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This week we received news of a case study involving a cell transplantation procedure that was performed during 2017/2018 on an American gentleman with Parkinson’s.
The operation (conducted in in Boston, USA) involved isolating skins cells from the individual who under went the surgery, then converting those cells into stem cells which were further encouraged to become dopamine neurons before being transplanted into his brain.
Although this is a single subject study (no control group), the result suggests that 2 years on the procedure is safe and well tolerated.
In today’s post, we will discuss the background of this research, review the published results, and explore other aspects of this story.
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On the 12th May, a story was posted on the online news site, STATnews.
I like STATnews (not an endorsement, just me sharing). They have lots of interesting stories covering a wide range of health and biotech topics.
But the story on the 12th May was different.
It focused on clinical study that involved just one participant – a gentleman from Southern California who was afflicated by Parkinson’s. He underwent a procedure called cell transplantation (Click here to read the STATnews story).
What is cell transplantation?
San Diego-based biotech firm Aspen Neuroscience recently announced that it has raised US$70 million in Series A funding to help its efforts to develop the first autologous neuron replacement therapy for treating Parkinson’s.
Cell replacement therapy represents a treatment approach that carries a lot of hope for the Parkinson’s community – providing new cells for the ones that have degenerated in the condition, and taking up lost function.
In today’s post, we will explore what “autologous neuron replacement therapy” means, look at what Aspen Neuroscience is doing, and discuss what will happen next.
In the SoPD ‘Road Ahead’ post at the start of this year (in which we discussed what is planned for Parkinson’s research in 2020 – click here to read that post), I briefly mentioned a biotech firm called Aspen Neuroscience.
It was one of the companies that I was going to be watching this year for signs of progress and development. I had no expectations, but was interested in what they are working on because it is in a rather exciting area of Parkinson’s research.
What does Aspen Neuroscience do?
The company works with stem cells.
It was co-founded by stem cell scientist Prof Jeanne Loring:
Prof Jeanne Loring. Source: SDT
She is a leading expert in the field of stem cell biology. Here is a video of Prof Loring talking about the potential of induced pluripotent stem cells:
What are induced pluripotent stem cells?
Given that the condition is defined by the loss of specific types of neurons in the brain, any future therapy for Parkinson’s should include some form of restorative treatment. Much of the current clinical research exploring restoration in Parkinson’s is focused on cell transplantation – replacing the cells that have been lost in the brain.
But the adult brain is very different to the developing brain. While youngsters have lots of nurturing and supportive protein floating around – encouraging plasticity and survival – once we reach adulthood, our brains appear to be full of inhibitory molecules that reduce rejuvenation in the case of injury.
What if we could re-introduce some of those supportive factors and remove the inhibitory proteins? Could this help with restorative therapies for Parkinson’s?
In today’s post, we will look at new research exploring how we may be able to reduce some of those inhibitory factors and provide a more supportive environment for restorative therapy in Parkinson’s.
As we regularly state here on the SoPD, any ‘curative therapy’ for Parkinson’s is going to require three core components:
- A disease halting mechanism
- A neuroprotective agent
- Some form of restorative therapy
Now, the bad news is (as far as I am aware) there is no single treatment currently available (or being tested) that can do all three of these things. By this I mean that there is no disease halting mechanism therapy that can also replace lost brain cells. Nor is there a restorative therapy that stop the progression of the condition.
That statement can obviously be read as bad news, but it shouldn’t.
Let me explain:
A curative therapy for Parkinson’s is going to need to be personalised to each individual, with varying levels of each of the three component listed above. It will be a multi-modal approach designed for each individual’s needs.
Making things personal. Source: Flickr
By this I mean, there is a great deal of heterogeneity (or variability) between individuals with regards to their symptoms and the amount of time that they have had the condition. Some folks are more tremor dominant, while others do not experience tremor at all. Likewise, some individuals have only just been diagnosed, while others have lived with the condition for many years.
The treatment needs of each individual will be different, and thus what we will require is different amounts of the disease halting mechanism component, the neuroprotection component, and the restorative therapy components for each affected person.
In today’s post we are going to explore some alternative approaches being tested for restorative therapy.
What do you mean ‘alternative’?
Cell replacement therapy is a key component of any “cure” for Parkinson’s – replacing the cells that have been lost over the course of the condition.
Cell transplantation of dopamine neurons has a long track record of both preclinical and clinical development and represents the most developed of the cell replacement approaches.
Two weeks ago, the biotech firm BlueRock Therapeutics announced an agreement under which the pharmaceutical company Bayer AG would fully acquire the company.
In today’s post we will discuss why this is major news for the Parkinson’s community and an important development for the field of cell replacement therapy.
On the 8th August, Bayer AG and BlueRock Therapeutics announced an agreement under which Bayer will “fully acquire BlueRock Therapeutics, a privately held US-headquartered biotechnology company focused on developing engineered cell therapies in the fields of neurology, cardiology and immunology, using a proprietary induced pluripotent stem cell (iPSC) platform” (Source).
