Myth vs reality of stem cell-based products

With stem cell therapy set to become an acknowledged treatment alternative globally for unmet medical diseases, BN Manohar, MD and CEO, Stempeutics Research, opines that it has become increasingly crucial to improve the level of knowledge among doctors

The traditional science of medicine has evolved exponentially. Today, there is a pill for almost everything that could possibly plague a human body. But, when it comes to critical and life threatening or debilitating/ degenerative diseases like diabetes, arthritis, muscle degeneration or organ disease, are these medicines offering complete treatment? Or just slowing down the progression of the ailment and creating an illusion of improving quality of life? Most often than not, the latter scenario is true.

This is when an innovative arm of medicinal science walks into the picture – stem cell treatments. This non-traditional approach is much more than just an intervention to give patients temporary relief or delay the effects of the condition. Research so far gives hope that in addition to possibly completely halting the progress of specific medical conditions, it may also be possible to actually reverse the degeneration of tissues/ organs, giving birth to the term ‘regenerative medicine.’

The world over, scientists and researchers are working aggressively towards unlocking the true potential of stem cell therapies and treatments. Clinical trials are successfully proving the efficacy of stem cells in repairing damaged tissue. Regenerative medicine, is a novel multi-disciplinary field that relies on cell therapeutics and bio-engineering techniques to enhance the functionality of organs and tissues. In technical terms, regenerative medicine as defined by the US NIH is the process of creating living, functional tissues to repair or replace tissues or organ function lost due to age, damage or congenital defects.

Stem cell research is continually advancing knowledge on how an organism develops from a single cell and how healthy cells are derived from precursor cells to replace damaged tissue. All this knowledge and data is then used in developing stem cell treatments for diseases that do not have a cure yet, with an aim to replace/ regenerate damaged cells with healthy cells.

Stempeutics Research, which is a group company of Manipal Education & Medical Group and a joint venture with Cipla Group, has invested in ground breaking research in this space. The company has spent the last nine years developing innovative stem cell products that could provide a cure for chronic and degenerative diseases. Stempeutics’ stem cell based technologies and products, are under various phases of clinical trials which will put India firmly on the global medical innovations map and potentially revolutionise the treatment protocols for a number of unmet medical conditions.

The most significant breakthrough has been the successful completion of phase II clinical trial of Stempeucel – a novel stem cells based medicinal product, designed to treat Critical Limb Ischemia (CLI) due to Buerger’s disease. Buerger’s Disease is a rare and severe disease affecting the blood vessels of the limbs. It is characterised by inflammation and occlusion of the vessels of extremities resulting in reduced blood flow to these areas, thus leading to severe rest pain and ulcers or necrosis, which finally leads to amputation of the limbs. Buerger’s Disease is a major unmet medical need in India and globally. Stempeucel treatment is designed to enhance the body’s limited capability to restore blood flow in ischemic tissue by reducing inflammation and improving blood flow to the affected limbs.

The phase II trial conducted by Stempeutics is the largest ever phase II trial in no-option patients with Buerger’s disease in the world. In the trial Stempeucel demonstrated statistically significant results in ulcer healing, rest pain reduction, increased ankle brachial pressure index (indicator of increased blood flow due to neoangiogenesis) and improvement in quality of life.

Stempeucel is developed from allogeneic pooled mesenchymal stromal cells extracted from the bone marrow of healthy, adult voluntary donors. The drug works by increasing blood flow which is the root cause of diseases and just does not give symptomatic relief, thus proving to be much more effective agent of treatment. Stempeutics’s proprietary pooling technology allows an efficient manufacturing process with minimum wastage of resources in order to provide the product at an affordable cost to patients. Stempeucel has a strong patent protection with 18 patent applications filed across the globe. The core patent has been granted in the US, Australia, New Zealand, Singapore, South Africa, China and Japan. In addition, Stempeucel has been given the Advanced Therapeutic Medicinal Product designation by the European FDA and Buerger’s disease has been granted the Orphan Drug Designation (ODD) by the same agency.

Challenges

Stem cell related research is in its infancy in India, similar to the IT/ ITES industry two decades ago. Stempeutics has faced several challenges including having to convince policy makers about the importance of aggressive R&D including clinical research in this area, ensuring that the company is self-regulated and works to the highest global standards and to invest in a technology that is not even understood well by most people.

As with any new or innovative treatment method, there are a number of myths and misconceptions surrounding stem cell research and treatment. These are mainly due to lack of knowledge among professional doctors as well as end consumers.

With stem cell therapy set to become an acknowledged treatment alternative globally for unmet medical diseases, it has become increasingly crucial to improve the level of knowledge among doctors about the importance of stem cells. This in turn will help doctors guide their patients better, give them complete and accurate information and secure a patient right to informed decision making.