1. Regenerative Medicine

Category Archives: Regenerative Medicine

Is the cell therapy sector outperforming the major indices?

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So here's what I did today.  I built a portfolio of public companies focused exclusively or predominately in the cell therapy space.  I excluded any companies that are in the sector but their products/services constitute less than a significant majority of their revenue and/or expenses.  The portfolio sits at 29 companies.  Here's the list:


Here's how the portfolio performs against the Dow Jones, Standard and Poor's, and NASDAQ indices so far this year.


When looking at the period 1 January 2012 to 10 August 2012, the cell therapy portfolio is up 42%, Dow Jones up 8%, Standard and Poor's up 12% and NASDAQ up 16%.

In the context of how much we hear about how harsh this sector is or has been on investors, I found today's analysis interesting and, honestly, pleasantly surprising.

This snapshot is useful but has its limitations. I'm relying on Google Finance for accuracy of the information provided.  Do your own due diligence. Invest accordingly.  I hope this helps.

--Lee

This snapshot has been brought to you by Cell Therapy Group: all cell therapy, all the time! 🙂

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http://feeds.feedburner.com/CellTherapyBlog

Two lessons I learned this week.

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I learned two valuable things this week I thought I'd pass on in a Friday afternoon post.  Actually strictly speaking these are likely things I've learned before but needed to re-learn or to be 'reminded' of their importance.
Please pardon a little stroll away from the typically strict focus on cell therapy -- but in a way that's the theme of today's post.
1.  Take time each week to read something from outside your specific profession or job focus.  
I'm not talking here about the importance of escaping in the evening with a fiction novel (also very important) but rather reading something professional but from well outside your area of focus.  Here are my examples.

I always read WIRED magazine.  Aside from GEN it's the only magazine I read.  Just reading something outside of cell therapy or biotech often infuses me with an idea that otherwise would have never occurred to me like the need for a cell therapy X Prize or cellular aggregates as microcarriers or tissue-engineered memory and processing devices or even just the conviction to better represent cell therapy to the broader world out there of scientists, engineers, journalists, policy-makers, or perhaps people with too much money looking to be inspired and wanting to make a difference.

Similarly, on a flight this week I reached into the seat pocket in front of me and discovered a recent copy of the Journal of the American Medical Association.  I read a fascinating article that has me excited about an idea for how we as a cell therapy industry might lead the way in addressing clinical trial and data transparency that would put our sector in a leadership position, lend the industry a much-needed spotlight, and has the potential to facilitate the kind of meta-analysis and data-mining that could only be done through data aggregation.  I believe the concept has the potential to be disproportionately significant for a sector defined by so many small, under-powered trials.
The idea may never see the light of day but the point is the source of the inspiration.  In order to 'think' outside the box one typically has to 'be' outside the box.  Lesson?  Spend some time outside your box.
2. It often takes something very small to make a disproportionately significant impact on someone.  
I was reminded recently through an exchange of simple kindnesses just how little it sometimes takes to make a big difference in someone's life.  For you what might be so easy to give might be of unparalleled value to someone for whom that is so unattainable.  
Lesson?  When the opportunity knocks for you to give something small or simple, take it.  This kind of charity almost always has the potential to be mre impactful than you might ever imagine.

Source:
http://feeds.feedburner.com/CellTherapyBlog

Cell-based Cancer Immunotherapies. Some metrics..

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Whatever one makes of Dendreon's challenges in bringing Provenge to market and then its ups and downs in the market, the whole affair has brought a much bigger spotlight to cancer immunotherapies and cell-based immunotherapies in particular.

This is true on all fronts.  Cancer immunotherapy conferences are popping up everywhere.  A growing number of of analysts are now covering a growing number of companies in the space with coverage ranging from bearishly critical to ebullient bullishness. Some venture capital firms are now loosening their purse strings for immunotherapy plays and both pharma venture funds and business development departments are now spending an increasing amount of time actively monitoring and exploring potential plays in the sector.



One of the best annual industry summaries of what is happening in the sector is sponsored by MD Becker Partners through its annual Cancer Immunotherapy: A Long Awaited Reality conference held each in New York, this year on October 4 and select video replays it hosts on its YouTube channel.

Some Segment Metrics
As part of our ongoing industry intelligence and consulting services we actively track the activity and progress of industry-sponsored clinical trials of all cell therapies in addition to the products already on the market.  Here's how our data stacks up regarding the cell-based immunotherapies segment of the sector:
Commercial:
  • Dendreon's Provenge
    • Autologous immunotherapy for prostate cancer (1 monthly dose for 3 months)
    • Efficacy: prolongs survival
    • Markets: only the United States (approved April 2010)
    • Next markets: submitted the marketing authorisation application to the EMA (European Medicines Agency) in early 2012 and hopes to introduce Provenge in the European market in 2013
    • 2011 Revenue $290,000
    • Projected 2012 Revenue: ~$380,000

Phase III or II/III:

Phase II or I/II

  • 50 industry-sponsored clinical trials of cell-based immunotherapies actively recruiting, active no longer recruiting, active not yet recruiting, or anticipated to commence yet in 2012
    • ~10 of these are expected to have readouts yet this year
    • Trial sites in US, Canada, UK, continental Europe, Israel, South Korea, India, Australia
    • Expected enrollment of 3,500+
Investment:

The following are notable cash infusions into the sector for 2012 to-date:
  • Bellicum Pharmaceuticals.  $20M series B. 
  • CellMedica. $15M grant from CPRIT in Texas.
  • Argos Therapeutcs. $25M Series D.
  • Northwest Bio.  $5.5M grant from German gov't Saxony Development Bank

Hope this is useful.

