Category Archives: Stem Cell Clinics

Europe’s guardian of stem cells and hopes, real and unrealistic – Yahoo News

Poland has emerged as Europe's leader in stem cell storage, a billion-dollar global industry that is a key part of a therapy that can treat leukaemias but raises excessive hopes.

Submerged in liquid nitrogen vapour at a temperature of minus 175 degrees Celsius, hundreds of thousands of stem cells from all over Europe bide their time in large steel barrels on the outskirts of Warsaw.

Present in blood drawn from the umbilical cord of a newborn baby, stem cells can help cure serious blood-related illnesses like leukaemias and lymphomas, as well as genetic conditions and immune system deficits.

Polish umbilical cord blood bank PBKM/FamiCord became the industry's leader in Europe after Swiss firm Cryo-Save went bankrupt early last year.

It is also the fifth largest in the world, according to its management, after two companies in the United States, a Chinese firm and one based in Singapore.

Since the first cord blood transplant was performed in France in 1988, the sector has significantly progressed, fuelling hopes.

- Health insurance -

Mum-of-two Teresa Przeborowska has firsthand experience.

At five years old, her son Michal was diagnosed with lymphoblastic leukaemia and needed a bone marrow transplant, the entrepreneur from northern Poland said.

The most compatible donor was his younger sister, Magdalena.

When she was born, her parents had a bag of her cord blood stored at PBKM.

More than three years later, doctors injected his sister's stem cells into Michal's bloodstream.

It was not quite enough for Michal's needs but nicely supplemented harvested bone marrow.

As a result, Michal, who is nine, "is now flourishing, both intellectually and physically," his mum told AFP.

A cord blood transplant has become an alternative to a bone marrow transplant when there is no donor available, with a lower risk of complications.

Stem cells taken from umbilical cord blood are like those taken from bone marrow, capable of producing all blood cells: red cells, platelets and immune system cells.

Story continues

When used, stem cells are first concentrated, then injected into the patient. Once transfused, they produce new cells of every kind.

At the PBKM laboratory, "each container holds up to 10,000 blood bags... Safe and secure, they wait to be used in the future," its head, Krzysztof Machaj, said.

The bank holds around 440,000 samples, not including those from Cryo-Save, he said.

If the need arises, the "blood will be ready to use without the whole process of looking for a compatible donor and running blood tests," the biologist told AFP.

For families who have paid an initial nearly 600 euros ($675) and then an annual 120 euros to have the blood taken from their newborns' umbilical cords preserved for around 20 years, it is a kind of health insurance promising faster and more effective treatment if illness strikes.

But researchers also warn against unrealistic expectations.

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Haematologist Wieslaw Jedrzejczak, a bone marrow pioneer in Poland, describes promoters of the treatment as "sellers of hope", who "make promises that are either impossible to realise in the near future or downright impossible to realise at all for biological reasons."

He compares them to makers of beauty products who "swear their cream will rejuvenate the client by 20 years."

Various research is being done on the possibility of using the stem cells to treat other diseases, notably nervous disorders. But the EuroStemCell scientist network warns that the research is not yet conclusive.

"There is a list of almost 80 diseases for which stem cells could prove beneficial," US haematologist Roger Mrowiec, who heads the clinical laboratory of the cord blood programme Vitalant in New Jersey, told AFP.

"But given the present state of medicine, they are effective only for around a dozen of them, like leukaemia or cerebral palsy," he said.

"It's not true, as it's written sometimes, that we can already use them to fight Parkinson's disease or Alzheimer's disease or diabetes."

EuroStemCell also cautions against private blood banks that "advertise services to parents suggesting they should pay to freeze their child's cord blood... in case it's needed later in life."

"Studies show it is highly unlikely that the cord blood will ever be used for their child," the network said.

It also pointed out that there could be a risk of the child's cells not being useable anyway without reintroducing the same illness.

Some countries, such as Belgium and France, are cautious and ban the storage of cord blood for private purposes. Most EU countries however permit it while imposing strict controls.

- Rapid growth -

In the early 2000s, Swiss company Cryo-Save enjoyed rapid growth.

Greeks, Hungarians, Italians, Spaniards and Swiss stored blood from their newborns with the company for 20 years on payment of 2,500 euros upfront.

When the firm was forced to close in early 2019, clients were left wondering where their stem cells would end up.

Under a kind of back-up agreement, the samples of some 250,000 European families were transferred for storage at PBKM.

The Polish firm, founded in 2002 with two million zlotys (around 450,000 euros, $525,000), has also grown quickly.

Present under the FamiCord brand in several countries, PBKM has some 35 percent of the European market, excluding Cryo-Save assets.

Over the last 15 months, outside investors have contributed 63 million euros to the firm, PBKM's chief executive Jakub Baran told AFP.

But the company has not escaped controversy: the Polityka weekly recently published a critical investigative report on several private clinics that offer what was described as expensive treatment involving stem cells held by PBKM.

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Europe's guardian of stem cells and hopes, real and unrealistic - Yahoo News

Why stem cells could be the medical innovation of the century – World Economic Forum

Right now, your bodys stem cells are working hard replacing your skin every two weeks, creating new red and white blood cells and completing thousands of other tasks essential to life. They are your own personalized fountain of youth.

Scientists generally agree that a stem cell should be able to do both of the following:

One theory of ageing suggests that between the ages of 30 and 50, our stem cells reach a turning point and start to decline in number and function. This results in the typical features associated with ageing.

There does not seem to be a single discoverer of stem cells. Accounts date back to the 1800s and even further, but the first successful medical procedure was a bone marrow transfusion in 1939. Advances in immunology led to donor matching, initially via siblings and close relatives. Unrelated donor matching flourished in the 1970s, alongside donor registries.

In the 1980s, scientists identified embryonic stem cells in mice, leading to the 1997 cloning of Dolly the Sheep. This created immense interest for human and medical applications and a backlash in the US as federal R&D funding was essentially halted in 2001.

In 2012, a Nobel Prize was awarded for the earlier discovery of induced pluripotent stem cells (iPS). Essentially, they return potency and self-renewal properties to mature non-stem cells, essentially making them act like stem cells again.

In the decade between 2010 and 2019, the first wave of stem cell start-ups emerged, alongside R&D programmes at many large pharmaceutical companies, leading to innovation and the first human clinical trials for iPS and other related therapies.

According to Q3 2019 data from the Alliance for Regenerative Medicine, there are 959 regenerative medicine companies worldwide sponsoring 1,052 active clinical trials; 525 of these companies are in North America, 233 in Europe and Israel, and 166 in Asia. In aggregate, $7.4 billion has been invested in regenerative medicine companies in 2019; $5.6 billion of which has been dedicated to gene and gene-modified cell therapy, $3.3 billion in cell therapy, and $114 million in tissue engineering.

Overview of the cancer stem cells market

Perhaps most excitingly, curative therapies are hitting the market and the results are astonishing: 60% of Acute Lymphoblastic Leukemia patients taking Novartis Kymirah showed a complete response (no traces of cancer) and were declared in full remission. Meanwhile, 75% of patients with Transfusion-Dependent -Thalassaemia treated with bluebird bios Zynteglo achieved independence from transfusions. Perhaps most astonishingly, 93% of spinal muscular atrophy patients treated with Novartis Zolgensma were alive without permanent ventilation 24 months after treatment. We should expect more medical breakthroughs in the coming years.

New science, new start-ups: several companies in the sector have gone public or been acquired. These exits led to the recycling of talent and capital into new companies. Because the science and commercial systems have also advanced, the companies in the next wave are pursuing bigger challenges, driving innovation, with even greater resources.

