Summary: The Main Takeaways
The amount of research being conducted is a very positive sign, and it suggests that 2022 will be an extremely productive year for Parkinsons research. The breadth of approaches now being applied to Parkinsons is also very encouraging, and in the next few years we may have answers to some fundamental questions regarding the biology that may be underlying many cases of PD .
There is certainly going to be a lot of clinical trial results being announced in 2022! The top 5 clinical trial results that I will be looking out for this year are:
- The Phase II UP study results evaluating UDCA in Parkinsons
- The Phase II Lixisenatide study results more data on GLP-1R agonists in PD
- The Phase II Liraglutide study results even more data on GLP-1R agonists in PD
- The Phase II Peptron study results lots of data on GLP-1R agonists in 2022
- The Phase II Deferiprone study results addressing an important question
And with bulging pockets, we can expect more acquisitions and partnering deals to come in 2022. Any fears that big pharma companies are quitting neurodegeneration appear to be misplaced with aging demographics in the Western world, the market opportunities are simply too big for these enormous companies to ignore.
All of this collective activity provides encouraging signs for future research focused on finding new therapies for slow, stopping and reversing Parkinsons.
But now its time to give the fingers a wee rest.
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Biolamina And Novo Nordisk Partner To Advance Stem Cell Based Therapies For Three Common Medical Conditions
BioLamina and collaborators have partnered with Novo Nordisk A/S , a Danish multinational pharmaceutical company, allowing Novo Nordisk to develop novel stem cell based therapies based on Biolaminins, human recombinant laminin cell culture matrices, developed and produced by BioLamina.
Malin Parmars research group at Lund University and Karl Tryggvasons research group at Duke-NUS in Singapore have developed potential cell therapies based on BioLaminas unique Biolaminin matrices. Both groups have signed research and potential licensing agreements with Novo Nordisk with the aim to develop these projects further together with Novo Nordisk, enabling a translation of cell therapy preclinical studies, to clinical trials and eventually towards patient treatments.
The timing for these licensing agreements coincides well with the launch of BioLaminas first cell therapy grade product a couple of weeks ago, assuring cell therapy developers worldwide that Biolaminas products can be used all the way from the bench to bedside. There is an ongoing trend that researchers using embryonic stem cell or induced pluripotent based protocols want to use BioLamina products in their product development to make their processes more robust and reproducible.
For more information, please contact:
BIOLAMINA AB
A Leading Laboratoy In Denmark
It is not only in academia reNew has caused excitement. Pharmaceutical companies jostling to enter the field of stem cells are also welcoming the research center. Many companies hope that the establishment of a research center of this caliber will attract sought-after researchers to Denmark and inspire valuable collaborations.
According to Rasmus Beedholm-Ebsen, Special Advisor within Life Sciences at Invest in Denmark, the foundation of reNew will have a huge impact on companies within the stem cell field. The research center will significantly support stem cell research and have a big impact on the education and employment of skilled people within the field. This will attract Danish and foreign researchers to Denmark and hopefully lead to new, important scientific discoveries.
The research center also aims to translate discoveries to tangible products, e.g. mini-organs. Potentially developed in cooperation with the pharmaceutical industry, mini-organs can be used to test new drug candidates. Rasmus Beedholm-Ebsen expects that some of the researchers at reNew will find partnerships with major drug companies.
To read more on the Danish life sciences industry, please head to our fact page here.
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Clinical Trials To Study The Efficacy Of Semaglutide In Treatment Of Nash
A phase-2 trial has been conducted on 320 patients with NASH and fibrosis to explore the efficacy and safety of daily-once s.c. semaglutide. The subjects were randomly assigned to be given 0.1 mg or 0.2 mg or 0.4 mg semaglutide or daily-once s.c. placebo for 72 weeks. All the three doses of semaglutide improved NASH without worsening fibrosis in 40%, 36%, and 59% patients respectively as compared to 17% in placebo. However, no significant difference between various treatment groups was observed in improving fibrosis. Semaglutide was well tolerated in all the three doses but without any significant improvement in fibrosis .
