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10 April
Invion Limited
Investor Insight
With a strong IP portfolio, deep clinical-stage pipeline, global collaborations and an experienced leadership team, Invion is poised for transformative value creation as it enters the next phase of clinical and commercial growth.
Overview
Invion Limited (ASX:IVX) is a clinical-stage Australian life sciences company focused on transforming photodynamic therapy (PDT) into a next-generation solution for treating cancer and infectious diseases. The company’s core value lies not just in preclinical promise, but in real-world clinical validation.
At the heart of Invion’s platform is Photosoft, a proprietary suite of next-generation photosensitizers that selectively target diseased cells and, upon light activation, induce a precise oxidative stress response to destroy them. Unlike conventional PDTs that suffer from toxicity and off-target effects, Photosoft compounds are engineered for enhanced safety, specificity, immune activation, and theragnostic utility. INV043, Invion’s lead cancer drug candidate, has demonstrated up to 80 percent tumor control in combination with immune checkpoint inhibitors, and is now in human trials.The company is actively progressing a broad pipeline across multiple indications, including:
- A Phase I/II trial in non-melanoma skin cancer (NMSC), now dosing in Australia
- An anogenital cancer trial in collaboration with Peter MacCallum Cancer Centre, scheduled to begin in 2025
- A completed investigator-led Phase II prostate cancer trial, showing a 40 to 44 percent response rate with a strong safety profile
These trials signal a critical shift for Invion from preclinical research to meaningful clinical validation, spanning both systemic and topical formulations. The safety data from the NMSC trial is expected to support accelerated development of the anogenital cancer program, given both use the same topical compound.
The clinical results so far have been consistent with the findings from the preclinical studies – showing INV043 to be very safe with promising efficacy signals. The next set of trials aim to further solidify the potential for the technology, where the one drug can treat multiple cancers, improve patient outcomes when using checkpoint inhibitors in combination with INV043 and demonstrate its diagnostic and theragnostic potential.
Beyond internally funded trials, Invion is strategically expanding its clinical and commercial footprint through non-dilutive, global partnerships. In South Korea, Hanlim Pharm is funding the preclinical development of Photosoft for glioblastoma multiforme — a highly aggressive brain cancer — and oesophageal cancer, both of which present major unmet needs. These programs are fully financed by Hanlim, with Invion retaining all intellectual property
Similarly, Dr. I&B Co. (Dr.inB), another South Korean group, is backing the development of Photosoft for human papillomavirus (HPV) in a new proof-of-concept trial. This collaboration not only funds a novel therapeutic area outside of oncology but underscores the versatility of Photosoft as a multi-indication, platform technology. Invion bears none of the clinical trial costs and maintains all rights to future commercialization.
In parallel, Invion is expanding the Photosoft platform into infectious diseases, where preclinical studies have demonstrated broad-spectrum antimicrobial activity. The compound has proven effective in vitro against:
- Antibiotic-resistant “superbugs”
- Fungal and bacterial infections
- SARS-CoV-2 (Omicron)
- Oral and periodontal conditions such as peri-implant mucositis
These results reflect Invion’s long-term vision of developing a scalable, accessible and affordable therapy platform that addresses both high-burden cancers and the growing global threat of antimicrobial resistance. With Photosoft, the company is building a foundation not just for treating disease — but for reshaping therapeutic accessibility and patient outcomes worldwide.
Company Highlights
- Clinical-stage Pipeline in Multiple Indications: Successfully completed Phase II prostate cancer trial, ongoing Phase I/II skin cancer trial, and anogenital cancer trial initiating in 2025. Multiple cancer and infectious disease programs underway.
- Photosoft Platform Technology: Combines cancer selectivity, immune system activation, and minimal toxicity. Preclinical studies show INV043 can regress multiple cancers, deliver superior safety and efficacy and improve tumour control to 80 percent in combination therapy studies with blockbuster ICIs (vs 12 percent with ICIs alone).
