Toward Treatments

An authoritative resource for stem cell research and development.

Every day, stem cells are redefining what may be possible to treat and potentially cure diseases. It’s exciting, but it can also be confusing. What treatments are available now? What breakthroughs should we expect soon?

  • ALS

    Are there stem cell treatments for ALS?

    While no stem cell therapy currently has Health Canada or FDA approval for treatment of ALS, the Canadian Stem Cell Foundation and many others are working to turn stem cell research into treatments as quickly and safely as possible.

    What are scientists hoping stem cells can do?

    Stem cells hold the potential of replenishing the supply of motor neurons destroyed by ALS and the cells that protect them. Scientists are also looking at ways to stimulate the body's own stem cells to produce new motor neurons.

    Are clinical trials currently underway?

    While stem cell research for ALS is moving along a number of different routes, only a few have been successful enough to warrant Phase 1 and 2 clinical trials testing the safety and tolerability of using stem cells in patients.

    When might a stem cell therapy be available?

    While the outlook is promising, understanding what causes ALS to prevent it from occurring likely will take many years. Meanwhile, scientists are studying how stem cells – with their regenerative capacity, flexibility to grow into hundreds of types of cells, and ability to make factors that protect cells – could be deployed to treat this devastating disease.

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  • Arthritis

    Are there stem cell treatments for arthritis?

    While no stem cell therapy currently has Health Canada or FDA approval for the treatment of arthritis, the Canadian Stem Cell Foundation and many others are working to turn stem cell research into treatments as quickly and safely as possible.

    How can stem cells help?

    Stem cells can make cells called chondrocytes that in turn make cartilage, the thin tissue layer that covers joints and eases friction. Scientists are trying to find ways to direct stem cells to make patient-derived chondrocytes for transplanting.

    Are clinical trials currently underway?

    While the use of stem cells to create transplantable chondrocytes is still in animal testing stage, researchers are conducting early-stage clinical trials to evaluate the use of mesenchymal stem/stromal cells as anti-inflammatories to support the regeneration of cartilage in affected joints. The results from these early phase clinical trials are encouraging.

    When might a stem cell therapy be available?

    Stem cells therapies for arthritis are still years away, but the wealth of information now being generated in laboratories around the globe will help with the transition from basic research to clinical application.

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  • Autism

    Are there stem cell treatments for autism?

    While no stem cell therapy currently has Health Canada or U.S. Food and Drug Administration approval for treatment of autism, the Canadian Stem Cell Foundation and many others are working to turn stem cell research into treatments as quickly and safely as possible.

    What are scientists hoping stem cells can do?

    Scientists are using stem cells to create models of autism for the purpose of testing the effects of possible new drugs. They are creating neurons for use as laboratory models using induced pluripotent stem (iPS) cells made from the skin of people with autism.

    Are clinical trials currently underway?

    Worldwide, there are few clinical trials currently underway to treat autism. For the most part, researchers are trying to get a better understanding of the mechanisms underlying the function of stem cells and the biology of autism.

    When might a stem cell therapy be available?

    A stem cell therapy for autism could be several years away. It will take considerable time for scientists to create better models in which to test various stem cells and drug therapies before clinical trials can go ahead.

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  • Blood Disorders

    Blood Disorders

    Are there stem cell treatments for blood disorders?

    Yes. Hematopoietic stem cell transplants from bone marrow, umbilical cord and peripheral blood are approved by Health Canada and the FDA to help 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 or SCID.

    How can stem cells help?

    Because hematopoietic stem cells normally make all the red and white blood cells that populate the bone marrow and blood, they are the perfect tool for restoring blood components in patients who have blood deficiencies. For example, scientists have learned that transplanting hematopoietic stem cells into a patient who has undergone chemotherapy or radiation can supply new, healthy red and white blood cells. Scientists are also hoping that basic research on cancer stem cells will shed light on cancer initiation and relapse, and that this knowledge will lead to the development of future therapies targeting blood-related cancers.

    Are clinical trials currently underway?

    Many clinical trials have already proven that hematopoietic stem cell transplants can play an important role for helping to treat blood disorders. Today, there are literally thousands of new clinical trials underway to explore how to improve hematopoietic stem cell transplantation, how best to combine it with other types of therapies, and which sources of hematopoietic stem cells will do the best job for patients with blood cancers and other blood disorders.

    When might a stem cell therapy be available?