--

This post has been brought to you by your friends at CTG.  All cell therapy. All the time. 🙂  

-- Lee @celltherapy

p.s.  As always we welcome your feedback, comments, and corrections.  












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http://feeds.feedburner.com/CellTherapyBlog

Clinical trial costs

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Based on a survey* of 21 drug makers, 12 biotechs, nine device makers and 23 contract research organizations, PharmaLot has recently published the following metrics for current clinical trial costs (see full article here)
Average per-patient trial costs across all therapeutic areas:
Phase I: $21,883
Phase II: $36,070
Phase IIIa: $47,523
Phase IIIb: $47,095
Phase IV: $17,042
Average cost per patient for a cardiovascular trial:
Phase II: $33,700
Phase IIIa: $21,750
Phase IIIb: $6,830
In oncology, the average per patient trial cost:
Phase II: $73,303
Phase IIIa: $57,207
Phase IIIb: $65,900
For central nervous system disorders:
Phase II: $28,197
Phase IIIa: $33,768
Phase IIIb: $41,824
For diabetes:
Phase II: $ 8,854
Phase IIIa: $12,667
Phase IIIb: $10,700
Anyone have any thoughts or data as to how this compares to cell therapy trials?
--Lee
*survey conducted by Cutting Edge Information.

Careers in cell therapy & regenerative medicine

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As you will see just below, this post falls under the category of a thinly veiled and somewhat shameless plug which I hope you'll tolerate if I'm transparent about it upfront and I add what may be a little useful commentary along the way.

I won't have to convince "regular" readers of this "irregular" blog that regenerative medicine is one of the fastest growing life science sectors.

Given the pace of the industry's growth, the amount of public money being "invested" in the sector, and the general enthusiasm that the science generates, it is no surprise that is increasingly an area of interest for those seeking a new career path.

One commonly held misconception is that career opportunities in the sector are limited largely to those in early-stage research.

While it is true that the industry is still in the nascent stages of commercial development compared to other more mature segments in the broader biopharmaceutical industry, careers in regenerative medicine span the full research and commercial spectrum from pre-clinical to clinical research in both academic and corporate settings, manufacturing, regulatory, quality, operations, logistics, finance, business development, marketing, sales, communications, and executive management.

As has been discussed here in the past, most analysts agree that in 2008 the industry passed the $1 billion mark in annual revenue generated from sale of approved therapeutic products falling under the regenerative medicine category (see here for more in-depth analysis).

Furthermore, most large multinational life science companies are now investing heavily into the sector on the promise that it will revolutionize healthcare in the coming decades.

I believe the 2008 decision by Pfizer to create a substantial new Regenerative Medicine division will be seen as a seminal turning point in corporate perspectives on the industry. Since that point most life sciences have begun investing in regenerative medicine strategies and the trend has even leaked outside of life science to companies such as Google Ventures which has informally identified regenerative medicine as one of their primary areas of interest when looking at companies in which to invest.

Most major universities have now created both academic and research programs dedicated to the emerging field of regenerative medicine such that there is a new class of graduates now emerging in both scientific and commercial disciplines with regenerative medicine as a primary focus.

However, one of the difficulties with finding a career in regenerative medicine is that it is comprised of what has heretofore been fairly discrete disciplines of stem cell biology, pharmaceutical sciences, biotechnology, tissue engineering, stem cell transplantation, device technologies, cell therapies, etc.

Furthermore, most recruiters and online job sites have very little experience or focus on the industry making recruiting difficult for positions in regenerative medicine companies or departments. The task is further complicated by the fact that the candidate pool is small and diversely spread over a multitude of disciplines, centers, and a multitude of small companies.

Finally there are only now emerging industry publications, organizations, online communities, and websites that truly represent the broad spectrum of regenerative medicine and that can be used as central resources for recruiting.

All this is why my partners and I saw the need for an online recruiting tool that really focused on regenerative medicine in a way that would support the industry's maturation. What we wanted to bring was a very simple solution that would create as much value as possible for those looking to recruit the right talent into their regenerative medicine efforts. This was the genesis behind RegenerativeMedicineJobs.com.

(here comes the shameless part....)

About RMJ

At RegenerativeMedicineJobs.com, we believe the growth trajectory of this industry requires a specialized, online recruiting system – one that is more than just a website for posting jobs but also plugged into the large and diverse network of associations, publications, and social media that serves this industry.

RegenerativeMedicineJobs.com is a specialized online job board focused on recruiting for positions in the rapidly growing field of regenerative medicine. The site is simple to use and focused in scope. But this is more than just another website.

