Webinar: Accessing Emerging Treatments for Childhood Dementia
This webinar was held on 8 September 2023. With the limited number of treatment options available for childhood dementia disorders, pathways such as Australia's special access scheme (SAS) are an important means to accessing emerging treatments.
This webinar covered a case where the SAS was used to obtain an experimental treatment for a child with dementia. It featured perspectives from the involved clinician, pharmaceutical company, and the family.
You can access a full transcript of the webinar here:
Get the transcript
Vicki Wong (Co-founder, Parasail)
“Antibody Enzyme Targeting for Degradation of Intracellular Aggregates: Special Access Treatment of VAL-1221 for Lafora Disease”
Dr Alan McDougall (Head of Neurology, Liverpool Hospital, South Western Sydney Local Health District)
“Importation and Use of Medication VAL1221 for Lafora Body Disease - Challenges of Obtaining a New and Unregistered Treatment”
Niki Markou (Parent and Advocate and Director Chelsea’s Hope Lafora Children Research Fund) will present:
“The Family Perspective”
Answers to questions we didn't get to during the webinar
VAL-1221 is specific for diseases where there is a build up of glycogen, for example Pompe disease and Lafora disease. Since glycogen build up does not occur in Sanfilippo syndrome, VAL-1221 is not expected to help with Sanfilippo syndrome.
There are enzyme replacement therapies for Sanfilippo syndrome being developed by other companies, more information is available on the Sanfilippo Children’s Foundation website.
How can we address clinical trials in rare childhood diseases when the number of cases is often n=1, unlike other common diseases where there are more patients available for clinical trials and more advocacy. Thank you - Dr Ramesh Narayanan
Thank you for your question Dr Narayanan, this is just about a topic for a whole other webinar! This is an issue that is only going to grow with the advent of more personalised medicines and I’m not sure anyone has an answer yet. More collaboration between clinical trial sites internationally with streamlined processes in place to set up new trial sites and utilising innovative clinical trial designs are some ideas. As always, funding is an issue, especially for expensive advanced therapies.
Bringing rare diseases together that have similar presentations (like childhood dementia) gives the opportunity for more effective advocacy for increased funding and clinical trials.
What is the first step if we are interested in finding a medication?
Keeping up to date with research news and understanding what opportunities there are can be a full time job and very daunting. Patient organisations, either locally or overseas, are often good sources of information. Asking your doctors and any other experts in the field whenever you can, may also be helpful.
It is important to note that accessing treatments through the special access scheme will not be possible for every patient. Opportunities where a company has a drug with adequate safety data that they are willing and able to supply, are quite rare. In this case, the drug was leftover from a previous trial. Manufacturing drugs is expensive and all of it is usually earmarked for clinical trials. Clinical trials are the most effective way to demonstrate a drug works, get it approved and bring it on the market for widespread use by the patient population, so this is usually the main focus of pharma companies. You can keep an eye on clinical trial opportunities on www.clinicaltrials.gov.
Queries about individual access should be directed to Parasail: email@example.com
Before you go: Join the Childhood Dementia Research Alliance
The many neurodegenerative disorders that cause childhood dementia collectively affect around 700,000 children and young people worldwide. We're driving accelerated action for all of these disorders and referring to them under the umbrella term ‘childhood dementia’.
We are bringing together the world’s brilliant health and medical researchers to work collaboratively to develop new effective treatments. The Childhood Dementia Knowledgbase is an example of the shared infrastructure and efficiencies in research Childhood Dementia Initiative is driving.
To stay up to date on the latest news and opportunities, to help us identify research priorities and increase opportunities for collaboration, join the growing Childhood Dementia Research Alliance.