How Stem Cells Support Personalized Medicine

Stem cells are transforming healthcare by enabling treatments tailored to individual needs. They can repair damaged tissues, model diseases, and test drugs in ways that were previously impossible. By using a patient’s own stem cells, doctors can avoid immune rejection and create therapies specific to the patient’s genetic and biological makeup.

Key Takeaways:

• What Are Stem Cells? They are "master cells" that can develop into various cell types and replicate themselves. Types include totipotent, pluripotent, and multipotent stem cells.
• Personalized Medicine: Focuses on creating treatments based on an individual’s genetic profile, reducing side effects and improving outcomes.
• Applications:
  • Disease Modeling: Stem cells help replicate diseases in the lab to study their progression and test treatments.
  • Drug Testing: Patient-derived cells allow for testing drugs on human tissues, predicting responses more accurately than animal models.
  • Regenerative Therapies: Stem cells are used to repair or replace damaged tissues, offering potential cures for conditions like Parkinson’s disease and heart damage.
  • Gene Editing: CRISPR and other tools enable fixing genetic mutations in stem cells, addressing root causes of diseases like sickle cell anemia.

Why Stem Cell Banking Matters:

• Collecting and preserving stem cells at birth ensures access to these powerful cells in their healthiest state for future treatments. Cord blood, tissue, and placental banking provide a "medical reserve" for personalized therapies.

Stem cells are reshaping medicine, offering safer, targeted, and more effective treatments. For families, preserving newborn stem cells provides a resource for future medical advancements.

Stem Cells and Personalized Medicine: The Basics

What Are Stem Cells?

Stem cells are often called the body's "master cells" because they form the building blocks for all organs and tissues. What sets them apart is their ability to self-renew (make endless copies of themselves) and differentiate (transform into specialized cells like neurons, muscle cells, or blood cells). Unlike specialized cells, which have a fixed role and rarely divide, stem cells can multiply extensively and adapt to different functions.

"Stem cells are defined by two properties – they can self-renew (make copies of themselves) and differentiate (develop into more specialized cells)." - Keith Alm, Author, About Stem Cells

Stem cells are classified by their potential to develop into different cell types. Totipotent stem cells, for example, can form both an embryo and a placenta. Pluripotent stem cells, such as embryonic stem cells and induced pluripotent stem cells (iPSCs), can become any type of adult cell. Meanwhile, multipotent stem cells are more restricted, typically limited to producing cell types within a specific tissue.

A groundbreaking discovery in 2006 allowed scientists to reprogram mature adult cells into iPSCs, which behave like embryonic stem cells. This innovation has been a game-changer for personalized treatments, as iPSCs can be genetically matched to individual patients. Today, the only FDA-approved stem cell products are blood-forming stem cells derived from cord blood.

With their regenerative and adaptive abilities, stem cells are paving the way for treatments tailored to the unique biology of each individual.

What Is Personalized Medicine?

Personalized medicine takes the capabilities of stem cells and applies them to create treatments designed specifically for each patient. Instead of using a generic approach for everyone with the same condition, personalized medicine focuses on your individual genetic profile, cellular traits, and lifestyle factors.

"Precision medicine is revolutionizing healthcare by transitioning from the traditional 'one-size-fits-all' approach to a more individualized strategy." - ScienceDirect

This approach relies on molecular profiling to predict which treatments will work best for you, reducing side effects and saving valuable time. Stem cells play a key role in this process. For example, scientists can use them to grow patient-specific "organoids" or tissue models in the lab. These models allow doctors to test how your disease responds to different therapies, giving them a clearer picture of what works for you. This shift moves healthcare toward being more proactive, predictive, and preventative.

Currently, there are over 174 approved companion diagnostics tied to specific therapies, and that number continues to rise as personalized medicine becomes more integrated into modern care.

