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May 03, 2006
May - Part 2
I had a call from someone asking about Project Catalyst. What is it? What are "hits"? What does it all mean? And I hear the silent "when†in their voice. In short, Project Catalyst is our work with PTC Therapeutics (www.ptcbio.com) in drug discovery. The idea for such a project started long ago – the need to develop relationships with industry and thankfully today, several companies have a muscle program.
Providing support to academic researchers is wonderful because basic research is critical to the field, provides the seeds for developing treatment, but actually delivering medicines to patients requires industry.
In 2003, we initiated Project Catalyst, a major effort focused on identifying compounds that could potentially lead to treatments for ALL individuals diagnosed with DMD. With a group of experts in the field, we identified five (5) targets. PTC researchers set up screens (High throughput screens – HTS). What happens is that several million compounds (compound libraries) are run against identified targets. (A scientific experiment is designed to identify compounds that cause a certain reaction.) “Hits†– or compounds that have a positive reaction to the designated target – are identified. These “hits†are then validated to make sure the compound identified did have the specific effect. As you might imagine there are positives (degree varies), false positives, false negatives and all sorts of things in between.
First question: Does it work? (Was it a true “hit�) In addition some preliminary toxicity is conducted.
Second question: Is it toxic? (Does it kill the mice, rats, dogs?)
Third question: Does it have a desired effect? (Animal studies to demonstrate benefit).
If the answers to questions one, two and three are “yes†– there’s a fourth question: Can it be given to people?
As you might imagine, as the process goes forward, the number of viable compounds significantly decreases. At the end of the day, we are hopeful one or more compounds on one or more targets will be selected as potential candidates for trial. It is a lengthy process and we are now in the stage of testing the ‘hits’. This stage is called Lead Optimization - validation and toxicity.
Project Catalyst is moving forward and we are hopeful that by filling this drug pipeline, we will accelerate the development of treatments for all.
Happy May.
Posted by ppmd at 05:06 PM | Comments (4)
May - Part 1
The days fly by. It is hard to believe April is over.
After those long grey days of winter, spring is always so welcome. Long ago, I planted lilies of the valley right next to our front steps. That day, which seems a lifetime ago, all the kids were playing in the front yard (Jenny, Michelle, Chris and Patrick) and I was sitting on the step watching. It would be several years before I heard the words Duchenne muscular dystrophy, though I do remember wondering why Chris and Patrick were reluctant to ride their Hot Wheels. It was just a flash, a quick ‘hmmm’ and that uncertain worry planted somewhere inside and then forgotten. To this day, every time I drive into the garage, I find myself looking for those lilies and thinking about carefree days before Duchenne formally arrived. To me, they will always be a sign, though the interpretation changes on any given day.
April is both wonderful and difficult at the same time. Jenny, Michelle and Chris all have April birthdays. Chris would have been 28 on April 14th and Patrick died on April 29th. Of course, birthdays are highlights, wonderful days but on certain days, sadness sometimes sticks to me like cotton on a wool sweater. The more I try to pick it off, the more I find it sticks. It feels like two lassos spinning at once and in different directions, one of joy and one of sadness and sometimes they get tangled together, so I stop, take a breath and begin again.
Posted by ppmd at 05:05 PM | Comments (1)