Our Solution: Q-Cells®

Q-Cells® are the first and only human glial-restricted progenitor (GRP) cell therapeutic

Glial progenitor cells are early descendants of neural stem cells that can produce two types of glial cells that are essential for supporting, maintaining or even restoring neuron health. Although glial progenitor cells are present in the adult brain (including the spinal cord), they are limited in number.

Q-Cells® are purified human glial progenitor cells that have been isolated and cultured from brain tissue at the precise stage of development when their ability to multiply, migrate and differentiate into glial cells is most potent.

Q Therapeutics is the only company developing this specialized cell type, which we believe to have the greatest potential for long-lasting CNS benefits.

Based on numerous, promising laboratory and animal studies, we are actively preparing for a clinical trial of Q-Cells® in human patients with amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s disease). In 2017, we received FDA clearance to proceed with a 30 patient, dose escalating trial.

Q-Cells® have been shown to successfully differentiate into:

  • Astrocytes – Specialized cells that perform a variety of neuroprotective functions
  • Oligodendrocytes – Specialized cells that produce the myelin needed for normal transmission of nerve signals in the brain and spinal cord

Q-Cells® have already been granted fast-track and orphan disease designation by the U.S. Food and Drug Administration (FDA) for ALS. Going forward, we will be pursuing similar designations in both Europe and Japan.

Existing safety and manufacturing data support multiple CNS indications for broader market penetration.

Early Evidence for the Safety and Efficacy of Q-Cells®

Q-Cells® have already been studied extensively in the laboratory and in rodent models of various CNS diseases, including:

  • Myelination deficiency, as is found in multiple sclerosis
  • Amyotrophic lateral sclerosis (ALS, a degenerative disease of the motor neurons)
  • Spinal cord injury
Rodent models are excellent models for many human CNS diseases because CNS mechanisms are highly conserved (have remained largely unchanged by evolution) between rodents and humans. The research results in these highly conserved models of human disease are promising for our planned phase 1/2a clinical trials in ALS.
 
Pre-clinical studies have shown:
  • No observed toxicity in more than 200 treated animals in six independent laboratories
  • Q-Cells® differentiate only into astrocytes and oligodendrocytes
  • Q-Cells® are capable of migrating to and targeting at-risk motor neurons in animal models of ALS
  • Implantation of Q-Cells® in animal models of ALS enhances motor function and survival*
  • Q-Cells® can mature into oligodendrocytes that are capable of producing normal myelin
  • A single implantation of Q-Cells® results in widespread myelin production, with associated improvement in survival

Projected Safety Benefits of Q-Cells®

Q-Cells® do not differentiate into neurons, eliminating the risk of uncontrolled new neuron formation or aberrant neuronal connections that could lead to seizures or other nerve conduction problems. In addition, Q-Cells® can be injected directly into the brain or spinal cord at the site of injury or disease, avoiding many of the off-target risks associated with oral medicines or medicines delivered through the bloodstream.

The Advantages of a Later-Stage, More Differentiated Cell



Unique Attributes of Q-Cells® in Comparison to Other Cell Therapies

  • Not harvested from the patient – Q-Cells® are an allogeneic cell therapy delivered to the site as an off-the-shelf pharmaceutical product that is ready to be injected for therapeutic use.
  • No genetic manipulation – Q-Cells® are isolated and purified from brain tissue, so they are natural CNS cells in their natural state performing their natural function.
  • Small-volume injection – Each vial of Q-Cells® contains millions of human glial progenitor cells, with the capacity to multiply and differentiate into millions of astrocytes and oligodendrocytes.
  • Long-lived in the CNS – Q-Cells® survive in the CNS longer than mesenchymal stem cells (MSCs), with the potential to provide long-lasting effects.
 
* Using the rat homolog of Q-Cells® in a rat model of ALS.

 

A culmination of 10 years of intense research and development, Q-Cells® are a novel therapeutic product that can potentially be used to treat a wide range of CNS disease and injuries.