How Stem Cells Create Customized Treatments

Stem cells are revolutionizing medicine by enabling treatments tailored to individual patients. The process begins with your own cells - either from a skin biopsy or a blood sample - which scientists reprogram into induced pluripotent stem cells (iPSCs). These iPSCs have the incredible ability to transform into any cell type your body requires, such as heart cells, neurons, or liver tissue. This approach allows researchers to closely study how your body reacts to diseases and treatments, speeding up the process of finding the most effective therapies. Below, we’ll explore how these techniques are used to model diseases, test drugs, and develop regenerative therapies.

Using Stem Cells to Model Diseases

To understand how a disease affects you personally, scientists can create what’s known as a "disease-in-a-dish" model using your stem cells. This method provides a window into how your unique genetic makeup influences disease progression and treatment outcomes. For example, in Parkinson’s disease research, patient-derived iPSCs are turned into dopaminergic neurons to study how alpha-synuclein accumulates in your specific cells. This is a critical improvement over animal models, as human cardiac action potentials, for instance, last 10 times longer than those in mice, making human stem cell models far more reliable for studying conditions like heart disease.

"iPSCs can capture the full myriad of genetic risk factors that contribute to sporadic PD, in contrast to the relative simplicity of the animal and cellular models." - Donna R. Trollinger and Priyanka Swamynathan, Thermo Fisher Scientific

The potential expands even further with organoids - 3D structures that mimic real organs. Researchers have developed organoids for over 14 different organs, including the brain, lungs, liver, and heart. These mini-organs offer a more accurate, near-physiological environment compared to traditional flat cell cultures, dramatically improving our ability to predict how treatments will perform in your body.

Testing Drugs with Patient Stem Cells

Once a disease model has been created from your stem cells, researchers can use it to test how your cells respond to various drugs. This process, called in vitro drug testing, involves exposing your stem cell-derived tissues to a range of compounds to determine which treatments are most effective while avoiding harmful side effects. For instance, in cystic fibrosis research, intestinal organoids have been used to evaluate CFTR modulators for rare genetic variants. This highlights how banking cord blood for rare diseases can provide a vital resource for future personalized treatments. These tests not only guided clinical decisions but also influenced insurance reimbursement policies for individual patients.

The implications for drug development are enormous. Currently, only 8% to 10% of oncology drugs that succeed in animal testing gain FDA approval, often due to unforeseen toxicity in humans. By testing drugs on patient-derived organoids, researchers can evaluate treatments on human tissues that match your genetic profile. In studies on metastatic gastrointestinal cancers, drug responses in organoids closely mirrored actual patient outcomes, highlighting their role in personalizing chemotherapy and targeted therapies.

"Patient-derived organoids (PDOs) can be used to predict individual drug responses, thus paving the way toward personalized medicine." - Giuseppe Novelli, Department of Biomedicine and Prevention, University of Rome Tor Vergata

Regenerative Therapies with Patient Stem Cells

Stem cells aren’t just for studying diseases - they’re also being used to repair damaged tissues. These regenerative therapies rely on your own cells, which your immune system recognizes as "self", reducing the risk of rejection. In 2018, Japanese researchers conducted the first human clinical trial for Parkinson’s disease using dopaminergic neurons derived from iPSCs. The goal? To replace the neurons lost in the substantia nigra and restore motor function.

Progress is also being made with more complex organs. Scientists have developed kidney organoids from hiPSCs that include glomerular and tubular structures. When transplanted into mice, these organoids became vascularized and even formed a filtration barrier, showing that lab-grown kidney tissue can function. Similarly, researchers have created human heart organoids that maintained spontaneous beating after implantation, with structural features resembling atrium and ventricle-like areas.

These breakthroughs signal a shift from merely managing symptoms to potentially curing diseases by regenerating tissues that are customized to your body’s unique anatomy and genetics.

