How stem cells may help children battling birth injuries

From time to time we invite patients or patient advocates to post a guest blog on the Stem Cellar. Today we are featuring Brigitta Burguess, a mother and writer from Michigan, who focuses on pregnancy, parenting, and children with disabilities. Brigitta writes for the HIE Help Center, a website that offers information and supportive resources for families of children with disabilities.

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Because stem cells are the building blocks of the immune system, they possess the ability to develop into other types of cells. You can use stem cells to help repair tissues, organs, and blood vessels, and even treat a host of different diseases. This is done through stem cell harvesting and stem cell therapy. In stem cell therapy, stem cells are injected into injured tissues in the hopes of replacing damaged tissue and preserving existing tissues.

Cord Blood

Every part of the human body contains stem cells. However, many areas of the body do not contain enough stem cells to make harvesting them worthwhile. Cord blood, the leftover blood collected from a baby’s umbilical cord or a mother’s placenta after birth, is especially beneficial because:

  • It provides a rich source of stem cells that can be changed into other types of cells and help to maintain and repair tissues
  • Its stem cells are immature and have not developed the ability to attack foreign cells, which makes them perfect for transplant
  • Its stem cells differ from embryonic stem cells in that they are considered adult stem cells and do not require the destruction of an embryo to harvest
  • It can be used to treat blood disorders, immune deficiencies, and certain cancers
  • Storing cord blood can help family and community members receive gene therapy treatment for the aforementioned conditions and diseases

The Applications of Stem Cell Therapy for Kids

Today, over 2,000 total cord blood stem cell transplants are performed annually, with the total number of cord blood banks worldwide reaching over 150. The innovations in stem cell therapy have made waves over the past four decades. Today, more than 80 difference diseases are being treated with cord blood stem cells.

In 2012, many clinical trials revealed that cord blood transplants were an effective treatment for cerebral palsy. Researchers also believe that cord blood stem cells have great potential in treating the neonatal brain injuries such as hypoxic-ischemic encephalopathy (HIE). As of right now, there is no indication that stem cell therapy can cure these conditions, but there is some evidence that it can lessen the severity of symptoms.

It is important to note that there is thus far no cure for hypoxic-ischemic encephalopathy (HIE) and resulting motor, cognitive, and/or intellectual disorders. Stem cell therapy seeks to limit the damage caused by HIE and reduce the severity of disabilities caused by HIE, but it is not a cure.

Because stem cell therapy is still in clinical trials, parents should think twice before going down this untested path, as no formal guidelines about administration protocol, dosages, safety, or treatment timeline have yet been established. Clinical trials are important for ensuring that treatments are safe and effective – unregulated treatments bear significant risk.

To learn more about stem cell therapy trials for hypoxic-ischemic encephalopathy, please visit the National Institute of Health’s (NIH) Clinical Trial Recruitment Center.

 

New stem cell approach targeting deadly blood cancers

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Every four minutes someone in the US is diagnosed with a blood cancer. It might be lymphoma or leukemia, myeloma or myelodysplastic syndromes (MDS). While we have made great strides in treating some of these over the years, we still have a long way to go. Need proof? Well, every nine minutes someone in the US dies from a blood cancer.

Because of that need, the CIRM Board last week approved $3.5 million to help fund the search for a more effective, more efficient way to treat people suffering from blood cancer.

The Board funded a program by Angiocrine Biosciences, a San Diego-based company that is developing a new method for transplanting cord blood into patients.

Now cord blood transplants have been around for decades and they can be very effective. But they can also cause serious, even life-threatening complications. And they have limitations. For example some cord blood units are small and don’t have as many stem cells as the doctors would like. As a result, patients may need to spend longer in the hospital recovering from the procedure, putting them at increased risk of viral infections or pneumonia. Alternatively, doctors could use more than one cord blood unit for each transplant and while that seems to be an effective alternative, some studies suggest it can also carry an increased risk for serious complications such as Graft-versus-host disease (GVHD) where the newly transplanted cells attack the patient’s body.

To get around these issues, Angiocrine is developing a product called AB-110. This takes stem cells from cord blood, uses a specialized manufacturing facility to expand their numbers and then mixes them with genetically modified endothelial cells, the kind of cell that forms the lining of blood vessels.

It’s hoped that AB-110 will reduce the complications and increase the chances the transplanted cells will successfully engraft, meaning they start growing and creating new, healthy, blood cells.

In a news release CIRM’s President and CEO, C. Randal Mills, PhD, says this program fits in perfectly with our mission of accelerating stem cell treatments to patients with unmet medical needs:

“This project aims to do precisely that, speeding up the body’s ability to create new white blood cells and platelets – both essential qualities when treating deadly diseases like leukemia and lymphoma. Under CIRM 2.0, we are trying to create a pipeline of products that move out of the lab and into clinical trials in people, and we’re hopeful this program will demonstrate it’s potential and get approval from the Food and Drug Administration (FDA) to begin a clinical trial.”

Everyone at Angiocrine and CIRM will work as hard as we can to move this research toward a clinical trial as fast as we can. But in the meantime there are tens of thousands of critically ill people in desperate need of a life-saving transplant.

One way of helping those in need is for new parents to donate their child’s umbilical cord blood to the state’s umbilical cord blood collection program. This is a safe procedure that doesn’t harm the baby but could save someone’s life.

The cord blood program is housed at the UC Davis Institute for Regenerative Cures – a facility CIRM helped build and where we fund many great projects. This program is particularly important because it collects and stores cord blood units that reflect the state’s diverse communities, and that are available to all those in need of a transplant.

The bank also is a rich source of cord blood units for research, particularly for stem cell research, which will hopefully lead to even more effective therapies in the future.