Clinical Trials and Medical Blog

This article is from Biosingularity:

One of the nation’s pre-eminent genetic researchers, Eric Hoffman, PhD, of Children’s Research Institute at Children’s National Medical Center, predicts that in relatively short order, medicine’s next innovation–individualized molecular therapies–will have the unprecedented ability to treat muscular dystrophies, and other disorders.

In the latest edition of the New England Journal of Medicine, Dr. Hoffman posits that the results of a small clinical trial involving a new treatment for Duchenne muscular dystrophy provides a proof-of-principle for personalized molecular medicine. Practical implementation of the ‘exon-skipping’ approach described in the co-published report of vanDeutekom et al. will require advances in systemic administration of large amounts of customized DNA-like drugs, and proof that long-term delivery is not toxic. However, these advances are likely to come in short order, with the oversight and regulations of the FDA critical for appropriate labeling and marketing of such personalized molecular target drugs.

Though this particular treatment remains in its early stages, within the foreseeable future the now-standard Phase I, II, and III pathway to drug approvals may need to be re-evaluated.

How can DNA-like drugs specific to a single patient’s mutation go through the existing approval process” Are the current standards of rodent and monkey toxicity studies relevant and appropriate for DNA-like drugs, when the animals do not have the same DNA target (or off-target) sequences as humans” These and other questions are certain to pose exciting challenges to both the approval and marketing processes of drugs.

Read the full article here

From Pulse Today

Government experts are calling for more children to receive the pneumococcal vaccine, as new figures show its introduction is already saving lives.

A year after the rollout of the pneumococcal vaccine programme, 300 immunised children have avoided serious illnesses such as meningitis, septicaemia and severe pneumonia, a Department of Health analysis shows.

The department estimates that out of these 300 children, 17 would have died, and around 30 would have been left with permanent disabilities.

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GPs have achieved record flu vaccine uptake for October in this year’s campaign as practices demonstrated what they are capable of in the absence of delivery delays.

Figures for the first month of this year’s campaign show 54.7% of over 65s were vaccinated, up from 34.2% last year and 48.9% in 2005/6 – the campaign with the highest overall uptake to date.

The impressive performance casts doubts on the Government’s desire to reform the way GPs order flu vaccines, indicating the blame for previous uptake problems lay firmly with delays in manufacture and distribution.

Uptake in at-risk individuals has nearly doubled since last year’s delay-hit campaign, stands at 28.5%, compared with 14.5% in 2006/7 and 25.0% in 2005/6.

The figures reflect the efforts of GPs to restore the momentum of the seasonal flu vaccination campaign after the problems of recent years.

Dr George Kassianos, RCGP immunisation spokesman, said: ‘The flu vaccine uptake for October is higher than the previous two years because of the hard work of the primary care teams.

‘Practices are well organised, vaccines are available to them, call systems are in place and many practices have carried out mass open-door immunisation sessions.’

 Read the full article here

A phase II trial on axitinib, a new experimental drug for treating patients with cytokine-refractory, metastatic kidney cancer who have a poor response to more traditional drugs has shown promising results according to a new study published in the The Lancet Oncology.

In an intention to treat analysis, the study produced the following results:

• Of the 52 patients, 2 responded completely and 21 partially.
• This equated to an objective response rate of 44.2 per cent.
• The median response rate was 23.0 months (range 4.2 to 29.8 months).
• But 12 of 23 initial responders progressed with response durations of 4.2 to 26.5 months.
• Also, 22 patients showed stable disease for more than 8 weeks, with 13 of them for 24 weeks or more.
• 4 patients had early disease progression, 3 had missing response data.
• Median time to progression was 15.7 months (range 0.03 to 31.5 months).
• Median overall survival was 29·9 months (range 2·4 to 35·8 months).
• Adverse events linked to treatment included diarrhoea, hypertension, fatigue, nausea, and hoarseness.
• High blood pressure was found in 30 patients.
• Of these, all but 8 responded to blood pressure treatment. 7 of the 8 had a history of high blood pressure at enrollment.

Read more here

A team at Newcastle University are beginning a trial this week that could give hope to thousands of sufferers of muscular dystrophy.

A new treatment called molecular patch therapy has been developed which has the potential to give boys born with Duchenne Muscular Dystrophy (DMD) the chance to preserve their muscle function and live into old age.

In a world first, the antisense oligonucleotide (AO) patches work by masking the faulty part of the gene (exon 51) and allowing shortened but functional proteins to be formed.

DMD affects one in 3,500 boys and is caused by reduced production of dystrophin protein - vital for muscle function. The progression of the condition is so severe that untreated boys lose the ability to walk by their early teens are only expected to live into their twenties.

