For many centuries, we have looked to medicine to heal us when we are sick or injured. Major breakthroughs, like vaccines and antibiotics, have improved quality of life, and, in some cases, led to the effective eradication of infectious diseases.
While modern medicine has certainly changed the human experience for the better, we remain at the mercy of disease. There are no vaccines for malaria or HIV, for example. And chronic diseases, like heart disease, Alzheimer’s, diabetes, and osteoporosis, although treatable, are relentless causes of suffering. There are no silver bullet for these conditions. Often the best we can do is manage the symptoms.
One key to changing that may be regenerative medicine, a field of research with its sights set on the root causes of diseases, including many being studied now at the Institute for Stem Cell and Regenerative Medicine (ISCRM).
As a discipline, regenerative medicine combines principles of biology and engineering to develop therapies for diseases characterized by cell depletion, lost tissue, or damaged organs. The broad aim of regenerative medicine is to engineer, regenerate, or replace tissue using natural growth and repair mechanisms, such as stem cells. Organoids, 3D organ printing, and tissue engineering are examples of biopowered technologies used in regenerative medicine.
Many common chronic diseases begin with harmful cell depletion. For example, Alzheimer’s disease is associated with a loss of brain cells, heart disease is often marked by a loss of healthy heart muscle, and type 1 diabetes occurs when cells in the pancreas fail to produce insulin. In the case of cancer, the problem is that cells grow too quickly. (Click here to read more about diseases being researched at ISCRM.)
For scientists, regenerative medicine is a way to fix the root causes of disease by harnessing the body’s natural capacity to repair itself – in other words, to regenerate lost cells and tissue and restore normal functioning. At the Institute for Stem Cell and Regenerative Medicine, researchers are studying how to jump start the growth of cells in the brain, heart, pancreas, liver, kidney, eyes, ears, and muscles.
Ultimately, the goal of regenerative medicine is to improve the daily wellbeing of patients with debilitating chronic diseases by developing a new generation of therapies that go beyond treating symptoms.
Stem cells are powerful tools of discovery used by researchers hoping to understand how regenerative medicine could be used to treat patients. Right now, ISCRM researchers are using stem cells to study how heart diseases develop, testing stem cell-based therapies that could regenerate damaged or lost heart tissue, and even launching heart tissue into space to study the effects of microgravity on cardiovascular health. Many ISCRM scientists use stem cells to create 3D organ models, known as organoids, that allow them to study diseases and test regenerative treatments without involving animals or human subjects.
Researchers in multiple ISCRM labs are pursuing novel approaches that can potentially cure rather than manage heart disease. In 2018, a study led by ISCRM Director Dr. Charles Murry demonstrated that stem cell-derived cardiomyocytes have the potential to regenerate heart tissue in large non-human primates, a major step toward human clinical trials. In another investigation, ISCRM faculty members Jen Davis, PhD and Farid Moussavi-Harami, MD are developing new tools to help cardiologists design personalized treatments for certain heart diseases.
ISCRM researchers are studying the mechanisms that regulate the development and function of beta cells in the pancreas that produce insulin – a key to future treatments for any type of diabetes. Vincenzo Cirulli MD, PhD, is screening for biological factors that could promote the growth of beta cells necessary for insulin production. Dr. Cirulli’s ISCRM colleague Laura Crisa MD, PhD is using a “disease-in-a-dish” model to study how islet cells falter and whether they can be regenerated, and eventually transplanted, into patients.
Researchers at the Institute for Stem Cell and Regenerative Medicine (ISCRM) are using stem cell-derived retinal organoids to study how diseases of the retina form and how they can be treated. Organoids closely approximate human tissue without many of the ethical questions and supply limitations that complicate the use of fetal tissue. Read more about recent efforts to validate stem cell-derived organoids as disease models here.
In an approach could someday be used to help repair the retinas in patients who have lost vision due to macular degeneration, glaucoma and diabetes, the Reh Lab has successfully induced non-neuronal cells to become retinal neurons. In an October 2021 study published in the journal Cell Reports, Reh and his team using proteins (known as transcription factors) that regulate the activity of genes to induce glial cells in the retina to produce neurons. The effort demonstrates that gene therapy could someday be used in clinics to help repair damaged retinas and restore vision.