Patients are eager: the current market for stem cell therapies is growing at 36% per year, though it will rapidly expand when a breakthrough occurs toward the treatment of a non-communicable disease (such as cancer, diabetes, heart disease) or a lifestyle factor (for example, growing hair in the correct places, expanding cognitive abilities or increasing healthy lifespan).

New R&D models: funding is flowing into the sector from large companies, VC funds, and institutions such as the California Institute for Regenerative Medicine (CIRM) and New York State Stem Cell Science programme (NYSTEM). Some of the leading university R&D platforms include the Center for the Commercialization of Regenerative Medicine in Toronto, the Stanford Institute for Stem Cell Biology and Regenerative Medicine, the Oxford Stem Cell Institute, and most notably, the Harvard Stem Cell Institute (HSCI).

Founded in 2004, HSCI has established a phenomenal track record. It provided the first $200,000 in funding to Derrick Rossis lab, which inspired the largest biotech IPO to date. HSCI scientists were also co-founders or principals in the three most prominent gene-editing companies (CRISPR Tx, Intellia and Editas), the combined $1.55-billion True North/iPierian acquisitions and the recent $950-million acquisition of Semma Tx, Frequency Tx, Fate Tx, Epizyme Inc., and Magenta Tx.

For the casual investor, Evercore ISI is building a Regenerative Medicine Index, which may be the simplest way to build a portfolio. For institutions and those with deeper pockets, regenerative medicine funds are forming, including the Boston-centric Hexagon Regenerative Medicine Fund, which aims to create companies out of the Harvard Stem Cell Institute.

Caveat emptor. Though patients needs are immediate, those seeking treatments should think very carefully about the risks. There are many dubious clinics touting expensive stem cell treatments and some patients have experienced horrifying complications. Dr. Paul Knoepfler of UC-Davis has written a practical and scientifically accurate guide, a strongly recommended read if you or a family member are considering treatment or a clinical trial.

The leading causes of death in 1900 were mostly infectious/communicable diseases. While the prevalence of most causes has diminished, the largest increases include heart disease (+40%) and cancer (+300%). Granted, this is partly due to doubling life expectancy and a lack of death from other causes. However, given time and resources, scientists and physicians may cure these challenging diseases.

Total disease burden by disease or injury

Today, six of the seven leading causes of death are non-communicable diseases (heart disease, stroke, lung diseases, cancer, Alzheimers disease and diabetes). Based on the early promise mentioned above, regenerative medicine may be our best hope to solve the great non-communicable diseases of our time, and perhaps the single most transformative medical innovation in a century.

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The views expressed in this article are those of the author alone and not the World Economic Forum.

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Why stem cells could be the medical innovation of the century - World Economic Forum

Orthobiology Market Innovative Technologies, Current And Future Trends –

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Orthobiology Market Innovative Technologies, Current And Future Trends -

Where Are They Now? Top 3 Biotech Startups From NextGen Bio Class of 2018 – BioSpace

Every year, BioSpace analyzes the biotech industry, looking for the hot new biotech startups to watch. We then produce the NextGen Bio Class of, twenty companies ranked based on several categories, including Finance, Collaborations, Pipeline, and Innovation. The companies were typically launched no more than 18 months before the list was created.

We thought it would be insightful to look back at our previous lists to see where some of those companies are today. Heres a look at the top three companies from the Top 20 Life Science Startups to Watch in 2018.

#1. BlueRock Therapeutics. Founded in 2016, BlueRock was #1 on our list of companies to watch in 2018. With facilities in Ontario, Canada; Cambridge, Massachusetts; and New York, New York, BlueRock launched in December 2016 with a $225 million Series A financing led by Bayer AG and Versant Ventures. The company focuses on cell therapies to regenerate heart muscle in patients who have had a heart attack or chronic heart failure, as well as therapies for patients with Parkinsons disease.

In October 2017, BlueRock and Seattle-based Universal Cells entered into a collaboration and license deal to create induced pluripotent stem (iPS) cell lines that can be used in the manufacture of allogeneic cellular therapies. Shortly afterwards, the company established its corporate headquarters in Cambridge, and in April 2018, established a research-and-development hub in New York City, as well as formalizing a sponsored research collaboration with the Center for Stem Cell Biology at Memorial Sloan Kettering (MSK) Cancer Center. The collaboration focuses on translating Ketterings expertise in creating multiple types of authentic neural cells from stem cells to address diseases of the central and peripheral nervous system. BlueRock also received $1 million from the State of New York and Empire State Development under its economic development initiatives program.

In April 2019, BlueRock partnered with Editas Medicine (which was on BioSpaces NextGen Bio Class of 2015 list) to combine their genome editing and cell therapy technologies to focus on novel engineered cell medicines. Part of the deal was to collaborate on creating novel, allogeneic pluripotent cell lines using a combination of Editas CRISPR genome editing technology and BlueRocks iPSC platform.

And finally, in August 2019, Bayer AG acquired BlueRock for the remaining stake in the company for about $240 million in cash and an additional $360 million in pre-defined development milestones.

#2. Prelude Fertility. Prelude Fertility is a bit of an outlier from the typical BioSpace NextGen company, because it isnt quite a biopharma company. It is a life sciences company whose business model is aimed at in vitro fertilization and egg freezing. It was founded with a $200 million investment by entrepreneur Martin Varsavsky. The investment was in the largest in vitro fertilization clinic in the Southeast, Reproductive Biology Associates of Atlanta, and its affiliate, My Egg Bank, the largest frozen donor egg bank in the U.S.

Since then it has expanded in various parts of the country, including adding San Francisco-based Pacific Fertility Center (PFC) to its network in September 25, 2017; partnering with Houston Fertility Institute and acquiring Vivere Health; partnering with the Advanced Fertility Center of Chicago; and in October 2018, partnered with NYU Langone Health.

In March 2019, Prelude merged with Inception Fertility to establish the Prelude Network as the fastest-growing network of fertility clinics and largest provider of comprehensive fertility services in the U.S. Inception is acting as the parent company, with the Prelude Network, both having board representatives from the previous organizations.

#3. Relay Therapeutics. Ranking #3 on our list for 2018, Relay Therapeutics launched in September 2016 with a $57 million Series A financing led by Third Rock Ventures with participation form D.E. Shaw Research. On December 14, 2017, it closed on a Series B round worth $63 million, led by BVF Partners, with new investors GV (formerly Google Ventures), Casdin Capital, EcoR1 Capital and Section 32.

The company focuses on the relationship between protein motion and function. It merges computational power with structural biology, biophysics, chemistry and biology. In December 2018, the company completed a $400 million Series C financing. It was led by the SoftBank Vision fund and included additional new investors, Foresite Capital, Perceptive Advisors and Tavistock Group. Existing investors also participated.

The company announced at the time it planned to use the funds to accelerate the implementation of its long-term strategy, expanding its discovery efforts, advancing existing programs into the clinic and improving its platform.

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Where Are They Now? Top 3 Biotech Startups From NextGen Bio Class of 2018 - BioSpace

Drugs, Biologics, and Regenerative Medicine in 2019: A Successful Year Ends with Promise of a More Challenging 2020 – JD Supra

Following up on our first post in this year-end series that discussed medical device regulatory activities at the Food and Drug Administration (FDA), the Mintz FDA teams second year-end post will provide an overview of 2019 with a focus on the drug, biologic, and regenerative medicine programs at the agency. In many ways, the past year could be called a business as usual year for the FDAs drugs and biologics centers in that they continued to make progress on all of large-scale programs and priorities initiated by former-Commissioner Scott Gottlieb, who left the agency in April. FDA has been under the leadership of an Acting Commissioner since that time, although Texas radiation oncologist Dr. Stephen Hahn will be taking the reins soon following his confirmation by the full Senate in a 72-18 vote on December 12, 2019. (The Senate HELP Committee advanced the nominee on December 3, 2019; see our blog post just prior to that committee vote here.)

