By Jessica Wong

Every year, dementia kills more Americans¹ than prostate cancer and breast cancer combined. Advances in life sciences are moving researchers, patients, and carers closer to enjoying a better quality of life for longer. The digital transformation of biotechnology and life sciences is key to transferring results from laboratories to family homes.

One in three seniors¹ in America is dying with a form of dementia, according to the Alzheimer’s Association. Dementias include Alzheimer’s disease, Parkinson’s disease dementia, Rett syndrome, and other related diseases.

Advances in life sciences are moving our society closer to managing dementia and offering patients greater quality of life with the illness. Some of the most promising treatments are in clinical trials right now. These precision medications are aiming to attack the disease at its source, by protecting nerve cells in the brain and the spinal cord.

Leading biotechnology and life sciences companies are developing medications that have the potential to make management of Parkinson’s and Alzheimer’s as simple as taking a pill. One of the most promising candidates currently undergoing clinical trials is ANAVEX®2-73. The drug works by binding with a protein known as the sigma-1 receptor.

All forms of dementia affect the brain and the spinal cord. They lead to a long, slow decline in the physical and mental health of the patient. Dealing with Alzheimer’s and other dementias puts a strain on family members, who often act as front-line carers. These diseases also have a wider detrimental impact on our economy.

The Alzheimer’s Association estimates that dementia care cost the economy more than $300 billion² in 2022 alone. These figures do not yet include the hours of unpaid care provided by family members. Unpaid carers are providing more than $270 billion worth of care every year.

What Future Dementia Treatment Could Look Like

Dementia develops when nerve cells in the brain, the spinal cord, and its connecting tissues are damaged or lost. Nerve cell damage happens most often because of underlying diseases, such as Parkinson’s or Alzheimer’s. Despite scientific advances, once nerve cells have been damaged, they cannot be regenerated.

With multiple functions in protecting nerve cells in the brain and the spinal cord, the sigma-1 receptor has emerged as one of the main targets in treating diseases like dementia. Those with dementia have been found to have a much lower volume of sigma-1 receptors. ANAVEX®2-73 binds to this receptor and increases its activity.
Research and early clinical trials have been supported by the Michael J. Fox Foundation for Parkinson’s Research. Since then, the drug successfully completed several stage-two trials that have shown that it can improve common symptoms of dementia.³

According to Lancet Public Health, by the year 2050, more than 150 million people worldwide will have dementia.⁴ The total healthcare costs for the treatment of Alzheimerʼs disease in 2020 was estimated by the American Journal of Managed Care to be approximately $305 billion, with the cost expected to increase to more than $1 trillion as the population ages.⁵ In the future, being able to take a simple pill prior to the onset of dementia is vital to relieving the financial and social burdens of dementia conditions on the global economy.

When envisioning the future of dementia treatment, trials of ANAVEX®2-73 suggest that society may be close to being able to effectively treat dementia conditions. Trials of this drug related to Parkinson’s disease dementia showed improvements in movement- and non-movement-related symptoms of the disease.⁶ Scientists linked the improvement in symptoms to higher activity levels in the patients’ sigma-1 receptors. Blood tests on trial participants also showed that the drug improved the function of genes that have been impaired in patients with Parkinson’s, Alzheimer’s, and other dementias.

How Can Digital Innovation Change the Course of Dementia

The biotechnology and pharmaceutical sectors have traditionally been slow in adopting digital advances. Even leading companies in the field resisted altering their operations to integrate technologies like artificial intelligence (AI) or the cloud.⁷

This slow approach changed during the coronavirus pandemic. Deloitte’s consultants
noted that the onset of the pandemic forced several companies to accelerate their digital transformation plans, often taking years off planned implementation targets. As a result, the face of operations changed. McKinsey notes⁸ that clinical trials, for example, have become “decentralised, virtual, and customer-centric”.

However, not every company required the restrictions of the pandemic in order to incorporate digital innovation into its strategic business practices. Smaller biotechnology companies like Anavex Life Sciences deployed decentralised clinical trials prior to the pandemic through the adoption of customer-centric practices.⁹

Additionally, biotechnology and biopharmaceutical companies leverage their websites to connect with a range of stakeholders. Rather than keeping information about research advances siloed to industry experts, companies are now publishing overviews of their most promising drug candidates on their websites, in addition to patient-centric landing pages. For example, Anavex Life Sciences has ten dedicated pages to the various conditions the company aims to treat.¹⁰

The use of digital platforms to both educate and collaborate with the patient and carer communities regarding the various stages of drug development is a pivotal step towards expediting innovation and satisfying the warranted demands of the patient and carer communities.

Deloitte’s research shows that biotech companies are now prioritising targeted digital investments, intending to bring them together for maximum impact. According to the consultancy’s survey of 150 industry representatives,⁷ the industry is looking to meet research goals faster than ever before and engage patients, partners, and other stakeholders more optimally.

