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Is There Hope for Parkinson’s Disease Patients?

By STEVEN ZECOLA

Congress will need to amend its faltering Parkinson’s disease legislation if it hopes to achieve meaningful results.

Why Change Is Necessary

For more than 50 years, the National Institutes of Health (NIH) has pursued Parkinson’s disease (PD) research with limited success. Nearly $5 billion has been spent, yet no approved treatment exists that slows, stops, or cures the disease.

This is not a new concern. By the mid‑1990s, Parkinson’s advocacy groups had grown frustrated by the absence of major therapeutic advances since L‑dopa’s approval 25 years earlier. That frustration led to three national plans launched in 2000, 2006, and 2014. None produced meaningful breakthroughs.

Congress to the Rescue?

Congress attempted to address this stagnation by passing the National Plan to End Parkinson’s Act (Public Law 118‑66), signed July 2, 2024. The Act:

  • Calls for an integrated national strategy to prevent, diagnose, treat, and cure Parkinson’s
  • Establishes an Advisory Council on Parkinson’s Research to deliver annual recommendations to the HHS Secretary, with the first report due within 18 months
  • Requires the HHS Secretary to issue a progress assessment within 24 months

What Happened Instead

The HHS Secretary delegated implementation of the Act to NIH. As a result, the Advisory Council and HHS missed their first statutory deadlines and produced no measurable progress. NIH convened the Council’s first meeting on June 29, 2026—two years after enactment.

The meeting materials omitted information essential for forming a credible strategy, including:

  • The time and cost of the current drug‑approval process
  • Lessons learned from the three previous national PD plans
  • The role of artificial intelligence and other emerging technologies
  • Regulatory barriers
  • A comparison of federal vs. non‑federal PD research funding and coordination
  • Funding allocations across projects
  • An assessment of where industry stands on potential cures and the remaining scientific steps
  • A plan for engaging external subject‑matter experts
  • Any economic analysis

Even basic administration proved difficult. NIH collected comments from 162 individuals but posted them a month late—and did not include my submission. Despite recommendations, NIH still does not use regulations.gov, which would eliminate many of these administrative failures.

More importantly, NIH shows no recognition that the regulatory system itself is broken and imposes massive societal costs. Parkinson’s disease alone generates over $80 billion annually in direct and indirect costs.

What the Advisory Council Is Likely to Recommend

Based on the 162 public comments, the Council’s top recommendation will likely be a substantial increase in PD research funding.

More funding would be logical—but only if used efficiently. Efficiency requires acknowledging that the technology, regulation, and process must change. NIH, as currently structured, cannot deliver on that acknowledgement or those changes.

A Better Approach

Congress should not wait for the fourth national plan to fail. Instead, it should establish a private investment vehicle, funded with federal research dollars and majority‑owned by the federal government. Executive leadership should be recruited from the healthcare industry and granted a 20% carried interest, aligning incentives and ensuring accountability for research outcomes.

Congress should also require the FDA to adopt a zero‑based regulatory framework for AI‑driven applications, including collapsing the multi‑trial model and incorporating real‑time data into the review process.

Conclusion

Human brain cells are complex and fragile, and central nervous system drugs have among the highest attrition rates in medicine—approved at less than half the rate of drugs in other therapeutic areas.

Layer on the length, cost, and complexity of today’s regulatory process, and the Advisory Council’s recommendations risk becoming the fourth demonstration of how not to solve the problem. Congress should anticipate this outcome and implement a system capable of delivering real progress for Parkinson’s patients.

Hope for PD patients exists—but only if Congress acts decisively and soon.

Steven Zecola is a former technology executive and government official.  He retired 24 years ago with a diagnosis of Parkinson’s disease.   He currently is an ardent patient advocate.

Will HHS Enhance or Stall the Promise of Artificial Intelligence for Healthcare?

By STEVEN ZECOLA

In its Strategy for Artificial Intelligence (V.3), the Department of Health and Human Services (“HHS”) acknowledges that: “For too long, our Department has been bogged down by bureaucracy and busy work.” HHS promises that it will accelerate artificial intelligence (“AI”) innovation, including “accelerating drug and biologic approvals at the FDA.”