At the same time, however, the final months of 2019 have exposed several challenges for various FDA programs that operate under the extensive drug and biologic authorities contained in the Food Drug & Cosmetic Act (FD&C Act) and the Public Health Service Act (PHS Act), respectively. The agency will be forced to grapple with many of these issues directly and deliberately in 2020as a result of deadlines of the agencys own making as well as external pressures coming from other parts of D.C. and from the rapidly changing nature of the U.S. health care system.

Business as Usual When Getting New Drugs, Generic Drugs, and Biosimilars to Market and Promoting Competition under Various Action Plans But Challenges Are Emerging Related to Accelerated Approval and Breakthrough Products

According to data presented by the Office of New Drugs in early December (see ONDs slides here), FDA had another extremely productive year when it comes to its approval of new molecular entities that address a unique blend of therapeutic areas. The agency approved 45 new molecular entities in FY 2019 (October1, 2018 to September30, 2019), of which 71%, or 32 products, received priority review status and 23 were designated as orphan drugs intended to treat rare diseases. As OND emphasized in the presentation, several of those new product approvals are notable for their uniqueness and therefore, in the agencys view, 2019 reflects not only quantity but [also] quality. The OND presentation also highlights a significant amount of other information on new molecular entity approvals and may be of interest to those readers who want to take a deeper dive into the data.

In addition to advancing important new drugs and biologics to market, former Commissioner Gottlieb is well-known for having spearheaded to development of a Drug Competition Action Plan (DCAP) and a Biosimilars Action Plan (BAP) during his nearly two-year tenure as head of the agency. Some of our prior coverage of the DCAP and BAP is available here. In general terms, the DCAP encourages market competition for generic drugs and helps to bring greater efficiency and transparency to the generic drug review process; the BAP aims to achieve similar goals for biosimilar products as the agency continues its implementation of the 2010 Biologics Price Competition and Innovation Act (BPCIA), including the critical drug-to-biologic transition that will occur by operation of law in March of 2020.

FDA continued to make progress on its various goals under these two initiatives during 2019. Some examples of this progress can be captured with these two data points:

With all of these wins, however, FDA still faces its fair share of challenges related to its expedited drug and biologic programs, especially as it appears to have accelerated its review of products intended for diseases with an unmet need to breakneck speed. Indeed, the OND presentation from early December also added that for FY 2020 and as of November 21, 2019, the agency had already approved 13 such drugs, suggesting that next year could be a record-breaking one in terms of innovative drug product approvals. A recent Bloomberg Law article (available here) used the phrase breakneck speed to describe the agencys actions in this space based on several recent FDA approvals of new molecular entities that have come months in advance of their assigned target dates. The Bloomberg Law article highlights that in response to FDAs speediness in reaching approval decisions on new drugs and biologics for diseases with unmet needs, patient advocates and, increasingly, insurers that have to pay for those treatments are starting to raise concerns that these products lack sufficient safety or effectiveness data.

Relatedly, there has been increasing pressure on FDA to remove certain accelerated approval drugs from the market following a failure by the drug product sponsor to confirm the efficacy or clinical benefits of the product in the required post-marketing confirmatory clinical trial. The most visible example of this regulatory challenge came in October 2019 when FDA convened an advisory committee to recommend whether it should withdraw accelerated approval from hydroxyprogesterone caproate injection (marketed under the brand name Makena for the prevention of preterm birth in pregnant women). FDAs Bone, Reproductive and Urologic Drugs Advisory Committee voted 9-7 to withdraw approval, with the dissenters favoring leaving Makena on the market while requiring the sponsor to conduct a new confirmatory trial. (Notably, no one voted for the option of leaving it on the market without requiring a new confirmatory trial.)Among the concerns of some members who voted to leave the product on the market with a new clinical trial obligation was that the drugs withdrawal would leave no safe treatment options for pregnant women at high risk of preterm birth. FDA will have to make a final decision regarding what to do about Makena in 2020, and it undoubtedly will face intense criticism (and potentially legal challenge) no matter what route it chooses to take for this public health quandary in which it finds itself.

In a similar vein, FDA official Dr. Richard Pazdur participated in a Senate briefing on December 10, 2019, in which he and other speakers defended the Breakthrough Therapy Designation program. FDA insisted again that the designation was intended to let the agency have earlier interactions with drug sponsorsand that it was not meant to be an early rating system for drugs or a signal of how they might do commercially. Given that the Breakthrough program was created in 2012 and is considered to be wildly successful, some speakers at the briefing expressed surprise that there was still any confusion about its purpose and function. Whether Congress picks up any of these emerging areas for consideration as part of FDAs 2022 user fee reauthorization packages remains to be seenas those negotiations will begin in earnest after the New Year, but the issues certainly are complex enough to allow for robust policy discussions to occur.

Finally, there are expected to be bumps in the road with the upcoming March 2020 transition of proteins previously approved under New Drug Applications (NDAs) to Biologics License Applications (BLAs)for which FDA only finalized its guidance for industry last year. The March 2020 transition date was established under the BPCIA and the agency does not have discretion in getting the transition done (only in how it handles the logistical and administrative issues created by transitioning approved products in this way). Check out our prior blog post on the final deemed to be a license transition guidance.

Business as Usual with the Rapid Pace of FDAs Issuance of Agency Guidance But Challenges Are Emerging Related to Judicial Deference to FDA Decision-Making

FDA guidance documents for all regulated product categories continued to be released on a regular basis this year, including several related to areas of agency priorities under the DCAP and BAP including the final biosimilar interchangeability guidance issued in May (see our blog post here) and a draft guidance on insulin interchangeability issued in November 2019. The latter also relates to the March 2020 NDA-to-BLA transition, as insulins are one of the largest class of products that will be transitioning into regulation as biologics, making them open to what is expected to be more efficient competition through the BPCIAs biosimilar pathway than what was possible in the past as insulin NDAs.

Despite the accelerated pace of the issuance of Agency guidance, however, FDA is beginning to face more challenges related to its decision-making and the scope of its exercise of agency discretion. In particular, a significant District Court for the District of Columbia ruling issued on December 6, 2019, Genus Medical Technologies, LLC v. FDA, provides hints of a potential shift in judicial deference to certain agency actions. The court vacated FDAs classification of a medical imaging liquid as a drug rather than as a device after determining that FDA did not have discretion to decide how to regulate a product merely because the definitions of drug and device overlap in the FD&C Act.

According to the district court judge, FDA was not interpreting the drug/device definitions in the statute properly, and Congress did not intend to allow the agency unfettered discretion to pick between the two categories. Rather, the court found that the text of the definitions are clear and do not create a gap or any ambiguity for FDA to fill with an exercise of agency discretion. This recently issued decision may indicate a potential shift in how courts are going to apply long-standing precedents related to judicial deference to agency decisions. If FDA decides to appeal the Genus ruling, it may end up at the Supreme Court as one of many expected challenges to the doctrines that established our current framework for judicial deference of an administrative agencys interpretation of an ambiguous statute.

Business as Usual with FDAs Comprehensive Regenerative Medicine Framework and Stepped up Enforcement Against Stem Cell Clinics Offering Unlawful Products But What Happens in November 2020 When the Enforcement Discretion Period Ends?

One of the first FDA press releases for 2019 was co-authored by former Commissioner Gottlieb and Center for Biologics Evaluation and Research (CBER) Director Peter Marks and was focused on the agencys new policies aiming to advance the development of safe and effective cell and gene therapies. In the press release, the agency leaders predicted that by 2020, FDA would receive more than 200 Investigational New Drug Applications (INDs) for cell and gene therapies each year. The agency has continued to work diligently to increase its staff in CBER to conduct clinical reviews for such INDs and to try to keep pace with the industrys development of these innovative technologies.

