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Discovery & Development Drug Discovery, Trends & Forecasts

The Clinical Cosmos

Vast and enigmatic, space has always been the focus of human obsession. It also offers a unique environment – a “hotspot”, if you will – for researchers. Space research not only helps us broaden our understanding of the cosmos, it lays the groundwork for a deeper examination of the challenges faced in Earth-based research. For example, Earth's gravitational forces impact cellular behaviors and interactions, making it challenging to study cells in their true three-dimensional context. There are many ways in which space could be a promising avenue for more accurate and accelerated research.

Microgravity: a new frontier for laboratories
 

In microgravity, cells experience minimal gravitational forces. They can grow in three-dimensional structures in a way that mimics their natural state within the human body,  facilitating more precise observations, particularly in understanding cellular interactions, growth dynamics, and treatment responses. A notable example is the growth of protein crystals, which were found to be larger and less flawed than those grown on Earth, offering deeper insights into their structure and functions. 

The absence of gravity brings about significant alterations in well-known physical phenomena. The settling of heavier particles – sedimentation – becomes negligible, as does convection, the movement of molecules driven by temperature differences. In contrast, diffusion, the movement of molecules from high to low concentration, becomes the dominant force in the microgravity environment of space. This shift has profound implications for cellular biochemistry. Cells adapted to Earth's gravitational pull may function differently, affecting processes such as nutrient absorption and waste expulsion. Recognizing these changes, the pharmaceutical industry is now exploring how space-based molecular interactions can pave the way for enhanced drug development on our planet, and new perspectives on areas such as drug interactions and stability. 

Off the top of my head, I can offer a couple of examples of organizations embracing off-planet research. First, Budapest-based space chemistry research corporation InnoStudio has conducted experiments on the International Space Station (ISS) to determine if microgravity could enhance the stability of remdesivir and broaden its applications by reducing the risk profile (1). The company is also looking at other novel APIs and how they behave in microgravity.  Second (and going back to protein crystallization in space), Merck, Sharp & Dohme has been investigating crystallization processes for biologics with a view to simplifying drug delivery (2). The study of proteins could help us to understand various health conditions  and prompt investigations into other pharmaceutical relevant areas, such as the effects of microgravity on our immune system. And back on Earth, a coordinated effort led by the University of Barcelona sought to simulate microgravity conditions using parabolic flights to discern the impact of this environment on our immune defenses (3). Preliminary findings suggest that microgravity might not significantly compromise our immune system – at least not during short exposures (4). This research not only has implications for future space missions, but also provides invaluable insights for space tourism and potential long-term human habitation in space. 

As we delve deeper into the effects of space on our physiology, the heart emerges as another critical area of study. NASA's experiments have revealed that microgravity can be beneficial for stem cells and their remarkable ability to develop into various specialized cell types. In this context, stem cells in space have shown to grow into a type of heart muscle cells known as cardiomyocytes – the cells responsible for the contraction of the heart, allowing it to pump blood effectively. One specific study, known as the MVP Cell-03 study (5), demonstrated that microgravity can boost the production of cardiomyocytes. These findings are not just academic; they have real-world implications. Such revelations could lead to innovative treatments for cardiac abnormalities, both those induced by spaceflight and those prevalent on Earth.

Similarly, space-based clinical research is shining a light on one of the most formidable health challenges we face here on Earth: cancer.

Every year, cancer claims 10 million lives, a number surpassing the total population of Switzerland. Could the unique conditions of microgravity provide fresh insights? A UK-based research initiative is exploring the three-dimensional growth and spread of cancer cells in the microgravity environment of the ISS (6). The weightlessness of space allows cancer cells to form tumor spheroids or organoids that closely resemble genuine growth patterns in the human body, providing researchers with a unique opportunity to examine cancer cell behavior, evolution, spread, and reactions to different treatments. In particular, this research team is interested in diffuse midline glioma, a devastating childhood cancer known for affecting critical areas of the brain and spinal cord, making it particularly challenging to treat. Children diagnosed with diffuse midline gliomas often die within a year after their initial diagnosis, and there are no effective treatments.

The insights gleaned from these space-based studies hold the potential to revolutionize our approach to cancer and other diseases.

The challenges in space
 

Conducting experiments in space requires intricate planning, coordination, and execution. The process of sending equipment and samples to and from is both time-consuming and expensive. In many cases, space-based research is inaccessible for smaller research entities. Moreover, the unique variables introduced by the microgravity environment can complicate the interpretation of experimental results. Other factors, such as increased radiation exposure, may also influence experimental outcomes, necessitating additional controls and considerations. Collaborations with private space companies can provide additional resources, expertise, and capabilities, but other solutions are also being developed.

