Improving Outcomes for Patients with NF1- Medical News

When 9-year-old Philip Moss ran out of treatment options for his neurofibroma tumors in April 2015, his doctors directed his family to NCI. NCI was leading the only treatment trial in the nation for children with tumors caused by neurofibromatosis type 1 (NF1), a genetic disorder in which painful and often disfiguring tumors of the nerves can grow on or under the skin. These tumors are generally benign, but about 10% of people with NF1 will develop a cancerous neurofibroma. There are currently no Food and Drug Administration-approved therapies for NF1.

Philip’s experience with NF1 began when he was 6 years old, with the detection, diagnosis, and removal of a tumor on his neck. “We’d never heard of NF1 before; our world changed,” said Renie Moss, Philip’s mother. Unfortunately, the tumor regrew over the next few years. After additional surgery, routine imaging scans revealed that the tumor was growing again, and, over a 6-month period, it grew from the size of a ping pong ball to the size of a tennis ball. In addition, other tumors had developed. The tumor on his neck was particularly concerning because it could obstruct his airway or cause other complications. “We felt like we were one step away from disaster,” recalled Renie.

Philip’s doctor referred them to a phase I clinical trial at the NIH Clinical Center in Bethesda, Maryland, that was testing an experimental targeted therapy called selumetinib. The trial was led by Brigitte Widemann, M.D., chief of NCI’s Pediatric Oncology Branch, and involved collaborators from Children’s Hospital of Philadelphia, Cincinnati Children’s Hospital Medical Center, and the Children’s National Health System.

Laboratory researchers had previously shown that mutations in the NF1 gene cause neurofibromas to grow through a chemical pathway inside cells called the RAS pathway. Selumetinib blocks a specific node in this pathway called MEK. In mouse models of NF1, MEK inhibitors reduced the growth of neurofibroma tumors. Other MEK inhibitors are safe and effective in other types of adult cancers. Based on these findings, researchers hypothesized that selumetinib might work in NF1 tumors in children.

In September 2015, Philip visited NCI for an intense medical evaluation, joined the trial, and began taking selumetinib twice a day. Because the drug is provided in pill form, Philip was able to return home for ongoing treatment and was monitored by his local pediatrician. He and his family learned to cope with the initial side effects of treatment, which included fatigue, headaches, and nausea. One year after joining the trial, Philip’s tumors were 36% smaller.

Although this trial was a small early-phase study, Philip was among the 71% of children whose tumor volumes decreased by more than 20%. “We saw tumor shrinkage in nearly every patient,” Brigitte remarked. “The shrinkage was measurable…something we had never seen before” among NF1 patients. These promising results are now being pursued in a phase II study.

Philip remains in the trial, and the tumor on his neck has shrunk by half. “Kids don’t ask what’s wrong with him anymore,” said Renie. “It’s meant the world to him and to our family.” Renie acknowledged the years of research that led to this trial. “Someone was planting the seeds in NF1 research so we would benefit from that work now.”

This content is provided by the National Cancer Institute (www.cancer.gov)
Syndicated Content Details:
Source URL: https://www.cancer.gov/publishedcontent/syndication/1124707.htm
Source Agency: National Cancer Institute (NCI)
Captured Date: 2018-09-24 12:10:14.0

Overcoming Acute Myelogenous Leukemia – Max’s Story



Max Harris, a twenty-nine year old patient at Mayo Clinic in Arizona, shares his experience battling a rare form of leukemia, acute myelogenous leukemia (AML). He describes undergoing a bone marrow transplant as treatment for AML. Max explains what helped him get through his treatment and how he is doing now following the bone marrow transplant.

To request an appointment, visit http://www.mayoclinic.org/departments-centers/transplant-center/sections/request-appointment/ptc-20203893?mc_id=us&utm_source=youtube&utm_medium=sm&utm_content=video&utm_campaign=mayoclinic&geo=national&placementsite=enterprise&cauid=100504

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How to Prevent a Urinary Tract Infection

UTI might be a woman’s least favorite acronym. Half of all women will get one… and it could be getting worse. Doctors are finding it harder and harder to treat urinary tract infections with antibiotics. Here’s what you can do to avoid the infection altogether.

