Information on Stem Cell Research: National Institute of Neurological Disorders and Stroke (NINDS)
02/20/2013 09:47 AM EST
The National Institutes of Health resource for stem cell research
Stem Cells and Diseases
The Promise of Stem Cells
Studying stem cells will help us understand how they transform into the dazzling array of specialized cells that make us what we are. Some of the most serious medical conditions, such as cancer and birth defects, are due to problems that occur somewhere in this process. A better understanding of normal cell development will allow us to understand and perhaps correct the errors that cause these medical conditions.
Another potential application of stem cells is making cells and tissues for medical therapies. Today, donated organs and tissues are often used to replace those that are diseased or destroyed. Unfortunately, the number of people needing a transplant far exceeds the number of organs available for transplantation. Pluripotent stem cells offer the possibility of a renewable source of replacement cells and tissues to treat a myriad of diseases, conditions, and disabilities including Parkinson's disease, amyotrophic lateral sclerosis, spinal cord injury, burns, heart disease, diabetes, and arthritis.
Clinical Trials for Stem Cell Therapies
Human embryonic stem cells:
Scientists have been able to do experiments with human embryonic stem cells (hESC) since 1998, when a group led by Dr. James Thomson at the University of Wisconsin developed a technique to isolate and grow the cells. Although hESCs are thought to offer potential cures and therapies for many devastating diseases, research using them is still in its basic stages.
The NIH funded its first basic research study on hESCs in 2002. Since that time, biotechnology companies have built upon those basic foundations to begin developing stem cell-based human therapies. There are currently two active clinical trials using cells derived from human embryonic stem cells, both being conducted by a biotechnology company called ACT. The company has laboratories in Marlborough, Massachusetts and corporate offices in Santa Monica, California. ACT has begun enrolling patients for Phase I (safety and tolerability) clinical trials of two hESC-derived stem cell products:
- The first ACT trial is testing the safety of hESC-derived retinal cells to treat patients with an eye disease called Stargardt's Macular Dystrophy (SMD).
- The second ACT trial is testing the safety of hESC-derived retinal cells to treat patients with age-related macular degeneration. In January, 2012, the investigators published a preliminary report on the first two patients treated with hESC-derived cells: http://www.ncbi.nlm.nih.gov/pubmed/22281388. A third patient was treated on April 20, 2012.
- A third clinical trial using hESC-derived cells was halted on November 14, 2011. The trial was being conducted by a biotechnology company called Geron, located in Menlo Park, California. Four patients with recent spinal cord injuries had been enrolled for its clinical trial of a hESC-derived therapy. The trial was testing the safety of using hESC-derived cells to achieve restoration of spinal cord function. Oligodendrocyte progenitor cells derived from hESCs were being injected directly into the lesion site of the patient's injured spinal cord. On November 14, Geron announced that it was discontinuing its stem cell programs to concentrate on cancer programs.
Bone marrow stem cells:
Bone marrow contains blood-forming stem cells (hematopoietic stem cells) that have been used for decades to treat blood cancers and other blood disorders. Umbilical cord blood is another source of hematopoietic stem cells that is being used in treatment. You can see a list of diseases that may currently be treated with hematopoietic stem cells at the website of the National Marrow Donor Program. You may also search for clinical trials testing "bone marrow stem cells" or "umbilical cord blood" on the ClinicalTrials.gov website.
Human spinal cord stem cells:
A biotechnology company called Neuralstem (corporate headquarters in Rockville, Maryland) is conducting a clinical trial testing the use of human spinal cord stem cells to treat Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s Disease. The company obtained FDA approval to conduct a Phase I trial (safety and tolerability study) and began enrolling patients in January 2010. Twelve participants have received lumbar transplants, and in March 2012, the second participant received an injection in the cervial region. Details about this trial are listed on the ClinicalTrials.gov website.
