Latest news with #stemcelltherapy
News.com.au
19-05-2025
- Business
- News.com.au
Dr Boreham's Crucible: Can this biotech emerge from Mesoblast's shadow and ride ‘stem cells 2.0'?
Investors now have three ASX-listed stem cell therapy companies to choose from – and we can thank sector big daddy Mesoblast for inspiring the third. Hitherto a developer of peptide-based drugs for neuroscientific conditions, NeuroScientific Biopharmaceuticals (ASX:NSB) s is in the process of acquiring Stemsmart, the stem-cell portfolio of the public unlisted Western Australia company Isopogen WA Ltd. In December, Mesoblast won US Food and Drug Administration (FDA) approval for Ryoncil, its stem cell treatment for paediatric graft versus host disease (GvHD). Neuroscientific describes this approval as 'momentous', as it was the first FDA assent for a therapy derived from donor bone marrow. Both the Ryoncil and Stemsmart therapies are based on mesenchymal stromal cells (MSCs). (The other company is Cynata, also working with mesenchymal stem cells but induced pluripotent ones called IPSCs.) 'Stem cell therapy is a cornerstone of modern medicine,' the company says. 'Stem cells have the unique ability to develop into different cell types in the body and are often hailed as the body's master cells.' Neuroscientific's initial focus is on the common auto-immune condition Crohn's disease. That makes sense, given Isopogen underwent a phase II trial for that condition that deemed the therapy to be 'potent, efficacious and safe.' About Neuroscientific To date, the Perth-based Neuroscientific has been developing peptide-based drugs for several neuro-degenerative conditions with high unmet medical demand. Neuroscientific was founded by former equities analyst Dr Anton Uvarov, on the back of the Emtinb peptide. This asset was developed by the University of Copenhagen and then acquired by the University of Tasmania. The company listed on July 25, 2018, having raised $6 million at 20 cents apiece. At the time, Emtinb was most advanced for Alzheimer's disease, but recently the board decided to focus on glaucoma. Before founding the company, Uvarov had a two-year stint a biotech analyst at Citigroup. He has also been on the board of several ASX listed biotechnology companies and non-biotechs, including Blinklab, Actinogen, Sun Biomedical (now Dimerix), Acuvax and Imugene. The company's portfolio includes Emtinb (initially targeting glaucoma) and Emtinac, Emtinan, and Emtinbn 'which have demonstrated similar therapeutic potential as Emtinb'. As part of the Stemsmart purchase, current Isopogen directors Robert McKenzie and Paul Fry will join the board. The force behind Stemsmart, Dr Marian Sturm joins as chief scientific adviser. An inaugural member of the local Therapeutical Goods Administration (TGA) advisory committee on biologics – among other things – Sturm is a leading expert in cell therapies in Australia (particularly in stem cell therapies). Peace deal: now for Ukraine and Gaza Uvarov says he had known about Sturm and her work for some time, given they both hail from Perth medical circles. In 2021, Perth's East Metropolitan Health Service (EMHS) launched a legal action over ownership of the stem cell intellectual property on which Isopogen was founded. Sturm was a long-time employee of EMHS's Royal Perth Hospital. The technology was developed in 2007 and registered in Sturm's and Isopogen's names. Neuroscientific swooped after Sturm and EMHS last year reached a 'mutually acceptable' confidential settlement. A joint statement said the peace deal provided the basis for an 'ongoing relationship' – so let's call it friends with benefits. Investors had been excited about Isopogen's prospects as a 'mini Mesoblast' – and an ASX listing seemed on the cards. But the prolonged and increasingly complex legal spat put paid to that. 'As soon as it was resolved we jumped on the opportunity,' Uvarov says. About Stemsmart Neuroscientific describes the Stemsmart cells as having 'potent anti-inflammatory and immune-modulatory properties'. This creates a 'multifaceted and complex interaction' with the body's immune system, dampening inflammation, moderating immune responses and encouraging tissue repair. 'It's quite an advanced program,' Uvarov says. 'Overall, more than 200 patients have gone through this therapy, so we know it's quite safe and active.' Derived from adult human donor marrow, mesenchymal stem cells (MSCs) are grown in a culture and then revved up with the patented cell manufacturing process. To date, patients have received Stemsmart on compassionate grounds for a variety of serious and life-threatening clinical conditions, with 'multiple strong positive clinical responses'. Crohn's disease Isopogen undertook a phase II trial of 18 patients with refractory Crohn's disease. The results were 'promising', with most patients experiencing clinical improvement and even clinical remission. The company's attention has turned to a small, 12-person, phase I trial for fistulating Crohn's disease under a TGA special access program. A severe complication of Crohn's disease, fistulas are abnormal tracts connecting the intestine to another organ or to the external surface of the body. Fistulating Crohn's disease is challenging to treat and sustained healing with standard therapies has been limited. 