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Posts Tagged ‘Stem Cells

Targeting the spread of unregulated Stem Cell and regenerative therapies

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By: Kellsye Fabian, PhD

Image by Darko Stojanovic from Pixabay

Advances in regenerative medicine research have generated significant public interest in therapies that have the potential to restore the normal function of cells, tissues and organs that have been damaged by age, disease, or trauma. Investment and enthusiasm in this field have propelled the development of regenerative therapies such as cell therapy, bioengineered tissue products, and gene therapy. While several hundreds of these treatments have progressed to clinical trials, the Food and Drug Administration (FDA) has approved only a few regenerative therapies. Of these, most are stem cell-based products derived from umbilical cord blood used to treat blood cancers and immune disorders, and three are gene therapies to treat cancer or blindness. 

Alarmingly, an increasing number of businesses and for-profit clinics have been marketing regenerative therapies, mostly stem cell products, that have not been reviewed by the FDA. In 2016, there were 351 stem cell businesses offering interventions that could be administered in 570 clinics. That number was estimated to have doubled in 2018. Most of these establishments tout that their products can treat or cure serious illnesses and/or provide a wide range of benefits. These claims are often unsubstantiated. Moreover, these unapproved interventions pose a great danger to patients and have resulted in serious complications including blindness, infections, cardiovascular complications, cancer and death.

 Some patients remain willing to take the risks, especially those with serious diseases who have exhausted all possible conventional treatment or those that may be searching for alternative therapies. These individuals often fall prey to the overly optimistic portrayals of stem cell products in the media advertisements from stem cell companies. 

For years, these unscrupulous businesses have avoided heavy regulations. Physicians, researchers and ethicists, have urged for stricter monitoring of regenerative therapies as the commercial activity related to these interventions expanded. In response, the FDA has increased its oversight of the field and has issued guidance relating to the regulation of human cells, tissues and cellular or tissue-based products (HCT/Ps) to ensure that commercialized regenerative therapies are safe and are founded on scientific evidence.

FDA increased oversight 

Since 2017, the FDA has increased oversight and enforcement of regulations against unscrupulous providers of stem cell products. In 2018, the FDA sought permanent injunctions against two stem cell clinics, California Stem Cell Treatment Center Inc and Florida-based US Stem Cell Clinic LLC, for selling unapproved stem cell products and for significantly deviating from current good manufacturing practice requirements that ensure the sterility of biological products. 

The case against California Stem Cell Treatment Centers began in August 2017, when the US Marshals Service, on behalf of the FDA, seized five vials of smallpox virus vaccine from a clinic affiliated with California Stem Cell Treatment Centers. The vaccine was provided by a company called StemImmune and was being combined with stromal vascular fraction (SVF), which are cells derived from patient adipose (fat) tissues that consists of a variety of cells, including a small number of mesenchymal stem cells. This combined product was then administered to cancer patients in California Stem Cell Treatment Centers intravenously or through direct injection into patients’ tumors. 

Cancer patients have potentially compromised immune systems and the use of a vaccine in this manner could pose great risks, such as inflammation and swelling of the heart and surrounding tissues, to the patients. In addition, California Stem Cell Treatment Center provided unapproved treatments to patients with arthritis, stroke, ALS, multiple sclerosis, macular degeneration, Parkinson’s disease, COPD, and diabetes. The injunction case against California Stem Cell Treatment Center is still pending.

US Stem Cell Clinic also marketed SVF to patients seeking for treatment for conditions such as Parkinson’s disease, amyotrophic lateral sclerosis (ALS), chronic obstructive pulmonary disease (COPD), heart disease and pulmonary fibrosis. Three women with macular degeneration, an eye disease that causes vision loss, went blind after receiving eye injections of SVF products from US Stem Cell Clinic. Following these events, in June 2019 a Florida judge ruled that the FDA is entitled to an injunction against US Stem Cell Clinic, meaning that the FDA has the authority to regulate them and stop them from providing potentially harmful products.

While this decision strengthened the position of the FDA as a regulatory body for regenerative medicine, businesses have found different tactics to continue selling unapproved products. After the court ruling, US Stem Cell Clinic stopped selling the fat-based procedure. However, it said that it would continue to offer other stem cell treatments. Instead of stem cells derived from fat, which was the topic of the injunction, the company would now harvest cells from patients’ bone marrow and other tissues to “treat” different conditions. Another company, Liveyon, was given a warning by the FDA in December of 2019 for selling unapproved umbilical-cord blood-based products that were tied to life-threatening bacterial infections. Liveyon has since halted the distribution of its products in the US but has opened a clinic in Cancun, Mexico where it has continues “treating” patients outside the scope of the FDA. Other companies have changed their terminology and marketing language to escape the FDA crackdown against stem cell clinics. Instead of using the phrase “stem cells” in their websites and advertising, they now use “cellular therapy” and “allografts.”

The FDA’s Regulatory Framework for Regenerative Medicine

The warnings and injunctions filed by the FDA against the aforementioned stem cell businesses were in conjunction with the comprehensive policy framework for regenerative medicine that the agency announced in November 2017. The policy framework aims to clarify which medical products are subject to the agency’s approval requirements and to streamline the review process for new regenerative therapies. 