A proof of concept, phase-2 trial explored the effectiveness of semaglutide, when taken alone or with firsocostat and cilofexor for treatment of NASH. This study involved 108 patients with NASH to take either weekly-once s.c. semaglutide , or semaglutide+daily-once oral 20 mg firsocostat, or semaglutide+daily-once oral 30 mg cilofexor, or semaglutide+daily-once oral 100 mg cilofexor, or semaglutide+daily-once oral 20 mg firsocostat+daily-once oral 30 mg cilofexor for 24 weeks. Significant improvement in NASH and fibrosis was observed with semaglutide monotherapy, but the combination regimen improved further reduction in hepatic steatosis. Semaglutide along with the combination regimen was well tolerated, but highest impact was observed in NAFLD activity score due to the combination regimen .
New International Research Center To Drive Future Stem Cell
The new international stem cell research center is a collaboration between the University of Copenhagen, Denmark, Murdoch Childrens Research Institute, Australia, and Leiden University Medical Center, The Netherlands. These three world-leading research institutes aims to achieve a deep understanding of stem cell biology in organ development, tissue repair and disease mechanisms, which is essential to harness the therapeutic potential in stem cell medicine.
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Nyscf Robertson Investigator Partners With Novo Nordisk To Produce Therapy For Parkinsons Disease
NYSCF Robertson Investigator Malin Parmar, PhD, Professor at Lund University, Sweden, is beginning a collaboration with Novo Nordisk, one of the worlds largest pharmaceutical companies, to develop a large-scale cell therapy for Parkinsons disease.
Dr. Parmars lab has spent the last ten years studying the cellular basis of Parkinsons disease and developing methods for turning stem cells into dopamine neuronsthe cells lost in the disease. This new partnership with Novo Nordisk will allow her to scale up the research and development of her stem cell therapy, accelerating its progress to clinical trials.
To develop a therapy that can benefit patients all over the world, you need financial resources, expertise in global regulation issues, resources to carry out clinical trials, and an infrastructure that enables large scale development and commercialization, explains Dr. Parmar. All of this is available within Novo Nordisk, and we are very happy to have them as a partner in driving our research through to clinical trials.
For more information, see the press release from Lund University.
One Clinical Trial After Another Has Failed
More than 30 years have elapsed since researchers seeking the cause of Parkinsons disease began investigating GDNF . The dopamine-producing neurons, a type of neurotransmitter essential for controlling human movement, degenerate in Parkinsons disease. This can be easily treated with oral dopamine in the short term, but over time this becomes less and less effective as the side-effects increase. In the 1990s, researchers discovered GDNF.
In animals, GDNF appeared to be able to protect the dopamine-producing neurons in the brain from cell death. Clinical trials were therefore quickly initiated. The first ones probably failed because of insufficient doses of GDNF being delivered to the brain tissue. Since then, one clinical trial after another has failed each time the suspected reason was that either the disease had progressed too far or that the doses were too low to be effective, explains Agnete Kirkeby.
However, expectations were high for the latest clinical study from the Bristol Medical School at the University of Bristol, which generated considerable publicity, including a BBC documentary The Parkinsons Drug Trial: A Miracle Cure? Nevertheless, once again, the results were disappointing. Admittedly, GDNF treatment seemed to improve the motor abilities of people with Parkinsons disease, but unfortunately the difference from the control group was not statistically significant.
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More Than 6 Million People Have The Highly Debilitating Parkinsons Disease Only The Symptoms Of Parkinsons Disease Can Be Alleviated And A Promising Cure Has Been Unsuccessful In Contrast Stem Cell Transplantation Offers A Glimmer Of Hope The First Clinical Trials Involving Patients Will Begin In 2021 And If These Replicate The Convincing Results From Preclinical Studies In Rats And Pigs Stem Cell Treatment Might Be Realistic In 57 Years
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The early signs are shaking, stiffness and slower movements. The central nervous system of people with Parkinsons disease degenerates slowly, and this often leads to difficulties in walking combined with sensory, behavioural and emotional problems. Daily medication can alleviate the symptoms, but the effect of the medicine diminishes around 7 to 15 years after disease onset, and most people with Parkinsons disease then have severely debilitating symptoms that cannot be alleviated without severe side-effects. Today, more than 6 million people worldwide have Parkinsons disease, and that number is increasing as global populations age. Researchers are therefore still struggling to understand and treat Parkinsons disease, but so far all these efforts have been in vain.
