Senescent Cell Clearance Therapies Could Have a Transformative Effect on Medicine

Senescent Cell Clearance Therapies Could Have a Transformative Effect on Medicine
Source: © koya979 - Fotolia.com

 

In a recent post in Signals, the blog hosted by the Canadian Centre for the Commercialization of Regenerative Medicine, I outline recent developments in the race to develop senescent cell clearance therapies.

The idea of developing biomedical technologies that can repair the underlying cellular and molecular damage that drives the aging process as a way of treating age-related diseases and extending healthy life span is rapidly gaining traction in the biotechnology community. This type of work is now being crowdfunded, part of a trend in crowdfunding regenerative medicine R&D.

The hypothesis that clearing senescent cells could have a rejuvenating effect received a huge boost this past winter:

In a landmark 2012 study, the Mayo Clinic’s Darren Baker and colleagues established the first proof of concept that senescent cells cause age-related functional decline. They genetically modified progeroid mice – which appear to age rapidly, much like humans affected with progeria – in a way that allowed them to selectively remove a class of senescent cells from the mice’s bodies by administering a drug that induced apoptosis (cell death) in these cells. Crucially, they found that clearing senescent cells on a regular basis delayed the development of age-related conditions in the mice’s skeletal muscle, fat and eyes.

 

While this study provided a proof of concept using a clever design, the extent to which the findings were generalizable to natural aging was unclear, so Baker and colleagues conducted a follow-up study using naturally-aged mice, published this past February in Nature. They found that clearing senescent cells every two weeks increased the median lifespan of male and female mice by up to 35%, and extended their healthspan by delaying the onset of cancer and attenuating age-related deterioration in the kidneys, heart and fat – all with no observed side effects.

You can read the rest of the post HERE.

The Science of Senescent Cells and Aging

For more on the science of senescent cells and their role in aging, watch this talk by Judith Campisi of the Buck Institute for Research on Aging, who is a leading scientist in the field and an advisor to senescent cell clearance startup UNITY Biotechnology.

Crowdfunding Senescent Cell Clearance Therapies

To do your part to accelerate the development of senescent cell clearance therapies, you can also donate to the Major Mouse Testing Program crowdfunding campaign:

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This Biotech Startup Is Crowdfunding its R&D After Turning Down a $1M Investment

This Biotech Startup Is Crowdfunding its R&D After Turning Down a $1M Investment
© Remedy Plan

 

In this guest post, Allison Crimmins (Director of Strategy at Remedy Plan) tells us why her biotech startup decided to raise $100,000 via crowdfunding instead of taking the $1 million in traditional risk capital that was on the table. The funds will help to finance its search for drug candidates that block cancer stem cells’ ability to multiply, thereby preventing metastasis, drug resistance, and the recurrence of tumors. You can support Remedy Plan’s research HERE.

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It’s not easy turning down a million dollars. Especially when there isn’t a line of investors beating down your door. And saying no to a big investment in favor of running a crowdfunding campaign may sound crazy, but I think we made the right decision.

I’m part of a company called Remedy Plan. We’re a startup biotech company with an innovative technology that allows us to develop drugs that stop the spread of cancer. Our crowdfunding campaign just launched on Monday at fund.remedyplan.com, and made it past 10% of our goal in the first day. We may not have originally planned on going the route of crowdfunding, but it now plays a very important role in our overall business strategy.


Back in 2013, the co-founders of the company, two former classmates at Berkeley, recognized that the work they were doing on embryonic stem cell biology had implications for measuring similar properties in cancer cells… and they dropped everything to focus on this big idea. Greg Crimmins, who invented a promising new way to treat cancer with immunotherapy (patent licensed by Aduro Biotech), immediately knew he had to switch career streams and build a company that would conduct these new experiments. Remedy Plan was born.

“The scope of it blew my mind,” recalls Greg, “I couldn’t imagine a more important series of experiments that needed to be done.” The series of experiments he had in mind could lead to the development of new cancer drugs that target the spreading properties in cancer cells. Rather than attacking both cancer cells and healthy cells, like traditional therapies do, these drugs will be used in Cancer Containment Therapy. Because the spreading properties they identified are shared by many types of cancers but not by healthy mature cells, Cancer Containment Therapy would be applicable to multiple types of cancer without being toxic, including cancers of the breast, lung, liver, prostate, skin, and brain.

Just one problem: that well known funding gap in the drug development process, dubbed the “Valley of Death”, that lies between preliminary tests on a big idea and identifying drug candidates. Or as NIH Director Francis Collins puts it: “where great ideas, unfortunately, go to die”.

stages of drug development

© Remedy Plan, used with permission.

Greg and his partner Ron Parchem pitched their companies to several San Francisco area investors, and at first were met with some good luck. With the backing of two respected National Academy scientists on their advisory board, Dr. Frank McCormick at UCSF and Dr. Dan Portnoy of UC Berkeley, they were offered several investments that totaled over $1M. But because the company was in such an early stage, the two founders were unable to negotiate acceptable investment terms. Unwilling to turn over the scientific direction of the company, they turned down the offers.

Realizing that they weren’t going to get the kind of terms they desired, the company took a serious turn: towards crowdfunding. While strengthening the core business (IP, business plan, talking to industry partners, etc) and finalizing a patent submission, the Remedy Plan team also spent the next year working through branding exercises, ironing out language and tone, developing a website, a communications strategy, and the foundations of a crowdfunding campaign.

