CARLSBAD, Calif., May 07, 2018 (GLOBE NEWSWIRE) -- International Stem Cell Corporation (OTCQB:ISCO) (www.internationalstemcell.com) ("ISCO" or "the Company"), a California-based biotechnology company developing novel stem cell based therapies and biomedical products, announced today that the United States Patent and Trademark Office (USPTO) has granted the Company a key patent (US9926529B2) on the method used to manufacture ISC-hpNSC. These are the cells administered in ISCO’s ongoing Parkinson’s disease clinical trial, and they can potentially be utilized in therapies to treat traumatic brain injury and stroke. This patent covers the procedure where human pluripotent stem cells (hpSCs), which have the potential to differentiate into essentially all cell types in the human body, are chemically differentiated into neural stem cells (NSCs), self-renewing cells that generate the neurons and glia of the central nervous system.
The new technique developed by ISCO’s scientists outperforms the traditional methods because it produces a more pure population of NSCs, generating NSCs that are over 95% pure, as opposed to traditional methods that only yield purities of around 80%. In addition, ISCO’s method is automatable and less labor intensive.
"Year after year, ISCO continues to strengthen its position as a leader in the field of regenerative medicine and explore the full potential of hpSCs," said Russell Kern, PhD, ISCO’s Executive Vice President and Chief Scientific Officer. "This new patent adds to the strong intellectual property of the company and positions us to extend our scientific reach to new heights," he continues.
Generating NSCs from hpSCs has proved to be a scalable method that is easily adapted for manufacturing under GMP (Good Manufacturing Practices). With this method, ISCO has developed both master and working cell banks of ISC-hpNSC® under GMP. These banks have undergone quality control release testing for sterility, purity, identity, potency, and safety before being utilized in the clinic.
The cells manufactured through this process are absent of residual pluripotent stem cells, have a normal karyotype and are negative for bacteria, fungal, mycoplasmal, and adventitious viral contaminants, which is critical to assure their safe use in the clinic.
ISCO also conducted a series of pre-clinical studies with these cells and demonstrated the absence of teratomas and tumorigenic cells, and these findings have been published in various prestigious peer-reviewed journals. This shows that the cells are not associated with any proliferative risk or other serious adverse event, which is critical for the translation of ISC-hpNSC® into viable therapies that could potentially be delivered to millions of patients suffering from Parkinson’s disease and other neurological diseases around the world.
About the clinical study
The Phase I clinical study is a dose escalation safety and preliminary efficacy study of ISC-hpNSC®, intracranially transplanted into patients with moderate Parkinson's disease. The open-label, single center, uncontrolled clinical trial will evaluate three different dose regimens of 30,000,000 to 70,000,000 neural cells. A total of 12 participants with moderate-to-severe Parkinson's disease will be treated. Following transplantation, the patients will be monitored for 12 months at specified intervals to evaluate the safety and biologic activity of ISC-hpNSC®. A PET scan will be performed at baseline, as part of the screening assessment, and at 6 and 12 months after surgical intervention. Clinical responses compared to baseline after the administration of ISC-hpNSC® will be evaluated using various neurological assessments such as Unified Parkinson Disease Rating Scale (UPDRS), Hoehn and Yahr and other rating scales. An extension phase of the study will evaluate patients every 6 months for 5 additional years.
About Parkinson's disease
Parkinson's disease is a degenerative disorder of the central nervous system mainly affecting the motor system. The motor symptoms of Parkinson's disease result from the death of dopamine-generating cells in the substantia nigra, a region of the midbrain. Early in the course of the disease, the most obvious symptoms are movement-related. These symptoms include shaking, rigidity, slowness of movement and difficulty with walking and gait. Later, thinking and behavioral problems may arise, with dementia commonly occurring in the advanced stages of the disease, and depression as the most common psychiatric symptom. Parkinson's disease is more common in people over the age of 50.
There are no approved treatments that restore the damaged dopaminergic neurons. Medications typically used in the treatment of Parkinson's disease, levodopa and dopamine agonists, improve the early symptoms of the disease. As the disease progresses and dopaminergic neurons continue to be lost, the drugs eventually become ineffective, while at the same time frequently producing a complication marked by involuntary writhing movements. There are over 10 million people afflicted by Parkinson's disease worldwide. In 2013 Parkinson's disease resulted in about 103,000 deaths globally, up from 44,000 deaths in 1990.
About ISC-hpNSC®
International Stem Cell Corporation's proprietary ISC-hpNSC® consists of a highly pure population of neural stem cells derived from human parthenogenetic stem cells. ISC-hpNSC® is a suspension of clinical grade cells manufactured under cGMP conditions that have undergone stringent quality control measures and are clear of any microbial and viral contaminants. Preclinical studies in rodents and non-human primates have shown improvement in Parkinson's disease symptoms and increase in brain dopamine levels following the intracranial administration of ISC-hpNSC®. ISC-hpNSC® provides neurotrophic support and neuroregeneration to the dying dopaminergic neurons of the recipient Parkinson's disease brain. Additionally, ISC-hpNSC® is safe, well tolerated and does not cause adverse events such as dyskinesia, systemic toxicity or tumors in preclinical models. International Stem Cell Corporation believes that ISC-hpNSC® may have broad therapeutic applications for many neurological diseases affecting the brain, the spinal cord and the eye.
About International Stem Cell Corporation
International Stem Cell Corporation (ISCO) is focused on the therapeutic applications of human parthenogenetic stem cells (hpSCs) and the development and commercialization of cell-based research and cosmetic products. ISCO's core technology, parthenogenesis, results in the creation of pluripotent human stem cells from unfertilized oocytes (eggs). hpSCs avoid ethical issues associated with the use or destruction of viable human embryos. ISCO scientists have created the first parthenogenetic, homozygous stem cell line that can be a source of therapeutic cells for hundreds of millions of individuals of differing genders, ages and racial background with minimal immune rejection after transplantation. hpSCs offer the potential to create the first true stem cell bank, UniStemCell™. ISCO also produces and markets specialized cells and growth media for therapeutic research worldwide through its subsidiary Lifeline Cell Technology (www.lifelinecelltech.com), and stem cell-based skin care products through its subsidiary Lifeline Skin Care (www.lifelineskincare.com). More information is available at www.internationalstemcell.com.
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Safe harbor statement
Statements pertaining to anticipated developments, clinical studies expectations (including timing and results), progress of research and development, the potential use of ISC-hpNSC in other therapies and other opportunities for the company and its subsidiaries, along with other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements. Any statements that are not historical fact (including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates,") should also be considered to be forward-looking statements. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in the development and/or commercialization of potential products, regulatory approvals, need and ability to obtain future capital, application of capital resources among competing uses, and maintenance of intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the company's business, particularly those mentioned in the cautionary statements found in the company's Securities and Exchange Commission filings. The company disclaims any intent or obligation to update forward-looking statements.
International Stem Cell Corporation
Russell Kern, PhD
Executive Vice President, CSO
(760) 940-6383
ir@intlstemcell.com