Clinical Trials For Alzheimers Disease
Animal Studies
Zhang et al. have demonstrated that daily-once semaglutide improved the motor impairment, reduced the oxidative injury, inflammation and apoptosis in methyl-4-phenyl-1,2,3,6-tetrahydropyridine mice model . A similar study carried out by the same research group has shown that semaglutide protects dopaminergic neurons from injury by reducing the aggregation of -synuclein and by enhanced expression of glial cell line-derived neurotrophic factor , a growth factor. It also augmented the dopamine levels by supplementing tyrosine hydroxylase levels in brain . Yang et al. have demonstrated the neuroprotective efficacy of semaglutide in the rat model of stroke through decreased inflammation, apoptosis and stabilizing the neurogenesis process .
Human Studies
Chang et al. have demonstrated the neuroprotective property of semaglutide in human neuroblastoma cell line against amyloid- plaques , possibly mediated by enhanced autophagy and inhibition of apoptosis . Various pre-clinical and phase-2 study results supported 14 mg oral semaglutide to proceed for phase-3 clinical trials on Alzheimers patients .
Two placebo-controlled phase-3 trials have been initiated to study the efficacy of oral semaglutide in patients with early Alzheimers disease. Change in clinical dementia rating, time taken to reach dementia, change in the Alzheimer’s Disease Composite Score are some of the important outcomes that would be assessed from these trials .
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International Stem Cell Research
I am very excited about the amazing opportunity that reNEW represents. Building on the stem cell research excellence that exists within all partner institutions, the Center will reach a critical mass that is required for translating fundamental discoveries into stem cell medicine. The international collaboration that forms the basis for the new Center will provide access to extensive technical and clinical translation expertise across all sites. Across the breadth of stem cell medicine this will lead to new drugs based on human stem cell models, cell and tissue therapies and novel cell and gene therapies
Melissa Little Professor, Murdoch Children’s Research Institute and CEO of the reNew partnership
Collaboration Between Lund University Researchers And Novo Nordisk Paves The Way For Large
One of the worlds largest pharmaceutical companies, Novo Nordisk, are starting a new stem cell program for the treatment of Parkinsons disease in close collaboration with Lund University.
In order to develop a therapy that can benefit patients all over the world you need financial resources, expertise within global regulation issues, resources and expertise to carry out clinical trials, and an infrastructure that enables large scale development and commercialization. All of this is available within Novo Nordisk, and we are very happy to have them as a partner in driving our research through to clinical trials. Says Malin Parmar, professor of cellular neuroscience and research team leader.
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Million Euros For New International Stem Cell Consortium
The Leiden University Medical Center , the Danstem Institute from the University of Copenhagen and the Murdoch Childrens Research Institute in Melbourne have received 300m euros from the Novo Nordisk foundation. The aim of this new international consortium is to bring stem-cell based therapies from the lab to the patient.
Stem cells offer a unique opportunity to repair tissue that has become damaged by disease or trauma. The possible applications of these cells are promising for the treatment of diabetes, metabolic diseases and hereditary disorders, but much work is still needed to get these stem-cell therapies to patients. To accelerate this, the Novo Nordisk Foundation is investing around 100m euros in each of the three institutes that together form the new reNEW consortium. The funding is for a period of ten years.
Component #3 Some Form Of Restorative Therapy
Once the condition has been slowed/halted and a neuroprotective/nurturing environment is in place to protect the remaining cells , a curative treatment for Parkinsons will require replacing some of the cells that have been lost.
And until we have developed methods that can identify Parkinsons long before the motor features appear , some form of cell replacement therapy is required to introduce new cells to take up lost function.Cell transplantation currently represents the most straight forward method of cell replacement therapy.
Cell Transplantation
Traditionally, the cell transplantation procedure for Parkinsons has involved multiple injections of developing dopamine neurons being made into an area of the brain called the putamen . These multiple sites allow for the transplanted cells to produce dopamine in the entire extent of the putamen. And ideally, the cells should remain localised to the putamen, so that they are not producing dopamine in areas of the brain where it is not desired .
Targeting transplants into the putamen. Source: Intechopen
Transplanted dopamine neurons. Source: Sciencedirect
The transplanted cells take several years to develop into mature neurons after the transplantation surgery. This means that the actually benefits of the transplantation technique will not be apparent for some time . Once mature, however, it has also been demonstrated that these transplanted cells can produce dopamine.
Source: Fujifilm
Ok, thats it.
I think we are done.
