FDA WARNS ABOUT STEM CELL THERAPIES FROM UNSCRUPULOUS PROVIDERS

Doris Tyler lay on the examining table as the doctor stuck a long, thin tube into her belly. The doctor pulled back a plunger, and the syringe quickly filled with yellow blobs tinged with pink.

“Look at that beautiful fat coming out. Liquid gold!” one of the clinic’s staff exclaimed in a video of the procedure provided to The Washington Post.

Hidden in that fat were stem cells with the amazing power to heal, the Stem Cell Center of Georgia had told Tyler. The clinic is one of hundreds that have popped up across the country, many offering treatments for conditions from Parkinson’s disease to autism to multiple sclerosis. WASHINGTON POST

Stem cells have been called everything from cure-alls to miracle treatments. But don’t believe the hype. Some unscrupulous providers offer stem cell products that are both unapproved and unproven. So beware of potentially dangerous procedures—and confirm what’s really being offered before you consider any treatment.

The facts: Stem cell therapies may offer the potential to treat diseases or conditions for which few treatments exist. Sometimes called the body’s “master cells,” stem cells are the cells that develop into blood, brain, bones, and all of the body’s organs. They have the potential to repair, restore, replace, and regenerate cells, and could possibly be used to treat many medical conditions and diseases.

But the U.S. Food and Drug Administration is concerned that some patients seeking cures and remedies are vulnerable to stem cell treatments that are illegal and potentially harmful. And the FDA is increasing its oversight and enforcement to protect people from dishonest and unscrupulous stem cell clinics, while continuing to encourage innovation so that the medical industry can properly harness the potential of stem cell products.

To do your part to stay safe, make sure that any stem cell treatment you are considering is either:

  • FDA-approved, or;
  • Being studied under an Investigational New Drug Application (IND), which is a clinical investigation plan submitted and allowed to proceed by the FDA.

Stem Cell Uses and FDA Regulation

The FDA has the authority to regulate stem cell products in the United States.

Today, doctors routinely use stem cells that come from bone marrow or blood in transplant procedures to treat patients with cancer and disorders of the blood and immune system.

With limited exceptions, investigational products must also go through a thorough FDA review process as investigators prepare to determine the safety and effectiveness of products in well-controlled human studies, called clinical trials. The FDA has reviewed many stem cell products for use in these studies.

As part of the FDA’s review, investigators must show how each product will be manufactured so the FDA can make sure appropriate steps are being taken to help assure the product’s safety, purity, and strength (potency). The FDA also requires sufficient data from animal studies to help evaluate any potential risks associated with product use. (You can learn more about clinical trials on the FDA’s website.)

That said, some clinics may inappropriately advertise stem cell clinical trials without submitting an IND. Some clinics also may falsely advertise that FDA review and approval of the stem cell therapy is unnecessary. But when clinical trials are not conducted under an IND, it means that the FDA has not reviewed the experimental therapy to help make sure it is reasonably safe. So be cautious about these treatments.

About FDA-approved products derived from stem cells

The only stem cell-based products that are FDA-approved for use in the United States consist of blood-forming stem cells (hematopoietic progenitor cells) derived from cord blood.

These products are approved for limited use in patients with disorders that affect the body system that is involved in the production of blood (called the “hematopoietic” system). These FDA-approved stem cell products are listed on the FDA website. Bone marrow also is used for these treatments but is generally not regulated by the FDA for this use.

Safety Concerns for Unproven Stem Cell Treatments

All medical treatments have benefits and risks. But unproven stem cell therapies can be particularly unsafe.

For instance, attendees at a 2016 FDA public workshop discussed several cases of severe adverse events. One patient became blind due to an injection of stem cells into the eye. Another patient received a spinal cord injection that caused the growth of a spinal tumor.

Other potential safety concerns for unproven treatments include:

  • Administration site reactions,
  • The ability of cells to move from placement sites and change into inappropriate cell types or multiply,
  • Failure of cells to work as expected, and
  • The growth of tumors.

Note: Even if stem cells are your own cells, there are still safety risks such as those noted above. In addition, if cells are manipulated after removal, there is a risk of contamination of the cells.

FDA Actions on Unapproved Stem Cell Products

When stem cell products are used in unapproved ways—or when they are processed in ways that are more than minimally manipulated, which relates to the nature and degree of processing—the FDA may take (and has already taken) a variety of administrative and judicial actions, including criminal enforcement, depending on the violations involved.

