Prior to 2005, the stem cell field was virtually nonexistent. Stem cells had recently been discovered as a powerful tool for understanding disease and developing new treatments, but the field lacked the funding and talent needed to capitalize on this technology. Because of this, Susan L. Solomon launched The New York Stem Cell Foundation with the mission of using stem cell research to accelerate treatments and cures for the major diseases of our time. This catalyzed the growth of the field, which now includes thousands of scientists worldwide conducting groundbreaking research across many diseases.
NYSCF’s primary goal has always been to accelerate new treatments and cures for the major diseases of our time, and we have seen many exciting therapies made possible by NYSCF moving into the clinic, including 21 currently in clinical trials. These successes demonstrate the power of stem cells for bringing about inventive new treatments, and we look forward to seeing many more move into the clinic soon.
Mitochondrial Replacement Therapy
A team of scientists from NYSCF and Columbia University Medical Center developed a technique called mitochondrial replacement therapy that prevents the inheritance of devastating mitochondrial diseases in children. With this technique, published in Nature, mitochondria carrying disease-causing mutations in a mother’s egg cell can effectively be replaced by healthy mitochondria, preventing her offspring from inheriting fatal mitochondrial diseases. Mitochondrial replacement therapy is currently used in the United Kingdom, where it is preventing children from inheriting potentially fatal diseases.
A Cell Therapy for Macular Degeneration
Our 20/20 clinical trial is a cell therapy that will provide a lasting cure for age related macular degeneration (AMD). This pioneering stem cell therapy will use a patient’s stem cells to create healthy retinal cells that then replace those lost to AMD and restore vision. We are leading this effort and working in collaboration with world-renowned retinal surgeons including Dr. Stanley Chang at Columbia University and stem cell scientists including Dr. Kapil Bharti at the National Eye Institute at the National Institutes of Health. This builds on pioneering work by NYSCF – Robertson Stem Cell Prize recipient Dr. Pete Coffey, whose clinical trial of a stem-cell-based AMD therapy has improved vision in two AMD patients. Construction is now complete at the NYSCF Research Institute for a state-of-the-art “good manufacturing practice” (GMP) facility in which we can create cells that are safe for transplantation into patients.
Discovering a New ALS Drug
An epilepsy drug, initially tested using a stem cell model of amyotrophic lateral sclerosis (ALS) developed with NYSCF support (which was named Time and Science Magazine’s #1 medical breakthrough of the year), has successfully reduced motor neuron excitability – a hallmark of the disease – in 65 ALS patients participating in a phase 2 clinical trial. This work, led by NYSCF Scientific Advisor Dr. Kevin Eggan and NYSCF – Robertson Stem Cell Investigator Dr. Brian Wainger, is the first clinical trial to result from a drug discovered in a stem cell model, setting a precedent for countless future discoveries.
Clinical Trials for Cancer and Parkinson’s Disease
NYSCF – Robertson Stem Cell Investigator Alumnus Dr. Ravi Majeti of Stanford University, is conducting clinical trials to test a class of cancer therapies called anti-CD47 treatments. These drugs strip away camouflaging agents in leukemia-affected cells, helping the immune system target them more effectively.
Additionally, NYSCF – Robertson Stem Cell Investigator Alumna Dr. Malin Parmar of Lund University has spent several years working with the pharmaceutical company Novo Nordisk on a cell therapy for Parkinson’s disease that aims to replace lost brain cells with healthy ones derived from a patient’s own stem cells.
The backbone of our mission lies with using stem cells to advance disease research, and our scientists have made many important discoveries that are paving the way for brighter futures for patients everywhere.
The First Personalized Embryonic Stem Cell Model of Disease
We created the first-ever disease-specific stem cell line using somatic cell nuclear transfer (SCNT). Our scientists used SCNT to create embryonic stem cells from a patient with type 1 diabetes. This work marked a major step forward in the effort to use stem cells to understand the development of, and eventually treat, a range of chronic diseases affecting tens of millions of people.
Creating All Cell Types in the Brain from Stem Cells
To understand brain diseases such as Alzheimer’s, Parkinson’s, and multiple sclerosis, it is important that scientists can examine all cell types in the brain. NYSCF’s Dr. Valentina Fossati has pioneered methods for creating a category of brain cells called glia – support cells in the brain that likely play significant roles in disease but are often overlooked in research and drug development. Dr. Fossati’s protocols for making glia (such as astrocytes, oligodendrocytes, and microglia) are now used by scientists around the world, accelerating research into how inflammation and the immune system drive neurological diseases and opening the door for entirely new classes of therapies.
Personalized Bone Grafts from Stem Cells
In a step towards personalized bone grafts to treat traumatic injury or congenital defects, NYSCF scientists led by Dr. Giuseppe Maria de Peppo discovered in 2013 how to create patient-specific bone substitutes from skin cells for repair of large bone defects. This advance facilitates the development of customizable, three-dimensional bone grafts on-demand, matched to fit the exact needs and immune profile of patients whose bodies may otherwise reject synthetic grafts or require frequent surgeries to get them replaced.
Disease Modeling and Drug Testing
NYSCF scientists have established methods for advanced cell manipulation, allowing us to create models of diseases that affect various human tissues, including the lungs, pancreas, bone, and more. Because of this, we can address a variety of diseases including rare and recently emerging diseases like COVID-19, for which our researchers are creating lung cells to study infection. The cells NYSCF creates can also serve as material for drug testing and toxicity evaluations, ensuring that the safest, most effective drugs are advanced toward the clinic.