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Stem Cell Therapies: What’s Approved, What Isn’t, and Why Not?

With acceptance of stem cell therapies growing, so have controversies surrounding regulations.

Desperate to heal sports injuries, top professional athletes have been known to pay tens of thousands of dollars for experimental stem cell treatments that many used to find controversial. But now, stem cell therapies have become more mainstream and are no longer limited to professional athletes. Stem cell clinics offer both medical and non-medical treatments with claims of improving aesthetics and quality of life.

One recent study found over 400 websites – with the largest portion in the United States – advertising stem cell-based therapies (1); another found over 570 U.S. clinics offering stem cell interventions (2), giving more evidence that the market for stem cell therapies in the U.S. is growing at an accelerated rate. Yet these therapies are too often based on unfounded claims and lack proper clinical trials or authorized regulation. Despite what some clinics claim, very few stem cell treatments are currently available that are actually approved by the Food and Drug Administration (FDA). Hematopoietic stem cells harvested from bone marrow are routinely used in transplant procedures to treat patients with cancer or other blood or immune system disorders. Banking of umbilical cord blood is FDA-regulated and its use is approved for certain indications. Otherwise, consumers should be wary of claims by stem cell clinics implying FDA-approval.

So why aren’t more FDA-approved stem cell therapies available?

The FDA has strict regulations on using stem cell products in humans. In most cases, stem cell-based products are categorized the same way as pharmaceutical drugs. Therefore, each new therapy must go through a rigorous process including pre-clinical animal trials, phased clinical studies, and pre-market review by the FDA prior to offering the treatment in the clinic.

And with stringent regulatory requirements comes prohibitive costs. Research animals, Phase I-III clinical trials, and the regulatory demands for good manufacturing practice (GMP) labs result in an extraordinarily costly process that may hinder the progress of new therapies. The cost of developing a new drug has even been estimated to reach billions of dollars.

Nevertheless, a complete lack of regulation of stem cell therapies – as is seen in many of the stem cell clinics springing up worldwide – is clearly problematic. Alarmingly, many clinics advertise claims related to medical diseases for which there is no scientific consensus that supports their safety or efficacy. Premature commercialization of unproven therapies not only puts patients at risk, but also jeopardizes the credibility of still-developing stem cell products.

One of the most exciting outlooks for stem cell therapy is the prospect of using one’s own stem cells for personalized medicine. Should the development of an autologous stem cell product really be regulated the same way as a pharmaceutical drug, which is aimed at treating huge populations of people? If not, how should stem cell products be regulated?

In an effort to make the transition of novel stem cell products to the clinic more seamless, some countries have made significant changes in regulations. For instance, in 2014, Japan broke out a separate regulatory system for stem cell products that softened legislation dramatically to require only limited safety and efficacy data. Some argue that countries with softer regulations and less stringent safety and efficacy milestones, such as Japan, have poised themselves to become the likely pioneers in the field of regenerative medicine.

Regulatory frameworks for the clinical application of stem cell products are still evolving in most countries, including the U.S. In March, the Reliable and Effective Growth for Regenerative health Options that improve Wellness (REGROW) Act was introduced to congress. This change in legislation would remove some of the regulatory hurdles that hinder the progress of biologic therapies.

Regardless, the FDA needs to establish a more reasonable regulatory system that can evaluate the safety and efficacy of stem cell products in a more efficient manner.


1.  Berger, I., et al., Global Distribution of Businesses Marketing Stem Cell-Based Interventions. Cell Stem Cell, 2016. 19(2): p. 158-62.
2.  Turner, L. and P. Knoepfler, Selling Stem Cells in the USA: Assessing the Direct-to-Consumer Industry. Cell Stem Cell, 2016. 19(2): p. 154-7.

 

Generation of Human Stem Cells under Good Manufacturing Practice: Facility Update

cGMP Facility on Nancy Ridge Dr.

Allele’s New cGMP Facility on Nancy Ridge Dr.