In August 2017, the FDA announced increased enforcement of regulations and oversight of stem cell clinics. To learn more, see the statement from FDA Commissioner Scott Gottlieb, M.D., on the FDA website.

And in March 2017, to further clarify the benefits and risks of stem cell therapy, the FDA published a perspective article in the New England Journal of Medicine.

The FDA will continue to help with the development and licensing of new stem cell therapies where the scientific evidence supports the product’s safety and effectiveness.

Advice for People Considering Stem Cell Therapies

Stem cell products have the potential to treat many medical conditions and diseases. But for almost all of these products, it is not yet known whether the product has any benefit—or if the product is safe to use.


If you’re considering treatment in the United States:

  • Ask if the FDA has reviewed the treatment. Ask your health care provider to confirm this information. You also can ask the clinical investigator to give you the FDA-issued Investigational New Drug Application number and the chance to review the FDA communication acknowledging the IND. Ask for this information before getting treatment—even if the stem cells are your own.
  • Request the facts and ask questions if you don’t understand. To participate in a clinical trial that requires an IND application, you must sign a consent form that explains the experimental procedure. The consent form also identifies the Institutional Review Board (IRB) that assures the protection of the rights and welfare of human subjects. Make sure you understand the entire process and known risks before you sign. You also can ask the study sponsor for the clinical investigator’s brochure, which includes a short description of the product and information about its safety and effectiveness.

If you’re considering treatment in another country:

  • Learn about regulations that cover products in that country.
  • Know that the FDA does not have oversight of treatments done in other countries. The FDA typically has little information about foreign establishments or their stem cell products.
  • Be cautious. If you’re considering a stem cell-based product in a country that may not require regulatory review of clinical studies, it may be hard to know if the experimental treatment is reasonably safe.

Last Updated: 11/16/2017

FDA Alerts Consumers of Nationwide Voluntary Recall of EpiPen and EpiPen Jr

 

SILVER SPRING, MD – The U.S. Food and Drug Administration is alerting consumers to Meridian Medical Technologies’ voluntary recalldisclaimer icon of 13 lots of Mylan’s EpiPen and EpiPen Jr (epinephrine injection) Auto-Injector products used for emergency treatment of severe allergic reactions. This recall is due to the potential that these devices may contain a defective part that may result in the devices’ failure to activate. The recalled product was manufactured by Meridian Medical Technologies and distributed by Mylan Specialty.

While the number of reported failures is small, EpiPen products that potentially contain a defective part are being recalled because of the potential for life-threatening risk if a severe allergic reaction goes untreated. Consumers should keep and use their current EpiPens if needed until they get a replacement. Consumers should contact Mylan at 800-796-9526 or customer.service@mylan.com with any questions.

As stated on the product label, consumers should always seek emergency medical help right away after using their EpiPens, particularly if the device did not activate.

At this time, the 13 lots identified – distributed between Dec. 17, 2015, and July 1, 2016 – are the only EpiPen lots impacted by the U.S. recall. Consumers who have EpiPens from lots that are not included in this recall, do not need to replace their EpiPen prior to its expiration date.

Product/Dosage NDC Number Lot Number Expiration Date
EpiPen Jr Auto-Injector, 0.15 mg 49502-501-02 5GN767 April 2017
EpiPen Jr Auto-Injector, 0.15 mg 49502-501-02 5GN773 April 2017
EpiPen Auto-Injector, 0.3 mg 49502-500-02 5GM631 April 2017
EpiPen Auto-Injector, 0.3 mg 49502-500-02 5GM640 April 2017
EpiPen Jr Auto-Injector, 0.15 mg 49502-501-02 6GN215 September 2017
EpiPen Auto-Injector, 0.3 mg 49502-500-02 6GM082 September 2017
EpiPen Auto-Injector, 0.3 mg 49502-500-02 6GM072 September 2017
EpiPen Auto-Injector, 0.3 mg 49502-500-02 6GM081 September 2017
EpiPen Auto-Injector, 0.3 mg 49502-500-02 6GM088 October 2017
EpiPen Auto-Injector, 0.3 mg 49502-500-02 6GM199 October 2017
EpiPen Auto-Injector, 0.3 mg 49502-500-02 6GM091 October 2017
EpiPen Auto-Injector, 0.3 mg 49502-500-02 6GM198 October 2017
EpiPen Auto-Injector, 0.3 mg 49502-500-02 6GM087 October 2017

 

The FDA asks health care professionals and consumers to report any adverse reactions or device malfunctions to the FDA’s MedWatch program, by:

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

WOMEN’S HEALTH ALERT – Public Meeting on Patient-Focused Drug Development for Sarcopenia

On April 6, 2017, from 1:00 pm to 5:00 pm, FDA is conducting a public meeting on Patient-Focused Drug Development (PFDD) for Sarcopenia. FDA is interested in obtaining patient perspectives on the impact of sarcopenia on daily life and patient views on treatment approaches. Registration to attend the meeting must be received by April 6, 2017.