    The road to finding new stem cell therapies for blood disorders is paved with many challenges that will take time to overcome. But the wealth of information generated from labs around the globe is converging to help with the transition from basic research to the clinic. The results are very promising and in time may point to new stem cell therapies that not only cure blood disorders but also target disease relapse.

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  • Breast Cancer

    Breast Cancer

    Are there stem cell treatments for breast cancer?

    While no stem cell therapy currently has Health Canada or FDA approval for the treatment of breast cancer, the Canadian Stem Cell Foundation and many others are working to turn stem cell research into treatments as quickly and safely as possible.

    What are scientists hoping stem cells can do?

    Scientists believe cancer is propagated by a small subset of cells with stem cell properties – and that these cancer stem cells must be eliminated to cure the disease. Researchers are working to identify and isolate cancer stem cells to detect breast cancer earlier, predict its prognosis, and provide drug therapy targets.

    Are clinical trials currently underway?

    Early clinical trials are using new ways to track cells and DNA in breast cancer patients before surgery, at the time of removing the cancer, and for years afterwards. Other studies are testing whether cancer stem cell biomarkers can predict patients' response to treatments.

    When might a stem cell therapy be available?

    Finding therapies presents challenges that will take time to overcome, but the wealth of information about breast cancer stem cells now being generated in laboratories around the globe will help with the transition from basic research to clinical application.

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  • Cerebral Palsy

    Cerebral Palsy

    Are there stem cell treatments for cerebral palsy?

    While no stem cell therapy currently has Health Canada or FDA approval for the treatment of cerebral palsy, the Canadian Stem Cell Foundation and many others are working to turn stem cell research into treatments as quickly and safely as possible.

    How can stem cells help?

    Scientists are hoping to mobilize stem cells into replacing or repairing the central nervous system cells that have been damaged by cerebral palsy.

    Are clinical trials currently underway?

    Researchers are conducting very early stage clinical trials using mesenchymal stem/stromal cells and neural stem cells. There are also lessons to be learned from the results using these stem cells in trials for spinal cord injury, multiple sclerosis, eye diseases and Parkinson’s.

    When might a stem cell therapy be available?

    Stem cells therapies for cerrebral palsy are some time away, but the wealth of information now being generated in laboratories around the globe will help with the transition from basic research to clinical application.

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  • Crohn’s Disease

    Crohn’s Disease

    Are there stem cell treatments for Crohn's?

    While no stem cell therapy currently has Health Canada or FDA approval for the treatment of Crohn’s disease, the Canadian Stem Cell Foundation and many others are working to turn stem cell research into treatments as quickly and safely as possible.

    How can stem cells help?

    Stem cells may be able to help reverse some of the gastrointestinal damage caused by Crohn’s disease by making new cells, inhibiting inflammation, stimulating tissue repair and dampening the immune response.

    Are clinical trials currently underway?

    Early stage clinical trials using various kinds of stem cells are underway. Autologous (from the patient) transplants of mesenchymal stem/stromal cells have shown some benefits. Also, small-sample trials for patients with fistulas (abnormal connections between the intestinal tract and adjacent tissues and organs) suggest adipose (fat) derived stem cells might work in treating Crohn’s.

    When might a stem cell therapy be available?

    Stem cells therapies for Crohn’s disease are some time away, but the wealth of information now being generated in laboratories around the globe will help with the transition from basic research to clinical application.

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  • Diabetes

    Are there stem cell treatments for diabetes?

    While no stem cell therapy currently has Health Canada or FDA approval for treatment of diabetes, the Canadian Stem Cell Foundation and many others are working to turn stem cell research into treatments as quickly and safely as possible.

    What are scientists hoping stem cells can do?

    The application of stem cells to treat diabetes is very promising because diabetes can be traced to the loss of a single cell type – the insulin-producing beta cell. Researchers hope to use stem cells as beta-cell producing factories or as cells that support beta cell repair.

    Are clinical trials currently underway?

    Canadian researchers have already proven that transplanted pancreatic islet cells can produce beta cells, but transplant tissue is in short supply. Researchers are working to use stem cells as an endless source of cells for transplants. Phase 1 and 2 clinical trials are underway to test the safety of using adult stem cells to generate and/or repair beta cells.

    When might a stem cell therapy be available?

    Researchers believe that a stem cell therapy for diabetes is much closer than for other diseases because diabetes is well understood and can be traced to the loss of a single cell type. Many groups around the world are working on new stem cell approaches. The outlook is promising as researchers investigate the use of stem cells to free diabetic patients from the daily dependence on insulin injections.