- Renowned Partners & Global Pharma-funded Collaborations: Working with distinguished research institutions like Peter MacCallum Cancer Centre and Hudson Institute of Medical Research. Further, Hanlim Pharm (GBM, oesophageal cancer) and Dr.inB (HPV) are funding multiple programs without requiring Invion to contribute capital or give up IP.
- Theragnostic Capability: Photosoft compounds enable both treatment and imaging, allowing for highly precise cancer targeting and enhanced surgical decision-making.
- Strong Clinical and IP Foundation: GMP-grade INV043 manufactured and patented in Australia, with global IP protection extending to at least 2041.
- Compelling Upside: Following a share consolidation and reduced overhangs, IVX offers significant re-rating potential with multiple clinical readouts expected over the next six to 12 months.
Key Programs
Non-melanoma Skin Cancer
Invion’s leading active trial is a Phase I/II adaptive clinical study targeting non-melanoma skin cancer — a condition that represents 98 percent of all skin cancers and constitutes a substantial public health burden in Australia. Using a topical formulation of INV043, the trial is designed with a 3+3 structure allowing real-time protocol optimization for safety and efficacy. The formulation offers significant cosmetic and pain-reduction benefits over existing approved PDT treatments like Metvix (by Galderma S.A.) and excisional surgery, both of which have limitations related to scarring and pain. Dosing of the first patient commenced in December 2024, with ongoing recruitment and interim data expected in the second half 2025 or early 2026. The skin cancer program not only represents a potentially fast path to market but also supports downstream programs, such as the anogenital cancer study, through shared formulation and safety data.
Anogenital Cancers (Peter Mac Collaboration)
The anogenital cancer program is a major upcoming milestone for Invion, developed in collaboration with the prestigious Peter MacCallum Cancer Centre in Melbourne, Australia, one of the leading oncology research centres in the world. Utilizing the same topical INV043 formulation as the non-melanoma skin cancer trial, this Phase I/II study will leverage safety data from the trial to accelerate approval timelines. The anogenital trial targets high-risk lesions and cancers with limited therapeutic options, and benefits from the scientific and operational expertise of Peter Mac. Preclinical data showed exceptional synergy when INV043 was used in combination with immune checkpoint inhibitors (anti-PD-1), achieving up to 80 percent tumor-free responses in models of anal squamous cell carcinoma, compared with a circa 12 percent response rate with aniti-PD-1 alone. This program exemplifies Invion’s theragnostic strength, where the compound also enables fluorescent visualization of tumor margins to aid surgical decision-making.
Prostate Cancer (Phase II Completed)
INV043 has completed a Phase II investigator-led clinical trial in prostate cancer, funded by the RMW Cho Group. The trial involved sublingual systemic administration of the compound followed by targeted light therapy. Following COVID-related disruptions, a second cohort of 16 patients was successfully treated and evaluated using PSMA-PET scans and RECIST criteria. Results were compelling: 44 percent of patients showed no detectable cancer via PSMA-PET scan three months post-treatment, while 40 percent demonstrated partial or stable response by MRI. The therapy was extremely well-tolerated, with no serious adverse events and only mild treatment-emergent side effects. These findings serve as clinical proof-of-concept for systemic delivery of INV043, highlighting its potential for deeper-seated cancers and reinforcing its safety and scalability.
Glioblastoma and Oesophageal Cancer (Hanlim Pharma Collaboration)
Through a strategic partnership with South Korean pharmaceutical company Hanlim Pharma, Invion is advancing preclinical programs for two highly aggressive and deadly cancers: glioblastoma multiforme and oesophageal cancer. Hanlim is funding both programs entirely, allowing Invion to retain all IP and avoid capital outlay. These programs are exploring INV043’s efficacy in some of the most treatment-resistant solid tumors, with early-stage studies underway and updates expected in 2025. If successful, this collaboration could lead to regional licensing or joint ventures in Asia, significantly expanding Invion’s global footprint.