In addition to providing input at the public meeting, stakeholders are invited to provide their perspectives on the discussion questions through the public docket. The docket closes on June 6, 2017.

Register for the meeting

Submit comments to the public docket

Sarcopenia, or the decline of skeletal muscle tissue with age, is one of the most important causes of functional decline and loss of independence in older adults.

FDA Approves First Treatment For Frequent Urination At Night Due To Overproduction Of Urine

 

March 3, 2017

SILVER SPRING, MD – The U.S. Food and Drug Administration today approved Noctiva (desmopressin acetate) nasal spray for adults who awaken at least two times per night to urinate due to a condition known as nocturnal polyuria (overproduction of urine during the night). Noctiva is the first FDA-approved treatment for this condition.

“Today’s approval provides adults who overproduce urine at night with the first FDA-approved therapeutic option to help reduce the number of times a night they wake up to urinate,” said Hylton V. Joffe, M.D., M.M.Sc., director of the Division of Bone, Reproductive, and Urologic Products in the FDA’s Center for Drug Evaluation and Research. “It is important to know that Noctiva is not approved for all causes of night-time urination, so patients should discuss their symptoms with their health care provider who can determine the underlying cause of the night-time urination and whether Noctiva is right for them.”

Nocturia (wakening at night to urinate) is a symptom that can be caused by a wide variety of conditions, such as congestive heart failure, poorly controlled diabetes mellitus, medications, or diseases of the bladder or prostate. Before considering Noctiva, health care providers should evaluate each patient for possible causes for the nocturia, and optimize the treatment of underlying conditions that may be contributing to the night-time urination. Because Noctiva is approved only for adults with nocturia caused by nocturnal polyuria, health care providers should confirm overproduction of urine at night with a 24-hour urine collection, if one has not been obtained previously. Health care providers should also be mindful of underlying conditions that can cause nocturia, but that make treatment with Noctiva unsafe, such as excessive drinking of fluids or symptomatic congestive heart failure.

Noctiva is taken daily, approximately 30 minutes before going to bed. It works by increasing the absorption of water through the kidneys, which leads to less urine production.

Noctiva’s efficacy was established in two 12-week, randomized, placebo-controlled trials in 1,045 patients 50 years of age and older with nocturia due to nocturnal polyuria. Although these trials showed a small reduction in the average number of night-time urinations with Noctiva compared to placebo, more patients treated with Noctiva were able to at least halve their number of night-time urinations, and patients treated with Noctiva had more nights with one or fewer night-time urinations.

Noctiva is being approved with a boxed warning and a Medication Guide because it can cause low sodium levels in the blood (hyponatremia). Severe hyponatremia can be life-threatening if it is not promptly diagnosed and treated, leading to seizures, coma, respiratory arrest or death. Health care providers should make sure the patient’s sodium level is normal before starting Noctiva, and should check sodium levels within one week and approximately one month after starting treatment and periodically thereafter. The lower Noctiva dose is recommended as the starting dose for those who may be at risk for hyponatremia, such as the elderly. Noctiva should not be used in patients at increased risk of severe hyponatremia, such as those with excessive fluid intake, those who have illnesses that can cause fluid or electrolyte imbalances, certain patients with kidney damage, and in those using certain medicines, known as loop diuretics or glucocorticoids.

Noctiva should also not be used in patients with symptomatic congestive heart failure or uncontrolled hypertension because fluid retention can worsen these underlying conditions. Use of Noctiva should be discontinued temporarily in patients with certain nasal conditions such as colds or allergies until those conditions have resolved.

Noctiva is also not recommended for the treatment of nocturia in pregnant women. Nocturia is usually related to normal changes in pregnancy that do not require treatment with Noctiva. Noctiva should not be used in children.

The most common side effects of Noctiva in clinical trials included nasal discomfort, cold symptoms (nasopharyngitis), nasal congestion, sneezing, high or increased blood pressure, back pain, nose bleeds, bronchitis and dizziness.

Although there are other FDA-approved medications that also contain desmopressin, none of those medications are approved to treat nocturia.

