Advantages of Adult Stem Cells for Sickle Cell Disease

Red Blood CellsSickle cell disease is a disabling and painful genetic blood disorder that primarily affects African-Americans. Fortunately, researchers at John Hopkins have found that there are many advantages of adult stem cells being used as a treatment for the disease.

The stem cell treatment corrects the alteration that causes sickle cell disease. The corrected stem cells were persuaded into immature red blood cells in a test tube and then turned into a regular version of the gene.

According to the stem cell research team, the work conducted in the laboratory is years away from being used to treat clinical patients. However, the advantages of adult stem cells therapies are that they provide tools for the development of gene therapies for sickle cell disease as well as a number of other blood disorders.

Researchers believe that they are yet another step in the right direction toward finding a realistic cure or a possible long-term option for treating patients with sickle cell disease. The disease is the result of a single letter change in the human gene responsible for adult hemoglobin, which is the primary protein in red blood cells that are necessary for the transporting of oxygen throughout the body.

When a person inherits two copies of the genetic alteration, which means they get one from each of their parents, the red blood cells take on a sickle shape as opposed to the usual round shape of everyone else’s red blood cells. Because the cells are misshaped, they clog the blood vessels, leading to fatigue, pain, organ damage, infection and even premature death.

Even though painkillers and other drugs are available to treat the symptoms of sickle cell disease, the only known cure is a bone marrow transplant. However, the majority of patients with sickle cell disease are African-American and only a few African-Americans are listed in the bone marrow registry. Therefore, it is difficult to find suitable donors.

Researchers at the John Hopkins Hospital isolated a single patient’s bone marrow cells and reprogrammed theses adult stem cells to act like embryonic stem cells. They then used a normal copy of the hemoglobin gene to replace the one that is defective through the advantages of adult stem cells engineering technologies.

The researchers used three hundred different samples of iPS cells to sequence the DNA to identify the ones that had the right copies of the hemoglobin gene and they only found four. However, three of the iPS cells did not pass assemblage in the following tests.

According to the professor of medicine and associate director of basic research for the Division of Hematology and a member of the John Hopkins Institute for Cell Engineering, Linzhao Cheng, Ph.D., “The beauty of iPS cells is that we can grow a lot of them and then coax them into becoming cells of any kind, including red blood cells.”

Accelerating Bone Growth Through the Use of Stem Cells

broken boneAnytime someone breaks a bone in their body, it is understood that they will be in a cast for a number of weeks while the injury heals. However, technology has been discovered with the use of stem cells that may actually cut the healing time for broken bones in half. Chemistry professor Qian Wang of the University of South Carolina in the United States has made enticing progress toward a faster recovery.

According to Wang and his co-workers, surfaces that have been coated with bionanoparticles have the potential of considerably accelerating the earlier phases of bone growth. In part, the coatings are developed on the basis of the Tobacco mosaic virus following genetic modification. The length of time it took stem cells to convert to bone nodules was reduced from two weeks to only two days.

The key to healing or growing bone at a faster rate with the use of stem cells is coaxing the natural process to speed up the pace. Wang said, “If you break a rib, or a finder, the healing is automatic.” He continued, “You need to get the bone aligned to be sure it works as well as possible, but then nature takes over.”

Healing is a very natural process. The body is continuously circulating and generating undifferentiated cells that can be converted to other tissues, such as muscle, skin or bone, depending on what is required by the body. The conversion of stem cells begins with external cues.

When it comes to healing bone, the body recognizes the injury on a cellular level and starts to convert stem cells to bone cells at the point of the break, thus bonding the break into a single unit. The natural process is incredibly slow so the fracture has time to set properly. However, the wait is at the very least inconvenient for the patient, and detrimental in some cases.

According to Wang, “With a broken femur, a leg, you can be really incapacitated for a long time.” He said, “In cases like that, they sometimes inject a protein-based drug, BMP-2, which is very effective in speeding up the healing process. Unfortunately, it’s very expensive and can also have some side effects.”

