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.

A new type of T cell capable of recognizing and killing most human cancer cells has just been discovered. Researchers at Cardiff University in Wales hit upon the entirely new class of T cell while examining immune cells at a local blood bank. ^[1] This T cell carries a novel T cell receptor (TCR) which recognizes most cancers, while ignoring healthy cells.

"File:Lab mouse mg 3216.jpg" by Rama is licensed under CC BY-SA 2.0

A recent publication out of UCLA cites using HemaCare mobilized leukopaks in the development of a new natural killer (NK) T cell-based therapy. ^[1]

The multi-departmental group of scientists, the majority of whom are based at UCLA, collaborated on the groundbreaking cancer research project. Their potential treatment relies on the unique properties of a powerful class of natural killer T cells.

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.