Cedars-Sinai Investigators Confirm Safety of Therapy Targeting Motor Neurons That Die in Patients With ALS
For the first time, an investigational therapy using stem cells and a protective protein has been shown to be safe in humans. The therapy targets motor neurons that die in patients with ALS. The treatment, which investigators developed at Cedars-Sinai, could potentially protect diseased motor neurons in the spinal cord of patients with amyotrophic lateral sclerosis (ALS), a fatal neurological disorder known as Lou Gehrig’s disease. In the trial, which was conducted using human cells in a lab dish and then again in a small group of patients with ALS, no serious side effects were observed. This is an important step forward in developing a potential new treatment for this devastating disease. The findings were published in the peer-reviewed journal Nature Medicine.
“We’re excited that we proved the safety of this approach, but we need more patients to really evaluate efficacy, which is part of the next phase of the study,” said Johnson, who is also the vice chair of Neurosurgery at Cedars-Sinai. “Proving that we have cells that can survive a long time and are safe in the patient is a key part in moving forward with this experimental treatment.” ―From Cedars-Sinai
Pablo Avalos, MD, Robert Baloh, MD, Ph.D., previously a professor of Neurology at Cedars-Sinai and now global head of neuroscience at Novartis, and J. Patrick Johnson, MD, co-medical director of the Spine Center at Cedars-Sinai, are co-lead authors on the publication.
“Using stem cells is a powerful way to deliver important proteins to the brain or spinal cord that can’t otherwise get through the blood-brain barrier,” said senior and corresponding author Clive Svendsen, PhD, professor of Biomedical Sciences and Medicine and executive director of the Cedars-Sinai Board of Governors Regenerative Medicine Institute. “We were able to show that the engineered stem cell product can be safely transplanted in the human spinal cord. And after a one-time treatment, these cells can survive and produce an important protein for over three years that is known to protect motor neurons that die in ALS.” ―From Cedars-Sinai
The engineered cells could be a powerful therapeutic option for those suffering from the disease that robs people of their ability to move and breathe.
The study used stem cells that were designed in Svendsen’s laboratory to produce a protein called glial cell line-derived neurotrophic factor (GDNF). This protein can help protect motor neurons, cells that send signals from the brain or spinal cord to a muscle so it can move.
“GDNF on its own can’t get through the blood-brain barrier, so transplanting stem cells releasing GDNF is a new method to help get the protein to where it needs to go to help protect the motor neurons,” said Pablo Avalos, MD, co-lead author on the paper and associate director of Translational Medicine in the Cedars-Sinai Board of Governors Regenerative Medicine Institute. “Because they are engineered to release GDNF, we get a ‘double whammy’ approach where both the new cells and the protein could help dying motor neurons survive better in this disease.” ―From Cedars-Sinai
Dr. Terrence Kim, Dr. Doniel Drazin, and Dr. J. Patrick Johnson
Everything Cool in Spine Surgery
Please join us for the 8th Annual SSF Robotics Course, “Everything Cool in Spine Surgery,” scheduled for Saturday, December 9, 2023, at the Seattle Science Foundation in Seattle, WA. This event promises to be an enlightening experience for professionals in the fields of neurological and orthopaedic surgery, with nationally recognized speakers focusing on provocative topics and in-depth debates regarding spinal navigation and robotics application in spine surgery.
Innovative Topics: Dive into the expanding frontiers of technology-assisted surgery, the role of artificial intelligence in pre-operative planning, and much more.
Hands-on Demos: Gain practical experience with demonstrations including navigated lumbo-pelvic fixation and robotic-assisted surgeries.
Engaging Discussions: Participate in debates about the latest trends in spinal navigation, robotics, imaging, and MIS techniques.
Networking Opportunities: Connect with peers and leaders in the field.
Details
Date & Time: Saturday, December 9, 2023, from 7:00 AM to 5:00 PM.
Dr. Johnson (left), Professor Alan Crockard (center), and Professor Adrian Casey.
Dr. Johnson was recently a guest of Professor Alan Crockard and Professor Adrian Casey of the National College of Neurology and Neurosurgery at Queen’s Square in London, England.
Dr. Johnson had studied under Professor Crockard, who was Chief of Neurosurgery in the 1990s in London at Queen’s Square and also studied at the National Pediatric Hospital at Great Ormond Street in London.
Dr. Adrian Casey is now the Chief of Spinal Neurosurgery at Queen’s Square and a longtime professional colleague of Dr. Johnson, having been on teaching and research programs focused on artificial disc technology and caring for patients around the globe.