HPV and Infectious Diseases (Dr.inB Collaboration)
Dr.inB, a leading PDT innovator in South Korea, is partnering with Invion to develop Photosoft-based treatments for HPV-related conditions, including genital warts and potentially HPV-linked cancers. The program, which includes proof-of-concept human trials, is entirely funded by Dr.inB, and Invion retains all IP and commercialization rights. Beyond HPV, Photosoft has demonstrated broad-spectrum antimicrobial potential against antibiotic-resistant bacteria, fungi and viruses, including SARS-CoV-2. This opens the door to applications in periodontal disease, peri-implant mucositis, and other infectious conditions. PDT’s unique mechanism of action — using light to generate oxidative stress — renders it immune to resistance development, making it an ideal candidate for combatting antimicrobial resistance, one of the top 10 threats to humanity identified by the World Health Organization.
Management Team
Thian Chew – Executive Chairman & CEO
Thian Chew brings over two decades of executive and advisory experience in healthcare and finance. He is the co-founder of Chronic Airway Therapeutics and a board advisor at Stanford Medicine’s Center for Asian Health Research and Education (CARE). Formerly an executive director at Goldman Sachs and director at KPMG Consulting, Chew combines strategic vision with operational rigor. He is an adjunct professor at the University College London and associate professor at HKUST, holding dual an MBA from Wharton School (Palmer Scholar) and an MA from the Lauder Institute, University of Pennsylvania.
Robert Ramsay – Scientific Advisor
A world-renowned expert in immunotherapy and translational cancer biology, Robert Ramsay is a senior scientist at Peter MacCallum Cancer Centre and has over 30 years of oncology research experience. He was instrumental in demonstrating the synergy between INV043 and checkpoint inhibitors, leading to his appointment as a key advisor. Ramsay also served as president of the Australian Society for Medical Research.
Scott Carpenter – Program Director
Scott Carpenter brings cross-functional expertise in regulatory affairs, business development and stakeholder engagement. He previously held leadership roles at Starpharma, AusBiotech and Bayer CropScience and holds an MBA from Melbourne Business School.
Sebastian Marcuccio – Medicinal Chemistry Lead
The co-inventor of Invion’s PDT patents, Sebastian Marcuccio is the founder of Advanced Molecular Technologies and has a deep background in pharmaceutical R&D, including with CSIRO. He is currently adjunct professor at La Trobe University and holds a PhD in organic chemistry.
Kim Steel – Clinical Trial Director
With more than 18 years of experience managing global Phase I-IV drug and device trials across 14 countries, Kim Steel has worked with Novotech and Pacific Clinical Research. She is managing director of SAPRO Consulting, leading operational delivery for Invion’s ongoing trials.
Alexander Bennett – Technical Advisor, Light Devices
A veteran of scientific instrumentation development, Alexander Bennett brings 35+ years of experience designing medical and forensic light systems. He led PDT light source trials at Peter MacCallum Cancer Centre and ensures the clinical precision of INV043’s light activation protocol.
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Revolutionizing Photodynamic Therapy (PDT) for cancer and infectious diseases
20 August
Orphan Drug Designation Granted by U.S. FDA for Anal Cancer
31 July
Appendix 4C and Quarterly Activities Report - June 2025
29 August
FY25 Preliminary Results Summary and Company Update
29 August
Appendix 4E and Preliminary Final Report
12 August
Top 5 Small-cap Biotech Stocks of 2025
The NASDAQ Biotechnology Index (INDEXNASDAQ:NBI) is trading at three year highs despite market volatility, responding to breakthrough innovations and increased deals involving NASDAQ biotech stocks.
After dropping to a low of 3,637.05 in October 2023, the index climbed to a nearly three year peak of 4,954.813 on September 19, 2024. While the index had pulled back to 4,530.69 as of August 5, 2025, further growth could be in store.
According to a Towards Healthcare analyst report, the global biotech market is expected to grow at a compound annual growth rate of 12.5 percent from now to 2034, reaching a valuation of US$5.04 trillion.
Driving that growth will be favorable government policies, investment in the sector, increased demand for synthetic biology and a rise in chronic disorders such as cancer, heart disease and hypertension.