Noctiva is marketed by Milford, Pennsylvania-based Renaissance Lakewood, LLC for Serenity Pharmaceuticals, LLC.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The Agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

Is Rinsing Your Sinuses With Neti Pots Safe

 

nettipotopti12417

January 24, 2017

SILVER SPRING, MD – Little teapots with long spouts have become a fixture in many homes to flush out clogged nasal passages and help people breathe easier.

Along with other nasal irrigation systems, these devices — commonly called neti pots — use a saline, or saltwater, solution to treat congested sinuses, colds and allergies. They’re also used to moisten nasal passages exposed to dry indoor air. But be careful. According to the U.S. Food and Drug Administration (FDA), improper use of these neti pots and other nasal rinsing devices can increase your risk of infection.

These nasal rinse devices — which include bulb syringes, squeeze bottles, and battery-operated pulsed water devices — are usually safe and effective products when used and cleaned properly, says Eric A. Mann, MD, PhD, a doctor at FDA.

What does safe use mean? First, rinse only with distilled, sterile or previously boiled water.

Tap water isn’t safe for use as a nasal rinse because it’s not adequately filtered or treated. Some tap water contains low levels of organisms — such as bacteria and protozoa, including amoebas — that may be safe to swallow because stomach acid kills them. But in your nose, these organisms can stay alive in nasal passages and cause potentially serious infections. They can even be fatal in some rare cases, according to the Centers for Disease Control and Prevention (CDC).

What Types of Water Are Safe to Use?

  • Distilled or sterile water, which you can buy in stores. The label will state “distilled” or “sterile.”
  • Boiled and cooled tap water — boiled for 3 to 5 minutes, then cooled until it is lukewarm. Previously boiled water can be stored in a clean, closed container for use within 24 hours.
  • Water passed through a filter designed to trap potentially infectious organisms. CDC has information on selecting these filters.

Safely Use Nasal Irrigation Systems

Second, make sure you follow instructions.

“There are various ways to deliver saline to the nose. Nasal spray bottles deliver a fine mist and might be useful for moisturizing dry nasal passages. But irrigation devices are better at flushing the nose and clearing out mucus, allergens and bacteria,” Mann says.

Information included with the irrigation device might give more specific instructions about its use and care. These devices all work in basically the same way:

  • Leaning over a sink, tilt your head sideways with your forehead and chin roughly level to avoid liquid flowing into your mouth.
  • Breathing through your open mouth, insert the spout of the saline-filled container into your upper nostril so that the liquid drains through the lower nostril.
  • Clear your nostrils. Then repeat the procedure, tilting your head sideways, on the other side.

Sinus rinsing can remove dust, pollen and other debris, as well as help to loosen thick mucus. It can also help relieve nasal symptoms of sinus infections, allergies, colds and flu. Plain water can irritate your nose. The saline allows the water to pass through delicate nasal membranes with little or no burning or irritation.

And if your immune system isn’t working properly, consult your health care provider before using any nasal irrigation systems.

To use and care for your device:

  • Wash and dry your hands.
  • Check that the device is clean and completely dry.
  • Prepare the saline rinse, either with the prepared mixture supplied with the device, or one you make yourself.
  • Follow the manufacturer’s directions for use.
  • Wash the device, and dry the inside with a paper towel or let it air dry between uses.

Talk with a health care provider or pharmacist if the instructions on your device do not clearly state how to use it or if you have any questions.

Nasal Rinsing Devices and Children

Finally, make sure the device fits the age of the person using it. Some children are diagnosed with nasal allergies as early as age 2 and could use nasal rinsing devices at that time, if a pediatrician recommends it. But very young children might not tolerate the procedure.

Whether for a child or adult, talk to your health care provider to determine whether nasal rinsing will be safe or effective for your condition. If symptoms are not relieved or worsen after nasal rinsing, then return to your health care provider, especially if you have fever, nosebleeds or headaches while using the nasal rinse.

Health care professionals and patients can report problems about nasal rinsing devices to the FDA’s MedWatch Safety Information and Adverse Event Reporting Program.

FDA Approves Eucrisa for Atopic Eczema

December 14, 2016

Silver Springs, MD – The U.S. Food and Drug Administration today approved Eucrisa (crisaborole) ointment to treat mild to moderate eczema (atopic dermatitis) in patients two years of age and older.