There are a few reasons for refocusing on crowdfunding. Primarily, the money raised over the campaign, along with some smaller private investments, will allow the company to rent lab space, purchase equipment, and run an initial drug screen. Armed with the proof-of-concept results of this initial screen—plus a cadre of supporters who believed in the idea enough to chip in their own money to see the research happen— Remedy Plan will be in a much better position to negotiate the terms of future investments needed to develop and optimize drug candidates prior to clinical trials. Crowdfunding is the bit of momentum needed to take the first leap across the “Valley of Death”.

But I also believe that there is a secondary benefit to crowdfunding scientific research. Crowdfunding works to tap into people’s inherent curiosity about the world and allows them to be a part of the discovery process. At Remedy Plan, we know that relying on the generosity of people comes not just with gratitude, but with the responsibility to be upfront and honest about our process. Crowdfunding campaigns like ours enable people to get directly involved in funding research, follow the progress of specific researchers, and share in their successes. It works to demystify science and make it accessible to a wider audience.

Our campaign just launched, so it’s still too early to tell if we’ll hit our $100K goal. Regardless, I’m convinced that launching a crowdfunding campaign has strengthened our company and that the mechanism is a viable, beneficial, and exciting tool that will only continue to become more prevalent in today’s biotech startup world.

 

 

 

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The Patient Empowerment Trifecta: Personalized Medicine, Digital Health, and Crowdfunding

The Patient Empowerment Trifecta: Personalized Medicine, Digital Health, and Crowdfunding
© ZIQUIU - Fotolia.com

 

The first summit organized by British Columbia’s Personalized Medicine Initiative took place last week, and I was blown away by the level of energy and excitement in the room. There were a number of really interesting talks, including Leroy Hood on the 100K Wellness Project, Julio Montaner‘s argument for treatment as prevention for HIV, Joel Dudley on incorporating genomic sequencing into clinical practice, Jim Kean on founding and growing Wellness FX, and Jill Kagenkord on 23andMe‘s vision for leveraging its rapidly growing collection of customer genetic and phenotypic data to advance medical research.

Three themes came through loud and clear at the conference, and point to how personalized medicine can lead to patient empowerment and to better health outcomes.

1. A Focus on Wellness, not Sickness

A majority of speakers highlighted the need to focus on maintaining and optimizing people’s wellness, rather than curing their sickness when wellness breaks down. Wellness is the medical equivalent of positive psychology, and is the key to generating the economic benefits that will power the adoption of personalized medicine, since preventing disease and maintaining health are likely to lead to substantial cost savings to both health insurers and to society at large.

2. Data is Power, so Patients Must Track

To turn health maintenance and wellness into a scientific enterprise, though, large amounts of data at the level of the individual are necessary. While the rapidly decreasing cost of whole-genome sequencing means that most of us will probably have our genetic data available within a few years, the key to connecting genomic information to health outcomes at an individual level is to dynamically track a wide variety of phenotypic and environmental data. This will allow researchers to identify actionable biomarkers that indicate increased risk of disease (or movement away from a healthy state), and allow patients to monitor those biomarkers and move them towards a healthier level.

This is why direct-to-consumer digital health technologies are likely to be a game-changer for personalized medicine. Fitness trackers, smart-watches, biosensors embedded in clothing, “tricorders”, and, ultimately, implantable biosensors, will generate a deluge of data that will facilitate data mining aimed at identifying key wellness biomarkers, and will help patients to monitor and modulate their own profiles.

3. Direct Financing by End Users can Accelerate Development

A third way in which the personalized medicine ethos can empower users is by allowing them, in turn, to empower the development of personalized medicine. New “democratized” financing tools like crowdfunding provide end users with an avenue to support the development of specific technologies that allow users to capture their own health data. Crucially, users can support this type of initiative both by providing capital to fund R&D and commercialization, and by providing early users who will help to refine the product or service. Joel Dudley mentioned two such technologies in his talk at the summit:

  • The Scanadu Scout – a “medical tricorder” competing for the Qualcomm Tricorder XPrize. Scanadu crowdfunded $1.66 million in 2013 on Indiegogo to fund development of the Scout, and subsequently raised $49 million in four investment rounds. A key part of the appeal of their crowdfunding campaign was that they hoped to enlist backers, who have begun to receive Scout prototypes, to participate in the clinical trial needed to obtain FDA approval of the Scout as a medical device.

 

  •  The uBiome project launched in 2013 with a crowdfunding campaign that raised $357,000 on Indiegogo. Campaign backers, and the company’s current customers, send a swab to the company and complete a range of surveys. They receive access to their microbiome sequencing data as well as to tools that allow users to compare their own microbiome to other users, track changes in their microbiome over time (via repeated sampling), and identify potential lifestyle interventions to achieve a more favorable microbiome profile. The company’s mission is to “equip all individuals with the tools they need, in order to empower them to learn about the unique balance of bacteria in their bodies”.

 

Direct-to-consumer companies like WellnessFX and 23andMe have not used crowdfunding, but they benefit from the same technological trends that allow them to offer biological tracking services at price points that are low enough to attract a large market of early adopters. In short, this convergence of trends is empowering patients – and we are all patients – to build the installed user base (the “big data”) necessary to make personalized medicine a reality.

It’s not only big government, big science, or big tech that will bring personalized medicine to you – it’s users proactively seeking the best ideas out there and supporting them directly. As Sean George of Invitae put it at the Personalized Medicine Summit – the 1970s are back in Silicon Valley, but this time the transformation will be in human biology and health, not semiconductors.

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