health

A Variety of Medications Can Be Used to Treat Essential Tremor

Ask the Doctors by by Eve Glazier, M.D. and Elizabeth Ko, M.D
by Eve Glazier, M.D. and Elizabeth Ko, M.D
Ask the Doctors | November 17th, 2017

Dear Doctor: I know that essential tremor doesn't signify a disease, but the condition is very embarrassing and sometimes limits my activities. I've tried propranolol, but it didn't help. Any suggestions?

Dear Reader: It sounds as if you understand the basics, which is great. That will help you focus your search for new options. For those who are unfamiliar with the condition, essential tremor -- unlike the tremor with Parkinson's disease -- occurs only with activity and only when holding a sustained position. In Parkinson's, the tremor occurs at rest.

Essential tremor was formerly termed "benign essential tremor," but there's nothing benign about it. For some people, it can be debilitating, affecting fine motor skills and limiting the ability to use utensils to eat, write, shave or apply makeup. A 1994 study found that 15 to 25 percent of people with essential tremor retire early as a result, and 60 percent fear looking for job or seeking a promotion because of it.

The condition is termed "essential," because the tremor is not associated with any other disease. It occurs in 5 percent of people worldwide and is more prevalent as people age. An estimated 30 to 70 percent of people with the condition have a family member who also has it, but the exact cause is unknown.

So what can you do? First, be aware that caffeine, nicotine and withdrawal from alcohol or opiates can make the tremors worse, as can stress and anxiety. Medications such as anti-depressants, amphetamines, steroids and lithium also might make essential tremor more noticeable. Alcohol may ease tremors in the short term, but patients develop a tolerance and the alcohol becomes less effective. And, as I mentioned, withdrawal leads to greater tremors.

The medication you tried, propranolol, is a beta blocker -- a blood pressure drug long used to treat essential tremor. It can be used as needed to decrease tremors in stressful situations or taken daily to suppress tremors overall. But it didn't work for you, so let's assess other options.

The anti-seizure drug primidone is also often used to ease essential tremor. It decreases nerve excitability and is as effective as propranolol, with improvement rates of about 70 percent in most people. However, many patients feel sedated, nauseated and have difficulty with balance when taking primidone. These side effects occur about 36 percent of the time.

Another anti-seizure drug, gabapentin, can also be effective, particularly at a daily dose of 1,200 mg. The problem with gabapentin is that for many people, especially the elderly, it can lead to drowsiness. It might be best to start at doses of 100 mg three times per day and increase that if necessary and if you can tolerate it. Topiramate, another anti-seizure medication, has also shown benefit in easing tremor, but it too causes drowsiness.

Also worth considering: Botox injections. Botulinum toxin can decrease head tremors, voice tremors and tremors involving the arms, but because the toxin paralyzes muscles, it can cause weakness in the injection areas.

When essential tremor is severe, surgical techniques such as deep brain stimulation may be an option. In this technique, electrodes are placed deep in an area of the brain called the thalamus and are connected to an electrical-pulse generator implanted in the chest wall.

But before you consider such a measure, start by avoiding medications and drugs that make your tremors worse, and ask your doctor about primidone or gabapentin. Also, don't give up.

(Send your questions to askthedoctors@mednet.ucla.edu, or write: Ask the Doctors, c/o Media Relations, UCLA Health, 924 Westwood Blvd., Suite 350, Los Angeles, CA, 90095. Owing to the volume of mail, personal replies cannot be provided.)

health

Newly Approved Gene Therapy a Breakthrough in Cancer Treatment

Ask the Doctors by by Eve Glazier, M.D. and Elizabeth Ko, M.D
by Eve Glazier, M.D. and Elizabeth Ko, M.D
Ask the Doctors | November 16th, 2017

Dear Doctor: Our daughter was successfully treated for leukemia as a child, but the chemotherapy she went through was pretty brutal. Now I've read that we have the first gene therapy for cancer, which is a huge deal. How does it work? How much does it cost? Are there side effects?

Dear Reader: We're so glad that your daughter's treatment was successful. As you mention, the therapies we currently rely on can take a steep physical toll. That's part of what makes new breakthroughs in precision medicine -- which includes targeted therapy, immunotherapy and gene therapy -- so promising and so exciting.

Before we get into the details of Kymriah, the name of the treatment you're writing about, it's important to note that gene therapy is still new and evolving. It's quite expensive, it doesn't help every person every time, and it can have side effects that range from challenging to life-threatening.

Kymriah is the first gene therapy to be approved by the Food and Drug Administration. It targets B-cell acute lymphoblastic leukemia, a cancer of the blood and bone marrow that affects children and young adults. The therapy, which received FDA approval in August 2017, was developed at the University of Pennsylvania, and is licensed by the drug company Novartis.

The therapy works by altering the genetic code within a patient's own T cells, which are part of the immune system. The T cells are extracted from the patient, frozen and then shipped to a Novartis lab in New Jersey. There, the patient's cells are reprogrammed to seek out and destroy the leukemia cells. They do this by targeting a specific protein that is unique to the leukemia cells.

