Q Therapeutics, Inc., a developer of clinical-stage cell therapies for central nervous system (CNS) diseases announced that its Q-Cells® product (human glial restricted progenitor cells and their progeny) has received the FDA’s orphan drug designation for the treatment of transverse myelitis.
“Orphan drug designation of Q-Cells for transverse myelitis is a great step toward our goal of advancing glial cell therapy for demyelinating and neurodegenerative diseases,” said Steven Borst, President and CEO of Q Therapeutics, Inc. “People who are hit hardest by transverse myelitis face a lifetime of paralysis and wheelchair confinement. Building upon the remarkable, disease-modifying results that have been achieved in pre-clinical studies, we aim to someday reverse that paralysis, not only in these patients, but all who have disabilities that result from damaged or destroyed myelin.
Last year, collaborators at The Johns Hopkins University showed that Q-Cells replaced missing myelin in mice, restoring the animals to normal function and life expectancy. These robust data supported swift FDA clearance of Q Therapeutics’ proposed TM clinical trial. Similar experiments in animal models of spinal cord injury also have demonstrated the restorative capabilities of Q-Cells. For the complete article, click here
(This is a summary of the original communications. For the complete article, click here https://bit.ly/2OqwCEn }
1.Who is conducting this study? The University of Texas Southwestern, Q Therapeutics, Inc., and The Transverse Myelitis Association are collaboratively sponsoring the first human safety study to treat central nervous system (CNS) disease.
2.What are Q-Cells®? Q Therapeutics developed a glial-restricted precursor cell, called a Q-Cell, that develops into oligodendrocytes. Oligodendrocytes produce myelin, the insulation around nerves, and other factors that are necessary for healthy CNS function. In mice born with nerve cells without myelin, Q-Cells have been shown to produce myelin and other factors that restored the mice to normal function.
3.What is the difference between these cells and stem cells? Stem cells are undifferentiated cells that can turn into other types of cells. Q-cells are not stem cells because they can only turn into oligodendrocytes and astrocytes, the support and repair cells of the CNS.
4.How is this study being funded and what are these funds going to be used for? The cost of the Phase I study is approximately $2.5 million. The CONQUER program at The University of Texas Southwestern secured a $1.5 million gift towards funding of the Phase I trial. The TMA through The Pauline H. Siegel Eclipse Fund and Q Therapeutics, Inc. have committed to raise the remaining funds.
5.Who can participate, and why were these inclusion criteria picked? This study is enrolling patients diagnosed with transverse myelitis who are between one and ten years from their event and remain unable to walk.
6.How can someone sign up to participate? Screening is scheduled to begin by the end of 2018. A link to the web site will be provided at that time.
7.How many people will participate? The Phase I trial will include nine non-ambulatory adult transverse myelitis patients.
8.How are the cells being used? The Q cells will be surgically implanted into the spinal cord at the level of a lesion. Participants will be followed for both safety assessments and multiple measurements to determine if the cells are inducing any level of repair.
9.What are the outcomes the study will look at? As a Phase I study, the majority of outcomes in this study are focused on assuring the safety of this treatment modality. We will look for any complications from surgery, evidence of immune rejection of the cells or abnormal growth of cells.
10.Are there any risks to participating? There are always risks to participating in research and those are greatest in Phase I studies, based on our limited experience.
11.What would cause the study to end early? Studies can be stopped early for many reasons, including concerns about safety, evidence of harm, or regulatory concerns.
12.What will it take to get the study to a Phase II or efficacy trial? If successful, the data from the Phase I study will be used to justify raising the funds necessary for a Phase II trial.
13.Is it possible this might cure TM? After an attack of monophasic transverse myelitis, damage is done to the spinal cord, which causes neurological symptoms such as weakness, paralysis, sensory changes, bladder, bowel, and/or sexual dysfunction etc. Therefore, a cure would be a therapy that repairs the damage to the spinal cord, which improves neurological symptoms. This trial is a safety trial, meaning the safety of this therapy is being assessed.
14.Since this study is only in transverse myelitis, how may it help people with neuromyelitis optica spectrum disorder, MOG antibody-associated disease, acute disseminated encephalomyelitis, or multiple sclerosis? If it is shown that this therapy repairs damage to the spinal cord in individuals with transverse myelitis, this therapy may be used to repair damage to the spinal cord from these others as well.
