Stem cell therapies

What conditions can be treated with stem cells?

Stem cell research is a global enterprise, and much of the work is being done in Europe. This page gives readers a good sense of the medical conditions stem cell researchers in Europe focus on, and the specific research challenges they address. This page also offers links to in-depth information about many of the research projects.

Stem cells have the ability to self-renew

Stem cells have remarkable potential for new medical therapies. But to understand what makes stem cells so special, you must understand exactly what they are, and what they’re capable of doing. This page offers an accessible introduction to stem cell biology and their unique properties.

How to turn any cell of the body into a stem cell

Scientists have developed techniques allowing them to 'reprogram' any cell in the body into an induced pluripotent stem (iPS) cell, which can be used to create either more stem cells or specialized cells for use in experimental medical treatments. This page gives readers a brief introduction to the history of iPS cells, what researchers are studying with iPS cells, and challenges associated with iPS research.

How do stem cells actually work?

Stem cells are unspecialized cells that can develop into various cell types while also making exact replicas of themselves. This amazing ability has made stem cells a candidate to treat a wide variety of diseases and conditions—though the vast majority of potential therapies remain in the research stage.

The spread of fraudulent stem cell treatments

All the coverage of research into promising (but often unproven) medical treatments involving stem cells has created conditions allowing some people to engage in activities regulators describe as scams. As this news article explains, attorneys general in at least seven states have taken legal action against businesses that promised (costly) stem cell treatments but allegedly engaged in fraud.

Stem cell therapy has shown promise in treating spinal injuries in an early-stage clinical trial.

Stem cells can reboot the ability of cells to repair themselves

Stem cells—special cells that can self-renew and evolve into new cell types—may provide a path to address aging. Instead of repairing aging cells, stem cells can replace a cell's "repair kit," effectively rebooting the internal processes of tissues to a more youthful state.

‘Hope for Healing’: The first-ever use of an in utero stem cell treatment

In 2023, researchers at UC Davis performed the first-ever fetal stem cell transplant, attempting to cure a case of spinal bifida prior to childbirth using stem cells derived from the placenta. The trial, and others including an effort to treat muscular dystrophy, highlight the potential power of stem cell therapy.

There are three types of stem cells, each with different properties

Embryonic stem cells are "blank slates" that can change into any other type of cell. Adult stem cells can become specialized cells to replenish the supply or repair injury, while induced pluripotent cells are "reset" by scientists and used in clinical and medical applications.

The scandal that rocked stem cell research

In 2014, a Japanese research group published a paper suggesting ordinary cells could be reverted to pluripotent stem cells by subjecting them to external stressors. The results generated interest but could never be replicated. The lead researcher was accused of fraud, and the group’s supervisor committed suicide.

Stem cells develop into a wide range of other specialized cells in the human body

Stem cells are different from other cells because they are capable of creating copies of themselves and producing other types of cells. For example, the ones found in our brains, bones, or muscles. Curious about stem cells? Here's a breakdown of how they work.

'Cells Making Cells': 'Science Sam' discusses the potential of stem cells

In this episode of the Ologies podcast, host Alie Ward picks the brain of science communicator Samantha Yammine—whose PhD work focused on neural stem cells. The conversation covers everything from the earliest days of stem cell research to what the future of stem cell therapies may look like.

The 'Armamentarium' is a set of new gene therapy tools—including viruses that could shuttle therapies into the body—designed to fight brain diseases.

Scientific breakthroughs funded by NASA, the NSF, and the NIH include GPS, sign language, bladeless LASIK surgery, and quantum dots—tiny semiconductor crystals used in many modern televisions.

Using stem cells for bone marrow transplants

Stem cells hold promise for many future medical applications, but only one type of stem cell is currently approved for use. This page offers a clear description of adult stem cells called hematopoietic stem cells, which create the full range of blood cells and are approved for use in treatments such as bone marrow transplants.

See this list of FDA-approved gene therapies, including offerings for inherited conditions as well as some forms of cancer.

Induced pluripotent stem cells are "reprogrammed" cells

Induced pluripotent stem cells are used in a great deal of stem cell research, but many people are still unfamiliar with exactly what “iPS” cells are. This video provides a clear (and short) explanation of iPS cells for non-experts, and offers insights into why scientists are interested in these cells.

