Addressing The Approaching Crisis Of Neurologic Disease
By Gene Kinney
According to a 2007 report from the World Health Organization, up to 1 billion individuals across the globe – nearly 1 in 6 people – suffer from a neurological disorder1, and neurological disorders currently represent the leading cause of death and disability.2 Consider further that the number of individuals in the United States alive today who have ever had lung cancer (the most common form of cancer) is approximately 541,000.3 By comparison, over 5.8 million Americans are living with Alzheimer’s disease.4 These statistics are staggering but not at all hard to believe given that nearly every family I know has been impacted in one way or another by Alzheimer’s or Parkinson’s disease – the two most common neurodegenerative diseases.
Nonetheless, the attention paid to neurological research as indicated by the 2020 fiscal year budget remains insufficient relative to the need. For example, the 2020 budget for the National Institutes of Neurological Disorders and Stroke (NINDS) was approximately $2.45 billion. By reference, the National Cancer Institute (NCI) budget for the same period was approximately $6.44 billion. Similarly, the combined investment in industry (including venture funding, IPO, and follow-on public offerings) during 2009-2018 aimed at developing treatments for neurological disorders was approximately $11.7 billion for neurological disease focused companies, whereas funding for oncology companies during the same period was approximately $22.7 billion.5 The point is not that oncology is overfunded, but rather that the focus on neurological diseases is underserved relative to the current and future impact on individuals and society. Investing appropriately in science delivers results. Following decades of investment in oncology research, between 2000 and 2018 breast cancer as a cause of death for people of all ages in the U.S. increased by 1.5 percent and today, the average 5-year survival rate for women with invasive breast cancer is 91 percent.4,6 By contrast, Alzheimer’s remains a 100 percent fatal disease and in that same time frame, Alzheimer’s as a cause of death has increased by 146 percent.3
Since the occurrence of many neurological disorders including Alzheimer’s and Parkinson’s disease increases with advancing age, and the worldwide population is aging at a rate never before observed, the magnitude and impact of the pending healthcare crisis is all too predictable and should be alarming to us all.
At Prothena, we are focused on developing treatment options for some of the world’s most devastating neurological diseases. We have been working to understand the pathology that is fundamental to the cause and progression of these diseases for decades and apply our experiences to develop new therapies that target the underlying cause of these diseases. In Alzheimer’s specifically we are working on a number of programs that span antibody immunotherapy, small molecule and vaccine approaches. We have been heartened by recent data from Biogen and others, which suggests that appropriate intervention with these types of treatments result in clinical efficacy consistent with the slowing of disease progression. Such therapeutics, if approved, have the potential to fundamentally change how we treat these otherwise progressive and unrelenting disorders. But, as history has shown in the treatment of other diseases, we believe that the first-generation therapies will require careful and iterative progress to improve efficacy, safety and accessibility. Improving patient access and the patient experience will be an immediate need following approval of first-generation treatments shown to slow the underlying progression of the disease. Approaches that account for the heterogenous nature of pathology, as is seen in Alzheimer’s disease, offer the possibility of improved efficacy. And, of course, moving from treatment to prevention of the disease altogether remains our ultimate goal.
Similar to the inspiring collaborative-focused approaches observed with the current COVID-19 crisis, it will take an “all-hands-on deck” approach of working together across industry, government, academia and patient groups to realize the full potential of interventions needed to quell the devastation of these diseases. Individuals and families affected by these diseases are counting on us to make these collaborations successful. Efforts like the Global Coalition on Alzheimer’s Disease which convenes efforts to accelerate biomarker research; attract public, private and philanthropic capital; increase global capacity and performance of clinical trials; and improve the readiness of healthcare systems to detect, diagnose and treat those with Alzheimer’s are examples of the kind of collaboration needed to speed new and better treatments.
We often hear that it is difficult to develop drugs for neurological disorders or that the failure rate is too high. Now is neither the time to throw up our hands because of failures nor to rest on recent successes. In fact, it is clear to many of us focused on these diseases that the prior experiences are now revealing a clear path forward and it is time to intensify our focus so that we can impact the trajectory of neurological disease before it is too late. The need has never been greater, and it is growing every day. Fortunately, the science has never been better understood, and the path has never been more clearly illuminated. We must prepare for the coming tidal wave. We must act now.
When bright minds come together to tackle a common goal, amazing things happen. As in many other fields, I am confident that science will continue to guide us toward better solutions for neurologic conditions.
Bio: Gene Kinney is president and CEO at Prothena Biosciences.
1Neurological disorders: Public health challenges, World Health Organization, 2007
2Lancet Neurol 2017, 16:877-97; Lancet Neurol 2019, 18:459-80
3Source: American Lung Association
4Source: Alzheimer’s Association
5Bio Industry Analysis, 2019 Emerging Therapeutic Company Trend Report, http://bio.org