteins sometimes referred to as paraproteins.

In multiple myeloma, malignant plasma cells are over produced and build up in the bone marrow where they crowd out healthy blood making cells. This causes serious complications for patients as their normal blood components such as immune cells, red blood cells and platelets are compromised.  The production of paraproteins by malignant plasma cells means they are ineffective at fighting infections and also causes complications clinically which can be numerous and potentially very serious and debilitating.  Complications include, but are not limited to, impaired renal (kidney) function, haematological problems (anaemia, bone marrow failure, bleeding disorders), infections, bone complications (pathologic fractures) and neurological complications (spinal cord and nerve root compression.

Multiple myeloma may be treated in several ways with a range of medications but if relevant, a stem cell transplant may be offered. With autologous stem cell transplantation, the stem cells used are derived from the bone marrow or peripheral blood of the patient themselves. Alternatively, patients may receive an allogeneic transplant where the cells come from a healthy matched donor and cord blood has occasionally been utilised in this setting.  Whether the patient is receiving an autologous or allogeneic transplant, they receive very high doses of chemotherapy designed to kill the myeloma cells (abnormal plasma cells) but inevitably any normal bone marrow function is severely damaged as a side effect. The transplanted stem cells are capable of restoring or rescuing the bone marrow function in the patient. In the case of allogeneic transplant, the healthy donor cells may also recognise any cancerous plasma cells in the patient and help to destroy them.

The length of survival for myeloma patients depends on many factors including the stage and the specific molecular abnormality of the disease, treatment already given and response to treatment. Remission from the disease following treatment with medication +/- transplantation can last for months or years, but unfortunately the myeloma is likely to come back eventually and will then need further treatment.

Cerebral palsy is the general term for a number of neurological conditions that affect movement and co-ordination. Neurological conditions are caused by problems in the brain and nervous system.

Specifically, cerebral palsy is caused by a problem in the parts of the brain responsible for controlling muscles. The condition can occur if the brain develops abnormally or is damaged before, during or shortly after birth.

It is estimated that 1 in 400 people in the UK is affected by cerebral palsy.

Autism spectrum disorder (ASD) is the name for a range of similar conditions, including Asperger syndrome, that affect a person’s social interaction, communication, interests and behaviour.

In children with ASD, the symptoms are present before three years of age, although a diagnosis can sometimes be made after the age of three. It’s estimated that about 1 in every 100 people in the UK has ASD. More boys are diagnosed with the condition than girls.(8)

Thalassaemia is an inherited condition, which means it can be passed on from parents to children. It’s not known exactly what causes the genetic mutations associated with thalassaemia. However, it’s likely they’ve they have persisted in certain areas of the world as carriers of the condition (both alpha and beta thalassaemia) are protected against malaria.

This is why thalassaemia and other related genetic blood disorders, such as sickle cell anaemia, are more common in parts of the world where malaria is a problem, including certain Mediterranean countries such as Greece, Cyprus and Italy, the Middle East, Asia and sub-Saharan Africa. (2)

Stem cells in the treatment of neurological damage including spinal injury

There is growing interest in the use of stem cells in the treatment of neurological damage including spinal injury. This category of regenerative medicine includes orthopaedic applications to help treat spinal joints and associated spinal nerves in addition to strategies to address other spinal cord damage.

Mesenchymal stem cells (MSCs) are the key players in regenerative medicine applications including cell-based therapies for neurological damage. MSCs used in this setting have been derived from various sources including cord blood, umbilical cord tissue, adipose tissue and bone marrow.

MSCs have the capacity to undergo differentiation into neuronal cells under certain circumstances but more importantly they can secrete a range of proteins and other factors at the site of injury. These proteins can damp down overactive immune responses, they have  anti-inflammatory properties, can help in regenerating blood vessels and can augment tissue healing. Overall, MSCs promote a microenvironment at the site of injury that enhances local repair and tissue regeneration.

Spinal cord injury  is complex so a combination of approaches rather than stem cell therapy alone may be beneficial. A comprehensive treatment plan may include injection of stem cells along with other materials or the use of biologically inert scaffolds to support stem cell therapy in addition to the use of drugs and physical therapy.  Results suggest that MSC transplantation for spinal cord injury results in better outcomes for some patients in terms of improved movement, sensation and quality of life compared with rehabilitation alone. However, further clinical trials are required to confirm the effectiveness and safety of these interventions.