In the case of cellular and tissue products/procedure, there is often a gray area concerning what should be considered medical products, which are under FDA oversight, and what should be considered an individualized treatment being performed by a doctor within their medical practice, which is not regulated by the FDA. Stem cell clinics have often used this ambiguity as justification to sell products without FDA approval. According to the new guidelines, for cells and tissue to be exempt from FDA regulation, several criteria should be met: 1) they must be taken from and given back to the same individual during the same surgery, 2) they must not undergo significant manufacturing (minimal manipulation), 3) they must perform the same basic function (homologous use) when re-introduced to the patient, 4) they must not be combined with another drug or device, and 5) the benefits and risks must be well understood. If any of these criteria are not met, the cell or tissue is considered a drug or biologic and is subject to pre-market review of the FDA. Some ambiguities still persist in the current form of the policy, such as what constitutes “minimal manipulation” and how to address nonhomologous use (i.e. the cells or tissues are used in ways other than its original function). The guidelines are an important starting point in determining which therapies are under the FDA’s purview and continued dialogue between the FDA and stakeholders involved in product development will provide more clarity about how products will be classified.

The policy framework also addresses how the FDA aims to implement the regenerative medicine provisions of the 21st Century Cures Act. Signed into law in 2016, the Cures Act is designed to expedite the development and review of innovative medical products. One of the new programs under this law is the Regenerative Medicine Advanced Therapy (RMAT) designation. A product is only eligible for RMAT designation if 1) it is a cell therapy, therapeutic tissue-engineering product, HCT/P, gene therapy, or combination product using any such therapy; 2) it is intended to treat, modify, reverse, or cure a serious condition; and 3) preliminary clinical evidence indicates that the therapy has the potential to address unmet medical needs for such condition. Stakeholders involved in product development strongly support the creation of this expedited review program. Meanwhile, others are concerned that the RMAT designation will lead to the approval of therapies based on fewer or smaller studies and, hence, treatment-related adverse events would emerge only after a product is on the market. But since RMAT therapies are intended to treat serious conditions, the risks may be acceptable and may be outweighed by the benefits to the patients. Nevertheless, postmarket studies would be essential and must be required to ensure the safety and efficacy of RMAT therapies. 

The establishment of these policy frameworks are definitely a step towards better regulation of the unbridled regenerative therapies. The increased enforcement of these new guidelines will hopefully dissuade unscrupulous businesses from taking shortcuts while encouraging legitimate companies to develop novel treatments. This will ensure that regenerative medicine will continue to be an exciting field that has the potential to provide innovative treatments that will improve human health. 

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Written by sciencepolicyforall

January 24, 2020 at 7:36 pm

Science Policy Around the Web – June 7th, 2019

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By: Mary Weston, Ph.D.

Source: Pixabay

Pfizer had clues its blockbuster drug could prevent Alzheimer’s. Why didn’t it tell the world?

Last Tuesday, the Washington Post reported that the biopharmaceutical company Pfizer had hints that their rheumatoid arthritis drug Enbrel might reduce the risk of Alzheimer’s disease, but chose not to report these findings to the public.

In 2015, after analyzing hundreds of thousands of insurance claims, a team of Pfizer researchers observed that their anti-inflammatory drug Enbrel might also decrease the risk for Alzheimer’s by 64%. They recommended that the company conduct a costly clinical trial to prove the link but, after several years of internal debate, the company decided not to pursue the lead.  The question remains: why did Pfizer not release these findings to the scientific community?

Pfizer claims they did not pursue the research due to scientific considerations – they argue that since Enbrel cannot cross the blood-brain barrier and directly reach brain tissue, it is unlikely to prevent the debilitating neurodegenerative disease. Further, Pfizer claimed that they did not to report the research because the statistical findings did meet “rigorous scientific standards” and were concerned about misleading researchers down a false path. However, Pfizer is also losing its patent protection on Enbrel soon, meaning that generics will become available and the drug will be much less profitable, reducing any financial incentive for further research or clinical trials (likely to cost around $80 million).

Some in the scientific community are questioning Pfizer’s justification. Keenan Walker, an assistant professor of medicine at Johns Hopkins, argues that the scientific community benefits when the data is available, stating that ““[w]hether it was positive data or negative data, it gives us more information to make better informed decisions.’’

Several scientists argue that Pfizer’s results should be release because they could provide clues to combating the disease and slowing cognitive decline in its earliest stages. Specifically, recent research is hinting that inflammation may promote Alzheimer’s disease. Further, neurodegenerative research is notoriously challenging and there are no major drugs that treat Alzheimer’s. Even several recent phase 3 clinical trials have been halted because the drugs were not effective. Due to a lack of progress in the field, a couple large pharmaceutical companies, including Pfizer, have just closed their neurology-related research programs.

 (Christopher Rowland, Washington Post)

Trump administration halts fetal-tissue research by government scientists

The Trump administration has announced that government scientists will stop using human fetal tissue for research and is placing new limitations on researchers in academic settings who use federal funding from the NIH.

It is not entirely known how many research projects will be affected by the new regulations. Government scientists will be allowed to continue their current work, but are prohibited from acquiring new tissue samples. Current extramural research at universities and privately funded work can continue but any new grant proposals or renewals of existing projects must be approved by an ethics advisory board that will be formed.

In addition to halting government fetal tissue research, the administration has decided to cancel an ongoing HIV research contract with the University of California San Francisco, effectively ending a 30-year partnership. The project involves using fetal tissue to develop mouse models with human-like immune systems to develop new HIV therapies.

Use of fetal tissue is essential to for studying certain human biological processes, such as kidney development. Often biomedical research uses mice as substitutes of people, but in this case, murine kidney development is too different from their human counterparts to be of use. Some researchers fear that these new restrictions will set back certain research for years to come. Important areas of research that depend on using fetal tissue including HIV, neurodegeneration, human organ growth and regeneration, Zika (determining how/why the virus affects developing fetuses so severely), and certain types of vaccine development.

