Pilot Trials for Duchenne Muscular Dystrophy
Work with partner organisations to run clinical trials of approved drugs that show promise for the treatment of Duchenne Muscular Dystrophy. Parents of boys spend a lot of money on supplements that are believed to provide benefit. Pilot Trials will provide the qualitative results they deserve.
January 2011 - January 2012
Charity information: Action Duchenne
Currently the only medication available is steroids which may (at best) keep diagnosed children walking for additional two years and are accompanied by negative side effects including radio weight gain and loss of bone density. Bottom line: the disease remains 100% fatal, with most boys dying in the teens or 20s. There is not one approved drug offered to those suffering with Duchenne.
We will run clinical trials on FDA approved drug IGF1 which shows promise in the treatment of Duchenne. By supporting this trial we are finally giving boys with Duchenne a chance to save their muscles by triggering continual regeneration of them and building muscle strength. This will help to slow the severe and fast muscle wasting which is the primary cause of early death.
Show if a drug commonly used for other conditions can provide benefit for children with Duchenne.
Activities» Running a pilot trial on 20 patients for 1 year to show if improved muscle strength occurs and no harmful side effects are experienced.
What success will look like
Success will be demonstrated in the results gathered by the clinical team of the trial of IGF1 on 20 patients.
If successful, to provide regulatory approval for this drug for use in treating Duchenne.
Activities» On the basis of the data from the trial, submission for approval to the FDA and MHRA in the UK for the drug to be licensed for use of patients.
What success will look like
Approval from the FDA and MHRA in the UK for the drug to be licensed for use of patients with Duchenne.
If the drug is shown to be safe and effective then long term administration will slow muscle wasting and extend life expectancy. For the first time ever in the history of Duchenne, groundbreaking new therapies are being developed. This is the first generation of children who have a real shot at benefiting from these therapies. While these drugs wind their way through the regulatory process, this project will keep these young people “in the game” until the new treatments are available.
There is a risk that the drug will show no benefit to those living with Duchenne. This is a risk associated with all clinical trials. Should this occur this information will be distributed worldwide and IGF1 can be ruled out as a treatment with focus going toward other FDA approved drugs such as sildenail (commonly known as Viagra).
Published in medical journals and disseminated by partner organisation members who are parents and boys living with Duchenne. Key milestones set with the clinical team and these updates will be provided in newsletters and online for donors and at the International Duchenne Conference in London.
Budget - Project Cost: £400,000Loading graph....
Amount Heading Description £70,000 Clinical Personel Clinical Personnel £70,000 Patients Direct Patient Support £60,000 Peronnel Other Personel (including project coordinator and data management) £40,000 Administration printing of reports and other general admin £60,000 Medicines eg IGF1 for 20 patients £100,000 Research Principal Investigator and other research personnel
Current Funding / Pledges
Source Amount Charley's Fund and International Consortium £175,000 Guaranteed Action Duchenne £185,000 Guaranteed
The project if successful will provide benefit worldwide to Duchenne patients (currently there are over 100,000 boys and young men living with Duchenne worldwide). Action Duchenne will work with the 2000 boys and young men in the UK to ensure the treatment is delivered and information disseminated.
Boys and young men worldwide will benefit. Parents will receive on the first treatments for this life limiting condition generating hope and buying time for their sons for the further advancements currently being made.
The charity remains parent and patient led with the majority of staff and all trustees directly affected by the disease. This ensures we remain rooted within the community we serve.
Action Duchenne is unique in that it is the only charity that works exclusively with Duchenne Muscular Dystrophy. Volunteers play a considerate role and are parents of young children with Duchenne, family members and young men living with Duchenne.
Read more about the Charity running this project.
Parent who founded Charley's Fund and has overseen $2.5 million of Duchenne clinical trials.
CEO of Action Duchenne. Since 2001 Nick has actively overseen and managed £4M of research and development into Duchenne.