Last year Allele dedicated a new building space for cleanroom operations to provide a cell banking service for personalized medicine. This facility will be the center of current Good Manufacturing Practice (cGMP) production of human induced pluripotent stem cells (iPSCs) using Allele’s proprietary synthetic mRNA platform. Over the past three months, progress to get the facility up and running has been substantial. Our facility includes four main modules: the reception area and doctors’ offices, a Fibroblast Isolation and Maintenance room, a Reprogramming and iPSC Maintenance room, and a Quality Control room. Air handling, which is a major component of the environmental control system, has been installed and validated. Equipment such as biosafety cabinets, incubators, and refrigerators have been installed and qualified, as well as equipment for performing essential quality control steps. To standardize personnel-related steps of cGMP processing, we have prepared rigorous SOPs and have extensively trained individual manufacturing operators. Overall, we are enthusiastic about the facility’s progress and are committed to delivering the best possible service as the industry leader in iPSC banking.

ScientistHood

iPSC roomCells

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Introducing EcoCulture Tissue Culture Plates

Allele Biotech is scoring one for the environment again. Our brand new EcoCulture Tissue Culture Dishes are designed with up to 40% less plastic than other brands, helping us attain our goal to minimize the menacing need for plastic consumables in the lab. Our high tech, environmentally friendly EcoCulture Dishes demonstrate much better imaging capabilities because of the thin lay of plastic at the bottom, and stronger physical strength even using only 60% plastic due to their patented design, aiding the environment by reducing energy consumption and decreasing the amount of plastic that will end up on our planet. An all inclusive environmental effort surrounds this brand new product line with our added commitment of donating 1% of profits from EcoCulture sales to an environmental aid organization (to be determined).

EcoCulture Dishes were a natural progression for Allele Biotech product design. For a long time our operations have included environmentally friendly endeavors; our recycling program which we conduct at a cost to us, our Box Swap program designed to reuse and reduce the need for Styrofoam, and our packaging methods that emphasize minimal use of materials as a long time company policy have all been executed in the interest of the environment. We have striven toward the belief that you do not have to sacrifice the planet in the name of research and the launch of our EcoCulture Dishes aims to spread our altruistic philosophies to our customers and partners in research!

Brochures and catalogue numbers of these products will become listed on our webpages shortly. Visit us often or follow us on Facebook, twitter, or myspace for updates on all our weekly promotions and new products of the week.

Promotion of the week: Falcon 96-well tissue culture grade plates giveaway–buy 1 bottle of our top quality FBS (validated for both mammalian and insect cells), receive 3 packages of 5×96 Falcon 3075 flat bottom tissue culture plates for free!

Promotion of the week 060710-06310: iPS factors with fluorescent protein tracers on ready-to-use high titer lentivirus, currently available-Oct3/4 with RFP and c-Myc with RFP, more to be added.

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Wednesday, June 9th, 2010 Open Forum, You have the power No Comments

Introducing Product-on-Demand Biological Research Reagents

The general order of operations in the bioreagent industry begins with a developer observing or forecasting a need and developing a product. The supplier then supplies that product to customers by showing that the product will suit their existing needs. An alternative order in our industry is after a new discovery in the form an enzyme reaction mechanism, affinity binding, or biological system is made in lab, someone realizes that discovery could be made into a product. If the idea is picked up by a commercial R&D team, the underlining mechanisms of the discovery are then exploited for particular use and reagents or kits will be built around it. The new products are introduced to the market by convincing potential users that they will make their research better, cheaper, or faster.

From a supplier’s point of view, if the current processes for developing new products have been working, what’s the incentive to change? From a researcher’s point of view, well, do they have any other choices? If something is not commercially available, someone will just make it in the lab if they need it. Some of us still remember the days when a graduate student needed to make his own restriction enzyme because NEB didn’t sell it. However, there is a disconnect between how much new knowledge is being gained every single day in tens of thousands of labs and how small a portion of that knowledge pool is being turned into more powerful tools to make the next round of research easier and more cost-effective. For instance, when an important gene’s promoter is recently defined by a functional study in 293T cells, how soon do you expect to test the signals that influence transcription from that promoter in the primary cells you are working on? Wouldn’t it be nice if you could simply buy a vector that will express a promoter-driven reporter ready to be introduced into the primary cells in your lab instead of having a graduate student design, construct, learn and try to make a lentiviral vector in the next few months?

And yes, there is the route called custom projects provided by a few bioreagent companies. The prices are often inhibiting for the reasons that the price needs to cover for labor on industry pay scale, materials, indirect, and profit. Additionally, since the service provider does not take ownership of the product, the work of researching the relevant pathways and making construct designs is left to the user.

