Digital Innovation Could Transform Rare Disease Treatment – But Short Attacks are Putting Companies and Patients at Risk

By Jessica Wong

Digital technologies are changing the way biopharmaceutical and life sciences companies are approaching drug development. As artificial intelligence (AI) and machine learning (ML) are finding their way into laboratories worldwide, patients stand to benefit from faster, cheaper drug development.

This new way of developing a drug candidate and bringing it to clinicians and patients has the potential to be a game-changer for some of the most common as well as rare diseases. Among the most promising current developments is a pill with the potential to treat dementia. Similar technologies could also bring hope to rare disease sufferers. Many of those developments are driven by small, innovative biopharma companies without the backing of industry giants.

Their ability to develop groundbreaking drugs is closely tied to their stock market value, making them vulnerable to so-called short attacks. Apart from jeopardizing a company’s future, these illegal attacks are putting patients’ lives at risk.

How Digital Innovation Can Change the Treatment of Dementia

Right now, one in three Americans die with Alzheimer’s disease or another form of dementia. These figures put dementia ahead of prostate cancer and breast cancer combined, making it by far the largest killer of American seniors.

But the mortality rate of dementia is not the only toll the disease takes on American society. More than six million people are currently living with the disease, and many of those are relying on unpaid carers. The Alzheimer’s Association estimates that more than 11 million Americans are caring for a loved one with dementia and are providing services with a total value of more than $270 billion.

Caring for patients with Alzheimer’s and other diseases costs the country over $320 billion in 2022 alone. Due to an aging population, these figures are projected to reach $1 trillion by 2050 – unless scientists discover a game-changing treatment.

The Michael J. Fox Foundation for Parkinson’s Research has been one of the leading supporters of research in the field for years. Among other projects, the foundation supports small, innovative biopharmaceutical companies researching potential treatments for Parkinson’s Disease Dementia (PDD) and related conditions.

One of the foundation’s recent research grants was awarded to New York-based Anavex Life Sciences, a company whose scientists are working on a drug to treat PDD, Rett syndrome, and other forms of dementia as well as rare diseases that impact the central nervous system. Known as ANAVEX®2-73, the drug binds to the sigma-1 receptor in a patient’s brain and spinal cord and stimulates it.

Research has shown that dementia patients have fewer of these receptors than their healthy counterparts. By stimulating the activity levels of existing sigma-1 receptors, ANAVEX®2-73 looks set to protect the health and function of nerve cells, thus reducing the symptoms of various forms of dementia. The foundation’s grant fully funded preclinical work and the drug candidate has since successfully moved on to advanced clinical trials.

From the beginning, Anavex’s approach to its research and trial phase had digital technology at its heart. The goal was to streamline the research process and shorten the time between laboratory and patient without compromising research integrity and quality.

The Role of AI and ML in Drug Development

Traditional clinical trials involve a relatively large volume of patients who need to travel to the location of the trial to receive their treatment. Technologies like artificial intelligence and machine learning are creating new ways for biopharmaceutical companies to develop potentially life-saving treatments faster and more efficiently. In addition, by integrating digital technology into their approach to research, life science companies are minimizing the environmental impact of their work.

Faster, More Cost-Effective Drug Development

Some of the most noticeable changes are a move away from large clinical trials. Instead, innovative pharma and biotech companies are leveraging machine learning to enhance the outcomes of smaller trials. Paired with precision medicine tools like whole genome analysis, developers have been able to draw significant conclusions faster.

In the case of Alzheimer’s drug development, researchers were able to analyze all available patient data and efficacy outcomes through unsupervised formal concept analysis (FCA). As a result, the scientists could draw significant conclusions from a comparatively small sample of dementia patients. Specifically developed for this particular clinical trial, the FCA was part of a more comprehensive knowledge extraction and management (KEM) environment.

Thanks to taking a digital approach, the scientists were able to draw meaningful conclusions from a relatively small sample. In addition, the technology allowed them to analyze larger amounts of data faster than humans alone could have done.

By being able to limit the size of clinical trials without compromising outcomes, biopharmaceutical companies can shorten the time to bring a new drug to market. In addition, they are also able to limit the costs of drug development significantly.

Environmentally Friendly Drug Development

Another ‘side effect’ of using digital technologies in drug development is the reduced environmental impact of clinical trials. When Anavex Life Sciences entered the trial phase for its Alzheimer’s drug candidate, the coronavirus pandemic had not yet hit. Still, the company knew that it wanted to minimize its carbon footprint and overall environmental impact.

Even before travel became difficult, Anavex introduced video conferencing and decentralized clinical trials. Video technology eradicates the need for non-essential travel almost entirely. Taking a decentralized approach to clinical trials makes trials more accessible for patients to participate from various locations by removing the burden of travel. Based on the success of early decentralized trials, scientists are now considering entirely new approaches to therapeutic delivery and overall clinical trial designs.

