Icelandic Cancer Patient Receives a Completely New Windpipe Made From His Own Stem Cells

Andemariam Beyene, an African cancer patient from Eritrea had made his peace with his life, and talked about his impending death. Doctors found a golf ball-sized tumor growing in his windpipe, and despite several treatments with radiation and surgery to remove it, it kept growing. He was in pain, and out of options.

As a last resort, he went to the Karolinska Instute in Stockholm, Sweden to visit Dr. Paolo Macchiarini, who wanted to make a new windpipe for Mr Beyene from his own cells and spun plastic. This seemed like the medical equivalent of a “Hail Mary” pass in football, but it seemed worth a shot.

There is a precedent for such a procedure, since between 2008 and 2011, Dr. Macchiarini fitted nine people with new tracheas. These tracheas were built from the patient’s own stem cells that were grown on scaffolds made from tracheas that had been completely stripped of all their cells. These “decellularized” scaffolds work well, but they have one large drawback: .they required the existence of a pre-existing trachea from a human or an animal. Therefore, for Mr. Beyene, Dr. Macchiarini used a different procedure that built the scaffold from scratch out of plastic.

For this procedure, a mold of Mr. Beyene’s trachea was made from porous, fibrous plastic. This mold was then seeded with bone marrow stem cells and grown in a bioreactor. The bioreactor contained culture medium that turned much like a rotisserie. After one day and a half of growth in the bioreactor, the trachea was implanted into Mr. Beyene’s body.

Even though this procedure had been successful performed in pigs, it had never been tried on a human. Mr. Beyene was convinced by Dr. Macchiarini to give it a try. Now, the 39-year old African who lives in Iceland is back with his wife and children, getting to know people he thought he would never know. His strength is improving every day and can even run a little.

The synthetic scaffold used for Mr. Beyene’s windpipe was made by scientists at University College London. This mold was made to exact specifications so that it would perfectly fit inside Mr. Beyene’s chest. Then stem cells from his bone marrow were cultured in the laboratory and dripped with a pipette over the scaffold, in the same way that you baste a turkey. Then this whole thing is grown in a bioreactor that circulated fresh culture medium at regular intervals while spinning the scaffold and the cells. Macchiarini is quite sure that the original cells that are seeded onto the scaffold are dead. He suspects that the dying stem cells leave a host of chemicals in their stead that summon other stem cells from the bone marrow to come and seed the scaffold.

The windpipe is lined with several different types of cells. Some of them secrete mucus, which serves as a kind of adhesive tape for dust particles and microorganisms that are inhaled. The mucus is then moved to the top of the throat by a host of cells with hair-like extensions that move back and forth like oars on a Viking boat. At the top of the throat, the mucus is swallowed and destroyed in the stomach. Also, the windpipe is well endowed with blood vessels to feed the tissues oxygen and nutrients from the bloodstream.

Mr. Beyene’s windpipe was found to contain some mucus-producing cells at fives months after the surgery. At follow-up, it was also clear that his windpipe bled when nicked. Mr. Beyene hopes to return to Eritrea some day, but for now he will stay in Iceland, since he is close to Stockholm for his regular check-ups. He had some scar tissue from the implant that was impeding his breathing, but that has been removed. He still needs regular check-ups, but he is doing well.

Five months after implanting Mr Beyene with his engineered windpipe, Dr. Macchiarini implanted an American patient named Christopher Lyles with a tissue engineered windpipe made from a plastic scaffold and his own stem cells. Mr. Lyles’ windpipe was made with an improved type of scaffold fabricated from smaller plastic fibers. Mr. Lyles returned home to Maryland, by died in March of 2012. While the cause of death has not been released, Dr. Macchiarini has been told that his implant was not the cause of his death and that up until the time of his death, it had been functioning normally. Dr. Macchiarini has also implanted engineered windpipes into two Russian patients who have been discharged fro the hospital and are doing well.