RMJ has developed a sophisticated system designed to leverage our extensive network in RM to ensure every post gets maximum attention and attracts the best candidates.

Traffic is pulled to the site and job postings are pushed to a targeted audience by focused social media and marketing campaigns. This is the latest way to recruit for any position in a regenerative medicine department, division, or company. This is regenerative medicine recruiting with a focus.

RegenerativeMedicineJobs.com (RMJ) is a collaboration between CTG Consulting, Co. and Pencilneck Software, Co. The personnel behind this collaboration created Cell Therapy News, Cell Therapy Blog, the LinkedIn Cell Therapy Industry Group, and have produced over a dozen organizational or event-based websites in the cell therapy, stem cell, and regenerative medicine space.

The site has been designed to be simple and intuitive with only one goal in mind: to be the best online tool for recruiting personnel into positions in the regenerative medicine industry.

We don't offer resume posting, career advice, assistance building your CV, hand holding, shoulders to cry on, or job postings outside of regenmed. We believe in the value of specialization.

The site is a listing of regenerative medicine jobs (period). All types of jobs for all types of organizations but only regenmed job (period).

We have only one rule: the jobs posted m
ust be for a position within the regenerative medicine space. We define regenerative medicine as anything involving cell therapy, stem cells as therapies or tools for discovery or toxicity testing, or non-cell based programs/products (e.g., small molecules, biologics, devices, etc) used to replace or regenerate cells, tissues, or organs to restore, repair, or establish normal function.

The site has a topical niche with no regional focus or bias. This is meant to be a global tool equally useful for a company or university in Poland, Taiwan, Brazil, the United States, etc.

The site is also meant to be useful for recruiting any positions scientific or commercial, business or technical, executive or laborer.

*

We hope you find it useful.

We hope it contributes to the industry's growth and maturation.

We hope you will provide us feedback on what we're doing well and what we can do better.



In vivo cell trafficking just took a leap forward

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Today Celsense, Inc. and the University of Pittsburgh Cancer Center announced that the FDA has authorized the use of the Cell Sense imaging reagent for use in a phase I clinical trial of a dendritic cell caccine to treat colorectal cancer patients.

This is the first FDA authorization of the use of Cell Sense in patients. Cell Sense is a novel perfluorocarbon tracer agent used to safely and efficiently label cells ex vivo without the use of transfection agents. Labeled cells are then transplanted into the patient enabling researchers and clinicians to non-invasively track the administration and migration of therapeutic cells using MRI. Applications include tracking cells in immunotherapy or regenerative medicine as well as the diagnosis of inflammatory sites by tracking selected populations of immune cells.

Cell Sense has been studied extensively in preclinical testing with many different human cell types including human cells in animals. For instance, in 2009 a paper was published in Informa's Cytotherapy, in which Celsense’s novel perfluorocarbon tracer agent (product “Cell Sense”) was used to label human DCs ex vivo for the purpose of tracking the cells in vivo post-transplant by 19F MRI. The paper provided an assessment of the technology and demonstrated that human DCs were effectively labeled without significant impact on cell viability, phenotype or function. Furthermore, the labeled dendritic cells were clearly detected in vivo by 19F MRI in a model system, with the labeled cells being shown to migrate selectively towards draining lymph node regions within 18 hours after transplant.

Many investigators looking at various ways to label cells to enable in vivo imaging have expressed concern that the FDA would delay the regulatory progress of their therapeutic candidates if an imaging modality was introduced.

This concern is based on numerous reports of MRI contrast reagents, such as the commonly investigated USPIO (ultrasmall superparamagnet iron oxide), deleteriously affecting the cells (see recent paper in Cell Transplantation).

"We believe that the authorization of this IND will alleviate such concerns and lower the barriers for adoption. The agency’s tangible support for bringing new technologies to bear in the translation of cell-based therapeutics is very encouraging,” s Charlie O'Hanlon, President and CEO of Celsense.

While there have been approved uses of imaging reagents (e.g., Feridex, etc) with cell therapies in other countries (e.g. Isreal), I believe this may be the first FDA-sanctioned use of a particle-based imaging label with a cell-based therapy. Other approaches to cellular imaging include nuclear imaging reagents and genetically modifying cells with reporter genes such as those provided by CellSight Technologies.

Imaging labels are capable of providing investigators with data demonstrating where the cells go, at what volumes, and for how long they stay at the target location.

The industry has been keen to see these kinds of technologies clinically employed but different cell-based labels have created their own technical, clinical, and/or regulatory hurdles. I'm hopeful that Celsense and others like them are now ushering us into a new era where we will eventually be able to use various technologies to monitor and collect valuable data concerning cells after they have been administered as a therapy to a patients.

Additional resources on the topic of imaging for cell therapies:

CIRM recently hosted a webinar - "CIRM/RMC Webinar: Imaging Technology for Cellular Therapies. One of the speakers, Dr. Shahriar Yaghoubi from CellSight Technologies, provides an overview of cell therapy imaging with emphasis on PET. Click hear for the archived playback.