Advanced robotics, for example, could eventually streamline the process of transporting and handling clinical samples, reduce human error, and increase efficiency. Robotic arms, such as the Canadarm2 (8) have already been instrumental in capturing cargo spacecraft and assisting with experiments aboard the ISS. Telemedicine and remote monitoring technologies can help manage physiological changes in astronauts, with wearable health monitors already being used to track astronauts' vitals in real-time and send data back to Earth for analysis (9). 

Advanced shielding technologies could also mitigate the effects of increased radiation exposure in space, providing a more controlled environment for these clinical research experiments. NASA's development of radiation shielding materials, for instance, can protect both equipment and astronauts from harmful cosmic rays. 

Beyond the technical and logistical challenges, another key question is: do we have a governance compass in place to balance research opportunities with the myriad of ethical, legal, and safety implications unique to the interstellar domain? As space emerges as a promising domain for clinical research, the imperative for a comprehensive governance framework becomes evident. The unforgiving nature of space demands rigorous safety protocols, so precautions must be in place to shield researchers and participants – and to handle emergencies.  

Data collection in space presents its own set of challenges, given the constraints on tools and technologies. How can we vouch for the accuracy and reliability of this data, and what measures are in place to ensure seamless data transmission to Earth? A comprehensive governance framework for space-based clinical research is imperative to address the multifaceted challenges this frontier presents. It should account for the unique physiological responses in microgravity, establishing guidelines that ensure research findings are both relevant and applicable across environments. Safety protocols must be rigorous. Collaborative efforts between nations, space agencies, private entities, and the scientific community will be crucial in establishing guidelines that promote clinical innovation while safeguarding the integrity of space and its explorers.

Space, once the final frontier, could be a pivotal platform for research and innovation in the future, but with the human body responding differently in space's microgravity, there can be questions about whether the findings are universally applicable or space-specific. Are we truly maximizing the cosmic potential to reshape the future of healthcare, or merely echoing terrestrial pursuits in the cosmic arena? There is potential for conducting research in space, and it is an exciting field, but we should proceed with caution and ensure the research is conducted to a high standard  – and with attention paid to ethics and responsibility. 

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  1. Innostudio, “Hungarians develop more effective COVID drugs in space” (2021). Available at: https://bit.ly/467cYC2 
  2. ISS National Laboratory, “Published results from crystallization experiments on the ISS could help Merck improve cancer drug delivery”, (2019). Available at: https://bit.ly/4670OJk 
  3. A Gorgori-González, et al, “Effects of rapid gravity load changes on immunophenotyping and leukocyte function of human peripheral blood after parabolic flight”, Acta Astronautica 210, 437 (2023). doi.org/10.1016/j.actaastro.2023.02.012 
  4. D Fornell, Cardiovascular Business, “Heart tissue heads to space for research on aging and impact of long spaceflights” (2023). Available at: https://bit.ly/45gFX5e 
  5. The Institute of Cancer Research, “Cancer cells set to be launched into space for microgravity experiment on the International Space Station” (2023). Available at: https://bit.ly/3PwUrb9  
  6. M Filbin, Dana-Farber Cancer Institute, “What does a diffuse midline glioma look like?” (2022). Available at: https://bit.ly/3RCA7I2 
  7. NASA, “Space Station Robotic Arms Have a Long Reach” (2019). Available at: https://bit.ly/46w5U1D Canadian Space Agency, “Bio-Monitor: Keeping an eye on astronauts' vital signs” (2021). Available at: https://bit.ly/46s5wRK 
  8. World Health Organization, Cardiovascular diseases fact sheet (date unknown). Available at: https://bit.ly/3t9FOTB  
  9. World Health Organization, Cancer fact sheet (2022). Available at: https://bit.ly/3t9G4C3 
About the Author
Deepika Khedekar

Deepika Khedekar is an Associate Centralized Clinical Trial Lead at IQVIA Inc, a global clinical research organization, where she spearheads clinical trial monitoring programs for major pharmaceutical companies. In her 12+ years in the pharmaceutical industry, she led phase I, II, and III clinical trial programs in the respiratory and gastrointestinal therapeutics, including drugs for leading US and Australia-based pharmaceutical organizations, such as Gilead Sciences, Macleods Pharma, Arrowhead Pharmaceuticals, NoNO Inc., EpimAb, and Impact Pharma. She started her journey in the field of pharmaceutical research at Pfizer and holds a Master’s degree in Pharmacy from the University of Mumbai.

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