Tag Archives: drugs

Long-term monkey tests back Oxford’s gene therapy

in.reuters.com

Long-term monkey tests back Oxford’s gene therapy

Long-term tests on monkeys using Oxford BioMedica’s gene therapy ProSavin suggest it can treat Parkinson’s disease without causing the jerky, involuntary movements associated with current drugs, researchers said on Wednesday.

Parkinson’s is caused by lack of the brain chemical dopamine. Standard treatment involves oral drugs that briefly raise dopamine levels — but levels of the chemical still remain unstable, leading to a movement disorder called dyskinesias.

By contrast, tests on macaque monkeys found the gene therapy safely restored concentrations of dopamine in the brain, corrected motor deficits and prevented dyskinesias — with no severe adverse side effects.

French researchers observed the animals for up to three and a half years in the study, after first inducing Parkinsonian syndrome by giving a neurotoxin and then treating them with gene therapy injections.

“Gene therapy-mediated dopamine replacement may be able to correct Parkinsonism in patients without the complications of dyskinesias,” Bechir Jarraya and colleagues wrote in the journal Science Translational Medicine.

Oxford BioMedica is currently conducting Phase I/II clinical trials with ProSavin and in July announced encouraging initial results.

ProSavin, which is administered directly to the striatum in the brain, delivers three genes required to convert cells that normally do not produce dopamine into cells that do.

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U.S. pre-term babies die despite medical care: study

in.reuters.com

U.S. pre-term babies die despite medical care: study

Very early pre-term babies kept alive with ventilators, chest tubes and drugs to support the heart may live a little longer than they did 10 years ago, but are just as likely to die before ever going home, U.S. researchers reported on Monday.

Their study suggests the emotionally taxing and expensive care given these tiny newborns, delivered at 22 to 24 weeks gestation, does not in the end save their lives. Babies born at 22 weeks included in the study all died as infants, regardless of care.

“This is a very difficult ethical dilemma for everyone involved,” Pamela Donohue of Johns Hopkins Childrens Center in Baltimore, who led the study, said in a telephone interview.

Most pregnancies last about 40 weeks, and babies born earlier than 37 weeks of pregnancy are considered premature.

Donohue’s team studied 160 women who gave birth at 22-24 weeks during separate two-year periods — 1993-1995 and 2001-2003.

Those who gave birth during the current decade were more likely to receive higher-level care around the time of delivery, including sonograms, antibiotics and steroids to help with fetal lung development.

After birth, their children were more likely to be put on ventilators, drugs to boost heart and blood pressure rates and to have chest tubes inserted.

Infants born in 2001-2003 lived longer on average — seven days, compared to two days in the 1990s.

But mortality rates did not fall, and the researchers urged greater discussion and further study both on intervention and the degree of suffering imposed on children, their families and healthcare providers.

Very early pre-term babies kept alive with ventilators, chest tubes and drugs to support the heart may live a little longer than they did 10 years ago, but are just as likely to die before ever going home, U.S. researchers reported on Monday.

Their study suggests the emotionally taxing and expensive care given these tiny newborns, delivered at 22 to 24 weeks gestation, does not in the end save their lives. Babies born at 22 weeks included in the study all died as infants, regardless of care.

“This is a very difficult ethical dilemma for everyone involved,” Pamela Donohue of Johns Hopkins Childrens Center in Baltimore, who led the study, said in a telephone interview.

Most pregnancies last about 40 weeks, and babies born earlier than 37 weeks of pregnancy are considered premature.

Donohue’s team studied 160 women who gave birth at 22-24 weeks during separate two-year periods — 1993-1995 and 2001-2003.

Those who gave birth during the current decade were more likely to receive higher-level care around the time of delivery, including sonograms, antibiotics and steroids to help with fetal lung development.

After birth, their children were more likely to be put on ventilators, drugs to boost heart and blood pressure rates and to have chest tubes inserted.

Infants born in 2001-2003 lived longer on average — seven days, compared to two days in the 1990s.

But mortality rates did not fall, and the researchers urged greater discussion and further study both on intervention and the degree of suffering imposed on children, their families and healthcare providers.

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Scientists develop antidote for new class of drugs

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Scientists develop antidote for new class of drugs

A new compound can quickly counteract the action of an emerging class of drugs, offering a way to reverse the drugs’ actions if a patient develops serious side effects, U.S. researchers said on Sunday.

They designed the compound to work with a new blood-thinner being developed for heart patients undergoing angioplasty to clear out blocked arteries.

Such patients need to take blood thinners to prevent blood clots during surgery, but bleeding is a common side effect.

Having an antidote on hand would make the treatments safer, said Bruce Sullenger of Duke University Medical Center, whose study was published in the journal Nature Medicine.

But instead of just reversing the effects of the blood thinner, the antidote agent appears to work against a whole new class of drugs called aptamers.

“Most drugs target proteins. The type of drugs we’re talking about are ribonucleic acids (RNAs) that target proteins,” Sullenger said in a telephone interview.

“Normally in our body we don’t have these types of molecules outside of cells,” Sullenger said.

The antidote agent takes advantage of this, targeting only nucleic acid compounds circulating freely outside the cells.

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