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  • Eye Diseases

    Eye Diseases

    Are there stem cell treatments for eye diseases?

    While no stem cell therapy currently has Health Canada or FDA approval for treatment of eye diseases, the Canadian Stem Cell Foundation and many others are working to turn stem cell research into treatments as quickly and safely as possible.

    What are scientists hoping stem cells can do?

    Stem cells have the capacity to provide an endless supply of transplantable cells to repair the eye, solving the problem of the shortage of donor tissue for corneal transplants. The identification of retinal stem cells has kindled hope that retinal damage might be reversible.

    Are clinical trials currently underway?

    Early Phase 1 and 2 clinical trials are underway and some are having strong results, such as one study in which limbal stem cells were transplanted into 100 patients whose cloudy, acid-burned corneas became clear and transparent. Meanwhile, technological advances are paving the way for studies with retinal stem cells.

    When might a stem cell therapy be available?

    The outlook is promising: remarkable advances in pre-clinical and clinical studies are providing the basis for a realistic future in which stem cell therapies are a viable option for restoring damaged vision.

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  • GVHD

    Are there stem cell treatments for Graft-Versus-Host Disease (GVHD)?

    Yes. Health Canada and the FDA have granted conditional approval for Prochymal, a mesenchymal stem cell product, for the treatment of steroid-resistant and/or immunosuppressant-resistant GVHD in pediatric patients. Final approval depends on the outcome of clinical studies that are underway.

    How can stem cells help?

    For patients who need donated transplants for cancer or other blood disorders, two types of stem cells being used to rebuild the blood supply can also help control GVHD: cord blood stem cells and mesenchymal stromal/stem cells (MSCs). Banked cord blood means physicians can perform more transplants with less chance of GVHD. MSCs’ ability to control the immune system, inhibit inflammation, stimulate blood vessel formation, repair tissue and help stem cells to engraft makes them an attractive therapy for reducing GVHD.

    Are clinical trials currently underway?

    Yes. Researchers are investigating how to improve on stem cell therapies already in use, for example trying double cord blood transplants to ensure sufficient numbers of stem cells are transplanted. They are also trying to understand the wide range of results observed in clinical trials using MSCs to treat GVHD.

    When might a stem cell therapy be available?

    Researchers are considering the logistics of MSC transplantation in more depth to see if certain ways of selecting, handling, growing and freezing MSCs from lab to lab are affecting outcomes. Current discrepancies in results will take time to overcome before new therapies become available.

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  • Heart Failure

    Heart Failure

    Are there stem cell treatments for heart failure?

    While no stem cell therapy currently has Health Canada or FDA approval for treatment of heart failure, the Canadian Stem Cell Foundation and many others are working to turn stem cell research into treatments as quickly and safely as possible.

    What are scientists hoping stem cells can do?

    With donor hearts for transplant in short supply, researchers believe that stem cells could become a ready source of precursor cells to promote the growth of new heart tissue and blood vessels and restore lost function.

    Are clinical trials currently underway?

    Research into muscle stem cells, bone marrow stem cells, cardiac stem cells and endothelial progenitors has advanced rapidly to early Phase 1 and 2 clinical trials. Over 1,000 patients have been transplanted with bone marrow stem cells and the procedure has shown to be safe and modestly beneficial.

    When might a stem cell therapy be available?

    Research teams around the globe working to develop stem cell therapies for heart failure. As clinical trials progress, their results will inform and refine future research questions and bring the field closer to frontline treatments.

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  • Liver Failure

    Liver Failure

    Are there stem cell treatments for liver failure?

    While no stem cell therapy currently has Health Canada or FDA approval for liver failure treatments, the Canadian Stem Cell Foundation and many others are working to turn stem cell research into treatments as quickly and safely as possible.

    How can stem cells help?

    Researchers are hoping that stem cells will be able to churn out limitless numbers of cells called hepatocytes used for transplant therapy to solve the shortage of livers available for transplant. Researchers are also investigating which tissues will be the best sources of stem cells for treating various types of liver failure.

    Are clinical trials currently underway?

    The majority of early phase clinical trials are testing whether bone marrow stem cells can safely and effectively alleviate damage from liver cirrhosis, liver scarring due to injury, or liver cancer. There have been some cases of beneficial effects for up to one year in patients with end-stage liver disease. These patients received their own bone marrow stem cells that were collected in peripheral blood after they received growth factors. Researchers are also investigating whether transplanting particular populations of bone marrow stem cells could work better than whole bone marrow transplants.

    When might a stem cell therapy be available?

