Diabetes Type 1

Diabetes is a disease in which the body’s immune system attacks the pancreas, such that the pancreatic beta cells that exist there are no longer able make enough of the hormone insulin to properly control the amount of sugar (glucose) in the blood. In type 1 diabetes, the amount of sugar is too high, which can cause permanent, long-term damage and death. The cause of type 1 diabetes is a mystery, but it is considered to be an autoimmune disease, where beta cells are the target of the immune system’s attack. The vast majority of people with type 1 diabetes manage the disease by testing their blood sugar and injecting themselves with insulin multiple times daily.

Currently there are no Health Canada or FDA approved stem cell treatments available for diabetes. However, because the disease is due to the loss of a single cell type – the beta cell – it is a perfect candidate for a regenerative stem cell therapy. Many recent advances in Canada and elsewhere have led to early stage clinical trials, suggesting that a stem cell-based therapy may soon be available for diabetes patients.

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Crohn’s Disease

Crohn’s disease is largely considered to be an autoimmune disease, where the body’s own immune system turns on the digestive tract, creating inflammation that eats away at the digestive tract tissues. It is one of two chronic inflammatory bowel diseases (the other is ulcerative colitis), and can strike anywhere along the digestive tract, from the mouth to the anus, but most commonly strikes the ileum (lower part of the small intestine), the colon (large intestine) and the area where the two connect. There is no cure for Crohn’s though many drug therapies exist to stifle its progress and reduce symptoms.

Currently there are no Health Canada or FDA approved stem cell treatments available for Crohn’s disease. While we know that inflammation is a key part of the development of Crohn’s, the exact cause and the mechanisms for its persistence are not known and researchers are looking at autoimmunity, bacteria, genetics and the environment for clues. Several pre-clinical and early stage clinical trials using stem cells to treat Crohn’s are underway and include approaches that use mesenchymal and/or fat-derived stem cells, as well as blood stem cells.

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Cerebral Palsy

Cerebral palsy (CP) is not a single entity, but rather describes a group of permanent disorders that cause a range of life-long motor and posture-related impairments. Individuals with CP experience problems controlling motor function, but may also have complications in behaviour, learning, epilepsy, communication, vision, hearing, perception and sensation. The causes of CP are complex and not entirely understood. Treatment often involves exercise, speech and/or occupational therapies, surgery and drugs, but there is no cure.

Currently there are no Health Canada or FDA approved stem cell treatments available for cerebral palsy. Because we do not fully understand the causes of CP, there is much research that yet needs to be done. However, scientists are looking at ways different types of stem cells can be coaxed to help repair the central nervous system that is damaged in CP in hopes that this may lead to a future therapy.

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Cancer – Solid Tumour

Tumours are any abnormal growth of cells, which can be benign (harmless) or malignant (cancerous). There are many different forms of cancerous tumours and they can grow in many different tissues in the body – the name they are given depends on where they are found: for example, carcinomas are found in the skin in the lining of internal organs; sarcomas can be found in bone and cartilage, fat, muscle, blood vessels or connective tissue; lymphomas develop in the immune system. If solid tumours are benign and localized, they can generally be removed and pose no long-term threat. However, solid tumours that have acquired aggressive properties are able to spread (metastasize) via the blood or lymphatic (immune) systems to another part of the body. Once cancers metastasize the prognosis for the patient becomes poor, but why some tumours metastasize and others do not is still a mystery.

Hematopoietic stem cell transplants (from bone marrow and umbilical cord blood) have approval from Health Canada and the U.S. FDA Administration to help treat some patients with solid tumours. But not all tumours can be treated this way and researchers are using stem cells in the lab to study the underlying cause of cancer (which may originate in the stem cells themselves) and to quickly and safely test drugs that might help curb the spread of the disease.

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Breast Cancer

Breast cancers develop initially as “solid” tumours and are classified according to the cells in the normal breast that they resemble most closely. Some appear abruptly, but most are slow-growing; a result of a series of many steps in which mutations accumulate in normal breast cells to form cancer. Early stage breast cancer indicates a tumour that has not yet crossed the basement membrane barrier that surrounds the mammary gland. Advanced breast cancers have crossed the basement membrane into surrounding tissues, where they may invade and establish new tumours (metastasize) in the bone, liver, brain and other organs. If confined to the breast, most breast cancers can be cured or treated through surgery, radio- chemo- and/ or hormone therapies.

