CARLSBAD, CA – International Stem Cell Corporation (OTCQB: ISCO), a California-based clinical stage biotechnology company developing stem cell-based therapies and biomedical products, announced today the positive six months interim results of the first cohort of Parkinson’s disease (PD) patients receiving 30 million ISC-hpNSC cells in the ongoing clinical study. All patients in the first cohort are currently meeting the primary endpoint, which is safety.
“We are already seeing some positive efficacy results six months post-transplantation in this 12-month study. While the relatively small sample size makes it difficult to observe statistically significant differences, the interim efficacy results are very encouraging. The first dose tested was used to determine the safety and tolerability of ISC-hpNSC therapy and is below the optimal therapeutic dose established in our preclinical studies,” commented Russell Kern, PhD, ISCO’s Executive Vice President and Chief Scientific Officer. “We are anticipating strong results in the second cohort (receiving a higher dose of cells) in which two patients have been treated already. These clinical results build a strong foundation to start phase II clinical trials in PD and traumatic brain injury, which are some of the biggest current unmet medical needs,” continued Dr. Kern.
The % OFF-Time, which is the time of day when levodopa medication is not performing optimally and PD symptoms return, decreased an average of 24% for the first cohort at six months post-transplantation. The % ON-Time without dyskinesia, which is the time of day when levodopa medication is performing optimally without dyskinesia, increased an average of 19% for the first cohort during the same period. One hundred percent of the patients improved their mood six months post-transplantation with an average improvement of 35% in the Beck Depression Inventory and 33% in the Emotional Wellbeing dimension of the Parkinson’s Disease Quality of Life Score-39 (PDQ-39). 100% of patients improved or retained the same cognitive abilities with an average improvement of 14% in the Cognitive Impairment dimension of the PDQ-39. Conducting routine daily activities seemed easier after six months as demonstrated by the average improvements of 22% in Activities of Daily Living and 15% in Mobility dimensions of PDQ-39. The Bodily Discomfort dimension of PDQ-39 also improved an average of 12% after six months. The Unified Parkinson’s Disease Rating Scale in the OFF period did not improve six months post-transplantation. Impulsive and compulsive disorders were diminished, as demonstrated by the 53% reduction in the Questionnaire for Impulsive-Compulsive Disorders in Parkinson’s disease. No test article related serious adverse events have been reported in the clinical trial. There is no evidence of tumors, cysts, enhanced inflammation or infection. Furthermore, no Human Leukocyte Antigen antibodies against the implanted ISC-hpNSC have been detected.
About the Clinical Study
The Phase I clinical study is a dose-escalation safety and preliminary efficacy study of ISC-hpNSC®, intracranially transplanted into 12 patients who have moderate-to-severe Parkinson’s disease. The open-label, single center, uncontrolled clinical trial will evaluate three different dose regimens of 30 million to 70 million neural cells. Following transplantation, the patients will be monitored for 12 months at specified intervals to evaluate the safety and biologic activity of ISC-hpNSC®. PET scan will be performed at baseline as part of the screening assessment, and at six 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 the Unified Parkinson Disease Rating Scale (UPDRS), Hoehn and Yahr, among others. Patients will be followed for 5 additional years to monitor safety and progression in neurological assessments.
About Parkinson’s Disease
Parkinson’s disease (PD) is a degenerative disorder of the central nervous system, mainly affecting the motor system. The motor symptoms of PD 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 walking. Later, thinking and behavioral problems may arise, with dementia commonly occurring in the advanced stages of the disease, with depression being the most common psychiatric symptom. PD most commonly occurs with people over the age of 50.
Currently, medications typically used in the treatment of PD, L-DOPA and dopamine agonists, improve only early symptoms of the disease. As the disease progresses and dopaminergic neurons continue to diminish, drugs eventually become ineffective while at the same time, frequently producing a complication marked by involuntary fidgeting movements. In 2013, PD resulted in about 103,000 deaths globally, up from 44,000 deaths in 1990.
ISCO’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 cell replacement to the dying dopaminergic neurons of the recipient PD brain. Additionally, ISC-hpNSC® is safe, well-tolerated, and does not cause adverse events such as dyskinesia, systemic toxicity, or tumors in preclinical models. ISCO believes that ISC-hpNSC® may have broad therapeutic applications for many neurological diseases affecting the brain, the spinal cord, and the eye.
Safe harbor statement
Statements pertaining to anticipated developments, expected results of clinical studies, potential applications of ISC-hpNSC® to other diseases, progress of research and development initiatives, 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 (including clinical trial results that differ from expectations based on earlier studies), 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.