Innovation in Medicine : New Ideas for Translation

One of the greatest accomplishments of mankind in this century is the tremendous improvement in health status cutting across social strata and geographical boundaries. Of particular note, is the general health of people in developing countries which has taken a turn for the better. The last half century has witnessed innovations in medicine and improvements in healthcare, plunging morbidity and mortality rates to an all-time low.

Today, the average life expectancy in these countries, too, has surged from a lowly 40 years to a lofty 65 years. These dramatic results are the outcome of improving health status among children under 5 years, who constitute a bulk of the population at risk and the aggressive efforts towards preventing and containing lifestyle diseases like diabetes, hypertension and stroke. It is unfair to attribute this epic success to economic growth and social improvements alone.

Innovation in medicine, including state-of-the-art healthcare technology, advanced medical research, key reforms in healthcare policy, comprehensive implementation of public health programmes and effective propagation of health awareness, is seen as the main driver of health improvement, particularly in low-income countries. It is specific actions within these domains that have led to tremendous strides in the global health observed over the past few decades.

Are We Playing God?

Today, the world of medicine is rife with examples of scientific discovery and technological advances that change our lives for the better. Not long ago, people died of preventable and curable diseases due to the lack of basic amenities and life-saving medicines. With relatively easy access to quality healthcare, however, the problem of plenty has now started to gnaw at our feisty lives even as most people live beyond 65 years despite a sedentary lifestyle.

The recent innovations in medicine, however, amaze even the sharpest minds, let alone the mundane lives of millions across the Indian subcontinent who benefit from it. This is particularly true when it comes to innovations in molecular biology and genetic engineering. With the profiling of the entire human genome, scientists are now capable of detecting various genetic defects wreaking havoc on the human body and even modifying it at the genetic level to such an extent that it performs a reparative function, thus providing newer horizons for therapy.

Such gargantuan developments are just a step closer towards “playing God” and opening the Pandora’s box of ethical and moral dilemmas that have also arisen alongside. Nevertheless, these quantum leaps were unimaginable just a few decades ago, and the sudden surge of knowledge was definitely propelled by parallel innovations in the field of bioengineering. Molecular genetics is all set to lead the way forward towards newer frontiers in medicine, so long as this new knowledge is not lost in translation. 

From Concept to Destiny – A Tedious Journey Unravels

The onus to convert raw science to practicable health solutions helping millions of people across the globe lies not only on a handful of academicians, researchers and innovators but on the political will of governments, relentless efforts of various government and non-government agencies towards good health service delivery, adequate funding, robust industry inputs, a trained workforce, and a collective will of the people.

The process of translation is often tedious and uncoordinated, taking about 25 to 30 years from initial scientific discovery to delivery of a therapy to its ultimate beneficiary, the patient. The onus of translating a basic discovery by researchers into a viable, usable end-product often lies on the medical products industry, with huge investment in terms of funding and resources, taking the putative therapeutic product through various predetermined phases of preclinical and clinical trials, approval from regulatory bodies, and ultimately releasing it for use by patients through the healthcare system. Often, the putative therapy fails to move through the stages of ethical clinical research and is prematurely withdrawn due to non-feasibility or potential harm to patients. The “novel” therapy, once approved for clinical use, is initially sold at a premium until its patent protection expires, and is then released for wider and lower-priced distribution by multiple manufacturers for benefit of the entire community.

Thus, the giant strides in the field of medicine cannot be undermined by silly criticism of a few failures that cost millions and much more in resources, when a single successful launch of a “novel” therapy can benefit, even save, millions of lives. While a significant contribution to a healthy and ageing society is attributed to clean water, hygienic living conditions, better nutrition and reduced mortality from infectious diseases, a remarkable contribution is made by translation of innovations directed towards prevention and treatment of various diseases.

Health Nirvana – Are We There Yet?

The present state of innovation in medicine is almost beyond reproach and enviable when compared to the sad state of affairs in the pre-antibiotic era. But, the quantum of research and innovation is still at a primordial stage and needs the final push to translate into usable scientific discovery. This calls for in-depth research, significant funding and efficient distribution of resources to answer basic research questions in the most amicable way. This requires the proactive and holistic intervention of various stakeholders in the healthcare industry. 

Over the recent years, there is a paradigm shift in the management of diseases – from treatment and palliation to prevention. While cardiovascular diseases were often treated surgically in the past, nowadays even serious cardiac ailments are being tackled conservatively without much difference in clinical outcome and quality of life. Besides, efficient dissemination of health awareness among the masses through various media channels has led to favourable outcomes of lower morbidity and mortality. Innovation in early diagnostics and its translation into patient-centric devices has yielded satisfying, if not staggering results.

Despite the tremendous rate at which biomedical science is advancing, translation of scientific breakthroughs into better health has failed to keep pace. Non-technological issues like impecunious funding, impractical regulatory norms, and socio-cultural barriers also contribute to this lag. The increasing disconnect between rewards to healthcare researchers and innovators, and the actual needs of the society is another broken cog in the wheel of scientific discovery. “Health Nirvana” is thus a far cry, but it remains the ultimate goal.

