Stem Cell Therapies for Parkinson's Disease on the Horizon

An upcoming phase 1/2a clinical trial using the transplantation of stem cells sparked hope for the treatment of Parkinson’s disease (PD) during a recent study.

The trial will take place later this year and has created a buzz online among patients who are debating if they should sign up.

“As with many such exiting news items, however, one should also react with caution, especially since the outcome of this trial can affect the development of other stem cell programs moving towards clinical trials,” said lead author Roger A. Barker, PhD, in a press release.

The collaborate initiative GForce-PD define criteria in order to gauge progress from experimental results towards clinical trials, while making sure that every step was conducted at the highest standard and trials are not initiated prematurely.

Authors will review the stem cell-based trial, but believe a discussion should be held prior to the start using 5 key questions.

The questions included:

• What is transplanted and what’s the proposed mechanism of action?
• What pre-clinical and efficacy data supports the use of the proposed product?
• Can arguments on ethics, risk mitigation, or trial logistics outweigh concerns on the expected efficacy of the cell and have a primary justification for choosing 1 cell over another?
• In regards to potential therapeutic value of the stem cell based therapy what is being claimed, better symptoms control or a cure?
• What’s the regulatory oversight of the trial and is it guided by experts input in the field?

Authors will review how the stem cell therapies for PD have evolved and discuss the early results. They also elaborated on these 5 key questions and expressed concern for possible missing or incomplete information available.

Concerns were especially raised for cell types being transplanted that might not function as they wanted, and that the safety and efficacy data hasn’t been made public. Additionally, the length of follow-up in the trial may be insufficient. The authors also stated that there is a commonality among exaggerated claims.

“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 co-author Patrik Brundin, MD, PhD. “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. 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 3 decades.”