Celavie Biosciences Product Development
Celavie Biosciences is developing restorative stem cell treatments for Parkinson’s disease (PD), Huntington’s disease, Friedreich’s ataxia (FA), amyotrophic lateral sclerosis (ALS), epilepsy, cerebellar ataxia and other disorders, such as type 1 diabetes.
|Disease||Candidate||Preclinical||Phase I||Phase II||Phase III|
|Amyotrophic Lateral Sclerosis||Human|
OK99 Stem Cells in Development to Treat Parkinson’s Disease
For treatment of PD, Celavie scientists are testing our OK99 allogeneic pluripotent stem cells with plans to file an Investigational New Drug (IND) with the FDA. At the conclusion of the Phase 1 trial in Mexico, a three-year study that has just reached a two-year follow up stage with encouraging results, we plan to begin a US-based clinical trial of our OK99 stem cell line.
OK99 Stem Cells, Phase 1 Trial, Mexico
Top row: A larger area of the brain 1 year after transplantation (colored in red) documents increased release of dopamine into the specific area of the brain that directly correlates with clinical improvement in PD in a sample patient.
Middle row: A smaller area of the brain 1 year after transplantation (colored in red) documents that a selective antagonist of dopamine, RAC, occupies a smaller area because of the increased competition from increased levels of dopamine. This correlates with clinical improvement in PD.
Bottom row: A larger area of the brain 1 year after transplantation documents increased production of dopamine in the brain, which directly correlates with clinical improvement in PD.
- 11C-DTBZ, marker for vesicular monoamine transporter 2 (VMAT2) activity
- 11C-RAC , marker for dopaminergic (D2) receptor density
- 18F-DOPA reflects level of dopamine synthesis.
- Higher DTBZ and F-DOPA uptake and lower RAC uptake together reflect increased dopamine production, which might underlie the patients’ improved scorings on the UPDRS.
As a part of preclinical safety evaluation of OK99 cells, Celavie examined immunogenicity of the stem cells in a same-species environment. Celavie contracted Seventh Wave Laboratories, LLC (Chesterfield, MO) to perform the study: “Does Intraparenchymal Injection of Canine Allogeneic Stem Cells into the Canine Brain Elicit Immune Response.”
Our canine stem cells that are derived in the same manner as human cells and express the same markers were transplanted into the brains of healthy beagles. The blood and brain tissue were examined for signs of immune reaction to the cells. No immune response to OK99 stem cells was detected.
Developmental Treatment for Friedreich’s ataxia with OK99 Stem Cells
Celavie researchers are also considering FDA submission for Friedreich’s ataxia, based on positive results from preclinical work in a rat model of Ataxia.
Friedreich’s ataxia is a rare, inherited genetic condition that occurs in 1 in every 50,000 people in the US. It causes nervous system damage and movement problems. Usually beginning in childhood, it leads to degeneration of the spinal cord, the peripheral nerves and an area in the cerebellum that coordinates balance and movement. It often leaves patients wheelchair-bound. It also causes heart problems, diabetes and death. There is no known cure. Potential treatments for Friedreich’s ataxia are eligible for FDA’s orphan drug status, which greatly speeds up approval.
The following two videos demonstrate the effects of OK99 injection on rats with a model of cerebellar ataxia. While the untreated rat is unable to move, the treated animal can run.
Two manuscripts, “Efficacy of Two Delivery Routes for Transplanting Human Neural Progenitor Cells (NPC) Into the Spastic Hans Wistar Rat, a Model of Ataxia,” and “Transplantation of human neural progenitor cells reveals structural and functional improvements in the spastic Han-Wistar rat model of ataxia” were published in Cell Transplantation Journal in February 2017 and in June 2017 correspondingly.
Treatments in the Pipeline
Celavie is currently conducting preclinical trials of OK99 undifferentiated stem cells in animal models of diabetes mellitus type 1, and further studies of cerebellar ataxia (effect of stem cell treatment on the animals’ longevity) in collaboration with California State University, Northridge. Our research plan includes preclinical studies using OK99 stem cells for animal models of epilepsy, stroke, and ALS.
In addition to OK99 allogeneic pluripotent stem cells, Celavie’s subsidiary, Celavet, is offering a faster path to market with cultured stem cell lines, including equine cells (OK100), canine cells (OK200), and feline cells (OK300).