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Medical Breakthroughs: Advances to Believe In (cont.)

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PPMS is the most progressive form of MS. The symptoms don’t disappear and relapse. Instead, the symptoms accumulate and get worse. Experts say Ocrevus would be the first approved treatment for PPMS.

Ocrevus is designed to target CD20-positive B cells, which are immune-system cells that are believed to be a key contributor to nerve-cell damage. This nerve-cell damage leads to disabilities for people who have MS. Based on preclinical studies, Ocrevus binds to CD20-positive surface proteins that are on B cells but aren’t on plasma cells or stem cells, which preserves and doesn’t block important functions of the immune system.

“To have anything is a big deal, and this treatment opens up the door to primary progressive multiple sclerosis to being a version of the disease you can treat,” says Tim Coetzee of NMSS. “It gives people with primary progressive multiple sclerosis significant hope.”

FDA granted to Ocrevus a priority-review designation, which is granted to medications that FDA determines to have the potential to improve the safe and effective treatment of a serious disease. Ocrevus also is the first MS medication that FDA granted a breakthrough-therapy designation.

In December 2016, New England Journal of Medicine published positive results from three Ocrevus Phase III
studies on RRMS and PPMS. The data from the studies showed consistent reductions in major markers of the disease’s activity and progression. Genentech says Ocrevus showed the capability to reduce the relapse rate of RRMS and to reduce the progression of symptoms for at least 12 weeks in PPMS.

“This is quite a remarkable result in robust clinical trials,” Peter Chin of Genentech says.

Genentech says Ocrevus will be available as soon as it’s approved, but the company tells us that it doesn’t comment on the price or insurance coverage of preapproved medications.

PAINFUL PROCESS. The 40 percent–50 percent of postmenopausal women who experience dyspareunia, which is moderate-to-severe pain when they have sex, can find relief through the use of the medication Intrarosa, which FDA approved in November 2016.

Manufactured by Endoceutics, Intrarosa is the first FDA-approved product that contains the active ingredient prasterone, which is a synthetic version of the steroid hormone dehydroepiandrosterone (DHEA) and is included in certain dietary supplements for which the efficacy and safety haven’t been established.

Dyspareunia is a symptom of vulvar and vaginal atrophy that results from menopause, when the sex hormones androgen and estrogen decrease. This hormone deficiency causes the vaginal wall to thin and lubrication to decrease.

Endoceutics researchers discovered that prasterone can create androgen and estrogen in the peripheral tissues of postmenopausal women. These androgen and estrogen hormones repair the hormone deficiency in postmenopausal women and don’t release active sex steroids elsewhere in the body. In other words, no risk exists of adverse effects in other tissues, such as those that are in the uterus.

“I think this is a new paradigm of treatment of menopause symptoms,” says Dr. Fernand Labrie of Endoceutics. “What this treatment does is give back to women what’s missing in them.”

Typically, dyspareunia is treated with estrogen that’s taken orally or applied locally in the vagina. However, despite the painful symptoms of dyspareunia, only about 3 percent of affected women seek treatment for it, because they fear estrogen-related side effects, such as heart attacks, breast cancer or stroke, according to Dr. Irwin Goldstein, who is the director of sexual medicine at Alvarado Hospital.

The safety and efficacy of Intrarosa, which is a once-daily vaginal insert, were established in two 12-week trials. Compared with a placebo, Intrarosa was shown to reduce the severity of pain that women experienced during sexual intercourse. The safety of the medication also was established in a 52-week trial. The most common adverse reactions are vaginal discharge and an abnormal Pap smear.

Intrarosa is considered to be a medical breakthrough, because Endoceutics took a medication that was available as a dietary supplement—although it wasn’t approved by FDA—and made it available in an approved suppository form, Goldstein says.

“The breakthrough is to make medical use out of something already out there for years, explain how things are happening endocrinologically and really help women who have menopausal symptoms,” Goldstein says.

DECREASING DELUSIONS. An estimated 50,000 Americans are diagnosed with Parkinson’s disease each year, and about 1 million Americans have the condition, according to FDA. Delusions or hallucinations occur in as many as 50 percent of patients who have Parkinson’s disease, FDA says.

“It’s often very frightening for patients and always disturbing for caregivers and family members,” Peter Schmidt of National Parkinson Foundation tells us.

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