'Nothing disease modifying exists now, so it's a massive market,' Uvarov says. 'If we do a phase I trial, the next step would be to move to a phase II/III study as a potential step towards a regulatory trial.' The study will aim for a closure of more than 50% of the fistula openings, or a decrease in fistula discharge of more than 50% in at least four patients. Addressable markets Over the next 24 months, the company envisages expanding its stem cells trial from Crohn's disease, to other inflammatory and immune-based disorders including lung disorders and acute kidney transplant rejection. Uvarov says Isopogen has 140 doses frozen and ready for use in further clinical studies. Several patients have received the therapy on compassionate grounds, including both children and adult with graft versus host disease (GvHD). The company cites a US$13.8 billion addressable market for Crohn's disease and a US$640 million opportunity for GvHD by 2026. There's a projected US$7.2 billion market for organ transplant immune-suppressants by 2030 - the majority for renal – and a forecast US$33 billion market for lung disorders by 2023. Legacy program lives on And let's not forget about Neuroscientific's pre-clinical legacy program. Emtinb targets Alzheimer's disease and advanced glaucoma. In 2023, Neuroscientific decided development should focus on the latter as a therapy administered locally, via intravitreal injection. Uvarov says the company moved to stem cells because the legacy program was not moving as fast as investors would have liked. And an Alzheimer's program would have required a much bigger trial. In June last year, the company met with the US Food and Drug Administration for a pre-investigational drug approval powwow, with the agency guiding on the pre-clinical studies required to progress Emtinb to a first-in-human trial. Based on this counsel, the company plans a pharmaco-kinetics rabbit study, by which Emtinb is intravitreally administered. In parallel, the company plans a 13-week study of 'ocular tolerance, systemic toxicity and pharmacokinetic following repeated intravitreal administration in pigmented rabbits'. Given there's shortage of animals for medical research as suppliers withdraw from the market, it's a case of when the company can obtain the specially bred bunnies. 'It's harder to do animal studies because of regulatory pressure,' Uvarov says. 'Regulators want to move from pre-clinical animal studies to cell-based research using animal organoids.' Organoids are three-dimensional structures that mimic the architecture and function of human organs and tissues. Finances and performance Neuroscientific must obtain the separate assent from all Isopogen holders, although Sturm is by far the biggest holder. The company has snared 51.4% of Isopogen holders and is confident of the remaining minority holders coming on board. Under the scrip deal, Isopogen holders receive 85,714,286 shares, deemed to be worth 3.5 cents. At today's values the deal is worth a tad over $4 million. Isopogen holders also receive 57,142,857 performance shares convertible to ordinary shares, subject to the aforementioned milestones. These must be achieved within three years of a shareholder meeting to approve the performance shares, scheduled for mid-June. All the issued shares will be escrowed for 12 months. Currently underway, the $3.5 million capital raising is by way of a placement of 100 million shares, at 3.5 cents a share. Post-raising, the company will have cash of $7.5 million. Of the funds raised, just over $2 million is expected to be used for Stemsmart-related stuff. A further $835,000 is earmarked for the Emtinb program. Over the last 12 months Neuroscientific shares have ranged between 3.3 cents (early January this year) and 5.5 cents (early May 2024). The stock peaked at 50 cents in mid-September 2021. The tightly held register is dominated by the Clough family office, of Clough Engineering fame. Clients of Westar Capital account for much of the remainder. Dr Boreham's diagnosis Uvarov says Isopogen's stem cells could be 'even more potent' than Mesoblast's cells, although they work more by way of immune modulation rather than regeneration. 