stem cell research

imagine driving your car along your typicalcommute, when suddenly you are in an accident. waking up in the hospital, you find out thatyour spinal cord was damaged. immediately, everyday tasks like walking,eating, and taking a shower become huge challenges. patients with spinal cord injuries can losethe ability to move their legs and upper extremities and even lose their sense of touch. but that’s not all: these patients oftensuffer from pain, ulcers, bowel issues, difficulty breathing, depression and an overall reducedlife expectancy. a team of scientists and clinicians at asteriasbiotherapeutics is currently conducting a clinical trial in patients with spinal cordinjuries, with the goal of restoring some

of the lost motor function and sensation,and, as a result, improving the quality of these patients’ lives. before we jump in, let’s understand whathappens in spinal cord injury. the spinal cord is a big bundle of nervesand supporting cells, including specialized nerve cells called motor neurons, which areresponsible for movement. nerves connect our brains to our bodies bycarrying sensory and motor signals from the brain, down the spinal cord, and to the musclesthroughout the body. the information is transmitted across largedistances through a part of the neuron called the axon, which acts like a wire.

axons are protected by a material called myelin,which you can think of as the insulation around the wire. this insulation helps the nerve signals moverapidly down the spinal cord to the muscle to allow for movement. when the spinal cord is injured, two thingscan happen: 1) the nerve axons can get damaged, blocking the nerves from sending importantmotor and sensory signals, and 2) the myelin insulation can also break down, leaving thenerves exposed and unable to function normally. the asterias team has begun a human clinicaltrial to test a new experimental therapy - called ast-opc1 - to see if it can be effective inrestoring function back to patients who have

suffered severe spinal cord injuries. because the major damage in these types ofinjuries is done to the nerves, and to their protective myelin insulation, the scientistsare tackling this first. their approach uses a cell type known as oligodendrocyteprogenitor cells (opcs). these opcs are made from human embryonic stemcells, which can become any type of cell in the body. the stem cell-derived opcs are introduceddirectly into the spinal cord at the site of the injury, where they can help repairdamaged nerves and can produce more myelin in the spinal cord.

this therapy is exciting because it utilizesa type of cell similar to one found naturally in the body, and harnesses its normal functionto reinsulate and repair the nerves damaged in spinal cord injury. with support from the introduced opcs, damagednerves at the sites of injury might recover their functional abilities to conduct motorand sensory signals between the brain and the hands, arms and fingers—and, hopefully,reverse some of the devastating paralysis that affects many patients with spinal cordinjuries. by utilizing the latest stem cell based technologies,scientists are hoping to significantly improve the quality of life of spinal cord injurypatients and restore their ability to interact

with the world around them! so far, asterias’ animal studies, and theirongoing human clinical trial, have shown promising results: safe implantation of the cells andas a result increased motor function. but it is important to remember these resultsare early, and this stem cell based therapy must be tested further. with the successes we will celebrate, therewill inevitably also be setbacks and obstacles along the way. however, even setbacks will provide learningopportunities for further scientific and medical exploration that will bring us closer to curingor mitigating devastating conditions such

as severe spinal cord injury. as we build on the progress enabled by california’sproposition 71 and the funding agency it created, the california institute of regenerative medicine,we must keep the momentum going: we are now at the point of seeing, for the first time,clinical research results in human patients with devastating conditions such as severespinal cord injury, and we can make even more progress as we learn from these ongoing studies. only with continued support for biomedicalresearch can we increase our understanding of the therapeutic potential of stem cellsand translate that understanding into meaningful treatments that help give patients their livesback.