As discussed in our previous blog “Ten Companies with Innovative Stem Cell Therapies in Clinical Trials – Part I,” we have reviewed ten companies that have made significant progress with stem cell therapies in clinical trials. In part I, we reviewed Advanced Cell Technology, Neostem, Aastrom, Pluristem and Athersys. In part II we will continue the review with five more companies. Our purpose is to give an overview of stem cell therapies in clinical trials, the types of diseases they offer treatments for, the technologies they employ, and the companies who are bringing these products to market. StemCells Inc. StemCells Inc. has two stem cell therapies in clinical trials. The first product is a treatment for Spinal Cord Injury currently in Phase I/II clinical trials in Switzerland. The product targets patients that have suffered an injury to their spinal cord and have reached a plateau in their recovery. There are 1.3 million Americans who suffer from Spinal Cord Injury.

Their second product is for Pelizaeus-Merzbacher Diseases (PMD) and is currently in Phase I clinical trials in the United States. PAD is a disorder cause by a genetic mutation that results in impaired growth of the myelin sheath (the fatty substance that covers and protects nerves in the brain). It is a rare disease that affects young children and causes a deterioration of neurological function. In milder cases the symptoms are poor coordination, motor control, and intellectual function, but in the more severe cases symptoms are more severe and can be fatal. There is currently no cure for the disease.

StemCells Inc. uses human neural stem cells in their allogeneic treatments. After harvest these cells are purified and expanded in culture, then they are cryopreserved until needed for treatment. Cells are manufactured in a cGMP compliant facility. In the Spinal Cord Injury trial, cells are injected into the spinal cord and in the PMD trial they are transfused into the patient. Bioheart Bioheart’s lead product, Myocell, is currently in Phase II/III clinical trials. The treatment is injected after heart attack to improve cardiac function. Bioheart uses muscle stem cells (myoblasts) and have announced a next generation version of this product called Myocell SDF-1. Myocell SDF-1 is similar to Myocell, but the cells have been modified to express angiogenic proteins to enhance blood vessel formation.

Bioheart manufactures its autologous Myocell by harvesting a small piece of thigh muscle from the patient, then isolating and expanding the myoblasts. The cells are then injected into the patient’s scar tissue in the heart. The procedure uses an endoventricular needle-injection catheter and is minimally invasive. Osiris Osiris’ lead product is Prochymal. Prochymal is currently part of several clinical trials for various disease states. Prochymal for treatment of Graft vs. Host Disease (GvHD) has been approved in Canada and New Zealand and is currently in Phase III clinical trials in the United States. It has received Fast Track review from the Food and Drug Administration (FDA). GvHD can occur during stem cell or bone marrow transplants. The immune cells present in the transplant view the cells in the patient receiving the transplant as foreign and begin to attack the patient’s tissues and organs. GvHD can cause serious disability and can even be fatal. It is a frequent complication and occurs in about half of the transplants.

Prochymal is also being evaluated in Phase II clinical trials for treatment of Crohn’s Disease, Type 1 Diabetes, Acute Myocardial Infarction and Pulmonary Disease. For treatment of Chron’s Disease, Osiris has received Fast Track status from FDA. Crohn’s disease is an inflammatory disease that affects the colon and small intestine. This disease can cause abdominal pain, weight loss, vomiting and diarrhea.

In Type 1 Diabetes, also known as juvenile diabetes, the immune system attacks islet cells that produce insulin to regulate the level of glucose in the blood, the result is high levels of glucose in the blood that can be very dangerous or deadly. Prochymal is currently being evaluated for its ability to reverse the cause of the underlying disease. Prochymal is also being evaluated for its ability to reduce scar formation and aid in restoring heart function after Acute Myocardial Infarction (heart attack).

Osiris also is in Phase II clinical trials of Prochymal for Chronic Obstructive Pulmonary Disease (COPD). COPD is a disease of the lung, which limits airflow in the airway. It includes both emphysema and chronic bronchitis and affects 12 million Americans. It is the fourth leading cause of death in the United States and there is no cure.

