Dr Rhonda Voskuhl Discusses Financing Early Trials of MS Therapies

The challenge with translating good science into treatments for patients with multiple sclerosis (MS) is getting the funding to get the trials done, said Rhonda Voskuhl, MD, Jack H. Skirball chair of multiple sclerosis (MS) research, director of the MS program, and professor of neurology at the University of California, Los Angeles (UCLA).

Transcript

What are some of the biggest challenges, and how can they be overcome, of translating clinical insights of multiple sclerosis mechanisms into care?

The biggest challenge in translating basic science insights into clinical care lies in really financing of the early trials. So, I’ve done 4 clinical trials now, based on science in our lab, to come up with novel agents and, fortunately, I was funded by the National MS Society and the National Institutes of Health [NIH], that said, it can take up to 5 years just to get the grant. And then you go in for another one. And then, the trials of course, will take 2 to 3 years. And then, after all that, you’ll need to do another trial. These trials don’t go to the FDA before they’re done repeatedly and successfully and then, ultimately, you’ll have to do a phase 3 trial that generally could be a thousand patients and it could cost $20 [million] or $40 million, and only pharmaceutical [companies] do that.

Now, if you’re trying to develop a drug, like we’ve done trials with estriol, and I can give you the basis for that, it’s because pregnancy is good for MS—it reduces relapses by 70%, it could also have neuroprotective effects—and it’s a safe estrogen, it’s safer than birth control pills or hormone replacement therapy. The problem is estriol is cheap, and so, we’ve don’t 3 trials with it and we’ve shown cognitive improvement with it, but trying to get a phase 3 trial is hard with pharmaceutical companies because they can only charge so much for estriol. And currently the trials right now, they’re running $60,000 to $80,000 a year. And so estriol costs $700 a year.

And so the challenge lies at 2 levels. One is when you have the basic science finding and you know something could be good, you then have to go to the small trials and that can be done and I’ve done it 4 times, but that said it does cost time—they only cost about $1 [million] to $5 million, it depends on how big they are—it does take a significant amount of time to go through all the process over and over, the grants, the application to get it. And finally, you get it and you can do it. And then even when that is successful, then to take it that leap to phase 3 is very difficult if it’s not a secret, patented, protected product coming out of a drug company.

So, we’ve had, again, good fortune at the early stages with donors to UCLA that helped us get the ball rolling early, that helped us then get the grants at the NIH. And now we have 2 things that are kind of stuck here and it’s like, we would like to go big with them. Another one is testosterone for men with MS, who have low levels, and they can benefit with slow brain atrophy. We’d like to do more trials with that. They call it the valley where things get lost, and a lot of it is due to finances.

I mean, my vision, honestly, if I could do whatever I wanted to do it would be to have a clinical trial center at UCLA and a couple of others, where we would work together to just have a pipeline where we would take these things that are safe and we know a lot about them, and the science says they’re going to be good, and they might be cheap, and that’s fine with me, and we would run them through those trials. And even if we could do a lot of those at just the phase 2 level, we could make a big impact, because there’s a lot of science out there that never goes to trials because it’s just so difficult. And if we had this center where we could have one after the other after the other, there’s a lot of good candidates.

And some people take things off label, like coenzyme q or testosterone or vitamin D. But these really do need to be tested in a trial versus a placebo, very rigorously done. And you really need to see, what does it affect? Does it improve walking? Does it improve cognition? Or maybe one versus the other? Maybe we shouldn’t pull them all together. I mean, I think I know how to do this, it’s just a matter of getting the infrastructure to do it.