UPCOMING MEDICAL REVOLUTION
The world is now at the threshold of a revolution in medical treatment in the form of cell and gene therapies which are fast emerging as new therapeutic modalities. Various developments surrounding the cell and gene therapies are being reported with scientists working on solving technical hurdles in the treatment, successes in using the therapies on different diseases, and companies establishing manufacturing plants.
A new study conducted by Stanford University researchers hopes to solve a major problem of gene therapy, an autoimmune attack. In an experiment on mouse they have shown that the gene therapy can be effective without causing a dangerous side effect of autoimmune reaction which the patient’s immune system is encountering for the first time. Several such hurdles in the gene therapy development are being eliminated with research and innovations.
There have been several reports in the last two years of the encouraging clinical results where the therapy was used in various diseases like haemophilia, sickle cell disease, multiple cancers, several neurodegenerative disorders and genetic diseases. A teenage boy in France was cured in March of a genetic disorder using experimental gene therapy. GSK’s gene therapy Strivelis is available since 2016 for a rare disease among the children. Spark Therapeutics’s Luxturna is approved for correcting genetic blindness. Three CAR-T cell therapies are already approved in US by the FDA to treat blood cancers. They include Novarit’s Kymriah, useful for rare blood cancer treatment, and Kite Pharma’s Yescarta.
Besides these, many more therapies are under clinical investigation. These developments are also reflecting in the stocks market. Sangamo Therapeutics’s shares increased by over 33 per cent previous month when it reported interim data for its haemophilia A therapy. As a result of these developments, MIT declared 2017 as the year of gene therapy breakthroughs.
Scientists feel that cell and gene therapy is fundamentally different from other regular therapies since instead of treating symptoms, it corrects the genetic cause of a disease. So far, these therapies have been introduced for rare diseases and hence the number of patients is small. But that will not be the case in the future. Successes in CART-cell therapies demonstrate the need for capacity expansion. Some developments are taking place on that front too.
Swiss biotech company Lonza opened its cell and gene therapy manufacturing plant in US in April. The 30,000 sq feet facility will be used for engineering viruses and gene therapy vectors since its shortages have created bottlenecks in development of the therapy. A month prior to that Sumitomo Dainippon Pharma completed its manufacturing plant for Regenerative Medicine and Cell Therapy, which has been built to produce cells for use in clinical trials.
Although various positive developments are being seen in cell & gene therapies, few challenges need to be addressed. Developing cell engineered products is one such challenge due to many aspects including production, delivery, regulatory and testing.
As the field is still young, one of the early challenges is the high price tag these therapies come with. uniQure’s Glybera, the first gene therapy, was withdrawn last year due to pricing issue. In order to overcome this hurdle some companies are offering full money back in case of failures or charging only those patients who are showing positive results in the first month. But it is a temporary solution that can only be adopted in the initial phases.
According to the experts, another hurdle related to the pricing is reimbursement of the treatment cost from the insurance company. Establishing health insurance reimbursement for a new category of products which do not fit the established healthcare delivery or insurance model is challenging and time consuming.
Once these obstacles and challenges are addressed, cell & gene therapies can become a common practice. It seems that a new revolution of medicine is around the corner.