    Finding a stem cell therapy for liver disease will take some time. As researchers continue to grapple with the challenges, they are focused on using stem cells to regenerate the liver, reduce the levels of inflammation, and alleviate the shortage of organs for transplantation.

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  • Multiple Sclerosis

    Multiple Sclerosis

    Are there stem cell treatments for multiple sclerosis?

    While no stem cell therapy currently has Health Canada or FDA approval for treatment of multiple sclerosis, the Canadian Stem Cell Foundation and many others are working to turn stem cell research into treatments as quickly and safely as possible.

    What are scientists hoping stem cells can do?

    Stem cells have an unparalleled regenerative capacity and the flexibility to grow into hundreds of different types of cells including neurons and cells that make the myelin sheath that are damaged by MS. Stem cells also modulate the immune system – MS is an autoimmune disease – and can make factors that protect neurons.

    Are clinical trials currently underway?

    Although scientists have long envisioned using stem cells to replace myelin-forming cells lost during MS, it is becoming increasingly clear that these cells help regulate the immune system. Phase 1 and 2 clinical trials are testing whether a patient's own bone marrow stem cells can be used to make healthy immune cells and arrest the progression of MS.

    When might a stem cell therapy be available?

    While clinical trials that use patients' bone marrow cells to produce healthy immune cells are producing encouraging results, the side effects currently limit the use of this therapy to those with aggressive MS. However, trials are also being considered for patients with less severe forms of the disease. As we learn more about stem cells, researchers are hopeful that the knowledge gained can be translated into new therapies.

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  • Muscular Dystrophy

    Muscular Dystrophy

    Are there stem cell treatments for muscular dystrophy (MD)?

    While no stem cell therapy currently has Health Canada or FDA approval for treatment for Duchenne's muscular dystrophy, the Canadian Stem Cell Foundation and many others are working to turn stem cell research into treatments as quickly and safely as possible.

    What are scientists hoping stem cells can do?

    Because the disease is caused by the deficiency of one gene product, there is hope that stem cells can provide a treatment option. Scientists have identified stem cells in skeletal muscle, bone marrow, blood, fat, and other tissues to potentially regenerate muscle tissue or to help replace or repair the defective gene.

    Are clinical trials currently underway?

    Research is proceeding steadily but so far only a few results have been translated into early Phase 1 clinical trials, the majority of which are small trials to test the safety and effectiveness of using muscle precursors from bone marrow to treat boys with Duchenne's.

    When might a stem cell therapy be available?

    While the future is promising, many challenges remain. Currently, a major focus is on working out the variables to push research forward and provide new and safe opportunities for testing stem cells in clinical trials.

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  • Parkinson’s Disease

    Parkinson’s Disease

    Are there stem cell treatments for Parkinson’s?

    While no stem cell therapy currently has Health Canada or FDA approval for treatment of Parkinson's, the Canadian Stem Cell Foundation and many others are working to turn stem cell research into treatments as quickly and safely as possible.

    What are scientists hoping stem cells can do?

    Parkinson's is a good candidate for a stem cell therapy because it is linked to the loss of one specific kind of cell – the dopamine neuron. Researchers are investigating two main ways to use stem cells to treat Parkinson's: as factories to churn out dopamine neurons or as a source of growth factors to protect neurons from being damaged.

    Are clinical trials currently underway?

    Early stage clinical trials with small numbers of patients are mainly focused on the safety of stem cell therapies and assessing the protection provided to patients. Researchers are also watching results from clinical trials using stem cells for stroke, spinal cord injury, cancer and other degenerative diseases that may apply to Parkinson's.

    When might a stem cell therapy be available?

    It will take an international multi-disciplinary network of scientists, clinicians and laboratories working together to find safe and effective protocols for transplanting stem cells into the brain. These types of collaborations are now underway and researchers are hopeful they will provide the basis for testing future stem cell-based therapies.

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  • Solid Tumour Cancers

    Solid Tumour Cancers

    Are there stem cell treatments for solid tumour cancers?

    Yes. Hematopoietic stem cell transplants from bone marrow and umbilical cord blood have been approved by Health Canada and the FDA to help treat patients with solid tumours. For children, who cannot undergo radiation because it would damage their still- developing brains, hematopoietic stem cells transplantation following chemotherapy is now standard and can increase survival rates for cancers arising from brain, bone, and immune cell tumours.

    How can stem cells help?