History shows that well-intended but cumulative regulatory intervention – more so than scientific complexity – is the primary deterrent to rapid technological progress. If AI is subject to the typical pattern of regulatory creep, its potential to accelerate drug discovery and development will be significantly reduced. To avoid this outcome, HHS should develop a plan that is premised on a zero-based regulatory approach. That is, each new technology such as AI should start with a clean slate and only the minimum requirements deemed necessary to show effectiveness and safety should be applied in the approval process for that technology.

The Pace of Innovation

Medical innovation has lagged the pace in the other sectors of the economy. As Dr. Scott Podolsky of Harvard Medical School observed: “Medicine in 2020 is much closer to medicine in 1970 than medicine in 1970 was to medicine in 1920.” Podolsky points to breakthroughs such as antibiotics, antihypertensives, antidepressants, antipsychotics, and steroids that have not been met with same impact as innovations in the later 50 years.

Two explanations have been offered for this phenomenon: 1) the inherent complexity of biological processes; and 2) the regulatory approval process.

As a benchmark for comparison to the following case studies, the development of 4G communications spanned less than a decade, with discussions starting around 2001, technical specifications being released in 2004, and the first commercial networks launching in 2009.

Regulatory Intervention in New Technologies

  1. The Human Genome (Great Science Leads to Regulatory Paralysis)

The Human Genome Project (HGP) ran from 1990 to 2003, and has been lauded as one of the world’s greatest scientific achievements. The project identified the specific location of genes and DNA, creating a “roadmap” of the human genetic code and facilitating the identification of disease-related genes.

The HGP focused on balancing rapid scientific progress with ethical safeguards. Oversight was primarily managed through internal ethical programs and international data-sharing agreements rather than a single overarching legislative or regulatory body.

Under this structure, the HGP beat its target date by two years. That is to say that the complexity of the problem did not cause any delays, and progress was not impeded by the standard drug-approval bottleneck.

However, once the genetic roadmap was handed off for drug discovery and development, progress slowed dramatically.

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The Dimensions of Artificial Intelligence in the Healthcare Industry

By STEVEN ZECOLA

On December 19th, the Department of Health and Human Services (“HHS”) issued a Request for Information seeking to harness artificial intelligence (“AI”) to deflate health care costs and make America healthy again.

As described herein, AI can be used in many dimensions to help lower healthcare costs and improve care. However, to achieve significant breakthroughs with AI, HHS will need to completely revamp the regulatory approach to drug discovery and development.

Dimension #1. Incorporation of AI into Drug Discovery

The biggest benefit to the healthcare industry’s performance from AI is achievable from drug discovery. Accounting for the costs of failures, the average FDA drug approval costs society almost $3 billion and takes decades to reach the market from its inception in the lab. 

In contrast, AI identifies potential treatments much faster than traditional methods by processing vast amounts of biological data, uncovering hidden causal relationships, and generating new actionable insights.

AI is particularly promising for complex, multifactorial conditions – such as neurodegenerative diseases, autism spectrum disorders, and multiple chronic illnesses – where conventional reductionist approaches have failed.

In the short-run, HHS should direct its grants toward AI-generated basic research, with a particular emphasis on the hard-to-solve illnesses. At the same time, the FDA should be putting into place a new approval system for AI-initiated programs to enable breakthrough treatments in a compressed timetable. 

Dimension #2. Incorporation of AI into the Drug Development Process

Simply relying on AI for drug discovery, while subjecting its advances to the current approval process would undermine the use of the technology. 

Rather, improvements from AI can already be had in fulfilling the exhaustive regulatory documentation requirements, which today add up to as much as 30% of the cost of compliance.

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Let’s get moving on AI-discovered treatments

By STEVEN ZECOLA

Recursion Pharmaceuticals announced results today for one its AI-discovered treatments. I was pleased to see the large, sustained reduction in polyps attributable to its treatment for Familial Adenomatous Polyposis.  Recursions’ oral medication will be viewed by the traditional scientific and regulatory community as “promising”.