The Scope of Innovation and Translation in Remedial Science

Scientific advancements in healthcare have long since relied on academic centres for basic research and on for-profit industry players for diffusion of technology. This has proven difficult to translate, particularly at junctures between discovery and commercialisation, and between commercialisation and public health.

However, new ideas for translation keep cropping up as long as basic research and innovation keep spawning across the globe. Newer state-of-the-art diagnostics and novel therapies are being developed and pushed into the market at an alarming rate with most of them being outdated or outwitted by a newer model or better therapeutic modality. The potential scope for innovation is often limited by lack of resources but the inherent possibility for translation of an already established innovation is immense and yet feasible.

New Ideas for Translation

Translation yields result from the foundations laid by basic scientific research or innovation. New ideas often arise from established ideas which are already tried and tested. The same principle applies to the development and discovery of new molecules/drugs from a widely used parent molecule/drug, with similar characteristics as a parent molecule/drug without the unwanted effects. The potential use of modern technology in preserving or enhancing our health is enormous. Following is just a sample of new ideas for translation that would help in enhancing human health:

1. Enhancing the potential of telemedicine: While telemedicine is not new, newer applications and innovations in its implementation has the potential for a larger impact on human health, particularly for remote and inaccessible areas where access to tertiary care is difficult, if not impossible.

Expert opinion through telemedicine has saved millions of lives across the globe, particularly in the management of heart diseases. Exceptional results to prompt treatment instituted based on remote consultation has spawned newer opportunities for speciality care like e-ROP (Evaluating Acute-phase ROP) to identify babies at greater risk of developing severe retinopathy of prematurity in underserved or remote areas potentially saving millions of premature babies from imminent blindness. This success may be translated to the early management of acute cerebrovascular events, remote radiological and pathological diagnoses, and acute psychiatric disorders.

2. Genomic interventions: There are 3 ways in which genetic research can be used to promote and to recover health, namely, using tests to identify genetic diagnosis or to identify genetic risk, application of gene-expression panels and other kinds of genetic technology that will enable improved disease classification, and innovative therapies.

Obstetric screening for Trisomy 21 is being increasingly used for early detection and treatment. Screening for carrier-status is another area where genetic testing is helping patients with Tay-Sachs disease, haemoglobinopathies, and cystic fibrosis with early diagnosis, genetic manipulations, or even a successful cure.

Gene therapy is working miracles with breast cancer and chronic myeloid leukaemia (CML) patients worldwide. Although such success is difficult to fathom in other cancers due to multiple loci of genetic defects, there are various trials in progress evaluating gene therapy for lung, prostate gland, gastrointestinal and brain cancers.

3. Robotic technology: Bioinspired soft robotics is the next step in wearable robotic devices that seamlessly integrate with the functioning of the human body to assist in normalising gait and sports activities. One such example is the soft exosuit for gait management in patients with a cerebrovascular accident. These are lightweight and flexible exosuits with integrated artificial muscles and refined robotic controls. The applications of this new technology can be extrapolated to assist children with cerebral palsy, adults with movement disorders such as stroke, Parkinson’s disease, elderly patients to maintain gait and among professionals requiring them to carry extremely heavy loads.

Robotic surgery, which has been tried with variable results, can be explored in areas requiring microsurgical precision like eye surgery, neurosurgery, and organ transplants.

4. Cell therapy: Cultured stem cells injected into the human body at various sites have the potential to replenish dead and degenerated cells and thus restore function in organs or structures that normally do not regenerate. One recent study that stands out is the injection of cultured corneal endothelial cells to replenish endothelial cells in cases of complete endothelial loss due to pseudophakic bullous keratopathy. The results are promising.

Similar cell therapies have been tried in various haematological cancers with variable success. The potential for cell therapy in dementia, heart failure, pancreatic cancer, diabetes and other diseases is looking bright. The momentum achieved in this field should be potentiated by further research into the genetic components of such cell therapy.

6. Big data analytics in healthcare: The role of big data in predicting lifestyle diseases is well-documented but its application in real life is abysmal considering the immense potential. There is much scope for using big data analytics in predicting and preventing life-threatening diseases like cerebrovascular accidents, evaluating the evolution of disease processes and analysing outcomes of various therapeutic measures. Big data can potentially explicate the natural history of diseases without ethical issues to boot.

7. 3-D tissue printing: There is immense scope for the futuristic concept of 3-D tissue printing, as various organs may be replicated for transplant without the need for living or cadaveric donors. Although in its primordial stage of development, it envisions hope for the future.  

Newer Horizons – The Road Ahead

The uncharted waters of potential discovery keep growing indefinitely as technological advances keep burgeoning. But, where does one draw the line? Rationalised distribution of resources is key and the decision to translate innovation into a novel diagnostic or therapeutic modality should be guided by an overall sense of social good and not just by profit alone.

The raison d’être is to reach a state of “Health Nirvana,” where the portentous disconnect of innovation to translation is bridged by a holistic approach from its conceptual stage to its ultimate destiny. Newer horizons lay ahead, unexplored roads to tread, but caution is warranted!