When the rewired T cells are returned to the patient's body, they don't only attack the cancer, but they also multiply in number. According to the university's scientists, a single engineered cell can generate 10,000 new cells, and can survive in the body for years. This benefits patients with refractory leukemia, which is when leukemia cells persist in the body even after intensive treatment.

Side effects of Kymriah can be severe. It's possible that the altered T cells will set off a positive feedback loop within the immune system, known as a cytokine cascade. Symptoms include high fever, severe fatigue, rash, lung congestion, neurological issues and a dangerous drop in blood pressure. In some cases, these can lead to death. Because of this, Kymriah therapy may only take place at designated treatment centers, which are equipped to deal with these side effects.

Customizing each individual patient's cells is expensive. At this time, treatment with Kymriah runs about $475,000. Novartis has said it won't charge patients who don't respond to the drug within a month of treatment. Some types of financial aid are also available.

If turning your own cells into a successful cancer drug sounds hard to believe, you're not alone. When the first patient treated with Kymriah in 2010 was shown to be cancer-free a month later, even the lead scientists who developed the drug had trouble accepting it. They ordered a follow-up biopsy just to be sure.

Meanwhile, the research continues. The hope is that, with FDA approval of the first gene therapy, advancing and refining the science can move forward even more rapidly.

(Send your questions to askthedoctors@mednet.ucla.edu, or write: Ask the Doctors, c/o Media Relations, UCLA Health, 924 Westwood Blvd., Suite 350, Los Angeles, CA, 90095. Owing to the volume of mail, personal replies cannot be provided.)

health

Firefighter Trying to Lose Excess Pounds in Chest and Stomach

Ask the Doctors by by Eve Glazier, M.D. and Elizabeth Ko, M.D
by Eve Glazier, M.D. and Elizabeth Ko, M.D
Ask the Doctors | November 15th, 2017

Dear Doctor: I'm a firefighter and in pretty good physical shape, even though I have Type 1 diabetes -- but my body doesn't show it. Specifically, I have flabby abs and man boobs. I walk, run, do push-ups and crunches, and I think I eat pretty well. What else can I do?

Dear Reader: First, I have some questions for you: How long has this been going on? Is the fat around the belly something new or something gradual over many years? Is the gynecomastia ("man boobs") something you have been dealing with since adolescence, or is it something you have only noticed recently?

Let us begin with gynecomastia. Gynecomastia occurs in adolescence due to a relative imbalance of estrogen and testosterone. The relatively elevated levels of estrogen compared to testosterone stimulate breast tissue in adolescent boys, leading to breast enlargement. The majority of adolescent boys will lose this breast tissue, but in 20 percent the enlarged breast tissue will persist into adulthood. In adult men, some estrogen is produced directly by the testes, but most is produced from the breakdown of testosterone and the adrenal hormone androstenedione. As in adolescent boys, adult men can develop gynecomastia if there is an imbalance between estrogen and testosterone levels.

Then, of course, there's obesity, which leads to increased fat in breast tissue. This can give the appearance of increased breast tissue, which is termed pseudo-gynecomastia. Because the fat in the breast tissue carries the enzyme that converts testosterone and androstenedione to estrogen, breast fat may actually increase estrogen levels and stimulate the formation of breast glands.

Because breast fat and the fat in your abdomen are related, the fundamental question is likely: Why are you storing fat?

First, blame the diabetes -- and the connection between the disease and your diet. As a Type 1 diabetic, you've probably been on insulin for many years. Insulin inhibits the breakdown of fat, but also stimulates the creation of fat. The higher your body's insulin level, the more your body will store fat. The more insulin you inject, the more fat you will store.

The higher your calorie intake and the higher your carbohydrate intake, the more insulin you will require to keep your blood sugar low. This will lead to a greater storage of fat. Look for foods that are low in glycemic index, meaning low in the food's ability to raise the blood sugar. Avoid high-sugar foods such as cookies, candies, cakes, sodas, ice cream and juices. These foods may no longer be part of your diet. But if they still are, and if your sincere desire is to remove the fat from your body, then eliminating these foods will help. I would also recommend limiting the amount of high-glycemic carbohydrates, such as bread, pasta, rice, corn and potatoes. Increasing fiber in your diet will also decrease your insulin requirements.

Then there's aerobic exercise. Walking, running, biking, rollerblading, hiking, swimming, using an elliptical machine -- all of these will help you lose and maintain a lower weight.

Of course, even with a good diet and exercise, you may have difficulty totally losing this unwanted fat. The important point is to be patient, diligent and more obsessive about your diet. This will decrease your insulin requirements, help control your diabetes and help you lose the fat.

(Send your questions to askthedoctors@mednet.ucla.edu, or write: Ask the Doctors, c/o Media Relations, UCLA Health, 924 Westwood Blvd., Suite 350, Los Angeles, CA, 90095. Owing to the volume of mail, personal replies cannot be provided.)

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