On August 23, 2010, Advocure NF2 was invited to participate in a conference call with both Bruce R. Korf, MD, PhD (Principal Investigator of the NF Consortium) and Dr. Roger Packer (Chair of the Consortium), in order to clarify the NF Consortium’s timeline on NF2’s inclusion for trials.
Summary of Issues/Responses
- The NF Consortium was formed following a meeting jointly sponsored by the DoD and NIH. The main impetus was recognition that clinical trials were difficult to conduct in any single institution, and the complexities of regulatory requirements make it desirable to have a central operations center to facilitate these issues. At the time of the original meeting, the NF2 researchers present encouraged the group to focus first on NF1, in part because at the time there were no drugs available to test in NF2 patients and in part because they recognized the complexity in establishing the NF Consortium.
- It was very complicated to get up and running, including getting significant funding. It has required several years to get the infrastructure up and running smoothly.
- The centers were chosen by DoD following an open competitive process – they made a significant contribution of funding.
- Most of the NF Consortium sites have their greatest expertise with children with NF1, as the original protocols were mainly tailored to this group (see point #1).
- Dr. Scott Plotkin’s proposed trial fits well as he wants it to initially be focused on 12-30 year olds. Genentech is financing some of this and wants as few sites as possible, to be able to adequately audit these sites. This effort has been between Scott and Genentech.
- If Dr. Scott Plotkin can get Genentech to agree to other additional sites, its fine with them. But again, there would need to be significant funding and coordination to get them up to speed.
- A site also must be prepared to cover some of the shared expenses not covered by the grant.
- They guesstimate that each hospital pays more than $100,000 for this operations support.
- Any center that participates has to pledge that NF Consortium protocols will be given priority, so as to avoid biases, e.g., keeping out the most difficult cases, to avoid artificial positive or negative results.
- Some centers deemed more appropriate for NF2 declined to be part of this, as they have other drugs they’re testing and don’t necessarily want to prioritize the NF Consortium drugs.
- Because of the orphan drug status, it is an easier step for a pharma company to stop/pull out of the trial if it becomes problematic.
- The way the NF Consortium works is that the centers that are part of it must be included in the study, they can’t be cut out. Other sites can be added on a case-by-case basis according to their ability to participate and the need to include them to achieve the aims of a particular study.
- In terms of a timetable, under normal circumstances this might be 6 months, but due to the complexity including contract negotiations that haven’t even begun yet between Genentech and DoD, this will be 8-9 months away at the earliest.
- DoD has been great and actually is helping streamline the process going forward.
- It is believed that centers will work with other hospitals nearby to advise NF2 patients of this trial. An example of this would be Cincinnati working with Ohio State.
Q & A with Dr Bruce Korf
Q: How will patient care be monitored during this trial if a patient from Ohio State participates with Cincinnati, for instance (or a patient from House participates with Utah?)
A: There are strict requirements for monitoring patients stipulated in the protocol. Some of these can be done at a remote site (like MRIs or blood tests); others require travel to the NF Consortium site.
Q: Will travel to a NF Consortium center be required? If so, who pays for the travel?
A: It is necessary to travel to the NF Consortium site to get access to medication and for some evaluations. Funds are not available to support travel. In some cases for other protocols we’ve been able to get free flights, etc., but otherwise the patients pay for this.
Q: Going forward, are there NF2 sites slated for inclusion, e.g., MGH, HRI, NYU, OSU?
A: It is my hope that we’ll be able to submit a proposal in 2011 – 2012 to renew the NF Consortium and in that process it will be possible to add new sites. I’d strongly favor including major NF2 sites in that proposal.
Q: Is there a patient database that will be available for other drugs/centers in the future?
A: There is no central patient database associated with the NF Consortium the funding is related to specific trials, not to creation of such resources. Each site tends to have its own database of patients whom they can call on.
Q: Is there collaboration with NIH, CTF, and NF Inc?
A: NIH is one of the sites involved in the NF Consortium. CTF and NF Inc are familiar with the consortium and tend to refer patients to us if they inquire. I believe that they link to our website as well.
“It would not be possible to conduct NF2 trials without including additional NF2 specific sites. The operating procedures of the NF Consortium include the ability to add sites as needed for specific studies and the Consortium is aware of the need for NF2 sites to support a trial.”
– Dr. Naba Bora, director of the NFRP, at CDMRP.
Q: Does the NF Consortium conduct trials for all kinds of neurofibromatosis?
A: Yes. The NF Consortium is now set up to conduct trials for patients with NF1, NF2, and Schwannomatosis.
As of spring, 2012, the NF Consortium sites are as follows:
- Ann & Robert H. Lurie Children’s Hospital of Chicago
- Boston/Harvard Center for NF and Allied Disorders
- Children’s Hospital at Westmead, University of Sydney
- Children’s Hospital of Los Angeles
- Children’s National Medical Center
- Cincinnati Children’s Hospital Medical Center
- House Research Institute (HRI)
- John Hopkins Hospital
- Massachusetts General Hospital (MGH)
- National Cancer Institute
- New York University Medical Center (NYU)
- Ohio State University Wexner Medical Center (OSU)
- University of Alabama at Birmingham
- University of Chicago
- University of Indiana
- University of Pennsylvania
- University of Utah
- Washington University