Known as one of the warmest members of the IndyCar community, her sudden loss has struck a heavy blow to her friends and family.
When Cody Unser became paralyzed at age 12 in 1999 with Transverse Myelitis, Shelley began a vigorous campaign of rehabilitation, fundraising and spreading the word to try and encourage others in their fight, and the Cody Unser First Step Foundation was formed. To read the complete article, click here: https://bit.ly/2wBMjhc
Editor - Kevin Weilacher
By Alison Rodriguez – AJMC Newsroom Aug 10, 2018
A recent study aimed to evaluate the burden of age of onset cardiovascular conditions in a US population with multiple sclerosis, neuromyelitis optica spectrum disorder, or transverse myelitis and found that cardiovascular conditions are as common in those with demyelinating diseases compared with unaffected individuals.
For the complete article, click on this link: https://bit.ly/2B7iquA
By Iora Mumal MS News Today July 23, 2018
People with multiple sclerosis are 48 percent more likely to have high blood pressure compared to the general population — and to people with other demyelinating diseases, a new study reports. Its researchers also emphasize that hypertension is already linked to poorer outcomes in MS patients.
The study, “Cardiovascular conditions in persons with multiple sclerosis, neuromyelitis optica and transverse myelitis,” was published in the journal Multiple Sclerosis and Related Disorders.
Like all demyelinating diseases, MS is marked by damage to the protective cover – called myelin – that surrounds nerve fibers in the brain, optic nerves, and spinal cord. For the complete article, click on this link: https://bit.ly/2JQjrpS
By Beth Galvin Fox 5 News – Atlanta July 7, 2018
ATLANTA - In Shepherd Center's Day Therapy gym, LaThea Adams is working hard to come back from a twist in the road she never saw coming, 13 years ago, when she was just 17. "It was the third day of my senior year in high school," Adams remembers.
She woke up with numbness and tingling in her arms. "I was rushed to the hospital, and within 24 hours, I was completely paralyzed, on a ventilator," Adams says, “with a diagnosis for transverse myelitis.”
For the complete article and video, click on this link: https://bit.ly/2Mhrz8a
By Lucy May & Katherine Nero – WCPO 9 Cincinnati - July 23, 2018
Alexandria, KY - Jen Stansbury Koenig can do laundry again. She can shop for groceries and clean her house and even take a few steps backwards without falling.
None of those things are a big deal for most people. But they are huge for Koenig, who couldn’t do any of them this time last year. “I haven’t fallen down for two months,” she said, smiling. “Before I was falling down at least once a week if not two or three times.”
“Before” was prior to October of 2017, when Koenig underwent an innovative stem cell transplant in Chicago to treat her advancing Multiple Sclerosis. The goal was to use Koenig’s own stem cells to help her body grow a new immune system and stop the progression of the disease that was stealing her mobility and limiting her life. The procedure appears to have done exactly that.
For the complete article and video, click on this link: https://bit.ly/2P3UScX
By Diogo Pinto MS News Today - Aug 1, 2018
Pregnancy, including successful delivery or miscarriage, worsens symptoms of multiple sclerosis (MS), as well as onset of the disease, a retrospective study shows.
Researchers found the same effect of pregnancy on neuromyelitis optica spectrum disorders (NMOSD), an inflammatory disorder of the central nervous system characterized by demyelination and damage of nerve fibers, especially in the eyes and spinal cord.
For the complete article, click on this link: https://bit.ly/2KM9Vog
By John Lundy - Duluth News Tribune - Aug. 7,2018
Paisley Forsell used to enjoy hiking, cliff jumping and competition body-building. She loves riding roller coasters. "I like adrenaline, I guess you'd say," said the 21-year-old, who lives in the city of Rice Lake.
But although she's looking forward to a trip to Valley Fair later this summer and now is going to the gym on good days, Forsell's activities have been sharply curtailed over the past year and a half.
She can manage a quick trip into a store to pick up an item, the Hermantown High School graduate said. A longer excursion requires her to use her wheelchair, which she ordered in a smaller size to fit her 5-foot frame.
Forsell was diagnosed in February 2017 with neuromyelitis optica spectrum disorder (NMO), a rare autoimmune disease that attacks the optic nerves and spinal cord. Since then, her life has been on something of a medical roller coaster. For the complete article and video, click on this link: https://bit.ly/2MF2EZe
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