'Our cells … have more growth factors they excrete when you culture them,' he says. He says the FDA's approval of Mesoblast's Ryoncil 'paves the way for renewed enthusiasm and global investment in clinical research of MSC therapies'. It's not just the company that's hyper-enthused: the International Society for Cell and Gene Therapy dubs the Ryoncil approval as 'a pivotal moment in the history of medicine shaping the future of therapeutics'. Uvarov adds that after years of being untrendy, stem cells again were the hot topic at JP Morgan's global biotech gabfest in January. 'We are at the beginning of stem cells 2.0,' he says. Given Mesoblast is worth around $2.3 billion and Neuroscientific is valued at not much more than its cash backing, Neuroscientific will be walking in the shadow of a giant. 'When Mesoblast was at a similar stage they had a market cap of several hundred million dollars,' Uvarov says. How soon the company emerges from this penumbra depends on the pace of its trials and – ultimately – its first port of call of initial TGA approval. 'It's complex and tricky so we are taking it slow,' Uvarov says. At a glance: ASX Code: NSB Share price: 4.8 cents Shares on issue: 144,604,870 * * Increases to 333,176,299 shares post-Isopogen WA acquisition and placement Market cap: $6.9 million Chief executive officer: Dr Anton Uvarov (founder) Board: Chris Ntoumenopoulos (chair), Dr Tony Keating, Clarke Barlow, Uvarov, Dr Linda Friedland Financials (March quarter 2025): revenue nil, cash burn $182,000, cash of $4.3 million (ahead of $3.5 million capital raising).
Forbes
13-05-2025
- Business
- Forbes
Somite AI Raises $47M Series A To Reinvent Cell Replacement Therapy
Regenerative medicine and therapeutic stem cell therapy to regrow damaged cells as treatment for ... More disease Startup Somite AI announced today that it has raised over $47 million in a Series A funding round, bringing its total funding to about $60 million. The round was led by Khosla Ventures, with participation from SciFi VC, the Chan Zuckerberg Initiative, Fusion Fund, Ajinomoto Group Ventures, Pitango HealthTech, TechAviv, Harpoon Ventures, and prominent angel investors such as Fidji Simo, the new CEO of Applications at OpenAI. Legendary investor Vinod Khosla said in a statement: 'Somite AI's foundation models, once fully developed and validated, will not only create value for their own pipeline, but have the potential to reshape the entire field of human cell therapy.' Somite's initial ambition was to design a 'life language model' to drive the development of Somites, the embryonic structures at the origin of the musculoskeletal system, and use them in cell replacement therapy targeting particular ailments such as Duchenne muscular dystrophy, the most common hereditary neuromuscular disease. With today's announcement, Somite is publicly broadening its scope, aiming to revolutionize the production of any human cell type through its AI foundation model platform, DeltaStem, to address a broad spectrum of diseases, including metabolic disorders such as Type 1 Diabetes, orthopedic conditions, muscular diseases, and blood disorders. When Somite was established just over a year ago, the founders asked themselves what they would be able to do if they could create any type of human cell. 'We'd be able to replace and replenish any diseased or damaged tissue in the body,' says co-founder and CEO Micha Breakstone, defining their mission as reverse engineering of stem cell biology with artificial intelligence. 'Today, we only know how to reliably produce about 10 types of cells, but there are over 5,000 types of cells in the human cell atlas,' says Breakstone. 'There are more possible ways of creating a specific cell than there are atoms in the universe.' To take a stem cell to a specific mature state, researchers adjust both its environment and the specific biological signals it receives—such as the concentration of a growth factor or a change in pH—learning through step-by-step experimentation the precise sequence of conditions that guide its developmental trajectory. How do you re-engineer a process that takes many years, tens of millions of dollars, and lots of trial and error, with no guaranteed result? As in other domains (e.g., quantum computing error correction), where the 'search space,' the initial set of potential solutions to a problem, is beyond human comprehension (e.g., larger than the number of atoms in the universe), AI could be of great help. However, you need vast quantities of data to successfully apply today's AI or deep learning. The first ingredient in Somite's recipe for developing a cell foundation model is its innovative method for efficiently generating data at scale in a completely natural way. Professor Alon Klein, another Somite co-founder, had a thinking-inside-the-box breakthrough moment when he asked, as Breakstone tells it, 'What would happen if instead of taking the signals to the cells, we take the cells to the signals?' The box or boxes in this case are semi-permeable capsules, each containing a few