Recently we wrote that the EMA made a remarkable decision by designating cannabidiol (CBD) as an orphan drug for the treatment of Leigh syndrome. It was all thanks to researchers from the Mitochondrial Neuropathology Research Group at the UAB Institut de Neurociències (INc-UAB): drs. Emma Puighermanal, Albert Quintana, and Elisenda Sanz.
We were talking with Albert Quintana Romero about this achievement and next steps of Leigh syndrome research.
CBD: potential outcomes
The longer lifespan of mice with Leigh syndrome in animal research was one of the most intriguing findings, as stated in our original article. We therefore posed the question of what potential implications this finding would have for patients. We asked if there are any current initiatives to use this discovery to the treatment of patients.
As dr. Albert Quintana noted at the start, there is currently no cure for Leigh Syndrome. The only options that are available are vitamins and supplements. Nevertheless, there is no proof that they help people with Leigh Syndrome in general. Even if they make sense from a biological perspective.
For this reason, according to Dr. Quintana, cannabidiol offers a novel treatment approach. CBD shows promise in a disease-related animal model that went through thorough testing. Since mitochondrial diseases like Leigh Syndrome are monogenic, he thinks that reexpressing the mutated gene will be necessary for a cure. He is certain, though, that CBD will be a first-in-class medication that helps to both delay and relieve the clinical symptoms.
Leigh syndrome: cannabidiol clinical trial
We asked about the specific steps involved in moving forward with the CBD clinical trial as a Leigh syndrome treatment. And, as dr. Quintana says cannabidiol has already been passed by both the FDA and EMA. Because of this, it’s possible that the new Leigh Syndrome indication will be exempt from a few regulatory requirements.
However, since the medication is approved, there is an opportunity to begin a small compassionate use clinical trial while researchers work on the regulatory plan. They think that in the upcoming year, they need to be ready to start accepting new patients:
“Our goal is to establish the efficacy of cannabidiol in this first set of patients, which will allow it to expand to a full-sized clinical trial.”
They are optimistic to be able to begin the first clinical trial in 2024. They want to complete it by the end of 2025. In order to achieve this, they intend to establish a spin-off company that will oversee the first clinical trial. They will be taking the required regulatory actions in the interim in order to have CBD for Leigh Syndrome approved.
Dr. Quintana additionally addressed the upcoming phases of the Leigh syndrome and CBD clinical trial studies. In the lab, researchers are concentrating on figuring out the mechanisms underlying CBD’s beneficial effects, which may lead to the discovery of new, more focused medications.
Still, researchers’ objective in terms of clinical development is to finish the first study proving CBD’s effectiveness. The next step will be to start an international multi-center phase III clinical trial. However, it will surely require the participation of associations, foundations, and pharmaceutical corporations.
Further clinical trial and research details
To date, the project’s preclinical efforts have been funded by the government, according to the researchers. Clinical trials, however, call for a significant infrastructure. Here, we talk about recruitment, hospitals, and the production and distribution of drugs. When it comes to a rare disease like Leigh Syndrome, a global network of hospitals and patients will probably need to work together. The expenditures of the experiment are greatly increased by all of these considerations.
For this reason, UAB Neurosciences Institute’s researchers have established connections with a number of associations and organisations. As dr. Quintana says they are beneficial in locating funding sources for the trial’s early stages. Furthermore, they never stop searching for possible financiers to support the trial. Long-term, though, pharmaceutical corporations would need to be involved.