POLITICO reports that this decision was made after much debate between the White and the Department of Health and Human Services (HHS), which wanted a less restrictive policy. In a statement released Wednesday, HHS said that “promoting the dignity of human life from conception to natural death is one of the very top priorities of President Trump’s administration.” HHS is now reviewing whether sufficient alternatives to human fetal tissue exist and will be supporting the development and validation of these models. However, good alternatives for certain fetal tissue research are elusive and many scientists say that the tissue is essential for some fields.

 (Sara Reardon, Nature)

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June 7, 2019 at 6:11 pm

Science Policy Around the Web – November 2, 2018

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By: Allison Cross, Ph.D.


Source: Flickr

Opioid Epidemic

Signing Opioid Law, Trump Pledges To End ‘Scourge’ Of Drug Addiction

The nation is facing a devastating opioid epidemic.  In 2017 alone, the CDC reported over 70,000 drug overdose deaths in the US, with about two-thirds of those deaths linked to opioids.  More strictly, it is estimated that more than 115 people die every day in America from opioid-related overdose. President Trump declared the opioid epidemic a national public health emergency in October of 2017. And now, on October 24th 2018, President Trump signed into law the Substance Use-Disorder Prevention that Promotes Opioid Recovery and Treatment (SUPPORT) for Patients and Communities Act.  The bill received bipartisan support, passing 393–8 in the House and 98–1 in the Senate.

 What does the new legislation do?

The new law is intended to combat the opioid crisis though Medicaid, Medicare and public health reforms. These will address over prescription of opioids, advance treatment options, encourage development of non-addictive painkillers and stop the flow of illicit drugs from outside the country.

What does the new law mean specifically for the FDA?

With the passing of the bill,  FDA Commissioner, Scott Gottlieb, released a statement that the new legislation grants the FDA additional authorities they believe will be meaningful in advancing their efforts to combat the opioid crisis. The FDA has taken new steps over the past 18 months to work towards combating the crisis, and has focused its efforts in 4 main areas; decreasing exposure to opioids, advancing innovation in low-risk pain medications, developing and implementing improved treatment for opioid use disorder and increasing efforts to prevent the illegal shipment of drugs including fentanyl into the US.

The new law gives the FDA authority to destroy illegally imported or hazardous drugs and halt the distribution of any drug that is deemed a public hazard. It also gives the FDA authority to prohibit all future drug importation from those convicted of a felony involving illegal import of drugs and from manufacturers, distributors, or importers who have shown a pattern of importing misbranded drugs. This will allow the FDA to expand and improve their oversight of international mail facilities and prevent drugs from entering the US illegally.

The SUPPORT Act also gives the FDA authority to order a recall for a controlled substance that is found to cause serious adverse health consequences or death.  Up to this point, a recall was a voluntary action taken by a company to remove their product from the market.

With the new legislation, the FDA has the ability to require specific packaging and disposal of drugs that pose a risk of abuse. This could help lower the number of opioids in the hands of patients by requiring packaging such as unit dose blister packs and requiring that opioids be dispensed with a mail-back pouch or other safe disposal option.

The SUPPORT Act clarifies the FDA’s authority to require post-approval studies of drugs that contain controlled substances.  These studies will help advance the understanding of opioid pain medicines including the long-term efficacy of opioids for the treatment of chronic pain.  Furthermore, the act requires that the FDA provide guidance for collecting data on opioid sharing and including this information on product labels.

Is it enough?

Despite bipartisan support, and most experts and advocates viewing the changes in the law as positive, some people are already voicing concerns that the bill is just not enough. The biggest criticisms come down to money.  The legislation makes a lot of legal and regulatory tweaks, but it does not provide a significant increase in spending for the opioid crisis.

(Ayesha Rascoe and Scott Horsley, NPR)

(FDA Commissioner Scott Gottlieb, FDA Press Announcements)


Officials: Number Of Measles Cases Doubles In Just Days, 33 And Climbing

As of October 19, 2018 there were 17 cases of measles reported in New York state linked to recent travel to Israel.  These cases, 11 of which were reported in Rockland County, sparked the Rockland health commissioner to require unvaccinated students attending schools in which confirmed cases were reported to remain home until November 3rd.  On October 28th, health officials announced that the number of confirmed cases had more than doubled, with a total of 33 confirmed cases.

This news comes at the same time as misleading headlines suggesting a multi-state measles outbreak in the United States.  Though the CDC has reported 142 individual cases of measles confirmed in 25 states and the District of Columbia as of October 6th 2018, the CDC recently tweeted that the “number of US-reported cases in 2018 is similar to recent years & in expected range.”  Despite the lack of a multi-state outbreak, the CDC does warn that the measles is still common in many parts of the world and people should get vaccinated.  The prevalence of measles across the globe allows it to be brought to the US by unvaccinated travelers, as it was in the recent local outbreak in New York.

A vaccine to measles first became available in United States in 1963, with an improved version developed in 1968. The vaccine, normally combined with the mumps and rubella vaccines, is commonly known as the MMR vaccine. With availability of a vaccine, disease rates were dramatically reduced and the disease was declared eliminated (absence of continuous disease transmission for greater than 12 months) in the United States in 2000.

Despite the availability of the MMR vaccine and disease elimination, local outbreak of measles (like other vaccine-preventable diseases) occur every year in the United States.  There are no US federal vaccination laws, but children attending public schools in all 50 states are required by state law to be vaccinated against measles.  All states, however, do allow medical exemptions, 47 states allow exemptions for religious reasons, and 18 states allow personal belief exemptions.

