The FACT Alliance

Progress through collaboration

by Jamshid Arjomand, PhD, Chief Science Officer

As a fan of home improvement shows, I’m fascinated by the evolution of going from an idealized dream home or floor plan to the ultimate result. Compromise is always a key factor in decision making, especially when resources and time are limited.

Scientific research is no different. Most scientists often need to compromise between the ideal set of experiments and the ones they can practically execute in a timely way. Pharmaceutical companies also operate within similar constraints, albeit with larger budgets but more urgent deadlines.

In tackling FSHD, most companies face similar questions and hurdles to move their therapeutic solutions forward. Left on their own, each A company will need to prioritize their resources and design practical experiments to address the most critical issues facing their programs. Often, these compromises fall short of the ideal, and their limited resources may even prevent them from addressing all the key questions they need answered. Another downside of this type of siloed approach is that results of these limited experiments are seldom made public, so different companies end up repeating the same ones, unaware that these questions may have already been addressed.

Breaking out of siloes

One way of accelerating therapeutic research and optimizing resources, while minimizing risk and redundancies, is to promote collaboration among pharmaceutical companies. To that end, the FSHD Society has developed a program called the FSHD Assessment and Clinical Tools Alliance, or FACT Alliance. The program is designed for pharmaceutical companies to join forces in tackling common and important questions pertinent to drug development in general.

For example, MRI scans have become an important tool for tracking muscle loss and fatty infiltration of muscle in FSHD. Unfortunately, datasets of MRI scans are not readily available, and although studies like MOVE+ are designed to collect new scans in an ongoing manner, researchers may not have access to the data for some time.

As an initiative of the FACT Alliance, the FSHD Society is working with key imaging researchers to see if historical scans from small natural history studies can be pooled together and re-analyzed with cuttingedge technologies to provide a more detailed “picture” of the changes in the muscles of FSHD patients.

The benefit of this approach is that expensive MRI scans from past studies would be available for immediate analysis, and newer technologies could be applied to re-analyze results from a larger number of study participants.

For pharmaceutical companies, a cooperative approach with other companies would allow them to get results from a larger study than they would have been able to obtain on their own. This would certainly be less expensive and faster than collecting new scans. For FSHD research in general, this type of approach also ensures that results are put in the public domain for every future company in the field to build on.

It takes a village

We know this approach works and is attractive to pharmaceutical companies. The proof of concept was already demonstrated last year with the successful launch of our genetic testing program, TestFSHD (see FSHD Advocate 2002, Issue 1). In this initiative, six pharmaceutical companies joined forces with the FSHD Society to co-fund a fully sponsored genetic testing program for the FSHD patient community. Although limited to 150 participants, the program was hugely successful and provided a muchneeded resource to the community Toward a better life while increasing the pool of eligible patients to enter clinical trials. Drug development is a team activity, and collaborative partnerships are essential to ensure steady progress.

As the saying goes, “It takes a village.” The FACT Alliance is designed to harness the power of collaborations among competing companies in areas of common interest that would benefit them all by reducing their risk, providing them with more comprehensive studies, and, most importantly, speeding up the development of treatment options for the FSHD community.

We are all part of this village. None of these activities would be possible without the brilliant FSHD researchers and clinicians, the dedicated biotechs, and the selfless support of the FSHD families.