Atopic dermatitis, a chronic inflammatory skin disease, is often referred to as “eczema,” which is a general term for the several types of inflammation of the skin. Atopic dermatitis is the most common of the many types of eczema and onset typically begins in childhood and can last through adulthood. The cause of atopic dermatitis is a combination of genetic, immune and environmental factors. In atopic dermatitis, the skin develops red, scaly and crusted bumps, which are extremely itchy. Scratching leads to swelling, cracking, “weeping” clear fluid, and finally, coarsening and thickening of the skin.

“Today’s approval provides another treatment option for patients dealing with mild to moderate atopic dermatitis,” said Amy Egan, deputy director of the Office of Drug Evaluation III in the FDA’s Center for Drug Evaluation and Research (CDER).

Eucrisa, applied topically twice daily, is a phosphodiesterase 4 (PDE-4) inhibitor, although its specific mechanism of action in atopic dermatitis is not known.

The safety and efficacy of Eucrisa were established in two placebo-controlled trials with a total of 1,522 participants ranging in age from two years of age to 79 years of age, with mild to moderate atopic dermatitis. Overall, participants receiving Eucrisa achieved greater response with clear or almost clear skin after 28 days of treatment.

Serious side effects of Eucrisa include hypersensitivity reactions. Eucrisa should not be used in patients who have had a hypersensitivity reaction to Eucrisa’s active ingredient, crisaborole. The most common side effect of Eucrisa is application site pain, including burning or stinging.

Eucrisa is manufactured by Palo Alto, California-based Anacor Pharmaceuticals, Inc.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

FDA Approves First Autologous Cellularized Scaffold For The Repair of Cartilage defects of the knee

Silver Springs, MD – The U.S. Food and Drug Administration today approved Maci (autologous cultured chondrocytes on porcine collagen membrane) for the repair of symptomatic, full-thickness cartilage defects of the knee in adult patients. Maci is the first FDA-approved product that applies the process of tissue engineering to grow cells on scaffolds using healthy cartilage tissue from the patient’s own knee.

Knee problems are common, and occur in people of all ages. Cartilage defects in the knee can result from an injury, straining the knee beyond its normal motion, or can be caused by overuse, muscle weakness, and general wear and tear.

“Different cartilage defects require different treatments, so therapy must be tailored to the patient,” said Celia Witten, Ph.D., M.D., deputy director of the FDA’s Center for Biologics Evaluation and Research. “The introduction of Maci provides surgeons with an additional option for treatment.”

Maci is composed of a patient’s own (autologous) cells that are expanded and placed onto a bio-resorbable (can be broken down by the body) porcine-derived collagen membrane that is implanted over the area where the defective or damaged tissue was removed. Administration should be performed by a surgeon specifically trained in the use of Maci.

Each Maci implant consists of a small cellular sheet containing 500,000 to 1,000,000 cells per cm2 (about 0.16 square inches). The amount of Maci administered depends on the size of the cartilage defect, and is trimmed to ensure that the damaged area is completely covered. Multiple implants may be used if there is more than one defect.

The safety and efficacy of Maci were shown in a two-year clinical trial designed to demonstrate reduced pain and improved function in comparison to microfracture, an alternative surgical procedure for cartilage repair. The trial included 144 patients (72 in each treatment group). A majority of the patients who completed the two-year clinical trial also participated in a three year follow-up study. Overall efficacy data support a long-term clinical benefit from the use of the Maci implant in patients with cartilage defects.

The most common side effect reported by people who received Maci were: joint pain, common cold-like symptoms, headache and back pain.

Maci is manufactured by Vericel Corporation, headquartered in Cambridge, Massachusetts.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

FDA Awards 21 Grants to Stimulate Product Development for Rare Diseases

 

Silver Spring, Maryland – The U.S. Food and Drug Administration today announced that it has awarded 21 new clinical trial research grants totaling more than $23 million over the next four years to boost the development of products for patients with rare diseases. These new grants were awarded to principal investigators from academia and industry with research spanning domestic and international clinical sites.

“We are proud of our 30-year track record of fostering and encouraging the development of safe and effective therapies for rare diseases through our clinical trials grant program,” said Gayatri R. Rao, M.D., J.D., director of FDA’s Office of Orphan Product Development, within the Office of Special Medical Programs. “The grants awarded this year will support much-needed research in 21 different rare diseases, many of which have little, or no, available treatment options.”

The FDA awards the grants through the Orphan Products Clinical Trials Grants Program to encourage clinical development of drugs, biologics, medical devices, or medical foods for use in rare diseases. The grants are intended for clinical studies evaluating the safety and effectiveness of products that could either result in, or substantially contribute to, the FDA approval of products.