As we discussed in our update on FDAs Comprehensive Regenerative Medicine Policy Framework earlier this year, the agency is prioritizing two parallel goals: (1) clarifying the regulatory criteria for product marketing and providing support and guidance to legitimate product developers; and (2) removing unapproved, unproven, and potentially unsafe products from the U.S. market. The second prong of this comprehensive plan for regenerative medicine products was the topic of one of Dr. Gottliebs very last statements as Commissioner before he left the agency, issued on April 3, 2019 in conjunction with CBER Director Dr. Marks, indicating how important this area is to the agencys current public health priorities.

In the April 2019 statement, Drs. Gottlieb and Marks acknowledged FDAs challenges and efforts to stop stem cell clinics and manufacturers from marketing unapproved products that put patients at risk, citing several Warning Letters issued to manufacturers that violated current good manufacturing practices (CGMPs) for human cells and tissue products. They noted that it was of particular concern given that the industry was nearly halfway through the period during which the FDA intends to exercise enforcement discretion for certain regenerative medicine products with respect to INDs and premarket approval requirements. Now that it is December 2019, that deadline is even closer with less than one year left. November 2020 is the end of the three-year period of enforcement discretion announced by FDA when it first articulated the policies and goals of this comprehensive framework in 2017. See our prior posts on the topic here and here.

Under the Comprehensive Regenerative Medicine Policy Framework, FDA appears to have stepped up the pace of issuing Warning and Untitled Letters to sellers of unapproved stem cell products during the second half of 2019. In conjunction with a Warning Letter issued on December 5, 2019 to two related companies for processing and marketing unapproved umbilical cord blood-derived cellular products, Dr. Marks of CBER reiterated the agencys concerns about safety and reminded the public of the upcoming compliance deadline: As evidenced by the number of actions that the agency has taken this month alone, there are still many companies that have failed to come into compliance with the [FD&C Act] and FDAs regulations.

Dr. Marks was referring to two Untitled Letters that were issued to stem cell product distributors on November 20 and November 25, 2019, respectively. The press release cited above also added that the agency had also recently sent 20 letters to manufacturers and health care providers noting that it has come to [FDAs] attention that they may be offering unapproved stem cell products, reiterating the FDAs compliance and enforcement policy.

FDA also prevailed this year in the U.S. District Court of the Southern District of Florida against a stem cell clinic charged with violating the FD&C Act and the PHS Act. In June 2019, the court held that the defendants adulterated and misbranded a stem cell drug product made from a patients adipose tissue without FDA approval and for significant deviations from CGMPs, issuing a permanent injunction as requested by the Department of Justice on FDAs behalf. The agencys statement on that important court win by the government is available here.

Lastly, on December 6, 2019, FDA issued a Public Safety Notification on Exosome Products. The safety notification informed the public of multiple recent reports of serious adverse events experienced by patients in Nebraska who were treated with unapproved products marketed as containing exosomes, which came to FDAs attention through the Centers for Disease Control and Prevention, the Nebraska Department of Health and Human Services, and others. There are currently no FDA-approved exosome products and, to be honest, we are not even sure what such a product would be since an exome consists of all the sequenced exons within a single human genome after the introns are removed. (So were the clinics administering complete exomes to patients? that seems unlikely.)But what we found noteworthy about this public safety notice is the forceful and direct language FDA used when describing the unscrupulous conduct of the sellers of these products:

Certain clinics across the country, including some that manufacture or market violative stem cell products, are now also offering exosome products to patients. They deceive patients with unsubstantiated claims about the potential for these products to prevent, treat or cure various diseases or conditions. They may claim that they these products do not fall under the regulatory provisions for drugs and biological products that is simply untrue. As a general matter, exosomes used to treat diseases and conditions in humans are regulated as drugs and biological products under the [PHS Act] and the [FD&C Act] and are subject to premarket review and approval requirements.

The clinics currently offering these products outside of FDAs review process are taking advantage of patients and flouting federal statutes and FDA regulations. This ultimately puts at risk the very patients that these clinics claim to want to help, by either delaying treatment with legitimate and scientifically sound treatment options, or worse, posing harm to patients, as evidenced by these recent reports of adverse events.

As we enter the final year of FDAs enforcement discretion period, perhaps these public notices and Warning/Untitled Letters will become even more frequent and the agency will become even more frustrated by the ongoing violations and medical practitioners who flout federal law. This area will see substantial activity in 2020 and we will be watching closely to see what changes, if anything, about FDAs approach in November when the deadline to come into compliance ends. Will there be widespread FBI raids on stem cell clinics engaged in this kind of bad behavior? Only time will tell.

Final Thoughts: A Few Other Business as Usual Activities in the Therapeutic Product Areas and Prescription Drug Advertising Enforcement

Although we have highlighted what we view as some important challenges for the FDA to address in the coming months, other areas continue to be business as usual without anything very new to report. FDA continues to invest significant resources into improving the quality of compounded drugs and ensuring compliance with Sections 503A and 503B of the FD&C Act. Drug compounding was another topic of one of Dr. Gottliebs very last statements as Commissioner on April 3, 2019, in which he laid out the 2019 compounding priorities that included maintaining quality manufacturing and compliance and regulating compounding from bulk drug substances.

Notwithstanding all the efforts by FDA and State regulators in this area over the past several years, the agency continues to see concerning activity when it comes to compounded drugs, such as problems related to the condition under which compounded sterile medicines are made, which raisesignificant risks to patients. As a result, FDA has made it an intense area of focus to take enforcement actions against compounders who fail to produce sterile drugs in compliance with the law. During the past year, for example, FDA has won at least four permanent injunctions against various compounders after the agency identified behavior that posed a significant risk to public health and safety.

In addition, in 2019 FDA also increased its activities towards reducing and mitigating the impact of drug shortages on the health care system. See our prior blog post on Drug Shortages.

Finally, after a fairly slow year of enforcement in the prescription drug advertising space, the last two months of 2019, at least as of December 12th, have given us three (!) letters two untitled and one warning from the FDAs Office of Prescription Drug Promotion (OPDP). Most interestingly, the Warning Letter issued on December 2, 2019 for omitting warnings about the most serious risks associated with [a medication-assisted treatment] drug from promotional materials was announced to the public via FDA press release, which is not a typical action for normal-course OPDP letters to industry. The drug in question, approved for the prevention of relapse to opioid dependence following opioid detoxification, is associated with several significant risks including potential opioid overdose. Given the countrys public health emergency that is the opioid epidemic, FDA appears to have felt the need to make the deficiencies in the advertisement and those risks more widely publicized. So another thing we will be watching for in the New Year is whether this OPDP action represents the beginning of a new trend by the agency to publicize these Warning Letters more directly, or whether its advertising enforcement activities may be picking up due to industrys evolving approaches to promoting therapeutic products.

If you made it to the end of this post, thank you for reading our tome, and we hope you found it helpful and interesting! Stay tuned next week for our third and final FDA year-end blog post.

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Drugs, Biologics, and Regenerative Medicine in 2019: A Successful Year Ends with Promise of a More Challenging 2020 - JD Supra

Automation in Cell Therapy Manufacturing Is Driving the Growth of the Cell Therapy Market over the Forecast Period (2019-2025) – Press Release -…

A recent report published by Infinium Global Research on cell therapy market provides in-depth analysis of segments and sub-segments in the global as well as regional cell therapy market.