A Nobel laureate discuss whether stem cells can extend lifespan

Listen to Nobel laureate Shinya Yamanaka discuss the potential of induced pluripotent stem cells to extend human lifespan. By reprogramming adult cells into embryonic-like stem cells, scientists might be able to rejuvenate tissues and treat age-related diseases—though ethical and safety concerns remain.

Support groups can offer insights into current and potential treatments

Beyond connecting with patients with similar experiences, support groups can also become centers for advocacy and for sharing research advances and medical experiences, including potential side effects from novel therapies or clinical trials.

In 2023, the FDA approved gene therapy for sickle cell disease, enabling one-time infusion bespoke treatments that use a patient's own stem cells to effectively cure a painful condition that disproportionately affects people of African American descent.

US federal researchers are blocked from putting certain human cells in animals

In 2015, US policymakers put a halt to any government-funded work that would insert human stem cells into animals. The reasoning was that human cells might spread through the animal's body and perhaps to its brain, blurring the line between humans and nonhumans. The ban remains in place today even as gene-edited pig parts are being placed in humans.

In vitro gametogenesis: using induced stem cells to fertilize eggs

Using stem cells from skin and mouth cells could save hopeful parents time and money and spare egg donors from significant amounts of discomfort throughout the process. There are potential consequences, not just for infertility treatment, but for the ability to create customized cells of any type.

Stem cells become specialized cells through the activation or silencing of specific genes

After fertilization, when a sperm cell fuses with an egg, the resulting stem cell is described as totipotent—capable of transforming into any type of body cell or embryonic structures (e.g., placenta). Once differentiated into multipotent cells, they can only specialize further within specific cell type families, such as hematopoietic cells that become platelets and red and white blood cells.

Bone marrow transplants were the first successful type of stem cell therapies

Bone marrow contains two types of stem cells: Skeletal (develop into bone, cartilage, and more) and hematopoietic, which create new blood cells. The latter are critical in bone marrow transplants, and give the recipient the ability to create healthy blood cells to fight diseases like leukemia.

Gene therapy can occur inside the body, but it can also be completed outside the body—with scientists treating cells in the lab, and then those corrected cells are transplanted back into the body.

A new type of immunotherapy focuses on healthy cells hijacked by tumors

Purdue University scientists are at work on a new cancer treatment that attacks immune system cells that feed and protect cancer cells rather than attacking them. The immunotherapy process promises to become a universal treatment for solid tumors. Thus far, the treatment has been tested and shown to work on human tumor cells in the laboratory and animal models.

Frequently asked questions on immunotherapy

The Cancer Research Institute takes a close look at immunotherapy and its potential for treating cancer. Immunotherapy teaches the patient's own immune system to recognize and fight cancer cells. It's especially promising as an alternative for cancers that resist chemotherapy and radiation treatment. Have all your questions about the process and its promise here.

New eye implant clinical trial recruiting in 2026

University of Southern California researchers have launched a phase 2b clinical trial of a stem cell implant designed to restore vision in people with advanced dry macular degeneration. Patients will be randomized to receive the stem cell implant or a simulated procedure and placebo implant. This type of larger, second-stage clinical trial is designed to confirm efficacy and dosing and to look for side effects. It's one of the stages that precedes the US Food & Drug Administration approval of a device.

Scientists using gene therapy recently managed to regenerate retinal cells in the lab—a breakthrough for mammals that could potentially lead to future treatments for human retinal diseases.

NASA began investigating the healing properties of red light therapy in the 1980s, when researchers studying how LED lights could aid in photosynthesis noticed that their hands seemed to heal faster than normal under the lights.

Limb regeneration research may lead to treatments for arthritis

For scientists pursuing the cure for arthritis, the holy grail of discoveries lies in how to regrow healthy cartilage inside human joints. While this feat has so far resisted our best scientific attempts, there are plenty of other human tissues—such as the liver and fingertips—that will successfully regenerate.

As of February 2025, there are more than 150 clinical trials using CRISPR technology

The clinical trials are designed to determine whether experimental CRISPR-related therapies are safe and effective. The majority of trials involve treatments for blood disorders, including beta thalassemia.

View a gallery of images and videos of over 200 types of cells

Funded by the National Institute of General Medical Sciences of the National Institutes of Health, the Cell Image Library houses over 60,000 pieces of media, including microscopic photographs, time-lapse videos of cellular activity, and 3D reconstructions of organic processes.