cells, with minuscule 'windows' allowing the signals to come in and affect the cells. The Somite lab team sends millions of these capsules through many signals or conditions, and each cell-condition encounter is recorded with a barcode. Put all the barcodes together and 'you get an address that correlates with exactly the specific trajectory that the capsule went through, a beautiful model of the development of the embryo,' explains Breakstone. This capsule technology generates cell state transition data at an unprecedented scale, achieving 1000x greater efficiency than existing methodologies. For Jonathan Rosenfeld, another Somite co-founder, this data serves as the basis for developing an AI foundation model for the human cell. Currently, Somite is running experiments at roughly a one-million-condition scale, mapping the developmental paths embryonic cells take under these varied conditions. It plans to reach 10 million conditions by the end of the year. Given the enormous size of the 'search space,' even 100 million would be just a drop in the vast ocean of 'protocols,' all the possible trajectories of cell development. 'However,' says Breakstone, 'it's a drop large enough to learn from, and that's the beauty of deep learning. From a relatively small amount of data, we can develop and train a working model to generate novel protocols that we haven't seen before.' With its DeltaStem platform, Somite aims to overcome key challenges of current lab-based stem cell development—the prohibitively expensive, slow, manual, trial-and-error methods researchers use to guide stem cells toward specific mature cell types. Major obstacles include purity (percentage of desired cells produced), scalability (efficient cell manufacturing at therapeutic scale), and reproducibility (consistent results across batches). 'What AlphaFold did by predicting protein structures, transforming structural biology, DeltaStem aims to achieve for stem cell biology—predicting the conditions needed to precisely generate mature human cell types at scale,' says Breakstone. Unlike biotech companies traditionally engaged in individual drug development, 'TechBio' companies like Somite focus on developing broad technology platforms addressing multiple indications. Their approach is data-driven, and their goal is to develop proprietary AI that reduces costs and risks, shortens drug and treatment development time, and accelerates the shift to more personalized medicine. The success of Moderna and the Nobel Prize awarded to AlphaFold have attracted increased venture capital and corporate investment in applying data and AI to current healthcare challenges, including improving regenerative medicine: 'Artificial intelligence presents broad utility in the discovery and development of new biotherapeutics.' Somite's AI foundation model and platform will help build a future 'where we can create any human cell on demand, like a supply of cellular spare parts to repair or replace diseased or damaged tissue,' says Breakstone.
Health Line
12-05-2025
- Health
- Health Line
Understanding the Potential of Stem Cell Therapy for Diabetes Type 2
Stem cell therapy is an emerging treatment for type 2 diabetes. Although it's not yet FDA-approved in the United States, it is a rapidly expanding and promising field of research. Stem cells are special human cells that can become many different types of cells. Because of this special property, doctors are investigating the potential of stem cell therapy to treat many conditions. People with type 2 diabetes don't make an adequate amount of insulin to manage their blood sugar. Stem cell therapy may potentially allow people with type 2 diabetes to start producing more insulin again and reduce the need for medications. The effectiveness and safety of stem cell therapy for treating diabetes are still under investigation, but some early studies have found promising results. What is stem cell therapy? Stem cell therapy involves taking stem cells from your body or another human's to help regenerate damaged tissues. Stem cells can be collected from fetal and adult tissues, such as: fat tissue umbilical cord bone marrow placenta How is stem cell therapy being used to treat type 2 diabetes? The diabetes epidemic remains one of the biggest health crises in the United States. Roughly 1 in 10 people in the United States has diabetes, with the majority having type 2. Researchers continue looking for new treatment options, and stem cell therapy has emerged as a promising option. Its use in humans is still being investigated, and more research is needed to assess its long-term effectiveness and safety accurately. According to the authors of a 2025 letter to the editor, their team identified 143 diabetes stem cell therapy clinical trials worldwide from 2000 to 2024. Of these, 19 trials were ongoing. Research on type 2 diabetes