Gene Editing and Stem Cells

Gene editing has made it possible to fix genetic defects directly in stem cells. Using CRISPR-Cas9, scientists can target and correct disease-causing mutations in your cells before they are used for treatment. This method works by guiding an enzyme to the specific DNA mutation, where it makes a precise cut. Your cell then uses a healthy DNA template, supplied by researchers, to repair the mutation. This approach aligns with the goals of personalized medicine, focusing on tailoring treatments to an individual's unique genetic makeup. These advancements are paving the way for therapies that address genetic issues at their core.

Fixing Genetic Defects

For conditions like sickle cell disease, doctors can harvest hematopoietic stem cells from your body, edit them in a lab, and then reintroduce them. A Phase 3 clinical trial in June 2023 demonstrated promising results: 29 out of 30 patients with severe sickle cell disease experienced 12 months without a crisis after receiving exa-cel treatment. This therapy, developed by Vertex Pharmaceuticals and CRISPR Therapeutics, involved editing the BCL11A enhancer in patients' CD34+ cells. The median time for neutrophil engraftment was just 27 days, and total hemoglobin levels reached an average of 12.5 g/dL within six months.

This technique is also being explored for other genetic disorders. In April 2020, researchers at Washington University School of Medicine used CRISPR-Cas9 to fix a mutation in the WFS1 gene in iPSCs (induced pluripotent stem cells) from a patient with Wolfram syndrome. These corrected cells were turned into pancreatic β cells, which reversed diabetes in mice for 6 months by producing insulin effectively. Similarly, for retinal conditions, scientists corrected 13% of RPGR gene mutations in patient-derived iPSCs, showing potential for personalized therapies for diseases like X-linked retinitis pigmentosa.

Newer tools like base editors and prime editors are making these procedures safer and more precise. Base editors can chemically change single DNA letters without breaking the strand, while prime editors use a “search-and-replace” system to fix various mutations, including insertions and deletions. For example, prime editing successfully corrected the sickle cell mutation in 15% to 41% of patient-derived blood stem cells. After transplantation into mice, an average of 42% of red blood cells expressed the corrected gene 17 weeks later.

Benefits of Gene-Edited Stem Cell Therapies

Using autologous (self-derived) gene-edited stem cells eliminates the need for donor matches and reduces the risk of graft-versus-host disease. Patient-specific iPSCs show a 40% higher engraftment rate and reduce complications by 70%, making them a safer and more effective option. Clinical evidence supports these advantages: 97% of patients treated with exa-cel avoided vaso-occlusive crises for at least 12 months, and 100% stayed out of the hospital for related issues during the same period.

"The combination of CRISPR gene editing technology with personalized stem cell therapy opens new possibilities for correcting genetic defects at the cellular level." - OmniStem

These therapies offer much more than symptom management. By addressing the root genetic cause, they hold the potential to provide a one-time curative treatment. For families considering newborn stem cell banking, it’s worth noting that these cells could serve as an ideal platform for future CRISPR-based therapies, especially for children with genetic conditions caused by single-gene mutations.

Why Newborn Stem Cell Banking Matters

With advancements in personalized therapies, newborn stem cell banking offers a lifelong resource for future medical treatments tailored to the individual.

What Is Newborn Stem Cell Banking?

Newborn stem cell banking involves collecting stem cells from birth tissues that are typically discarded. This process is quick, non-invasive, and painless, taking just five minutes after delivery. Cord blood is collected using a closed-system blood bag to ensure sterility, while cord and placental tissues are carefully cleaned, sterilized, and prepped for long-term preservation. These samples are then processed to concentrate the most potent cells and frozen in liquid nitrogen at temperatures near -180°C, where they can remain usable for decades.

The stem cells collected at birth are in their healthiest state - untouched by aging, environmental factors, or health issues. Cord blood is rich in hematopoietic stem cells (HSCs), which are essential for treating blood and immune system disorders. Meanwhile, cord and placental tissues contain mesenchymal stem cells (MSCs), which play a key role in regenerative medicine and tissue repair. Using one’s own banked cells eliminates the risk of immune rejection and avoids reliance on immunosuppressive drugs. These preserved cells not only support current medical treatments but also pave the way for future regenerative therapies.