To further limit the environmental impact of pharmaceutical research and development, smaller biopharmaceutical companies have started to move away from owning wet laboratories which can stay idle for months at a time. Instead, they are limiting their wet laboratory work to animal toxicology studies required by regulatory agencies as well as other necessary preclinical work. By reducing the overall time and costs involved in the development of life-saving drugs, patients and their families benefit.

The Impact on Rare Disease Drug Development

In the United States, a rare disease is an illness that affects less than 200,000 people¹ in the country. The European Union’s definition is somewhat different, but patient concerns are similar. From the traditional biopharmaceutical company’s perspective, drugs to treat rare diseases have limited commercial potential, simply because there is a limited number of patients that would benefit from the drug.

Despite medical advances, only 10% of rare diseases in the U.S. currently have an FDA-approved treatment². Cost – and time-savings made possible by using AI and ML in drug development are bringing hope to the rare disease community. These savings are making it easier for drug developers to invest in innovative treatments even for small rare disease communities.

How the Stock Market is Influencing Life Sciences Innovation

Many smaller biotechnology companies rely heavily on the stock market to fund their research and operations. On one hand, the stock market allows these companies direct access to funding. On the other hand, it leaves them vulnerable to the potentially detrimental impact of market manipulation.

One of the most detrimental developments is so-called short attacks³. Not to be confused with fully legal short selling, where a broker bets on a stock’s price to fall, short attacks equate to illegal market manipulation. When a stock is being attacked in this way, deliberate misinformation and negative press coverage are planted to drive down the price of the stock in question. Large, well-established pharmaceutical companies are often able to ride out a period of volatility.

However, the same degree of financial instability can be enough to threaten the health of small biopharmaceutical companies. The ones suffering the most are patients whose complex and rare disease clinical trials are put at risk by short attacks and who may not receive life-improving or life-saving treatments as a direct result.

Conclusion

In today’s world, digital technologies like artificial intelligence and machine learning have found a path into all aspects of our lives, and the biopharmaceutical industry is no exception. These technologies have the potential to enhance data gathered during clinical trials, among other benefits. Technology is leveling the playing field between smaller, more agile, and innovative pharmaceutical and biotech companies and their larger, more established competitors.

Smaller players may lack the resources of their larger counterparts. However, their size allows them more flexibility to adapt to new technologies and processes. Patients stand to benefit from innovative drugs for diseases like Alzheimer’s and other dementias coming to market faster, thanks to a more streamlined development process and more targeted clinical trials. Technology has the potential to cut the costs and time involved in drug development. These innovations are also bringing hope to rare and complex disease sufferers.

As researchers and scientists continue to integrate digital technologies into their work, we are destined to see the biopharmaceutical sector transform almost beyond recognition within the next few years.

About the Author

Jessica Wong, Founder and CEO of Valux Digital, is a digital marketing expert and experienced PR executive with over 20 years of success driving results for clients. Using cutting-edge technology, Jessica and her team at Valux Digital helped corporations and startups achieve digital transformation and develop robust data-centric marketing campaigns. They strive to improve efficiency, affordability, rapidity, quality, and – most importantly – results.

As a digital expert, Jessica was invited to publish thought leadership articles on Forbes as an official member of the Forbes Communications Council. She also provides business advice to millions of Entrepreneur.com readers. She was named the Most Influential CEO by CEO Monthly magazine.

The Women in IT Awards have named Jessica a finalist for the Digital Leader of the Year. MARsum USA has recognized her as one of the Top 100 Marketing & Advertising Leaders. In recognition of her work with Valux Digital, The Female CEO of the Year Awards recognized her as the Best Digital Marketing & Public Relations Agency CEO. The Global100 Awards have also named Jessica CEO of the Year.

Through her extensive digital experience in pharmaceutical, healthcare and biotechnological, Jessica has been named as the authorized digital transformation advisor for the Rare Advocacy Movement (RAM), the first community-based decentralized autonomous organization dedicated to the best interests of people diagnosed with life altering rare diseases and their families.

References

1. Kimmel L, Conti RM, Volerman, A, Chua, KP.  Pediatric Orphan Drug Indications: 2010-2018. Pediatrics (2020) 145 (4). https://doi.org/10.1542/peds.2019-3128
2. Haendel M, Vasilevsky N, Unni D, Bologa C, Harris N, Rehm H, et al. How many rare diseases are there? Nat Rev Drug Discov. 2020;19:77–8. doi: 10.1038/d41573-019-00180-y.
3. Mitts, Joshua, Short and Distort (February 13, 2020). Columbia Law and Economics Working Paper No. 592. http://dx.doi.org/10.2139/ssrn.3198384

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