🔬 “Aspen Neuroscience looks to combine its expertise in stem cell biology, genomics and neurology to offer the first autologous cell therapy for Parkinson’s disease—while others in the space have pursued allogeneic routes, or therapies derived from donors other than the patient.
“We can say without any equivocation that we can produce the population of cells necessary to transplant, and in a short enough period of time to have a potential beneficial impact on the evolution of the disease,” said Federoff, who has also served as chair of the NIH’s Recombinant DNA Advisory Committee and helped lead the U.S. Parkinson’s Disease Gene Therapy Study Group.
“We envisage that this will set back the clock on patients who have Parkinson’s, unlike any other therapy that we know of,” he told FierceMedTech in an interview.
“We anticipate that they will manufacture and release dopamine in a manner that is consistent with synaptic neurotransmission and the process of communicating from cell-to-cell,” said Federoff. “They will take up dopamine from synapses when it has done its business, bring it back into the cell, and prepare it for another synaptic release.”
“These are not just dopamine pumps, they’re real neurons,” added Loring. “They will genuinely replace the cells that have been lost in every way.
“Aspen plans to pursue two courses of therapy, for the two major types of Parkinson’s disease. Their lead candidate is for idiopathic, or sporadic Parkinson’s, while their second is a CRISPR-edited version of the therapy designed to address one of the disease’s most common genetic mutations, linked to about 5% of cases.
“This would not only aim to restart dopamine production in this orphan indication, but also restore the damaged enzyme GBA, which is seen as an underlying cause. Federoff and Loring expect their sequencing-based quality check system will also help catch any off-target edits linked to the use of CRISPR-Cas9.
“After it receives its go-ahead from the FDA, Aspen plans to hit the ground running, enrolling at least 176 participants in a phase 1/2 study that includes a randomized stage to determine clinical benefits.”
Sounds wonderful. And good to have something different as a genuine news story. Jeanne Loring has been around a while making big claims that are outside the mainstream, and dismissive of the regular stem cell research. It would be interesting to see some independent scientific review of this stuff. But, meantime, keeping my fingers crossed that she's the real deal
JANVAN, “The company has yet to secure permission from the FDA to officially launch clinical trials, but the agency has signed off on Aspen’s plans to prepare a ‘trial-ready cohort’ of Parkinson’s disease patients in the meantime. This would include the initial stages of recruitment and testing, including the selection of patients capable of having their skin cells made into pluripotent stem cells.”
*The ‘initial stages of recruitment and testing’ are supposed to take place early 2020 - hopefully within a few months.
While stem cells are a hopeful way of restoring dopamine producing cells, we still need to find a way to stop PD's progressive killing of those cell, so that we do not repeat the process over again.
Absolutely, once the IP stem cells have restored normal dopamine levels and "reset your clock", knowing the precise mechanics behind that long, slow, 20-30 year progression that preceded the whole ordeal will come in real handy (just in case you live beyond your second 20-30 year progression window).
Both strands are important. There is a lot of activity in the" stop progression" field. Its been a bit quieter in the reverse the damage field. The frustration is the lead times. If this is the real deal and kicks off research in 2020 its likely to be late 2023 before the phase 1,2 reports and then another 4 years phase 3. With the best of luck IF it is successful I might be able to book surgery for 2028.
Still, it's the job that's never started takes longest to finish...
At least there seems to be some urgency behind this
Great news; but the article I read said that "the company expects the earliest it would get the go-ahead from FDA to start human trials would be 2021." Wish they could hurry this along!
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>>> how points of view can cross each other <<<
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