The second day of the World Medical Innovation Forum continued to build momentum on the Forum’s mission — providing a venue for imperative conversation among academic, private, government and patient/consumer communities. The agenda set forth today brought together stakeholders across disciplines — patients living with neurologic conditions and leaders in academic medical research, private biotechnology, and government. The one-on-one panel format paved the way for candid insight into where the field of neurosciences has been for the past 30 years and where it is heading, at an accelerated pace, via collaboration and conversation.
The first session started early and strong with demonstrations from two of neuroscience medicine’s most promising tools, the AMIGO Suite and NeuroSwitch. The AMIGO Suite is a $20 million multi-modality operating room pioneered at the Brigham and Women’s Hospital (BWH). This robust theater is intraoperative imaging at its best, the concept first conceived at BWH in the 1990s. It is equipped with CT, MR, fluoroscopy, among others, and the world’s only intraoperative PET/CT scan. Suites featuring intraoperative imaging are now adopted widely across the United States and the world.
The goal of these suites is to make surgery safer by assisting surgeons in distinguishing normal versus cancerous tissue. This guidance, along with superior imaging technique, allows neurosurgeons to contour a mass with unprecedented approximation and aids in real-time clinical decision-making. Suites like AMIGO are multi-specialty and can be applied to systems other than the brain and therefore, engage a wide array of the clinical community. Transforming a maximally invasive procedure into a minimally invasive one and being able to operate on a formerly inoperable patient are the underlying incentives for using such suites.
What are brain surgeons currently excited about? More precision for better outcomes. Metabolite-guided surgery through mass spectroscopy, delivery of drugs into the central nervous system (CNS), and laser hyperthermia to destroy tumors. The AMIGO Suite is able to use any type of instrument, metallic or not. A versatile OR is a vehicle of the future, and the world’s eyes are on BWH and the AMIGO suite.
NeuroSwitch is unlike the typical communication aids available on the market. It gets past the physical interface (buttons, keyboards, clickers) and uses movements and EEG data to control a computer device. Patients with locked-in syndrome (LIS), most commonly found in ALS, are able to use NeuroSwitch to regain control of their self-expression. The market size is growing – 35,000 patients suffer from ALS in the United States, with 5,000 new diagnoses annually. The ability for patients to communicate with not only their loved ones, but also with the world online is paramount for them. Patients who use NeuroSwitch gave live demonstrations, raving about NeuroSwitch and how it allows them to keep their passions, like book blogging or 3D printing, alive. When motor control diminishes, sophisticated, hands-free communication aids are mandatory. With features like the ones NeuroSwitch boasts, patients are able to stay connected with their families and communities.
Serving as the official launch of the Ann Romney Center for Neurological Diseases at BWH, Ann Romney spoke to Forum attendees about her personal journey with multiple sclerosis (MS). She was diagnosed in 1998 and expressed how fortunate she was to have received early and aggressive treatment at BWH. She is now currently in remission and on no medication, a state of health not realized by many other patients who suffer from neurologic disease worldwide. She maintains her stellar health by rigorous fitness regimens and diets, as well as a spiritual and mental wellbeing that is fostered by her passion for horseback riding. She credits both physicians at BWH and her dedicated supporters who were there for her on the campaign trail during her husband’s past presidential bid.
The Ann Romney Center for Neurological Diseases will support research and innovation in MS, Alzheimer’s disease (AD), Amyotrophic Lateral Sclerosis (ALS), Parkinson’s Disease, and brain tumors. She hopes that this horizontal model will instigate interdisciplinary collaboration. The overlap in many neurological illnesses calls for a different approach and to break down the silos of disease cures and therapy research and development efforts. There is power in numbers, and she encourages all who are affected by neurological disease to join her new social media campaign by using the hash tag, #50MillionFaces, on Twitter.
The Alzheimer’s Phoenix: New Technologies, New Economics
This panel of esteemed and diverse AD experts shed light on the current theories of disease causes and what to expect on the journey to a cure. All were very optimistic and agreed that we are close to disease-modifying therapies that could be marketable as soon as five years from now. It has taken decades to validate the two-tiered pathology of AD in amyloid-beta (a-beta) and tau protein aggregation. The future lies in combining these theories into parallel therapies to enhance clinical effect. Base inhibitors have been found to be very promising and are currently the most competitive targets in the industry. When given to compliant patients for a year, a-beta was eliminated almost entirely. This is the epitome of reverse translational medicine – aiding the immune system in recognizing these destructive protein aggregates.
As the underlying causes of AD become clearer, early and personalized treatment for patients has become more realistic. In the autopsies of people who died of suspected AD, almost 1/3 of their brains were seen to be damaged. According to the speakers, clinicians must realize that AD is malicious organ failure that starts decades before being symptomatic, if at all. The heterogeneity of the population with AD in years to come will help evolve treatment. It will not be long before we are treating AD patients based on their own specific symptoms and biology. Today’s speakers advised that clinicians need to start utilizing a risk-assessment profile to plan beyond the long prodromal period and optimistically provide a risk “score” as early as age 40.
