Considering that BioMarin has scrapped their similar program, and that Dr. Davies is at Oxford, I'm very concerned that the next phase of trials for SMT C1100 will happen in Europe or elsewhere outside the U.S.
In my opinion when this happens the FDA uses it as an excuse to take "wait and see" approach and for those of us with boys in their teens, that is of great concern.
My son in particular will likely not benefit from exon-skipping or other mutation-specific treatments. Utrophin-based treatments appear to be the most directly applicable to him.
What can I, as a parent, do NOW to help ensure that he can participate in the first human trials? We just don't want to wait and see any more.
It is certain that there has been more support for exon skipping even though the % of boys who would benefit from it is low. One would think that the most money would go to support research/trials that would benefit all the boys rather than a small subgroup.
Summit is looking for funds for phase 1, possibly around a million dollars. It would seem that between MDA/PPMD/European organizations, that should be possible. The challenge for us (parents of non-exon-skipping boys) is to ensure that it happens.
I'm pretty sure but not certain BioMarin dropped their program. There were press releases to that effect last year, and it is not longer listed on their pipeline page
My concern is where the money goes. We need this in the US. Beyond helping raise the money, what can a parent do to encourage this trial to come to the US as soon as possible?
Not sure whether Biomarin is in the picture at all. Summit is the one now leading SMT-C1100.
Not sure at what stage PTC's utrophin program is. But Summit is certainly at the point where they can start phase 1 which will likely take at least an year to get to the clinic even AFTER they have the funds. They already did some studies in healthy volunteers and had promising results.
Yes, they believe the molecule will work, but there was a "formulation problem" at BioMarin.
The Biomarin trial had variability in the exposure levels in the blood streams of individuals. Summit believes that alternate formulation of the drug would correct the variability for consistent results and that would be the focus of the next trial.
Summit is currently looking for outside funds either via donations or partnering with another company to see utrophin upregulation through, not an uncommon event with biopharms anywhere. Even US biopharm companies take their trials elsewhere so geographical location of any company (ex Acceleron's trial in Canada) dosen't promise trials in any given location. Previously Biomarin had an agreement with Summit to work together on utrophin but released themselves from the menu, my guess is when there was a chemical set back & Biomarin didn't have enough funds to push further. Thank God Summit stuck with the program as they are committed to seeing this drug go into trials.
No matter what country trials take place, ALL boys benefit throughout the world. FDA has the ability to legally allow treatments to take place here in the states, if a drug is approved in another country especially if its related to a life threatening condition. We parents might have to make FDA do this but ok, what ever it takes.
Why do we believe that utrophin regulation works? We don't, not until we see what the trials bring. Why should we believe it, or any other chemestry, might work? Because we have to. That's all we have right now.
Keep in mind the ever changing landscape of DMD. The past few years has shown researchers & biopharm's working constantly. Prior to that there were no clinical trials for DMD. Progress, although never fast enough, IS being made. Something I am eternally grateful for even though my son is 12.5 and declining fast. Alex may not make it for some treatments coming down the pipeline but I know we parents will live to see this monster, Duchenne, whipped.