A very interesting article posted today on Harvard's StemBook website. "In-vivo Stem Cell Imaging - Regulatory Challenges and Advances". Nice overview intel from J. Bulte and a snapshot into E. Wirth's (of Geron) perspective re: stem cell imaging.

A new book from CRC Press edited by Dara Kraitchman and Joe Wu will be out soon. It gathers together different methods for comparison. The issue will remain the sensitivity of the methods to track few cells. "Stem Cell Labeling for Delivery and Tracking Using Non-Invasive Imaging".

MRI contrast agents can change stem cell proliferation

There s also a very informative discussion thread on the topic in the Cell Therapy Industry group on LinkedIn.

Stem cells for a Webby!

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I know I haven't been a very good blogger for quite some time but I wanted to pass on a letter I just received from my friend James Price as a Charter member of the Canadian Stem Cell Foundation. They're going for a Webby award. You don't have to be Canadian to support their cause - you just have to:

  • believe in the power of interactive, online network-based activism,
  • support the potential of stem cells to change people's lives, and
  • wanna have a little fun raising awareness for our otherwise-sometimes-stodgy-science!

Go vote - it's good for you and the world! (WARNING: you might find out some things about some pretty cool stuff nominated in the other categories while you're there).

CSCFHeader

Dear Lee,

I'd like to thank everyone who has voted and helped spread the word about the Webby Awards nomination. We think a big part of the Foundation's success, and especially this nomination, is due to your enthusiasm, creativity and support.

That support has taken us a long way. Right now, we're in the lead for Best Activism Website. But our lead is narrow, only 3%, so we need you to pull out all the stops. There are only two days left to vote, so let's make sure it's a win for stem cells!

Why Activism?

A large part of what makes our website and social media pages work is the Charter community. All the material we create - the Stem Cell Charter, "Rock Star Scientists" video and all the posts on our social media pages - are designed to give the stem cell movement a voice - your voice. You are the most important part of getting the word out about stem cell science and helping people see the amazing potential of the field.

Voting and encouraging others to vote is a perfect way to do this. Tweet, post, blog, email and shout your support from the rooftops. Let's show the world how important we think stem cell science is.

How to vote

Step 1: Click HERE and fill in your email address and a password.

Step 2: You will receive an email from the Webby Awards - click the link to activate your account.

Step 3: Go HERE TO VOTE and choose RENEW THE WORLD.

Voting closes at midnight on April 29th. We have two days left!

webby banner

Thanks for your support,


James Price
President & CEO



Latest Cell Therapy Approval by FDA. Dendreon's Provenge.

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It has been a long-time coming. It has been hyped and scoffed, bet against and hoped for, but now none of that matters. It's here. Dendreon has brought Provenge to market. Here, in the word's of the FDA...

FDA NEWS RELEASE

For Immediate Release: April 29, 2010

FDA Approves a Cellular Immunotherapy for Men with Advanced Prostate Cancer

The U.S. Food and Drug Administration today approved Provenge (sipuleucel-T), a new therapy for certain men with advanced prostate cancer that uses their own immune system to fight the disease.

Provenge is indicated for the treatment of asymptomatic or minimally symptomatic prostate cancer that has spread to other parts of the body and is resistant to standard hormone treatment.

Prostate cancer is the second most common type of cancer among men in the United States, behind skin cancer, and usually occurs in older men. In 2009, an estimated 192,000 new cases of prostate cancer were diagnosed and about 27,000 men died from the disease, according to the National Cancer Institute.

“The availability of Provenge provides a new treatment option for men with advanced prostate cancer, who currently have limited effective therapies available,” said Karen Midthun, M.D., acting director of the FDA’s Center for Biologics Evaluation and Research.

Provenge is an autologous cellular immunotherapy, designed to stimulate a patient’s own immune system to respond against the cancer. Each dose of Provenge is manufactured by obtaining a patient’s immune cells from the blood, using a machine in a process known as leukapheresis. To enhance their response against the cancer, the immune cells are then exposed to a protein that is found in most prostate cancers, linked to an immune stimulating substance. After this process, the patient’s own cells are returned to the patient to treat the prostate cancer. Provenge is administered intravenously in a three-dose schedule given at about two-week intervals.

The effectiveness of Provenge was studied in 512 patients with metastatic hormone treatment refractory prostate cancer in a randomized, double-blind, placebo-controlled, multicenter trial, which showed an increase in overall survival of 4.1 months. The median survival for patients receiving Provenge treatments was 25.8 months, as compared to 21.7 months for those who did not receive the treatment.

Almost all of the patients who received Provenge had some type of adverse reaction. Common adverse reactions reported included chills, fatigue, fever, back pain, nausea, joint ache and headache. The majority of adverse reactions were mild or moderate in severity. Serious adverse reactions, reported in approximately one quarter of the patients receiving Provenge, included some acute infusion reactions and stroke. Cerebrovascular events, including hemorrhagic and ischemic strokes, were observed in 3.5 percent of patients in the Provenge group compared with 2.6 percent of patients in the control group.

Provenge is manufactured by Seattle-based Dendreon Corp.