Apart from patient and carer engagement, other applications of digital technology in the field include advanced analytics through the use of AI, and improved access to data thanks to cloud-based storage and handling. Put together, digital advances in the field of biotechnology and pharmaceuticals can bring drugs to market faster and, ultimately, improve patients’ lives.

How Will Patients Benefit?

Dementia affects every aspect of a patient’s life, and the disease does not spare their families either. Early symptoms of mild cognitive impairment (MCI) may go unnoticed or simply attributed to normal signs of ageing. However, as dementia takes hold, sufferers experience disorientation, more significant memory loss and changes to their personality.

The illness also makes it harder to use language and might lead to problems with day-to-day activities such as personal hygiene or getting dressed. Over time, dementia worsens and leads to physical and psychological challenges for patients, their carers, and their families.

Experts predict that dementia will become more prevalent over the next two to three decades. The increase is mostly due to the ageing of the baby-boomer generation, which will change the look of America’s society. Alzheimer’s alone is currently affecting more than six million Americans. By 2050, the Alzheimer’s Association believes1 the number will rise to nearly 13 million people aged 65 or over.

Statistics from the Centers for Disease Control and Prevention (CDC) corroborate these estimates. The CDC’s projection assumes that there will be 14 million dementia sufferers in the United States by 2060.⁷

Aside from the human cost of the diseases, there is the pressure on a struggling economy. With the projected costs of dementia likely to reach $1 trillion in this country alone, it is clear that there is a need for effective dementia therapeutics.
Current clinical trials suggest that drugs like ANAVEX®2-73 can have a significant impact on a wide range of dementia symptoms, ultimately improving the quality of life of patients.

This is a significant achievement on its own and will have positive rippling effects across the economy. As dementia sufferers can manage their symptoms better, they are less likely to need care. Consequently, family members who are now caring for a relative with dementia could remain in the workforce, relieving the strain on the economy.

Rare Diseases Targeted

There is another benefit to digitally driven research for drugs like ANAVEX®2-73. Instead of exclusively investigating drugs for their potential to treat diseases affecting millions, scientists are also considering the effectiveness of these drugs in treating rare diseases. To that effect, the Rare Advocacy Movement (RAM) works with a variety of therapeutic manufacturers through the rare360 programme to assist these biopharmaceutical and biotechnology companies in identifying the most appropriate rare diseases to target.

The goal is to use digital technologies to obtain accurate, real-world, community-based insights. These insights are extremely valuable, as they expedite the process of moving a therapy from its pre-clinical stages to the commercial market.

Traditional rare-disease patient advocacy has slowed the pace of therapeutic development through privileged silos and the bottlenecks of territorial 501(c)3 non-profit groups. However, RAM’s novel community-based platforms and the rare360 programme¹² are the first-ever tools available to the drug development continuum that take a wider approach.

Pros and Cons

PROs

Digital transformation speeds up drug development and time to market.
Digital technology supports decentralised clinical trials and enables enhanced analytics and data sharing.

Digitalisation enables patient and carer engagement early in the process.
Patients diagnosed with a neurodegenerative condition (like dementia) and/or a neurodevelopmental disease (like Rett syndrome, a rare disease) benefit from improved quality of life and access to precision medicine.

CONs

Digital transformation requires changes to the status quo of drug development.
Pharmaceutical companies need to commit to initial investments and amend institutional processes and protocols.

The adoption of digital technologies is key to driving drug development and improving the lived experiences of patients diagnosed with debilitating conditions like dementia. Advances in precision medication capabilities will change the course of how dementia is treated in the future.

Key Takeaways

• The detrimental economic impact of dementia runs into hundreds of billions every year and is projected to triple by 2050.
• Scientists are on the brink of offering simple, yet powerful treatments to improve disorders of the brain and spinal cord. Anavex Life Sciences is an industry-leading biotechnology company that is dedicated to finding a successful treatment for conditions that impact the brain and spinal cord.
• Digital technology is critical to bringing therapies to the market faster, engaging with healthcare professionals and the patient and carer communities respectfully, and in revolutionising the value chain of the entire drug development industry.

This article was originally published on 3 December 2022.

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 bottom-line results for clients through innovative programmes aligned with emerging business strategies.

As a digital expert, Jessica was invited to publish thought-leadership articles on Forbes.com as an official member of the Forbes Communications Council. She also provides business advice to millions of Entrepreneur.com and Franchise500.com readers.

The Women in IT Awards also named Jessica a finalist for the Digital Leader of the Year in both 2018 and 2020. MARsum USA 2021 has recognised her as one of the Top 100 Marketing & Advertising Leaders.

