RARITAN, N.J.—Aimed at helping children diagnosed with autism—and their families—have access to a state-of-the-art information highway, Janssen Research & Development has created an internal incubator program to nurture highly innovative ideas of cutting-edge research, possibly leading to new platforms and innovative technologies.
The driving force of the Janssen Incubator is its Autism Knowledge Engine, a first-of-its-kind, digital, integrated system designed to facilitate research and clinical trials for the development of new medicines which may ultimately help children with autism curb obsessive behavior and become more focused.
With the Center for Disease Control (CDC) estimating that one in 88 U.S. births is diagnosed with autism spectrum disorder (ASD), time is of the essence.
Collaborating with Microsoft, the Janssen venture brings together talent and expertise from a key information technology organization to develop a robust approach to therapeutic assessment and development, Janssen stated in a news release.
The Microsoft HealthVault software provides a personalized electronic health record system to track detailed treatment and developmental milestones—a boon to ASD parents, often mired in paperwork, doctors and therapy visits, meds changes, lengthy health insurance phone calls and more.
Although ASD may be genetic and present at birth, the symptoms usually don’t start until the ages of between 18 and 24 months old when the child stops “using his or her words,” making eye contact and displaying repetitive behavior. Researchers believe an early diagnosis and speech and occupational therapy may stem the effects of autism by the time the child starts school.
But as yet, the theory that early intervention could rewire a child’s brain to lessen the effects—and affects—of autism, has not been proven.
To date, the Janssen Incubator has invested in six ventures, one being the Autism Knowledge Engine, which ultimately addresses the symptoms of autism and investigates new medicines to treat ASD, a collection of diseases for which therapeutic unmet needs remain high.
The other five venture teams are focused on the following areas: lupus; staphylococcal infections; chronic pain; a new technological approach in small molecule research; and a platform to develop therapeutics with reduced side effects, says Dr. Rob Willenbucher, head of the Janssen Incubator.
The Janssen venture team takes an integrated collaborative approach, working with Autism Speaks, a leading global autism science and advocacy organization, to provide scientific guidance through an advisory panel, and it leverages the experience, capabilities and leadership of the Janssen R&D Neuroscience Therapeutic Area.
“Janssen Research & Development created the Janssen Incubator, which has been established to identify and nurture highly innovative ideas arising through our scientific community,” Willenbucher says.
“It also places special emphasis on gathering detailed information on patients living with ASD across care providers via web and mobile applications,” according to Gahan Pandina, venture leader for Janssen R&D, and it “provides symptom tracking and detailed information on clinical and medical history to help identify subpopulations for proof-of-concept studies and to inform development of new treatment options.”
“Venture teams are created under an entrepreneurial operating model and strive to incubate and deliver high-value opportunities through internal and external networks and collaborations,” notes Willenbucher. “These venture teams are focused on new spaces or areas outside of the current Janssen disease focus areas and are staffed and led by Janssen scientists.”
Pandina says that the “integrated personal electronic health record system that utilizes Microsoft HealthVault is designed specifically for children with ASD, their families and their care teams.”
The Autism Knowledge Engine will soon have home- and lab-based biosensors linked to the core and associated symptoms and underlying biological processes of ASD, to improve sensitivity of assessment of treatment outcomes—and finally, a de-identified research data warehouse with data analytic tools, he adds.
The Janssen virtual warehouse actually “creates a unique, integrated database of information, while preserving patient anonymity,” according to Pandina, adding: “The vision is to utilize the Autism Knowledge Engine to understand who best to enroll in a particular clinical trial, to evaluate a particular medicine, to use the system in clinical trials, to improve the ability track treatment outcomes and understand the factors that may affect efficacy, safety and tolerability.”
Janssen is in the process of designing the system, now, with the intention to learn from individuals living with ASD and caregivers to determine which components may be most effective in improving research methods in clinical trials, he said.
“For individuals who participate in future clinical trials in ASD, equipment or medication that may be necessary for the research project would be provided,” Pandina explains. “We believe this system will help to improve clinical trials’ efforts to evaluate new medicines for the core and associated symptoms of ASD. We are still determining which devices and technologies we would need to be provided to patients/caregivers who participate in such clinical trials as part of the initial testing of the system.”
“We believe the system, initially, will be focused on developing new medications and learning more about how patients respond to treatments,” Pandina continues. “We may understand how a child living with autism thinks and learns via the tools we are developing. However, that is not the primary focus of the system.”
Autism Speaks presented a Microsoft HUG award to Janssen for its creativity in building the Autism Knowledge Engine, according to a Feb. 24 news release.
“Janssen’s work in creating this Autism Knowledge Engine highlights the increasing importance of innovative technologies in facilitating the future discovery and development of treatment options for individuals living with autism spectrum disorders,” noted Autism Speaks Chief of Science Rob Ring.
“It is also designed to help identify subgroups of children who might best benefit from clinical studies of promising new treatments”, Ring said. “It even integrates wearable biosensors that can record autism-related symptoms and body responses at home, as well as in a clinical setting. These biosensors are designed to provide objective measures of symptoms in ways that will improve assessment of treatment benefits.”
The platform “will combine anonymous patient information to create a powerful database to guide further research,” he added.
Pandina concludes, “This award represents the important collaborative work that is underway to learn more about a constellation of autism-related disorders that affect the lives of children, families and caregivers, and through a greater understanding and advance treatments in the future.”