You know what they say. If it isn't broken, don't try to fix it! Somehow, us humans always tend to break things. Luckily, the technology that helps us heal is advancing every day.
After focusing on smart cities and precision agriculture, it’s time to focus on ourselves, and this preview of Deep Tech Trends addresses personalized medicine.
This year, based on the over 5,000 applications we received worldwide for our Global Startup Challenge and our years of experience in monitoring, shaping, and developing the deep tech ecosystem, we can say we are in a unique position to predict how the future will shape up. In the continuation of our article, you can find some examples of carefully selected Deep Tech Pioneers from the 2019-2020 Global Challenges.
Keep reading to learn more about what we predict!
Everyone’s biology is unique. Now, let’s examine targeted therapies supported by high probabilities!
The complete genetic code of the human chromosome was first revealed in 1999 as part of the Human Genome Project. At that time, many expected to see a complete transformation in therapeutic medicine within 20 years. [1] This did not happen for one main reason: the initial hopes for personalized medical treatments based on everyone’s genome were based on a baseless assumption that each disease is directly tied to only a few genetic factors. It is true that some serious diseases are caused by an individual mutation; Huntington's disease is one of them. However, most debilitating diseases result from combinations of mutations; some consist of hundreds of genes, each of which has a small effect that accumulates over a lifetime. Thus, the dream of using gene therapy to treat diseases, that is, replacing a mutated gene with a normal copy, would only be feasible for a small number of disorders.
“Correcting” the mutated gene for these diseases can be done using engineered enzymes like the well-known CRISPR/Cas or through virus-mediated gene transfer. While cutting-edge technology is much more advanced now, it has already received approval to treat certain single-gene mutation-based diseases. Beyond the high costs of these types of treatments, the technology is still limited by two factors: the patient’s immune response to the viral vector and the inefficient gene transfer from the virus to the target cells. Chameleon’s vector offers immune inhibitor molecules that reduce the immune response against the viral vector, overcoming one of the two major limitations related to neutralizing antibodies that are naturally present in many individuals.
Despite these advances in the used advanced technology, these developments raise many ethical questions. Human germline engineering, which refers to altering a trait that can be passed on to offspring, has profound implications that are difficult for us to comprehend. To prevent unethical use of these technologies, a societal consensus on germline genome editing will be necessary.
Genomic profiling will help clinicians select the best care for you
While it may not completely transform medicine, the Human Genome Project still has a significant impact on many studies and, most importantly, has facilitated the development of rapid, accurate, and inexpensive DNA sequencing and manipulation. For example, the low costs of DNA sequencing have enabled clinicians to sequence the genome of tumor tissue, thus allowing them to build large genomic databases. These databases are increasingly used to establish a relationship between a person's unique genetic profile and their susceptibility to specific diseases and treatments. To understand the relationship between genetic factors and the development of a disease, scientists and companies are increasingly developing machine learning-based solutions based on its progression and response to current treatments. For instance, Cambridge Cancer Genomics and Oncohost match the genome of newly diagnosed cancer patients with clinical trials and FDA-approved treatments that have been shown to work best for specific genomic molecules.
Similarly, the Nalagenetics algorithm prevents adverse drug reactions through pharmacogenomic tests. Bilhi Genetics is developing algorithms that can predict the genetic risk of developing hepatocellular carcinoma after being infected with the Hepatitis C virus. It takes into account 29 genetic markers along with non-genetic factors such as gender or alcohol consumption. Although it has not yet defined the causal relationship between genomic profiling and disease, these algorithms will help doctors select the best available treatment based on probability charts indicating a patient's susceptibility to a specific genetic treatment.
The great potential of cell therapies
In the meantime, we are also witnessing incredible advancements in cell therapies. Instead of correcting a single mutated gene, cell therapy aims to replace faulty cells, organs, or specific cell types by transferring live cells. Cells can be genetically engineered and sourced from the patient (autologous cells) or from a donor (allogeneic cells). The most common and successful cell therapy used to date involves the treatment of certain forms of leukemia with engineered T cells. T cells are normally a crucial part of everyone’s immune system that fights viral and bacterial infections. However, instead, the designed T cells attack leukemia.
Similarly, Cutiss designs autologous dermo-epidermal skin cells to treat large skin defects caused by burns or serious skin diseases. Once a healthy skin cell is identified, it is cultured and transformed into a pluripotent stem cell, creating a live skin graft that can produce many new skin cells to replace the defective skin.
The main challenges of this process are engineering the cells and automating the procedure. Both are being addressed by GC Therapeutics and TreeFrog Therapeutics, which respectively develop new stem cell differentiation and culture techniques. Overcoming critical issues related to manufacturing will enable millions of patients suffering from organ disorders, such as type 1 diabetes, to benefit from cell therapy. Others like Ternion Bioscience are developing stem cell technologies to better replicate the human body's response for testing drug toxicity. While cell therapies may one day fulfill the promise of personalized medications, they are still far from being automated and therefore widely applicable.
More targeted therapies = Fewer side effects!
Nevertheless, even if they are not personalized, therapies are becoming more focused. Lumento Therapeutics aims to enhance existing cancer drugs using a photosensitive protective group that renders them inactive until activated by a specific wavelength of light. If a focused light source from outside the body marks the cancer tissue, the drug is activated locally, allowing for higher doses since the cancer drug remains inactive in other parts of the body. Similarly, RS Research is developing a network technology that will only allow cancer drugs to regain activity once within the targeted tumor cell. If combined with the technology of Nanology Labs, which utilizes manganese nanoparticles to specify the precise position of early-stage (brain) tumors using MRG, it could truly provide a solution. Theranostics refers to a combination of specific targeted therapies and diagnostic tests that will likely become a more common tool in medicine, paving the way for delivering personalized medicine.
[1] Medical and Societal Consequences of the Human Genome Project (1999), N Eng J Med 341:28-37, Collins FS