What is BlueRock Therapeutics?
Versant Ventures is a leading venture capital firm that specializes in investing “in game changing biopharmaceuticals, medical devices, and other life science opportunities”. Leaps by Bayer is an effort by the Pharmaceutical company Bayer at “spearheading a movement to make paradigm-shifting advances in the life sciences – targeting the breakthroughs that could fundamentally change the world for the better”.
The news on the 8th August means Bayer will acquire the remaining stake for approximately US$240 million in cash (to be paid upfront) and an additional US$360 million which will be payable upon the achievement of certain pre-defined development milestones.
Given that Bayer currently holds 40.8% stake in BlueRock Therapeutics, this announcement values the company at approximately US$1 billion.
Interesting, but what exactly does BlueRock do?
On the 12th and 13th November, Parkinson’s UK held their biennial research conference in York.
It is not only an opportunity for the charity to showcase some of the research that they have funded over the last few years, but it was also a chance for members of the Parkinson’s research community to come together to share ideas, network and form new collaborations.
I was lucky enough to attend the event this year, and wanted to share some of the take away messages from the conference with the readers.
In today’s post, we will review Parkinson’s UK 2018 research conference (#Parkinsons2018).
Parkinson’s UK is the largest Parkinson’s research and support charity in the United Kingdom. Since 2015, they have invested over £18 million in a variety of research projects focused on all aspects of Parkinson’s – from new experimental treatments to the Parkinson’s UK Brain Bank.
Every two years, Parkinson’s UK holds a conference highlighting some of the research that the organisation has funded over the last few years. The meeting is usually held in the beautiful walled city of York – lots of history and narrow streets to explore.
Th “The Shambles” in York. Source: hauntedrooms
Parkinson’s is a neurodegenerative condition. This means that cells in the brain are being lost over time. Any ‘cure’ for Parkinson’s is going to require some form of cell replacement therapy – introducing new cells that can replace those that were lost.
Cell transplantation represents one approach to cell replacement therapy, and this week we learned that the Japanese regulatory authorities have given the green light for a new cell transplantation clinical trial to take place in Kyoto.
This new trial will involve cells derived from induced pluripotent stem cells (or IPS cells).
In today’s post we will discuss what induced pluripotent stem cells are, what previous research has been conducted on these cells, and what we know about the new trial.
Source: Glastone Institute
The man in the image above is Prof Shinya Yamanaka.
He’s a rockstar in the biomedical research community.
Prof Yamanaka is the director of Center for induced Pluripotent Stem Cell Research and Application (CiRA); and a professor at the Institute for Frontier Medical Sciences at Kyoto University.
But more importantly, in 2006 he published a research report that would quite literally ‘change everything’.
In that report, he demonstrated a method by which someone could take a simple skin cell (called a fibroblast), grow it in cell culture for a while, and then re-program it so that it would transform into a stem cell – a cell that is capable of becoming any kind of cell in the body.
The transformed cells were called induced pluripotent stem (IPS) cell – ‘pluripotent’ meaning capable of any fate.
It was an amazing feat that made the hypothetical idea of ‘personalised medicine’ suddenly very possible – take skin cells from anyone with a particular medical condition, turn them into whatever cell type you like, and then either test drugs on those cells or transplant them back into their body (replacing the cells that have been lost due to the medical condition).
Personalised medicine with IPS cells. Source: Bodyhacks
IPS cells are now being used all over the world, for all kinds of biomedical research. And many research groups are rushing to bring IPS cell-based therapies to the clinic in the hope of providing the long sort-after dream of personalised medicine.
This week the Parkinson’s community received word that the Pharmaceuticals and Medical Devices Agency (PMDA) – the Japanese regulatory agency that oversees clinical trials – have agreed for researchers at Kyoto University to conduct a cell transplantation trial for Parkinson’s, using dopamine neurons derived from IPS cells. And the researchers are planning to begin their study in the next month.
In today’s post we are going to discuss this exciting development, but we should probably start at the beginning with the obvious question:
What exactly is an IPS cell?
Chinese researchers recently published pre-clinical research demonstrating the use of their protocol for generating stem cell-derived neurons for cell transplantation in Parkinson’s.
The data represents the last step/proof-of-principle stage for taking this procedure into clinical trials (which are ongoing).
In today’s post, we will discuss what cell transplantation is, we will review the new data, and we will consider some of the issues associated with taking this procedure to the clinic.
Brain surgery. Source Bionews-tx
As we have discussed before, any ‘cure’ for Parkinson’s requires 3 components:
- A disease halting mechanism – slowing or stopping the progression of the condition
- A neuroprotective agent – a treatment that will protect and support the remaining cells
- Some form of cell replacement therapy – introducing new cells to replace the ones that have been lost
Now, the bad new is that there is no ‘silver bullet’ on the horizon that provides all three (for example, there is no neuroprotective agent that also replaces lost cells).
