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.

HemaCare Corporation’s latest publication in BioPharm International ^[1] sheds light on a topic that has been preoccupying some of the best minds in cell therapy research; the need to ensure a reliable supply of top-notch starting materials for up-and-coming cell and gene therapies.

Unlike traditional medical treatments, the raw materials for these therapies cannot simply be manufactured. Instead, the supply of starting materials relies on a steady influx of voluntary donors willing to take the time to undertake a fairly complex screening process and donate peripheral blood or bone marrow for altruistic reasons. Nor is a reliable donor pool the only criteria necessary to supplying materials for a successful cellular therapeutic. Starting material quality and consistency ultimately determine the reliable efficacy of the final product.

HemaCare is pleased to announce the publication of our latest article ^[1] in the journal International Clinical Trials (ICT). The paper, entitled “The Landscape of Clinical Approval” describes how changes in the clinical drug approval process are impacting starting material suppliers, and what suppliers are doing to meet increased demands.

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.

“As the industry and regulatory landscape continues to evolve, it’s vital that as an industry, we take a lifecycle approach to risk management –to ensure that we mitigate as much risk as possible all the way from discovery through to commercialization.” - Dr. Dominic Clarke, Global Head of Cell Therapy, HemaCare

Cell and Gene Therapy Insights has just published HemaCare’s white paper on how to manage starting material quality and stability to maximum effect during cell therapy manufacturing. ^[1] Quality cell therapies can only be created from quality starting materials, yet paradoxically, starting materials are the single greatest source of variability in the cell therapy manufacturing process. Mitigating risk requires minimizing that variability, and also managing shelf-life limitations and biopreservation logistics.