The doctor says – “twelve to fifteen months.”
Less than two years, this is the prognosis for patients diagnosed with glioblastoma. Without treatment? Patients may only live three to four months. While intervention can extend this out to 15 months, the numbers still paint a grim picture.
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Maurice R. Ferré MD Maurice R. Ferré MD is CEO and Chairman of the Board of Directors at Insightec, a global medical technology innovator of incisionless surgery. Dr. Ferré brings over 20 years of experience in the medical device industry. Before Insightec, Dr. Ferré served as Chairman of the Board and CEO of MAKO Surgical […]
While extensive research is underway to identify potential treatments for challenging diseases like glioblastoma, there have been limited meaningful breakthroughs. In the meantime, Senator Kennedy, Senator McCain and thousands of others have passed away from this devastating disease. Medicine has made great strides in cure rates for many illnesses, yet there is still a long way to go.
For patients, diagnosis is half the battle. They are plagued by vexing symptoms that can take months, or sometimes years, for a physician to accurately assess, but some measures of relief can be swift once the correct diagnosis is made. Neurology is a notable exception. Despite the fact that over 600 neurological diseases have been identified, patients living with these conditions often face limited treatment options. In other fields of medicine this is not the case; the life expectancy after being diagnosed with HIV has increased to well over 50 years with antiretroviral drugs, and immunotherapy has made melanoma and many other deadly cancers treatable. However, innovative technology is driving progress, positioning 2020 as the start of “The Century of the Brain.”
Frontpage headlines feature failed drug trials and high-cost therapies to treat society’s most burdensome diseases, such as Alzheimer’s and cancer. Immunotherapy, using a patient’s own immune cells to fight disease, is demonstrating promising early results. However, a drug or agent is only part of the story. Identifying a mechanism to deliver the right concentration of a specific drug to a specific target presents a unique challenge in the brain.
This gap between diagnosis and effective treatment has an element of irony in the neuroscience space: the brain has an excellent set of defenses, so good that it often thwarts treatment efforts. The brain is protected by the blood-brain barrier (BBB), a layer of cells which prevents toxins and other infectious agents from diffusing into brain tissue and also limits the effective delivery of drugs and other treatments. While chemotherapy is the standard of care for treating many types of cancer, because of the BBB, many novel and promising new therapies cannot be precisely delivered to their target into the brain.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
While diseases are treated with medications or medical procedures, the delivery of treatment is an equally important part of the equation. The end of this decade has seen major advancements in surmounting this obstacle. Focused ultrasound, essentially concentrated sound waves, have been used in early-stage clinical trials to temporarily disrupt the blood-brain barrier, creating a window of time where a therapeutic treatment could be delivered in a targeted manner.
The potential impact of this advancement is enormous, with clinical trials evaluating focused ultrasound BBB disruption to treat Alzheimer’s disease, ALS, Parkinson’s dementia and brain tumor treatment—including glioblastoma. For glioblastoma, clinical trials are currently evaluating the application of low intensity focused ultrasound to temporarily increase the permeability of the BBB during adjuvant temozolomide (TMZ) chemotherapy to allow the delivery of an agent for targeted therapy. While the blood-brain barrier is not the only challenge hindering effective treatments, the progress that has been made is a true watershed moment, setting the stage for continued research that will mark this new era of the brain.
The next phase in revolutionizing neurological treatment is improvement in patient experience. Much like the changing landscape of infusion centers, technology is helping to drive major surgical operations to friendlier patient and caregiver environments.
Let’s consider essential tremor, the most common movement disorder, affecting an estimated 10 million in the U.S. alone. The uncontrollable shaking of one’s hands caused by the condition has a major impact on the ability to pursue hobbies or hold a job, and patients who don’t respond to medications are left with limited options—traditionally open surgery to implant electrodes in the brain, or living with the debilitating symptoms. Focused ultrasound is an FDA-approved treatment option, allowing medication-refractory patients to receive care that requires no incisions, anesthesia, implanted hardware, and often allows them to return home the same day as treatment. In this new era of neuroscience, the question being asked is not just how can we effectively treat patients, but how can we treat them with few side effects, and in a way that has minimal disruption to their daily life?
In some cases, it may require completely reinventing how we treat, and even think about, a disorder. In the US an estimated 1.7 million people suffer from substance abuse related to prescription opioid pain relievers, resulting in more than 47,000 deaths from overdoses in 2017. Addiction is often thought of in psychological terms as opposed to physiological ones, and addressing opioid addiction typically consists of therapy and enrollment in a 12-step program. While methadone treatment is a common method for addressing addiction, treatment often has to be maintained for life in order to avoid relapse.
Researchers and clinicians are exploring addiction in a new light to see how interventions can be used to affect the brain’s “reward system,” which responds strongly to opioids. Clinical trials today are evaluating surgical interventions that can stimulate this specific target within the brain, either by implanting electrodes, or delivering sound waves. This sort of innovative thinking will be a hallmark of the century of the brain— looking at today’s most challenging medical problems through a new lens, rewriting the book when it comes to treatment approaches.
This vision will not become a reality without action. A recent $106 million donation by Joan and Sandy Weill to create a “Neurohub”—a shared grant between the UCSF, UC Berkeley and the University of Washington which will focus on projects in imaging, engineering, data analysis, and genomic and molecular therapies— is the type of endowment that drives real change, recognizing the importance of cross-disciplinary collaboration. Another example is a $50 million pledge to the Alzheimer’s Drug Discovery Foundation, made by a coalition of philanthropists including Bill Gates, Jeff and Mackenzie Bezos and Leonard Lauder. This donation creates the “Diagnostics Accelerator,” a new fund from the foundation which recognizes that Alzheimer’s research is greatly hindered by the current diagnostic methods, which are limited to an invasive spinal tap or an expensive brain scan. Both of these philanthropic efforts, as well as those from the Michael J. Fox Foundation, the American Brain Tumor Association and many others, exemplify the sort of concerted effort needed to usher in the Century of the Brain—uniting minds from across disciplines to decipher the intricacies of the brain, and carefully analyzing the roadblocks to effective treatment in order to focus investment where it can make the greatest impact.
Picture: mrspopman, Getty Images
Maurice R. Ferré MD is CEO and Chairman of the Board of Directors at Insightec, a global medical technology innovator of incisionless surgery. Dr. Ferré brings over 20 years of experience in the medical device industry. Before Insightec, Dr. Ferré served as Chairman of the Board and CEO of MAKO Surgical Corp, a transformational robotic surgical company that he co-founded in 2004. The company was IPO’d in 2008 and sold to Stryker Corp. for $1.65 billion in 2013. Prior to MAKO, Dr. Ferré was Founder, CEO and President of Visualization Technology Inc. (1993-2002). VTI became the world leader in image guided surgery for ENT, cranial and orthopedic procedures. The Company was acquired by GE Healthcare in 2002.
Dr. Ferré received his Doctor of Medicine and Master of Public Health from Boston University in 1992. He was also the recipient of the prestigious Ernest & Young 2007 Entrepreneur of the Year Award. Dr Ferre is currently active on the following boards: Advamed, The Everglades Foundation and Boston University.
