13 April, 2022
White Paper (Preclinical Studies)
CREATIO PARTICIPATES IN THE GUIDELINES TO BRING RESEARCH CLOSER TO THE CLINIC FOR HUNTINGTON PATIENTS
Creatio’s staff are involved in the white paper that sets the challenges to translate the substantial advances made in cell therapy to patient benefit. The report has been published this month at the prestigious journal Brain.
Dr. Josep M. Canals, Dr. Unai Perpiña and Dr. Cristina Salado participated, as Creatio representatives, with the definition of the 7 challenges to overcome for the clinical translation of cell therapy in Huntington’s disease. The report, published in Brain, is focus on the accelerated progress in stem cell therapies and its potential to reach clinical trials. However, it reinforces the need for harmonisation across all stages, from preclinical studies to clinical application. These range from choosing the right animal models, scaling-up, designing appropriate clinical trials and patient selection frameworks to post-transplantation management of these patients. Expert’s conclusions reinforce the need for international collaboration and harmonization to ensure that the knowledge gained in pre-clinical studies can be applied in clinical trials.
Creatio, the Production and Validation Center of Advanced Therapies, is a member of the Stem Cells for Huntington Disease (SC4HD) and the European Huntington Disease Network Advanced Therapies Workshop Group (EHDN), both involved in the development of this white paper. These international networks bring experts from a broad range of fields to share experiences, develop guidance and highlight future directions towards the treatment of Huntington’s disease.
The Director of Creatio, Dr. Josep M. Canals, was hopeful that this report “would set a clear path of the issues that ought to be resolved in our collaborative efforts to treat Huntington’s disease, not just to alleviate its symptoms”. Dr. Canals emphasised that regenerative medicine in neurodegenerative disorders has a restorative goal. Unlike drug treatments, cell therapy aims to recover already lost brain cells. This is of particular importance as once the disease is diagnosed, there is already significant neuronal damage that cannot be reversed. Hence, cell therapy should be seen as a complimentary option for the treatment of Huntington patients.
Huntington’s disease is a progressive brain disorder that causes uncontrolled movements, emotional problems, and loss of thinking ability (cognition). Adult-onset Huntington disease is usually in a person’s thirties or forties, with serious social, economic and emotional implications for patients and their loved ones. Moreover, Huntington’s disease affected patients have features of the major pathophysiological hallmarks of the most prevalent multi-genic and/or multifactorial neurodegenerative diseases. Added to the availability of genetic tests and multiple cell and animal models, Huntington’s disease is an excellent candidate in which to test, optimise and translate cell therapy. Thus, experts hope that these guidelines will also pave the way for other complex neurodegenerative disorders such as Parkinson’s or Alzheimer’s diseases.