Why CAR-T therapy has become one of the main focus areas for autoimmune diseases

The therapy, originally targeting cancer, involves taking a patient's own T cells, genetically reprogramming them to recognize and attack a specific target and then infusing them back into patients. For autoimmune diseases, the therapy instead programs them to target immune cells that are causing problems, restoring immune system tolerance.

Minorities are largely underrepresented in medical research, which compromises our understanding of how conditions and treatments may affect various populations, but recent efforts, like the African Ancestry Neuroscience Research Initiative and new genome research studies, are seeking to close that gap.

The difference between embryonic and adult stem cells

The main difference between embryonic and adult stem cells lies in their potential and origin. The former can become any cell type in the body, while the latter are limited to specific cell types. Embryonic stem cells also carry ethical concerns when used in medical or clinical applications.

Red light therapy—a treatment harnessing a specific form of energy that penetrates the top layers of skin—is marketed for skincare and wellness, but has its roots in NASA plant growth research.

A gallery of medical illustrations from NIH

The National Institutes of Health provides educational materials for broad use. The institute hosts a Flickr account with many different image galleries, including this one (with over 70 images to date) that illustrate and annotate curious phenomena, from the life cycles of a malaria parasite to the comparison of male and female bladders. Explore the gallery here.

Explore the National Cancer Institute's archive on cancer treatments, including complementary and alternative medicine.

High-powered red light therapy—delivered directly to nerve fibers via a fiber optic needle—has been used in recent experimental research to suppress pain.

A snapshot of progress in cancer treatments

The American Association for Cancer Research's "Snapshot of a Year of Progress" offers an overview of significant advancements in cancer research and treatment over the past year. Take a quick look at the rapid pace of scientific discovery and the translation of research findings into clinical practice. In 2024 alone, health regulators approved 15 new anticancer therapies alone.

Biohacking to boost personal health has become a concept that encompasses everything from vitamin regimens and cryochambers to brain implants and red light therapy.

Scientific advances are unlocking a wide range of alternatives to IVF

From refined artificial insemination and microsurgical sperm extraction to experimental uterine transplants, scientific innovation is rapidly advancing potential fertilization technologies. But they come along with costly trials, some ethical dilemmas, regulatory issues, and more.

See this 201-level explanation about mitochondria and mitochondrial replacement therapy

This 2022 academic presentation from Stanford University's Dr. Gregory Enns offers a detailed overview about mitochondrial therapy approaches. Enns, a pediatric geneticist, explains how well-functioning mitochondria support the human body.

Neural interfaces offer major medical potential but raise serious ethical concerns

Although brain-computer interfaces can help people recover lost functions and allow direct brain-to-computer communication in the future, surveys have shown little public support for using them to boost abilities like memory or focus in healthy people.

An overview of the National Science Foundation and the technologies it has helped fund

Established in 1950, the federal agency has provided billions of dollars to over 300,000 researchers, entrepreneurs, and students to conduct research across all seven continents. These funds have contributed to major technological achievements, from the invention of the internet to the first image of a black hole.

Researchers are investigating if nanoparticle treatments could combat allergies

In rodent work, Northwestern University’s Evan Scott (now at the University of Virginia) and his colleagues have been trying to attack allergies with specially designed nanoparticles—miniscule lab-made objects that can penetrate cells. They stud the nanoparticles with a tiny bit of the allergen specific to the allergy (i.e. cashew protein for cashew allergy) and antibodies that shut down allergic response processes.

Gene therapy, a hope of the Human Genome Project, has successfully treated a baby born with a rare urea cycle disorder that causes toxic ammonia level buildup.

How personalized gene-editing saved a baby's life

KJ Muldoon had a 50% chance of surviving a genetic mutation until doctors developed a one-of-a-kind gene-editing therapy. In an interview with one of the researchers behind the treatment, learn what it will take to make personalized genetic medicine possible for everyone.

Four frozen embryos in the US were created via 3-parent IVF technique

Columbia University researchers created four embryos using mitochondrial replacement therapy for four female patients who didn't want to pass on mitochondrial diseases to their children. Yet US law restricts the researchers from implanting the embryos so they remain frozen. (Some users may encounter a paywall.)