Dr. Johnson and Visiting Prof and longtime friend and colleague Dr. Alex Vaccaro.
Dr. Vaccaro was recently a Visiting Professor with us at Cedars Sinai and lectured at the Spine Center Grand Rounds on the current treatment of spinal trauma, of which he is a renowned authority. He and Dr. Johnson have been close professional colleagues over their entire careers.
Dr. Vaccaro has been the President of Rothman Orthopedic Institute since 2014 and Professor of Orthopedics at Thomas Jefferson University in Philadelphia. His spinal care leadership and clinical research excellence earned him the Leon Wiltse award from the North American Spine Society (NASS) and past presidencies at prominent spinal and orthopaedic associations. Currently, he sits on the American Academy of Orthopaedic Surgeons (AAOS) board. Dr. Vaccaro also co-directs the Regional Spinal Cord Injury Center of the Delaware Valley.
J. Patrick Johnson, MD is a renowned neurosurgeon specializing in spinal disorders and has served as the Director of the Institute for Spinal Disorders at Cedars Sinai Medical Center since 2001 and previously as the Director of the UCLA Comprehensive Spine Center from 1993-2001.
Dr. J. Patrick Johnson, alongside orthopaedic oncology expert Dr. Earl Brien, is conducting a computer-guided resection of an abnormal lesion located in the coccyx region, at Cedars-Sinai Medical Center. This procedure showcases the integration of advanced technology in modern surgical practices to enhance precision and patient outcomes.
J. Patrick Johnson, MD is a renowned neurosurgeon specializing in spinal disorders and has served as the Director of the Institute for Spinal Disorders at Cedars Sinai Medical Center since 2001 and previously as the Director of the UCLA Comprehensive Spine Center from 1993-2001
The information provided on this website should not be used to diagnose or treat any medical condition and is not meant to take the place of professional medical advice. If you think you have a medical problem, please seek the advice of a physician. Call 911 for all medical emergencies.
A recent study published in the journal Neurology reveals concerning findings about the long-term effects of concussions, even when suffered only once, on cognitive health in individuals. The results indicate that concussions may be linked to increased cognitive decline later in life, regardless of whether the individual seemed to have fully recovered from the injury.
Study Overview
The research followed twins who had served during World War II. An important highlight of this study was that many of these twins were identical. The genetic makeup of identical twins is almost the same, allowing for an exceptional opportunity for medical comparison. By studying one twin who had experienced a concussion and comparing their cognitive progression to their twin who hadn’t, scientists could observe the closest possible “what if?” scenario in medicine.
Why This Study is Significant
Control for Genetic Variability: Given that identical twins share the same genetic code, the study effectively controls for genetic variations. This means that differences observed between the twins (one with a concussion and the other without) are more likely attributed to the injury itself rather than genetic predispositions or other external factors.
A Window into Long-Term Effects: Although concussions and their immediate effects have been extensively researched, there is still a gap in our understanding of their long-term consequences. This study provides essential insights into the potential lasting effects of concussions on cognitive health, which may manifest years or even decades after the injury.
Implications for Clinical Practice: Understanding the long-term risks associated with concussions can guide clinicians in developing effective treatment and monitoring strategies. Patients who have experienced concussions may benefit from regular cognitive assessments throughout their lives to detect and address potential issues early on.
Key Takeaways
Even a single concussion can potentially have long-term consequences on an individual’s cognitive health.
Those who seem to have fully recovered from the immediate symptoms of a concussion are not necessarily free from the long-term risks.
Further research is essential to determine the exact mechanisms through which concussions impact cognitive health and to develop interventions that may mitigate these risks.
Concussions, though often seen as mild and temporary injuries, might carry with them long-term implications that we are only beginning to understand fully. This research underscores the importance of timely diagnosis, careful monitoring, and ongoing research to further our understanding of concussions and their potential lasting impacts on the brain.
Dr Johnson on sidelines at recent LA Rams game as NFL concussion consultant at halftime with new fellows Drs Cameron Murphy and Karim Shafi .
J. Patrick Johnson, MD is a renowned neurosurgeon specializing in spinal disorders and has served as the Director of the Institute for Spinal Disorders at Cedars Sinai Medical Center since 2001 and previously as the Director of the UCLA Comprehensive Spine Center from 1993-2001. Dr. Johnson is a well-known concussion and sports medicine expert and a sideline concussion doctor with the NFL.
The information provided on this website should not be used to diagnose or treat any medical condition and is not meant to take the place of professional medical advice. If you think you have a medical problem, please seek the advice of a physician. Call 911 for all medical emergencies.
