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.

Researchers are studying the importance of white adipose tissue and its role in storing memory T cells.

The idea that if you don’t eat enough, you’ll get sick may be true in some ways, but there is new information available revealing that the immune system may have a strategy to keep fighting infectious agents even when food is scarce.

Traditionally used to treat cancer, new research shows how immunotherapy could also be useful for treating other diseases as well.

The goal of immunotherapy is to modify or influence the immune system in such a way to enhance its disease-fighting ability. Immunotherapies have been extensively studied and developed for the treatment of various forms of cancer. One type of immunotherapy used to target cancer cells is chimeric antigen receptor T (CAR-T) cell therapy.  In CAR-T therapy, a patient’s T cells are harvested, genetically engineered to target cancer cells, and grown under laboratory conditions. These cells are then injected back into the patient. There are a number of research endeavors that are now investigating the effectiveness of CAR-T cells for non-cancer diseases, including autoimmune conditions.

In addition to fetal tissue tolerance, T cells and NK cells also play a role in fetal-maternal metabolic homeostasis.

Immune system adaptations in the maternal-fetal environment are essential for a successful pregnancy. When this adaptation is hindered or inadequate, infertility, miscarriage, pregnancy complications, and adverse fetal outcomes can occur. Specific immune cells have been studied to better understand their role in pregnancy and to know what this information can provide overall regarding the development of new immunotherapeutic approaches for disease states.

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.