This press release was orginally distributed by SBWire

Pune, India -- (SBWIRE) -- 12/13/2019 -- The Infinium Global Research analyzes the "Cell Therapy Market (Cell Type - Stem Cell, and Differentiated Cell; Therapy Type - Allogenic Therapies, and Autologous Therapies; Application - Autoimmune Diseases, Oncology, Dermatology, Musculoskeletal Therapies, and Other Applications; End-users - Hospitals and Clinics, Biotechnology and Pharmaceutical Companies, and Research Institutes): Global Industry Analysis, Trends, Size, Share and Forecasts to 2025."

For More Details Get FREE Sample Pages of this Research Report@

Cell therapy is the transplantation of human cells to replace or repair damaged tissue or cells. With new technologies, products, innovative, and limitless imagination. Cells used for cell therapy often stem cells, cells that can mature into different types of specialized cells. The most common type of cell therapy has been the replacement of mature, functioning cells through blood and platelet transfusions. Cell therapies treat cancer, improving a weakened immune system, autoimmune disease, rebuilding damaged cartilage in joints, urinary problems, and infectious disease, repairing spinal cord injuries, and helping patients with neurological disorders.

Automation in Cell Therapy Manufacturing Providing Intensive Opportunities in the Cell Therapy Market

The rising occurrence of chronic diseases across the globe is the major driver for the growth of the global cell therapy market. Moreover, increasing the geriatric population, increase in cell therapy transplantation rate, and replacement of animal testing model are some of the key factors fueling the market growth. Furthermore, increasing awareness of stem cell therapy and the development of cell banking facilities contributing to the growth of the global cell therapy market.

Moreover, technological advancements and improvements in the regulatory framework continuously provide to the growing market. However, challenges in research and development, lack of proper and advanced healthcare in developing regions may hinder market growth. Furthermore, automation in cell therapy manufacturing providing intensive opportunities in the cell therapy market in the coming years.

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Stem Cell Therapy is Expected to Hold Maximum Share in the Cell Therapy Market

The global cell therapy market is segmented on the basis of type, therapy, application, and end-user. Types are further segmented into stem cells and differentiated cells. Stem cell therapy is expected to hold maximum share in the cell therapy market. Stem cell therapies having several advantages like regenerating the body organisms and other cells, which contributes to the growth of the stem cell therapies market.

By therapy, the cell therapy market is segmented into allogeneic therapies and autologous therapies. On the basis of application, the market is further divided into autoimmune diseases, oncology, dermatology, musculoskeletal therapies, and other applications. Based on end-users, the market is hospitals and clinics, biotechnology and pharmaceutical companies, and Research Institutes.

North America is Leading the Market Share in the Cell Therapy Market

The cell therapy market is segmented regionally into North America, Europe, Asia-Pacific, and the Rest of the World. North America is leading the market share in the cell therapy market due to the high rate of cancer and blood-related disorders coupled with high investments in the research and development of novel technologies. North America has a firm healthcare organization that acts as an added advantage for the growth of the market in this region.

Asia-Pacific region is anticipated to stimulate the growth of the cell therapy market due to a large number of surgeries performed in this region. Rising awareness about advanced medicinal therapies contributes to the growth of the market in the Asia-Pacific region.

Browse Detailed TOC and Description of this Exclusive Report@

Cell Therapy Market: Competitive Analysis

The leading players in the cell therapy market are NuVasive, Inc., Kolon TissueGene, Inc., JCR Pharmaceuticals Co., Ltd., Osiris Therapeutics, Inc., Stemedica Cell Technologies, Inc., MEDIPOST, Stemedica Cell Technologies, Inc., Celgene Corporation, ANTEROGEN.CO., LTD, Vericel Corporation. These companies are adopting strategic partnerships to enhance their product portfolio.

Reasons to Buy this Report:

=> Comprehensive analysis of global as well as regional markets of the cell therapy.

=> Complete coverage of all the product type and application segments to analyze the trends, developments, and forecast of market size up to 2025.

=> Comprehensive analysis of the companies operating in this market. The company profile includes analysis of product portfolio, revenue, SWOT analysis and the latest developments of the company.

=> Infinium Global Research- Growth Matrix presents an analysis of the product segments and geographies that market players should focus to invest, consolidate, expand and/or diversify.

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Automation in Cell Therapy Manufacturing Is Driving the Growth of the Cell Therapy Market over the Forecast Period (2019-2025) - Press Release -...

Bioethics experts call on GoFundMe to ban unproven medical treatments – The Verge

A bioethics study published on December 8th calls on crowdfunding platform GoFundMe to ditch campaigns for unproven and unsafe medical procedures.

People turn to GoFundMe for help paying for all sorts of medical interventions. These campaigns have brought in over $650 million since 2010. But a subset of the money raised is spent on unproven and even illegal operations. Unregulated stem cell therapies, for example, attract harsh condemnation from the Food and Drug Administration, and Google even banned ads for the procedures. But the public fundraisers still appear on GoFundMe.

In the new paper, published in the peer-reviewed bioethics journal The Hastings Center Report, the authors argue that GoFundMe enables misinformation that enriches bad actors and can harm patients sick with cancer or other serious conditions. Between November 2017 and November 2018, GoFundMe campaigns raised over $5 million for unregulated neurological stem cell procedures, according to a recent study. Those campaigns were shared over 200,000 times on social media.

They know this is happening. It cant happen without their involvement, says Jeremy Snyder, a bioethics researcher at Simon Fraser University and co-author of the report. I think they should be ashamed of themselves for taking part in it.

This report comes days after The Washington Post reported that an unregulated stem cell treatment center based in Tampa, Florida, openly coached patients to take out loans and crowdfund thousands of dollars for risky procedures.

I think its absolutely beyond time for them to stop, Snyder says about GoFundMes inaction. And an instance of them running counter to what the rest of the tech sector seems to be doing.

Tech companies are facing more scrutiny for enabling clinics that push pseudoscience, and major players like Facebook and Google have taken action. Facebook is removing sensational health claims, and Google recently banned predatory ads for unregulated cell therapies. But GoFundMe has yet to act in a comparable way when it comes to similar treatments.

Alison Bateman-House, an assistant professor at New York Universitys Langone Health and a bioethics expert who is unaffiliated with the report, says its perfectly reasonable to bar unproven treatments from fundraising.

Bateman-House is concerned that GoFundMe allows misinformation, suggesting it messes with patients abilities to make informed decisions by not policing false medical claims. We know that most Americans are not medically literate, she says. Where there is money to be made, some will prey on the hopes and misunderstandings of others.

In response to questions from The Verge, a GoFundMe spokesperson shared a company statement related to its policies on stem cell therapy. The statement says it is reaching out to experts and medical regulatory authorities to understand the effect on their customers, but that ultimately it is up to the GoFundMe community to decide which campaigns to donate to.

Every campaign on GoFundMe whether its for regulated or unregulated treatments is an opportunity for the site to make money. When someone donates to a cause, the platform gives donors an option to add a voluntary tip to the company, which defaults to 10 percent.

The paper, written by Snyder and his co-author, Harvard Law professor I. Glenn Cohen, suggests steps GoFundMe may take to upend its ethical problem. They concede that expecting the platform to independently evaluate evidence for medical claims would be expensive and difficult. Instead, they propose a white list approach, only allowing people to raise money for regulated treatments or those cleared by the FDA for a special program called expanded access.

There may be some challenges to implementing, says Patricia Zettler, a law professor formerly with the FDA who is unaffiliated with the report. But, as they say, we shouldnt let the perfect be the enemy of the good...these are sensible suggestions.

Another option the authors propose is to compile a black list of egregious procedures. They encourage GoFundMe to partner with organizations like the American Cancer Society to create the lists, in addition to the FDA, which frequently sends warning letters to problematic clinics.

