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To make a difference, adrenal cancer requires research and advocacy collaboration
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Today, the only approved treatment for adrenal cortical carcinoma is mitotane, a derivative of DDT — a toxic pesticide banned since 1972. This is a problem. This needs to change.
However, in today’s society, where return on investment for government agencies and big pharma is measured by constituent lives saved and money earned, it is difficult to make a case for orphan diseases, including most endocrine cancers. What’s needed are: mechanisms to share resources such as patient databases and tissue samples to facilitate the advancement of research; and the formation of clinical networks to develop management guidelines and protocols that both define “standards of care” and facilitate the involvement of these small patient populations in clinical trials.
Gary D. Hammer
Researchers and clinicians with vested interest and expertise in endocrine cancers are integral to the efforts, serving as advocates for patient support, patient care and research dollars. This is best done in the halls of Congress and by educating the public and our fellow endocrinologists, oncologists and researchers about our orphan disease work.
Network building must involve our professional societies in conjunction with the increasingly empowered patient groups and foundations that bring awareness and advocacy to both big pharma and government agencies through public outreach and lobbying efforts.
Researcher collaboration
With rare diseases in particular, one of the most important things to realize is that practicing physicians and researchers need to collaborate.
In 2003, a handful of adrenal cancer researchers and clinicians came together from around the world to develop a consensus statement on adrenal cancer. Due to a paucity of evidence-based clinical data, we instead committed ourselves to collaboration in the research and clinical arenas. Coordinated in Europe, the First International Randomized trial in locally advanced and Metastatic Adrenocortical Carcinoma Trial, or the FIRM-ACT, was born.
Although this study of more than 300 patients (a huge number for such a rare disease) showed only minor superiority of one chemotherapy regimen over another, it more importantly showed that we can develop our own collaborative network that could bring leverage to the scientific and clinical fronts and navigate interactions with the government and the pharmaceutical industry.
The FIRM-ACT was instrumental to the formation of the European Network for the Study of Adrenal Tumors (ENSAT) and a similar American cooperative that together serve as a platform of shared resources, collaborative research and clinical trial coordination. Major initiatives include the ongoing deep sequencing of 100 adrenal cancer samples by The Cancer Genome Atlas (TCGA) of the National Cancer Institute and recent industry-sponsored national and international trials of new targeted therapies for adrenal cortical carcinoma (ACC).
Importantly, it was only after completion of the FIRM-ACT, after we in the ACC community began to uncover some of the biology of adrenal cancer and after we had proven that we have a functional collaborative network, that drug companies began inquiring about the possibility of performing a trial.
Over a number of years, collaborating laboratories in Europe, the United States and Brazil had published genetic, cellular and modeling data implicating the insulin-like growth factor pathway in ACC. There have now been industry-sponsored trials targeting this pathway in ACC, including one international trial involving more than 100 patients.
Although none of these trials were blockbusters that got the drug approved, the targeted therapies showed biologic and clinical effects in small subsets of patients, pointing the teams to important signatures of tumoral responsiveness. In addition, they illustrated the value of a collaborative network in understanding this ultra-rare disease and engaging big pharma.
Patient advocacy
Our ability to make headway in rare diseases like adrenal cancer is dependent upon patient understanding, patient involvement in research, patient-driven advocacy campaigns and patient-driven funding mechanisms. What I’ve learned is that real people — grateful patients and families — make profound differences in the rare cancer arena.
The extremely limited funding currently available for biomedical research, let alone rare disease research, demands that we find nontraditional ways to fund research.
Grateful patients and families want to make a difference, to level the playing field for others afflicted, and they know they can. Since the rise of the Internet, patients with endocrine cancers are more informed, empowered with information and finding a united voice. From patient support groups to disease-oriented advocacy groups, to formal foundations for fundraising, patients are front and center in the fight. These patient-oriented groups often link directly or indirectly to the centers of excellence engaged in the rare cancer work.
Some of the foundations, professional organizations and funding mechanisms now demand that scientists have patient advocacy embedded within grant applications and research networks. This has been transformational. Adrenal cancer advocacy groups have been instrumental in funding endowed chairs, recruiting outstanding adrenal cancer researchers, funding young scholars, creating databases and tumor repositories and, overall, pushing the research agenda, advocacy and awareness forward.
Of course, this is a feed forward win-win. The patient groups play a huge role in recruiting patients to trials and it is through the patient networks that patients come to centers of excellence specializing in research and clinical care associated with any given rare disease. Through these groups, we not only see acceleration in research findings due to the infusion of funding, but we see our patient volume double, triple and more. These patients then participate in the ongoing clinical trials associated with the network and populate the databases and tumor repositories that then fuel the research. The iterative process continues and a center of excellence is born.
Willing to go the extra step
A specialized center that focuses on all aspects of a given rare endocrine cancer is an essential ingredient in the new recipe for success. Such centers logically become the hubs for the successful research and clinical networks.
Because patients often come long distances to a center of excellence for adrenal cancer, a multidisciplinary clinic requires real engagement with the patient, the patient’s family and their referring physicians that all records, all pathology, all radiology are received and reviewed by the treating doctor, endocrine pathologist and radiologist before the visit.
It is ideal to be able to provide the patient with the multitude of interactions with all associated specialists during one unified visit — a one-stop shop. This is often difficult and may take years to coordinate a joint clinic where endocrinologist, endocrine surgeon, medical oncologist, radiation oncologist and geneticist share a staff room and see patients together with enough nimbleness to change/add who a patients sees during a visit that often takes a combined 2 to 3 hours.
Having the clinical trial team present to review trial eligibility and consent with patients links the clinic experience with the ongoing research essential to progress. Negotiated standing slots on the CT, MRI or PET schedule can facilitate patient-centered service excellence and the initiation of therapies on site. A real-time “review board” that discusses the cases of the day after clinic is also essential to provide concrete guidelines and recommendations on how to move forward before the patient departs.
There is an increasing desire for telemedicine and remote second opinions as patients are desperately seeking experts knowledgeable in managing their rare cancers. The Internet and patient support and advocacy groups have fueled this desire. We have developed a mechanism to work remotely with patients around the country and internationally. The Remote Second Opinion (RSO) program for adrenal cancer involves the formal review of a clinical case, along with pathology and radiologic review followed by a case presentation at the review board. The rules of telemedicine are being revised regularly, but when done well and within the law, they provide great value for patients.
Making a difference
If we’re going to make a difference, we must develop specialized centers dedicated to the multidisciplinary care of patients with endocrine cancer.
If we’re going to make a difference, we must build cooperative networks for these rare diseases.
If we’re going to make a difference, we must engage with patient support groups who advocate for funding and access to specialized centers.
With disease networks that couple centers of excellence and patient advocacy groups to provide access to care, research will flourish. Granting agencies are beginning to provide mechanisms of support for orphan diseases. Big pharma is slowly becoming less risk averse to rare diseases. The result is that clinical care is advancing.
If we want to make a difference, we have to do things a little differently.