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Biological Complexity of ALS to Be Addressed by the Development of New Stem Cell Models
The ALS Therapy Development Institute (ALS TDI), LifeArc, and Axol Bioscience launched the Patient induced pluripotent stem cell (iPSC)-based Research to Improve Sporadic ALS Modeling (PRISM) initiative, a collaborative effort to expand access to patient-derived stem cell models.
ALS is a heterogeneous disease. While 10-15% of cases are linked to inherited mutations, nearly 85% are sporadic, according to a PRISM ALS official, who adds that much of ALS drug discovery has relied on models representing a limited number of rare genetic subtypes. This mismatch has constrained target discovery, limited therapeutic testing across patient populations, and contributed to the high failure rate of clinical trials, maintains the spokesperson.
This initiative plans to provide a high-quality and accessible source of sporadic ALS/MND models for use in research. PRISM ALS aims to develop, evaluate, and make available a diverse panel of well-characterized, patient-derived induced pluripotent stem cell (iPSC) models that capture both genetic and sporadic forms of ALS.
For researchers and drug developers, those standardized, human-relevant models are expected to allow them to better understand disease mechanisms, identify therapeutic targets, and evaluate treatments across distinct biological subtypes. For people living with ALS, it might lead to the development and testing of therapies in models that more closely mirror their own biology, increasing the likelihood that discoveries will translate into meaningful treatments.
The stem cells used in PRISM ALS are derived from samples contributed by people living with ALS through ALS TDI’s ALS Research Collaborative (ARC) Study, the longest-running longitudinal patient study in ALS.
“We know that ending ALS will require delivering the right treatments to the right individuals,” says Fernando Vieira, MD, CEO, and CSO of ALS TDI. “By characterizing iPSC-derived motor neurons from sporadic ALS and making these cells broadly accessible, PRISM ALS will facilitate global drug discovery. This program is only possible thanks to the people living with ALS who contributed samples and data through the ARC Study.”
“We’re delighted to participate in this consortium to develop multiple iPSC-derived end point cell types from sporadic ALS iPSC lines that reflect for the first time, real-world variability across age, sex, and genotype,” notes Sapna Vyas, head of scientific programs at Axol Bioscience. “By leveraging Axol’s scalable manufacturing infrastructure, we will facilitate access to standardized iPSC-derived cells that empower researchers to stratify patients, assess subgroup responses to therapies, and reduce late-stage clinical trial failures.”
“Our hope is that the stem cell models we produce can unleash a new generation of treatments that could be effective against this disease by slowing its progression and, ultimately, curing it,” adds Paul Wright, head of motor neuron disease (MND) at LifeArc. “We need to do more for people living with MND/ALS, and PRISM ALS brings together leading organizations to help make that happen.”
The post Biological Complexity of ALS to Be Addressed by the Development of New Stem Cell Models appeared first on GEN – Genetic Engineering and Biotechnology News.
Hosted by Drug Discovery World and supported by Sartorius and BioIVT, this webinar will explore the opportunities and challenges that exist within cancer research drug discovery and development.
You will hear from Dr Sudha Rao, Chief Scientific Officer of Kazia Therapeutics, Karol Budzik, PhD, Business Development Associate at Vyriad Therapeutics and Lars van der Veen, Chief Scientific Officer at iOnctura.
Presentations will cover how cancer treatments have shifted towards reprogramming the biology driving tumour growth, immune escape and treatment resistance, the trajectory that in vivo CAR-T treatments are taking, and how challenging tumours burdened by stroma and immune-mediated resistance can be tackled.
Q&A with the speakers follows the presentations.
The post New webinar: Tackling drug discovery challenges in cancer research appeared first on Drug Discovery World (DDW).
Amid the rise of psychedelics in the mental health space, researchers have begun to explore psilocybin as a treatment for chemotherapy-induced peripheral neuropathy.
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New Spectrometry Technique Could Aid Formulation Development
New Spectrometry Technique Could Aid Formulation Development
A new technique combining two forms of spectrometry could help biopharmaceutical companies improve their choice of formulation buffer for antibody manufacturing by revealing how molecular forms and three-dimensional shapes of complex biologics respond to their environment. That’s the view of Christian Bleiholder, PhD, a professor at Florida State University who helped develop the technique.
According to Bleiholder, what happens structurally when a complex biological molecule, such as an antibody or viral spike protein, binds to its target is currently poorly understood.
“This is where [this approach] can help with the bioprocessing and formulation,” he says, as structural changes “can affect the lifespan [of the product] and lead to issues, such as aggregation.”
Because antibodies are complex, existing techniques tend to be powerful at different levels of complexity, he explains. Mass spectrometry is particularly powerful for distinguishing molecular composition, while structural approaches such as X-ray crystallography and cryo-electron microscopy can provide high-resolution structural information.
The challenge is understanding the link between these things within a heterogeneous sample, he says.
To overcome this, Bleiholder and his team worked with Bruker Daltonics to develop Tandem-Trapped Ion Mobility Spectrometry (Tandem-TIMS). This combines tandem ion mobility spectrometry with tandem mass spectrometry to disentangle three overlapping layers of molecular complexity: molecular form, three-dimensional shape, and binding or assembly state, he says.
He explains that, if the proteins have different structures, they can be characterized with tandem ion mobility spectrometry, and then mass spectrometry can be used to look at their molecular forms and binding states.
Going forward, Bleiholder hopes the technique can be used for formulation development but also earlier, during drug discovery of new products, such as multi-specific antibodies, to determine which molecular states are important and how those change when a biologic engages its target. He also plans to look at automating the technique.
Bleiholder spoke about using Tandem-TIMS at the Bioprocessing Summit in Boston earlier this year.
The post New Spectrometry Technique Could Aid Formulation Development appeared first on GEN – Genetic Engineering and Biotechnology News.
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