In recognition of her work with Valux Digital, Jessica was awarded CEO of the Year for 2021 & 2022 by AI Global Media and the Most Influential CEO of the Year 2022 (New York, USA) by the CEO Monthly magazine based on an in-depth review and thorough research.

The Vaux Digital team has notably been accepted into Rare 360. This is a programme hosted by the Rare Advocacy Movement (RAM), focused on addressing the issues that affect people suffering from rare diseases and are part of the rare disease community.

References

1. https://www.alz.org/alzheimers-dementia/facts-figures#:~:text=More%20than%206%20million%20Americans%20of%20all%20ages%20have%20Alzheimer’s,older%20(10.7%25)%20has%20Alzheimer’s.
2. Link to press release mentioned in your document or media outlet having picked it up.
   “GBD 2019 Dementia Forecasting Collaborators”, Lancet Public Health. 7(2):E105-E125; Jutkowitz
3. E, Kane RL, Gaugler JE, MacLehose RF, Dowd B, Kuntz KM. “Societal and Family Lifetime Cost of Dementia: Implications for Policy”, Journal of the American Geriatric Society. 2017 Oct;65(10):2169-75.
4. W Wong, “Economic Burden of Alzheimer Disease and Managed Care Considerations”, American Journal of Managed Care. 2020;26:S177-S183.
5. Kulisevsky J, et al. ANAVEX®2-73 (blarcamesine): “Analysis Of Movement (MDS-UPDRS) And Cognitive (CDR System) Pharmacodynamic-Biomarker Outcome Measures of Placebo-Controlled Phase 2 Trial in 132 Parkinson’s Disease Dementia Patients”. Presented at AD/PD 2022, Barcelona, Spain, 15-20 March 2022.
6. https://www.rareadvocacymovement.com/post/great-news-for-rett-syndrome-and-other-similar-rare-diseases-on-the-autism-spectrum
7. https://www2.deloitte.com/us/en/insights/industry/life-sciences/biopharma-digital-transformation.html
8. https://www.mckinsey.com/industries/life-sciences/our-insights/five-ways-that-life-science-companies-can-build-tech-talent
9. https://www.anavex.com/therapeutic-candidates
10. https://www.anavex.com/our-dedication
11. https://www.cdc.gov/aging/dementia/index.html
12. https://www.rareadvocacymovement.com/rare360

Gene therapy is one of the most fascinating areas of biotechnology, both in terms of the signals of success that are being made in the field right now and the possibilities that lie ahead. 

The developments in technology that have made it possible to change the human immune system, control the distribution of nucleic acid, and define the scope of human genome manipulation have sparked the development of entirely new subfields within the field of a medical study. 

Recent advances in gene therapy have provided a platform upon which further generations of technology can be built. The potential of gene therapy is the primary subject of this article.

How Gene Therapy Works

Sometimes a gene is defective or missing in all or in part from birth, or a gene might change or mutate as an adult. Any of these differences can interfere with the production of proteins, leading to illnesses or other health issues.

Depending on the issue at hand, scientists can use gene therapy to accomplish a variety of goals. They can add genes to help the body fight or treat disease, switch off genes that create issues, or replace a gene that causes a medical condition with one that doesn’t.

Scientists utilize a “vector,” genetically tailored to convey the gene, to directly introduce new genes into cells.

For instance, viruses can be utilized as vectors since they naturally possess the ability to transfer genetic material into cells. However, a virus must first be altered to remove its capacity to spread an infectious disease before being utilized to deliver therapeutic genes into human cells.

Cells inside or outside of the body can be modified using gene therapy. A doctor will perform the procedure within the body by injecting the vector containing the gene directly into the area of the body that has damaged cells.

In gene therapy, which modifies cells outside of the body, a patient’s blood, bone marrow, or other tissue can be removed, and in platforms like lentiviral vector company, particular cell types can be isolated. These cells are then provided with the vector containing the interest gene. 

After being allowed to multiply in the lab, the cells are injected back into the patient, where they continue to grow and finally have the intended effect.

How Gene Therapy is Going to Change the World

Next-generation technologies are significantly increasing the impact of such medications on treating human disease, even while it is fascinating to think about how these early gene therapy triumphs might be generalized to other conditions and patient populations. 

For instance, the immunological response to foreign transgenes and their products continues to be a major barrier to wider deployment. As a result, some of the most significant research will take place in the near future in immune system management.

In the near future, it will also be necessary to replace some expensive treatments for specific conditions with less expensive ones. The ability to evaluate the safety profiles of the treatment has been greatly exceeded by the rate of technological progress in genes. 

According to current regulatory frameworks, a sizable sample of patients is necessary to demonstrate the treatment’s safety and effectiveness. For both common and unusual disorders, new genetic medicines will be created in the future. Similar to biologics, gene treatments are anticipated to experience major advancements in the years to come.

This article was originally published on 27 July 2022.