Roundtable on cGMP Stem Cell Manufacturing

Allele Biotechnology & Pharmaceuticals is hosting a cGMP Stem Cell Manufacturing Roundtable to discuss ways to accelerate stem cell-based therapies toward clinical development and commercialization. The roundtable will bring together top minds from academic settings, cGMP facilities, and biotech industries in an informal setting to explore partnerships and avenues for developing effective and marketable iPSC-derived therapies.

The meeting will be held on April 20th at The Hilton on Torrey Pines and will consist of four sessions covering (1) existing cGMP facilities, (2) manufacturing and quality systems, (3) regulatory concerns, and (4) business strategy.

Meeting highlights will be produced to summarize the presentations and discussions. For inquiries, contact info@allelebiotech.com or call 858-587-6645.

Allele-iPSC News Translate: iPS clinical research encounters a problem. Doctors say “the problem is not caused by iPSCs”

Kobe City Medical Center General Hospital and Riken Research Institute announced and reported to the Japanese government that a patient who had received allogeneic iPSC-derived cells developed an “epi retinal membrane”, which they subsequently removed by operation. Dr. Masayo Takahashi at Riken says “the problem is caused by the transplantation procedure, but not by iPSCs. This will not affect future clinical research that uses iPSCs.”

The laws that govern regenerative medicine in Japan mandates that the deaths and hospitalizations that occur during treatment need to be reported to the government as “serious harmful effects”. This is the first such report involving iPSC clinical research.

The problem occurred to a man in his 70s, who is at the risk of blindness due to “wet age-related macular degeneration”. Last June, he received a transplantation of the solution containing allogeneic iPSC-derived retinal pigment epithelium (RPE) in his left eyes. Last October, the epi retinal membrane and swelling started to develop and the membrane was removed on January 15.

The possibility exists that the solution leaked from the needle hole during the transplantation, and the leaked cells might have formed the membrane. The transplanted cells inside the retina are stable and there has been no decline in his eyesight.

Dr. Takahashi says “although this event qualifies as a serious harmful event, the patient’s condition has not worsened and there has been no rejection of transplanted cells”. Dr. Yasuo Kurimoto, a surgeon who performed the operation, says “the procedure was the problem. We would like to improve the method, in order to make iPSC therapy a common treatment.”

The current clinical trial targets patients with wet age-related macular degeneration and is run by the Kobe City Hospital, Riken, Osaka-University Hospital, and Kyoto-University CiRA (Dr. Shinya Yamanaka). Between last March and October, five patients have received the transplantation.

Original News Credits: https://www.kobe-np.co.jp/news/iryou/201801/0010902012.shtml

Tags: AMD, eye disease, iPSC, Japan iPSC, RPE, stem cells, Takanishi, Yamanaka

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

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.

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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.

Tags: biotech and planet, c-Myc RFP, environmental charity donation, environmentally conscious business, Oct3/4 promoter GFP, tissue culture plates