Blog | HemaCare

As Phase 3 clinical trials begin for Lenti-D as a treatment for patients with X-ALD, the drug also shows promise for treating a range of neurodegenerative diseases.

X‐linked adrenoleukodystrophy (X‐ALD) is a peroxisomal disorder resulting from a mutation in the human ATP-binding cassette, sub-family D, member 1 gene (ABCD1). The ABCD1 gene codes for the adrenoleukodystrophy protein (ALDP) needed for very-long-chain fatty acids (VLCFAs) to enter peroxisomes for degradation. When this process is disrupted, the VLCFAs accumulate in various tissues of the body, including the brain. VLCFA accumulation and demyelination of brain white matter lead to the severe neurological symptoms seen with X-ALD.

Scientists in New York studying allergic disease have just published work citing the use of HemaCare-sourced bone marrow mononuclear cells for their research into the mechanisms of immunological memory.

The immune system is a wonderfully complex assembly of interacting cells and tissues that protect us from disease. To function properly, the cells within this system must detect and remember a wide variety of triggering agents.

Stromal cells are becoming a viable candidate for cell therapy due to their immunosuppressive properties and ability to treat autoimmune diseases.

Advances in the development of treatments for autoimmune diseases and tissue repair are being realized through cell therapy approaches. Research is ongoing to overcome common limitations to successful clinical use, such as transplantation rejection and low therapeutic efficacy. Stromal cells, or mesenchymal stem cells, are becoming a very viable candidate for cell therapy due to their unique characteristics in comparison to other stem cell types. Mesenchymal stem cells (MSCs) are pluripotent stem cells that are self-renewable; however, they have immunomodulatory features that increase the success of their utility for immune disease therapy and regenerative medicine.

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.