On the other hand, I was disappointed not to see/hear any reference to the savings of the cost to society from this treatment and a vague reference to working with the FDA in 1H2026.  Quite frankly, the urgency seemed to be lacking.

Currently, treating FAP is an expensive, lifelong endeavor for the 50,000+ survivors. Early detection strategies cost $10k+ and late detection $37k+. The cost to treating metastatic colorectal cancer (for which FAP predisposes) can be extremely high, up to $300,000.  Overall, the cost to society from FAP easily exceeds $1 billion per year, or more than $15 billion on a present value basis.

This medication should not be subject to any further regulatory delay.  There is enough information now on efficacy and safety to have Recursion more forward with a broad application of this treatment, while continuing test dosage levels and stratifying the patient population.  The alternative is more needless cost and suffering.

Steve Zecola sold his web application and hosting business when he was diagnosed with Parkinson’s disease twenty three years ago.  Since then, he has run a consulting practice, taught in graduate business school, and exercised extensively

Artificial Intelligence Renders the FDA’s Current Drug Approval Process to be Obsolete

By STEVEN ZECOLA

Artificial intelligence (“AI”) has taken root in the field of drug discovery and development and already has shown signs of running past the traditional model of doing research.  Congress should take note of these rapid changes and: 1) direct the Department of Health and Human Services (“HHS”) to phase down the government’s basic research grant program for non-Ai applicants, 2) require HHS to redirect these monies to fund nascent artificial intelligence applications, and 3) require HHS to revamp the roadmap for drug approvals of AI-driven trials to reflect the new capabilities for drug discovery and development.

Background

There are four distinguishing features of the U.S. healthcare industry.

First, the industry’s costs as a percentage of GNP have increased from 8% in 1980 to 17% today, and are expected to exceed 20% by 2030.  The federal government subsidizes roughly one-third of these costs.  These subsidies are not sustainable as healthcare costs continue to skyrocket, especially in the face of an overall $37 trillion federal deficit.

Second, the industry is regulated under a system that results in an average of 18 years of basic research and 12 years of clinical research for each drug approval.  The clinical cost per newly approved drug now exceeds $2 billion.  The economics of drug discovery are so unattractive to investors that the federal government and charitable foundations fund virtually all basic research.  The federal government does so to the tune of $44 billion per year.  When this cost is spread among the 50 or so drug approvals per year, it adds a cost of roughly $880 million to each drug, bringing the total cost to over $3 billion per drug approval. Worse yet, the process is getting slower and more costly each year.  As such, drug discoveries under the current research approach will not be a significant contributor to lowering the overall healthcare costs.

Third, the Trump administration has undercut the federal government’s role in healthcare by firing several thousand employees from HHS.  Thus, the agency can no longer effectively administer its previously adopted rules and regulations, and therefore, cannot be expected to shepherd drug discovery into lowering healthcare costs.

Fourth, on the positive side, artificial intelligence software combined with the massive and growing computational capacity of supercomputers have shown the potential to dramatically lower the cost of drug discovery and to radically shorten the timeline to identify effective treatments.

Enter Artificial Intelligence (AI) into Drug Discovery

For the past decade, a handful of companies have been exploring advanced automation techniques to improve the many facets of the drug discovery process. Improvements can now be had in fulfilling regulatory documentation requirements, which today add up to as much as 30% of the cost of compliance.  More significantly, Ai can be used to accurately create comprehensive clinical documents from raw data with citations and cross-references – and continually update and validate the documentation.

The top Ai drug discovery companies include Insilico Medicine, Atomwise, and Recursion, which leverage Ai to accelerate various stages of drug development, from target identification to clinical trials. Other notable companies are BenevolentAI, Insitro, Owkin, and Schrödinger, alongside technology providers like Nvidia that supply critical Ai infrastructure for the life sciences sector.

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The Healthcare Industry Needs a Course Correction

By STEVEN ZECOLA

The United States healthcare system has failed by any measure.

First, costs are out of control. For example, 17% of the country’s GDP is spent on healthcare. This percentage was less than half that amount in 1980. It is expected to continue growing to 20% by 2032. Seventy-five percent of these costs are attributable to chronic diseases.

