The United States Food and Drug Administration (FDA) has recently cleared the Investigational New Drug (IND) application of Cellenkos, Inc. The company can now proceed with a phase I clinical trial of its IND first-in-class CK0801, allogeneic cord blood-derived regulatory T cells, in patients with treatment-resistant Guillain-Barré Syndrome (GBS).
Cellenkos also holds an earlier FDA approval to proceed with a phase I study of CK0801 in bone marrow failure syndromes.
Founded in 2016 by Simrit Parmar, MD, Associate Professor at The University of Texas MD Anderson Cancer Center, and based in Houston, USA, Cellenkos is an early stage biotechnology company that focuses on cord blood-derived T regulatory cellular therapies for the treatment of autoimmune diseases and inflammatory disorders.
CK0801 was developed using Cellenkos’ proprietary manufacturing platform, and can be infused at the point of care. CK0801 will be manufactured in the Cellenkos FDA registered GMP facility located in Houston, Texas.
GBS is a rare disorder that destroys the protective covering of the peripheral nerves (myelin sheath), preventing the nerves from transmitting signals to the brain. According to the US National Institute of Neurological Disorders and Stroke, the rate of GBS occurrence is about one person in every 100,000 each year.
The exact cause of GBS is unknown. But it is often preceded by an infectious illness such as a respiratory infection or the stomach flu.
The first symptoms of GBS include varying degrees of weakness or tingling sensations in the legs. In many instances the symmetrical weakness and abnormal sensations spread to the arms and upper body. These symptoms can increase in intensity until certain muscles cannot be used at all and, when severe, the person is almost totally paralyzed.
GBS can affect anybody. It can strike at any age and both sexes are equally prone to the disorder. A cure has not been developed.
Adoptive therapy with CK0801 is intended to result in the infused healthy regulatory T cells preferentially trafficking to the area of inflammation; engaging with the key regulators of immune dysfunction and secreting suppressor cytokines. This is intended to inhibit proliferating auto-reactive effector T cells and restore immune homeostasis. Ultimately it is intended that this would halt nerve damage leading to clinical improvement.
A Phase I clinical trial of CK0801 will be led by Kazim Sheikh, MD, Professor and Director of the Neuromuscular Program at the University of Texas Health Sciences Center at Houston (UTHealth), Houston, Texas.
Phase I trials are primarily intended to ensure that the IND candidate is safe for people to receive, however, the resulting data can often give an early indication of the IND’s efficacy.
“The timely clearance of this IND and commencement of the Phase I trial will unlock the tremendous potential of cellular therapy application for neuro-inflammatory disorders. This is yet another significant milestone for Cellenkos, and marks an important step forward in finding effective treatments for autoimmune diseases”
“For a rare and debilitating disease with no known cure, this is an exciting opportunity to bring a novel cellular therapy, CK0801, that works to replace and replenish the defective regulatory T cells and protects the neurological system from the constant attack as a result of uncontrolled inflammatory signals. Such an interruption of the neural insult loop can allow for reinstating the immune balance and support neuron regeneration leading to clinical improvement”. – Simrit Parmar, MD, Cellenkos’ Founder and Chief Medical Officer
“The US FDA’s clearance of this IND continues to validate our commitment and progress towards creating an ‘off-the-shelf’ cord blood-derived regulatory T cell therapeutic platform and reinforces our strive to bring forth ‘on-demand’, affordable, point-of-care delivery cellular therapies that can have a tremendous impact on patient care by providing novel, non-pharmacologic options”. – Tara Sadeghi, Senior Clinical Director at Cellenkos