What’s Holding Cell Therapies Back? A Look Into The Evolution And Future Of Stem Cell Tech

Salvatore Viscomi, M.D., is the Chief Medical Officer at GoodCell, a life sciences company, offering Personal Biobanking.

Cell and gene therapies have grown in popularity and effectiveness over the past several years, reaching 27 approvals by the U.S. Food and Drug Administration (FDA) by December 2022. What’s more is that the FDA anticipates approving another 10 to 20 therapies each year by 2025.

While cell therapies have already come a long way, there is much to be done to streamline clinical trial submissions and approvals. The first step toward improvement is to first understand the historical evolution of stem cell technology and where cell therapy advancements are today.

The Evolution Of Stem Cell Technology

While they may seem relatively new, cell therapies have actually been treating rare forms of blood cancer for decades. In fact, stem cell research dates back to over 60 years ago and continues to be one of the most heavily saturated fields of scientific research.

Early Cell Therapy Breakthroughs

One of the earliest attempts at stem cell research can be traced to Dr. Edward Donnall Thomas, who studied the possibility of using bone-marrow transplantation to treat life-threatening diseases. After Dr. Thomas’ early advancements, a monumental breakthrough occurred in 2006 when Dr. Shinya Yamanaka of Kyoto University established a method that reprogrammed adult cells into induced pluripotent stem cells (iPSCs) through the expression of embryonic transcription factors. These factors—later called the Yamanaka factors—and his reprogramming process successfully obviated the world’s dependency on controversial embryonic stem cell research.

Today’s Advancements In Patient Treatments

Since then, there has been staggering momentum of iPSC and other cell therapy approvals accompanied by potentially life-changing results. In 2021, for example, Vertex Pharmaceuticals successfully treated a type 1 diabetic patient using VX-880—a stem cell-derived pancreatic islet cell replacement therapy. More recently, biotech companies Sernova and Evotec announced that the first two patients in the second cohort of their active diabetes clinical trial have been implanted with off-the-shelf, induced pluripotent stem cell (iPSC)-based islet replacement therapy.

Arguably one of the most beneficial aspects of iPSC therapy is its use of a patient’s own cells. This type of treatment, called autologous therapy, may eliminate the need for a donor match, thereby saving valuable time and negating immunosuppressant usage. Immunosuppressants have historically been used to ensure a patient’s body does not reject the donor cells but unfortunately also increases their susceptibility to other diseases.

FDA Efforts To Streamline Cell Therapy Approval Process

Unsurprisingly, the flood of iPSC and other cell therapy clinical trials is being accompanied by severe delays in FDA approvals. The FDA has recently announced, however, the reorganization of its Office of Tissues and Advanced Therapies (OTAT) to become a Super Office within the Center for Biologics Evaluation and Research (CBER). There are plans to increase staff to more than 400 employees and rename the organization to the Office of Therapeutic Products (OTP). This restructuring intends to significantly expedite the approval process for all involved parties.

In addition, the FDA is looking to expedite the identification of alternative product versions that may be safer and more effective for patients. Those who are interested in studying multiple versions in an early-phase clinical trial for a single disease are encouraged to take an umbrella style approach and read the FDA’s Guidance for Industry document for more information.

Foreseeable Challenges And Proposed Solutions

These FDA updates demonstrate a collective effort to improve the rate at which cell therapies are approved, proving their undeniable importance. Despite this, several foreseeable challenges are gaining attention among researchers and medical professionals alike and require immediate solutions.

Scaling Autologous Cell Therapies

Autologous iPSC therapies offer numerous advantages to the health of the patient but present several obstacles that may prevent them from reaching their full potential. When compared to allogenic therapies, or those dependent on donor cells, there are manufacturing disadvantages that make the autologous cell therapy manufacturing process costly and time-consuming.

To scale production, novel techniques for cost-effective manufacturing are needed to modernize the process. In addition, stronger relationships with data management and analytics system providers must be built in order to optimize production science and consequently product design.

Reducing Associated Health Risks

A primary clinical challenge with current therapies is the risk of toxicity-related events such as cytokine release syndrome (CRS) and CAR-related encephalopathy syndrome (CRES). CRS is known to cause flu-like symptoms in patients, whereas CRES can be more severe, resulting in speech and memory complications for several days.

To mitigate cell therapy related events such as CRS and CRES, novel diagnostics to identify high risk patients and target them with prophylactic therapies are required. In addition, the engineering of CAR-T cell therapies that either lessen the likelihood of CRS and CRES development or regulate them entirely have been proposed as viable solutions.

The Future Of Cell And Gene Therapies

While iPSC and other cell therapies require time to reach FDA approval—which may be sooner than expected given the FDA’s recent streamlining efforts—estimates suggest that regenerative medicine may eventually benefit almost 1 in 3 people in the United States alone.

As the sheer number of affected people continues to climb, cell and gene therapy markets continue growing in tandem. According to the 2022 Cell & Gene Therapy Clinical Trials Market Research Report, the global cell and gene therapy market size was estimated at $5.2 million in 2021, then $5.9 million in 2022 and is projected to reach $10.8 million by 2027. The private equity and venture capital investment has also skyrocketed from $362 million in 2020 to nearly $68 billion in 2021.

Given the undeniable financial interest and escalation in cell therapy breakthroughs and countless more healthcare advancements are on the horizon, and the future of precision and regenerative medicine has only just begun.


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