There’s no doubt that one of the best ways to find new treatments is through the use of clinical trials. However, when it comes to rare diseases, finding enough people to participate in a trial can be extraordinarily difficult. Take, for example, mesothelioma, an extremely uncommon form of cancer that can form in the linings of the lungs, abdomen, or (very rarely) the cavity around the heart. Caused by the inhalation or ingestion of asbestos, only about 2,000 to 3,000 people are diagnosed with mesothelioma each year. It’s almost always caught at a late stage, and the life expectancy of mesothelioma patients is usually measured in months rather than years.
Given all of these factors, finding enough individuals to participate in a mesothelioma clinical trial can be challenging. When patients are found, they may be too ill to travel to the clinic where a trial is being conducted, and even those who can travel may not fit within the parameters of the trial’s criteria.
From the patient end of things, there are plenty of roadblocks as well. Given the poor prognosis of mesothelioma, many patients who have the disease are willing to try experimental treatments of all kinds, even if they offer little hope of overcoming the disease. However, most patients are older (the average age of a mesothelioma patient is 74), and many are not savvy enough to navigate online clinical trial information, such as the database available at ClinicalTrials.gov. Even with help from a younger family member or caregiver, finding a trial that can help them is a difficult task – one which often has to be completed while undergoing an existing treatment plan.
Enter the Moonshot
The Cancer Moonshot Initiative, announced earlier this year in President Barack Obama’s State of the Union address, is looking to alter the clinical trial landscape in a way that could change the dynamic for those who suffer from rare diseases. One of the biggest initiatives is to improve collaboration between patients, practitioners, researchers, advocates and organizations who are all looking for the same thing – a way to cure cancer.
While this cooperation takes a myriad of forms – from making it easier to share study results to “big data” projects such as Cancer Genomics – one of the opportunities that will affect patients directly is the plan to revamp the way clinical trials are shared with the public.
As part of a partnership between the National Cancer Institute and the White House Presidential Innovation Fellows, an entirely new way of presenting trial information has been created. The new model builds on previous government data-sharing projects, such as weather information and GPS coordinates, both of which have been around for decades. While the new system has yet to be perfected, the idea is to improve it over time, making the system an even more effective way to connect patients to clinical trials. In fact, the government is seeking ideas from the public – particularly from patients who will benefit the most from an improved system.
Another form of collaboration is the push to provide standard guidance about how to conduct research and orchestrate trials. Programs like QUILT will develop a set of guidelines regarding the testing of new drug combinations, vaccines, chemotherapy, immunotherapy and various other types of treatments, both conventional and investigational. Standardizing and coordinating these types of studies will both fill in the gaps and expand the horizons of current clinical research.
Why This Is Important for Rare Diseases
These new areas of collaboration will be very meaningful for rare diseases, such as mesothelioma. By making clinical trial information more accessible, there is more opportunity for patients to connect with a trial that could lead to longer-term survival, or perhaps even remission. From simply a humanitarian perspective, giving new hope to people with a disease that has a historically poor prognosis is something that everyone should be able to get behind.
Furthermore, as more patients of rare diseases become available for such studies, there is a greater likelihood of success – or at least a greater opportunity for informative results – for the trials themselves. In the long run, having access to more data from larger studies is the only sure way that we can move closer to realizing a cure for cancer of any kind.
Finally, when it comes to rare diseases, awareness is a huge hurdle to overcome. Cancer awareness is important at every level, but large campaigns around some of the better known cancers – such as breast cancer and prostate cancer – tend to overshadow other cancers that could benefit greatly from some of that attention, in the form of both dollars and recognition. Better cooperation among medical professionals, patients, advocates, politicians and others can help bring much-needed consciousness to lesser-known diseases like mesothelioma.
We are still in the early days of the Cancer Moonshot Initiative, and ideas are still flying around about how to tackle a lot of these issues. Nobody knows if it’s possible to cure cancer by 2020, but there’s no reason not to aim for it. How it all shakes out remains to be seen, but whatever the outcome, we hope everyone will be included in the effort, regardless of how many people have the same disease.
For more content on the Cancer Moonshot initiative, rare disease research and similar industry topics, click the link below to view our educational materials for clinical research professionals.