Second, notwithstanding the highest percentage of GDP spent on healthcare of the top ten high-income countries, the US has the worst performance outcomes whether measured on life expectancy, preventable mortality through disease management, and even access to care through insurance coverage or other means.

Third, the agency overseeing the healthcare industry is the Department of Health and Human Services. HHS is organized by functions such as Clinical Health Services and Behavioral Health Services rather than organized by disease management. The five strategic imperatives of its 4-year strategic plan do not contain benchmarks for improving the health status of the population, nor concrete steps to achieve the benchmarks. There is no mention of costs.

Fourth, the industry is huge and has many different components from healthcare providers to equipment manufacturers, to researchers, to pharmaceutical companies, to genetic companies, to insurance companies and so on. Over 16 million people are employed in the industry, with 60,000 in HHS alone. At this level of aggregation, leadership and management prowess becomes watered down and there can be no driving force for across-the-board improvements in disease management.

Fifth, the industry spends about $100 billion per year on R&D in pursuit of FDA approvals. The cost of this development translates into more than $2 billion per approved drug. Once approved, the drug effectively gains a barrier against unfettered competition. Independent analysts have estimated the costs of this regulatory scheme vastly exceed the benefits. Yet the FDA holds firm in its approach, given that its primary objective is safety.

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The Long and Tortured History of Alpha-Synuclein and Parkinson’s Disease

By STEVEN ZECOLA

This study tracks the decades-long journey to harness alpha-synuclein as a treatment for Parkinson’s disease. Steven Zecola an activist who tracks Parkinson’s research and was on THCB last month discussing it, offers three key changes needed to overcome the underlying challenges.

A Quick Start for Alpha-Synuclein R&D

In the mid-1990’s, Parkinson’s patient advocacy groups had become impatient by the absence of any major therapeutic advances in the 25 years since L-dopa had been approved for Parkinson’s disease (PD).

The Director of National Institute of Neurological Disorders and Stroke (NINDS) set up a workshop in August 1995 that featured scientists with expertise in human genetics who might open novel avenues for PD research.

One such scientist, Robert Nussbaum, made the following remarks at the workshop:

“…finding genes responsible for familial Parkinson’s should be helpful for understanding all forms of the disease. Techniques now available should allow researchers to find the genes responsible for familial Parkinson’s disease in a relatively short time.”

Two years later in 1997, Spillantini et al. showed that alpha-synuclein (A-syn) was a major contributor of abnormal clusters of proteins in the brain, not only in patients with synuclein mutations but, more importantly, in patients with sporadic Parkinson’s disease as well.

As Nussbaum had predicted, progress had occurred rapidly. President Clinton in his 1998 State of the Union address, said:

“Think about this, the entire store of human knowledge now doubles every 5 years. In the 1980’s, scientists identified the gene causing cystic fibrosis. It took 9 years. Last year scientists located the gene that causes Parkinson’s disease in only 9 days.”

The NIH is Asked to Take a Leadership Role

Shortly after President Clinton’s call to action, a Senate Committee asked the National Institutes of Health (NIH) to develop a coordinated effort to take advantage of promising opportunities in PD research.

In response, the NIH and the National Institute of Neurological Disease and Stroke (NINDS) held a major planning meeting that included all components of the PD community. The group’s recommendations formed the basis of a five-year PD Research Agenda.

The Research Agenda was codified in a comprehensive 42-page report that covered all aspects of research from better understanding the disease, to creating new research capabilities, to developing new treatments, and to enhancing the research process.

Noting the “remarkable paradigm shift in Parkinson’s disease research” from the discovery of the effects of alpha-synuclein, the report stated that:

“New insights into the role of synucleins in the pathobiology of Parkinson’s disease would accelerate discovery of more effective therapies and provide fresh research opportunities to advance our understanding of Parkinson’s disease”.

NIH invested nearly $1 billion from FY 2000 to FY 2004 to implement the PD Research Agenda.  A-syn research would be funded out of the funds allocated to the categories of Genetics and Epidemiology, with both categories targeted to receive about 15% of the overall spending.

Overall, there were 19 broad categories with spending authorizations, including $32.7 million allocated to Program Management and Direction.

