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].

 

Read Full Article

Researchers Restore Hearing in Deaf Mice by Inserting a Normal Copy of the Mutated Gene

Two recent publications in the Nature Biotechnology journal show that researchers were able to help deaf mice hear again after inserting a gene, Ush1c, into their ears. Initially, the mice were given Usher Syndrome type IC, which also causes deafness in humans. To combat the deafness this caused in the mice, the mice were then given a normal copy of the mutated gene. The mice, who were profoundly deaf, began to hear. This research has important implications for humans with hearing loss, in that approximately half of the cases of hearing loss in humans has a genetic component.

From wired.co.uk, “Biologists help deaf mice hear again by inserting healthy genes into their ears”

Excerpt from article:

“[T]he work – and the field in general – is trying to answer one big question: ‘Can you manipulate the system to cure things that are wrong?’”

Read Full Article

 

 

Cuba Discovers Cancer Treatment, Yet Embargo Keeps It Out of Reach for U.S. Patients

The fast-growing biotechnology industry in Cuba has created a new cancer drug called Cimavax. The drug targets epidermal growth cells and allows the body to attack the cancer on its own. Cancer patients who use the drug are expected to live 3 to 5 months longer.  Yet, patients in the United States have limited access to the new treatment. The United States has imposed a trade embargo on Cuba that prevents the importation of goods such as medication and, therefore,  requires patients to travel to Cuba in order to receive the new treatment.

From NYTimes.com, “A Souvenir Smuggled Home From Cuba: A Cancer Vaccine”

Excerpt from article:

“The trial could take years, but American cancer patients are not waiting. Over the past couple of years, dozens have slipped into Havana and smuggled vials of the vaccine in refrigerated lunchboxes back to the United States, sometimes not even telling their doctors. Talk about Cimavax on cancer patient networks online has been escalating steadily as relations between the two countries have warmed and more patients are making preparations to go…”

Read Full Article

 

Tags: Cancer, Treatment, News, Research

Editing Embryos: Scientist Begins Research Involving Modifying Genes of Human Embryos

In an article dated September 22, 2016, it was revealed that a Swedish scientist has become the first known researcher to begin attempts to modify genes in healthy human embryos—a practice which has been, and for many still is, considered “taboo.” Critics are concerned that people will begin to use such technology to create “designer” babies, or that such research could unintentionally create a new genetic disease; however, the scientist involved says he plans to use the embryos for no more than 14 days, in which time he may be able to discover new ways to treat infertility, prevent miscarriages, and use stem cells to treat various diseases.

From NPR.org, “Breaking Taboo, Swedish Scientist Seeks to Edit DNA of Healthy Human Embryos”

Excerpt from article:

“Lanner is planning to methodically knock out a series of genes that he has identified through previous work as being crucial to normal embryonic development. He hopes that will help him learn more about what the genes do and which ones cause infertility.”

Read Full Article.

 

FDA Takes the Advocates’ Advice: Approves Drug from Muscular Dystrophy

In an article dated September 19, 2016, Muscular Dystrophy patients and advocates, with the help of select members of Congress, celebrated the FDA’s approval of Eteplirsen, a drug manufactured by Sarepta Therapeutics for treatment of a type of muscular dystrophy that predominantly affects boys in their childhood. Advocates fought heavily for the approval even though there are limited clinical trials to confirm the drug’s effectiveness. While the drug may help up to 12,000 Americans affected by the disease, critics worry that the FDA has set a “dangerous precedent” by its decision.

From NYTimes.com, “F.D.A. Approves Muscular Dystrophy Drug That Patients Lobbied For”

Excerpt from article:

[I]t was taken as a deeply troubling sign among drug policy experts who believe the F.D.A. has been far too influenced by patient advocates and drug companies, and has allowed the delicate balance in drug approvals to tilt toward speedy decisions based on preliminary data and away from more conclusive evidence of effectiveness and safety. . .

To read full article:   F.D.A. Approves Muscular Dystrophy Drug That Patients Lobbied For

 

 

DNA Leftovers: Research and Difficulties in Obtaining Consent

What happens to a blood sample, or tissue from a biopsy, once the procedure is done and a nurse walks away with it? Most patients simply assume that it is used for whatever medical test their doctor ordered, but they do not consider what happens afterwards. Contrary to what most assume, tissue and other bodily matters that are the “leftovers” of a medical procedure can be used for research purposes. In fact, under the old Common Law Rule, researchers do not need to notify or obtain permission from patients before using such leftovers. The only limitation is that the bio-specimen must be non-identifiable. However, in recent years, scientists have discovered a way to re-identify anonymous samples by relying on DNA testing and publicly available information. Ethical concerns about re-identifying bio-specimens are reminiscent of concerns that surround the famous case of Henrietta Lacks, who died in 1951; yet, her so-called “immortal cells” are still used in laboratories to this day.

In order to address these concerns, the Department of Health and Human Services (“HSS”) has recently proposed a rule that would change the consent requirements. In particular, the rule would require researchers to obtain very broad consent for use of all bio-specimens, even those that are anonymous. In short, patients would be consenting to the use of their tissue and other bodily matters in all potential and unknown future research. The consent would also include permission for the researchers to access the patient’s identifying information. HHS has not yet released a template for the consent form. And while this rule would be a step forward in mitigating ethical concerns, critics say that even stricter requirements must be in place to ensure truly “informed” consent.

