Severe and prolonged neutropenia is a common consequence of cancer chemotherapy and is associated with an increased risk of severe infections. Transfusion of donor neutrophils is a viable option to combat this, but obtaining matched donors is a dilemma. There are pharmacologic interventions aimed at shortening the duration of neutropenia and combatting infection; however, infection risk remains high due to a lack of response to these treatments in many patients.

Natural killer (NK) cells are immune cells that can be used as a form of immunotherapy and are particularly effective in the treatment of acute myeloid leukemia (AML). NK cells can be obtained from peripheral or cord blood, but with low yields. This limits their use for patients that need multiple treatments. They also have a short survival time and do not proliferate or remain viable after injection into patients. The availability of NK cells for therapy can be enhanced by stimulating their production from hematopoietic (CD 34+) stem cells.

Dilated cardiomyopathy occurs when the heart muscle stretches and becomes weakened, leading to less efficient pumping of the blood throughout the body. It is the most common form of non-ischemic cardiomyopathy (not due to coronary artery disease). Dilated cardiomyopathy may be associated with damage to the heart muscle from a variety of causes such as inflammation, infections, and toxic substances. Diastolic dysfunction seen in patients with dilated cardiomyopathy refers to insufficient relaxation and filling of the ventricles during the second part of the heart-pumping phase (leading to increased pressure in the ventricles).

Sickle cell disease refers to a group of genetic blood disorders mainly affecting people of African, Mediterranean, Middle Eastern, and Caribbean decent. Those with sickle cell disease carry the abnormal hemoglobin S in their red blood cells due to a mutation of the β-globin gene. Instead of having flexible, disc-shaped red blood cells, affected individuals have characteristic sickle-shaped, more rigid cells that cause disturbances in blood flow and tissue oxygenation.

The leading cause of death for U.S. citizens is cardiovascular disease, which affects nearly a third of the U.S. population. There is an active and dedicated search for effective therapies to address this serious medical issue. The use of immunotherapy approaches for a number of diseases and conditions is continuously gaining momentum. Included in this is the use of CD34+ stem cells from a patients’ own blood (autologously derived) to treat cardiovascular disease. CD34+ is derived from bone marrow and other tissue types. However, CD34+ stems cells are more widely known for their hematopoietic origin.

Hematopoietic progenitor and stem cells, which express CD34 antigen, are widely used clinically in the treatment of a number of conditions and diseases such as vascular disease, spinal cord injury, multiple myeloma, and more. However, emerging research data indicates that CD34 stems cells are not exclusively hematopoietic. CD34 antigen is expressed on a number of other cell types including keratocytes, multipotent mesenchymal stromal cells, and interstitial dendritic cells, among others.

Incredible advances continue to occur with the application of cell therapies. Stem cell research is ongoing in a number of areas including for cancer treatment, neurological conditions, spinal cord injury, diabetes, and more. Stems cell therapy is showing promise as a successful treatment for many conditions due to their ability to develop into many different cell types, thus having regenerative functions leading to repair of damaged cells and tissues. Due to controversies attached to the use of embryonic stem cells, research using mesenchymal stem cells from bone marrow, cord blood, and peripheral blood is very active.

Nearly 7 million people in the U.S. (about 2% of the total U.S. population) suffer some level of stroke-related brain damage and related health problems. Studies in animals suggest that cell-based therapies can improve post-stroke outcomes. To determine the safety of cell-therapy approaches in humans, researchers from Stanford University conducted a clinical trial to study the safety of a procedure to transplant donor stem cells in the brains of patients with chronic stroke. 

Patients with angina suffer with chronic, severe pain in the chest and other parts of the torso due to low blood supply to the heart muscle. Although there are standard treatments that work for many with angina, some have a more debilitating form that does not respond to treatment (refractory angina). About 1 million people in the U.S. have refractory angina. However, there is hope for those who are without viable treatment options. Researchers have observed that CD34+ stem cell treatment may help improve angina in patients who do not respond to other treatments.

The successes and failures that led to the state of stem cell therapy have provided a framework for other types of molecular therapies now being studied and advanced. An example of this begins with the development of a new type of therapy for the immunometabolic disorder, ADA-SCID. The therapy, strimvelis, was developed by scientists in Milan, Italy at the San Raffaele Hospital and Telethon Institute (TIGET). They accomplished this by combining the science of gene therapy with stem cell transplantation.