Human mesenchymal stem cells:
Osiris Therapeutics (Columbia, Maryland) is conducting three different Phase 2 clinical trials with a product from adult mesenchymal cells (called Prochymal). The three trials are for:
- protecting pancreatic beta islet cells in adults and children with newly diagnosed type 1 diabetes (in partnership with the Juvenile Diabetes Research Foundation),
- repair of heart tissue following a heart attack, and
- the repair of lung tissue in patients with chronic obstructive pulmonary disease (COPD).
Osiris is also conducting Phase 3 testing of Prochymal for acute graft versus host disease (GvHD) and Crohn's disease.
Adult Eye Stem Cells for Eye Disease or Damage:
The limbus is the marginal region of the cornea of the eye that contains stem cells. Stem cells from the limbus are called limbal stem cells, and they normally serve to replace cells to maintain the cornea. Limbal stem cells are being tested as possible treatments for human eye conditions. Currently, scientists are testing whether limbal stem cells can help repair damage to the cornea and whether they can help replace cells that are lacking due to limbal stem cell deficiency.
Participating in Clinical Trials
Scientists are testing the abilities of many different types of stem cells to treat certain diseases. You can search for clinical trials using stem cells (or other methods) to treat a specific disease at ClinicalTrials.gov.
Stem Cells and Diseases [Stem Cell Information]
Stem Cell Transplants
Stem cells are cells in the body that have the potential to turn into anything, such as a skin cell, a liver cell, a brain cell, or a blood cell. Stem cells that turn into blood cells are called hematopoietic stem cells. These cells are capable of developing into the three types of blood cells:
- red blood cells that carry oxygen
- white blood cells that fight infection
- platelets that help blood to clot
Hematopoietic stem cells can be found in bone marrow (the spongy tissue inside bones), the bloodstream, or the umbilical cord blood of newborn babies.
A stem cell transplant (sometimes called a bone marrow transplant) can replenish a child's supply of healthy hematopoietic stem cells after they have been depleted. It's used to treat a wide range of diseases, including cancers like leukemia, lymphoma, neuroblastoma, Wilms tumor, and certain testicular or ovarian cancers; blood disorders; immune system diseases; and bone marrow syndromes.
Transplanted hematopoietic stem cells are put into the bloodstream through an intravenous (IV) line, much like a blood transfusion. Once in the body, they can produce healthy new blood and immune system cells.
Types of Transplants
The two main types of stem cell transplants are autologous and allogeneic. The type of transplant needed will depend on the child's specific medical condition and the availability of a matching donor.
- Autologous hematopoietic stem cell transplant. With this type of transplant, patients act as their own donor. That is, a child who is about to undergo cancer treatment will have his or her own stem cells removed (harvested) and frozen for later use. After the child receives chemotherapy and/or radiation, the stem cells are thawed and put back into the child's body.
This procedure may be done once or many times, depending on the need. Sometimes doctors will use extra-high doses of chemotherapy during treatment (to kill as many cancer cells as possible) if they know a patient will be getting a stem cell transplant soon after. - Allogeneic hematopoietic stem cell transplant. With an allogeneic transplant, the stem cells come from a donor — often a sibling but sometimes another volunteer — whose cells are considered a "match" for the patient. The process of finding a match is called tissue typing (or HLA [human leukocyte antigen] typing). HLA is a protein on the surface of blood cells. Basically, the more "HLA markers" a child and the potential donor have in common, the greater the chance that the transplant will be successful.
Unlike with an autologous transplant, there is a risk of a child's body rejecting the donated cells. Sometimes, despite the donor being a good match, the transplant simply may not take. Other times, the donor cells can begin to make immune cells that attack the recipient's body. This condition is called graft-versus-host disease, and can be quite serious. Fortunately, most cases are successfully treated with steroids and other medications.
Sometimes, an upside of graft-versus-host disease is that the newly transplanted cells recognize the body's cancer cells as different or foreign, and actually work to fight them.