The top NASDAQ biotech stocks have seen sizeable share price increases over the past year. For those interested in investing in biotech companies, the best-performing small-cap biotech stocks are outlined below.
Data was gathered on August 5, 2025, using TradingView’s stock screener. Small-cap biotech stocks with market caps between US$50 million and US$500 million at that time were considered for this list.
1. Tiziana Life Sciences (NASDAQ:TLSA)
Year-to-date gain: 227.8 percent
Market cap: US$256.36 million
Share price: US$2.26
Tiziana Life Sciences is a clinical-stage biopharma which is developing therapies for autoimmune and inflammatory diseases, degenerative diseases, and cancer-related to the liver. Its pipeline of candidates is built on its patent drug delivery technology that provides a possible alternative to intravenous delivery.
Tiziana’s lead candidate is intranasal foralumab, a fully human anti-CD3 monoclonal antibody.
Tiziana Life Sciences shares hit US$1.69 on March 7 after the company filed an investigational new drug application with the US Food and Drug Administration (FDA) for a Phase 2 clinical trial in amyotrophic lateral sclerosis (ALS), which is supported by the ALS Association. However, by early April it had fallen back to US$0.78 per share.
Positive news flow later in the spring gave Tiziana shares another boost. In April, John Hopkins University and the University of Massachusetts commenced dosing of the biotech company's intranasal foralumab in Phase 2 trials for patients with non-active secondary progressive multiple sclerosis. On May 7, the company shared positive results from the use of its lead candidate in improving the quality of life for patients with that form of multiple sclerosis.
Tiziana is also studying the use of intranasal foralumab for treating moderate Alzheimer’s disease. On May 9, it announced that PET scans of a patient with moderate Alzheimer's showed a significant reduction in microglia activation associated with neuroinflammation after three months of treatment.
Shares of Tiziana reached US$1.62 on May 13. On July 21, the company announced an "unexpected discovery" following immunologic analysis of the patient with Alzheimer's disease:
"In an unexpected discovery, the analysis revealed an increase in phagocytosis markers in classical monocytes, suggesting that nasal foralumab may enhance their ability to clear amyloid plaques. This unexpected effect may open new avenues for treating Alzheimer’s Disease by targeting both inflammation and amyloid accumulation."
Tiziana shares climbed through the remainder of the month, hitting a year-to-date high of US$2.50 on July 31.
2. Palvella Therapeutics (NASDAQ:PVLA)
Year-to-date gain: 224.98 percent
Market cap: US$416.08 million
Share price: US$37.64
Palvella Therapeutics is a clinical-stage biopharma developing treatments targeting rare genetic skin diseases for which there are no FDA-approved therapies. Its pipeline centers on its patented QTORIN platform, which has an initial focus on rare genetic skin diseases. Its lead product candidate, QTORIN rapamycin, is currently in a Phase 2 clinical trial in cutaneous venous malformations, and a Phase 3 clinical trial in microcystic lymphatic malformations (LM).
QTORIN rapamycin has been granted breakthrough therapy designation, orphan drug designation and fast-track designation from the FDA for the treatment of microcystic LMs.
After starting the year at US$12, shares of Palvella had surged to US$20.99 by February 18. About a week earlier, the company had shared plans to expand the Phase 3 trial to include pediatric patients from three to five years of age. That momentum in Palvella's share price continued to rally to US$29 per share on March 13.
June produced a number of significant milestones for Palvella. On June 9, the company received initial proceeds from a grant issued by the FDA Office of Orphan Products Development for its Phase 3 trial, and on June 23, it completed enrollment for the trial with 51 subjects, 25 percent over its target.
The company closed out the month with news that it had been added to the broad-market Russell 3000 Index (INDEXRUSSELL:RUA) and the Russell 2000 Index (INDEXRUSSELL:RUT).
The company said it remains on track to deliver top-line Phase 3 data in Q1 2026 to support its planned new drug application submission later that year. While the company didn't release news in July, its share price climbed significantly through the month to hit a year-to-date high of US$39.87 on July 28.