Since its creation in 1983, the Orphan Products Clinical Trials Grants Program has provided more than $370 million to fund more than 590 new clinical studies and supported the marketing approval of more than 55 products. Five of the studies funded by this grants program supported product approvals in 2015 alone, including much needed treatments for neuroblastoma, lymphangioleiomyomatosis, hypoparathyroidism, and hypophosphatasia.

Consistent with the tenor set by Vice President Joe Biden’s National Cancer Moonshot Initiative to accelerate cancer research, 24 percent of the new grant awards fund studies enrolling patients with cancer; 40 percent of these studies target devastating forms of brain cancer, one of which recruits children with recurrent or progressive malignant brain tumors.

Forty-three percent of this year’s awards fund studies that enroll pediatric patients as young as newborns. Of these, two focus on research in transplantation and related issues.

In addition, one funded project is a medical device trial to develop a fully implantable neuroprosthesis for grasp, reach, and trunk function in individuals with spinal cord injury with the potential to enable these patients to use their hand, arm, and trunk more independently.

A total of 68 grant applications were received for this fiscal year, with a funding rate of 31 percent (21/68). The grant recipients for fiscal year 2016 include:

Drugs/Biologics:

  • Chemigen, LLC (Zionsville, Indiana), Yansheng Du, Phase 1 Study of CC100 for the Treatment of Amyotrophic Lateral Sclerosis — about $243,000 for one year
  • Chemocentryx, Inc. (Mountain View, California), Petrus Bekker, Phase 2 Study of CCX168 for the Treatment of Anti-Neutrophil Cytoplasmic Auto-Antibodies Associated Vasculitis — $500,000 for one year
  • Columbia University Health Sciences (New York, New York), Elizabeth Shane, Phase 2B Study of Denosumab to Prevent Bone Loss in Idiopathic Osteoporosis in Premenopausal Women Treated with Terripatide — about $1.6 million over four years
  • DNATRIX, Inc. (Houston, Texas), Frank Tufaro, Phase 2 Study of DNX-2401 for the Treatment of Glioblastoma — $2 million over four years
  • Elorac, Inc. (Vernon Hills, Illinois), Scott Phillips, Phase 3 Study of Naloxone Lotion for the Treatment of Pruritus in Mycosis Fungoides — about $2 million over four years
  • Johns Hopkins University (Baltimore, Maryland), Pamela Zeitlin, Phase 1/2 Study of Glycerol Phenylbutyrate for the Treatment of Cystic Fibrosis — $750,000 over three years
  • Oncoceutics, Inc. (Hummelstown, Pennsylvania), Wolfgang Oster, Phase 1/2 Study of ONC201 for the Treatment of Multiple Myeloma — about $1.7 million over four years
  • Oregon Health and Science University (Portland, Oregon), Kevin Winthrop, Phase 2 Study of Clofazimine for the Treatment of Pulmonary Mycobacterium Avium Disease — about $1.8 million over four years
  • Santhera Pharmaceuticals (Liestal, Switzerland), Thomas Meier, Phase 1 Study of Omigapil for the Treatment of Congenital Muscular Dystrophy — $246,000 for one year
  • Scioderm, Inc. (Durham, North Carolina), Jay Barth, Phase 3 Study of SD101 for the Treatment of Epidermolysis Bullosa — $500,000 for one year
  • Seattle Children’s Research Institute (Seattle, Washington), Leslie Kean, Phase 2 Study of Abatacept Combined with Calcineurin Inhibition and Methotrexate for Prophylaxis of Graft Vs Host Disease — $99,630 for one year
  • Sloan-Kettering Institute Cancer Research (New York, New York), Katharine Hsu, Phase 1 Study of Humanized 3F8 MoAb and NK cells for the Treatment of Neuroblastoma — about $750,000 over three years
  • Taimed Biologics USA Corp (Irvine, California), Stanley Lewis, Phase 3 Study of Ibalizumab for the Treatment of Patients with Multidrug Resistant HIV — $500,000 for one year
  • University of Alabama (Birmingham, Alabama), Gregory Friedman, Phase 1 Study of HSV G207 & Radiation for the Treatment of Pediatric Brain Tumors — about $750,000 over three years
  • University of California, San Diego (La Jolla, California), Donald Durden, Phase 1 Study of PI-3 Kinase/BRD4 Inhibitor SF1126 for the Treatment of Hepatocellular Carcinoma — $750,000 over three years
  • University of Florida (Gainesville, Florida), Peter Stacpoole, Phase 3 Study of Dichloroacetate for the Treatment of Pyruvate Dehyrugenase Complex Deficiency — about $2 million over four years
  • University of Michigan (Ann Arbor, Michigan), Kathleen Stringer, Phase 2 Study of Inhaled Activase for the Treatment of Acute Plastic Bronchitis — $2 million over four years
  • University of North Carolina Chapel Hill (Chapel Hill, North Carolina), Matthew Laughon, Phase 2 Study of Furosemide for the Prevention of Bronchopulmonary Dysplasia in Premature Infants — about $1.4 million over four years
  • Vanderbilt University Medical Center (Nashville, Tennessee), Cyndya Shibao, Phase 2 Study of Atomoxetine for the Treatment of Multiple System Atrophy — about $1.6 million over four years
  • Wilson Wolf Manufacturing Corporation (New Brighton, Minnesota), Sunitha Kakarla, Phase 1 Study of Viralym-A for the Treatment of Adenovirus Disease — about $750,000 over three years