Sources: http://mobile.nytimes.com/2015/12/30/opinion/your-cells-their-research-your-permission.html?smid=tw-share&referer=&_r=2; http://www.hhs.gov/ohrp/humansubjects/regulations/nprmhome.html

The Use of Fetal Tissue From Abortions For Research: Acceptable or Exploitation?

Planned Parenthood was subject to national attention and scrutiny in late 2015 after videos surfaced that allegedly showed Planned Parenthood employees discussing the sale of fetal tissue from abortions for research. In response, an investigative panel was formed by the House of Representatives. In its first hearing, the panel engaged in a heated debate on the use of any fetal tissue, not just from abortions, for research purposes. While it seems that everyone can agree that this practice raises ethical concerns, there is sharp disagreement about what should and should not be permissible. Especially in the case of elective abortions, many argue that fetal tissue should not be used for research under any circumstances, even with the woman’s consent. One advocate even took the position that a woman who elects to have an abortion should not have the ability to give consent in the first place. Other opponents of fetal tissue research argued that such research could create an industry that may exploit a vulnerable and voiceless population. On the other hand, those who support the research took a utilitarian approach and explained the importance of tissue research for scientific advancements that could benefit a vast number of people.

While the panel’s focus may seem narrow to some, it implicates a host of ethical and legal questions: What right should a patient have to dictate what happens to his or her bodily tissue after a medical procedure? Should the answer depend on whether the procedure was elective or medically necessary? Should the potential benefit of fetal tissue research outweigh the risk of exploitation? What is our duty to protect vulnerable populations, such as women seeking abortions and their unborn fetuses? For now, these questions remain unanswered. The panel, however, will meet again in the next few months to hear arguments of additional witnesses and further discuss the issues at hand.

Sources: http://www.bioedge.org/bioethics/panel-on-infant-lives-meets-in-washington/11793; https://www.washingtonpost.com/news/powerpost/wp/2016/03/02/house-fetal-tissue-research/; http://docs.house.gov/meetings/IF/IF04/20160302/104605/HHRG-114-IF04-Wstate-LeeP-20160302.pdf

Prion Proteins Gone Rogue: Looking at the Impact of John Collinge’s Study on Transmissibility

A study conducted in January 2015 by John Collinge and his team of pathologists has opened the door to the theory that rogue proteins, known as amyloids, might share properties of misfolded proteins, known as prions, including their transmissibility.

The study was conducted on autopsied brains from four individuals who were previously injected with growth hormones. All four individuals died in their 40’s or 50’s as a result of Creutzfeldt-Jakob disease, a rare disease caused when the brain is contaminated by a prion. The study discovered that while the individuals died at relatively young ages, their brains were scarred with white plaque that is typically seen in people with Alzheimer’s, a disease that generally affects much older individuals. Collinge and his team concluded that this white plaque was a result of prion transmission from the growth hormone injections. This conclusion has stirred some controversy in the scientific field, as it suggests that Alzheimer’s may be transmissible from one person to another. It raises the possibility that certain aspects of amyloid-β protein can be transferred through medical procedures where fluid or tissue from one person is introduced into another, such as a blood transfusion or organ transplant.

Collinge published his findings in the September issue of Nature. Careful to not stir panic in the public and scientific community, Collinge stressed that the study did not conclude that Alzheimer’s is contagious, but rather brought to light how some medical procedures can transfer amyloid-β proteins without our knowledge. The results of the study have trickled down to worldwide academic discussions and a push for further understanding of putative amyloid seeds and varying strains of amyloids. Some members of the scientific community raised concern about creating premature panic when there could be other biological explanations. Collinge’s study used a small number of subjects, the subjects had no symptoms of Alzheimer’s prior to death, and their conditions may have been caused by the presence of other proteins.

It is unquestionable that Collinge’s study has highlighted the unknown and the need for further research. If his transmissibility theory proves to be true, it means amyloid seeds stick to metal surgical instruments and are not removed by normal sterilization. Consequently, the seeds may be transferred during surgery, whereby they remain in the body for years, spreading into plaque and inducing other pathological changes that cause neurodegenerative diseases.

Source: http://www.nature.com/news/the-red-hot-debate-about-transmissible-alzheimer-s-1.19554

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.

Source: http://www.andovertownsman.com/news/business/andover-company-develops-modern-organ-transplant-system/article_a0061efe-fa07-59ff-a292-9bb6bad96fba.html

New Research from Johns Hopkins University Explores How Stem Cells are Affected by their Surroundings

Researchers from Johns Hopkins University studied how immediate surroundings affect stem cells. Stem cells can develop into differentiated cells from their original state and therefore, hold a “promise of being used to replace damaged organs and muscle.” This study showcases how the application of stem cells could be greater, with more “reliable techniques to control how they take on specialized functions.” It was discovered that an enzyme, called aminopeptidase, located in the stem cell niche, helps keep stem cells in their original state by promoting specialized cells to transform into stem cells. This aids in the creation of more stem cells. However, it is still unclear how the stem cell niche performs this role.

Nevertheless, the results of this study could be crucial to future advances in medicine. Because it is possible for cell fate to change, in that specialized cells become stem cells by aminopeptidase, either from cues from the stem cell niche or randomly, then it could also be possible that random cell fate change can be a leading cause of cancer or other diseases.

Source: http://www.sciencedaily.com/releases/2015/10/151007185037.htm