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How the Aging Brain Affects Thinking

 

The brain controls many aspects of thinking—remembering, planning and organizing, making decisions, and much more. These cognitive abilities affect how well we do 3 older men playing chess in the parkeveryday tasks and whether we can live independently.

Some changes in thinking are common as people get older. For example, older adults may have:

  • Increased difficulty finding words and recalling names
  • More problems with multi-tasking
  • Mild decreases in the ability to pay attention

Aging may also bring positive cognitive changes. People often have more knowledge and insight from a lifetime of experiences. Research shows that older adults can still:

  • Learn new things
  • Create new memories
  • Improve vocabulary and language skills

The Older, Healthy Brain

As a person gets older, changes occur in all parts of the body, including the brain.

  • Certain parts of the brain shrink, especially those important to learning and other complex mental activities.
  • In certain brain regions, communication between neurons (nerve cells) can be reduced.
  • Blood flow in the brain may also decrease.
  • Inflammation, which occurs when the body responds to an injury or disease, may increase.

These changes in the brain can affect mental function, even in healthy older people. For example, some older adults find that they don’t do as well as younger people on complex memory or learning tests. Given enough time, though, they can do as well. There is growing evidence that the brain remains “plastic”—able to adapt to new challenges and tasks—as people age.

It is not clear why some people think well as they get older while others do not. One possible reason is “cognitive reserve,” the brain’s ability to work well even when some part of it is disrupted. People with more education seem to have more cognitive reserve than others.

Some brain changes, like those associated with Alzheimer’s disease, are NOT a normal part of aging. Talk with your healthcare provider if you are concerned.

Brain Regions

The brain is complex and has many specialized parts. For example, the two halves of the brain, called cerebral hemispheres, are responsible for intelligence.

The cerebral hemispheres have an outer layer called the cerebral cortex. This region, the brain’s “gray matter,” is where the brain processes sensory information, such as what we see and hear. The cerebral cortex also controls movement and regulates functions such as thinking, learning, and remembering.

For more information about parts of the brain, see Know Your Brain from the National Institute of Neurological Disorders and Stroke, part of the National Institutes of Health.

How Brain Cells Work

The healthy human brain contains many different types of cells. Neurons are nerve cells that process and send information throughout the brain, and from the brain to the muscles and organs of the body.

The ability of neurons to function and survive depends on three important processes:

  • Communication. When a neuron receives signals from other neurons, it generates an electrical charge. This charge travels to the synapse, a tiny gap where chemicals called neurotransmitters are released and move across to another neuron.
  • Metabolism. This process involves all chemical reactions that take place in a cell to support its survival and function. These reactions require oxygen and glucose, which are carried in blood flowing through the brain.
  • Repair, remodeling, and regeneration. Neurons live a long time—more than 100 years in humans. As a result, they must constantly maintain and repair themselves. In addition, some brain regions continue to make new neurons.

Other types of brain cells, called glial cells, play critical roles in supporting neurons. In addition, the brain has an enormous network of blood vessels. Although the brain is only 2 percent of the body’s weight, it receives 20 percent of the body’s blood supply.

See more resources about cognitive health.

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What Causes Psoriasis?

The root of this chronic condition lies beneath your skin. What does psoriasis look like, what causes it, and how do you treat it?

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Questions to Ask Before Participating in a Clinical Trial

 

The following are some questions to ask the research team when thinking about a clinical trial. Write down any questions you might have and bring your list with you when you first meet with the research team.

Older man asking a researcher questions about clinical trials

  • What treatment or tests will I have? Will they hurt?
  • What are the chances I will get the experimental treatment?
  • What are the possible risks, side effects, and benefits of the study treatment compared to my current treatment?
  • How will I know if the treatment is working?
  • How will you protect my health while I am in the study?
  • What happens if my health problem gets worse during the study?
  • How will the study affect my everyday life?
  • How long will the clinical trial last?
  • Where will the study take place? Will I have to stay in the hospital?
  • Will you provide a way for me to get to the study site if I need it?
  • Will being in the study cost me anything? If so, will I be reimbursed? Will my insurance cover my costs?
  • Can I take my regular medicines while in the trial?
  • Who will be in charge of my care while I am in the study? Will I be able to see my own doctor?
  • Will you follow up on my health after the end of the study?
  • Will you tell me the results of the study?
  • Whom do I call if I have more questions?
  • How will you keep my doctor informed about my participation in the trial?
  • Does the study compare standard and experimental treatments?
  • If I withdraw, will this affect my normal care?
  • What are the chances that I will receive a placebo?
  • What steps ensure my privacy?