Medical Devices:

  • Case Western Reserve University (Cleveland, Ohio), Kevin Kilgore, Phase 2 Study of a Networked Neuroprosthesis for Grasp, Reach, and Trunk Function in Cervical Spinal Cord Injury — about $2 million over four years

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

FDA Approves First Automated Insulin Delivery Device for Type 1 Diabetes

 

The U.S. Food and Drug Administration today approved Medtronic’s MiniMed 670G hybrid closed looped system, the first FDA-approved device that is intended to automatically monitor glucose (sugar) and provide appropriate basal insulin doses in people 14 years of age and older with type 1 diabetes.

The human pancreas naturally supplies a low, continuous rate of insulin, known as basal or background insulin. In patients with diabetes, the body’s ability to produce or respond to insulin is impaired.

medtronicloopdevice92816“The FDA is dedicated to making technologies available that can help improve the quality of life for those with chronic diseases – especially those that require day-to-day maintenance and ongoing attention,” said Jeffrey Shuren, M.D., J.D., director of the FDA’s Center for Devices and Radiological Health. “This first-of-its-kind technology can provide people with type 1 diabetes greater freedom to live their lives without having to consistently and manually monitor baseline glucose levels and administer insulin.”

The MiniMed 670G hybrid closed looped system, often referred to as an “artificial pancreas,” is intended to adjust insulin levels with little or no input from the user. It works by measuring glucose levels every five minutes and automatically administering or withholding insulin. The system includes a sensor that attaches to the body to measure glucose levels under the skin; an insulin pump strapped to the body; and an infusion patch connected to the pump with a catheter that delivers insulin. While the device automatically adjusts insulin levels, users need to manually request insulin doses to counter carbohydrate (meal) consumption.

According to the U. S. Centers for Disease Control and Prevention, approximately 5 percent of people with diabetes have type 1 diabetes. Also known as juvenile diabetes, type 1 diabetes is typically diagnosed in children and young adults. Because the pancreas does not make insulin in people with type 1 diabetes, patients have to consistently monitor their glucose levels throughout the day and have insulin therapy through injection with a syringe, an insulin pen or insulin pump to avoid becoming hyperglycemic (high glucose levels). In addition, management of type 1 diabetes includes following a healthy eating plan and physical activity.

“As part of our commitment to improving diabetes care, the FDA worked interactively with Medtronic from the earliest stages of development to assist in making this technology available to people with type 1 diabetes as quickly as possible,” said Alberto Gutierrez, Ph.D., director of the Office of In Vitro Diagnostics and Radiological Health in the FDA’s Center for Devices and Radiological Health. “We encourage companies to work closely with the agency to ensure scientifically sound, highly efficient clinical study designs, helping to expedite the FDA’s evaluation and subsequent approval of novel devices that can make a difference for patients.”

The FDA evaluated data from a clinical trial of the MiniMed 670G hybrid closed looped system that included 123 participants with type 1 diabetes. The clinical trial included an initial two-week period where the system’s hybrid closed loop was not used followed by a three-month study during which trial participants used the system’s hybrid closed loop feature as frequently as possible. This clinical trial showed that the device is safe for use in people 14 years of age and older with type 1 diabetes. No serious adverse events, diabetic ketoacidosis (DKA) or severe hypoglycemia (low glucose levels) were reported during the study.