But the good news is that we have a great deal of clinical research being conducted in all three of these areas. This video provides an overview of just some of the many different ways we are approaching all three components:
Recently a research report focused on a cell transplantation (a form of cell replacement therapy) approach for Parkinson’s was published by a group of researchers in China. They have proposed that the results presented in the report justify their efforts to take this approach forward into clinical testing.
What is cell transplantation?
The great ice hockey player Wayne Gretzky once said “A good hockey player plays where the puck is. A great hockey player plays where the puck is going to be” (the original quote actually came from his father, Walter).
At the start of each year, it is a useful practise to layout what is planned for the next 12 months. This can help us better anticipate where ‘the puck’ will be, and allow us to prepare for things further ahead.
2017 was an incredible year for Parkinson’s research, and there is a lot already in place to suggest that 2018 is going to be just as good (if not better).
In this post, we will lay out what we can expect over the next 12 months with regards to the Parkinson’s-related clinical trials research of new therapies.
Charlie Munger (left) and Warren Buffett. Source: Youtube
Many readers will be familiar with the name Warren Buffett.
The charming, folksy “Oracle of Omaha” is one of the wealthiest men in the world. And he is well known for his witticisms about investing, business and life in general.
Warren Buffett. Source: Quickmeme
He regularly provides great one liners like:
“We look for three things [in good business leaders]: intelligence, energy, and integrity. If they don’t have the latter, then you should hope they don’t have the first two either. If someone doesn’t have integrity, then you want them to be dumb and lazy”
“Work for an organisation of people you admire, because it will turn you on. I always worry about people who say, ‘I’m going to do this for ten years; and if I really don’t like it very much, then I’ll do something else….’ That’s a little like saving up sex for your old age. Not a very good idea”
“Choosing your heroes is very important. Associate well, marry up and hope you find someone who doesn’t mind marrying down. It was a huge help to me”
Mr Buffett is wise and a very likeable chap.
Few people, however, are familiar with his business partner, Charlie Munger. And Charlie is my favourite of the pair.
Recently I was invited to speak at the 6th Annual East Midlands Parkinson’s Research Support Network meeting at the Link Hotel, in Loughborough. The group is organised and run by the local Parkinson’s community and supported by Parkinson’s UK. It was a fantastic event and I was very grateful to the organisers for the invitation.
They kindly gave me two sessions (20 minutes each) which I divided into two talks: “Where we are now with Parkinson’s research?” and “Where we are going with Parkinson’s research?”. Since giving the talk, I have been asked by several attendees if I could make the slides available.
The slides from the first talk can be found by clicking here.
I have also made a video of the first talk with a commentary that I added afterwards. But be warned: my delivery of this second version of the talk is a bit dry. Apologies. It has none of my usual dynamic charm or energetic charisma. Who knew that talking into a dictaphone could leave one sounding so flat.
Anyways, here is the talk – enjoy!
I hope you find it interesting. When I have time I’ll post the second talk.
Two months ago a research report was published in the scientific journal ‘Nature’ and it caused a bit of a fuss in the embryonic stem cell world.
Embryonic stem (ES) cells are currently being pushed towards the clinic as a possible source of cells for regenerative medicine. But this new report suggested that quite a few of the embryonic stem cells being tested may be carrying genetic variations that could be bad. Bad as in cancer bad.
In this post, I will review the study and discuss what it means for cell transplantation therapy for Parkinson’s disease.
For folks in the stem cell field, the absolute go-to source for all things stem cell related is Prof Paul Knoepfler‘s blog “The Niche“. From the latest scientific research to exciting new stem cell biotech ventures (and even all of the regulatory changes being proposed in congress), Paul’s blog is a daily must read for anyone serious about stem cell research. He has his finger on the pulse and takes the whole field very, very seriously.
For a long time now, Paul has been on a personal crusade. Like many others in the field (including yours truly), he has been expressing concern about the unsavoury practices of the growing direct-to-consumer, stem cell clinic industry. You may have seen him mentioned in the media regarding this topic (such as this article).
The real concern is that while much of the field is still experimental, many stem cell clinics are making grossly unsubstantiated claims to draw in customers. From exaggerated levels of successful outcomes (100% satisfaction rate?) all the way through to talking about clinical trials that simply do not exist. The industry is badly (read: barely) regulated which is ultimately putting patients at risk (one example: three patients were left blind after undergoing an unproven stem cell treatment – click here to read more on this).
While the stem cell research field fully understands and appreciates the desperate desire of the communities affected by various degenerative conditions, there has to be regulations and strict control standards that all practitioners must abide by. And first amongst any proposed standards should be that the therapy has been proven to be effective for a particular condition in independently audited double blind, placebo controlled trials. Until such proof is provided, the sellers of such products are simply preying on the desperation of the people seeking these types of procedures.