Searching for a better alternative four years ago, Wang and his colleagues discovered that plant viruses were accelerants of bone growth. Initially, they intended for the viruses to be controls. Glass surfaces were coated uniformly with the Tobacco mosaic virus and the Turnip yellow mosaic virus as a starting point for the study of other possible variants.

Surprisingly, the researchers discovered that the coatings reduced the time it took bone nodules to grow from stem cells. Ever since, they have been working to refine their approach to defining exactly how the use of stem cells with the plant virus enhances bone growth.


Stem Cell Transplantation May Improve Emphysema Damage

Emphysema LungResearchers have discovered that adult stem cells taken from a patient’s, or in this case the sheep’s, own body and used for stem cell transplantation into thirteen adult sheep with emphysema provided evidence of tissue regeneration with an increase in blood perfusion as well as additional cellular matrix content following the transplant. The researchers working on the project determined that this approach, which was published in Cell Transplantation, just might represent a useful alternative to traditional stem cell therapy for the treatment of the condition.

According to the study author, Edward P. Ingenito of Brigham and Women’s Hospital Division of Pulmonary and Critical Care Medicine, “Mensenchymal stem cells are considered for transplantation because they are readily available, highly proliferative and display multi-lineage potential.” He also said,” Although MSCs have been isolated from various adult tissues—including fat, liver and lung tissues—cells derived from bone marrow (BM) have therapeutic utility and may be useful in treating advanced lung diseases, such as emphysema.”

On the other hand, according to researchers who have conducted previous studies on stem cell transplantation, most of which delivering the transplanted stem cells via an intravenous method have indicated that bone marrow Mensenchymal stem cells have only been slightly successful in the treatment of lung disease. However, the therapeutic responses for such research have been limited to experiments on animal models with inflammatory lung ailments, like acute lung injury and asthma.

In an attempt to find answers to the numerous questions about the usefulness of bone marrow Mensenchymal stem cell transplantation for the treatment of advanced emphysema, which is a disease that is characterized by the loss of structural integrity and the destruction of tissue in the lungs, researchers have isolated proliferative adult stem cells from functional tissue in the lungs. They delivered the tissue through an endoscopic system combined with the use of a scaffold, which compromised the natural extracellular components.

Ingenito explained, “LMSCs display efficient retention in the lung when delivered endobronchially and have regenerative capacity through expression of basement membrane proteins and growth factors.” He continued, “However, despite the use of autologous cells, only a fraction of the LMSCs delivered to the lungs alveolar compartment appeared to engraft. Cell death likely occurred because of the failure of LMSCs to home to and bind within their niche, perhaps because the niche was modified by inflammation or fibrosis. These cells are attachment-dependent and failure to attach results in cell death.”

However, the results of the researchers’ experiments did provide evidence that the LMSCs were proficient in contributing to the remodeling of the lung. The stem cell transplantation led to functional improvement in the lungs, as opposed to scarring almost a month following the procedure.

In conclusion, Ingenito said, “Although the data is from a small number of animals, results show that autologous LMSC therapy using endoscopic delivery and a biocompatible scaffold to promote engraftment is associated with tissue remodeling and increased perfusion, without scarring or inflammation.” He went on to say, “However, questions concerning mechanism of action and pattern of physiological response remain topics for future investigation.”

Use of Adult Stem Cells for Stroke Recovery

The use of adult stem cells to aid in the recovery of stroke victims by injecting the cells into their brain is becoming a rather hot topic these days. Many people are wondering if it is safe and if it is something that really can be done. Well, researchers at the University of Pittsburgh are looking for the answers.

Dr. Lawrence WechslerAccording to Dr. Lawrence Wechsler, a UPMC neurologist, “Because these are cells that have not been injected into the brain before, we need to know whether it is safe to do so.”

