Cellular Dynamics Announces Launch of World’s Largest Public Stem Cell Bank With 300 Available iPSC Lines


The Madison, Wisconsin-based biotechnology company, Cellular Dynamics, has announced the launch , of the world’s largest public stem cell bank and the availability of the first 300 iPSC lines.

Cellular Dynamics International, Inc. (CDI), a FUJIFILM company, has announced the launch of the world’s largest publicly available stem cell bank, the California Institute for Regenerative Medicine (CIRM) hPSC Repository.  Additionally, CDI has also announced the availability of the first 300 induced pluripotent stem cell (iPSC) lines in September, 2015.  These high quality, disease-specific iPSC resources are now accessible to academic and industry researchers to model diseases, target discovery and drug discovery.

In 2013, CDI was awarded $16 million by the CIRM to create induced pluripotent stem cell (iPSC) lines for each of 3,000 healthy and diseased volunteer donors across 11 common diseases and disorders to be made available through the CIRM hPSC Repository.

In September, the CIRM hPSC Repository will make the first 300 iPSC lines available to researchers.  These lines include cells from healthy donors and donors suffering from non-alcoholic steatohepatitis (NASH), dilated cardiomyopathy, diseases of the eye and autism.  The number of available cell lines is expected to increase to 750 by February 2016.

iPSC lines can be created from any individual, and thus provide a powerful tool for understanding disease as well as studying genetic variation between individuals. Patient samples are collected primarily from standard doctor’s office blood draws.

The iPSC lines are accompanied by detailed demographic and clinical data and each line was derived from tissue samples collected from living donors.

iPSCs have the potential to be differentiated into any cell type in the human body, and CDI already routinely manufactures 12 iPSC-derived cell types, including high-quality heart, neural, liver and endothelial cells, in high quantity, and at very high levels of purity.  Through CDI’s MyCell® Product portfolio, researchers can order iPSC line(s) of interest and have them differentiated into the cell type of choice.

Researchers can obtain undifferentiated iPSC lines through the Coriell Institute for Medical Research.

What are researchers saying about this advance:

Kaz Hirao, CDI Chairman and CEO, said, “iPSCs are proving to be powerful tools for disease modeling, drug discovery and the development of cell therapies, capturing human disease and individual genetic variability in ways that are not possible with other cellular models. We’ve seen a dramatic increase in the availability of iPSC lines. We’re pleased to be the vendor of choice for creating high quality iPSC lines and enabling scientists from academia and industry to better understand and help develop treatments for major diseases. The lines available from the CIRM stem cell bank directly complement CDI’s ability to provide differentiated cells corresponding to each of these iPSC lines, which will allow researchers to model the diseases represented, better understand disease progression, perform more targeted drug discovery, and ultimately lead to better treatments.”

Jonathan Thomas, Ph.D., J.D., CIRM Chairman, said, “We believe the bank will be an extraordinarily important resource in helping advance the use of stem cell tools for the study of diseases and finding new ways to treat them. While many stem cell efforts in the past have provided badly needed new tools for studying rare genetic diseases, this bank represents common diseases that afflict many Californians. Stem cell technology offers a critical new approach toward developing new treatments and cures for those diseases as well.”

Michael Christman, Ph.D., president and CEO of Coriell said, “Coriell Institute is a leader in managing large and complex biospecimen collections and distributing samples and data worldwide to promote research. We are very pleased to be part of this CIRM initiative and advance stem cell research for several devastating yet common diseases.”

City of Hope Launches Alpha Clinics – A New Stem Cell Clinic


Cancer patients usually have three different options: surgery, chemotherapy and radiation therapy. None of these options elicits a great deal of confidence. A new study at City of Hope will officially open the Alpha Clinic for Cell Therapy and Innovation. At this center, patients battling cancer and other life-threatening diseases will have another option: stem cell-based therapy.

The Alpha Clinic, which officially opened March 19, is funded by an $8 million, five-year grant from the California Institute for Regenerative Medicine. This grant will also be supplemented by matching funds from City of Hope. The Alpha clinic will combine the uniquely patient-centered care for which City of Hope is well-known with the most innovative, stem cell-based therapies being studied to date. This approach will hopefully revolutionize the treatment of not only cancer, but also AIDS and other life-threatening diseases.

“We are in a new era of cellular therapy,” said John Zaia, M.D., the Aaron D. Miller and Edith Miller Chair in Gene Therapy, chair of the Department of Virology and principal investigator for the stem cell clinic. “The California Institute for Regenerative Medicine recognizes this, and they have been leading the field. Alpha Clinics like ours aim not only to provide research to benefit patients in the future, but also to get these innovative treatments running in real-life clinics to benefit patients now.”

