$70,000 pledged to new drug

Yesterday, Alex’s Wish pledged $70,000 to support the next step in bringing VPB15 (a new novel anti-inflammatory steriod drug) to boys with Duchenne Muscular Dystrophy.  The drug is currently in Phase 1 clinical trials and this money will help support the work needed to take the drug into Phase 2a and Phase 2b, with the possibility of gaining accelerated approval at this stage, should the results prove to be successful.

The upside of VPB15 is that it has far less side effects as seen with other steriods treatments currently available on the market including stunted growth, boned fragility and immunosuppression meaning that more of the drug can be administered.  This new treatment is likely to be used for other conditions, not just Duchenne Muscular Dystrophy.

Alex’s Wish is very excited to be supporting this exciting project and hopes the new drug, if the trials are successful, becomes available in the next few years.  Alex is currently on daily steriods and has to attend regular bone density scans at Birmingham Hospital as well as regular weight check ups to monitor weight gain. Emma Hallam says ‘We are already noticing Alex putting on weight which is a real concern as this makes it more difficult for him to move around and also puts extra pressure on his heart.  It would be absolutely amazing to have a new steriod available in the near future that will eliminate such side effects.’

We would like to thank all of our Wish Warriors for supporting our cause, without you we would not be able to fund these exciting new clinical trials.

Posted in Funding