US-based biotechnology firm Q Therapeutics has received orphan drug designation from the US Food & Drug Administration (FDA) for its Q-Cells product (human glial restricted progenitor cells and their progeny) for treatment of amyotrophic lateral sclerosis (ALS) patients.
ALS is also referred to as motor neurone disease (MND) in most commonwealth countries and as Lou Gehrig’s disease in the US.
Each year around 5,600 people in the US are diagnosed with ALS and most patients die two to five years after diagnosis, as no effective therapies exist, according to the ALS Association.
With the orphan drug status, the company will benefit from financial incentives from the FDA, including seven years of market exclusivity after product marketing clearance and access to federal grant funding opportunities to pay clinical trial costs, as well as assistance with clinical protocols and federal tax credits.
Q Therapeutics president and chief executive officer Deborah Eppstein said designation of Q-Cells as an orphan drug for the treatment of ALS is a significant milestone for ALS patients, who have few treatment options.
"As we move toward our Phase 1/2a clinical trial with Q-Cells in ALS in 2014, orphan drug designation opens up new federal grant opportunities to help us advance our goal of providing efficacious treatment for ALS patients," Eppstein said.
"Orphan drug designation also complements our intellectual property portfolio. The potential for seven years of market exclusivity protection after product launch augments Q-Cells’ proprietary position."
According to the company, in many neurodegenerative conditions such as ALS, glial cells in the brain and spine are damaged or diseased, and if untreated may result in damage to and eventual death of the neuronal cells that glia are meant to support.
Q Therapeutics’ answer to ALS was providing the diseased/damaged central nervous system with healthy glial cells, trademarked Q-Cells.
The companies’ research collaborators at Johns Hopkins University Medical School have shown that a single treatment with the rat homolog of Q-Cells doubled the survival time after ALS disease onset in a rat model of ALS.
Q-Cells are currently under development for the treatment of multiple sclerosis, transverse myelitis, traumatic spinal cord injuries and ALS.
According to the company, Q-Cells technology aims to treat neurodegenerative diseases by supplementing the damaged or missing glia in the CNS with new, healthy cells that can help maintain and/or restore neuron function to a more robust state.
Q Therapeutics’ collaborators at The Johns Hopkins University have previously secured $1.3m in a third year of grant funding from the National Institute of Neurological Disorders and Stroke (NINDS) of the National Institutes of Health (NIH) for manufacturing and pre-clinical safety studies for Q-Cells.
Q Therapeutics is engaged in the development of new cell-based therapeutics to treat debilitating diseases of the central nervous system.
Image: MRI (axial FLAIR) demonstrates increased T2 signal within the posterior part of the internal capsule, consistent with the clinical diagnosis of ALS. Photo: courtesy of Frank Gaillard.