The christening of City of Hope’s Alpha Clinic is the culmination of a decade of planning and visionary thinking. When the state of Californian voted to found the California Institute for Regenerative Medicine, the funds now became available to start the institute. New stem cell therapies are ready for clinical trials, and City of Hope is home to one of three Alpha Clinics in the state. The other two clinics are at the University of California San Diego and a joint clinic by University of California Los Angeles and University of California Irvine.

City of Hope’s first trials will study stem cell-based therapies for leukemia, and the use of neural stem cells to deliver treatments to brain tumors. The first such study will modify a patient’s own immune cells so that they can recognize and fight cancer cells. Cancer researchers hope the modified cells will be able to attack existing cancer cells, and also be able to attack the cancer again should it recur.

Brain cancer patients will also be able to enroll in a study that uses neural stem cells, which have an innate ability to home to tumor cells, as a delivery mechanisms for cancer drugs. Genetically engineered neural stem cells can bring targeted therapies across the blood-brain barrier, and can potentially deliver drugs directly to tumor sites, which eliminates systemic toxicity.

The US Food and Drug Administration (USFDA) has already approved a new HIV trial that will be conducted at the City of Hope Alpha Clinic. This trial will use “molecular scissors” known as zinc finger nucleases to edit the blood cells of HIV patients and remove a specific gene. Without this particular gene, the cells are unable to produce a protein that HIV requires in order to invade cells and replicate. The approach has the potential to eliminate HIV from the body.

“As we move forward with our Alpha Clinic, we will also be defining a new discipline in nursing of cellular therapy,” said Shirley Johnson, R.N., senior vice president, chief nursing and patient services officer at City of Hope. “This clinic is a unique opportunity to provide patients with the most leading-edge treatments while still giving them the compassionate comprehensive care City of Hope patients expect.”

The Alpha Clinic launched officially on March 19. Future trials will include T cell immunotherapy for blood cancer, new brain cancer therapies, treatments for breast cancer metastases and ovarian cancer treatments. Zaia said the clinic also plans to work with City of Hope’s diabetes researchers to introduce treatments for diabetes, exploring both the potential of pancreatic stem cells and preventing the immune system from attacking insulin-producing cells.

StemCells, Inc., Sued by Former Employee Who Says Their Stem Cell Treatment is Unsafe


A California stem cell company, StemCells, Inc., that is developing cell-based therapies for several different neurological and eye conditions, is being sued by a former employee (whistleblower) who claims that the company did not follow proper protocols in the preparation of their treatments. Rob Williams, who was once a senior manager at StemCells, Inc., has alleged that the company fired him after he brought these problems to the attention of senior management.

According to the Courthouse New Service, Williams in the lawsuit stated that he “noted poor sterile technique, failure to adhere to current Good Manufacturing Practices in the company’s manufacturing process, and substantial deficiencies in the company’s Manual Aseptic Processing of HuCNS-SC (Human Central Nervous System Stem Cells) cell lines—failure and deficiencies that put patients at risk of infection or death during ongoing clinical trials.”

Ken Stratton, who serves as the general counsel for StemCells, Inc., has told the California Stem Cell Report that Williams’s employment “was terminated for performance deficiencies, and [the company] finds no merit to the allegations.” Stratton also said that “the elements of manufacturing practices that concerned Mr. Williams were immediately and carefully reviewed by the company.”

It might be worth noting that this lawsuit coincides with the departure this past April or May of StemCells, Inc.’s Executive VP of Manufacturing Operations and Regulatory Affairs, Stewart Craig, who took a position at Sangamo Biosciences.

Unfortunately for StemCells, Inc., this particular lawsuit comes soon after a second bit of bad press. Embryologist Alan Trounson led the California Institute for Regenerative Medicine (CIRM) until June of this year, but has joined the board of StemCells, Inc., shortly after leaving the state stem cell research funding agency. According to an opinion article written by Ron Leuty, who is a reporter for the San Francisco Business Times, Trounson has recused himself from discussions regarding a loan StemCells, Inc., received from CIRM in 2012 because of his close relationship with the company’s founder. “But the speed of his appointment to the StemCells board has raised questions” about a possible conflict of interest, Leuty wrote.

CIRM has been marred by conflicts of interest accusations since California voters in 2004 birthed CIRM through Proposition 71 and the subsequent sale of $3 billion in state bonds. Now it has one more strike against it.

Leuty called the situation an embarrassment for CIRM. “If the public perceives that individuals—researchers or CIRM employees or company executives—are feeding at the trough of the semiautonomous public agency, it isn’t going to help CIRM get more cash from that very same public that foots the bill.”

Phase 2 Clinical Trial that Tests Stem Cell Treatment for Heart Attack Patients to be Funded by California Institute for Regenerative Medicine


A new stem cell therapy that treats heart attack patients with cells from a donor has been approved to begin a Phase 2 clinical trial.

