Stem Cell Therapies: U.S. Changing Lanes

The U.S. Food and Drug Administration (“FDA”) has been vocal about their efforts to curtail stem cell programs. Yet, in a surprising turn of events, the FDA commissioner, Dr. Scott Gottlieb, and the director of FDA’s Center for Biologics Evaluation and Research, Dr. Peter Marks, have recently co-authored a paper that promotes more accurate research regarding the use of stem cell therapy in medicine. The paper sets forth a detailed plan for assessing the effectiveness of stem cell therapy.

Stem cells have the abilities to mimic other cells in the human body, cells that may not be working properly or at all in patients with various diseases and, therefore, can have significant impacts on the outcome of health. Most promising about Drs. Gottlieb and Marks’ paper is that it opens the door for expanded use of stem cell therapy in the U.S. which was long thought to be closed. In short, it appears that stem cell programs will no longer be given a cold hard “no,” in both the medical and regulatory context.

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Tags: Science, Stem cells, Health, Law, FDA


An Increasing Demand For The Right-To-Try

In recent years, thirty-three U.S. states have passed “right-to-try” laws aimed at increasing access to experimental treatments for terminally ill patients. Many such patients feel it is their right to try experimental medications, which have not been fully approved by the Food and Drug Administration (“FDA”) and are otherwise unavailable. On the other hand, some doctors and medical ethicists stand firm in their belief that FDA procedures and clinical trial processes should be adhered to as they were put in place for good reason – to ensure that drug manufacturers are producing a safe product. Furthermore, some argue that “right-to-try” laws do not expand patients’ access to effective drugs since pharmaceutical companies are not obligated to distribute experimental medications.

Excerpt from Article:

Right-to-try laws offer patients an alternative to the compassionate-use, or expanded-access, program the FDA has had in place for more than two decades. As many as 1,821 applications are filed each year, and about 99 percent of them are approved, according to the [FDA].


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A Potential End to the “Ice Age” of Transplant Organ Transportation

An innovative means for transporting organs may soon debut in the United States. It has the potential to bring organ transportation out of the “ice age.” TransMedics, a company headquartered in Andover, Massachusetts, has developed the Organ Care System, also known as the “heart in a box.” of the system can keep human organs “alive, beating, and breathing” during transport. Rather than transporting disconnected organs via ice cooler, the Organ Care System plugs hearts, lungs, livers, and other organs into a system that keeps blood, air, and other fluids circulating while the organ is maintained at body temperature. Organs – transported alive – have been successfully transplanted up to twenty-four hours after removal, which is roughly six times the current average life span for an organ outside the body. Waleed Hassanein, founder and president of TransMedics, has asserted that as long as an organ remains alive and “perfused in [the Organ Care System] . . . there really is no time limitation [for successful transplantation.]” Even more astonishing is the device’s ability to revive organs from recently deceased patients. Thus far, seventeen people have successfully received hearts from patients whose hearts had stopped beating for thirty minutes or more.

Although the Organ Care System has been in use for many years in Europe and Australia, the FDA has yet to approve its use in the United States. However, this may soon change: TransMedics is scheduled to introduce its system to the FDA on November 18, 2015. If FDA approves the system’s use, the “ice age” of transplant organ transportation may come to and end.


Shortcomings in Genetic Testing as Revealed By the New England Center for Investigative Reporting

Several new companies have introduced genetic tests aimed at making treatment of psychiatric illnesses more predictable and effective. Test makers assert that psychiatric genetic testing minimizes patients’ risk of adverse side effects by enabling physicians to select appropriate medications with less trial and error. However, a recent investigative report conducted by the New England Center for Investigative Reporting (the “Report”) raises significant questions about the effectiveness of these genetic tests.

Among the concerns raised in the Report, the lack of independent oversight and financial conflicts of interest are the most serious. The Report suggests that test makers may be overstating findings and have failed to seek outside review of their unproven products. The Report also alleges that financial incentives may be causing improper referrals because test makers have been paying medical professionals to speak favorably about their tests, which have yet to be proven effective. Furthermore, as a result of a highly controversial FDA exemption, the majority of these genetic tests have been introduced into the marketplace without FDA review. Although the FDA recently announced plans to regulate these tests, no timeline has been established.

Despite the ethical concerns raised in the report, psychiatric genetics may still hold the key to unlocking a definitive approach for identifying the drugs most likely to be effective for individuals suffering from a multitude of psychiatric conditions. Only time will tell if the analysis of an individual’s genetic make-up can be used to effectively determine drug response.


FDA Approves Cheaper Version of Popular Drug Prescribed for Chemotherapy Patients

In early March 2015, the Food and Drug Administration (“FDA”) approved Zarxio, the first “biosimilar” drug, to be introduced into the U.S. market. Biosimilars are expected to offer cheaper versions of existing biologics. They are nearly identical to the originals, and are used to treat the same conditions. Biosimilar drugs are expected to create a more competitive market by providing people with comparable, less expensive prescription medication.