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Immunotherapy and Triple Negative Breast Cancer – Mayo Clinic

A promising new study from Mayo Clinic, in conjunction with Caris Life Sciences, points to immunotherapy as a possible treatment option for patients with the difficult-to-treat triple negative breast cancer mutation. The study was presented this week at the 50th annual meeting of the American Society of Clinical Oncology in Chicago.

“This study may change our ability to treat triple negative breast cancer patients,” says Barbara Pockaj, M.D., lead investigator of the study and Mayo Clinic surgeon. “We may have signs that these patients can be treated with immunotherapy. We don’t have a lot of options for these patients and this would really expand our options.”

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Clinical Trials: Benefits, Risks, and Safety

 

You may ask yourself, “Why should I try something that researchers are not sure will work?” That is a good question. Being part of a clinical trial may have risks, but it may also have benefits.Older couple listening about the benefits, risks, and safety protections of clinical trials

Benefits of a Clinical Trial

  • You may get a new treatment for a disease before it is available to everyone.
  • You play a more active role in your own health care.
  • Researchers may provide you with medical care and more frequent health check-ups as part of your treatment.
  • You may have the chance to help others get a better treatment for their health problems in the future.
  • You may be able to get information about support groups and resources.

Risks of a Clinical Trial

  • The new treatment may cause serious side effects.
  • The new treatment may not work or it may not be better than the standard treatment.
  • You may NOT be part of the treatment group (or experimental group) that gets the new treatment—for example, a new drug or device. Instead, you may be part of the control group, which means you get the standard treatment or a no-treatment placebo.
  • The clinical trial could inconvenience you. For example, medical appointments could take a lot of time or you might be required to stay overnight or a few days in the hospital.

How Will My Safety Be Protected?

This is a very important question. The history of clinical research is not perfect. Based on many years of experience and learning, Congress has passed laws to protect study participants. Today, every clinical investigator is required to monitor and make sure that every participant is safe. These safeguards are an essential part of the research. Research abuses like the Tuskegee Syphilis Experiment, which began in 1932, before safeguards were in place, will NOT happen again.

Researchers are required to follow strict rules to make sure that participants are safe. These rules are enforced by the Federal Government. Each clinical trial also follows a careful study plan or protocol that describes what the researchers will do. The principal investigator, or head researcher, is responsible for making sure that the protocol is followed.

An Institutional Review Board, or IRB, at each study site must approve every clinical trial in the United States. The IRB is made up of doctors, scientists, and lay people, like yourself, who are dedicated to making sure that the study participants are not exposed to unnecessary risks. The people on the IRB regularly review the study and its results. They make sure that risks (or potential harm) to participants are as low as possible.

Along with the IRB, many clinical trials are closely supervised by a Data and Safety Monitoring Committee. The Committee is made up of experts in your condition who periodically look at the results of the study as it is in progress. If they find that the experimental treatment is not working or is harming participants, they will stop the trial right away.

The informed consent process also helps protect participants. Before joining a clinical trial, you will be told what to expect as a participant and all the things that might happen. For example, someone from the research team will explain possible side effects or other risks of the treatment. As part of the informed consent process, you will have a chance to ask questions about the trial. The clinical trial coordinator will be happy to answer your questions.

After getting all this information, you can think about whether or not you want to participate. If you decide to join the trial, you will be given an informed consent form to sign. By signing the form, you show that you have been told all the details and want to be part of the study. The informed consent form is NOT a contract. You can leave the trial at any time and for any reason without being judged or put in a difficult position regarding your medical care. Researchers must keep health and personal information private.

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How to Get Rid of Ear and Nose Hair

Hair in your ears and nose is a good thing. It keeps junk in the air from getting in your body. It can start getting a little crazy as we get older, though – and no one knows why exactly. If you’re tempted to trim that unruly nose and ear hair, here are some of the best tips on how to go about it.

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