A recent study published in PLOS Medicine examining the relationship between non-medical expectation (NME) rates and actual vaccine coverage showed that states with higher NME rates exhibited lower rates of MMR vaccination among kindergarteners.  The 2015 measles outbreak linked to the Disneyland Resort in Anaheim, California was believed to be the result of substandard vaccination compliance, with a 50%–86% vaccination rate reported among the exposed population.  Following this outbreak, the California State Legislation passed a statewide bill banning NMEs beginning in January 2016, resulting in an increase in the state’s kindergarten vaccination rate.

In 2017, the CDC reported that 91.1% of children between 19-35 months received the MMR vaccine nationwide.  With measles being a highly contagious disease, a study from 2015estimated that vaccination rates need to be as high as 96 to 99% are to preserve herd immunity and prevent outbreaks.  This raises the question of whether stricter legislative action should be taken to put an end to NMEs in order to protect children from the measles and other vaccine-preventable diseases.


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Written by sciencepolicyforall

November 2, 2018 at 2:22 pm

Science Policy Around the Web – October 30, 2018

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By: Ben Wolfson, Ph.D.



What a massive database of retracted papers reveals about science publishing’s “death penalty”

One of the biggest issues facing research is the high number of retractions of published papers. Almost a ten years ago, members of the scientific community began to realize that there the number of retractions was increasing. The blog Retraction Watch was formed in 2010 to report on the phenomenon, and in October 2011, Nature published an article finding that retractions had increased 10-fold in the past decade. These and other sources sparked introspection within the scientific community and calls for structural reforms, however these have yet to result in systematic change. In part this is due to the inherent difficulty of examining the retraction phenomenon. While the increase in retraction was alarming, the lack of consistency in how journals reported them confounded statistics, making direct analysis and subsequent action difficult. One strategy for dealing with this has been for third parties to create their own databases. Over the past decade of reporting, Retraction Watch compiled a list of retractions, which was made public as a searchable database this past week. 

Through analysis of the database, Science magazine made several conclusions, including that the rate of increase of retractions has slowed, and that the a high percentage of the growing number of retractions is likely due to improved oversight rather than increased research misconduct. Moreover, of the retractions due to misconduct, a disproportionate number of them are from a small population of authors, demonstrating a potential pattern of “bad-actors” within the scientific community. 

In addition to the confounding statistics, the stigma associated with retractions makes it more difficult for reforms to be carried out. Authors whose papers are retracted often do not want to cooperate, and journals are sometimes hesitant to retract a paper, knowing that such action bears serious implications for the author.

Retraction Watch’s database is currently the most detailed source on retractions and an official database is necessary infrastructure for the future of scientific research. While the conclusions drawn by Science are a good first step, they also indicate the breadth of the work yet to be done.

(Jeffrey Brainard, Jia You, Science)

Stem cell clinics

Federal Trade Commission takes action against stem cell clinics

On October 12th the Federal Trade Commission took its first action against stem cell clinics when they filed a complaint concerning unsupported health claims made by two Californian stem cell clinics. The clinics had advertised that their amniotic stem cell therapy was capable of treating a variety of diseases, including heart attacks, autism, macular degeneration, Parkinson’s disease, cerebral palsy, and multiple sclerosis, none of which currently have FDA approved stem cell therapies. After negotiation, the companies have settled the complaint, agreeing to pay a fine and not engage in false advertising in the future.

Hundreds of stem cell clinics have come into existence in recent year, selling treatments for a multitude of conditions. While stem cell research is a current hot topic and researchers hope that stem cells may one day be used to repair damaged tissues and organs, the treatments sold by these clinics are not proven or FDA approved. As of 2016, there were 570 stem cell clinics throughout the United States. These clinics advertise autologous stem cell procedures, wherein a patient’s own stem cells are harvested from their adipose tissue (fat) or bone marrow and reinjected into the patient. Stem cell clinics claim that these treatments should not be FDA regulated as they are made up of patient’s own cells, and that they are merely facilitating patients utilization of their own natural healing ability. 

This action by the FTC follows the Food and Drug Association seeking a permanent injunction against two clinics in California and Florida in May of 2018 as a result of reports of misconduct by both clinics. The California clinic, Stem Cell Treatment Center, created an experimental cancer treatment from a non-commercially available smallpox vaccine, and procedures conducted by the Florida-based US Stem Cell clinic resulted in 3 women becoming legally blind in 2015. 

These are not the only cases of patients being harmed by stem cell treatments, and experts hope that this FTC and FDA activity indicate a new beginning for regulation of U.S. stem cell clinics. 

(Ashley P. Taylor, The Scientist)

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October 30, 2018 at 4:36 pm

Science Policy Around the Web – July 6, 2018

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By: Kelly Tomins, BSc


source: pixabay

Genetic privacy

Could DNA Testing Reunite Immigrant Families? Get the Facts.

Since the enactment of the Trump administration’s “zero tolerance” immigration policy, over 2300 children have been separated from their families at the border. The policy caused widespread outrage throughout the US, and over 400,000 people protested the policy at the “Families Belong Together” march last week. Although the policy has since been redacted, the government has shown little transparency on how they plan to reunite families. Could DNA testing be a solution?

DNA testing companies, MyHeritage and 23andMe, seem to think so. They have offered thousands of testing kits to help reunite migrant children to their families. Scientifically, these tests are very reliable, and can detect direct relations by 99.9% accuracy. However, the science is the least complicated aspect of this situation.