In fact, some experts say that one way to avoid these crowdfunding issues would be to not only push platforms to act, but also to give bodies like the FDA more power to regulate them. Were in a moment right now where theres a lot of push to deregulate everything, says Aziza Ahmed, an expert in health law who is not affiliated with the study. I do think we need to press these tech companies to act a lot more ethically, but at the same time we need to do a better job of beefing up the FDA.

GoFundMe has banned campaigns in the past. The site removed anti-vaxxers in March, and it banned fundraising for a high-profile and highly controversial German cancer clinic in July. But many controversial treatments such as LGBTQ conversion therapy and unproven treatments for brain conditions are not yet prohibited by GoFundMe.

I think their first step would be to seek ethical advice, says Cohen. Crowdsourcing platforms could try white or black list approaches, and either would be superior to the status quo free-for-all.

Neither Snyder nor Cohen could predict whether their report will lead to change, but Snyder is certain that change is overdue. I just dont see that GoFundMe can continue to stick their head in the sand and pretend this isnt a problem on this platform.

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Bioethics experts call on GoFundMe to ban unproven medical treatments - The Verge

Must-Reads Of The Week From Brianna Labuskes – Kaiser Health News

Happy Friday! Hope everyone had a lovely, restful Thanksgiving. And heres your periodic (and Im sure very appreciated and not at all tiresome) reminder not to be one of the nearly 40% of Americans who plan to skip their flu shot.

Now on to our jam-packed week of news! Heres what you might have missed.

There seems to be some conflicting narrative around what exactly a new health care spending analysis means, but one thing is clear: We are now spending $11,172 per person and that is uh a lot, to say the least. Spending on health care grew at a slower pace than the economy overall. But the spending didnt increase because people were going to the doctor more. Instead, price hikes made up for the slower usage growth found by the HHS analysis.

If you want a good plain-English breakdown of what this means, check out this Axios summary of it all; they do a better job than I could. (No one told me thered be math!) Or listen to KHNs What The Health podcast by our fabulously knowledgeable Julie Rovner.

The New York Times: Health Spending Grew Modestly, New Analysis Finds

Axios: Health care spending grew at a faster rate in 2018

KHN: What The Health?: We Spend HOW MUCH On Health Care?

Speaking of outrageous hospital prices, in a move that shocked no one, hospitals officially filed a suit against the Trump administration rule that would compel them to share secretly negotiated prices for procedures.

The New York Times: Hospitals Sue Trump to Keep Negotiated Prices Secret

There has been an absolute flood of news out of the Trump administration this week, so buckle up.

Nearly 700,000 Americans are slated to lose their food stamp benefits under a new rule that tightens the work requirements in the Supplemental Nutrition Assistance Program (commonly known as SNAP). The move will purportedly save the government $5.5 billion over five years. The Agriculture Department defended the decision to crack down on waivers that extend the time a beneficiary can receive aid as, essentially, if not now (when the economy is good), then when?

Critics were quick to point out the economic and moral pitfalls of this kind of decision, among them being: Most beneficiaries work, and many of the ones who dont usually have a reason beyond wanting the $1.83 per meal that they receive under SNAP; its been shown that SNAP spending helps cushion the economy during a recession; and the people being cut off are among the most vulnerable in our society.

The Associated Press: 668,000 will lose food stamp benefits under new work rules

As part of the administrations goal to eradicate the HIV epidemic, HHS announced that uninsured Americans can now get free HIV-prevention drugs. While PrEP has been shown to be wildly successful, many people who are at high risk of contracting the virus arent taking the drug for one reason or another.

(I wonder if this is all a bit awkward, considering that HHS and PrEPs maker are locked in a bitter patent battle.)

The New York Times: 200,000 Uninsured Americans to Get Free H.I.V.-Prevention Drugs

Attorney General William Barr made waves when he suggested that communities upset with police brutality might lose protections from the cops theyre protesting. The remarks which seemed to encourage abandonment as a form of retribution for those seeking criminal justice reform were quickly condemned as dangerous.

The New York Times: Barr Says Communities That Protest the Police Risk Losing Protection

A new investigation from Reuters found that the FDA ignored warning bells about the dangers of talc as early as the 1970s. The agency for decades deferred to the industry over outside experts advice.

Reuters: FDA bowed to industry for decades as alarms were sounded over talc

And a disturbing video obtained by ProPublica contradicts the Border Patrols account of the death of a sick 16-year-old who was being held in U.S. custody. The video shows the boy staggering to the toilet and collapsing on the floor, where he remained in the same position for the next four-and-a-half hours. According to ProPublica, The video shows the only way CBP officials could have missed Carlos crisis is that they werent looking. Border Patrol also said it was agents who found his body but in reality a cellmate alerted them to his death.

ProPublica: Inside the Cell Where a Sick 16-Year-Old Boy Died in Border Patrol Care

Meanwhile, new documents reveal how a powerhouse consulting firm proposed money-saving methods for the detention centers that included proposed cuts in spending on food for migrants, as well as on medical care and supervision of detainees.

ProPublica: How McKinsey Helped the Trump Administration Detain and Deport Immigrants

Over on the presidential campaign trail, Medicare for All continues to trip up the candidates, including Sen. Elizabeth Warren (D-Mass.), whose fate seems to have become tied to the proposal, which wasnt even hers to start with. Politico takes us all the way back to a town hall in 2017 hosted by Sen. Kamala Harris (D-Calif.) to figure out how we got to where we are today.

Politico: The most consequential moment of the 2020 primary

The Washington Post: How a fight over health care entangled Elizabeth Warren and reshaped the Democratic presidential race

Meanwhile, some Dems in the race are touting a public option as a moderate alternative to Medicare for All, but dont let that fool you. That kind of shift could still send an earthquake through the health care landscape.

The New York Times: Why the Less Disruptive Health Care Option Could Be Plenty Disruptive

Gains made by the anti-abortion movement in recent years are often attributed to a well-executed ground game by the right. But there have also been crucial missteps on the other side of the abortion wars. Critics say that the national abortion rights movement lost touch with the ways access was steadily eroding in Republican strongholds, and that leaders grew overconfident during Barack Obamas presidency. As one director of a clinic in Atlanta told The New York Times: We were screaming at the top of our lungs, everything is not fine, please pay attention.

The New York Times: How a Divided Left Is Losing the Battle on Abortion

Theres been a ton of buzz around the first major gun case the Supreme Court took up in nearly a decade. But will it all be for naught? Arguments in the case, which centers around a NYC handgun ordinance, focused on the fact that the city got rid of the contested limits in July.

Reuters: U.S. Supreme Court justices debate whether to dismiss major gun case

I have to say I did not expect to read the phrase we are feared as a tiger with claws, teeth and balls when I kicked off my workweek, but here we are. As seems to be the case every time we get unsealed documents dealing with the Sackler family, the newest ones reveal how deeply involved Richard Sackler was in the aggressive push to market OxyContin.

Stat: Purdues Richard Sackler proposed plan to play down OxyContin risks, and wanted drug maker feared like a tiger, files show

So often opioid news focuses on the big players and court cases these days. But in this article, people who were high school kids at the time the epidemic was really starting to brew talk about how their lives have been irrevocably changed by the crisis.

The New York Times: The Class of 2000 Could Have Been Anything. Until Opioids Hit.

Despite the fact that it seems warnings are everywhere about the dangers of vaping, a new survey reveals that nearly 1 in 3 teens have used a tobacco product recently. Whats more, many students said they did not consider intermittent smoking of any product to be harmful.

The New York Times: Nearly a Third of Teens Use One or More Tobacco Products

In the miscellaneous file for the week:

In Rhode Island, 11 patients over a two-and-a-half-year span died because of a misplaced breathing tube (something thats never supposed to happen in emergency medicine). The state is the only one in New England to allow responders other than the most highly trained paramedics to place the tubes. But advocates say that, if they tighten those rules, it will cost even more lives, because fewer patients will have access to the equipment.