The ROI on pant-wearing and other social media tips

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With many things in life, there is a payoff for doing them.  Do the dishes and the kitchen is cleaner, your household is more functional, and hopefully one or more family members notice and appreciate you for it.



For other things, however,  the people around you have such high expectations you’ll do them that you only lose points if you don’t but gain very little if you do.  For you, this may be true of the dishes.  Certainly I’ve always maintained this is true for Valentine’s Day.  Get flowers and you simply maintain the relationship’s status quo; fail to do anything and you lose big points fast.  


Similarly, at some point certain things become so ubiquitous that they are expected as a baseline.  This is true of putting on your pants.

The global head of social media for Ford Motor Company, Scott Monty, once asked, “What’s the ROI* of putting your pants on in the morning?“.   The truth is that there is very little benefit to putting on your pants other than to avoid the significant cost of not doing so.

Certainly this is true now of having a website or an email address for your company.  Unlike a couple decades ago, no company gets kudos for having a website or email addresses but it would certainly raise eyebrows of criticism if your company failed to have them.

Arguably social media participation is not quite there yet but it is, I submit, fast approaching.  Someday in the not-too-distant future you will receive the cringe of shame if your company is not active in the leading social media platforms of the day.  Today – for companies – these are LinkedIn, Twitter, and Facebook.  This will be true irrespective of whether yours is a B2B or B2C company.
Recently I was invited to write an article for Future Medicine’s special issue for the World Stem Cell Report.  I was asked to make the case for why and how participating in social media stood to benefit the scientists, companies, executives, employees, academics, activists, and other stakeholders in the cell therapy industry.  

The result is “Why the stem cell sector must engage with social media“.  What I attempted to succinctly outline are the ways social media primarily benefit one’s career and organization or company.


“I can tell you without the slightest hesitation of conviction – having experienced it myself and seen it repeated countless times – is that active and successful social media engagement translates into:
  • Unparalleled learning: accessing more information relevant to your discipline, specialty and company than you otherwise will. 
  • Enhanced profile: higher profile within your industry, profession, specialty and community. Social media is not the only way to build a profile but it can be very effective.
  • Wider network: more touch points and meaningful relationships with people than you otherwise will accomplish by any other means combined.”
The measurable impacts and benefits are real and certainly include:
  • Traffic: “For companies, increased traffic equals increased opportunity to call readers/viewers to your intended action – interaction, citation, linking, investing, buying or engaging in some other action you solicit. For individual professionals, increased viewers translate into more chances for collaboration, citation, engagement, etc.”
  • Collaboration: “There is an intrinsic correlation between one’s profile and the opportunities one has for collaboration. For companies this means finding the right partnerships, joint ventures, strategic alliances, collaborators, employees, management and so on. For individual professionals, this means more and/or better quality invites to speak, write or collaborate in other ways. It also means finding quality grad students, faculty, employees and interns
  • Revenue/Income: This is about translating a broader knowledge base and a wider network over which you have some level of influence (if only just that they are listening) into more money for your company, organization and yourself. For companies, this means finding the right partners, investors, customers and so on. For organizations this means finding the right donors, impressing the right grant reviewers and/or recruiting the right rain-maker faculty. For individual professionals this translates into promotions or job offers.”

As I conclude my article I will conclude here:

    “In order to create the kinds of perceptions and solicit the kinds of actions we want from the world around us, we must engage the world around us. The world around us is engaging online. 

    For all kinds of selfish and selfless reasons you, your company or organization and your career will benefit from you engaging there too.”

    and this prediction:

    “…in less than the blink it took for the commercial world to accept websites and email, it will seem similarly ridiculous for professionals, academics and companies to operate and succeed without actively using social media.”

    ____________


    If this topic is of interest to you, here are some great resources particularly focused on the value of social media to those in life sciences.


    Canaday, M. Is Life Science Social Media Worth It Yet? Three Tenets Behind Its Relevance To Your Business. Comprendia. 6 December 2012. 


    Bersenev A. Scientific blogging as a model for professional networking online. Cellular Therapy and Transplantation. 2010;2(7). 10.3205/ctt-2010-en-000084.01. 


    Bersenev, A. Scientific blogging as a model for professional networking online. 4 August 2010. StemCellAssays.com 


    Bersenev, A. Who’s Who in the Stem Cell Blogosophere.  27 June 2011. StemCellAssays.com 


    Bishop, D.  How to bury your academic writing. Bishop’s Blog. 26 August 2012. 



    Buckler, L. If You’re Breathing, You’re in PR. Cell Therapy Blog. 11 June 2010.  

    Buckler, L. Don’t feel the pain of ignoring social media? Just wait a minute…. CellTherapyBlog.com 22 October 2008.    

    Jewell, T. Survey: How our scientists use social media. AZHealthConnections.com. 12 February 2012. 


    Knoepfer, P. Top ten tips for blogging for scientists. 2 August 2012. IPScell.com   


    Shipman, M. Why Scientists Should Publicize Their Findings – for Purely Selfish Reasons. Scientific America. Blog. 18 June 2012. 
         