Diabetes occurs either when the pancreas does not produce enough insulin or when the body cannot effectively use the insulin it produces. Insulin is a hormone that regulates blood sugar. Hyperglycemia, or raised blood sugar, is common in uncontrolled diabetes and over time leads to serious damage to many of the body’s systems, especially the nerves and blood vessels. Type 1 diabetes which can affect people at any age, but usually develops in children or young adults. The disease is caused by cells of the immune system called T cells that become inappropriately activated and kill the insulin producing beta cells in the pancreas, resulting in reduced insulin levels and hyperglycaemia. Type 1 diabetic patients need daily injections of insulin to control their blood glucose levels. There has been considerable interest in innovative stem cell therapies to help treat diabetic patients. These treatments are designed either to replace beta cells in the pancreas or to down regulate the immune cell destruction of these cells and promote healing.

Brain damage may be due to several causes including but not limited to brain injury due to ischemia which is a condition where which blood flow (and thus oxygen) is restricted or reduced to the tissues.  Ischemia at birth can cause severe damage to the  brain leading to conditions such as cerebral palsy in children. Ischemia is also the cause of approximately 85% of strokes and occurs when blood flow to a region of the brain is reduced. Both cerebral palsy and stroke result in neurological tissue damage and inflammation. It is believed that transplanted cells used to treat these conditions migrate to the damaged tissues and secrete a complex range of proteins that provide anti‐inflammatory and neuroprotective effects, thus enhancing the brain’s repair mechanisms.

Cerebral palsy is the general term for a number of neurological conditions that affect movement and co-ordination. Neurological conditions are caused by problems in the brain and nervous system.

Specifically, cerebral palsy is caused by a problem in the parts of the brain responsible for controlling muscles. The condition can occur if the brain develops abnormally or is damaged before, during or shortly after birth.

It is estimated that 1 in 400 people in the UK is affected by cerebral palsy.

Sickle cell anaemia is a serious inherited blood disorder where the red blood cells, which carry oxygen around the body, develop abnormally.

The disorder mainly affects people of African, Caribbean, Middle Eastern, Eastern Mediterranean and Asian origin. In the UK, sickle cell disorders are most commonly seen in African and Caribbean people. (7)

The leukaemias are a group of cancers of the blood and bone marrow. Broadly speaking, these diseases can be classified as acute or chronic. Some leukaemias, particularly in the acute category, can progresses rapidly and aggressively and thus requires prompt treatment, whereas others are slower to progress.

The leukaemias are classified according to the cell type and chromosomal problems associated with the cancer.

Leukaemia incidence is strongly related to age, with the highest incidence rates being in older men and women. In the UK, around 9,000 people are diagnosed each year with leukaemia. (3)

Autism spectrum disorder (ASD) is the name for a range of similar conditions, including Asperger syndrome, that affect a person’s social interaction, communication, interests and behaviour.

In children with ASD, the symptoms are present before three years of age, although a diagnosis can sometimes be made after the age of three. It’s estimated that about 1 in every 100 people in the UK has ASD. More boys are diagnosed with the condition than girls.(8)

Infantile (HSV) Encephalitis is an uncommon but serious condition caused by a virus. This can lead to inflammation and damage to the brain. (4)

Thalassaemia is an inherited condition, which means it can be passed on from parents to children. It’s not known exactly what causes the genetic mutations associated with thalassaemia. However, it’s likely they’ve they have persisted in certain areas of the world as carriers of the condition (both alpha and beta thalassaemia) are protected against malaria.

This is why thalassaemia and other related genetic blood disorders, such as sickle cell anaemia, are more common in parts of the world where malaria is a problem, including certain Mediterranean countries such as Greece, Cyprus and Italy, the Middle East, Asia and sub-Saharan Africa. (2)

Severe combined immunodeficiency (SCID) is the name given to a group of rare inherited disorders which cause severe abnormalities of the immune system.

This happens when white blood cells, responsible for fighting infection, are missing or working poorly. Their absence or poor function results in serious and often life-threatening infections. (6)

Hypoxic Ischemic Encephalopathy (HIE) is a birth injury that describes oxygen starvation that happens to the infant brain. There can be severe neurological impairment and damage to other organs as a result. (5)