International Stem Cell Corporation is starting clinical trials on for Parkinson
International Stem Cell Corporation (OTCQB:ISCO) has been granted authorization to start its phase I/IIa clinical trials on 12 participants with moderate to severe Parkinson’s disease. At first glance, the news might not spark too much attention. However, the implications of the trials could spell a world of difference for those suffering from Parkinson’s disease. That is, the clinical could trials could mean there is a cure on the horizon for those affected by the disease.
ISCO started enrolling patients for its clinical trial in early March after having received approval from the Melbourne Health Human Research Ethics Committee. The approval clears the study’s initiation at the Royal Melbourne Hospital.
“Enrollment in this trial is an important milestone. Promising preclinical results support our expectation that ISC-hpNSC will bring a long-needed solution for patients suffering from Parkinson’s disease.” Russell Kem, executive vice president ant chief scientific officer said in a company statement.
The first round of human trials will look to determine the dosage levels required. The company notes that following the transplant, the patients will be monitored for 12 months at specified intervals, in order to evaluate the safety and biologic activity.
Concerns remain on stem cell therapies remain
The Mayo Clinic describes Parkinson’s disease is a progressive disorder of the nervous system that impacts movement. The disorder develops gradually, with the most well known sign of Parkinson’s being a tremor, that starts off barely noticeably in one hand. However, as symptoms progress, the resulting effect is often stiffness or slowing of movements.
Though the cause of Parkinson’s disease is largely unknown, there are several factors that contribute to the disease, including genetics, environmental triggers like exposure to certain toxins can increase the risk of an individual’s likelihood of developing Parkinson’s later in life. Also of not, there are changes within people’s brain, the presence of Lewy bodies –clumps of specific substances within brain cells–, and alpha-synuclein, a widespread protein, found within Lewy bodies.
Still, while the prospect of a cure on the horizon for a degenerative disease that affects somewhere in the are of 7 million people world wide is a happy prospect, there are still those in the scientific community concerned with the company’s plan.
“This is an exciting prospect but should only be undertaken when all the necessary pre-clinical data and regulatory approvals have been obtained and verified and the criteria for moving those cells to trials fully resolved and met,” said Patrik Brundin, co-author of the paper and editor-in-chief of the Journal of Parkinson’s Disease. “Acting prematurely has the potential not only to tarnish many years of scientific work, but can threaten to derail and damage this exciting field of regenerative medicine.”
In the paper published in the Journal of Parkinson’s Disease, there are five key question that are going to be addressed before the trial begins.
- What is being transplanted, and what is the proposed mechanism of action?
- What are the pre-clinical safety and efficacy data supporting the use of the proposed stem cell product?
- Can arguments concerning ethics, risk mitigation, or trial logistics outweigh concerns regarding the expected efficacy of the cell and constitute a primary justification for choosing one cell type over another in a clinical trial?
- What is being claimed regarding the potential therapeutic value of the stem cell-based therapy – better control of symptoms or a cure?
- What is the regulatory oversight of the trial and is it guided by input from experts in the field?
Medical Express explained that in the commentary,the authors discuss “how cell-based therapies for PD have evolved and discuss some of the early results”, “the ethical issues concerning fetal stem-cell use.”
Furthermore, in addressing the key questions, the authors expressed some concern that there is some missing information from the company specifically the “particular cell types being transplanted may not function as desired, and that supporting safety and efficacy data have not been made public. The consortium also suggests that the length of follow-up in the proposed trial may not be sufficient.”
“Hopefully, in 2016, we are ready to take a more careful approach as we strive to repair the PD brain with stem cell-based therapies, avoiding many of the mistakes that have dogged this field over the last three decades.” Brundin stated.
Securities Disclosure: I, Vivien Diniz, hold no investment interest in any of the companies mentioned.