![]() The findings led the researchers to conclude that it may be possible to develop human iPSCs that stably produce GDNF to safely provide the benefits of the current clinical trial candidate without the constraints on manufacturing output. No rodents grew tumors or developed other health problems for several months after treatment. Importantly, the cells continued to produce GDNF and were safe. The cells survived, integrated into the spinal cord, and formed astrocytes, a subtype of glial cells that malfunction in ALS patients. Similarly, when transplanted into the spinal cords of rodents with ALS the cells helped protect motor neurons. Injecting the cells into the eyes of rodents with retinal degeneration led to protection of the cells in the eye that support vision. The results, which were published on Thursday in Stem Cell Reports, suggest that the approach has potential. To explore if iPSCs are suitable for their GDNF treatment, the researchers engineered iPSC-derived neural progenitor cells to produce the protein and studied the resulting cell therapy candidates in rodents. However, one downside is the potential for iPSCs to cause tumors to develop. The supply constraints led Svendsen and his collaborators to investigate iPSCs, cells that, because of their strong self-renewal ability, could provide a virtually unlimited source of cells. We just don't have endless product," said Svendsen. "The cell lines we are using in the clinic are coming from a single source and are going to eventually run out. However, supply problems could arise as the one-time treatment moves through the clinic and is needed in larger volumes to meet demand, Clive Svendsen, PhD, executive director of the Cedars-Sinai Board of Governors Regenerative Medicine Institute, explained in a statement. With the study finding that the treatment is safe, the researchers embarked on further development. The clinical trial studied the effect of cells engineered to produce glial cell line-derived neurotrophic factor (GDNF), a protein that can promote the survival of motor neurons. doi:10.1016/j. year, scientists at Cedars-Sinai Medical Center published early-phase clinical trial data on a potential cell therapy treatment for ALS. Safety and tolerability of bosutinib in patients with amyotrophic lateral sclerosis (iDReAM study): a multicentre, open-label, dose-escalation phase 1 trial. Transplantation of human neural progenitor cells secreting GDNF into the spinal cord of patients with ALS: a phase 1/2a trial. Immunoablation and stem cell transplantation in amyotrophic lateral sclerosis: the ultimate test for the autoimmune pathogenesis hypothesis. A phase I/IIa clinical trial of autologous hematopoietic stem cell transplantation in amyotrophic lateral sclerosis. New developments and opportunities in drugs being trialed for amyotrophic lateral sclerosis from 2020 to 2022. In one study, participants with ALS who received the drug showed a decrease in markers of ALS damage, which were measured with blood tests. Bosutinib, a pluripotent stem cell (iPSC)-based drug used to treat blood cell cancer, is being evaluated as a possible treatment for ALS.A tissue analysis of these participants showed graft survival and GDNF production, which is considered a good outcome. Of the 18 participants, 13 participants died of disease progression. At one year, there was no negative effect. The stem cells were injected into the participants’ lower spinal cord. One study used human neural progenitor cells (which can form nervous system cells) modified with glial cell line-derived neurotrophic factor (GDNF), which protects astrocytes.In this procedure, blood-forming stem cells from a person are harvested from their blood or bone marrow and returned after the person has received treatment to stop bone marrow blood-producing activity. In a clinical trial using autologous hematopoietic stem cell transplantation for people with ALS, the procedure was well tolerated, but there was no evidence of a significant modification in disease progression.
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