When the PD Research Agenda reached the end of its 5-year span, NINDS sponsored a second PD Summit which was held in June 2005.  It brought together an industry-wide consortium to assess the progress over the previous five years and to develop future directions for PD research.

The participants generated more than fifty specific recommendations.  NIH considered these plans and the unmet goals from previous efforts and developed a 3-year Plan.

A major focus of that Plan was to identify and intervene with the causes of PD.

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A New Day for Parkinson’s Disease Research Is Near

By STEVEN ZECOLA

The U.S. Department of Health and Human Service (“HHS”) is responsible for a wide range of activities relating to medical and public health. It has 60,000 employees and a $1.7 trillion annual budget with approximately $140 billion for discretionary spending. For the past 13 years, HHS has been spearheading a National Plan for addressing Alzheimer’s disease – with some notable successes.

Given its resources, expertise and charter, HHS should launch a National Plan to cure Parkinson’s disease patterned after its approach on Alzheimer’s disease.

Legislation, or Not

The U.S. House of Representatives has passed H.R.2365, the National Plan to Cure Parkinson’s Disease.

The bill would establish HHS as the central point for strategic direction and coordination of PD research.  It would require formation of a broad-based Advisory Panel to provide strategic advice and any on-going course corrections.

There is nothing preventing HHS from putting the structure of H.R. 2365 into effect now, and it should do so without waiting for Senate action or inaction. There is no incremental funding required to implement this National Plan, nor is any Congressional approval necessary.  This approach would mark an important step towards finding a cure for Parkinson’s disease, and is well within HHS’s charter.

A Cross-Section of Policy and PD Research

For those who have studied the application of regulatory policies to Parkinson’s disease research, it does not provide a productive narrative.

Levodopa was first discovered in 1910. In 1975, after 14 years of its “miraculous” treatment of PD symptoms, the FDA approved the drug. Levodopa does not cure or delay the progression of the disease. Yet, it has remained the gold standard of treatment of PD for the past fifty years. That is not to say there has been insufficient research or inadequate FDA approvals.  Rather, it’s a question of where the research dollars have been funneled. It turns out that levodopa becomes less effective over time and eventually produces uncontrolled shaking. Therefore, research dollars have been targeted toward drugs that delayed the need for levodopa or controlled its side effects.

An exception to this approach was Geron, which became a leader in embryonic stem cell research. It had raised $100 million to conduct clinical trials. However, most of that money was consumed by undertaking thousands of experiments on mice under the “guidance” of the FDA. Nevertheless, Congress saw the potential of embryonic stem cells, and passed the Stem Cell Research Enhancement Act.

While Congress cheered, the Evangelical movement viewed embryonic stem cell research as barbaric and akin to murdering a human life. It didn’t matter that embryonic stem cells could not become a living being unless they were implanted in a woman’s womb, and this step wasn’t part of the research efforts.  Notwithstanding, the Evangelicals convinced George W. Bush to veto the legislation, and a promising path for PD research was shut down.

More recently, the House has passed bills for a National Plan to Cure Parkinson’s in its last two sessions, but the Senate has failed to act, despite a myriad of sponsors of a bill with similar provisions.

Building Upon Lessons from the Past

In 2011, Congress passed legislation establishing a National Plan to Address Alzheimer’s disease (“NAPA”).  Thirteen years later, there are many lessons to be learned from that effort that can be applied in a National Plan for PD. Of particular note, the original plan had five objectives including to “Prevent and Effectively Treat AD/ADRD by 2025”. 

The first report by the Advisory Council specified that the current “level of resource commitment falls drastically short of the funding needed to accelerate the pace of research on prevention, cures, and treatments for AD”. It also recommended that the Secretary examine “[h]ow HHS uses existing authorities to reduce drug development barriers and accelerate development of new therapies” and specifically called for recommendations to “accelerate the FDA review process”.

What happened?  While funding was increased substantially and hundreds of potential treatments have been identified, only two drugs have been approved by the FDA under an “accelerated” review process.

