Work-in-progress: Bio-therapies & the Future of Medicine
Wednesday, December 7, 2011 at 7:15PM
Gordon Laird

Occasionally, I write books. My current project began as an investigation into stem cell research, but has expanded to all emerging biological therapies -- including immunotherapy, oncolytic viruses, genomics -- as well as policy and society considerations in medical tourism, drug research and commercialization, ethics, and health policy.

Tentatively entitled The Universe Inside, this book is an up-close look at this historic movement in science and medicine. Why medsci? My books are an ongoing (sometimes accidental) study of disruptive change and the impact of innovation, both positive and negative. More directly, I am active in the medical world as a board member at Canada's first preschool for immune-compromised children facing cancer, solid organ transplant, bone marrow transplant, and blood disorder. Having already written about various kinds of change in our physical world -- climactic, economic, cultural -- it's exciting and compelling to investigate the inner world of the human organism, where existing medical options and scientific knowledge are always framed by greater questions and mysteries.

This is how the story goes:

At first, it seemed like an impossible goal. Since the early 1900s, medical researchers and clinicians have experimented with chemotherapy as a treatment for cancer. And for much of the 20th century, survival rates were negligible, and a diagnosis of cancer usually meant inevitable death, something that affected at least one in five people, as it still does today. Treatments with mustard gas derivatives and arsenic sometimes gave reprieve to early cancer patients, and newer drugs targeting fast-growing cells increasingly achieved temporary cures without severe toxic effects. Yet it wouldn’t be until the 1960s that patients could actually hope to be cured of common blood cancers like leukemia, where childhood treatments now approach 80 per cent success. Overall, survival rates improved incrementally for many cancers and blood disorders as patients often lived many more months or years in remission. Yet amazingly, early drugs like methotrexate and vincristine remain the cornerstone of many cancer treatments. While targeted therapies and combination drugs have improved cancer outcomes, the basic fact is that basic treatment modalities have not changed for decades. And with the exception of childhood leukemia, many cancers remain hard to treat and cure within exisiting treatment paradigms, leading some patients, researchers and clinicians to question the efficacy of cytotoxic (cell-killing) chemotherapy as the standard response to much of the developed world's critical illness.  

Yet at the same time that medicine was exploring chemotherapy in the 1950s, another paradigm emerged. Researchers discovered that fatal doses of radiation could eliminate certain kinds of disease and, more importantly, the patient could be at least temporarily saved by infusing their body with hematopoietic (blood) stem cells derived from bone marrow. A new person could be engineered, in essence, by grafting a healthy immune system into a diseased body. And despite many early failures, stem cell transplant eventually began to result in a small but growing number of durable cures for relapsed and other hard-to-treat cancers. 

Truth is, if humans are ever saved by a cloned heart, kidney or lung, or have their immune system retrained to fight cancer, it will because of stem cell research and stem cell transplantation that began in the 1940s and 1950s. Transplant pioneers like Donnall Thomas or stem cell researchers McCulloch and Till helped to discover a new frontier, the realization that human tissue or other biological materials could themselves become the durable cure for otherwise incurable disease. 

In scientific and medical terms, it is a potentially huge paradigm shift. For example: if chemotherapy for cancer is cure by poison – chemical war on unwanted, uncontrolled cells that emerged from the 1950s – then stem cell transplant (via bone marrow or cord blood) is cure by transformation, grafting DNA onto DNA, a high-impact treatment for hard-to-treat cancers that gained broader clinical acceptance in the 1970s. “The metamorphosis [of transplant] is not yet fifty years old,” writes medical historian Julie M. Fenster. “It is possible that people still don’t fully understand what transplantation means, not even those who have worked within the field for decades.” 

Consequently, today's world of biological medicine reads a lot like science fiction. On numerous fronts, we are moving to adopt the unique power of the human body, viruses, engineered tissue to multiply, differentiate, illuminate, and, potentially, regenerate. Stem cells, the immune system and non-traditional mediums play a significant role in much of today's leading research: already, we can create blood from skin cells, and culture 3-D lung tissue, kill leukemia (MLL) cancer stem cellsdestroy human cancers with viruses, and even model mammal cells after salamander genes to encourage regeneration of lost and damaged limbs. Since the 1990s, cell-based or biological therapies have been investigated as novel treatments for heart disease, cancer, auto-immune diseases, multiple sclerosis, autism, diabetes, spinal and brain injury (including stroke), regeneration of injured organs, rheumatoid arthritis, deafness, blindness – even baldness. "What differentiates this new generation of therapeutics is that investigators are no longer simply processing cells or tissues, but rather designing, engineering and manufacturing cell-based products," writes Toronto biomedical engineer Peter Zandstra

Biological therapies are potentially quite different than what you might find at your local hospital. Stem cells, for example, have been hypothesized as the master source, or cellular engine, of all complex life forms on earth. To the degree that we actually understand them, stem cells can transform into nearly any kind of tissue (depending on stem cell type), modulate immune response, hormones, and inflammation, as well as be replicated on a large scale, either for human therapy or study of biology and disease. They are present in the embryo onward, and through various mechanisms, help create tissue for every aspect of our body, and repair and renew organ systems essential to continued life. Stem cells are also thought to play a leading role in cancer, and play a critical role in our understanding of disease in general, and the ongoing mapping of the human genome. In other words, employing stem cells, viruses or even the patient's native immune system is an approach to treatment of disease quite unlike nearly any drug or medical intervention ever devised by humans. 

There is much that we still don't understand about using tissues and cells to cure people; and frequently high rates of mortality within existing transplant medicine reflect this experimental aspect. While most of the risks of established transplant medicine are well-documented, newer bio-therapies pose unique and potentially serious side effects, such as tumorous growths resulting from stem cell injections (documented in patients treated in offshore clinics in Russia and Thailand). Artificially induced stem cells, pluripotent cells developed without human embryos, offer new ways to customize and manufacture cells, but also create new challenges, since severely reprogrammed cells do not always behave as intended. “The worry is that reprogramming might shove cells so far from what is physiologically normal that they become pathological,” reported Nature in April 2009. Perhaps one of the greatest threats is that of public expectation: since the 1970s, patients have been promised victory in the war on cancer, yet with many common cancers we remain decades away from significant, lasting cures. 

From yesterday's pioneering stem cell transplant doctors to tomorrow's human organs cultivated in bio-reactors, cell-based and transplant medicine is a bridge, an experimental mode of treatment that links past, present and future. My work is at one level a genealogy of transplant as the next major (biological) movement in medicine: exploring early efforts in stem cell transplant and linking this early work and the complexities and promise of current treatment to future-looking efforts to translate cell-based therapies into durable cures. Through profiles, documentary and investigative reporting, this project will explore the complexities and possibilities of the human immune system, the strengths and weaknesses of existing medical treatments and drug options, the mysteries of life at the cellular level, and, despite everything, the irrepressible aspect of hope that is part of every medical experience. In other words, the book is a personal yet big picture attempt to explore the current trajectory of science and medicine, as it pertains to some our most serious diseases.

Today's research points to the strong likelihood of biological treatments playing a significant role in the future of medicine. Yet lack of public awareness and current public policy suggest that we may not be ready for miracle cures, even if they arrived tomorrow. One thing is certain: the history, current reality, and future of our own cells are not well enough understood. 

Keep posted in the coming months for new work on this topic. 

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