Wednesday, August 31, 2016

History with a Window Seat: How the Cancer Moonshot Impacts Clinical Trial Opportunities for Rare Disease Patients


Clinical trials represent one of the most effective ways to find new treatments for cancer patients. However, trials for rare cancers can face significant difficulties, such as attracting an adequate level of funding or finding enough patients who meet the criteria to participate.

An example of this conundrum is mesothelioma research and treatment. Mesothelioma is a very rare form of cancer that can form in the linings of the lungs, abdomen, or the cavity around the heart. Only about 2,000 - 3,000 people are diagnosed each year. Most of the time it is caught at a late stage and life expectancy for patients is usually measured in months not years.

Given all of these factors, finding enough individuals to participate in a mesothelioma clinical trial can be extraordinarily difficult. Patients who are eligible can be too ill and cannot travel to where the trial is being held. 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.

With most patients being advanced in age (the average is 74 years old), most have trouble navigating online clinical trial information. Databases like ClinicalTrials.gov and NASA are tough nuts to crack for many users, and finding a trial that can actually help them in this deluge of information has proven to be very difficult.
Introducing the Moonshot

The Cancer Moonshot Initiative was announced earlier this year as part of President Obama’s State of the Union speech. One of the primary focuses of this effort is to improve cooperation between patients, researchers, and organizations that all are now all pulling in the same direction. This is already showing some interesting (and promising) results, for example, with the recent release of NASA-funded research on PubMed, including 140 papers on various types of cancer.

One of the biggest ways this will affect patients is by redesigning the process of how patients find clinical trials suitable for them, and how clinical trial administrators find eligible patients for their studies. 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 innovative model builds on previous government data-sharing projects, such as weather information and GPS coordinates, both of which have been around for decades. The system is still a work in progress, but its premise would help thousands of patients navigate this previously difficult task.


The improved collaboration manifests in other programs like QUILT, which is a private effort to develop a set of guidelines around testing new combinations of drugs, vaccines, chemotherapy, immunotherapy, and various other types of conventional and experimental treatments. Standardizing and coordinating these types of studies will expand the horizons of current clinical research, while filling in some of the gaps in which some rare diseases like mesothelioma find themselves.

Why this matters for Rare Diseases

Without taking away from the significance of Cancer Moonshot projects for major diseases, these new areas of cooperation will be even more meaningful for rare diseases.

By making clinical trial information more accessible, there is more opportunity for patients to actually connect with a trial that could lead to longer-term survival, or perhaps even remission. Many rare diseases carry a poor prognosis, but this effort could give people hope where there was little to none before.

Furthermore, as more patients participate in such studies, there is a greater likelihood of success – or at least a greater opportunity for informative results – for the trials themselves. This could result in having more access to data from much larger studies, which is the way to move closer to finding actual cures.

We are still in the early days of the Cancer Moonshot Initiative, and many ideas are still in the planning stages. The mission is incredibly difficult but that’s no reason to not try to achieve it. Whatever the outcome this is an exciting time in cancer research as we try to do the impossible - find a cure for cancer.

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