Editorial Feature

Nanomedicine for Rare Diseases

Nanomedicine is a rapidly advancing field that combines nanotechnology with medical applications. The versatility of this innovative technology has made it a popular research area for many scientists and pharmaceutical developers.

Nanomedicine for Rare Diseases

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Researchers are promisingly pursuing the use of nanomedicine for rare diseases. With high-level technology enabling surface modification, targetability, and even the crossing of the blood-brain barrier (BBB) for advanced treatment, they may be able to target these diseases for the first time.1

This article will provide insight into how nanomedicine can be utilized for rare diseases.

Introduction to Nanomedicine and Rare Diseases

The development of nanomedicine began in the early 1990s as a promising technique with many therapeutical benefits, with applications in areas such as neurodegenerative and infectious diseases, as well as cancer.1

The term “nano” describes objects that are on a nanometric scale of 1-1000 nm, including nanoparticles, nanocarriers, and nanodrugs. These components can effectively interact with biological systems to treat various disorders, especially those related to the central nervous system (CNS).1,2

Approximately 30 million Americans, half of whom are children, suffer from a rare disorder. Rare diseases are typically caused by significantly distinct genetic mutations. They are characterized by having a prevalence of less than 200,000 people within the United States or 1 in 2,000 people in the European Union.1

Neurological disorders can impact the nervous system, including the brain, spinal cord, and all the nerves that run throughout the body. These disorders encompass many rare diseases, with pathologies categorized as either targeting the CNS or the peripheral nervous system (PNS).

The National Institute of Health database includes more than 1,000 nervous system pathologies, with most being considered rare diseases.1

Challenges in Treating Rare Diseases with Conventional Medicine

The BBB is a complex structure composed of specialized endothelial cells tightly connected by tight adherens junctions. These junctions are highly restrictive—they limit the entrance of most pathogens and large and hydrophilic molecules, while permitting the entry of small non-polar and hydrophobic agents.1

The BBB poses a significant challenge for 98 % of neurotherapeutic drugs that are unable to reach the brain, making the treatment of rare neurological diseases difficult. Without crossing the BBB, CNS-related rare disorders cannot be treated effectively, leading to poor prognoses and high mortality for patients.1

Additional challenges associated with conventional therapies involve their predominantly symptomatic and palliative care approach for rare diseases, lacking curative solutions due to poorly understood pathologies. These therapies may also inadvertently lead to life-threatening and severe disabilities.

Unfortunately, the combination of these two elements can be challenging for conventional medicine, which requires early awareness and knowledge of the disease to achieve an effective recovery.1

Traditional medicine often lacks specific therapeutic indications for commercially available drugs with established dosages. This becomes particularly problematic for rare diseases, as they are frequently not included in drug trials. As a result, determining the effectiveness of conventional drugs for treating specific rare diseases becomes challenging.1

Advantages of Nanomedicine in Treating Rare Diseases

Nanomedicine has spearheaded the progression and advancement of the treatment of rare diseases with a strategy that effectively overcomes the obstacle of the BBB.1

Drug design of effective agents can be manipulated with nanomedicine approaches, such as the use of nanocarriers, to allow these molecules to cross the BBB efficiently.1

Nanocarriers are perfect candidates for treating rare neurological diseases due to their beneficial characteristics, including versatility, ability to display tridimensional shapes, and suitable dimensions that favor translocation across the BBB.1

Other characteristics of nanoparticles and nanomedicine include surface modification, whereby their surface properties can be altered. This includes tuning the surface charge or adding ligands, which enable effective interactions with their target cells for a positive result.1,2

Interestingly, nanoparticles and nanocarriers can also increase sustained drug release, a critical feature for effective treatment, as it ensures that the drug is available for longer.1,2

With a lack of relevant disease models available for less researched rare diseases, researchers are concerned about the path to enhance in vivo predictive validity and pre-clinical trial efficiency.