While HHS may express pride in the accomplishments from the Alzheimer’s National Plan, it should conclude that the process to get an effective treatment identified and approved takes too long. For example, the FDA provides “guidance” to researchers even before clinical trials are submitted. It also regulates the provision of genetic tests. These actions needlessly slow development and reduce innovation.  

Similarly, the FDA’s regulation of Phase 1 and Phase 2 trials slows down development and does little to benefit the public interest. The FDA points to multiple ways that it has accelerated the drug approval process.  But the reality is that progress from PD research has been lacking.

On the other hand, in 2019, researchers issued a report – based on real-world observations — that Terazosin resulted in a lower incidence of PD and a slower development of the disease when it did occur.  Terazosin has been used for over 35 years to treat other maladies. Yet the drug underwent a 13-person Phase I trial to determine if it is safe. This phase 1 trial took several years to complete. This approach was a distraction that caused unnecessary delay and cost under the FDA’s regulatory regime.

The FDA will say that its rules do not require 3 (or more) trials nor does it mandate a particular trial design. This is disingenuous. Companies spending hundreds of millions of dollars on research cannot afford the risk of shirking the FDA’s standard procedures.

Taken as a whole, the HHS should limit the FDA’s involvement in PD research to approval of Phase 3 trials. Such an approval process will speed development and foster innovation yet maintain adequate safety controls by the FDA. Research organizations would be less constrained in developing their strategies and would be held to more responsibility for their approach to research.

A Multivariate Solution Is Likely to be Required

PD is a complex disease that has different manifestations when looked at from a genetic, diet, exercise, environmental (pesticides/pollution/solvents), vitamin, drug, electronic, radiation and possibly other perspectives. As such, a multivariate solution is likely to be required to successfully treat PD. 

Such a solution will not be well accommodated by the current FDA review process, with each different combination of therapies being subjected to regulatory review and intervention.  The process could drag on for decades.

HHS should recognize the need for a multivariate solution and plan accordingly, as described below.

Data Collection to Identify Multivariate Solutions

In 2010, The Michael J. Fox Foundation launched the Parkinson’s Progression Markers Initiative (PPMI) to find the biological markers of Parkinson’s onset and its progression. That study led to the impressive finding of a tool that can detect pathology not only of people diagnosed with Parkinson’s, but also in individuals that are at a high risk of developing it. However, after ten years, that study has only a few thousand participants. HHS should endorse and expand the scope of that study.

The “second version” of PPMI should be an overlay study designed with the end game in mind. That is, it should produce a mapping of individual people’s PD “score” over time against all relevant explanatory variables that could possibly impact PD for each individual. Such an approach is superior for identifying multivariate solutions.

To accomplish this objective, each participant would establish and maintain a unique portal for his/her own explanatory PD variables. The portal would include a series of hard-coded entry requirements covering scores of inputs. The initial set-up could be completed in piece-part (with the availability of outside assistance) and would auto-populate with each quarterly update (allowing for input of any changes that occurred after the initial set-up). The portal would interface with the growing number of portals of individual healthcare providers and would collect the diagnostic information from those systems. Personal “meters” of this sort are now actively being deployed in the field of Alzheimer’s disease given that certain therapies and drugs have shown progress against that disease.

As the above information from participants is collected over time, artificial intelligence software would be used to identify combinations of diet, exercise, supplements, genetics, sleep habits, therapies, electronics, radiation and drugs that point towards promising results. New treatments such as those undertaken in clinical trials would be added to the participant’s portal as they as are pursued by those individuals. All of the patient’s existing drugs would be analyzed in the context of all other relevant explanatory variables for that participant – over time.

As importantly, a comparative, quantifiable measurement of PD over time for each individual is required. The PPMI was originally focused on identifying a marker for PD and therefore uses a series of qualitative questions to gauge the patient’s development of PD symptoms over time. In contrast, the emphasis for this data collection effort should shift to the explanatory variables affecting PD progression over time.

In terms of the participant’s PD score, I believe a modified version of the Fitness program currently designed for the computer game “Wii” (which provides a quantitative estimate of an adult’s age based on how that person performed on certain activities) would provide more reliable results. Each participant would provide his/her own age estimator from the computer program on a quarterly basis as well as provide any updates for the various explanatory variables.

