Running through a lot of different conversations here at the conference is a common thread that reflects the progress being made in the search for therapies to treat neuromuscular diseases.
Case(s) in point:
Clearly, it’s exciting times, and much of that does have to do with the number of clinical trials going on — or soon to be going on — for neuromuscular diseases.
But, first things first. There’s a lot that goes on in getting drugs to market that comes prior to testing in people — and that was the subject of the vast majority of today’s talks. Preclinical testing in preclinical (cellular and animal) models is not only valuable in terms of making sure the best therapeutic candidate — a safe and potentially effective candidate — moves forward. It’s necessary. And as with every other stage of drug development, it’s not without its own set of challenges.
One of those challenges — perhaps one of the most significant — is overcoming the differences between disease models (particularly mouse models) and humans. These differences underlie the numerous cases where drugs have shown promise in mice, only to fail to demonstrate efficacy in human clinical trials.
Those differences also form the basis for the oft-asked question: Is the mouse a good model for human disease?
A number of world-class researchers offered their thoughts on these questions today. For those of you reading this post, I thought I’d share what Cathleen (Cat) Lutz at the Rare and Orphan Disease Center, Jackson Laboratory, had to say.
Cat noted in her presentation that whether or not the mouse makes a good model of human disease depends on several things, including the research questions that are being asked, the particular model that is being used and how the experiment is conducted.
Mice and humans are 98 percent identical in gene composition, she noted, but there have been millions of years of divergence ...”so get over it!”
In order to make the best use of a model, Cat said, researchers must:
Finally, she added, “Do not fall in love with your preclinical data!”
“Testing therapies in a mouse model is very helpful to determine if a therapy will possibly be beneficial in patients, and it’s exciting when we see marked benefit in mouse models,” she explained. “With that said, mice are not humans, and while strong preclinical data is encouraging, we need to be cautious in not overpredicting the data.”
Cat’s aren’t the first and final words on mouse (and other preclinical) models in neuromuscular disease research, but they provide a brief overview of prevailing thought — at least here; at least for today. Curing a mouse is not the same thing as curing a person, but it’s helping us get there!