Consent and privacy are several of the most troubling aspects of the use of these tests. Due to medical privacy rules, children would need a designated legal guardian or representative to have their DNA tested, which is clearly a problem. In addition, adults likely cannot give informed consent, especially since they are in distressing conditions and many do not speak English. Migrants may feel pressured to have the sequencing done if they believe it is the only way to be reunited with their children. DNA sequencing reveals private information about health and paternity, and sequencing data stored in databases has been used to genetically track criminals. It is difficult to imagine that detainees would be given enough information about DNA sequencing and its’ implications to make an informed decision.

Despite these concerns, according to an unnamed federal official, DNA testing has already begun. Jennifer K. Falcon, communications director for RAICES, a nonprofit in Texas that offers free and low-cost legal services to immigrants and refugees, is extremely against DNA testing in this context. In addition to her concerns regarding consent, she argues that the government will have access to extremely personal data that could be used for future surveillance. Although 23andMe and MyHeritage have assured that the genetic data will only be used for reunification, it is unclear what will happen to the DNA samples and data afterwards.

Beyond the ethical and logistical hurdles in this case, DNA sequencing is not a quick fix. 23andMe state on their website that sample processing takes 6-8 weeks. It would also be a logistical nightmare to obtain and match DNA samples from all the detainees currently in custody, especially when matching results from two different genetic testing companies. Critics point out that registering the identity and locations of migrant parents and children would have circumvented the need for such invasive testing. Although genetic tests are cheaper and more accessible than ever, they require unique consideration to address issues of privacy and consent.

(Maya Wei-Haas, National Geographic)

Endangered species

Rhino Embryos Made in Lab to Save Nearly Extinct Subspecies

Thousands of northern white rhinos once inhabited the grasslands of east and central Africa, but habitat loss and poaching led to the population’s swift demise. All hope for the survival of the rhino subspecies seemed lost when the its’ last remaining male, Sudan, died earlier this year.  There are now only two surviving individuals of the subspecies, a mother-daughter pair named Najin and Fatu, both of whom are infertile. Remarkably, a new breakthrough in reproductive technology has reignited the possibility of saving this subspecies.

In a recent study published in Nature Communications, Dr. Thomas Hildebrant, a wildlife reproductive biologist, and his team show for the first time that rhino embryos can be created using in vitro fertilization (IVF). Although there are no remaining living males of the subspecies, there are four samples of frozen sperm that could potentially be used for reproduction. The research group created four hybrid embryos by combining frozen northern white rhino sperm and eggs from southern white rhinos. The scientists plan on implanting these hybrid embryos into surrogates, to see if they survive to birth. If that is successful, the scientists aim to extract eggs from the remaining female northern white rhinos and create pure-blood northern white rhinos in the lab.

Since there is a limited supply of northern white rhino gametes (only four sperm samples and two egg samples), Hildebrant and his team are also pursuing a technology called induced pluripotent stem cells (iPSC). iPSC are a type of stem cell that can be created from adult cells, such as skin or blood. These iPSC can then be reprogrammed into various cell types. iPSC have already been created from northern white rhinos, and scientists are now figuring out how to convert them to sperm and eggs. Since the San Diego zoo has skin cells from 12 northern white rhinos, the future conversion of these cells into gametes could provide more genetic diversity to any future population.

While many conservation scientists applaud the use of technology to save the subspecies, many wonder whether the resources should rather be spent protecting habitats for remaining rhinos on-the-ground. In a study in Nature Ecology and Evolution, scientists show that de-extinction efforts can lead to a net biodiversity loss, since resources could be spent on endangered species. As Dr. Bennett, a conservation scientist at Carleton University, puts it “if the person is couching de-extinction in terms of conservation, then she or he needs to have a very sober look at what one could do with those millions of dollars with living species — there’s already plenty to do.”

(Steph Yin, New York Times)

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July 6, 2018 at 3:11 pm

FDA stem cell therapy crackdown: a stem-free clinic

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By: Belinda Hauser, Ph.D.


source: pixabay

The building blocks of life are stem cells, they don’t kill or cure anything, but they promote regeneration. Stem cells are classically defined as an undifferentiated cell capable of giving rise to more stem cells or differentiating into any cell type. Stem cells have given scientists insight into understanding how cells function and dysfunction in development. Moreover, research in stem cell development has lead to promising treatment possibilities; it is believed that stem cells have the potential to repair or replace damage caused by age, injury or disease. However, stem cell therapies have been controversial, arising from the practice of isolating and culturing stem cell derived from human embryos, and later, introducing pluripotent stem cells from previously differentiated cell types. This controversy is entrenched in both political and ethical debates, broadly affecting the regulation of cord blood harvesting, human cloning and clinical trials.

Today, common stem cell therapy uses include blood transplants or bone marrow transplants. The Food and Drug Administration (FDA) has only approved hematopoietic progenitor cells, derived from umbilical cord blood, for use in the United States. Harvesting of cord blood is considered safe for the mother and baby since the blood is collected after birth. Stem cells collected from the blood of the cut cord are used to treat a variety of diseases including blood cancers such as leukemia, and lymphomas, and blood diseases of the immune system. Given the scarcity of approved options, patients desperately seeking therapy may turn to treatments that are illegal and potentially harmful. The FDA has gone to great lengths to evaluate the potential risk associated with new and current products through both animal and human studies in order to ensure safety in the use of biological products. Thereby, to determine the effectiveness and safety of new investigative products, well-controlled human studies must be designed and executed. This attention is applied to all clinical trials and is well documented. For example, the federal government requires all clinical trials to be cited and it is standard protocol for the National Institutes of Health (NIH) to list all clinical trials being conducted via This promotes awareness and gives consumers an opportunity to be well informed of all trials being conducted.