So far, two people at UPMC have been injected with stem cells that were derived from the bone marrow of adult donors. One of these patients is named Roger Hill, who suffered a stroke in August of 2009. When he woke that morning, he noticed he could not see but half of the world and shortly after, he lost the use of his left side. He said, “Something happened with my left leg. I fell down. I couldn’t feel my left knee.”

The problem with stroke victims lies in the brain. Most commonly, a stroke occurs due to a blocked artery. The lack of blood and oxygen causes a portion of the brain to die. For this reason, stokes are among the leading causes of disability and death.

Most of the focus on stroke research has been on prevention and treating patients within the initial few hours after a stroke occurs. Other than physical therapy, not much else can be done to treat someone who has suffered a stroke. That is what doctors thought before the use of adult stem cells came to mind as a potential treatment.

According to Hill, “They said they would cut a flap in your scalp, pull it away, drill a hole through your skull, put a probe in my brain, and then run a needle down in there and inject stem cells in eight different locations.”

At the time being, the correct dose and technique to produce a clinical benefit is not known. This is something that will come along further down the line as the use of adult stem cells to aid stroke recovery is explored.

The first six patients in the study were given 2.5 million stem cells, while the next six were given 5 million and the remaining six patients were given 10 million stem cells. These patients will be monitored on a regular basis for the next two years.

Dr. Wechsler said, “Not everyone is interested in trying something as new as the injection of these kinds of cells.” He also said, “There’s no way at this point for us to distinguish the effect of the cell injection from the natural course of recovery from a stroke.”





New Stem Cell Treatments for Eye Patients

Recently, seven patients were treated by an American retinal surgeon with a groundbreaking procedure, which he developed for the administration of stem cell treatments for eye disease. A consultancy that is known for their help is getting patients access to stem cell providers in Europe and the United States, MD Stem Cells, recently announced, “Our affiliate retinal surgeon traveled to Vienna, Austria to provide the new treatment to an international group of patients.” The organization’s senior consultant, Dr. Steven Levy, said, “We are extremely pleased that this new procedure is now available and being provided on a routine basis.”

Patients came from various parts of the world to receive the stem cell treatments, including Austria, Romania and the United States. These patients, who ranged in age from two to eighty-seven, were all suffering from some type of eye disease. These diseases included:

  • Age Related Macular Degeneration, or AMD
  • Myopic Macular Degeneration
  • Bulls Eye Retinopathy, which is a genetic retinal disease
  • Retinitis Pigmentosa
  • Optic Nerve Disease

All of the patients that received the stem cell treatments did well during the procedure and were quite pleased with the results.

For these stem cell treatments, adult stem cells were taken from the patients’ own bodies, which does not pose the risk of rejection associated with the controversial use of embryonic stem cells. Adult stem cells are usually derived from bone marrow because of the high concentration of cells that are useful in the treatment of a variety of diseases. However, sometimes stem cells are taken directly from the patient’s blood. Stem cell treatments have been proven in recent years to aid in the recovery of a number of different medical conditions, including:

  • Diabetes
  • Renal failure
  • Stroke
  • Cerebral palsy
  • Autism
  • Parkinson’s disease
  • Multiple sclerosis
  • COPD
  • Heart disease
  • Liver disease

Before this study, the stem cells were injected behind a patient’s eye in order to treat their ophthalmic issues. However, the exact location and the distance from the target were unknown. This led to variability in response to the treatments.

Dr. Steven Levy, MDAccording to Dr. Levy, “Now with the new technique the stem cells can be placed precisely adjacent to the eye in a very safe manner.” He also said, “Our surgeon notes that his approach avoids the risks of surgery yet allows him to precisely place the stem cells adjacent to the diseased part of the eye or the optic nerve for best effect.”

Dr. Levy said, “We’re fortunate to be working with such an experienced retinal ophthalmologist. He completed two retinal fellowships at Harvard, MIT and New England Deaconess Hospital. His strong training and prior experience with stem cell treatments inspires confidence in patients and reassures physician who may be referring or supporting their patients.”