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Researchers Think These Factors May Be Linked To Parkinsons
These results are an encouraging and critical step in our pursuit of therapies that slow or stop Parkinsons progression, Patrik Brundin, MD, PhD, a professor of neuroscience and the director of the Parkinsons Disease Center at Van Andel Institute in Michigan, said in a press release
Both the Van Andel Institute and the U.K.s Cure Parkinsons funded the trial.
We believe diabetes medications, such as liraglutide, hold particular promise and look forward to additional results from the trial, added Brundin, who is also chair of Cure Parkinsons International Linked Clinical Trials program, which aims to repurpose approved medications to treat Parkinsons and supported the liraglutide study.
Additional trial results are expected later this year and in 2023.
Increasing evidence suggests that GLP-1R agonists, which can reduce appetite and help to control sugar levels in the body by promoting insulin production, may be potential therapies for Parkinsons.
Previous studies showed that GLP-1R agonists have neuroprotective effects resulting in motor and non-motor improvements in animal models of Parkinsons and suggest that people taking such medications are at a lower risk of developing Parkinsons.
In addition, data from a previous Phase 2 clinical trial showed that exenatide, a short-acting GLP-1R agonist sold as Byetta, was superior to a placebo at aiding motor function in Parkinsons patients on standard therapy.
Component #2 A Neuroprotective Agent
Once a drug or a treatment has been determined to slow down the progression of Parkinsons, it will be necessary to protect the remaining cells and provide a nurturing environment for the third part of the cure .
Neuroprotection is the area of research that has had the most attention over the years. Drug companies have employed vast resources in this area in the hope of discovering a treatment which will work across conditions , and thus provide them with tremendous profits. Unfortunately, conditions of the brain have proven to be a lot more complicated than first perceived and cross-condition therapies seem unlikely as we move towards greater stratification and personalisation of disease and treatment, respectively.
But there has been the hint of a potential neuroprotective effect in one class of drugs for Parkinsons: GLP-1R agonists.
Neuroprotective approach: GLP-1R agonists
Exenatide is a glucagon like peptide-1 receptor agonist. This is a class of drug that has traditionally been used for treating diabetes, but has recently been repurposed for Parkinsons.
After multiple studies suggested neuroprotective properties in models of Parkinsons, a clinical trial program was intiated, and in 2017, a Phase II Exenatide trial reported the stablisation of Parkinsons motor features over the course of the 48 week trial .
Reduction in motor scores in Exenatide group. Source: Lancet
In late 2019, we saw the initiation of a Phase III clinical trial for .
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Cell Therapies For Parkinson’s Disease
CopyrightClinical and Translational Sciencecited by
Today we can treat many diseases symptomatically using both small molecules and biologics, and although effective, our repertoire of medicines that focuses on treating the cause of the disease is limited. Few therapeutic approaches have been designed to truly restore function. If done right, they can be very effective, often bringing lifelong therapeutic benefit to the patient. In this commentary, we will discuss how these principles are applied to living cell therapies for Parkinson’s disease.
As Several Big Pharma Companies Drop Parkinsons Projects Attention Turns To Gene Therapies And Repurposing Diabetes Drugs
Parkinsons disease research has ended in numerous dead ends despite substantial efforts over many years. Recently, Biogen and Sanofi scrapped their Parkinsons candidates, cipanemab and venglustat respectively, owing to lack of efficacy, and a disease-modifying therapy has yet to materialise.
But the push to find drugs that help beyond reducing symptoms continues, and Evaluate Vantage has delved into the pipeline of projects in active late-stage clinical trials. This year is shaping up to be crucial for the field, with 10 studies expected to yield data or to complete in 2021.
One target that crops up multiple times is GLP-1 this approach, traditionally employed in type 2 diabetes, is also being tested in Alzheimers. Among other avenues of research, it is hoped that gene therapy could offer a one-time cure for Parkinsons.
Repurposing
Research has suggested that GLP-1 agonists have neuroprotective benefits, and several trials of marketed diabetes drugs, as well as new GLP-1-targeting projects, are under way in Parkinsons. Some of these studies are investigator sponsored, including the most advanced, a UCL-run phase III trial of Astrazenecas Bydureon called Exenatide-PD3.
In the meantime, data are expected from several phase II studies of GLP-1 agonists, including a trial of Novo Nordisk’s Victoza, being run by Cedars-Sinai Medical in collaboration with the Danish company and The Cure Parkinson’s Trust. That study is set to complete in September.
Gene therapies
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