    Shipman, M. A gentle introduction to Twitter for the apprehensive academic. Scientific America. Blog.  14 June 2011.  


    Small, G. Time to Tweet. Nature 2011. 479 141 2 November 2011 


    Wilcox, C. Social Media for Scientists Part 1: It’s Our Job. Scientific American Blog. 27 September 2011.  


    Wilcox, C. Social Media for Scientists Part 2: You Do Have time. Scientific American Blog. 29 September 2011.  


    Wilcox, C. Social Media for Scientists Part 3: Win-Win. Scientific American Blog. 10 October 2011.  

    Wilcox, C. Guest Editorial: It’s time to e-Volve. Taking Responsibility for Science Communication in a Digital Age. Biol Bull. 22285-87. (April 2012)  

    The Rules of Social Media.  Fast Company.  8 August 2012. 


    Source:
    http://feedproxy.google.com/~r/CellTherapyBlog/~3/k7ANTnLXNjc/the-roi-on-pant-wearing-and-other.html

    Source:
    http://www.longevitymedicine.tv/the-roi-on-pant-wearing-and-other-social-media-tips/

    Commercialization of Regenerative Medicine: Learning from Spin-Outs

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    The meeting “Commercialization of Your Regenerative Medicine Research: Lessons from Spin Out Successes” was hosted by the Oxbridge Biotech Roundtable (OBR) (Oxford, UK) at the University of Oxford in February, 2013, and attracted a multi-stakeholder audience spanning academia and industry. 


    The event featured case studies from Gregg Sando, CEO, Cell Medica (London, UK), John Sinden, CSO, Reneuron (Guilford, UK), and Paul Kemp, CEO and CSO, Intercytex (Manchester, UK). 


    OBR is a student-led initiative with over 7000 members across eight different UK and US locations with a mission to foster a conversation about the healthcare and life sciences industry. 


    Anna French and David A. Brindley, along with some of my assistance, captured and have now published the main themes of the meeting and the major questions facing the regenerative medicine industry and its rapidly emerging subsets of cellular and gene therapies. 


    Notably, we discuss the compatibility of regenerative therapies to the existing healthcare infrastructure, biomanufacturing challenges (including scalability and comparability), and the amenability of regenerative therapies to existing reimbursement and investment models. Furthermore, we reiterate key words of advice from seasoned industry leaders intended to accelerate the translation path from lab bench to the marketplace.


    To read the review see: Commercialization of Regenerative Medicine: Learning from Spin-Outs


    Anna French, R. Lee Buckler, and David A. Brindley. Rejuvenation Research. April 2013, 16(2): 164-170. doi:10.1089/rej.2013.1423.

    Source:
    http://feedproxy.google.com/~r/CellTherapyBlog/~3/4Uv2o54_hWQ/commercialization-of-regenerative.html

    Source:
    http://www.longevitymedicine.tv/commercialization-of-regenerative-medicine-learning-from-spin-outs/

    FDA 1. RSI 0. Regenerative Sciences (Regenexx) vs FDA (2012)

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    As followers of this blog will know I've been blogging about Regenerative Sciences and predicting their eventual run-in with the FDA since my first post in September 2008 (Cell Therapy is Not the Practice of Medicine) and again in February 2009 (Regenexx vs the FDA 2009).  When the FDA finally proceeded with an injunction against RSI in August 2010,I helped spread the news (here).

    I've watched the development of the fight between RSI and the FDA with interest.  In September 2001 I posted a rather lengthy commentary about the potential impact of the case (Potential far-reaching implications of the ongoing fight over point-of-care autologous cell therapy.

    Since then I have welcomed other bloggers and commentators who are now following and commenting on the case much more closely and frequently than I including @LeighGTurner (on Twitter) and Paul Knoepfler (@PKnoepfler on Twitter and his Knoeplfer Lab Stem Cell Blog).  Recently I enjoyed being interviewed by Paul on the issue of unregulated stem cell activity and touched on the case for his blog.

    Consequently I read with interest yesterday's federal court ruling upholding the FDA's injunction against RSI and the immediate commentary from the New Scientist, Stanford's Scope Blog and Knopfler's multiple posts (here and here). As a long-term follower of this case, I've been asked to comment.  Here is my brief reaction:

    This is a case that was always destined for the appellate courts regardless of which way the initial court ruled.    The fact the federal court ruled in the FDA's favor certainly now sets the onus on RSI and what is anticipated to be a gamut of intervenors but taking this case to the appellate courts is what the legal team have anticipated and legal arguments designed for all along.

    This is just the beginning of what will be a long and interesting battle.  The ruling was nothing more than the granting of an injunction in response to the government's motion for summary judgement.  In granting the injunction the court  agreed with the government's position that it was acting under the authority given it under the Federal Food, Drug, and Cosmetic Act, 21 U.S.C. § 321(g) but it provided little-to-no rationale for its ruling.

    The court chose, in its wisdom, not to address the bulk of the RSI's legal arguments which are largely jurisdictional in nature. These are the kinds of arguments which the lower courts prefer be dealt with by appellate courts and frankly the judge did us all a favor by ruling quickly, succinctly and punting the case where we all knew it was inevitably headed.