Currently there are no Health Canada or FDA approved stem cell treatments available for breast cancer. While some forms of cancerous tumours can be treated with a hematopoietic (blood) stem cell transplant, not all tumours can be treated this way and breast cancer is one that, up to now, has been resistant to this type of therapy. Researchers are using stem cells to study the underlying cause of breast cancer (which may originate in the stem cells themselves), to see how they are different from other types of solid tumour cancers and to quickly and safely test drugs that might help curb the spread of the disease.

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Blood Disorders

There are many kinds of blood disorders – too many to review in detail, but broadly, they can be caused by congenital or inborn deficiencies (for example, sickle cell anemia), immune deficiencies (SCID), autoimmune mechanisms (immune thrombocytopenia purpura), and cancer (leukemia, myeloma, lymphoma). The most common therapies include blood transfusions, drug therapies and hematopoietic (blood) stem cell transplants.

Hematopoietic stem cell transplants (HSCT) were first performed in the 1950s and are the most common form of stem cell therapy used today, and are routinely used to treat a variety of different blood-based cancers including multiple myeloma, leukemia and lymphoma, and other blood disorders, including anemia, thalassemia and severe combined immune deficiency. However, HSCT is an aggressive therapy, not without risks, and not all patients are cured. Research is continuing to improve the outcomes of HSCT and make it available for a wider range of patients.

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Autism

Autism, also referred to as Autism Spectrum Disorder (ASD), describes a spectrum of neurodevelopmental disorders most often diagnosed in young children and includes classic autism, Asperger’s disorder, childhood disintegrative disorder, and pervasive developmental disorder. Each of the autism spectrum disorders affects brain development and impairs or alters normal communication, social and behavioral skills. There is no cure for ASD, which affects boys more than girls, and testing presents a number of challenges, however, early identification in the first 36 months allows interventions that can mitigate ongoing symptoms.

Currently there are no Health Canada or FDA approved stem cell treatments available for any of the disorders on the autism spectrum. Because so little is known about what causes autism spectrum disorder, a clinical stem cell treatment may well be years or decades away. The focus of research at present is to use stem cells to create models of ASD in order to study how it develops and what possible treatments might be beneficial in the future. Scientists are also investigating how the immune system and inflammation are involved in the development of ASD, and assessing whether stem cells could help play an anti-inflammatory role by modulating the immune system in patients.

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Arthritis

Over 100 conditions fall under the term arthritis, with varying causes but similar symptoms: chronic pain in the joints, muscles and bones, stiffness and swelling. Disability can be mild or crippling. Arthritis can be degenerative, as with osteoarthritis, which is caused by wear and tear on the joints due to genetic and/or mechanical stresses or injury. Arthritis can also be inflammatory, as with rheumatoid arthritis, where the cause is an autoimmune attack on the lining of the joints. There is no cure for arthritis and current treatments are focused on reducing pain and minimize the loss of function.

Currently there are no Health Canada or FDA approved stem cell treatments available for arthritis. However, research looks very promising and some early phase clinical trials have emerged. Currently, most research is focused on using stem cells to increase the body’s ability to regenerate cartilage, on methods to grow and graft replacement joints, and on identifying stem cells that can boost our resistance to inflammation.

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ALS

ALS, also known as motor neurone disease or Lou Gehrig’s disease, is a fatal disease that attacks a particular type of nerve cell (motor neurons) in the brain and spinal column. ALS causes the motor neurons to slowly degrade and waste away, impairing their ability to transmit the important signals that control movement, eating, speaking, and eventually, breathing. There is no cure for ALS, and researchers agree that both genetic and environmental factors contribute to the onset of the disease.

Currently there are no Health Canada or FDA approved stem cell treatments available for ALS. There is still much that is not known about the causes of ALS and a clinical stem cell treatment may well be years or decades away. However, researchers are using various types of stem cells to learn more about ALS and to see if the regenerative capacity of stem cells can repair or replace the damaged motor neurons, in the hopes of discovering a possible treatment.

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