3. OKYO Pharma (NASDAQ:OKYO)
Year-to-date gain: 163.03 percent
Market cap: US$117.35 million
Share price: US$3.13
OKYO Pharma is a clinical-stage biopharma developing therapies for the treatment of neuropathic corneal pain and dry eye disease. Its lead candidate is urcosimod, a non-steroidal anti-inflammatory and non-opioid analgesic.
So far in 2025, the company has achieved multiple milestones related to its Phase 2 trial of urcosimod for treatment of neuropathic corneal pain. On April 30, OKYO announced plans to end the trial early to analyze the data from the patients who had completed the trial, with the goal of accelerating its clinical development and expanding the program.
Supporting the decision was the fact that urcosimod had previously demonstrated safety in OKYO's completed Phase 2 trial of the candidate to treat patients with dry eye disease.
The next day, news broke that the FDA granted urcosimod fast-track designation for the treatment of neuropathic corneal pain. OKYO's stock price reached US$1.57 on May 1.
On July 17, OKYO posted strong top-line data from its Phase 2 clinical trial, and stated it is planning a meeting with the FDA to discuss next steps for its lead drug candidate. The following day, OKYO received US$1.9 million in non-dilutive funding to support its clinical development of urcosimod.
Shares of OKYO hit a year-to-date high of US$3.17 on August 5.
4. IO Biotech (NASDAQ:IOBT)
Year-to-date gain: 129.47 percent
Market cap: US$144.28 million
Share price: US$2.16
IO Biotech is developing immune-modulating therapeutic cancer vaccines based on its T-win technology platform, designed to activate T cells to target both tumor cells and the immune-suppressive cells.
The clinical-stage biopharmaceutical company's lead cancer vaccine candidate, IO102-IO103, which has the brand name Cylembio, is currently in clinical trials. IO102-IO103 has breakthrough therapy designation from the FDA when used in combination with Merck's (NYSE:MRK) anti-PD-1 therapy KEYTRUDA for the treatment of advanced melanoma based on positive Phase 1/2 first line metastatic melanoma data.
At the start of the year, IO Biotech completed enrollment in its Phase 2 trial of IO102-IO103 with KEYTRUDA as a treatment given before and after surgery for resectable melanoma or head and neck cancer.
On February 4, the company published results from a preclinical study of its second immune-modulatory therapeutic cancer vaccine candidate, IO112, targeting arginase 1, which plays a key role in immune suppression.
In mid-March, IO Biotech was named to Fast Company’s list of the world’s most innovative companies of 2025.
The following month, the company presented new preclinical data for its lead candidate IO102-IO103, as well as IO170, which targets transforming growth factor beta. In its Q1 financial results and business highlights, released on May 14, IO Biotech shared that a readout of primary endpoint data from its pivotal Phase 3 trial of its lead investigational therapeutic cancer vaccine in patients with advanced melanoma is expected in the third quarter of 2025.
Shares of IO Biotech reached a year-to-date high of US$2.40 on July 28.
5. Spero Therapeutics (NASDAQ:SPRO)
Year-to-date gain: 110.95 percent
Market cap: US$124.12 million
Share price: US$2.22
Spero Therapeutics is developing novel treatments for rare diseases and multi-drug resistant bacterial infections with high unmet need. The company’s lead drug candidate is tebipenem pivoxil hydrobromide (HBr), a late-stage development asset developed in collaboration with pharma giant GSK (NYSE:GSK).
HBr is used to treat complicated urinary tract infections (cUTIs), including pyelonephritis. Spero has an exclusive license agreement with GSK for the development and commercialization of the drug candidate in all ex-Asia markets. The FDA has granted tebipenem HBr qualified infectious disease product and fast-track designations.
Shares of Spero traded below US$1 for much of the first half of 2025. However, the stock surged 245 percent on May 28 to reach US$2.35 after Spero reported that its Phase 3 trial evaluating tebipenem HBr for treating cUTIs met its primary endpoint and stopped early for efficacy. GSK plans to include the findings in a filing to the FDA during H2.
