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Posts Tagged ‘stem cell therapy

FDA stem cell therapy crackdown: a stem-free clinic

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By: Belinda Hauser, Ph.D.

20180107_Essay

source: pixabay

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

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

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

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

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

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Written by sciencepolicyforall

January 17, 2018 at 11:43 am

Science Policy Around the Web – July 21, 2017

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By: Rachel F Smallwood, PhD

Source: pixabay

Cancer

Engineered Cell Therapy for Cancer Gets Thumbs Up from FDA Advisers

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

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

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

Stem-Cell Therapy

Unapproved Stem-Cell Treatments Touted on Federal Database Clinicaltrials.Gov

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

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

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

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

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Written by sciencepolicyforall

July 21, 2017 at 10:08 am

Science Policy Around the Web – June 13, 2017

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By: Nivedita Sengupta, PhD

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

Stem Cell Therapy

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

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

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

Research Funding

NIH Scraps Plans for Cap on Research Grants

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

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

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Written by sciencepolicyforall

June 13, 2017 at 7:08 pm