Cell and gene therapies are fast emerging as one of the most prospective and keenly anticipated areas of biologic medicine. Their buzz can be attributed both to the immense promise that these technologies generate for patients afflicted with rare and often deadly inherited diseases, as well as the profound value additions for payers associated with what is being seen as a potentially ‘one-time only’ cure.
Part of the allure is no doubt linked to the way in which this modality of healthcare seeks to deliver highly personalized, precision treatment while simultaneously harnessing and mastering the body’s own defence systems to heal. “The gate is being opened to a brave new era which is unprecedented in the entire history of modern medicine. Cell and gene therapies herald a thrilling turning point in how we conceive of fighting off disease,” declares André Choulika, chairman & CEO of the French immune-oncology specialist, Cellectis.
The term ‘cell and gene’ actually encompasses multiple recent breakthrough innovations, though “the two treatments are commonly referred to together as one because they tend to operate best in conjunction,” explains Anne McDonald Pritchett, SVP Policy and Research at the Pharmaceutical Research and Manufacturers of America (PhRMA). “The most effective cell therapies generally entail removing cells from the body, treating them with gene therapy – such as inactivating the disease-causing gene or replacing it with a health copy – and then transferring the cells back into the patient again,” she elaborates.
Amid all the fanfare around this rapidly emergent field of medical science, however, “three specific developments stand out as especially promising and significant: namely the deployment of stem cell technologies in regenerative medicine, gene-editing tools such as the much-acclaimed CRISPR-Cas9 technique, and chimeric antigen receptor (CAR) T-cell approaches,” observes David H. Crean, managing director of the investment banking firm Objective Capital Partners.
Stem cells, which are special unassigned human cells that possess the ability to replicate and morph into many different cell types – from muscle cells to brain cells – are exciting because of their potential use in the reparation of damaged tissues and the shift of emphasis from ‘treatment’ to ‘healing.’
“The predominant paradigm in the pharmaceutical space has for a great many years been palliative care. Most of the time, healthcare has been fixated upon attacking the symptoms rather than grappling with the base mechanism of the disease, but this is about to change,” predicts Yuzo Toda, former chairman of the Forum for Innovative Regenerative Medicine (FIRM) in Japan. He believes that cellular science-centric approaches that leverage the power of stem cells augur a considerably more enlightened, less invasive, restorative pathway. “Future medicine can be regenerative and operate along very different lines to the norms that we have become so used to,” he insists.
CRISPR-Cas9, meanwhile, much akin to a pair of molecular scissors, is redrawing the boundaries of gene engineering as it allows, for the very first time, what David Crean describes as “an efficient and precise insertion, deletion, modification or replacement of a particular gene in the genome thus paving the way for preventative healthcare.”
“CRISPR’s game-changing innovation stems from its accuracy, cost-effectiveness, and sheer simplicity to use… All of a sudden the ability to precisely delete undesirable traits and, potentially, add desirable ones is becoming feasible,” agrees Jongmoon Kim, CEO of ToolGen, a South Korean outfit famed for having deployed the technology in livestock to create the world’s first muscle strengthening pig, with a view to increasing the leanness of the meat! Already scientists have successfully managed to edit down the risk of sickle-cell anaemia and genetic deafness in rodents raising hopes of similar future feats in its application for humans.
Chimeric antigen receptor T-cell therapies, for their part, raise the possibility of taming aggressive cancers by re-engineering the body’s immune system to proactively seek out and neutralise any abnormal and malignant cells. Increasingly described as the “fifth arm” in the war on cancer following on from the more conventional arsenal of surgery, radiotherapy, chemotherapy and targeted biologics, CAR-T has been credited with triggering complete remission in a significant proportion of terminally ill patients suffering from leukaemia and certain cancers of the lymphatic system.
“The way it actually works is that a subset of a patient’s white blood-cells, T-lymphocytes, are extracted from their blood and shipped to a sophisticated manufacturing site where experts alter their genome to equip them with a new receptor, the Chimeric Antigen Receptor or CAR, that they do not naturally possess. The resulting artificially engineered CAR T-cells are subsequently shipped back to the patient and administered by a single infusion from which they go on to attack the cancer over a period of many months,” explains Emanuele Ostuni, Head of Cell and Gene Therapy for Novartis Oncology in Europe.
Little wonder, then, that the international healthcare community seems so enthused by the unfolding opportunities. “Frankly, We’re entering the golden age of genomics, where genetic design is being supercharged by new understanding and technologies… it’s certainly a great time to be in healthcare,” exclaims Andrew Hessel, CEO of Humane Genomics.