Arizona State University to Grow Human Neurons in Search of New Therapies for Neurodegenerative Diseases

Arizona State University to Grow Human Neurons in Search of New Therapies for Neurodegenerative Diseases

Arizona State University will soon launch a new biomanufacturing platform to grow human neurons in vitro to develop and test new therapies for neurodegenerative diseases, including amyotrophic lateral sclerosis, Alzheimer’s disease, and Parkinson’s disease.

The effort includes the development of several types of neurons on a large scale to test the effectiveness of small therapeutic molecules.

The $5 million project is financed by the U.S. Department of Defense’s Advanced Regenerative Manufacturing Institute and is a collaboration between the laboratory of David Brafman, assistant professor of engineering at Arizona State University (ASU), Biogen, one of the world’s largest pharmaceutical companies, and Trailhead Biosystems, an Ohio-based biotechnology company. ASU will receive $1.4 million.

“There is much excitement about the potential of human pluripotent stem cells to treat numerous devastating diseases. While the public is more familiar with the tissue and organ replacement aspects of pluripotent stem cell research, these cells also have tremendous utility in developing pharmacological interventions to combat these diseases,” Brafman said in a press release.

“As such, the focus of this project will be to develop the biomanufacturing processes needed to engineer the various neural cell types needed for drug screening,” he added.

“ASU’s growing leadership in this field, combined with the strengths of our partners Biogen, Inc., and Trailhead Biosystems, will bring innovative solutions to the fore impacting the future of treatments for neurodegenerative diseases,” said Sethuraman Panchanathan, executive vice president of Knowledge Enterprise Development and chief research and innovative officer at ASU.

One of the strategies researchers may use to develop the different neuronal lines for future research is generating human induced pluripotent stem cell lines from peripheral blood mononuclear cells.

As part of his research in Alzheimer’s disease, Brafman already has used blood cells from Alzheimer’s patients and reprogrammed them back into an embryonic-like pluripotent state. These cells can then be transformed into virtually every cell in the body and constitute an unlimited source of any type of human cell needed for therapeutic purposes.


    • Charlie says:

      ‘in vitro’ is the clue here.

      Growing human neurons is the interesting bit. Once they grow them in the labs it will maybe be a case of how they get them into a human, in the right place, and then it will be a case of whether those home-made neurons can impact on an existing pALS neurons- presumably they will want to see if they can replace the dead neurons and get functionality back.
      But what do I know……very little and I am speculating.
      Many years of lab work ahead on this one I think, but to me it seems very promising for future pALS.

  1. Charlie says:

    “Shares of gene therapy companies fell sharply Tuesday, after a report by a prominent scientist revealed the safety concerns that led him to resign recently from a scientific advisory board.

    Gene therapy researcher James Wilson, a giant in the field, quit Solid Biosciences Inc.’s advisory board ahead of the company’s initial public offering which was completed last week, because of the possible risks of high systemic dosing of AAV, the company’s delivery system for its cell therapy, according to the company’s IPO prospectus.

    Just hours before Solid Biosciences SLDB, priced its IPO, it revealed that the U.S. Food and Drug Administration had placed a partial clinical hold on the company’s lead product, SGT-001, a treatment for Duchenne muscular syndrome that involves high-dose AAV gene therapy.

    On Monday, Wilson, a University of Pennsylvania professor and head of its gene therapy program, and his team published a paper that revealed that monkeys and pigs used in an animal trial were sent into toxic crisis when given a high dose AAV delivery of a corrective gene.

    “We believe today’s publication likely rounds out the explanation for his (Wilson’s) departure,” said RBC analysts in a note.

    The paper has implications for companies developing gene therapy treatments, including AveXis Inc. AVXS, which is working to develop a treatment for spinal muscular atrophy, said the note. AveXis has already dosed at least a dozen patients with its AVXS-101 product and has not observed toxic reactions, “so it is possible such AEs are not applicable to humans, are only related to the specific variants Dr. Wilson’s lab described, and/or are related to manufacturing impurities – though it could help explain FDA’s apparent scrutiny in analyzing much of AVXS’s preclinical and manufacturing data ahead of filing/approval for the drug,” said the note.

    Biogen Inc. BIIB, is planning to move its spinal muscular atrophy gene therapy into the clinic last this year and may use the AAV delivery system, as Wilson has worked on that program.

    “Regardless, our sense has been that BIIB does not plan to assess their gene therapy as a high-dose systemic IV and is more likely to pursue intrathecal or other local delivery to the CNS, which could potentially avoid this possible toxicity,’ said the analysts.

    Sarepta Therapeutics Inc. SRPT, is also working to develop a gene therapy treatment for Duchenne muscular dystrophy in competition with Solid Biosciences, they said.”

    God -sorry, I mean Science- doesn’t always give with both hands.

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