Currently in Brussels for the Treat NMD Advisory Committee for Therapeutics Conference.

How many times have we all received Google Alerts suggesting a certain drug "is potentially promising," “improves function in the mdx mouse,” or ‘halts degeneration in the mdx”? And then, silence.

And how many of us have discussed the need for a certain drug to go to trial or how many parents/families have tried to persuade or were successful at persuading their physician to write the script for a drug based on a single paper about a mouse?

Some time ago, we were asked to recommend a drug for trial in Duchenne. What drug? What evidence? What does the trial look like? How will we measure success? What should we expect? A band-aid? Better? Better than steroids? Will it end Duchenne?

Now there is a process in place in order to objectively review what is known about a certain compound, what level of evidence, what barriers/obstacles might exist in the process of clinical trial development. At the end of the day, this process will help us prioritize drugs for trial and to the degree humanly possible, improve our likelihood for success.

TACT – Treat NMD Advisory Committee for Therapeutics

Treat NMD has established a committee and process to review drugs for trial in Duchenne. This committee provides broad-based expertise to help identify the potential of therapeutics and guide promising ideas to a realistic development path.

Drug development has dramatically changed in the last 12-18 months. The era of the blockbuster is coming to a close and is replaced by drugs targeted to subgroups with the goal of better outcomes for patients. Tools exist to revisit molecules used for other diseases and/or previously considered not useful. It is important for proof of concept trials which are crucial in exposing failure (of the molecule) before raising patient’s expectations and spending vast sums of money.

It is good timing for the TACT process because orphan drugs in chronic diseases with previously underestimated markets, are generating deals. (PTC/Genzyme; Prosensa/GSK; Summit/Biomarin)

The process of TACT will be highly educational and transparent. It will provide a comprehensive approach for review and a perspective so that plans for clinical trials can move forward with some surety. At the moment, TACT has a list of 46 that could be considered potential candidates for trials. The TACT will ensure a level of objectivity so that the most promising compounds are moved into trials. We have a limited number of boys and want to work really hard to do the best job, testing the best drugs with a high likelihood for success.

TACT process will include:
• Consistent standards of drug development considerations for an informed review
• Scientific rational (objective - preclinical support)
• Study design
• Study conduct
• Did the right experts and groups have input?
• Will it inform next steps (go/no go) and the field in general?
• Plans for Funding, initial and beyond

The goal is to objectively review and provide clear answers for developers to bring drugs to patients (Faster to clinic vs best informed to clinic).

Who can use TACT?
Researchers, funders, clinicians, industry

You can check out an application at:

I'm extremely enthusiastic about TACT - It will help us make the RIGHT decisions about moving drugs into trial.

More to come...

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Comment by Ali Rashidkhan on November 19, 2009 at 12:09am
I am one parents who has a boy with 10 years old boy who has DMD and waiting and hoping

that researchers will find a real drug for my son and all boys which have this disease.

What do you mean (you have a limited number of boys)?

I am very glad that you are working hard and i only can pray for you.

Comment by Jacobs Mommom on November 18, 2009 at 1:38pm
Once again we thank you for being there for our boys:)
Comment by JUAN PEDRO ARBULU on November 18, 2009 at 10:39am
Thank you Pat for giving us the feedback of what is happening right sure you have an "army" behind you waiting for news every day.
chau (goodbye) Duchenne !!!
Comment by RAKTIM SINGH on November 18, 2009 at 12:40am
Thanks Pat for being there and updating us all (alomost on a real-time basis). It is very necessary that we have a proper structure in place which can objectively review all the potential drug candidates and can bring an efficiency into the process.TACT is really doing that.
Will be eager to hear more from you on all the actions of Brussels.
Comment by Anne Barbano on November 17, 2009 at 9:50pm
Hi Pat Delighted to find you here And to know you are so on top of all this. Always think of our quick deep talk in Burlington VT at VT Family Network Parents as Partners Conference. I send out your resourceful poignant interview we did to any that will listen. Keep up the fight and I wish you were on TWITTER but... found you!

Anne Barbano
Comment by Ofelia Marin on November 17, 2009 at 4:40pm
Hi Pat, Thanks for the update. Please let us know what/if new results are presented by Prosensa and AVI (I noticed that they intend to presents something).

14:30 – 16:00 Developing Novel, Disease Targeted Therapies and Systemic Delivery
Chairpersons: Serge Braun (AFM, France) and Kenneth Fischbeck (NIH/NINDS,
We will highlight novel gene directed therapies for neuromuscular diseases:
expected therapeutic benefits, delivery issues, and regulatory challenges. Exon
skipping is taken as a model approach for gene‐specific therapy. Invited speakers
will present the latest results of cutting‐edge pre‐clinical and clinical trials.
14:30 – 14:40 New results from Prosensa’s Exon Skipping trial (Judith Van Deutekom)14:40 – 14:50 Limb perfusion gene delivery (Jon Wolff)
14:50 – 15:00 New results from AVI’s Exon Skipping trial (Francesco Muntoni)15:00 – 15:10 New results from systemic AAV total body delivery (Luis Garcia)
15:10 – 16:00 Panel Session: Judith van Deutekom (Prosensa, The Netherlands)
Jon Wolff (Roche, USA)
Hong M. Moulton (Oregon State University, USA)
Luis Garcia (Institute of Myology, France)
Annemieke Aartsma‐Rus (Leiden University, The
Francesco Muntoni (University College London, UK)
Dominic Wells (Imperial College London, UK)
Alessandra Ferlini (University of Ferrara, Italy)
Brian Kasper (Nationwide Children’s Hospital, USA)

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