MORE ON THIS TOPIC
Information on Stem Cell Research
Research @ NINDS
Stem Cell
| Highlights |
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| Submit a hESC line for NIH review (9/21/09) NIH Opens Website for Human Embryonic Stem Cell Lines for Approval and Announces Members of Working Group |
| Resource Links |
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| Highlights of Stem Cell ResearchStem Cell Research at NIH |
| Contacts |
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| David F Owens, Ph.D. Program Director do47h@nih.gov |
Introduction
Stem Cells are unique in that they have the potential to develop into many different cell types in the body, including brain cells, but they also retain the ability to produce more stem cells, a process termed self renewal. There are multiple types of stem cell, such as embryonic stem (ES) cells, induced pluripotent stem (iPS) cells, and adult or somatic stem cells. While various stem cells can share similar properties there are differences as well. For example, ES cells are able to differentiate into any type of cell, whereas adult stem cells are more restricted in their potential. The promise of all stem cells for use in future therapies is exciting, but significant technical hurdles remain that will only be overcome through years of intensive research.
The NINDS supports a diverse array of research on almost all stem cells, from studies of the basic biology of stem cells in the developing and adult mammalian brain to studies focusing on nervous system disorders such as ALS or spinal cord injury. For example, investigators are looking at how ES cells can be used to derive dopamine-producing neurons that might alleviate symptoms in patients with Parkinson’s disease or how somatic stem cells can generate myelin producing oligodendrocytes for remyelination following acute and chronic brain injury. Although there is much promise for using stem cells to treat neurological diseases in humans, there is much work to be done before stem cell-based therapies are ready for the clinic.
The NIH Stem Cell Information Web page provides additional information about stem cell research at NIH. Also, see MedlinePlus for more health information regarding stem cells.
To learn more about investigational therapies, including stem cells, one can search the National Institutes of Health (NIH) online clinical trials database, which has information about federally and privately funded clinical research studies on a wide range of diseases and conditions. You can access this database at ClinicalTrials.gov to learn about the location of research studies in need of participants, as well as their purpose and criteria for patient participation. The NIH also maintains a clinical research website that has additional information and can be found here: NIH Clinical Research Trials and You
NINDS Repository
The NINDS also supports a repository that offers human induced pluripotent stem cell (iPSC) lines for research on neurological disorders. A list of available cell lines can be found here: Human Induced Pluripotent Stem Cells
NINDS Stem Cell Research on CampusThe Intramural Research Program of NINDS is one of the largest neuroscience research centers in the world. Investigators in the NINDS intramural program conduct research in the basic, translational, and clinical neurosciences. Their specific interests cover a broad range of neuroscience research including stem cell biology. Listings of NINDS intramural researchers by laboratory affiliation and research areas are available online.
NIH Policy and ImplementationThe Director of the NINDS, Dr. Story Landis is the Chair of the NIH Stem Cell Task Force, which was created to enable and accelerate the pace of stem cell research and to seek the advice of scientific leaders in stem cell research. For comprehensive information on NIH policies related to stem cell research, visit the NIH Stem Cell Information web page.
NIH Center for Regenerative Medicine (NIH CRM)NIH CRM is a community resource that works to provide the infrastructure to support and accelerate the clinical translation of stem cell-based technologies, and to develop widely available resources to be used as standards in stem cell research. The Center provides services and information to both the intramural and extramural NIH communities that facilitate the use of stem cell technologies for therapeutic purposes and for screening efforts. Further information about NIH CRM can be found here: NIH Center for Regenerative Medicine
Funding OpportunitiesNINDS supports a wide array of stem cell research, both basic and disease-related. Funding mechanisms supported by NINDS can be found here: Funding Mechanisms
Additionally, those interested in targeted funding solicitations can search the NIH Guide for Grants and Contracts. One can do key word searches for entries such as “neurological disease” and “stem cell” or “regenerative medicine.” A link to the NIH Guide can be found here: NIH Guide for Grants and Contracts
NINDS Contact InformationDavid Owens, Ph.D.
Program Director
do47h@nih.gov
Phone: (301) 496-1447
Last updated February 12, 2013
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