Once this revised format is established, the HHS should establish a goal of enrolling 100,000 PD participants into the study within two years.

A Better Approach for PD Research Is Available Now

HHS can – on its own accord – dramatically improve the efficiency and effectiveness of Parkinson’s research by: 1) adopting the industry-wide structure it utilized for Alzheimer’s disease, 2) embracing and expanding upon the current PPMI study and 3) limiting the FDA’s involvement in research to the approval of Phase 3 clinical trials.

Steve Zecola sold his web application and hosting business when he was diagnosed with Parkinson’s disease twenty three years ago.  Since then, he has run a consulting practice, taught in graduate business school, and exercised extensively

The FDA’s Culture: Should Safety Dominate All Practices?

By STEVEN ZECOLA

An organization’s culture is an internal set of shared values, attitudes and practices. The cohesiveness of the organizational culture will affect whether the entity will meet its vision, purpose, and goals.

One type of organizational culture is hierarchical in nature.   Unlike a risk-taking culture, this structure features policy, process and precision. It is best suited for mature and stable organizations.

The disadvantage of a hierarchal culture is that its stability and control can turn into rigidity. In many cases, the organization develops a negative attitude towards ideas supplied by third parties. It paints itself as having the perfect answer for every issue, no matter how large or small.

My interactions with the FDA suggest that its cultural practices are focused on safety, seemingly to the exclusion of all other issues.  This practice may be appropriate in the regulation of food, but not for drug research where flexibility and creativity are required to cure complex diseases.

Over the past decade, I have witnessed an excessive adherence to its existing practices in the context of BRCA1-related breast cancer, metastatic cancer, precision medicines, “Big Data” and Parkinson’s disease. While the rulings were directed at me, the FDA’s position on these issues has impacted millions of people for the worse.

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The Case to Realign Parkinson’s Disease Research

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By STEVEN ZECOLA

If asked, the leaders of the research organizations working on Parkinson’s disease would say that they have made tremendous progress and are optimistic on finding a cure for the disease. 

In truth, this viewpoint understates the magnitude of the challenge and results in insufficient resources being devoted to PD. Given the size of the challenge versus the available resources, most research studies today don’t even include finding a cure for PD as part of their objective.

The time is ripe to get everybody on the same page when it comes to the objectives, resources, and timelines for PD research.

What We Know About Parkinson’s Disease

Parkinson’s disease (PD) is a chronic, progressive movement disorder that affects the lives of almost one million Americans. Roughly 50,000 of the inflicted people die each year, often by injuries from falling.  The incidence of PD is expected to expand to 1.6 million in the U.S. by the year 2037.

The characteristic motor symptoms of PD are tremors, stiffness, slowed movement and impaired balance. Over time, people with PD also experience non-motor symptoms including changes in mood, problems with attention and memory, sleep disturbances, fatigue, and changes in bowel and bladder function.  PD has a considerable impact on the quality of life.

The cost to treat PD has been estimated to be $50 billion a year, split equally between the direct cost of care and the indirect costs of lost opportunities for the patients and caregivers.

PD is a complex disease which is thought to result from an interaction between genetic and environmental risk factors.  More than 20 genes have been identified as having an impact on the onset of PD.  However, genetic variation is estimated to contribute only about 25% to the overall risk of developing PD. Moreover, like the majority of neurodegenerative disorders, little insight is available on how specific sequence variations contribute to disease development and progression.

In short, the exact cause of PD is unknown.  However, we know that that there is more than one manifestation of the disease. We can also reasonably conclude that more than one single element or therapy will be required to cure the disease.

What We Know About Parkinson’s Disease Research

PD was first discovered and described by James Parkinson in 1817 in London, England.

In 1911, the efforts of Kazimierz Funk, a Polish biochemist, paid off with the identification of Levodopa as a potential treatment.

By 1970, the FDA approved the use of Levodopa combined with Carbidopa for the treatment of PD. Since then, this combination has remained the gold standard for treatment.

During the last 50 years, many attempts have been made to improve this treatment and avoid its long-term complications.  While several enhancements have been approved by the FDA and have helped patients, no treatment has cured or slowed the progression of the disease.

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