Shahid Akhter, editor, ETHealthworld, spoke to B N Manohar, MD and CEO, Stempeutics Research, to know more about the challenges and opportunities associated with stem cell therapy.

Medical Therapies: ChallengesThe increased prevalence of cardiovascular diseases, metabolic disorders, and cancer and the emergence of a more virulent form of the existing indications have posed many challenges to the existing medical therapies. As a result, the industry and medical community are looking for something novel that can reverse the disease's trend and determine whether it is possible to repair and regenerate diseased tissues, organs, or cells.

Cellular Therapy: Market and OpportunitiesThe Indian bioeconomy was about $80 billion in 2021. The bioeconomy consists of revenues generated from various segments like vaccines, therapeutics, diagnostics, Covid vaccine and Covid testing, which was about 14.5 billion dollars in 2021. In biotherapeutics, the revenue was about $10 billion. But these revenues are coming from biosimilars and biologics. The revenue generated from cell and gene therapies in India is very small.

The landscape of cellular therapies outside India has changed dramatically over the past few years and is likely to continue to do so over the next decade. There is a big opportunity for India to showcase cell and gene therapies for many unmet medical needs, and I strongly feel cell and gene therapy will be an emerging area of science and stem cells will be a major branch of medical treatments and will be a standard of cure and practise in the years to come. India has the opportunity to become a leading global player for stem cell products and services, and the government needs to react with speed in implementing a clear policy for cell therapy. Also, India can become a good manufacturing hub for cellular therapy.

Stem Cell Therapy: Care to Cure The current medicines available today only address the symptoms, making it only a symptomatic treatment. The medicines are not addressing the root cause of the disease. For example, in diabetes, the beta islet cell degenerates due to genetic defects or other issues, resulting in insufficient insulin production and the need for insulin injections.But we are not fixing the root cause of the disease, where we had to regenerate the degenerated cells. Here, cell and gene therapies tried to address the root cause of the disease, thereby regenerating the beta islet cells so that we do not have to take repeat insulin injections.

So far, we have been using stem cells like MSCs (mesenchymal stromal cells), which are available in various parts of the body. Currently, Stempeutic's major focus is on bone marrow-derived MSCs. We have got a patented technology for isolating MSCs from bone marrow. We have the technology to multiply, expand, and ultimately produce the end product. But the current trend is moving towards engineered cells. So far, we are working on non-engineered stem cells or on god-given stem cells and figuring a way to use them for multiple indications. Now, to further improve the overall efficacy of the cell therapy product, we are working on engineering MSCs so that they can overexpress the relevant growth factors and cytokines, which are required for treating the required indications.

Stempeutics: Journey so farStempeutics is a leading regenerative medicine company with a stem cell platform technology for the treatment of immune-mediated and inflammatory diseases. We have been working in this field for the last 14 years. We are an MSC company. We focus on mesenchymal stem cells or mesenchymal stromal cells. In the last 14 years, we have been working on R&D, preclinical studies, and clinical studies to see what the potency of these MSCs is. Also, we have undertaken numerous studies in terms of testing the safety profile of this compound. We tried many indications, and ultimately, we succeeded on 2 of them: one is the peripheral vascular disease called clinical limb ischemia (CLI), and the other is knee osteoarthritis (OA).

After going through multiple phases of DCGI-approved clinical trials, the Stempeucel product has been granted manufacturing and marketing approval for the treatment of CLI and OA by the Drug Controller General of India (DCGI). Stempeucel becomes the first allogeneic cell therapy product to be approved for commercial use in India and the first stem cell product to be approved globally for CLI indications. Stempeucel product has been licenced to Cipla for CLI indication (marketed under the brand name Regenacip) and to Alkem Labs for OA indication (marketed under the brand name StemOne) for the India territory.

Stempeutics has put India on the world map of regenerative medicine. The first company to develop a drug, which was designed and developed by Indian scientists, wasStempeucel has strong patent protection, with more than 20 granted across the globe. The core Stempeucel patent has been granted in the USA, Australia, New Zealand, South Africa, Japan, China, Singapore, the UK, France, Germany, Italy, and the Philippines. Stempeucel has been classified as an "advanced therapy medicinal product" by the European Medicinal Agency and designated as an orphan drug for the treatment of CLI due to Buergers Disease. Stempeutics is the first company to come up with a cell-based drug that has gone from bench to bedside. Additionally, it is the first company to have taken the product from concept to being commercially available for treating patients.

Now that we have commercialised two indications in India (CLI and knee OA), Stempeutics is seeking partners for further clinical development and commercialization of Stempeucel in the US, Europe, and Japan for the knee OA product, whose prevalence is huge globally. In knee OA, we are taking patients who are grades 2 or 3 on the X-ray or MRI scale. Existing treatment options focus on providing temporary pain relief and reducing inflammation during the early stages of the disease without affecting its course. Stempeucel-OA has the potential to provide best-in-class pain reduction, improve stiffness, improve physical function, improve quality of life, and have the potential to regenerate or maintain cartilage and stall further disease progression for Grade 2 and Grade 3 osteoarthritis patients based on the Kellgren and Lawrence radiographic criteria.

We are happy that we could commercialise our product, Stempeucel, in India. Now our goal is to take this product from India to developed markets like the US, Europe, and Japan. In that context, we have initiated conversations with the US FDA, the EMA in Europe, and the PMDA in Japan. There are a few challenges in terms of commercialising the product in developed markets, but we are trying to improve our product based on the inputs shared by these regulators on our chemistry, manufacturing, and control.

Stem Cell: ChallengesThe three key challenges we are facing are:

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For a range of unmet medical needs, India offers a fantastic opportunity to push cell and gene therapies: B .. - ETHealthWorld