Preceding the FDA’s goal to develop and license stem cell therapies for patients and prevent consumer exploitation is their concern for consumer safety and education. In March 2017, the FDA provided materials to clarify the benefits and risks of stem cell therapies. They warned that when injected, unproven stem cell treatments present the risk of mobility of implanted cells, i.e. metastasis, risk of excessive proliferation, i.e. tumor growth, contamination, stem cell failure, or reaction of the injection site. Therefore, new investigative products must go through a rigorous protocol to determine their effectiveness and safety in well-controlled human studies.

In August 2017, the FDA cracked down on unscrupulous stem cell clinics, announcing increased enforcement of regulations and oversight of stem cells clinics across the country. For example, the FDA seized five vials of (live) smallpox virus vaccine from the California stem cell treatment centers in Rancho Mirage and Beverly Hills, California.  A Florida clinic, now called U.S. Stem Cell Clinic of Sunrise, Florida, caught the attention of the FDA after stem cell treatments it delivered to women with macular degeneration, an eye disease, caused permanent damage. Staff member used stem cells from fat isolated from each patient’s stomach and then injected cells into their eyes. A common practice of clinical trials is to pay human subject-volunteers to participate in studies. However, to receive this unproven treatment patients were required to pay $5,000 to receive the stem cell injections. Permitting patients to pay for participation is a topic of ethical debate for even the most scrupulously designed trials. The FDA issued a notice warning U.S. Stem Cell Clinic for marketing products without FDA approval and condemning their exploitation of consumers. An inspection performed  by FDA investigators found evidence of significant deviations from good manufacturing practices in manufacturing of at least 256 lots of stem cell products produced by the clinic. In an attempt to impede the investigation, the U.S. Stem Cell Clinic attempted to refused access of the FDA investigators to the employees of the clinic.  Ultimately, the clinic was cited for failure to establish appropriate written procedures to prevent contamination, risking infection of human subjects. It is required that U.S. Stem Cell Clinic comply and correct the failures stated in the warning letter. If the clinic fails to address the outlined issues, actions will be taken by the FDA, these include seizure, injunction and or prosecution.  Moreover, U.S. Stem Cell Clinic  administered the product both intravenously and directly into the spinal cord of patients hoping to treat a number of serious diseases (Parkinson’s disease, amyotrophic lateral sclerosis (ALS) heart disease, pulmonary fibrosis, and chronic obstructive pulmonary disease (COPD), all without FDA review or approval. In fact the FDA has not approved any biological products manufactured by U.S. Stem Cell Clinic for any use.

Overall, the challenge of regulation and compliance continues to loom over all stem cell clinics in the U.S.; however, the FDA is dedicated to enforcing continuous regulation, while educating and protecting U.S. consumers. The building blocks of life are stem cells, manipulated properly, they have the ability to treat disease without posing unacceptable risk. Safely figuring out how will take time.

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January 17, 2018 at 11:43 am

Science Policy Around the Web – October 10, 2017

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By: Kseniya Golovnina, PhD.


source: pixabay

Gene Therapy

In a First, Gene Therapy Halts a Fatal Brain Disease

 The first historic gene therapy approval by the U.S. Food and Drug Administration in August 2017 opened a new era for the treatment of serious and life-threatening diseases. One month later Bluebird Bio announced the successful start of Lenti-D therapy in the clinical trial and gave a flutter of hope to cure cerebral adrenoleukodystrophy (CALD) also known as Lorenzo’s Oil.

Adrenoleukodystrophy (ALD), a rare disorder that affects one in 21,000 male births worldwide, is caused by mutations in the ABCD1 gene that lead to the subsequent accumulation of very long chain fatty acids in tissues, including the myelin of the central nervous system. The most severe form of ALD, known as cerebral ALD (CALD), involves the progressive destruction of the protective sheath of the nerve cells in the brain that are responsible for thinking and muscle control. This leads to deafness, blindness, seizures, loss of muscle control and dementia, resulting in permanent disability or death. Symptoms of CALD progress rapidly if untreated and the only current treatment is stem cell transplantation.

Gene therapy is a technique for correcting defective genes responsible for disease development.  It utilizes viruses to deliver unmutated copies of the genes, such as the ABCD1 gene, to the cells of the patient’s body. First, blood from the patient is collected by apheresis, depleting immune response T cells and enriching progenitors of all blood cells (hematopoietic stem cells, HTS). The HTS cells are then infected with a virus carrying a functional copy of the gene, before returning the cells to the body.

Bluebird Bio is now pursuing Lenti-D therapy which uses lentiviruses to deliver a functional copy of the ABCD1 gene to patients with ALD. Results published in the New England Journal of Medicine, reported that 15 out of 17 patients (88%) were free from major functional disabilities two years after the hematopoietic stem-cell gene therapy. These results demonstrate the therapy’s efficacy over the 76% benchmark established by radiotherapy-free survival at 24 months. Bluebird’s Chief Medical Officer David Davidson expressed excitement about the patients’ progress. He announced that the first four patients treated in the expansion cohort are also doing well, as measured by their amount of the functional ABCD1 gene.

