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Stem Cell Clinical Trials in 2024: Risks, Trends, and Success Chances

Regenerating damaged organs, curing chronic diseases, and self-healing abilities are no longer sci-fi. Stem cell therapy is the closest we’ve been to “superhuman.”


But the complexity of regenerative medicine makes clinical trials an uphill climb.


Stem cell clinical trials are certainly not talked about enough.


So, what do clinical leaders need to know about stem cell clinical trials? What are the latest trends? And what pitfalls to avoid?


We are Rubix LS, an AI-powered research catalyst (CRO+) specializing in Phase I clinical trials. This article will guide you through everything we know about stem cell clinical trials in 2024: success chances, pitfalls, state-of-the-art, and market trends.


Keep reading, or choose a section to jump ahead


What does the stem cell therapeutics market look like?


In theory, the possibilities are limitless. Simply because stem cells, by definition, can replace any tissue.


In practice, so far, regenerative medicine has shown promise in cancer, diabetes, spinal cord injuries, and Parkinson's disease.


The interest and investment in the field justify the stem cell market projection to reach a staggering $31.6 billion by 2030.


But despite the optimistic projection, we see other sobering statistics:


  • Despite the high number of studies, only one stem-cell-based therapy has been reviewed and approved by the FDA. That is hematopoietic stem cell transplantation to treat blood and immune cancers and disorders.

  • Regulatory authorities are putting effort into educating patients about stem cell therapies and warning against unapproved stem cell products

  • The FDA overtly warns against engaging with ads about stem cell therapies that are not:

  • FDA-approved

  • Under an IND application

  • Some experts encourage the International Society of Stem Cell Research (ISSCR) to consider banning unapproved products


With tight regulations and low approval, you might wonder:


What are the success chances of stem cell clinical trials?


Before taking your stem cells to Phase I, consider the upsides and downsides:


Pros of stem cell therapy clinical trials


1. Safety


Stem cell therapies tend to perform well on safety. Phase I and II RCT show that cell therapy is safe for cardiac and coronary artery disease (CAD).


2. Recruitment


Patients can be motivated to enroll in stem cell clinical trials for several reasons:


  • The novelty of the treatment

  • The lucrative benefits like the ability to stabilize conditions for a year (or even longer)

  • Stem cells’ ability to treat rare diseases—which are difficult to address with small molecules and traditional drugs

  • Historical treatment safety of regenerative medicine trials


3. Success rate


Early trials on stem cells are often positive and show promising results.


Phase I-II data on the first stem cell therapy in cardiovascular medicine suggest feasibility and safety for both acute and chronic ischemic heart disease. After phase I-II RCT, it is clear that BMMC transfer after AMI has the potential to improve the recovery of LV systolic function beyond what can be achieved by current interventional and medical therapies


4. Efficacy


DVC Stem posted preliminary data from their clinic showing that 87.5% of their total patient population reported sustained improvement in their condition within three months of treatment.


The benefits included:


  • More stamina

  • Higher libido

  • Sharper cognitive functions (memory and decision-making)


5. Trial endpoints


Hard endpoint studies require many participants, making them expensive and time-consuming.


Regenerative medicine researchers often relied on surrogate endpoints in early regenerative medicine trials. This helped get trial costs down and advance the niche.


Cons of stem cell therapy clinical trials


1. Cost


Cost immediately comes up when talking about stem cell therapy.


Cellular products and viral vectors have high manufacturing costs.


Disease specificity also means that the trial will target a small patient population, leading to high prices per patient.


2. Trial endpoints


Yes, getting away with surrogate endpoints can lower costs. But it might not tell the whole story.


Moving forward with surrogate metrics can be a fragile foundation for scaling Phase II-III. This depends on the therapeutic niche.


We only know of a few trials that have used hard endpoints to evaluate, like the REPAIR-AMI and the BALANCE studies. 


Looking for help to start clinical trials for your IND? Contact Rubix LS


3. Outcome variability


Outcomes can vary widely based on the:


  • Stem cell type

  • Treated condition

  • Trial design

Some early trial results even showed mixed outcomes and transitory improvements.


4. Need for advanced-phase data


Stem cell research is still young.


As of 2022, 71% of trials for GVHD mesenchymal cell therapy were still in Phase I-II.

This shows a need for advanced-phase testing to have accurate success rates.


Regulatory advice: what to consider before a stem cell therapy clinical trial?


Regulatory and ethical authorities constantly provide and update recommendations.


Here’s what you need to keep in mind before starting stem cell clinical trials:


1. Clinical consideration


Fundamental pre-trial principles include:

  • Using quality-controlled cells

  • Demonstrating efficacy and safety in animal models

  • Involving stem cell expertise in protocol review

2. Trial design


During trial design, it’s crucial to have expertise in mechanisms of action, efficacy endpoints, dosage optimization, and safety assessment.


The design should also consider the key metrics of the trial phase like:


  • Safety assessment targeting detecting adverse events

  • Efficacy through surrogate endpoints with a focus on clinical benefits


Early design is foundational for later trials and phases. Make sure you have the right expertise on board.


3. Recruitment


In Phase I-II trials, you should enroll participants in the late stages of serious illness. Yet not so ill that they are at greatly increased risk for adverse events.

4. Review


Establish centralized review processes involving representatives from each institution to ensure appropriate risk-benefit assessment.


Your review process should also have stem cell expertise.


5. Comprehension


Participants should grasp key trial features beyond standard informed consent. Administer clear questionnaires to patients to ensure detailed comprehension.


6. Publishing


Publish timely results (including negative outcomes) in peer-reviewed journals. Credibility from the scientific community is key on the journey to the market.


7. Medical writing


You need scientific acumen, writing proficiency, and attention to detail to make it to the market.


Your protocols, reports, safety updates, and regulatory submissions tie in all of your trials together. This facet is especially important in regenerative medicine research.


Make sure your writing is taken care of by experts.


Looking for help to start clinical trials for your IND? Contact Rubix LS


What are the latest trends in stem cell clinical trials?


Despite the challenges, we see no shortage of new trends that offer hope for the future.


Here’s where the biggest regenerative medicine opportunities lie in 2024:


3D bioprinting


Creating 3D bio-printed scaffolds or organoids that mimic the natural environment of target tissues can improve cell survival, differentiation, and therapeutic efficacy.


This application showed promise in osteogenesis by mimicking and repairing bone defects.


Gene Editing


Combining stem cell therapy with gene editing tools promises to correct genetic defects and treat diseases at their source.


A 2020 study showed that induced pluripotent stem cells (iPSCs) with CRISPR/Cas9 might be able to treat primary immunodeficiency diseases (PIDs).


Engineered Microenvironments


Beyond therapeutics, we can employ stem cells to model disease progression.

iPSC-based disease can mimic how genetic and rare diseases manifest.


Disease-specific iPSC can help us develop treatments against them for diseases that have no known cure.


Looking for help to start clinical trials for your IND? Contact Rubix LS

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