Under FDA’s animal rule, Prochymal is being evaluated as a treatment for Acute Radiation Syndrome in Phase III clinical trials. FDA’s animal rule allows animals to be used in place of humans in circumstances where human clinical trials are not possible for ethical or safety reasons.

Osiris also has the product Chondrogen, which is being evaluated in Phase II clinical trials for its ability to regenerate meniscus and for the prevention of osteoarthritis in the knee. Meniscus is a C-shaped cartilage disc that cushions the knee joint and helps to keep the knee steady by balancing weight in the knee.

Osiris’s Prochymal and Chondrogen are allogenic therapies made up of mesenchymal stem cells derived from donor bone marrow. The cells are isolated and expanded and Osiris states that one donation can yield 10,000 doses. Doses are preserved for storage, after which, they have a two-year shelf life and are delivered to the patient when needed. Cells are infused into the patient and treatment takes thirty minutes to infuse one dose. Neuralstem Neuralstem has a treatment for Amyotrophic Lateral Sclerosis (ALS) in Phase I clinical trials in the United States and in a Phase I/II clinical trial in Mexico. ALS (also known as Lou Gehrig’s Disease) is a fatal neurological disease that progresses rapidly attacking the nerve cells responsible for controlling voluntary muscles. Eventually patients lose the ability to move limbs and the ability to breathe without ventilation. Patients ultimately die of respiratory failure and there is no cure. In the United States 20,000-30,000 people have ALS and approximately 5,000 new cases are diagnosed each year.

Neuralstem uses human neural stem cells that are injected directly into the grey matter of the patient’s spinal cord in the cervical and lumbar regions. In order to deliver their therapy correctly, Neuralstem had to develop a specialized surgical device. Neuralstem states “Neuralstem’s proprietary Spinal Cord Delivery Platform and Floating Can­nula were designed specifically by Neuralstem’s ALS trial neurosurgeon, Nicholas M. Boulis, MD, for the world’s first intraspinal delivery of stem cells.” Mesoblast Mesoblast’s lead product is a treatment for Congestive Heart Failure, which affects about 6.2 million Americans with 670,000 new cases diagnosed annually. Congestive Heart Failure causes patients to have an enlarged heart and insufficient blood flow to the extremities. Mesoblast’s product called Revascor is designed to improve heart muscle function in moderate to severe cases and is in Phase III clinical trials.

Mesoblast also has a treatment for early Type 2 Diabetes in Phase II clinical trials. Type 2 Diabetes (also called adult-onset) is responsible for 90-95 percent of diabetic cases. According to the American Diabetes Association, 25.8 million people in the United States have diabetes and 1.9 million new cases were diagnosed in 2010. With Type 2 Diabetes the body does not produce enough insulin or the cells don’t respond to insulin, resulting in a build up of glucose in the blood. When glucose levels get elevated complications can set in. Complications include, heart attacks, high blood pressure, blindness, kidney disease, nervous system disease and amputation. The aim of the Mesoblast treatment is to enhance the ability of pancreatic beta cells to produce more insulin and thus lower blood glucose levels.

Mesoblast has an orthopedic product for Degenerative Disc Disease (DDD) that has completed a Phase II clinical trial. DDD is the degeneration of intervertebral discs in the spine. Disc degeneration happens as part of aging, but for some people the degeneration is more severe and results in severe and chronic pain, primarily in the lower back, and can result in disability. Mesoblast states that DDD “affects between 15 and 45 percent of the population and often results in spinal surgery.’ The company has completed a Phase II clinical trial for a treatment that injects adult mesenchymal precursor cells into a degenerated disc resulting in a less invasive therapy alternative to surgery.

Mesoblast allogenic stem cell treatment consists of adult mesenchymal precursor cells derived from bone marrow or other tissue sources. Mesoblast has contracted with Lonza for cGMP manufacturing of their mesenchymal stem cell product.