Spero shares reached a year-to-date high of US$3.04 on July 9.
Don’t forget to follow us @INN_LifeScience for real-time news updates!
Securities Disclosure: I, Melissa Pistilli, hold no direct investment interest in any company mentioned in this article.
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12 August
SVN-SDN-14 PTSD Programme - Positive pre-clinical results with three high-performing candidates advancing to in vivo studies
Solvonis Therapeutics plc (LSE: SVNS), a clinical-stage biopharmaceutical company developing novel medicines for addiction and mental health disorders, today announces positive results from pre-clinical screening in its SVN-SDN-14 programme, identifying three high-performing candidates to advance into in vivo studies.
The SVN-SDN-14 series is a novel class of serotonin ("SERT"), dopamine ("DAT") and noradrenaline ("NET") modulators designed to enhance pro-social behaviour and improve therapeutic outcomes for people living with Post-Traumatic Stress Disorder ("PTSD"), which the Company estimates affects over 20 million people across UK, US, and main EU markets. By modulating neurochemical pathways associated with trust, empathy, and social bonding, these compounds aim to help patients rebuild interpersonal relationships, reduce social isolation, and engage more effectively in therapy.
In-vitro screening for SERT, DAT, and NET modulation activity identified three of six synthesised candidates with robust, balanced modulation profiles across all three neurotransmitter systems. These are precisely the mechanistic attributes the programme is targeting to deliver a new class of medicine for trauma-related mental health conditions such as PTSD.
While the Company had previously anticipated selecting a single lead candidate at this stage, the positive performance of three candidates has delivered an optimal outcome - a strong, competitive shortlist with multiple best-in-class contenders. This outcome increases optionality, strengthens the dataset and enhances the probability of selecting a best-in-class candidate with strong commercial potential.
The three shortlisted candidates will now advance to the final stage of the current work package - in vivo preclinical studies.
Professor David Nutt, Chief Scientific Officer of Solvonis, commented: "It is encouraging to see three candidates with equally strong, balanced SERT, DAT, and NET modulation profiles. This gives us a solid basis from which to make an informed choice. The in vivo data will enable us to select the candidate with the best potential to advance as a new treatment option for PTSD."
Anthony Tennyson, Chief Executive Officer of Solvonis, added: "These results represent an important step forward for the programme. Having three high-performing candidates at this stage, rather than a single clear lead, provides us with greater flexibility and a stronger position from which to select the most promising option for PTSD."
Enquiries:
Solvonis Therapeutics plc (LEI: 2138005PH7OJRCRPUD88)
Anthony Tennyson, CEO & Executive Director
anthony@solvonis.com
Allenby Capital Limited (Financial Adviser and Joint Broker)
Nick Naylor / Nick Athanas / Ashur Joseph (Corporate Finance)
Guy McDougall (Sales & Corporate Broking)
+44 (0) 20 3328 5656
Singer Capital Markets (Joint Broker)
Phil Davies
+44 (0) 20 7496 3000
About Solvonis Therapeutics plc
Solvonis Therapeutics plc (LSE: SVNS) is a clinical-stage biopharmaceutical company developing novel medicines for addiction and mental health disorders. Headquartered in London and listed on the main market of the London Stock Exchange, Solvonis is advancing a differentiated pipeline of repurposed and novel compounds targeting high-burden neuropsychiatric conditions with significant unmet need.
The Company's lead programmes address Alcohol Use Disorder ("AUD") and Post-Traumatic Stress Disorder ("PTSD")-conditions affecting over 80 million people across the UK, US, and EU4. Its lead asset, SVN-001, is currently in Phase 3 for severe AUD in Europe and the UK. SVN-002 is preparing for a Phase 2b trial in the US targeting moderate to severe AUD. Solvonis also has a preclinical PTSD programme leveraging novel serotonin-dopamine modulators designed to enhance pro-social behaviour and long-term outcomes.
In addition, Solvonis is advancing an AI-supported discovery platform built on a proprietary CNS compound library, with initial focus on depression and stimulant use disorders. This initiative expands the Company's R&D pipeline into earlier-stage innovation while maintaining strategic focus on comorbid and underserved neuropsychiatric conditions.
