A trio advancing trials

Taking a look at three recently announced technologies aimed at making clinical trials run more effectively

Jeffrey Bouley
Efforts to improve clinical trials have looked at many factors—among them better recruitment, more diversity and patient-centered policies—but a lot of focus lately is also on how to use technology to improve data gathering and make for better outcomes. Here is a roundup of three companies that have recently announced their work on that front.
 
Apple seeks to change the clinical trial landscape with ResearchKit
 
The buzz for Apple in recent weeks has largely revolved around its new Apple Watch, but there has also been talk in the life-sciences and drug development communities around ResearchKit, a software medical platform that seeks to turn the iPhone and the HealthKit app into diagnostic tools. Users who opt to work with ResearchKit will be able to send data gathered through the HealthKit app to medical researchers who partner with Apple. One of the hopes is that ResearchKit will make recruiting for clinical trials from diverse populations much easier.
 
As part of this effort, the Icahn School of Medicine at Mount Sinai and LifeMap Solutions, a subsidiary of BioTime, announced in March the launch of a large-scale medical research study that uses the new ResearchKit framework to make it easy for individuals who suffer from asthma to participate in studies right from their iPhone.
 
The Asthma Health app in this study is designed to facilitate asthma patient education and self-monitoring, promote positive behavioral changes and reinforce adherence to treatment plans according to current asthma guidelines. The study tracks symptom patterns in an individual and potential triggers for these exacerbations so that researchers can learn new ways to personalize asthma treatment.
 
“We are delighted to be amongst a select group of institutions to pioneer the use of this new ResearchKit framework,” said Dr. Eric Schadt, the Jean C. and James W. Crystal Professor of Genomics at the Icahn School of Medicine and founding director of the Icahn Institute for Genomics and Multiscale Biology. “This app is the first of a series of disease-related medical research apps we plan to develop, incorporating electronic consent or ‘e-consent’ to enable us to recruit, consent and enroll research participants remotely via the app without direct, in-person, contact during any phase of the study. Now we can reach all corners of the globe to recruit research volunteers and conduct medical research with sample sizes that are orders of magnitude greater than previously possible for a fraction of the cost.”
 
When granted permission by the user, the Asthma Health app can access health data from HealthKit to track asthma inhaler use measured by third-party devices and apps, or can take advantage of the accelerometer, microphone, gyroscope and global positioning system sensors built into iPhone to gather other relevant health data.
 
“This innovative study will explore how researchers can conduct clinical studies at an unprecedented scale by unshackling science from brick-and-mortar constraints,” said Corey Bridges, CEO of LifeMap Solutions. “The benefits of flexibility and portability that have made mHealth apps so successful for consumers in the past have the same potential to significantly improve the health of patients and revolutionize medical research.”
 
Global research and consulting firm GlobalData has mixed feelings about the technology at this point, but sees promise. According to Niharika Midha, GlobalData’s analyst covering medical devices, “GlobalData believes that Apple’s ResearchKit, which allows researchers to create apps and collect data for disease management by recruiting subjects via iPhones, has the potential to redefine the medical research dynamics, especially for chronic disease management.”
 
Midha sees the ability to recruit a large patient pool using iPhones as a way to both reduce the costs associated with these studies and enable continual and convenient monitoring. Also, the ability to link apps to other external wearable or monitoring devices, such as inhalers, is another distinct highlight of the technology.
 
“However, there are also some potential pitfalls with the ResearchKit, apart from the privacy concerns already voiced in several media reports,” according to Midha. “Firstly, it is uncertain whether this approach to medical research, in which physicians do not actually engage with participants, will add any valuable insight. This is because there are risks in assuming high participant compliance and formulating disease management conclusions based solely on the data collected by the ResearchKit. GlobalData believes that this approach can only supplement traditional research.
 
“Secondly, study participants can readily drop out when the app’s novelty wears off. This would make the previously-collected data unusable for the purposes of statistical analysis, although some form of incentive might sustain participant commitment. We therefore anticipate that the trade-offs between a large participant pool and data accuracy will be the top concerns of using this platform.
 
“Finally, GlobalData has reservations about the ResearchKit’s future penetration in international markets outside the U.S. The varied regulatory landscape in other geographies with regards to monitoring such apps may hinder this new platform’s adoption in regions such as the EU and Asia.”
 