ProPublica: EMS Crews Brought Patients to the Hospital With Misplaced Breathing Tubes. None of Them Survived.

Every once in a while were hit with a story that reminds us that relying on technology especially when it comes to health care is a dangerous game despite the benefits it brings. This time it was a glitch with diabetes monitors.

Stat: A glitch in diabetes monitors serves as a cautionary tale for health tech

Patients who are desperate for a miracle are being given tips by stem cell clinics on how to raise enough money to afford unproven, and sometimes dangerous, treatments. Theres always GoFundMe, the clinics say when met with the patients financial barriers.

The Washington Post: Clinic pitches unproven treatments to desperate patients, with tips on raising the cash

Thats it from me! Have a great weekend.

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Must-Reads Of The Week From Brianna Labuskes - Kaiser Health News

Examining the ethics of embryonic stem cell research …

Last year, President Bush cast the first veto of his presidency when Congress tried to ease the restriction on federal funding of embryonic stem cell research.

Following the recent passage by both houses of Congress of the Stem Cell Research Enhancement Act of 2007, which would permit federal funding of research using donated surplus embryonic stem cells from fertility clinics, the president has once again threatened a veto.

Because neither the House nor the Senate had sufficient votes to override a presidential veto, it appears unlikely this new bill will be enacted into law, further stalling the pace of this research. This bill crosses a moral line that I and others find troubling, stated Bush, following the Senates vote.

SCL: What are the main arguments for and against embryonic stem cell research? MS: Proponents argue that embryonic stem cell research holds great promise for understanding and curing diabetes, Parkinsons disease, spinal cord injury, and other debilitating conditions. Opponents argue that the research is unethical, because deriving the stem cells destroys the blastocyst, an unimplanted human embryo at the sixth to eighth day of development. As Bush declared when he vetoed last years stem cell bill, the federal government should not support the taking of innocent human life.

It is surprising that, despite the extensive public debatein Congress, during the 2004 and 2006 election campaigns, and on the Sunday morning talk showsrelatively little attention has been paid to the moral issue at the heart of the controversy: Are the opponents of stem cell research correct in their claim that the unimplanted human embryo is already a human being, morally equivalent to a person?

SCL: Considering that the moral and political controversy over embryonic stem cell research centers on this very question, why do you think there is so little attention being paid to it? MS: Perhaps this claim has gone unaddressed because stem cell proponents and many in the media consider it obviously falsea faith-based belief that no rational argument could possibly dislodge. If so, they are making a mistake. The fact that a moral belief may be rooted in religious conviction neither exempts it from challenge nor puts it beyond the realm of public debate. Ignoring the claim that the blastocyst is a person fails to respect those who oppose embryonic stem cell research on principled moral grounds. It has also led the media to miss glaring contradictions in Bushs stem cell policy, which does not actually live up to the principle it invokesthat destroying an embryo is like killing a child.

It is important to be clear about the embryo from which stem cells are extracted. It is not implanted and growing in a womans uterus. It is not a fetus. It has no recognizable human features or form. It is, rather, a blastocyst, a cluster of 180 to 200 cells, growing in a petri dish, barely visible to the naked eye.

SCL: What are the contradictions in Bushs stance? MS: Before we address that, it is important to be clear about the embryo from which stem cells are extracted. It is not implanted and growing in a womans uterus. It is not a fetus. It has no recognizable human features or form.

It is, rather, a blastocyst, a cluster of 180 to 200 cells, growing in a petri dish, barely visible to the naked eye. Such blastocysts are either cloned in the lab or created in fertility clinics. The bill recently passed by Congress would fund stem cell research only on excess blastocysts left over from infertility treatments.

The blastocyst represents such an early stage of embryonic development that the cells it contains have not yet differentiated, or taken on the properties of particular organs or tissueskidneys, muscles, spinal cord, and so on. This is why the stem cells that are extracted from the blastocyst hold the promise of developing, with proper coaxing in the lab, into any kind of cell the researcher wants to study or repair.

The moral and political controversy arises from the fact that extracting the stem cells destroys the blastocyst. It is important to grasp the full force of the claim that the embryo is morally equivalent to a person, a fully developed human being.

For those who hold this view, extracting stem cells from a blastocyst is as morally abhorrent as harvesting organs from a baby to save other peoples lives. This is the position of Senator Sam Brownback, Republican of Kansas, a leading advocate of the right-to-life position. In Brownbacks view, a human embryo . . . is a human being just like you and me; and it deserves the same respect that our laws give to us all.

If Brownback is right, then embryonic stem cell research is immoral because it amounts to killing a person to treat other peoples diseases.

SCL: What is the basis for the belief that personhood begins at conception? MS: Some base this belief on the religious conviction that the soul enters the body at the moment of conception. Others defend it without recourse to religion, by the following line of reasoning: Human beings are not things. Their lives must not be sacrificed against their will, even for the sake of good ends, like saving other peoples lives. The reason human beings must not be treated as things is that they are inviolable. At what point do humans acquire this inviolability? The answer cannot depend on the age or developmental stage of a particular human life. Infants are inviolable, and few people would countenance harvesting organs for transplantation even from a fetus.

Every human beingeach one of usbegan life as an embryo. Unless we can point to a definitive moment in the passage from conception to birth that marks the emergence of the human person, we must regard embryos as possessing the same inviolability as fully developed human beings.

SCL: By this line of reasoning, human embryos are inviolable and should not be used for research, even if that research might save many lives.MS: Yes, but this argument can be challenged on a number of grounds. First, it is undeniable that a human embryo is human life in the biological sense that it is living rather than dead, and human rather than, say, bovine.

But this biological fact does not establish that the blastocyst is a human being, or a person. Any living human cell (a skin cell, for example) is human life in the sense of being human rather than bovine and living rather than dead. But no one would consider a skin cell a person, or deem it inviolable. Showing that a blastocyst is a human being, or a person, requires further argument.

Some try to base such an argument on the fact that human beings develop from embryo to fetus to child. Every person was once an embryo, the argument goes, and there is no clear, non-arbitrary line between conception and adulthood that can tell us when personhood begins. Given the lack of such a line, we should regard the blastocyst as a person, as morally equivalent to a fully developed human being.

SCL: What is the flaw in this argument? MS: Consider an analogy: although every oak tree was once an acorn, it does not follow that acorns are oak trees, or that I should treat the loss of an acorn eaten by a squirrel in my front yard as the same kind of loss as the death of an oak tree felled by a storm. Despite their developmental continuity, acorns and oak trees differ. So do human embryos and human beings, and in the same way. Just as acorns are potential oaks, human embryos are potential human beings.

The distinction between a potential person and an actual one makes a moral difference. Sentient creatures make claims on us that nonsentient ones do not; beings capable of experience and consciousness make higher claims still. Human life develops by degrees.

SCL: Yet there are people who disagree that life develops by degrees, and believe that a blastocyst is a person and, therefore, morally equivalent to a fully developed human being. MS: Certainly some people hold this belief. But a reason to be skeptical of the notion that blastocysts are persons is to notice that many who invoke it do not embrace its full implications.

President Bush is a case in point. In 2001, he announced a policy that restricted federal funding to already existing stem cell lines, so that no taxpayer funds would encourage or support the destruction of embryos. And in 2006, he vetoed a bill that would have funded new embryonic stem cell research, saying that he did not want to support the taking of innocent human life.

The distinction between a potential person and an actual one makes a moral difference. Sentient creatures make claims on us that nonsentient ones do not; beings capable of experience and consciousness make higher claims still. Human life develops by degrees.