Risks associated with use of the system may include hypoglycemia, hyperglycemia, as well as skin irritation or redness around the device’s infusion patch. This version of this device is unsafe for use in children 6 years of age or younger and in patients who require less than eight units of insulin per day.

As part of this approval, the FDA is requiring a post-market study to better understand how the device performs in real-world settings. While the device is being approved today for use in people 14 years of age and older with type 1 diabetes, Medtronic is currently performing clinical studies to evaluate the safety and effectiveness of the device in diabetic children 7-13 years old.

The MiniMed 670G hybrid closed looped system is manufactured by Medtronic, headquartered in Dublin, Ireland.

The FDA, an agency within the U.S. Department of Health and Human Services, promotes and protects the public health by, among other things, assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

Head Lice Every Parent’s Nightmare

Silver Spring, MD – A year-round problem, the number of cases seems to peak when the kids go back to school in the fall and again in January, says Patricia Brown, M.D., a dermatologist at the Food and Drug Administration (FDA).

An estimated 6 to 12 million cases of head lice infestation occur each year in the United States in children 3 to 11 years of age, according to the

headliceopti
Grooming in the bath with a nit comb to remove head lice.

Centers for Disease Control and Prevention. Head lice are most common among preschool children attending child care, elementary school children, and household members of children who have lice.

Contrary to myth, head lice are not caused by poor hygiene, Brown says. They are spread mainly by direct head-to-head contact with a person who already has head lice. You cannot get head lice from your pets; lice feed only on humans.

Lice don’t fly or jump; they move by crawling. But because children play so closely together and often in large groups, lice can easily travel from child to child, especially when they touch heads during playing or talking

Blood-Sucking Bugs

Head lice are blood-sucking insects about the size of a sesame seed and tan to grayish-white in color. They attach themselves to the skin on the head and lay eggs (nits) in the hair.

According to Brown, you can check for head lice or nits by parting the hair in several spots. You can use a magnifying glass and a bright light to help spot them. Because head lice can move fast it may be easier to spot the nits. Nits can look like dandruff, but you can identify them by picking up a strand of hair close to the scalp and pulling your fingernail across the area where you suspect a nit. Dandruff will come off easily, but nits will stay firmly attached to the hair, Brown explains.

FDA-approved treatments for head lice include both over-the-counter (OTC) and prescription drugs, such as Nix and Rid, in the form of shampoos, creams and lotions. “Many head lice products are not for use in children under the age of 2, so read the label carefully before using a product to make sure it is safe to use on your child,” Brown says.

Although OTC drugs are available for treatment of head lice, Brown says your health care professional may prescribe drugs approved by the FDA, such as Ulesfia (approved in 2009), Natroba (approved in 2011) or Sklice (approved in 2012).

Steps for Safe Use

Follow these steps to use any head lice treatment safely and appropriately:

  • After rinsing the product from the hair and scalp, use a fine-toothed comb or special “nit comb” to remove dead lice and nits.
  • Apply the product only to the scalp and the hair attached to the scalp—not to other body hair.
  • Before treating young children, talk with the child’s doctor or your pharmacist for recommended treatments based on a child’s age and weight.
  • Use medication exactly as directed on the label and never more often than directed unless advised by your health care professional.
  • Use treatments on children only under the direct supervision of an adult.

Heading Off Head Lice

  • Teach children to avoid head-to-head contact during play and other activities at home, school, and elsewhere (sports activities, playgrounds, slumber parties, and camps).
  • Teach children not to share clothing and supplies, such as hats, scarves, helmets, sports uniforms, towels, combs, brushes, bandanas, hair ties, and headphones.
  • Disinfest combs and brushes used by a person with head lice by soaking them in hot water (at least 130°F) for 5–10 minutes.
  • Do not lie on beds, couches, pillows, carpets, or stuffed animals that have recently been in contact with a person with head lice.
  • Clean items that have been in contact with the head of a person with lice in the 48 hours before treatment. Machine wash and dry clothing, bed linens, and other items using hot water (130°F) and a high heat drying cycle. Clothing and items that are not washable can be dry-cleaned or sealed in a plastic bag and stored for two weeks.
  • Vacuum the floor and furniture, particularly where the person with lice sat or lay. Head lice survive less than one or two days if they fall off the scalp and cannot feed.
  • Do not use insecticide sprays or fogs; they are not necessary to control head lice and can be toxic if inhaled or absorbed through the skin.
  • After finishing treatment with lice medication, check everyone in your family for lice after one week. If live lice are found, contact your health care professional.