With a capital-efficient model, dual development strategy, and near-term partnering opportunities, Solvonis is positioned to generate value through innovation in neuropsychiatry.
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11 August
New Harvard Study Links Lithium Deficiency to Alzheimer’s
Lithium, a naturally occurring trace element in the brain, may be able to unlock a key medical mystery: why some people develop Alzheimer’s disease and others don’t, despite similar brain changes.
In a recently published study, scientists at Harvard Medical School state that lithium not only exists in the human brain at biologically meaningful levels, but also appears to protect against neurodegeneration.
Additionally, their work shows that lithium supports the function of all major brain cell types.
The decade-long study drew on mouse experiments and analyses of human brain and blood samples across the spectrum of cognitive health. The Harvard team discovered that as amyloid beta, the sticky protein associated with Alzheimer’s, begins to accumulate, it binds to lithium and depletes its availability in the brain. This drop in lithium impairs neurons, glial cells and other brain structures, accelerating memory loss and disease progression.
“The idea that lithium deficiency could be a cause of Alzheimer’s disease is new and suggests a different therapeutic approach,” said Bruce Yankner, who is the senior author of the study.
Yankner, a professor of genetics and neurology at Harvard Medical School who in the 1990s was the first to show that amyloid beta is toxic to nerve cells, said the new findings open the door to treatments that address the disease in its entirety, rather than targeting single features like amyloid plaques or tau tangles.
To explore this possibility, researchers screened for lithium compounds that could evade capture by amyloid beta.
They identified lithium orotate as the most promising candidate. In mice, the compound reversed Alzheimer’s-like brain changes, prevented cell damage and restored memory, even in animals with advanced disease.
Crucially, the effective dose was about one-thousandth of that used in psychiatric treatments, avoiding the toxicity risk that has hampered lithium’s clinical use in older patients.
“You have to be careful about extrapolating from mouse models, and you never know until you try it in a controlled human clinical trial,” Yankner cautioned. “But so far the results are very encouraging.”
The path to these findings began with access to an unusually rich source of brain tissue.
Working with the Rush Memory and Aging Project in Chicago, the team examined postmortem samples from thousands of donors, from cognitively healthy individuals to those with mild cognitive impairment and full-blown Alzheimer’s.
Using advanced mass spectrometry, they measured trace levels of about 30 metals. Lithium stood out as the only one whose levels dropped sharply at the earliest stages of memory loss.
The pattern matched earlier population studies linking higher environmental lithium levels, including in drinking water, to lower dementia rates. But unlike those correlations, the Harvard team directly measured brain lithium and established a normal range for healthy individuals who had never taken lithium as medication.
“Lithium turns out to be like other nutrients we get from the environment, such as iron and vitamin C,” Yankner said. “It’s the first time anyone’s shown that lithium exists at a natural level that’s biologically meaningful without giving it as a drug.”
To test whether this deficiency was more than an association, the researchers fed healthy mice a lithium-restricted diet, lowering brain lithium to levels seen in Alzheimer’s patients.
The animals developed brain inflammation, lost connections between neurons and showed cognitive decline; however, replenishing them with lithium orotate reversed these changes. What's more, mice given the compound from early adulthood were protected from developing Alzheimer’s-like symptoms altogether.
The findings raise several possibilities. Measuring lithium levels in blood could become a tool for early screening, identifying people at risk before symptoms emerge. Furthermore, amyloid-evading lithium compounds could be tested as preventive or therapeutic agents, potentially altering the disease course more fundamentally than existing drugs.
For now, researchers stress that no one should self-medicate with lithium supplements.
The team emphasized that the safety and efficacy of lithium orotate in humans remain unproven, and clinical trials will be needed to determine whether the dramatic benefits seen in mice translate to people.
Don’t forget to follow us @INN_Resource for real-time news updates!
Securities Disclosure: I, Giann Liguid, hold no direct investment interest in any company mentioned in this article.
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