The development of stem-cell based therapies has been slow even though there are many theoretical applications and benefits associated with these therapies. The challenges of translating potential drugs from clinical phases to bedside applications appear at both the microscopic and macroscopic levels.
The microscopic challenges include aspects such as the source of the stem cells and compatibility issues. The generation of embryonic stem cells requires the destruction of the embryo. In order to avoid this, there is a focus on using adult stem cells for the purpose of achieving similar regeneration outcomes. The immune-compatibility issue can be significantly subverted by using autologous cell lines—stem cells from the patient’s own body; however, this raises the question of how much starting material can be obtained, which is an important factor when considering scaling-up the product volume. Macroscopic challenges appear at the manufacturing process, as every stage of the process needs to strictly adhere to current good manufacturing practice (cGMP) guidelines. Inherent variation within the human cell population poses process validation challenges, and the lack of a clear and harmonized regulatory framework on stem cell treatments also poses a challenge to development in this area. All of these factors contribute to a long development timeline and associated developmental costs.
Despite the developmental challenges of stem-cell based products, the hype associated with the use of these products often outpaces the rate at which purpose-driven regulations have been developed—specifically, regulations that provide the finer details of what product types are qualified for approval by the FDA for use in the market. In November 2017, the FDA announced a comprehensive regenerative medicine policy framework, which had the objective to provide a clear structure for companies intending to develop products in the regenerative medicine landscape. The development and enforcement of these regulations are expected to drive innovation in this space and help the industry to establish the safety and efficacy of these novel products.
According to GlobalData, there are currently 29 stem-cell based products in the pipeline —Phase I, II, or III—that are being studied for potential use in the central nervous system. More than half of these candidates are currently in Phase II trials. A closer analysis of these product profiles reveals a wide range of indications for which these drugs are being developed, including Alzheimer’s disease, Parkinson’s disease, and spinal cord injury. This is a promising blueprint of development for neurology, considering the fact that there is only one currently marketed stem-cell product in this therapy area: Neuronata-R, which is only available in South Korea and is indicated for Amyotrophic Lateral Sclerosis (ALS).
As the regulations become clearer and more pipeline products enter clinical trials, GlobalData expects that the FDA’s recently announced regulatory developments will help to establish a wider ecosystem that will help to clarify the approval process of safe stem-cell based products. The launch of this new framework is also expected to help other countries develop similar policies in line with what has been set by the FDA.