Most research to date has examined stem cell therapy as a treatment for type 1 diabetes. In 2008, a team of researchers recruited a small group of 25 people with type 2 diabetes for a phase 1 clinical trial examining the effectiveness of stem cell therapy combined with hyperbaric oxygen treatment. The researchers found that all measured markers, such as fasting glucose levels and insulin requirement, improved at a one-year follow-up. Since then, more studies have continued to find evidence that stem cell therapy may potentially improve blood sugar management and reduce the need for diabetes medications. Further ongoing trials have also shown that stem cell therapy may help improve diabetic complications such as: diabetic foot ulcers erectile dysfunction diabetic peripheral neuropathy Modern research In 2024, researchers reported a groundbreaking case of a 25-year-old woman with type 1 diabetes who started producing insulin within three months of receiving a stem cell transplant, effectively reversing her diabetes. The results of current trials for type 2 diabetes look promising. Future research will focus on analyzing long-term results and safety in larger groups. Many clinical trials are currently underway. For instance, one clinical trial that's currently recruiting is examining the effectiveness of stem cell therapy for treating type 2 diabetes that hasn't responded to other treatments. A phase II clinical trial that isn't yet in the recruiting phase is examining the effectiveness and safety of a type of stem cell therapy that involves taking cells from your own body. How does stem cell therapy compare to traditional treatments for type 2 diabetes? Treatment for type 2 diabetes typically consists of making lifestyle changes and sometimes taking medications. Commonly prescribed medications include: metformin sulfonylureas glinides GLP-1 receptor agonists DPP-4 inhibitors Thiazolidnediones These medications help improve your blood sugar management, but they need to be taken continuously to be effective. Stem cell therapy is an attractive alternative since it can potentially be curative after one treatment. Who is a good candidate for stem cell therapy for type 2 diabetes? Stem cell therapy is still an experimental treatment. Researchers are continuing to see who might make a good candidate. For now, researchers may offer it as an experimental treatment for people who haven't responded to other treatments. What are the risks of having stem cell therapy for type 2 diabetes? Researchers are continuing to examine the safety of stem cell therapy for type 2 diabetes. Some concern is that it could encourage tumor formation. Side effects reported in clinical trials included: fever bruising nausea vomiting headache hypoglycemia What is the success rate for stem cell therapy as a treatment for type 2 diabetes? Improvements in blood sugar management have been reported in as little as weeks. For example, in a 2024 case study, researchers observed improvements in a 59-year-old man with severe diabetes complications within 2 weeks. Studies consistently show improvements in blood sugar management, but larger studies are needed to understand the actual success rate. Where can you have stem cell therapy for type 2 diabetes? Stem cell therapy for type 2 diabetes is still under investigation. In the United States, thousands of clinics have appeared offering unlicensed and unproven stem cell therapies. The FDA hasn't yet approved stem cell therapies for diabetes. What does cell therapy for type 2 diabetes cost? Stem cell therapy isn't yet available for treating type II diabetes in the United States. Because it's not approved for use, it's also not covered by insurance. To get a rough idea of the cost, in one study, researchers estimated that the average price of stem cell therapy for diabetes might range from $5,000 to $50,000. What's the outlook for people who have stem cell therapy for type 2 diabetes? Little research examines the long-term effectiveness of stem cell therapy for diabetes. Most current studies have had follow-ups shorter than 1–2 years. Although it shows a lot of promise, more research is needed to judge long-term results accurately. Can you still have traditional treatment during or after stem cell therapy? Some people seem to reduce their need for medications or can stop taking medications altogether after stem cell therapy. Takeaway Stem cell therapy is being investigated as a potential treatment for type 2 diabetes. In theory, it may allow the body to produce more insulin to better regulate blood sugar levels. Stem cell therapy isn't yet FDA-approved in the United States for treating diabetes, but researchers continue examining its safety and effectiveness. The field of stem cell therapy is expanding rapidly, so it's likely that many more studies will be completed in the next few years.