    In my opinion, other than chalking one up in the government's win column there is little to be gleaned from this ruling in terms of how RSI's arguments will be received in appellate court.  The interesting day is yet to come.

    In terms of a short-term practical impact, frankly I see very little.  RSI has already ceased distributing Regenexx within the US so there will be little-to-no impact there.  As for the potential impact on other companies or clinics who might be operating on the fringes of FDA regulation within the US, I suspect it will be business as usual.

    Most of the clinics/companies offering cell-based treatments/products which are arguably in contravention of FDA regulation are operating under the clear knowledge of what they are doing and where the FDA stands with respect to the treatments/products they offer and yet they persist and continue.


     For the truly fraudulent there is the risk of criminal charges and/or litigation but for those companies or practitioners who are operating in this shade of grey which are not shady (and they do exist), the  risks associated with this practice are barely higher than in the routine practice of medicine. 


    In reality, with the exception of the most fraudulent examples, it takes a fair long-time for the FDA to catch up with these folks and there is good money to be made in the interim.  When they get caught, they will stop. If they've recouped their initial investment (which is nominal and the margins are high) there is very little penalty to this course of action.  Perhaps they set up shot elsewhere or simply enjoy the proceeds.  I doubt we will see much of a slow-down of this kind of activity.  Indeed it may strengthen the resolve of those committed to the cause.

    In my opinion yesterday's ruling was in interesting and important milestone in a continuing evolution in the debate of how best to regulate the use of cells in treating people but I'm not sure it's the seminal pivot point that some believe.  I suspect we will not see any radical shift in terms of FDA or industry activity until (if then) the appellate courts rule.

    Just my two cents....

    --Lee

    Source:
    http://feeds.feedburner.com/CellTherapyBlog

    A proposed 6-step platform for the cell therapy industry to consider in combating non-compliant cell therapy treatments

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    Further to my recent post, “Six steps to fighting non-compliant cell therapy treatments. The stuff of grey shades, spades, ivory towers and (ahem) balls.“, I have crafted a 6-point platform that I propose to submit (with potential edits based on preliminary feedback) to several of the leading  industry and professional organizations for their consideration including ARM, ISCT, ISSCR, FACT, AABB  ICMS, and perhaps, in due course, to patient groups, physician groups, disease-specific professional organizations (e.g, cardiology, oncology, neurology, cosmetic, etc).



    I welcome comments and feedback. 


    1. In addition to helping patients distinguish between compliant and non-compliant treatments (and providers) we must do more to help patients distinguish between non-compliant cell therapy treatments (and providers) which are more or less risky. 


    2. Whatever we do in response to this issue should be done with an eye to being practical and helpful to patients in the real-life context of their decision about whether or not to buy a non-complaint cell therapy.


    3. Our response to this issue should be based on a risk-based approach recognizing that not all non-compliance is created equal.  We should create a framework for risk-based analysis (both for us and our audiences) and focus initiatives around those which present the highest risk.


    4. We recognize the problem of non-compliant cell therapies is not just a problem that exists in jurisdictions with little, no, or poor regulation but that is a growing problem even in the most highly regulated jurisdictions meaning the solution cannot be regulated it depends on education and enforcement.


    5. We recognize regulatory agencies cannot enforce non-compliance on their own.  We as an industry need to complement their efforts through our own standards and enforcement.


    6. Stakeholder groups should support the formation of a multi-organizational  initiative to, based on a risk-based assessment, spotlight the categories or signs of highest-risk offenders for use by patients and/or their physicians in identifying  whether or not treatments (and providers) they may be considering fall into the that category associated with the highest level of risk.


    What do you think?

    Source:
    http://feedproxy.google.com/~r/CellTherapyBlog/~3/wkSMxAV9938/a-proposed-6-step-platform-for-cell.html

    Source:
    http://www.longevitymedicine.tv/a-proposed-6-step-platform-for-the-cell-therapy-industry-to-consider-in-combating-non-compliant-cell-therapy-treatments/

    Another > $100M month for companies in the cell therapy space

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    Last month we reported here on this blog that March was more than a $100M month for companies in the stem cell and cell-based regenerative medicine space in terms of monies raised.  

    What we missed was a $15M grant from Cancer Prevention and Research Institute of Texas (CPRIT) for UK-based CellMedica.  This pumps last month's total to just under $140M.

    This month, according to our sources, betters even March's better numbers by coming in at just over $170M though that is largely on the back of one large deal in Asia.  Here's how the numbers break down.

    Allocure kicked off the month with a decent $25M Series B round from new syndicate member Lundbeckfond Ventures, as well as previous investors SV Life Sciences and Novo A/S.  Allocure is headed into phase 2 for acute kidney injury with an allogeneic mesenchymal stem cell therapeutic they currently call AC607.  


    Little-known Canadian-based, Sernova then announced a $3.6M PIPE to fund continued development of its proprietary Cell Pouch System(TM), and, in particular, to fund the upcoming first-in-man clinical trial for patients with diabetes receiving an islet transplant.  The application to proceed with this trial is currently under review by Health Canada.


