BioTech
Gene Therapy: Unlocking Innovations in Cancer Treatment, Detection, and Drug Development
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The history of using genes as a tool to treat humans goes back nearly five decades, marking a pivotal moment in medical science.
In 1972, the concept of gene therapy was first considered as a form of treatment. This groundbreaking idea was introduced in a paper titled “Gene therapy for human genetic disease?” published in the journal Science and written by T Friedman and R Roblin.
In that paper, the writers claimed that “gene therapy may ameliorate some human genetic diseases in the future.” They called for continued research “directed at the development of techniques for gene therapy.” This call for research marked the beginning of a new era in genetic medicine.
Indeed, it took another 18 years for the first gene therapy trial to happen on humans in 1990. This trial was a significant step forward, paving the way for further advancements. In 2003, another major milestone was achieved with the completion of the Human Genome Project.
Launched in October 1990 and completed in April 2003, the Human Genome project is considered one of the greatest scientific achievements in human history for generating the first sequence of the human genome.
Following this path of scientific progress, another milestone was reached when China approved the first Gene Therapy for treating head and neck cancers.
Since this development marks the beginning of the line of treatment possibilities we discuss here, let us look at this in greater detail.
The First Approved Gene Therapy Product for Cancer
In 2003, the China Food and Drug Administration (CFDA) approved Gendicine, a recombinant human p53 adenovirus, developed by Shenzhen SiBino GeneTech Co. Ltd. It came into the commercial market as a gene therapy product to treat head and neck cancer. The therapy is designed for delivery via minimally invasive intratumoral injection as well as by intracavity or intravascular infusion.
A scientific review that looked into the therapy's performance for the first 12 years found the company to have manufactured 41 batches of Gendicine and 169,571 vials. Impressively, the therapy could be delivered without any serious advances being noted, except for a vector-associated transient fever in 50-60% of patients that persisted for a few hours only.
Subsequent Gene Therapy Trials
After China's successful application of the Gene Therapy trial, the advancements continued. In 2005, the first gene therapy trial in hemophilia B started using AAV Vector technology.
AAV vectors serve as bioengineered tools to transport and deliver genetic material safely to affected tissue and cells using a non-enveloped virus. Then, in 2017, the United States approved the first gene therapy for a genetic disease known to have caused blindness.
With further scientific advancements to be made in this field, it is obvious that gene-based treatments will open up new horizons for treating malignancies as severe as those caused by different forms of cancer.
But before delving deeper into this space, it is vital to know what different gene-based therapy-related terms mean, which include genetic engineering, genome, genomics, and genome sequencing.
What is Genetic Engineering?
The National Human Genome Research Institute defines genetic engineering as a process that “uses laboratory-based technologies to alter the DNA makeup of an organism.”
Altering the DNA makeup may happen in different ways, encompassing a range of techniques. It may change a single base pair, choose to delete an entire region of DNA to replace it with a new segment, or even work between species where a gene from one species is introduced into an organism of a different species. This versatility of genetic engineering has made it a vital tool in the hands of cancer therapy solution providers.
What is Genomics?
Genomics is the study of genes and focuses on understanding how all of a person's genes interact with each other and with the person's environment. This field involves an in-depth study of various components, including DNA, cells, histones, nucleosomes, and chromosomes. The term ‘genomics' is derived from ‘Genome,' which refers to an organism's complete set of DNA. Every cell in the human body is noteworthy for containing a full-set copy of nearly 3 billion DNA base pairs.
Click here to learn all about investing in genomics and CRISPR.
What is Genome Sequencing?
Genomes are genetic codes that help distinguish one organism from the other. Like each human has different fingerprints, all organisms have a unique genetic code. Therefore, genome sequencing helps recognize the unique identification patterns for each distinct organism, be it a bacteria, vegetable, or mammal.
Four main components add up to make genome sequencing work as a wholesome process:
- DNA shearing
- DNA barcoding
- DNA sequencing
- Analyzing the data
With all these gene examination tools developed over the years, the scientific and medical community has achieved much success in treating life-threatening conditions, including cancer. In the next segments, we will see how gene-based maneuvering is leading us in our cancer detection and treatment efforts.
The Menace of Cancer: The Emperor of All Maladies
Indian-born American physician and oncologist Siddhartha Mukherjee aptly termed cancer as the emperor of all maladies in his 2010 book, a description that remains poignant given the disease's impact. Echoing this sentiment, the American Cancer Society estimates close to two million new cancer cases and more than 600,000 cancer deaths were experienced by the US alone in 2022.
According to another 2020 study, Lung cancer was the leading cause of cancer death globally, with an estimated 1.8 million deaths (18%), followed by colorectal (9.4%), liver (8.3%), stomach (7.7%), and female breast (6.9%) cancers.
On a more concerning note, the study anticipated a 47% rise in the global cancer burden compared to the 2020 levels. It projected 28.4 million cases in 2040, more than 9 million than the 19.3 million cases observed in 2020.
The numbers paint a grave picture, and it is essential to strengthen innovative efforts in detecting cancer early so that physicians and doctors get enough time to address their root causes and cure them. Promisingly, multiple gene-based efforts have yielded positive results in that direction.
Companies Leveraging Genome Sequencing for Cancer Detection and Treatment
1. SeekInCare
SeekInCare, a solution from SeekIn Inc., is a blood-based multi-cancer detection test capable of detecting more than twenty cancer types. This advanced solution leverages whole-genome sequencing from cell-free DNA, a technology central to its diagnostic capability.