    Hematopoietic stem cells are responsible for making all the red and white blood cells in the body. Much of the current research involves evaluating new ways to apply hematopoietic stem cells and finding other sources of these cells for transplants. Researchers are also investigating the role that cancer stem cells play in the formation and spread of tumours to develop therapies that target them and prevent relapse.

    Are clinical trials currently underway?

    Early phase clinical trials are underway to test the broadening applications of hematopoietic stem cells to treat different types of solid tumours including those found in cancer of the esophagus, stomach, colon/rectum, liver, pancreas, lung, breast, prostate, bone, and kidney.

    When might a stem cell therapy be available?

    The road to finding a stem cell therapy for solid tumours is paved with many challenges that will take time to overcome. But the wealth of information generated from labs around the globe is converging to help with the transition from basic research to the clinic. The results are very promising and in time may point to additional stem cell therapies for the wide variety of solid tumours and to prevent relapse from occurring.

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  • Spinal Cord Injury

    Spinal Cord Injury

    Are there stem cell treatments for spinal cord injury?

    While no stem cell therapy currently has Health Canada or FDA approval for treatment of spinal cord injury, the Canadian Stem Cell Foundation and many others are working to turn stem cell research into treatments as quickly and safely as possible.

    What are scientists hoping stem cells can do?

    With their regenerative capacity and the flexibility to grow into different cell types, stem cells could supply new cells and products to restore nerve function and prevent further spinal cord damage. The Canadian discovery of neural stem cells has sparked hope that these cells could help generate new nerve cells or guide in the regrowth of severed nerves.

    Are clinical trials currently underway?

    Early Phase 1 and 2 clinical trials are mainly testing the safety of transplanting adult stem cells into patients to treat spinal cord injury and the results should yield information about the viability of this kind of therapy.

    When might a stem cell therapy be available?

    Further clinical trials are needed to answer the question of whether stem cell therapy can improve nerve function. The wealth of information now being generated in labs around the globe is converging to help speed the the transition from basic research to clinical application.

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  • Stroke

    Are there stem cell treatments for stroke?

    While no stem cell therapy currently has Health Canada or FDA approval for treating strokes, the Canadian Stem Cell Foundation and many others are working to turn stem cell research into treatments as quickly and safely as possible

    How can stem cells help?

    Scientists are investigating ways to stimulate stem cells already present in the brain and other parts of the body to heal stroke-damaged brain tissue. A second strategy involves harvesting stem cells,purifying them, and partially or completely turning them into more mature cells for transplants to replace cells lost to stroke.

    Are clinical trials currently underway?

    Stem cell research for stroke has transitioned into very early clinical trials to prove the safety and effectiveness of potential treatments. The majority of trials are testing neural stem cells and different populations of stem cells from the bone marrow. For example, clinical trials are testing a growth factor that can push blood stem cells from the bone marrow into the bloodstream where they track to areas of the brain damaged by stroke.

    When might a stem cell therapy be available?

    Scientists expect it will take at least five to 10 years to determine if stem cell therapies can significantly improve the outcome for stroke victims. Meanwhile, they are investigating whether stem cell therapies can extend the window of opportunity for using drugs that salvage tissue damaged when blood flow to the brain is interrupted, limit the core zone of dying tissue, break apart clots, and minimize inflammation or damage to brain tissue that can occur when blood flow is restored.

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  • Wound Healing

    Wound Healing

    Are there stem cell treatments for wound healing?

    While no stem cell therapy currently has Health Canada or FDA approval for wound healing treatments, the Canadian Stem Cell Foundation and many others are working to turn stem cell research into treatments as quickly and safely as possible.

    What are scientists hoping stem cells can do?

    Stem cells reside in different layers of the skin, each contributing cells that maintain normal structure and function. With their regenerative capacity and ability to grow into different types of cells, including all the components of the skin, it is hoped they can produce a vast supply of skin cells for therapies.

    Are clinical trials currently underway?

    Researchers are trying to find ways to tap the built-in power of skin stem cells to repair wounds and are making headway at the pre-clinical stage. Small clinical trials have shown that mesenchymal stem cells from bone marrow help wounds close better and these results have opened the door to larger trials. A small pilot study that looked at using fat stem cells for chronic wounds caused by radiation treatment for cancer has shown positive results.

    When might a stem cell therapy be available?

    Work being done in labs around the globe is contributing to the transition of basic research to the clinic. The results are very promising and in time may point to stem cell therapies to addresses the ongoing issues of limited donor material for skin grafts, graft rejection and the need to regenerate the interior dermal skin layer crucial for optimal wound healing.

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