The magic of cell therapy 

Marjie Hadad talks to cell therapy expert and Kadimastem CEO Asaf Shiloni about the potential of cell therapy. 

“I am attracted to cell therapy because traditional medicine has reached its limitation in treating the ailments of the 21st century,” says Asaf Shiloni, 20-year biotechnology executive, cell therapy expert and CEO of Kadimastem. 

He continues: “It’s known today that most diseases develop because of cells that were injured, died or malfunctioned for genetic and/or pathological reasons. If we can replace the dead or damaged cells, we can decrease or overcome the loss of functionality or malfunctioning. Rather than treat diseases, we can try to ‘cure’ them, by replacing the faulty cells with ‘healthy’ ones or with cells that will support ‘healthy’ physiology.” 

MH: What’s so special about cell therapy in comparison to other approaches? 

AS: For organisms to thrive, cells must build and maintain tissues and organs must function properly. Unlike current remedies, like small chemical entity drugs or biologics like monoclonal antibodies and gene therapies, cell therapies use living cells that react to changing physiology and/or their environment. In other words, cells are small ‘computers of health’ that respond to respective needs as part of the therapy. This gives an advantage over other remedies.  

MH: Is cell therapy attractive to institutional investors? 

AS: Institutional investors understand the medical importance of cell therapies as well as their immense economic and ROI potential. It’s no wonder that the Cell Therapy Market is projected to reach  US$14.5 billion by 2030 and is on its way to becoming one of the highest valued markets within the biopharmaceutical industry. (Journal of Cyber Policy).  

For example, in September 2019, Semma Therapeutics, a cell therapy company that develops a treatment for diabetes, was acquired by Vertex Pharmaceuticals for $950 million. In August 2019, Blue Rock, a cell therapy company that develops a treatment for neurological conditions such as Parkinson’s disease, was acquired by Bayer, at a valuation of more than $1 billion. And most recently, in February 2021, Sana Biotechnology, a US cell therapy company, raised about $600 million through an IPO, with a market valuation of about $5 billion. 

Additionally, vaccine, diagnostics and gene editing technologies have become even more attractive to investors in light of Covid-19. These technologies have yielded high returns and many funds are now looking for the ‘next big thing’ in these fields. 

We believe the next big thing in these fields will be based on differentiated cells derived from human pluripotent stem cell research. Companies with success in this area will see an increase in valuation as they continue through clinical trials and secure market approvals.  

MH: What are the biggest challenges faced by cell therapy CEOs today? 

AS: I think there are four main challenges: 

  1. Awareness – raise awareness among pharma and financial sector opinion leaders of the medical potential of cell therapy to treat unmet clinical needs. This is important to secure funds for the development of cell therapy products.  
  2. Clinical Development –the need to develop cell therapy products fearlessly, timely, and with vision.  
  3. Production –the need to produce cellular products diligently, while maintaining strict quality standards and verifying safety and efficacy. 
  4. Clinical Trials –assiduously conduct the lengthy process of clinical trials, which are a crucial step toward the validation of the efficacy and safety of the treatment. The goal is to reach market approval as fast as possible, while managing patient and investor expectations. We need to maintain communication, as well as hope and excitement throughout the long journey. 

MH: What does Kadimastem do?   

AS: Kadimastem is a clinical stage biotechnology company with a platform technology for cell therapy that enables the production of off-the-shelf cell-based products, sourced from Human Embryonic Stem Cells (hESCs). Kadimastem is currently developing two cell therapies: AstroRx, for the treatment of Amyotrophic Lateral Sclerosis (ALS) and IsletRx, a potential cure for Insulin Dependent Diabetes. 

With AstroRx, Kadimastem is transplanting healthy brain glial cells (Astrocytes) to support the survivability of nerve cells, responsible for sending the necessary signals to the muscles for the body to move.   

We completed testing AstroRx cells in a first in-human, Phase I/IIa clinical trial on ALS patients. The results of the clinical trial demonstrated a high safety profile of AstroRx cells as well as that a single injection could slow disease progression for a period of three months.  

The therapy is scalable and optimised to be commercialised as a stable ‘off-the-shelf’ product to reduce the cost of treatment. Kadimastem will be in touch shortly with the FDA to discuss the best way to design an Investigational New Drug (IND) for clinical study in the US, which we plan to begin in 2022. 

Kadimastem’s IsletRx contains pluripotent stem cells that are differentiated, expanded, cultured, and purified in bioreactors to create large quantities of insulin and glucagon-secreting, human pancreatic islet-like cells intended to hopefully cure Type 1 diabetes.  

Our cells are not only small factories of insulin (beta cells) or glucagon (alpha cells), but they are also able to sense blood sugar levels and secret insulin when blood sugar is too high and glucagon when blood sugar is too low. Importantly, unlike available drugs, or insulin pumps, these cells act identically to the islets in the pancreas, and are able to compute external data and react accordingly. 

In our preclinical proof-of-concept study, IsletRx cells demonstrated the ability to balance blood sugar levels and maintain normal levels over time in immunocompetent diabetic mice. The functions of IsletRx are protected by patents worldwide.   

Kadimastem is encapsulating these cells to provide them with the required protection from the patient’s immune system, allowing the cells to function for a long period of time. Once finalised and approved, this therapy will replace the need for daily glucose monitoring and insulin injections and will show for the first time how cell therapy can cure a disease. 

MH: What are the latest developments in cell therapy? 

AS: The potential of cell therapy is endless. The latest key developments include: 

Stem cell derived cell therapy products –products such as the company’s differentiated cells derived from human pluripotent stem cells are reaching clinical trials and the advanced stages of clinical trials. Approval of these treatments and cures will mark a new era for cell therapy. 

Immune Therapy (CAR-T) – one of the latest cancer treatments, immune therapy is training a patient’s T-Cells to fight tumours. There are several companies already offering such a therapy and more at different stages of clinical trials. This technology is changing the landscape of cancer treatment and biotechnology.   

Gene Therapy –gene therapies and technologies using adult stem cells, like haematopoietic stem cells and mesenchymal stem cells, are getting closer to completing phase III clinical trials and reaching the market.  

3D Bioprinting – 3D bioprinting involves the printing of biological materials and cells to form tissue and with the vision to print complete organs. Cells are the key component, and cell therapy companies are exploring ways to adopt this technology. 

MH: What will the cell therapy industry look like in 10 to 15 years? 

AS: I strongly believe that by the end of this decade, several cell therapy products that are currently in development or in clinical trials, will reach commercialisation. Once these treatments become a reality, I anticipate a major shift of attention and capital toward cell therapy and stem cells. 

Within five-10 years, stem cell derived healthy and functional cells will replace, restore, and repair functionality of diseased and malfunctioning cells in various degenerative diseases. Cultured cell derived tissue and even complete organs will reach clinical trials and will be commercialised. Within 10-15 years, complicated organs, such as bones, kidneys, lungs, and maybe even full hearts, will be 3D printed. Companies like ours are mastering stem cell differentiation and providing the building blocks supporting the creation of such organs that will enable people to live longer with healthy tissue and organs. 

Volume 22, Issue 4 – Fall 2021

About the author

Marjie Hadad is a veteran publicist with 20 years of experience in medical public relations including pharmaceuticals, medical technology and digital medicine. She is the general manager of Must Have Communications and the international press representative at Kadimastem. 

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