While these procedures are remarkable feats of stem cell biology, tissue engineering and medical intervention, we must admit that these techniques are still experimental and are hugely expensive (half a million dollars per procedure). Dr. Macchiarini’s dream it to some day design drugs that induce the stem cells to build a new trachea within the patient from the inside out. This way, no surgery is required and the patient would have a rebuilt windpipe.

FDA Approves Clinical Trial that Used Cord Blood to Treat Autism

The Sutter Neuroscience Institute in Sacramento, California has announced its collaboration with the Cord Blood Registry, the world’s largest stem cell bank in what promises to be the first FDA-approved clinical trial to assess the use of a child’s own cord blood stem cells to treat selected patients with autism. This placebo-controlled study is the first of its kind and will evaluate the efficacy of cord blood stem cells to help improve language and behavior in autistic children.

The Centers for Disease Control (CDC) places the frequency of autism in the US as one in 88, but for boys, the rate is even higher (1 in 54). Autism, today, is part of a series of conditions that are collectively defined as autism spectrum disorders (ASPs). ASPs include individuals with very different symptoms, and include everything from autistic disorder (also known as classic autism, Asperger syndrome and pervasive developmental disorder not otherwise specified (also known as atypical autism). These conditions are thought to have multiple risk factors that include genetic, environmental and immunological components.

With regard to this study, Michael Chez, M.D., director of Pediatric Neurology with the Sutter Neuroscience and principal study investigator of this clinical trial said: “This is the start of a new age of research in stem cell therapies for chronic diseases such as autism, and a natural step to determine whether patients receive some benefit from an infusion of their own cord blood stem cells. I will focus on a select portion of children diagnosed with autism who have no obvious cause for the condition, such as known genetic syndromes or brain injury.”

This clinical study will enroll 30 children between the ages of two and seven who have been diagnosed with autism, and meet all the criteria for inclusion in the study. Over the course of this clinical trial, all enrolled participants will receive two infusions over the course of 13 months. One of the infusions will contain the child’s own cord blood stem cells, and the other infusion will contain a placebo. The participants and the lead investigators will not know the content of each infusion. To ensure the highest quality and consistency in cord blood stem cell processing, storage and release for infusion, Cord Blood Registry is the only family stem cell bank that provides umbilical cord blood units from clients for the study.

A newborn’s umbilical cord blood contains several unique populations of stem cells. Scientists and physicians have been used for more than 20 years in medical practice to treat certain cancers, blood diseases and immune disorders. When patients undergo a stem cell transplant for such conditions, the umbilical cord blood stem cells effectively rebuild the blood and immune systems.

According to Dr. Chez, “A focus of my research has been the complex relationship between a child’s immune system and central nervous system. We have evidence to suggest that certain children with autism have dysfunctional immune systems that may be damaging or delaying the development of the nervous system. Cord blood stem cells may offer ways to modulate or repair the immune systems of these patients which would also improve language and some behavior in children who have no obvious reason to have become autistic. The study is similar to other FDA-approved clinical trials looking at cord blood stem cells as a therapy for cerebral palsy.”

Heather Brown, vice president of scientific & medical affairs at Cord Blood Registry, said: “It’s exciting to partner with thought-leading medical researchers and clinicians, like Dr. Chez, who are pursuing a scientifically-sound approach in evaluating new therapeutic uses for cord blood stem cells for conditions that currently have no cures. Families who made the decision to bank their stem cells to cover the unknowns and what ifs in life are gaining access to this and other important clinical trials while playing an important role in the advancement of science.”

A co-investigator of the study is Michael Carroll, M.D., who is the medical director of the Blood and Marrow Transplantation and Hematological Malignancies Program at Sutter Medical Center, Sacramento. According to Dr. Carroll, “There is a vast amount of unchartered territory when it comes to how stem cell therapies may help patients living with these conditions. I’ve seen how stem cell therapy has changed my field of medicine and how I care for my blood cancer patients. I am eager to see how our work can open new doors for patients and families dealing with autism.”