As defined by the Centers for Disease Control and Prevention, whole genome sequencing is a laboratory procedure to determine the order of bases in the genome of an organism. Nucleotide bases are what constitute the unique genetics of an organism.
The researchers surveyed the efficacy of the test on 617 patients with cancer, achieving notable results. They were able to identify 404 of them, with a sensitivity rate of 65.5% and a specificity of 97.9%. The results showed that the higher the cancer stage, the higher the test's sensitivity. While it was 46.9% for stage I cancer, this rate increased to 81.8% for stage IV cancer.
In a prospective real-world study carried out among 1203 patients, the test offered a sensitivity of 60.0% and specificity of 96.1%.
Founded in early 2018, SeekIn Inc. is a biotech company that focuses on the early detection of blood-based pan-cancer. It believes that early detection of cancer can lead to a reduced mortality rate by a margin of 15%.
Apart from SeekInCare, it also has solutions like SeekInCure and SeekIn Clarity. SeekInCure monitors cancer recurrence and evaluates the risk for post-surgery cancer patients. And to fulfill its objective, the solution relies on monitoring the variation of the cancer genome in the cell-free DNA of the patients.
SeekInClarity helps in treatment response monitoring. In achieving what it set out to, SeekInClarity maps the panoramic cancer genome using shallow whole-genome sequencing (sWGS) data.
Headquartered in China, SeekIn is a privately held company and received angel funding from Green Pine Capital Partners and one more investor in 2018. The funding amount has been undisclosed.
2. Editas Medicines
Editas Medicine leverages gene editing to develop medicines for cancer and other serious diseases. Its SLEEK gene editing methods help develop the next generation of cell therapy medicines for cancer. Editas has its proprietary engineered AsCas12a nuclease, helping it deliver high efficiency, multi-transgene knock-in of induced pluripotent stem cells (iPSCs), T cells, and natural killer (NK) cells.
Editas Medicine, Inc. (EDIT +0%)
Recently, Editas published its Q3 2023 results, providing insight into the company's financial performance during this period. For the three months that ended on September 30, 2023, the company recorded more than US$5 million in collaboration and other research and development revenues. Additionally, for the nine months that ended on September 30, 2023, the revenue stood at more than US$18 million.
3. Beam Therapeutics
In December 2022, the US Food and Drug Administration (FDA) lifted the clinical hold and cleared the Investigational New Drug (IND) application for BEAM-201 to treat relapsed/refractory T-cell acute lymphoblastic leukemia (T-ALL)/T-cell lymphoblastic lymphoma (T-LL). Beam-201 was an anti-CD7, multiplex-edited, allogeneic chimeric antigen receptor T-cell development candidate from Beam Therapeutics, known for developing precision genetic medicines through base editing.
Beam Therapeutics Inc. (BEAM +3.87%)
For the three months ending on September 30, 2023, Beam Therapeutics Inc. registered license and collaboration revenue of more than US$17 million. For the nine months that ended on September 30, 2023, the company's revenue was more than US$61 million, a substantial increase from nearly US$41 million earned during the first nine months of 2022.
Apart from well-known public companies like Beam and Editas carrying out genetically engineered solution tests for cancer, scholarly and organizational research is also underway. Many of them promise to open up exciting opportunities for substantially curing or reducing the menace of cancer in the future.
An Early Detection Method for Pancreatic Cancer
A team of researchers from the United States, China, South Korea, and Japan have developed and tested a biomarker panel capable of detecting small amounts of RNA genetic material that goes off from the pancreatic cancer cells to circulate in the bloodstream. These are called circularRNA or circRNA.
The researchers have leveraged genome-wide expression profiling methods to carry out a comprehensive review of patient samples, leading to pancreatic cancer biomarkers discovery, based on circRNA. It would help distinguish pancreatic cancer tumors from normal tissue specimens in the bodies of early-stage pancreatic cancer patients. According to the study, the test “exhibited robust diagnostic accuracy.”
Erkut Borazanci, MD, research director of the Cancer Research Division at HonorHealth Research Institute, elaborated on the significance of the method further. He said:
“When pancreatic cancer is caught early, the chance of survival is so much greater than when diagnosed in the advanced stage. Therefore, there is a great need to develop novel ways to detect pancreatic cancer.”
This method is the right step towards developing such a novel method.
Gene-based Discoveries and the Future of Cancer Treatment
Undoubtedly, genes are at the core of our physiological existence, underscoring the complexity and importance of genetic study. Working with genes, therefore, requires sophisticated methods. But, when done carefully, approaches like genetic engineering, genome sequencing, and other gene-based therapies have the potential to free us from the risk of cancer to a significant extent.
However, it is not only about cure, as it also helps detect many cancers early. And early detection is often what helps eradicate cancer from its roots.
For gene-therapy-driven treatments to become more successful in the long run, it would have to address some challenges. It would have to look after challenges relating to samples, challenges specific to next-generation sequencing, and the sequencing of a nonmalignant comparator. Computational analyses and variant interpretation may also pose roadblocks.
However, all these challenges can be addressed efficiently with innovative research and collaborative efforts between research institutes, physicians and oncologists, healthcare centers, and drug manufacturers. The treatment must not affect the non-cancerous regions and should not introduce physiological changes that could prove harmful in the long run, even if the cancer is cured in the short term.
In the days to come, we can expect to see more R&D funds flooding in the area of gene-based therapies for cancer, with pharmaceutical industries globally expected to invest more. This is crucial as the scope is significantly large, and we need scalable and safe solutions.
Click here for the list of five best genome sequencing companies.