Where are there room for improvements in AD care? There has to be greater coordination among payers to coordinate cost-effectiveness. The late-stage AD patient requires 24-hour care, multiple caregivers, and an extensive clinical team. The advances to come will hopefully allow patients to maintain more independence and a greater quality of life and have care at an as-needed basis. Investigators need to focus their efforts on finding the right genes and biomarkers for drug development. Non-pharmacological interventions should be integrated into care and be viewed just as importantly as drugs. Poor diet and scarce exercise cannot be compensated for by better drugs. The stratification of AD patients as at-risk, non-symptomatic, early-stage, or late-stage, require individualized care and treatment.
Regeneration, Cell Therapy, and Neurocare: Products? Delivery?
Regenerative medicine is recreating cells that are deficient, diseased, or missing. The use of stem cells and cell-based therapies is still controversial and presents moral and ethical issues as well as regulatory hurdles. However, it is undeniable that the brain is incredibly “plastic” and boasts an elegant biofeedback system. To use cells to correct a dysfunction requires great specificity. Cell-based therapies have the potential to be applied across disciplines in spinal cord injury, diabetes, and blood dyscrasias. In spinal cord injury, “cross-correction” can produce enough of a missing enzyme in defective neurons for them to remain viable. Diabetics can be unleashed from a life support of administered insulin with the replacement of defunct beta cells in the pancreas. In inherited disorders like sickle cell anemia and thalassemia, gene editing can reprogram cells to be embryonic, enabling patients to essentially “get their cells back.” The targeted diseases for stem cell research are typically ones that have no treatments; any work in this field, therefore, will address a large unmet need.
There are two types of cells used in regenerative therapies: embryonic, or immature cells derived from embryos, and “adult” cells that come from a formed organ. Embryonic cells can potentially produce any cell type in the body – providing a wealth of possibility yet also result in the chance of tumorgenecity, or the primitive cell developing into a tumor. There are ways to address this by pre-determining cells or turning on “suicide genes” to kill the cell once it starts to express tumorgenecity. This controversial technique has the potential to save tens of thousands of lives but will not be adapted into practice if headway cannot be made by industry and academia, together, in addressing the treatment risks.
CEO One-on-One Sessions
The personal one-on-one accounts continued throughout the day with candid conversations between leaders in the private biotechnology industry and government. Robert Bradway, CEO of Amgen, and George Scangos, Ph.D., CEO of Biogen, reflected on the challenges and successes to date in identifying and producing novel therapies for devastating neurologic diseases.
Bradway said that Amgen is a leader in drug development across various diseases like chronic kidney disease, cardiovascular disease, inflammation, and the company is increasingly interested in getting more involved in the neurosciences. Some drugs in their current pipeline are early-stage treatments for schizophrenia, AD, and chronic pain. Amgen is currently active in genetics, and Bradway sees a sustaining trend in favoring genetics for informing drug development.
Bradway discussed Amgen’s acquisition of Decode Genetics at length, emphasizing the imperative role collaboration plays in translational research—an interdisciplinary research style that is currently guiding the industry. Bradway noted there has been recent feverish collaboration as government withdraws more funding for basic research. We are seeing this withdrawal because of a decades-long steady monetary decline. More investment in genetics can reverse this by quickening the drug development process and making it more efficacious. Per Bradway’s account, Amgen is looking ahead to identify and develop therapies based on the genetic and molecular understanding of a disease and its pathway. As industry at large is seen further reducing its commitment to discovery research, Amgen proclaims it is not shrinking back.
Scangos’ account provided insider details into Biogen’s past and current Phase 2 and 3 trials for ALS, MS and AD therapies. Scangos praised the role academic collaborations have had in driving the talent, direction, and mission of successful biotechnology research and development. Scangos also shed light on Biogen’s biggest concern for the next half decade—the current environmental context that medical research operates in. Thoughtful public policy will be necessary to fulfill the potential of this pivotal time in medical history—with aligned talent, resources, and a greater understanding of neurologic diseases than ever before.
The one-on-one conversation with Honorable Sylvia Matthews Burwell, Secretary of the U.S. Department of Health and Human Services, echoed Scangos’ glimpse into the importance of public policy. Secretary Burwell emphasized the similar priorities all entities hold—affordable, accessible, life-changing therapies for patients. By changing health care pay systems, implementing integrated care systems, and moving forward with data collection, analytics, and protection, the Department of Health and Human Services is optimistic about the next phases of neuroscience care and beyond.
Neurostimulation: First Line Therapy?
What is neuromodulation and why does it work? Neuromodulation is the use of various kinds of electric signals to restore the brain to a more normal state. It is a broad and emerging field; we are on the verge of a revolution with treating diseases such a Parkinson’s, epilepsy and OCD. This type of treatment has been around for decades but has recently re-emerged through new findings in neuroscience research.
Speakers said that in the past, neuromodulation treatments worked, but nobody knew why. Although the field is much more developed now, patients are still hesitant to embrace this technology due to its highly invasive nature. Panelists mentioned that up to 15 percent of Parkinson’s patients receive these implants, but there still needs to be additional education and a shift in mindset in order for this type of treatment to be more widely adopted – a challenge that may be overcome with time.