Capricor Therapeutics Inc. a regenerative medicine company, has developed this treatment, which extracts donor stem cells from the heart called “cardiosphere-derived cells,” and then infuses them into the heart of the heart attack patient by means of a heart catheter procedure, which is quite safe. These stem cells are introduced into the heart to reduce scarring in the heart and potentially replace dead heart muscle cells. One clinical trial called the CADUCEUS trial has already shown that cardiosphere-derived cells can reduce the size of the heart scar.

In a previous phase I study (phase I studies typically only ascertain the safety of a treatment), cardiosphere-derived cells were infused into the hearts of 14 heart attack patients. No major safety issues were observed with these treatments, and therefore, phase 2 studies were warranted.

Alan Trounson, Ph.D., president of the California Institute for Regenerative Medicine (CIRM), which is funding the trial, said this about the phase 2 trial approval: “This is really encouraging news and marks a potential milestone for the use of stem cells to treat heart disease. Funding this type of work is precisely what our Disease Team Awards were designed to do, to give promising treatments up to $20 million dollars to develop new treatments for some of the deadliest diseases in America.”

Capricor was given approval by the National Heart Lung and Blood Institute (NHLBI) Gene and Cell Therapy (GST) to move into the next phase of clinical trials after these regulatory bodies had thoroughly reviewed the safety data from the phase 1 study. After NHLBI and GST determined that the phase 1 study met all the required goals, CIRM also independently reviewed the safety data from the Phase 1 and other aspects of the Phase 2 clinical trial design and operations. Upon successful completion of the independent review, Capricor was given approval to move forward into the CIRM-funded Phase 2 component of the study

Capricor CEO Linda Marbán, Ph.D., said, “Meeting the safety endpoints in the Phase 1 portion of the trial is a giant leap forward for the field and for Capricor Therapeutics. By moving into the Phase 2 portion of this trial, we can now attempt to replicate the results in a larger population.”

For the next phase, an estimated 300 patients who have had heart attacks will be evaluated in a double-blind, randomized, placebo-controlled trial. One group of heart-attack patients will include people 30 to 90 days following the heart attack, and a second group will follow patients 91 days to one year after the incident. Other patients will receive placebos and neither the patients nor the treating physicians know who will receive what.  This clinical trial should definitely determine if an “off-the-shelf” stem cell product can improve the function of a heart attack patient’s heart.

The California Institute for Regenerative Medicine (CIRM) is funding this clinical trial, and for this CIRM should be lauded.  However, when CIRM was brought into existence through the passage of proposition 71, it sold itself as a state-funded entity that would deliver embryonic stem cell-based cures.  Now I know that director Alan Trounson has denied that, but Wesley Smith at the National Review “Human Exceptionalism” blog and the LA times blogger Michael Hiltzik have both documented that Trounson and others said exactly that.  Isn’t ironic that one of the promises intimated by means of embryo-destroying research is now being fulfilled by means of non-embryo-destroying procedures?  If taxpayer money is going to fund research like this, then I’m all for it, but CIRM has to first clean up its administrative act before they deserve a another penny of taxpayer money.

California Stem Cell Report Includes No Critics


Wesley Smith at his blog notes that the California Stem Cell Report, which will include public testimony to the Institute of Medicine (IOM), an arm of the National Institutes of Health (NIH), will include scientists who were awarded lucrative grants by the California Institute for Regenerative Medicine (CIRM), but no critics of the program.  His source is a very critical Los Angeles Times article.

The critics of CIRM are not pro-life advocates who oppose embryonic stem cell research on principle.  Instead critics include the Little Hoover Commission, which issued this blistering report of CIRM, and the Oakland-based Center for Genetics and Society.  These organizations were afraid that there were too many conflicts of interest on the grant-awarding panel.  In the words of the Little Hoover Commission:

CIRM’s 29-member oversight committee includes representatives from institutions that have benefitted from grants the committee approved. This structure, along with overly long terms and the inability to nominate its own leaders or hold them accountable, fuels concerns that the committee never can be entirely free of conflict of interest or self-dealing, notwithstanding a court ruling that established the legality of such a structure. Legal is not necessarily optimal, however, and litigation over this issue delayed CIRM from beginning its work. As long as the board remains in its present form, its structure will draw scrutiny, diverting CIRM resources.

No representatives from either of these critical institutions are on the witness list.  Why aren’t members of the public allowed to address the IOM?  According to the LA Times,  the proprietor of the California Stem Cell Report, David Jensen, says he asked the IOM why no objective witnesses were on the hearing list, and an IOM public relations person directed him to a survey form members of the public could fill out (though the link for the form on the IOM’s website was dead when I checked it).  Apparently, members of the public will also be permitted to address the IOM panel at Tuesday’s hearing. They’ll each get up to five minutes.

CIRM is selling the people of California a bill of goods.  In 2014, CIRM will be back to the people of California with their hand out for more money.  If the process is so objective, then what do they have to hide?  3 billion dollars later and little to show for it except for lots of dead human embryos.  People will be more than a little miffed; and they should be.