(Gina Kolata, The New York Times)

Drug pricing

FDA acts to encourage generic competition for complex drugs

What kind of feelings do you have when pharmaceutical companies announce their prices for upcoming exciting gene therapies and other innovative, life-changing bio pharmaceuticals? Positive news about development and success of first-of-their-kind drugs can be undermined by anxieties that patients will not be able to afford them. High drug prices can prevent accessibility of new therapies vital for many patients, and market analysts predict rapid inflation in healthcare spending over the next few years due to the aging US population.

The FDA is conscious of the stress expensive drugs put on both patients and the entire healthcare system. Under the leadership of current Commissioner, Dr. Scott Gottlieb, one of key goals of the the FDA is to bring more competition from generics to help drive prices down. On October 2, 2017 the agency prepared draft guidance specifically aimed at copycats of complex therapies and therefore trying to clear the way for generic drug makers to the market.

Gottlieb wrote in his blog post that the agency is looking for more efficient regulatory pathways with robust reviews and communication with pharmaceutical companies for abbreviated new drugs applications (ANDAs). He highlighted that “early and better meetings between FDA and sponsors can improve development timelines. The agency acknowledged that the complexity of the approval process may be discouraging to generic drug makers. The new guidance aims to clarify these complexities by outlining how genetic makers can prove “sameness” by showing that there is no difference in response to generic drug compared with the original. This is the second update for ANDAs process and Dr. Gottlieb assured that FDA will continue to progress in this reformation.

(Linda A. Johnson, The Associated Press)


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October 10, 2017 at 10:11 pm

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Science Policy Around the Web – July 21, 2017

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By: Rachel F Smallwood, PhD

Source: pixabay


Engineered Cell Therapy for Cancer Gets Thumbs Up from FDA Advisers

A panel of advisers has recommended that the FDA approve chimeric antigen receptor T-cell (CAR-T) therapy for treatment of acute B-cell lymphoblastomic leukemia. The committee unanimously agreed that the risk to benefit ratio was favorable enough to proceed with approval of the drug (tisagenlecleucel), manufactured by Novartis. CAR-T therapy utilizes a patient’s own immune cells to find and attack cancer cells. In a recent trial in humans, 82.5% of patients went into remission following treatment with the drug; there have also been promising results from its use in glioblastoma treatment. The treatment would specifically be for pediatric and young adult patients who did not respond well to initial treatments or who relapsed from being in remission.

Despite have strong positive effects, there are potential risks posed by CAR-T therapy. In the study mentioned above, almost half of the patients experienced an inflammatory reaction called cytokine release syndrome. Although all of those cases were treatable, the condition can be life-threatening. Novartis also reported neurological problems. Other CAR-T trials have had several deaths due to brain swelling, but those were in adult populations and were some differences in the therapies.

The FDA often does take the recommendations of its advisers, but there is much to consider in this decision. It would essentially be approving a living drug that is individualized to each patient; the patients’ own blood cells are sent to a manufacturing center, where they are genetically engineered to target leukemia cells. The cell population is then allowed to proliferate, and the entire process takes around twenty-two days. This process presents a quality assurance and control problem to the FDA. However, the target population typically has a poor prognosis and very few options, so the panel considers the potential for increased survival and quality of life to be worth the risks. (Heidi Ledford, Nature News)

Stem-Cell Therapy

Unapproved Stem-Cell Treatments Touted on Federal Database Clinicaltrials.Gov is an online database, curated by the National Library of Medicine and the National Institutes of Health, that logs clinical studies occurring around the country and allows them to be searched by patients, family members, healthcare providers, and researchers. The information on the site is provided by the researchers or sponsors of the individual studies themselves. It allows patients and healthy people to become aware of opportunities to participate in medical research. These studies involve a wide range of treatments, including drugs, devices, behavioral therapies, and procedures.

A recent study found that the database is being abused by clinics advertising for stem cell trials. These trials target individuals looking for treatment for a variety of conditions, and all of them charge for participation. There are very few FDA-approved stem cell therapies, and most clinics that utilize stem cell therapies assert that they do not need FDA approval since they are practicing medicine and do not substantially alter the stem cells (although that is disputed).  Since the researchers themselves indicate in the database whether they need FDA approval, there is little oversight to ensure these studies are correctly representing the risks and benefits of their treatment.

Although a disclaimer was added this spring that informs visitors that the presence of a trial in the database does not indicate government endorsement of it, many people do not realize that they could potentially be participating in a for-profit procedure that does not have the proper oversight to ensure patient safety. In one such case, three women were blinded who paid to receive stem cell therapy for macular degeneration. Most legitimate research studies will not require payment for participation, although travel and lodging costs associated with participation may be incurred.

While many patients may receive treatment at one of these clinics without an adverse event or even with a positive result, critics of these types of clinics are calling for regulation of entries into the system. They assert that a federal resource for medical research should not be used to advertise for for-profit clinics that are utilizing therapies that have not been studied or reviewed for safety and efficacy. (Laurie McGinley, Washington Post)

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July 21, 2017 at 10:08 am

Science Policy Around the Web – June 13, 2017

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By: Nivedita Sengupta, PhD

By Mikael Häggström, used with permission. [Public domain], via Wikimedia Commons

Stem Cell Therapy

Texas on Track to Become First State to Explicitly Back Stem Cell Therapies

On 30th May, Texas passed a bill  authorizing unapproved stem cell therapies, making Texas the first state to openly recognize experimental treatments. The bill will make the use of unapproved stem cell therapies legal for patients and is currently awaiting the approval of Governor Greg Abbott, who already supports the measure. Experimental stem cell therapies for terminal and chronic conditions have struggled for years to gain support without much success. Until now, no state has provided legal validation for these kind of therapies and the current stem cell procedures are mostly done under strict regulations.