    Next up was NeoStem closing a $6.8M public offering for "expanding" their contract manufacturing business, Progenitor Cell Therapy, and "enrolling the PreSERVE AMR-001 Phase 2 clinical trial for preserving heart function after a heart attack".  


    The biggest deal of the month was a $65M convertible debt financing of China Cord Blood by none other than global powerhouse Kohlberg Kravis Roberts (KKR) through it KKR China Growth Fund L.P., a China-focused investment fund managed by KKR.  We believe this is deal is certainly an investment in the future of China's healthcare market potential but that it is bigger than that.  We believe a significant driver for this deal may likely have been the opportunity to consolidate this sector globally - to use a significant operation and 'war chest' to fund mergers and acquisitions on both the public and private cord blood banking sector worldwide.


    The only classic first-round venture raise this month was a milestone-based $5M Series A by Bay City Capital into Phil Coelho's new company, SynGen, to fund his latest iteration of stem cell processing devices.


    Forbion Capital then announced that it was leading a series D round, joined by fellow existing investors TVM Capital, Lumira Capital, Intersouth Partners, Caisse de depot et placement du Quebec, Morningside Group, and Aurora Funds, of $25M into Argos Therapeutics in order to kick them into their phase 3.  The hope here is that with some early phase 3 data they may be able to attract the elusive partner they couldn't land with a mere bucket of phase 2 data.


    Innovacell landed the only European deal by announcing an 8.3M Euro (~$11M) investment by Buschier, Fides, HYBAG, and Uni Venture.  This will be used for the continued clinical development of its cell-therapy (ICES13) for the treatment of stress-urinary incontinence currently in a ph 3 study in several European countries.


    ReNeuron announced a private placement also open to existing shareholders that brought in just under $10M (£6.1M) to support their phase 1 trial in stroke and other pre-clinical, clinical, and regulatory milestones. 


    Finally, the Bio-Matrix Scientific Group, in an apparent ongoing quest to continuously reinvent itself, announced at month's end that they had formed a new subsidiary named Regen BioPharma and that they had raised $20M in a financing commitment from Southridge Partners II to purchase its common stock as required over the term of the agreement at a price set by an agreed formula.  This money is said to be dedicated to the acquisition of discovery-stage intellectual property and driving it through to phase 2 trials in an exercise of maximum value creation over a period they claim to be as short as 18-24 months.


    ..


    So in the end, the month saw companies in the space raise just over $170M and even if you back out the stem cell banking deal its still over $100M for cell therapy companies.  


    Over the 2 months, then, we've seen just over $311M raised through a variety of means by companies at every stage of maturity and for intended purposes ranging from acquisition, consolidation, early stage clinical development, and phase 3 testing.


    --Lee


    p.s. If you are aware of other deals in the sector this month, let us know and we'll update this accordingly.


    Source:
    http://feeds.feedburner.com/CellTherapyBlog

    Bioreactor Design and Bioprocess Controls for Industrialized Cell Processing

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    A short and sweet note to point you to a great article on bioreactor technologies related to cell therapy bioprocessing by CTG consultant and Director of Stem Cell-based Drug Discovery, John E. Hambor, who you can now follow on Twitter @StemCellonDrugs.


    "Bioreactor Design and Bioprocess Controls for Industrialized Cell Processing" was published in the June issue of BioProcess International.  


    The BPI team has made a real and meaningful commitment to representing cell therapy bioprocessing and we applaud them for their contribution to this emerging discipline.




    If this is a topic of interest to you, I recommend you also check out a conference being held this Fall by BPI's sister company, IBC LifeSciences, entitled "Cell Therapy BioProcessing" to be held September 11-12 in Arlington, Virginia.





    Source:
    http://feeds.feedburner.com/CellTherapyBlog

    Inactive and recently failed or terminated phase III or II/III cell therapy trials

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    In the two previous posts I have outlined what I believe to be the active phase III and II/III cell therapy trials, as well as the cell therapy products to have 'recently' obtained formal regulatory market approval in some jurisdiction.


    In the course of doing that work, I came across the following industry-sponsored phase III cell therapy trials which appear to be inactive and those which failed or were terminated.


    INACTIVE INDUSTRY PHASE III    

    •     Aastrom              BRCs
    •     Aldagen              ALD-101
    •     Arblast               AMT-301
    •     Avax                  Mvax
    •     HepaLife Tech      HepaMate
    •     KeraCure             KeraPac
    •     t2cure                BMCs
    •     TVAX                 TV-Brain
    •     TVAX                 TV-Kidney-1

       
    RECENTLY FAILED /TERMINATED INDUSTRY PHASE III   

    •     ABH (now Shire)        Dermgraft
    •     Cellerix                    Cx401

    I don't imagine this is an exhaustive list but as I have encouraged in previous posts, I welcome feedback as to errors, corrections, or omissions.   I'm using the 2009-11 time-frame here.  I'll update the post accordingly.

    Source:
    http://feeds.feedburner.com/CellTherapyBlog


    1. Regenerative Medicine