Project Catalyst : what is the present status of their utrophin upregulation drug candidate

Initiated by PPMD in 2003 Project catalyst was born out of a belief that present generation of young boys deserves a decisive and positive response to the challenges they face.

                     We are in the end of q1 2012, but there are not a single clinical trial by them. I emailed them about utrophin upregulation drug and they replied me on 07/11 that they expect utrophin compound in discovery to be in preliminary trials in the next 18 to 24 months.

                             My son ( my only child , 6y old) is not eligible for either " exon skipping " or "stop codon". After his diagnosis  I am listening about exon skipping trial news. My sincere wishes with them. But the rest of 85% boys including my son also deserve a positive treatment other than steroid.So, my only hope is Utrophin upregulation drug. ( considering the fact, that ace 031 is on hold)At this point I can see only SUMMIT's motivation that they are starting ph 1 of their reformulated drug. Other utrophin drug like TAT Utrophin and DR. Khurana's compound are still in preclinical stage( as per my knowledge).

             Pat , you are our strength. Could you please influence project catalyst's utrophin drug to be in clinical trial in 2012. The field is ripe, the horizon is nearer ( with all your effort) ,so what for we are waiting?

I am all ears to listen something from you or Sharon.

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Too late for that. I am already in the club and lost any hope that my son will benefit from most of these. I think this generation of boys is used for testing these drugs and have a low chance to benefiting from them. Things are moving very slowly w/o any explanation and I am not buying that these companies are doing all they can to advance these drugs as fast as possible.

Rupjani B said:


That is my point also.I think we must concentrate only on utrophin drug.Because

1. there will not be any immunological issue.

2. will be beneficial for all boys

3. much simple process

we need any positive drug immediately so that we can buy some time for our boys and then focus on other treatment.We don't want to expand "frustrating parents " club.


I know and respect that PPMD is working to change the system. There is no other organization that I know of that works harder in that regard.

But since my son was 6 (as Rupjani's is now) I was consumed with such dates for this project and that trial, and this conference, etc. I don't want to even think about all the energy I spent tracking those projects and looking for press releases when the dates would come around. I was realistic - I understood there would be delays, no stranger to medicine.

7 years later it's clear to me it was all a waste. I want those hours back. It seems to me that unless one has access to great wealth (their own or via foundation), and can directly control the drug development, these dates are simply meaningless numbers that have a purpose other than to inform those interested in the project. They are all simply wild guesses, that have more to do with the current year's conference schedule than the reality of the development process.

So I say to parents advocate, donate, raise money, do what you can today, this week, this month. But forget about the future dates of this trial, the next. Let the PPMD staff lose all the sleep. It will only serve to dampen your spirit, and rob you of time better spent away from the computer. ;-)






Mission accomplished, lol!  I do lose considerable sleep over these deadlines. 

And I'm pretty sure that Pat never sleeps at all. I think the only thing I would take issue with is the idea that you can control the time lines if you own the drug/company.  One interesting model that is kind of a hybrid is the idea of a "non-profit" biotechnology company.  We've thought about that at PPMD.  The ALS Therapy Development Institute follows this model (  The advantage is that you are not driven by shareholders or investors (who do put pressure on companies to do things fast but are also capable of putting pressure on companies to change directions). This is a little bit different from a family owned company because it's not for-profit and it's not focused on a single child.  You still have to raise money, but all the drug development work is done in-house and you can be very transparent about where you are in that process.  The disadvantage is that you are supporting a lot of infrastructure that can be hard to shut down if it's not working (with a grant or milestone investment you can just cancel your funding and go fund someone else).  But it's an interesting idea. 

I actually hate it whenever a company puts up any kind of time line because I know they don't really know how long a project will take.  You are right--they are all guesses.



I dont mind companies putting a timeline, even if it is an 'educated guess'....what bothers most of us is not putting any information out and maintaining complete silence or repeating meaningless phrases once things go out of schedule (ace31) is irresponsible at best and criminal at worst.

I started this discussion to hear" some news" from PPMD not a "forward looking statement".PPMD did a lot, we are here only for them. But which is most alarming is that the pace of drug development. We are still there where David was 7 yrs back. I am not a drug development expert , I don't want to be. How long it take to develop a drug to treat DMD like severe medical need. I think for a serious fatal disease only time matters. We have world class, dedicated scientists in our community.They are simply "the best."After all we are not living in the polio or chicken pox drug age. We are living in the

e-age where everything is possible with a finger touch. What I mean to say if A group of researchers faces a hurdle they can share with  B group and can pass on their information to the C group so that they don't have to start from the begining.

How many years we will console ourselves with research news, conferences, events etc....After how many lives the policy makers of drug companies will sit with a coffee cup and tell, well, now its time for DMD DRUG !!

Amit -

Daniel Kahneman, a nobel prize winner in economics and a reknown pyschologist, wrote a great book called "Thinking, Fast and Slow" where he explains the "expert fallacy." He argues that tor anything except very short-term estimates, the experts have no better ability to estimate dates than a monkey. That's not an insult to the experts. It's simply statistics. In other words, the "educated guess" is just a good as a random guess generated by you or me.

After reading the book I came to the conclusion that the same thing applied to this domain. We are listening to experts about things they CAN say (drug worked, drug did not, medical processes, etc.) and expecting them to also provide us with some idea of what the future holds. But, statistically speaking, their guess about future dates of the project is as good as ours; worthless.

Rupjani -

It has been my experience that PPMD has worked hard to improve the development process as you suggest. I wish we as parents could do more, but I wouldn't know where to begin.

But when it comes to information about dates or "pace", they will only repeat what the core project team has already said publicly. They can't give "inside info" even if they had it. This information is very, very closely guarded. Just the posting of comments by parents on this forum and others like it have riled feathers of companies with projects in the works (as if anyone would listen to us!).

I hate the way that such information is guarded. But to date, I've found no way to change that.


You may be interested in this url as it is very interesting.  All of these nonprofits who raise money have to agree to transparancey and to share results and problems they encounter.  This came about in part when a DMD parent gave a researcher $10,000 for a DMD project then to find out later this researcher was fully funded by NIH for that project.  It also has a streamline single application for researchers.

 Hi Cheri 

Thanks for posting this. So, we have everything; a very strong community, world class scientist, funding, everything. But we don't have two crucial things; TIME and TREATMENT for our boys. I just can't believe this!


This alliance has just recently started and has made great strides.  One of the best things is the non profits and NIH all had different application forms and this was very time comsuming.  The allieance is working with NIH to have a single application that can NIH and 21 DMD non profits can use.  Hopefully you will hear more about it at the PPMD conf this summer.  Our son will be 21 this fall and so time is not on our side either and we have been though a ton in trying to get a medication for him which is a very long story, google my name if you'd like to read more.  You might want to look at the way SMA is funded at NIH.  They used a differernt route and was one of the first to fund based on aggessive timelines.  You don't meet the timeline you don't get funded.  I am very wary of putting all you apples in one basket so to speak as who know what will work.   I have spoken with Dr Rusty Katz the head of the nueropharm div of the FDA and I believe they are doing the best with what they are given and would like to see changes to benefit the patient and keep the patient safe..  Note the drug companies not the FDA determine the endpoints and how fast they get the data to the FDA.  When i worked with the FDA they had a 30 day turnaround time for our application which is very fair.  Keep pressing and getting informed!  Cheri

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