Amendments were added to the bill, which require that the treatments be delivered by doctors with the approval of an institutional review board, which deals with human research. It will also add another amendment that will allow patients to have authority to sue in case the treatments go wrong. Many scientists and advocates opposed the measure stating that unapproved stem cell therapies can be harmful rather than beneficial. They state that though the amendments add protection to the patients, there are a few aspects of the bill that make them uncomfortable. Two other bills focused on patient access to experimental therapies, also known as “right-to-try” policies, failed to pass in the Texas Senate. (Andrew Joseph, STATNews)

Research Funding

NIH Scraps Plans for Cap on Research Grants

US National Institutes of Health (NIH) decided to drop the controversial proposal of capping the number of grants that an investigator can have at a time. The initial capping attempt was suggested to gather funds for younger researchers by NIH in May. The proposal was based on studies that suggested that a lab’s productivity decreases once it holds too many grants. Younger scientists often face more difficulties in obtaining NIH RO1 grants compared to their older more experienced colleagues. As a result, many researchers applauded the NIH’s effort to provide more funding for younger scientists. Yet the capping proposal received major adverse response from the scientific community stating that the NIH’s interpretation of the productivity study data does not apply to all labs, especially to the collaborative lab groups with four or five R01s that are more productive than labs with only one. Researchers also complained that the proposed point-based scoring system will also make collaborations difficult thus hampering productivity in the long run.

NIH director Dr. Francis Collins stated that the original idea was still a work in progress and NIH is going to put a hold on it. Instead of the cap, on 8th June, NIH announced the creation of the special fund, the Next Generation Researchers Initiative (NGRI), starting with US$210 for funding young researchers. The initiative will focus on investigators with less than 10 years of experience as NIH- funded principal investigators, and on high score grant proposals that were rejected because of lack of money. The initiative will grow up to $1.1 billion over the next five years. According to NIH principal deputy director Larry Tabak, NIH will immediately start creating an inventory of investigators who meet these criteria and expects that this approach will allow more than 2,000 additional R01 grants to be funded to younger scientists compared to the cap-based plan, which would have supported only 1600 awards. Nonetheless, the current proposal is still going to generate controversy as it will affect the older researchers because of NIH’s diversion of funding. (Sara Reardon, Nature News)

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June 13, 2017 at 7:08 pm

Science Policy Around the Web – May 12, 2017

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By: Liu-Ya Tang, PhD

Source: pixabay


Basic Scholarship in Biosafety Is Critically Needed

While a significant amount of money funds primary research in life sciences, the portion allotted in biosafety assessment is almost neglected, which can be detrimental to biomedical research. In a recent paper in mSphere, an open-access journal published by the American Society for Microbiology (ASM), the authors reported the status of practicing biosafety in U.S. labs and pointed out the urgent need for funding in this field.

They identified human errors as the dominant component of laboratory biosafety risk, but there was limited data to support a quantitative analysis of human failure rates. Publicly available risk assessments were only focused on mechanical failure rates. They also found that historical biosafety incident data is not adequate, and incidents reporting systems are not sufficiently standardized. So the same mistakes could likely happen in multiple labs. In contrast, other industries, such as the power and transportation industries, have been investing heavily in maintaining safety records and have benefited from doing so. The authors cite an example from the airline industry to address the importance of incident reporting system. After a flight crash outside Washington’s Dulles airport in 1974, the Federal Aviation Administration (FAA) created a no-fault system of reporting aviation incidents and mistakes. FAA has maintained this system ever since, which has helped reduce accident rates by two-thirds compared to that in the early 1970s.

Even though funding for biosafety assessment is much less than that in other industries, the consequences of a potential infectious disease outbreak can be much bigger than any other accidents. Therefore, such funding is urgently needed for three aspects: “(i) development of a national incident reporting system, (ii) primary research programs focused on human reliability assessments, equipment failures, and decontamination efficiencies, and (iii) sharing of best practices.” Investing in biosafety and biorisk management will help enhance laboratory safety practices and improve work performance of our research enterprise in the long run. (Ryan Ritterson and Rocco Casagrande, mSphere)

Human Stem-Cell Research

Attitudes Towards Stem-Cell Research in Europe, Canada and the United States

Human embryonic stem-cell research has caused many political and public debates over moral concerns while providing benefits to human health. In science policy making, public opinion has great impact. To investigate factors that affect international public opinion towards stem-cell research, Allum N. and colleagues analyzed representative sample surveys in Europe and North America, fielded in 2005, when it was a highly contested issue.

The authors found that public attitudes towards stem-cell research has been affected by government decisions, especially in the U.S. During the Bush administration, federal funding only allowed the use of a small number of existing cell lines in stem-cell research. These limitations were removed by an Executive Order from President Barack Obama that expanded NIH support for human stem-cell research. In response to government guidance, public support for stem-cell research in the U.S. rose from 40 percent in 2002 to around 65 percent in 2010. About 65 percent of Europeans and Canadians supported human stem-cell research on the condition that it is tightly regulated. The other influential factor is religion. The authors showed that in all the regions examined, approval for stem-cell research decreased with increasing religious commitment. This pattern was more pronounced in the U.S. and Canada than in Europe. But interestingly, half of even the most religious public supported stem-cell research, which indicated that perhaps the benefits of stem-cell research are being more appreciated. Overall, the majority of people in the surveyed areas hold positive attitudes towards human stem-cell research. (Nick Allum et al, PLOS ONE)

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May 12, 2017 at 11:07 am