Amyloidosis and stem cell therapy

Stem Cell Therapies

Stem Cell Therapy

Amyloidosis is a disease that happens when abnormal proteins, called amyloids accumulates in the tissues. This could present with various types of signs and symptoms. Some of the symptoms of amyloidosis include loss of weight, fatigue, glossitis, diarrhea, bleeding, orthostatic hypertension, fainting and so on. There are many types of amyloidosis, and according to studies, there are about thirty varieties of this disease. This disease is mostly caused by genetics, while other causes are acquired. The most predominant type of amyloidosis is a light chain, inflammation, dialysis, hereditary and old age.

It is usually diagnosed by checking for the abnormal protein in the urine, abnormal enlargement of the organs, and multiple neuropathies. The diagnosis is mostly confirmed by doing tissue biopsy. The focus of the treatment of amyloidosis is to reduce the number of abnormal proteins in the body. Autologous blood stem cell transplant is also another alternative to chemotherapy. This is sometimes done by eliminating the cause of the disease. This disease is predominant in people between the ages of fifty-five to sixty years old. The life expectancy of people affected by this disease is usually around half a year to about four years, if the disease is not treated.

What are the signs and symptoms of amyloidosis?

The signs and symptoms of this disease are numerous, and it largely depends on what part of the body the proteins are accumulating. However, the kidneys and the heart are the most commonly affected. Below are some of the symptoms of this disease;

  1. Renal system: The accumulation of abnormal proteins, also known as amyloids in the kidney can lead to the development of nephrotic syndrome. This occurs as a result of the inability of the kidneys to filter the amyloid proteins. This syndrome is usually associated with a rise in the level of creatinine and the blood urea.
  2. Cardiovascular system: The accumulation of the abnormal proteins in the heart can lead to many cardiac disorders, some of these disorders include arrhythmia, heart failure and even heart blocks. These proteins reduce the ability of the heart to contract, and relax, similar to a restrictive pattern defect. However, inflammation amyloidosis usually doesn’t affect the heart.
  3. Nervous system: Amyloidosis do not affect the central nervous system, but it can cause problems with the sensory and autonomic systems. Patients might present with orthostatic hypotension, which might be evident in disorders like nausea, and other gastrointestinal problems.
  4. Hepatic system: The deposition of amyloids in the liver usually leads to the increased amount of liver enzymes such as alkaline phosphatase, and aminotransferase. In addition, patients commonly do have an enlargement of the liver.
  5. Spleen: The enlargement of the liver is not common. It represents less than 5 percent of all amyloidosis cases.
  6. Gastrointestinal problems: Patients do present with gastrointestinal problems such as diarrhea, which might be mixed with blood, and constipation. In addition, patients might have difficulties swallowing food.
  7. Enlargement of tissues: The amyloid proteins deposit in tissues and causes their enlargement. An example of this can be seen in the tongue. Some patients do present with glossitis, swollen joints, such as the shoulder joints and so on.
  8. Endocrine system: It has been also been observed that amyloids also deposit in the thyroid and adrenal glands. The deposition of the abnormal proteins in the thyroid gland usually leads to hypothyroidism. On the other hand, the adrenal gland can be infiltrated, and patients might present with symptoms such as orthostatic hypotension, and hyponatremia. The amyloid proteins also deposit in the pancreas, and they could lead to the development of diabetes mellitus, however, there is no enough evidence to prove this yet.

What are the causes of Amyloidosis?

Generally, amyloidosis is caused by the accumulation of abnormal proteins, also known as amyloids in the tissues and organs. Amyloids are produced in the bone, from where they travel to different parts of the body and accumulate. There are many types of this disease, and the cause of amyloidosis is largely dependent on the type of amyloidosis you have. Below are some of the types of amyloidosis and what causes them;

  1. AL amyloidosis: This is also known as the light chain amyloidosis. It is the most predominant variant of this disease. It usually affects organs such as the heart, kidney, liver and the endocrine organs which includes the adrenal, pancreas, thyroid and so on. This disease occurs when the bone marrow abnormal proteins that cannot be degraded or broken down. These proteins then get transported out of the bone marrow to other parts of the body, where they get accumulate, and alters the function of tissues and organs.
  2. AA amyloidosis: This type of amyloidosis mostly affects the kidney, but can also affect other systems too, such as the gastrointestinal system.  It is usually accompanied by other secondary diseases, such as rheumatoid arthritis.
  3. Hereditary amyloidosis: This type of amyloidosis is usually inherited from parents. It affects organs such as the liver, renal system, heart and the nerves. The type of gene mutated to a large extent would determine the kind of symptoms the patient will present with. Usually, the symptoms of this disease worsen as time passes by.
  4. Dialysis-related amyloidosis: This happens when the abnormal; proteins in blood are deposited in the tendons, and also in the joints. This usually leads to the rigidity of the joints. Patients might also complain of pains. This type of amyloidosis is predominant in people that are undergoing dialysis on a long-term basis.

How is amyloidosis currently treated?

The treatment of amyloidosis depends on the type the patient is affected by, however, this disease is generally treated with high dose chemotherapy

Stem cell therapy of Amyloidosis

Stem cell therapy has been a solid alternative to chemotherapy. Autologous blood stem cell transplant is commonly used to treat this disease. Basically, stem cells are extracted from the patient, and then re-transplanted into the patient. The stem cells are usually extracted from the blood and stored. Chemotherapy procedures are then embarked upon. After this procedure, the stem cells are then re=introduced into the body. This procedure is important for people whose heart is already affected by the abnormal proteins.

References

Dedo, H. and Izdebski, K. (2004). Laryngeal Amyloidosis in 10 Patients. The Laryngoscope, 114(10), pp.1742-1746.

Lee, S., Brauneis, D., Stern, L. and Sanchorawala, V. (2016). Optimal dosing of high-dose melphalan prior to autologous hematopoietic stem cell transplantation in a patient with AL amyloidosis and a solitary kidney. Hematology/Oncology and Stem Cell Therapy, 9(2), pp.86-88.

Madarasingha, N., Satgurunathan, K. and De Silva, M. (2010). A rare type of primary cutaneous amyloidosis: amyloidosis cutis dyschromica. International Journal of Dermatology, 49(12), pp.1416-1418.

Roy, V. (2012). Autologous Stem Cell Transplant for AL Amyloidosis. Bone Marrow Research, 2012, pp.1-5.