domingo, 24 de febrero de 2013

Stem Cell Transplants

Stem Cell Transplants

Information on Stem Cell Research: National Institute of Neurological Disorders and Stroke (NINDS)

02/20/2013 09:47 AM EST

Source: Nemours Foundation
Related MedlinePlus Page: Stem Cells

02/20/2013 09:47 AM EST

Stem Cells and Diseases [Stem Cell Information]

Source: National Institutes of Health - NIH
Related MedlinePlus Page: Stem Cells

02/20/2013 09:47 AM EST

Stem Cell Transplants

Source: Nemours Foundation
Related MedlinePlus Page: Stem Cells

Stem Cell Transplants



  Stem Cell Information

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:

  1. 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).

  2. 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: third patient was treated on April 20, 2012.

  3. 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 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 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:

  1. protecting pancreatic beta islet cells in adults and children with newly diagnosed type 1 diabetes (in partnership with the Juvenile Diabetes Research Foundation),

  2. repair of heart tissue following a heart attack, and

  3. 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
Stem Cells and Diseases [Stem Cell Information]

Stem Cell Transplants

Stem Cell Transplants




Information on Stem Cell Research

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 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
Phone: (301) 496-1447

Last updated February 12, 2013

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