Merrilyn Datta, president and general manager at Definiens—a provider of image analysis and data mining solutions for quantitative digital pathology in the life sciences, diagnostic biomarkers and healthcare industries—spoke with DDNews about the technology and is also upbeat about the potential, while cognizant of the downsides right now.
 
As far as advantages, she notes that “It is remarkably difficult to recruit people to clinical trials; about one-third of the budget for trials is spent on that, so this offers cost benefits through channel reach. Second, it could give more ability for longitudinal and trending studies than you get interrogating patients one time—you can obtain data at various points. Finally, it plays into the Big Data scheme and you get more personalized data from different types of patients, which will hopefully mean data more representative of various populations. You need that for personalized medicine—a gateway into Big Data pulling information into clinical trials in a more collaborative way.”
 
She is concerned about the data privacy issue, particularly with Health Insurance Portability and Accountability Act regulations. “Also there is the aspect of getting adoption from more than just early adopters, but they’ve made it open source, so I think that will help get more applications for ResearchKit soon and help with adoption,” she says. “But in the end, I see a lot of advantages, especially synergy with other things happening like telemedicine, heart-monitoring wearables and the like. ResearchKit isn’t a diagnostic but I see it being very synergistic with diagnostic technology.”
 
Consortium advances prostate cancer tech
 
Epic Sciences and the Prostate Cancer Clinical Trials Consortium (PCCTC), a leading consortium for Phase 1/2 and Phase 2 clinical trials in prostate cancer, announced recently they have entered into a collaboration to apply Epic’s circulating tumor cell (CTC) technology in multiple investigator-initiated clinical trials to advance the development of new prostate cancer treatments.
 
“Liquid biopsies could help accelerate the clinical development of new targeted therapies to treat prostate cancer by providing rapid access to ongoing and timely information about a patient’s cancer on a cellular and molecular level,” said Jake Vinson, CEO at PCCTC. “We have been impressed with Epic’s circulating tumor cell technology’s capacity to help develop drugs in more precisely defined and biologically relevant patient groups.”
 
Epic is developing diagnostic tests that can detail the dynamic changes in gene and protein expression that occur during the course of cancer treatments. From a simple blood test, Epic’s “no cell left behind” technology examines approximately six million cells across more than 90 parameters to analyze all possible types of CTCs.
 
“Circulating tumor cells can describe the global heterogeneity of cancer in patients at multiple time points and can be used to track the genetic and proteomic changes that occur under therapeutic selection pressure,” said Dr. Murali Prahalad, president and CEO of Epic Sciences. “Collaborating with the PCCTC, we can help advance clinical trials by using Epic’s technology to monitor treatment effectiveness and understand acquired resistance.”
 
Epic has more than 30 collaborations with pharmaceutical companies and leading cancer research centers spanning 67 clinical studies.
 
ACD’s RNAscope supports trial of Merrimack’s MM-121
 
Advanced Cell Diagnostics Inc. (ACD) announced recently that Merrimack Pharmaceuticals Inc. is using ACD’s RNAscope technology to select patients for its Phase 2 clinical trial of its product candidate MM-121. Merrimack will use RNAscope to identify patients with heregulin-positive, locally advanced or metastatic non-small cell lung cancer, making this Merrimack’s first MM-121 trial to include only patients with a high heregulin biomarker profile. In previous MM-121 Phase 2 clinical trials, heregulin-positive patients, determined by RNAscope, have demonstrated significant therapeutic benefits when treated with a combination of MM-121 and standard therapies.
 
“These trials have repeatedly demonstrated the potential of heregulin as a promising biomarker in predicting the therapeutic benefits of MM-121.” said Gavin MacBeath, senior vice president of translational research at Merrimack. “We believe that the high sensitivity and specificity that RNAscope provides are necessary to identify the patients most likely to benefit from MM-121 in this Phase 2 clinical trial. RNAscope’s ability to detect heregulin within the confines of tumor cells, compatibility with existing automation instruments and with small tissue specimens such as fine needle aspirates and core needle biopsies were all important considerations in our decision to work with ACD.”

Jeffrey Bouley

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