Blog | HemaCare

Mesenchymal Stem cells (MSCs) derived from mouse muscle tissue and bone marrow were used in an experimental cell therapy for Duchenne Muscular Dystrophy.

Duchenne muscular dystrophy (DMD) is an incurable, progressively debilitating muscular-skeletal and cardiac disease caused by a mutation in the dystrophin gene. The encoded dystrophin protein is part of a larger protein complex involved in anchoring the muscle cytoskeleton to components of the extracellular matrix. Loss of dystrophin leads to muscle wasting, and heart disease is a major cause of death in patients with DMD. Therefore, the availability of effective treatments to address cardiac function in people with DMD is vital.

A new study shows that we may be able to boost the grafting ability in cells for people receiving a bone marrow transplant.

Allogenic stem cell transplantation is a treatment approach in people with diseases that are destructive to the bone marrow, such as multiple myeloma, leukemia, and Hodgkin´s lymphoma. The goal is to restore bone marrow after total-body chemotherapy and irradiation. The transplantation procedure involves transferring hematopoietic stem cells from compatible, healthy donors to a patient. An important complication of this procedure is rejection and destruction of the donated stem cells by the recipient’s immune system before engraftment can occur.

While no cure exists for ALS, researchers are finding ways to use unmodified human bone marrow CD34+ cells for spinal restoration.

The renowned physicist Stephen Hawking influenced how countless scientists worldwide view the universe. He also demonstrated his remarkable ability to survive amyotrophic lateral sclerosis (ALS) for 55 years. He is thought to be the longest survivor of ALS. According to the ALS Association, 50% of those with ALS survive for about 3 years after diagnosis, 20% survive for 5 or more years, and 10% survive for 10 years or more. There is no cure for ALS, and therapies that can slow or delay progression, decrease symptoms, and repair damaged spinal-cord tissue are continuously sought.

An independent publication cites the use of bone marrow and mobilized peripheral blood sourced from HemaCare in a study aimed at improving the efficiency of resource consumption in gene therapy.

The publication, published in the journal Molecular Therapy, focuses on the use of lentiviral vector (LV) to introduce corrected versions of defective genes into stem cells. The goal of gene therapy is to transplant these stem cells into people suffering from genetic disease, in hopes of restoring the function of the faulty gene. One of the main challenges in this clinical strategy is the cost; substantial amounts of LV are needed to modify a therapeutically effective dose of stem cells.

An independent study cites the use of HemaCare-sourced human bone marrow-derived CD34+ stem cells for research into a potential new therapy for sickle cell disease. ^[1]

Sickle cell is a serious disease that affects millions of people throughout the world. People affected by sickle cell anemia have rigid, sickle-shaped red blood cells that can’t adequately carry oxygen to the body’s tissues. As a result, sickle cell patients suffer from a variety of medical complications, which can shorten their life span. Contemporary cell therapy treatments have focused on a gene therapy approach that uses lentiviral vectors to supply CD34+ bone marrow-derived stem cells with an unimpaired version of the hemoglobin gene. While this tactic has met with some promise, finding healthy, matched bone marrow donors is difficult. Researchers at Biogen, a well-known biotech firm headquartered in Cambridge, Massachusetts, are trying to discover cell and gene therapy methods that they hope will provide an alternative solution, and thereby improve treatment success rates.