However, with nanotherapeutic tools, this may be overcome as they can be used to assess whether the integrity of the BBB is retained before, during, and after administration, which would be significant when treating rare CNS disorders.1

Case Studies: Successful Applications of Nanomedicine in Rare Diseases

Approval of nanomedicine products for various applications and indications has been slow compared to non-nanomedicine products. However, they are growing due to a high volume being in development.1

The Food and Drug Administration (FDA) approved Elzonris in 2018 as the first treatment for plasmacytoid dendritic cell neoplasm (BPDCN)—a rare blood disorder—in adult and pediatric patients.3,4

The approval of this drug was based on an open-label, multicenter, multicohort, single-arm clinical trial, in which seven out of thirteen patients in a pivotal cohort with untreated or refractory/relapsed BPDCN showed a complete response after a median follow-up of 11.5 months.4

Future Directions and Potential Impact of Nanomedicine for Rare Diseases

The nanopharmaceuticals market, comprising nanomedicine development, has spread throughout the globe due to providing added value in comparison to conventional pharmaceuticals.1,3

With varying definitions of nanomedicine in different countries, it can be challenging to estimate the global market size for these products. It has been reported that the global nanomedicine market reached US$ 263.9 billion in 2023, with a market forecast of US$ 719.5 billion in 2032 and a compound annual growth rate (CAGR) of 11.4 % during 2024–2032.5

The development of common clinical formulations within nanomedicine seems to be aimed at reducing toxicity rather than improving the efficacy of agents.

Additionally, with the potential to cross the BBB (unlike conventional drug design) and its high targetability, nanomedicine has the potential to revolutionize the treatment landscape. This is true for treating both common and rare diseases related to the brain, as well as more holistic conditions such as blood diseases.1

More from AZoNano: Harnessing Nanomedicine to Fight Infectious Disease

References and Further Reading

  1. Faouzi, A., Roullin, VG. (2021). Think big, start small: How nanomedicine could alleviate the burden of rare CNS diseases. Pharmaceuticals. 2021. doi.org/10.3390/ph14020109
  2. Farjadian, F., Ghasemi, A., Gohari, O., Roointan, A., Karimi, M., Hamblin, MR. (2019). Nanopharmaceuticals and nanomedicines currently on the market: Challenges and opportunities. Nanomedicine. doi.org/10.2217/nnm-2018-0120
  3. Zhang, N., Wei, M-Y., Ma, Q. (2019). Nanomedicines: A potential treatment for blood disorder diseases. Frontiers in Bioengineering and Biotechnology. doi.org/10.3389/fbioe.2019.00369
  4. Center for Drug Evaluation and Research. (2018). FDA approves tagraxofusp-erzs for blastic plasmacytoid dendritic cell neoplasm. [Online] U.S. Food and Drug Administration. Available at: https://www.fda.gov/drugs/fda-approves-tagraxofusp-erzs-blastic-plasmacytoid-dendritic-cell-neoplasm#:~:text=On%20December%2021%2C%202018%2C%20the%20Food%20and%20Drug,and%20in%20pediatric%20patients%202%20years%20and%20older. (Accessed March 24, 2024)
  5. Impactful Insights. (2024). Nanomedicine market size, share, growth, report 2024-32. Size, Share, Growth, Report 2024-32. [Online] Impactful Insights. Available at: https://www.imarcgroup.com/nanomedicine-market#:~:text=The%20global%20nanomedicine%20market%20size%20reached%20US%24%20263.9,US%24%20719.5%20Billion%2C%20CAGR%20of%2011.4%25%20during%202024-2032. (Accessed March 24, 2024)

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Marzia Khan

Written by

Marzia Khan

Marzia Khan is a lover of scientific research and innovation. She immerses herself in literature and novel therapeutics which she does through her position on the Royal Free Ethical Review Board. Marzia has a MSc in Nanotechnology and Regenerative Medicine as well as a BSc in Biomedical Sciences. She is currently working in the NHS and is engaging in a scientific innovation program.


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