European Cavernoma Alliance
- a network of national patient advocacy associations -
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RESEARCH NEWSFLASH
RESEARCH NEWSFLASH
New study: Drug compound reduces abnormal blood vessels in the brain
New study: Drug compound reduces abnormal blood vessels in the brain
In a new study from Uppsala University, researchers have identified a compound that reduces blood vessel malformations in the brain associated with cerebral cavernous malformation (CCM). The compound is already used to treat other conditions, potentially offering a relatively fast route to a drug treatment for CCM—and, researchers hope, an alternative to surgery, currently the only treatment option.
The researchers found that the compound could reduce both the number and size of these vascular malformations.
“We screened more than 5,000 compounds in cultured blood vessel cells from a mouse model of CCM to identify candidates that could restore normal function in the damaged cells responsible for the disease. We then investigated the most promising compounds in cultured human brain blood vessel cells and animal models of CCM. Dabrafenib produced the best results, effectively counteracting the disease-related changes and slowing the development of malformed blood vessels. Most notably, dabrafenib affected two separate signalling pathways involved in disease progression,” says Maximiliano Arce, a researcher at the Department of Immunology, Genetics and Pathology and the study’s first author.
To investigate dabrafenib’s potential as a treatment for CCM, the researchers examined its effects in a mouse model of the disease. In addition to reducing the number and size of vascular malformations, treatment with dabrafenib resulted in more stable blood vessels with less leakage. It also repaired malformed blood vessels in the retina, which can also occur in some people with CCM.
One advantage of dabrafenib is that it is already used as a medicine, including to treat low-grade glioma, a type of brain tumour in children. The findings suggest that it could have a new application in the treatment of CCM.
“Because dabrafenib is already approved by the US and European medicines regulators and has an established safety profile, it could potentially be brought into clinical use relatively quickly to stop the growth of cerebral cavernous malformations,” says Peetra Magnusson, who led the study.
The study was conducted in collaboration with researchers at the SciLifeLab BioImage Informatics Unit and has been published in Science Translational Medicine.
Link to the study: https://www.science.org/doi/10.1126/scitranslmed.adr0448
[Text: Kerstin Henriksson, Institute for immunology, genetics and pathology (IGP), Uppsala University]
European Cavernoma Alliance: Organisations and patient groups
European Cavernoma Alliance: Organisations and patient groups
Our vision, mission, values & goals
Our vision, mission, values & goals
We envision a future:
We envision a future:
in which every cavernoma patient in Europe will easily find access to specialist doctors who are well-informed about the disease AND about ongoing research
in which researchers, doctors and patients have joined forces to improve care and find a cure.
Mission:
Mission:
As a European network of national patient advocacy organisations, it’s our mission to empower cavernoma patients in Europe by providing them with high-quality information, resources, tools and emotional support. We promote creative, yet strategic interventions and stimulate productive collaborations between researchers, patients and clinicians in order to accelerate better care, treatment options and ultimately to find a cure for cavernoma/cerebral cavernous malformations (CCM).
Values:
Values:
Our values are to be Collaborative, Informative, High quality, Empowering, Supportive and Trustworthy.
Goals:
Goals:
European Cavernoma Alliance is a network of cavernoma patient advocacy groups that are working together to improve the lives and prospects of cavernoma patients (and their carers) by adopting the following goals:
To promote the creation of CCM centres of expertise across Europe
To promote therapeutic care pathways at European level and national emergency guidelines for better early intervention after a haemorrhage stroke caused by a cavernoma
To stimulate research and development of treatments by increasing the communication between patients, researchers and clinicians
To help countries without a local cavernoma patient association to form their own association
To raise awareness of cavernoma among the medical community and public.
About Cavernoma
About Cavernoma
Fast Facts (Alliance to Cure Cavernous Malformation, USA)
Spinal cavernoma (Cavernoma Alliance UK)
Patient brochure (Alliance to Cure Cavernous Malformation, USA)
Information leaflets (Cavernoma Alliance UK)
CCM Care Guidelines (Alliance to Cure Cavernous Malformation, USA)
Questions to ask your doctor (Alliance to Cure Cavernous Malformation, USA)
Questions to ask your doctors (Cavernoma Alliance UK)
The science of cavernoma (Cavernoma Alliance UK)
Treatments in development (Alliance to Cure Cavernous Malformation, USA)
Webinars and videos (Alliance to Cure Cavernous Malformation, USA)
Webinars and videos (Cavernoma Alliance UK)
Bergholtz_Final_28feb.pdfInsights
Insights
People with cavernoma (also known as cavernous angioma or cerebral cavernous malformations, CCM) typically arrive at emergency care with stroke-like symptoms such as paralysis and neurological deficits (e.g., vision or speech impairment). Others have epileptic seizures or serious headaches. It often takes a long time until we get the right diagnosis, because an MRI scan is needed (a CT scan is not enough!).
In this article, Jana Bergholtz (founder of Cavernöst Angiom Sverige-CASE, Sweden and currently leading the efforts of the European Cavernoma Alliance) talks about what cavernomas are, our search for equal access across Europe, more clinical research and better person-centred care. Even though there is still no treatment except neurosurgery, we are certain there is a solution, not only for us, but many others with cavernoma, still undiagnosed.
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