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Medical Breakthroughs: Advances to Believe In (cont.)

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That’s why Schmidt says FDA’s April 2016 approval of Acadia Pharmaceuticals’ Nuplazid (pimavanserin), which is the first antipsychotic medication that’s approved to treat the delusions and hallucinations that are associated with Parkinson’s disease, is an important breakthrough.

FRIGHTENING. Delusions or hallucinations occur in as many as 50 percent of patients who have Parkinson’s disease, FDA says.

FRIGHTENING. Delusions or hallucinations occur in as many as 50 percent of patients who have Parkinson’s disease, FDA says.


Parkinson’s disease typically occurs in people who are at least age 60 when brain cells that produce dopamine become impaired or die. Parkinson’s medications are designed to relieve the disease’s motor symptoms (such as stiffness and trouble walking) by increasing the brain’s dopamine levels. Unfortunately, increased dopamine levels cause delusions and hallucinations. Before Nuplazid was approved, doctors typically prescribed FDA-approved antipsychotics for bipolar disorder and schizophrenia to treat Parkinson’s-related delusions and hallucinations.

Nuplazid, which a patient takes as a tablet once per day, binds to serotonin receptors and reduces their stimulation. Other antipsychotic treatments for Parkinson’s disease, such as Haldol and olanzapine, bind to and stimulate serotonin receptors. In other words, they might reduce hallucinations, but they also worsen the primary motor symptoms of Parkinson’s disease, such as difficulty walking.

In a clinical trial, Nuplazid was found to decrease the frequency and severity of delusions and hallucinations but not to worsen the primary motor symptoms of Parkinson’s disease. The most common side effects that were reported are confusion, nausea, and swollen ankles, legs and feet because of the accumulation of excessive tissue fluid.

Nuplazid isn’t approved to treat patients who have dementia-related psychosis that’s unrelated to Parkinson’s disease psychosis. Nuplazid costs $1,950 for a 30-day supply and generally is covered by insurance, Acadia Pharmaceuticals says.

TREAT CANCER. Researchers at Dana-Farber Cancer Institute announced in November 2016 that they identified genomic changes that might predict how testicular cancer develops. The changes might help researchers to understand why the majority of testicular-cancer tumors are highly curable through chemotherapy, unlike most solid tumors. Researchers believe that this will help to explain the chemosensitivity of other types of cancerous tumors and the evolution of chemoresistance in other germ-cell tumors. In other words, the findings might unlock new ways to treat all cancer patients.

“[This study] is a good lesson in doing a deep dive and a large genomic dissection of rare cancers in a whole subset of patients who don’t do well and still need help,” says Dr. Eliezer Van Allen of the Dana-Farber research team. “The real breakthrough is setting the stage for what will be a big breakthrough of truly understanding how this process occurs.”

American Cancer Society (ACS) estimates that in 2017, 8,850 new cases of testicular cancer that have germ-cell tumors will occur in the United States. Of those cases, 410 men will die, ACS says. Most of the tumors are highly sensitive to chemotherapy, so at least 80 percent of patients who have germ-cell tumors are cured. However, a significant number of tumors become chemotherapy-resistant, and about 10 percent of patients who have chemotherapy-resistant germ-cell tumors that spread to other parts of their body die as a result.

Previous studies of testicular-tumor genomes revealed mutations and chromosomal damage but didn’t identify specific alterations that were linked to chemosensitivity or chemoresistance. Dana-Farber researchers found a type of chromosomal damage that might be linked to the development of chemosensitive germ-cell tumors. Those tumors have a gene that directs cells to make a tumor-suppressing protein. In most types of cancer, this gene is mutated or lost.

The researchers also found that, unlike most types of cancer, testicular tumor cells are poised to self-destruct by apoptosis, which is the body’s quality-control process that eliminates abnormal and unneeded cells. Many cancers block apoptosis.

“It’s a little ways away,” Van Allen says of finding a treatment for these cancers.

Memorial Sloan Kettering Cancer Center researchers say the genomic profiling of patients who have advanced germ-cell tumors could help researchers to discover cancer treatments.

“It’s up to us now to figure out how to better identify more-aggressive tumors and work on therapies to treat them,” says Dr. Joshua Meeks, who is an assistant professor of urology and obstetrics at Northwestern University Feinberg School of Medicine.

Lea Radick has reported on health-care issues for 11 years. Her work has appeared in American Health & Drug Benefits, American Libraries magazine and The Washington Post’s Express.

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