Zarxio, the first of what is likely to be many biosimilar drugs to receive FDA approval, will compete with Neupogen, a drug prescribed to chemotherapy patients. Zarxio promises to be cheaper than Neupogen and similar enough to serve all five of Neupogen’s current uses.  However, questions remain as to how effective biosimilars will be in lowering the cost of drugs. In fact, the makers of Zarxio have not announced how much cheaper the drug will be.  Secondly, there is no telling whether clinicians will embrace biosimilars.

Amgen, the makers of Neupogen, asked a California federal judge for an injunction blocking the launch of Zarxio.  They allege that the makers of Zarxio failed to follow the rules in seeking regulatory approval.   A court hearing is set for March 13, and Novartis, the maker of Zarxio, has agreed not to sell Zarxio until the hearing or April 10, whichever is earlier.

Doctors Continue Use of Tool Which FDA Claims May Have 1 in 300 Chance of Spreading Cancer

Months after the Food and Drug Administration (“FDA”) warned against using laparoscopic morcellators, a gynecological tool used to remove fibroids during hysterectomies, many doctors have continued to use the device. The FDA warned that the tool, used to slice uterine tissue into fragments for removal, may leave behind benign and undetected malignant cancers. In fact, the FDA has assed the risk of the device spreading cancer at 1 in 300 to 1 in 1000. of.

Gynecologists who have decided to continue using the morcellator argue that the FDA’s data has been exaggerated, and it is getting in the way of patient treatment. They believe the benefits of using the tool outweigh the risks, claiming patients who undergo a morcellator procedure have lower bleeding risks, less infection, and recover more quickly than those undergoing other minimally invasive procedures.

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FDA Approves New “At Home” Colon Cancer Screening Test

The FDA recently approved a new “at home” colon cancer screening test known as Cologuard after it outperformed fecal immunalchemical testing (FIT), which was previously “the most reliable stool test on the market.” In a study comparing the two tests, Cologuard “accurately detected cancers and advanced adenomas” more often than FIT, and is the first test that detects “DNA mutations, as well as blood” to screen for cancerous growths. However, Cologuard detects more abnormalities that end up not being cancer than FIT. In fact 13% of those who used Cologuard received a false positive compared to FIT’s 5%.

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Promising Heart Valve Procedure Isn’t Without Skeptics

An alternative to open-heart surgery, transcatheter aortic valve replacement (TAVR) is a less-invasive heart-valve procedure for the treatment of severe aortic valve stenosis.   The procedure, which involves threading a catheter through patients’ arteries to implant new aortic valves, has received high praise as a “technological leap” in treatment.

TAVR has been used as an alternative for patients where open-heart surgery is too risky. Despite the early success of the procedure, TAVR has higher rates of some serious complications, including blood vessel damage and stroke than traditional treatments.

Despite its initial praise, some doctors worry that the early excitement regarding TAVR can lead physicians to rely on the procedure even when it is not the best option.  Currently the FDA requires that before patients can undergo the TAVR procedure, they must show that they “are not the right candidate” for traditional surgery. However, recent data show that more than half of Medicare patients who received catheter valves did not meet the FDA threshold for the procedure.

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Approximately 50% of Americans Believe Health Conspiracy Theories

JAMA Internal Medicine published a research letter by authors J. Eric Oliver, PhD and Thomas Wood, MA, which indicates that almost half of the nation believes in at least one health conspiracy. For example, thirty-seven percent of Americans believe that the Food and Drug Administration is concealing natural cures for cancer in response to industry pressure from pharmaceutical companies. The authors conducted an online survey of 1,351 adults; the survey posited four health conspiracy theories. Forty-nine percent of those polled indicated that they believe at least one of the proposed theories, and eighteen percent believe in at least three. According to Oliver, the takeaway from the survey “is that people who embrace these conspiracies are very suspicious of traditional evidence-based medicine.

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Antibiotics in Food Animals May Pose “High Risk” to Humans

The Natural Resources Defense Council (NRDC), an environmental advocacy group, has compiled a report on the safety of antibiotics used by American farmers to treat food animals. The report reflects the finding of Food and Drug Administration (FDA) scientists that 18 out of 30 antibiotics being given to the animals pose a “high risk” to human beings, by exposing them to antibiotic-resistant bacteria in the food supply. In addition, all 30 of the antibiotics studied, when used for non-therapeutic purposes (such as increasing an animal’s size), expose humans to antibiotic-resistant bacteria, and violate the FDA’s own safety guidelines on non-therapeutic use.

The NRDC claims that FDA efforts to “phase out” the administration of non-therapeutic drugs to food animals are not swift enough to protect the public. In December 2013, the FDA stated that it is taking action to promote “the judicious use of important antimicrobials to protect public health, while ensuring sick and at-risk animals receive the therapy they need.”

Read more here and here.