The Center for Neural Tumor Research
Director: Marco Giovannini, Ph.D.
In January 2008 Dr. Marco Giovannini founded the Center for Neural Tumor Research at House Research Institute with a mission to translate research on the cellular and molecular basis of neurofibromatosis type 2-related tumorigenesis into tools relevant for the treatment of the disease. To accomplish this programmatic vision, the members of the Center have begun to develop several research projects and initiatives in close partnership with clinicians at the House Clinic, and with a network of collaborators worldwide that are pivotal for accomplishing and widening the mission of the Center. The Center has attracted an international team of young, enterprising researchers who have been brought to this program by their unique interest in cancer research. To help advance the mission of the Center, Dr. Giovannini has recently been appointed as the Director of the Division of Clinical and Translational Research at the House Research Institute.
Need:
Neurofibromatosis type 2 (NF2) is a rare autosomal dominant genetic disorder associated with neurologic, ophthalmologic, and cutaneous abnormalities. The disease is caused by inactivation of the tumor suppressor gene, NF2, which encodes the protein merlin/schwannomin. Interestingly, though NF2 affects only 1:33,000 persons with a prevalence of 5466 people alive in the US population studies suggest up to 1:300 people will develop a tumor with an underlying sporadic NF2 mutation in their lifetime. This includes patients with unilateral vestibular schwannomas (VS) and meningiomas. Therefore, what we learn in developing therapies for NF2-related VS could have much broader clinical applications. Typically, NF2 patients present between the ages of 17 and 21 with symptoms such as hearing loss, tinnitus, visual impairment, or imbalance. Surgery is the primary treatment option, yet surgical removal of all tumors is not possible or advisable; furthermore, surgery often introduces significant post-operative risks, including hearing loss, facial weakness, and dysphagia. Thankfully things have progressed with increasing understanding of NF2’s genetic basis, as well as emerging molecular parallels with various cancers for which drug therapies are in development or already clinically available. Nevertheless challenges lie ahead for NF2, including candidate drug prioritization, patient recruitment and integrating new therapies into NF2 clinical care.
The Center for Neural Tumor Research:
The NF2 field is at a critical juncture and we believe the experience the Institute has accumulated provides us with a broad set of tools to efficiently tackle the challenges of NF2 translational research. Components of the Center for Neural Tumor Research that are currently being implemented or are in development include:
1) Preclinical NF2 drug testing
2) Clinical Trials for NF2
3) An integrated biospecimen bank and multidisciplinary database for NF2
4) Program and facility for genetic analysis of NF2-related tumors
5) Development of personalized treatments for NF2 patients
Thus, the Center for Neural Tumor Research is an ideal environment to integrate medicine and science through clinical and research trials that may directly benefit NF2 patients.