The Myelin Repair Foundation, a non-profit group that focuses on multiple sclerosis research, will use web-based software from Collaborative Drug Discovery to improve how it collaborates with its sponsored academic researchers, the organizations said this week.
CDD’s software will specifically allow MRF-sponsored researchers at five US research universities to selectively share data with each other to identify and validate targets for myelin repair drug targets — and potentially with biotechnology or pharmaceutical partners to further develop the targets into new MS treatments, a foundation representative said.
Based in Saratoga, Calif., MRF is one of a growing number of non-profit medical research foundations that is attempting to bridge the so-called development gap between early-stage basic biomedical research and mature drug targets or candidate compounds that industry partners might want to develop and commercialize.
However, MRF believes its model is unique among research foundations in that its Accelerated Research Collaboration program is providing an organizational structure akin to a biotech of pharmaceutical company with the goal of supporting highly collaborative and applied research at academic research institutions.
When MRF introduced the ARC model in 2003, the goal was to “change the way the basic science was conducted in academia in order to make the nature of that research more outcome-focused, and really get top-flight basic scientists who heretofore had been concerned with publishing to think about doing things that would really have some practical application from a therapeutic standpoint,” MRF COO Rusty Bromley told BTW this week.
According to Bromley, MRF founder and MS patient Scott Johnson became interested in myelin repair as a new alternative to treating the disease after a breakthrough report on Schwann cell transplantation emerged earlier this decade.
“We are, from one standpoint, an orphan indication,” Bromley said. “There has been lots of research on MS, there are several drugs on the market, and sales of those drugs exceed $5 billion per year. But they’re all focused on modulating the immune system. Very little was being done … to look at neural repair in neurodegenerative diseases, and in MS in particular.”
But when Johnson began exploring what leading academic researchers were doing in the area of myelin repair, he found what Bromley called a “dysfunctional” system that failed to facilitate the type of collaborative research that could more quickly develop potential treatments for MS.
Part of that, Bromley said, was identifying potential areas of intellectual property development and commercialization opportunities early on — something that university tech-transfer offices would ideally handle, but often lacked the resources or funding to do.
“We volunteered to take that responsibility because we’re working with the researchers to plan experiments, we’re following the execution of those experiments, so we’re going to identify IP opportunities that they would have never recognized,” Bromley said.
“We volunteered to take that responsibility, because we’re working with the researchers to plan experiments, we’re following the execution of those experiments, so we’re going to identify IP opportunities that they would have never recognized.”
He said that MRF will “help with the invention records, hire the patent counsel to file the patents, [and] prosecute and protect those patents,” and in return the foundation wants two things: The ability to consolidate any resulting IP into a pool that MRF manages and actively promotes to potential industry partners; and a 50-percent cut of any revenue that might come out of such commercial partnerships.
Bromley said that several people told him “we were out of our minds” to suggest such a model and that no one would ever be interested in participating. “Yet the first three universities signed up in 120 days, partly because we sat down with them to talk about how we could make this a win-win situation,” Bromley said.
MRF now has a core research group comprising prominent myelin repair scientists from Stanford University, Case Western Reserve University, Northwestern University, and the University of Chicago. It also recently added a neuropathology expert from Johns Hopkins University.
Bromley said that since the research collaborative was formed, it has identified 18 putative drug targets for myelin repair and has chopped from 20 years to five years the time it expects it would take to validate a target that could attract industry. Furthermore, he said, the group has reached a point where approximately 80 percent of experiments being designed by its members “cannot be completed by one of the four labs alone.”
Recently, as the foundation turned its attention toward validating some of the myelin repair targets it found that its collaborative research strength had become a bit of a hindrance.
“What this does is impose a different level of communication” between laboratories, something that academic researchers traditionally are not known for, Bromley said.
Early on, he said, MRF envisioned web-based software like that offered by CDD, but some of the foundation’s industrial consultants recommended against it. “The concern was that if we gave people too much functionality, and they didn’t see the immediate benefit of the functionality, it would languish,” Bromley said. So the group tried traditional data-sharing conduits such as e-mail, web and teleconferencing, and face-to-face meetings.
“We spent a fair amount of time looking around to see who could build us a system that would enable us to manage those three activities: the discovery of new therapeutic targets, the validation of those targets to industry standards, and then, ultimately, a coordinated effort for the early stage of drug discovery,” Bromley said. MRF eventually found those capabilities in CDD’s software, he added.
According to CDD, its software allows participating researchers to share data with a spectrum of permissions: either selectively with just a few specific colleagues, openly with the entire scientific community, or not at all. This flexibility, it claims, encourages data sharing where appropriate while protecting intellectual property.
The software also excels at capturing and organizing fragmented data that would otherwise remain dispersed across multiple laboratories, CDD said. Foundations like MRF can set up and manage collaborations involving multiple research groups, and the central database ensures real-time access to the results of sponsored research through a web portal.
“We allow different scientists at their own discretion to decide if, when, and, to an extent, with whom they want to share data,” Barry Bunin, founder and president of CDD, told BTW this week. “You may have some data that is 100 percent private within your own group, or you could have a group where you could share one particular target or set of drug leads with a collaborator. That could be an academic working with a startup company, or one company or institution working with us on all their data.”
Financial terms of the agreement were not disclosed.
CDD, based in Burlingame, Calif., works with a number of non-profit research and academic institutions, as well as a small number of biotechnology companies, to manage their data using its software. Bunin said that in the past year the company has tripled its customer base. The company currently lists 34 organizational customers on its website.
The reason the collaboration with MRF was so important, Bunin said, is because it’s “such a good fit because our philosophies and values are so aligned.”
The MRF ARC program tells researchers that “we’ll fund you, but you have to be willing to work together more than you traditionally would,” Bunin said. “CDD can take that to the next level in terms of finding out what the best targets are for myelin repair – they can now instantaneously share their data with each other securely through the web.
“In MRF’s case, there are two different groups – one for validation to pick out the right targets, and the other to pick out the best drug candidates,” Bunin added. “That’s a microcosm of what we’re doing with researchers at other institutions.”
The end result, MRF’s Bromley said, is that the foundation can use the centralized data to pick out “the best of the best assays” from within the group, standardize those, and contract their development with outside labs so the foundation can validate them both internally and externally.
“Now when we show up at a pharmaceutical or biotech company and say, ‘Here’s a target we’ve identified, and here is some preliminary lead information about where we might find a therapeutic;’ when they review that data, it will meet their quality standards,” Bromley said.