But it is a striking feature of the presidents position that, while restricting the funding of embryonic stem cell research, he has made no effort to ban it. To adapt a slogan from the Clinton administration, the Bush policy might be summarized as dont fund, dont ban. But this policy is at odds with the notion that embryos are human beings.

SCL: If Bushs policy were consistent with his stated beliefs, how, in your opinion, would it differ from his current dont fund, dont ban policy? MS: If harvesting stem cells from a blastocyst were truly on a par with harvesting organs from a baby, then the morally responsible policy would be to ban it, not merely deny it federal funding.

If some doctors made a practice of killing children to get organs for transplantation, no one would take the position that the infanticide should be ineligible for federal funding but allowed to continue in the private sector. In fact, if we were persuaded that embryonic stem cell research were tantamount to infanticide, we would not only ban it but treat it as a grisly form of murder and subject scientists who performed it to criminal punishment.

SCL: Couldnt it be argued, in defense of the presidents policy, that Congress would be unlikely to enact an outright ban on embryonic stem cell research? MS: Perhaps. But this does not explain why, if the president really considers embryos to be human beings, he has not at least called for such a ban, nor even called upon scientists to stop doing stem cell research that involves the destruction of embryos. In fact, Bush has cited the fact that there is no ban on embryonic stem cell research in touting the virtues of his balanced approach.

The moral oddness of the Bush dont fund, dont ban position confused even his spokesman, Tony Snow. Last year, Snow told the White House press corps that the president vetoed the stem cell bill because he considered embryonic stem cell research to be murder, something the federal government should not support. When the comment drew a flurry of critical press attention, the White House retreated. No, the president did not believe that destroying an embryo was murder. The press secretary retracted his statement, and apologized for having overstated the presidents position.

How exactly the spokesman had overstated the presidents position is unclear. If embryonic stem cell research does constitute the deliberate taking of innocent human life, it is hard to see how it differs from murder. The chastened press secretary made no attempt to parse the distinction. His errant statement that the president considered embryo destruction to be murder simply followed the moral logic of the notion that embryos are human beings. It was a gaffe only because the Bush policy does not follow that logic.

SCL: You have stated that the presidents refusal to ban privately funded embryonic stem cell research is not the only way in which his policies betray the principle that embryos are persons. How so? MS: In the course of treating infertility, American fertility clinics routinely discard thousands of human embryos. The bill that recently passed in the Senate would fund stem cell research only on these excess embryos, which are already bound for destruction. (This is also the position taken by former governor Mitt Romney, who supports stem cell research on embryos left over from fertility clinics.) Although Bush would ban the use of such embryos in federally funded research, he has not called for legislation to ban the creation and destruction of embryos by fertility clinics.

SCL: If embryos are morally equivalent to fully developed human beings, doesnt it then follow that allowing fertility clinics to discard thousands of embryos is condoning mass murder? MS: It does. If embryos are human beings, to allow fertility clinics to discard them is to countenance, in effect, the widespread creation and destruction of surplus children. Those who believe that a blastocyst is morally equivalent to a baby must believe that the 400,000 excess embryos languishing in freezers in U.S. fertility clinics are like newborns left to die by exposure on a mountainside. But those who view embryos in this way should not only be opposing embryonic stem cell research; they should also be leading a campaign to shut down what they must regard as rampant infanticide in fertility clinics.

Some principled right-to-life opponents of stem cell research meet this test of moral consistency. Bushs dont fund, dont ban policy does not. Those who fail to take seriously the belief that embryos are persons miss this point. Rather than simply complain that the presidents stem cell policy allows religion to trump science, critics should ask why the president does not pursue the full implications of the principle he invokes.

If he does not want to ban embryonic stem cell research, or prosecute stem cell scientists for murder, or ban fertility clinics from creating and discarding excess embryos, this must mean that he does not really consider human embryos as morally equivalent to fully developed human beings after all.

But if he doesnt believe that embryos are persons, then why ban federally funded embryonic stem cell research that holds promise for curing diseases and saving lives?

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Examining the ethics of embryonic stem cell research ...

Stem of the problem | Columns | Journal Gazette – Fort Wayne Journal Gazette

I have been reading a book, The Man Who Wouldn't Die, that satirizes Silicon Valley and the venture-capital start-up culture. It's reasonably funny. I was reminded, however, that sometimes culture is crazy.

At one point a character casually describes how stem-cell therapy is obviously going to make 50-year-olds feel like 20-year-olds. Grow a new liver and you're good to go, so to speak! The author clearly meant the idea as a joke. However, that idea is out there and taken seriously. Stem cells made national headlines in the late '90s and early 2000s because of some scientific breakthroughs and their promise for future medical treatments.

Just about all of our body is made up of specialized cells. These are cells that have grown to perform some specific function. Some examples are heart muscle cells, say, or red blood cells.

Stem cells are different; they have not yet specialized. That is, they have not developed many of the specific properties that are unique to the different parts of our bodies.

Given the right environment the right chemicals, for examplethey can grow into specialized cells.

There are, of course, all sorts of important details for how stem cells grow, when they specialize and how much flexibility they have in specializing. Somehow, however, there is now an industry that has skipped all that work and is marketing stem cells as a general cure. Alzheimer's disease and joint pain are frequently mentioned. If that makes you suspicious that these clinics are targeting the elderly, you would be right.

A typical treatment involves taking stem cells from a patient's bone marrow or fat and injecting it back into a sore knee or hip or whatever needs fixing. The idea, somewhat vaguely, is that the stem cells will grow to replace whatever is worn down by age.

As is true of all good scams, this one has a good story, one that can pass as proven medicine for many patients. The treatment also benefits by treating a problem with symptoms that can come and go irregularly, so a patient can honestly claim that they recovered in, say,six months.

The body is a complicated machine. It is hard to predict what will cure or not cure any specific problem. Just because a story involving stem cells sounds reasonable is not a sufficient reason to try something.

For a typical patient, we should have good, strong, positive evidence that a treatment will help. A good story is not strong, positive evidence. In the language of science, it is a hypothesis. Potentially true.

It is worth remembering that many, many things are potentially true. We don't try them all out on ourselves when we are sick.

Many of these ideas have in fact been tested in rigorous, peer-reviewed studies. So far, the evidence for positive effects is weak.

One reason these clinics have managed to skirt the rules is that extracting material from a patient then reinjecting requires less oversight for safety. Depending on the details, this treatment is considered, for Food and Drug Administration regulations, something like plasma donation (where blood is extracted, platelets removed, then reinjected into the body).

Just because a process may be safe does not make it good medicine. Medicine is not and should not be a free market. Patients will almost never have enough knowledge to reasonably choose between treatment options.

The FDA is the federal agency in charge of ensuring companies do not offer useless or harmful procedures. Unfortunately, in 2017 the FDA decided to allow stem cell clinics a three-year grace period to describe their procedures for the FDA's evaluation. As a result of that open window, hundreds of clinics have opened nationwide, offering services for which there is no strong evidence.

In the past year the FDA has realized where the industry has gone with this treatment and begun trying to crack down. Some clinics have unsafe procedures; others are misleading patients about treatments. Many clinics are still operating, unfortunately, and it can be difficult for typical patients to recognize the difference between these treatments and proven remedies. Furthermore, these bad actors could potentially make people skeptical of stem cell-based technology entirely.

That would be a shame because there is still great promise for stem cells. That may be cold comfort for those who want a treatment immediately, but that desire is the exact motivation that has led to terrible medicine for centuries.

Christer Watson, of Fort Wayne, is a professor of physics at Manchester University. Opinions expressed are his own. He wrote this column for The Journal Gazette, where his columns normally appear the first and third Tuesday of each month.

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Stem of the problem | Columns | Journal Gazette - Fort Wayne Journal Gazette