There is a better way. A company can plan product groups, lines, and packages based solely on the demonstrated importance of a system such as signal pathway or a family of molecules like miRNA. The plan can project to use the most advanced technologies, even accompanied with full product descriptions and vector maps. However, it would be a great waste of money and material if nobody would ever need it, right? One way of dealing with the initial cost is that we make the first kit upon the first order. The customer that places the first order of a new product will get a deep discount off the shelf-product price on what used to be a custom project. They might even have the opportunity to provide input on the product design prior to production. From a supplier side, we will benefit by having an opportunity to initiate a new product without major investment, which in turn would keep our overall prices low for such innovative and advanced products.

This model should help speed up the commercial application of any new biological findings, lower the cost and price of bioreagent products, and encourage interaction between researchers who normally do not work with each other to produce better products for increasing the efficiency of research.

Discount of the week 060110-060710: Any virus packaging project initiated this week gets additional 10% discount that can be used with first time discount and other pricing advantages. http://www.allelebiotech.com/allele3/Services_Lentiviral_Retroviral_Packaging.php

New product of the week 060110-060710: Columns for Miniprep and Gel Purification, ABP-PP-COLM100. If you can make your own buffers or have leftovers from any miniprep or gel purification kits, get these high capacity columns and lower your costs by up to 70%!

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How I started my company and why–Inaugural Event by San Diego Entrepreneurs Exchange (SDEE)

For current graduate students, postdocs, and holders of other “in-transient” positions in bioscience-related fields today, a persistently resounding question on our minds is “What path should I follow at the end of a long and ragged journey of training?” Interestingly in our industry, like downhill skiing you see in the Winter Olympics, once you start one path it is not an easy switch to get on another.

Many of the Ph.D.s in biomed share the general view that an independent research position typically at an academic institute or non-profit organization such as San Diego’s local Salk, Scripps, or Sanford—Burnham, is the goal of the many years of training. Others soon realize that there are numerous research jobs at biotechnology and pharmaceutical companies that will make good use of their expertise, experience, and unique background knowledge in a particular field. And of course there are those who “defect” to different industries that may or may not directly relate to their extensive experience in wet labs, such as working in intellectual property laws, clinical trial management, biomedical sales, business development and management.

Research in major pharmaceutical companies (big pharma) normally focuses on a project with set goals, milestones, and layers of monitoring and management. That is how a large team can function together and get the tasks done in a timely manner. Working in smaller biotech companies can be much more flexible, researcher-initiated, and in many ways fun. On the other hand, you will be required to do much more than reading papers, designing experiments, obtaining and interpreting results. Starting a small biotech company is by no means an easy path to take, but if done correctly with some luck and a lot of determination, it can be a very rewarding career. You will get to utilize to the maximum extent of all your intelligence, knowledge, vision, and personal relations. You also have the opportunity to do real cutting-edge research in various areas, and see the fruits in journal publications, grant awards, as well as in the wild wide market.

The San Diego Entrepreneurs Exchange (SDEE) was founded by local San Diego entrepreneurs in order to provide a voice for the early stage technology startup, to encourage new entrepreneurs, and to sponsor networking and educational events that help develop the skills necessary to bring funding and business to the San Diego area.

The inaugural SDEE event to be held Wednesday March 10th at 5pm. It will help answer some of the questions you may have been thinking about regarding starting or working in a startup biotech company. Allele Biotech’s founder and CEO Dr. Jiwu Wang will be among the speakers. Ten years ago Dr, Wang was a postdoc at UCSD with an NIH fellowship, right before he started Allele with a number of NIH small business innovative research grants. He will talk about the ultimate “academic freedom”–doing any research you want but completely at your own risk– as the reason to start a technology-focused company, and the lessons he learned the hard way about running a lab vs organizing a business. Other speakers include CEOs from a number of San Diego biotech companies with great stories to share with postdocs and others. The talks will be brief yet informative, and on-site interactions are encouraged. The Sanford-Burnham building 12 is outside the main campus, with plenty of free parking. Click here for more details about the event. http://www.allelebiotech.com/allele3/SDEE-First-Event-Announcement.pdf (at AlleleNews). Let us know if you are coming by emailing to events@sdentrepreneurs.org

New Product/Service of the Week 02-15-10 to 02-21-10: Viral shRNA design and packaging services, packaging 2ml virus at 10e8 TU/ml for less than $1,400.

Promotion of the Week 02-15-10 to 02-21-10: FREE spreading beads (ABP-CE-CCCSB100, 500) to go with any competent cell order.

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