"The ability to store one's stem cells from birth... means that individuals have a personalized 'medical reserve', which can be accessed in the future should the need arise." - Fang Wu, Tsinghua University

Banked stem cells act as a type of biological insurance for emerging therapies. For example, by December 2018, the Cord Blood Registry had released over 500 cord blood units for clinical use, with nearly 80% utilized in regenerative medicine trials - many for neurological injuries occurring at or near birth. Given that one in three people in the U.S. could benefit from regenerative medicine during their lifetime, these preserved cells are becoming increasingly important as medical advancements continue. Newborn stem cell banking supports the shift toward personalized medicine by safeguarding these powerful cells for future use.

How Americord Registry Supports Families

Americord Registry turns this potential into reality by providing advanced stem cell banking services. Their offerings include the preservation of cord blood, cord tissue, placental tissue, and exosomes. Using their CryoMaxx™ Processing system, which employs FDA-cleared, closed technology, they maximize cell recovery while maintaining sterility. Samples are stored in 5-compartment vials, allowing families to use portions of the cells for specific treatments without thawing the entire sample. This flexibility is particularly important for therapies like gene editing or regenerative treatments.

Americord offers several storage plans to meet different family needs. The Essential Family Plan focuses on cord blood banking, while the Complete Family Plan includes cord blood, cord tissue, and placental tissue - providing access to both HSCs and MSCs for a broader range of potential treatments. Americord also prioritizes transparent pricing and individualized customer support, helping families make well-informed choices about preserving their child’s biological resources. For more information on pricing and services, visit the Americord Registry website.

Conclusion

Stem cells are transforming healthcare by making treatments more personal, precise, and forward-thinking. These cells allow for therapies tailored to an individual's genetic makeup, aiding in disease modeling, drug testing, and tissue repair. Using a patient’s own stem cells (autologous) not only avoids immune rejection but also eliminates the need for donor matching, making treatments safer.

"Stem cell banking is an exciting frontier in personalized healthcare, offering the potential for highly individualized, effective treatments for a range of medical conditions." - Fang Wu, Department of Biomedical and Health Engineering, Tsinghua University

For families, newborn stem cell banking offers a chance to preserve cells at their peak health. With stem cells already used to treat over 80 serious conditions and ongoing research pushing boundaries into previously untreatable diseases, banking these cells at birth secures a valuable resource for future therapies. These cells are a perfect genetic match for the child and often compatible with siblings or close relatives.

Americord Registry is helping families take advantage of these advancements by providing services to preserve cord blood, cord tissue, placental tissue, and exosomes. With cutting-edge processing and flexible storage options, they ensure families have access to potentially life-saving treatments whenever they’re needed.

FAQs

How do doctors turn my cells into iPSCs?

Doctors generate induced pluripotent stem cells (iPSCs) by reprogramming somatic cells, such as skin or blood cells. To achieve this, they introduce specific genes - like OCT4, SOX2, KLF4, and c-MYC - which reset these mature cells back to a pluripotent state. Once reprogrammed, these cells gain the potential to develop into virtually any cell type. This breakthrough has become a cornerstone for personalized medicine, offering opportunities for customized treatments, regenerative therapies, and advanced disease modeling.

Can organoids predict which drug will work for me?

Organoids can play a role in identifying which drug may work best for you. By replicating your body's unique response in a lab environment, they contribute to personalized medicine, allowing treatments to be customized to suit your specific needs.

When should I bank my newborn’s stem cells?

The best time to prepare for banking your newborn’s stem cells is during pregnancy, ideally between 28 and 34 weeks of gestation. This timeframe gives you ample opportunity to coordinate with your healthcare provider and the birthing facility, ensuring the collection process is seamless and doesn’t disrupt delivery. Planning early also allows you to handle all the required paperwork and finalize arrangements well before your due date.