From the Newcastle University website:

“This is a major breakthrough for the treatment of DMD,” said Professor Francesco Muntoni, head of the neuromuscular unit at Imperial College Healthcare NHS Trust, “as conventional gene therapy approach for this disorder has proven to be problematic. Animal work has suggested that the molecular patch has worked well and showed a very significant restoration in dystrophin function”.

Professor Kate Bushby and Professor Volker Straub from the Institute of Human Genetics at Newcastle University will be recruiting nine young men from across the country and preparing them for the treatment. The trial participants will need to be aged 12 – 17 with DMD and they will have the molecular patch administered by injection into a small muscle in the foot. Subject to the trial’s success, there is already a plan to proceed with another trial to deliver the molecular patch under the skin, so that all muscles in the body could be treated.

Professor Bushby of Newcastle University said ”This is very important work to be involved with and a reflection of the long -standing interest in muscular disease here in Newcastle. We’re one of only three specialist centres in the country and this is the first step to what might be a gene based therapy for Duchenne Muscular Dystrophy.”

Read the full story here

Zyban contain the active ingredient “bupropion hydrochloride” and is one of the newest drugs on the market that can help smokers in their battle to give up smoking. Rather than being a nicotine replacement therapy product, it acts in the brain. It does not replace the nicotine that is obtained from smoking a cigarette with but uses bupropion to affect neurotransmitters in the brain stored in nerve cells and are involved in transmitting messages.

Bupropion prevents two of these neurotransmitters, noradrenaline and dopamine, from being reabsorbed back into the nerve cells. Noradrenaline and dopamine are responsible for moderating mood and various other processes in the brain. It is thought that bupropion helps people to quit smoking by increasing the amount of noradrenaline and dopamine free to act in the brain.

Zyban is the trade name of the drug “bupropion” and it was approved as a stop smoking aid in 1997. The same drug, bupropion, was previously and still is known as an anti-depressant and is manufactured under the name of Wellbutrin. The manufacturing of Zyban came about after smokers, who were taking the anti-depressant medication Wellbutrin, declared that they no longer had such a desire or craving to smoke cigarettes. Further research, based upon these claims, found that bupropion was in fact a highly effective tool in helping people to stop smoking.

The starting dose is one tablet once a day for six days, increasing on day seven to one tablet twice a day. There should be an interval of at least eight hours between doses. It is best to take your first dose when you get up in the morning and your second dose at least eight hours later. Try to avoid taking your second dose at bedtime, as difficulty sleeping (insomnia) is a common side effect of the medicine.

Personally I’ve tried everything to give up smoking and I’ve still not tried Zyban, it would have to be the very last thing I try as it does appear to have several side effects I’d rather not risk.

Alan Johnson, the Health Secretary, has initiated a task force to investigate the possibility of making every UK citizen have “Presumed Consent” unless they specifically opt out or object.

This move follows on from a report published by Prof. Sir Liam Donaldson earlier this year, however Dr. Harry Brown from Scotland rejected the proposal and there are patient groups in England who are against the plans.

This move will be reviewed by “The Organ Donation Taskforce”, set up in 2006, who will weight up the ethical issues involved which includes giving the family of the deceased individual the final say in the matter.

In Britain around 8,000 people require a transplant, but only 3,000 are carried out leaving 4,000 people every year to die waiting for an organ. Volunteer schemes fall far short of the requirements and only a quarter of the UK populations have signed up to it, especially low in Black and Asia donors even though they are more likely to require organs due to a higher rate of diabetes.

At the moment due to the Human Tissue Act 2004, it’s forbidden to remove the organs or tissue without the consent of the dead person or his/her relatives, which is crazy when you look at how many people are dying from the lack of donors.

While there is still over one person dying every day from the lack of organ donors, this move can only prove positive. It makes sense to put the onus on the donors to opt out rather than in.

WebMD has a good article on the benefits and risks of clinical trials. It’s a good place to start if you’re thinking of volunteering.

People participate in a clinical trial for many reasons. Healthy people may join clinical trials to contribute to medical science and improve medical knowledge and care for others. Clinical trials also may provide free medical care during the trial, which appeals to some people.

Definitely worth a read: www.webmd.com

The US is considering allowing terminally ill patients to trial and use new drugs that have passed Phase I testing but are not yet approved for general use.

Obviously this engenders strong feelings on both parts - Is it ethical? Should terminally ill patients be allowed to choose? Will their illness prejudice any decisions they make?

An interesting article in two parts at www.bmj.com

Yes, says Emil J Freireich, patients with limited life expectancy should have the same privileges as all individuals in a free society and be able to judge risks for themselves.

No, says Dean Gesme, the allure of promising new drugs engenders false hope, and using experimental drugs outside trials will damage both individuals and science.

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New government proposals will give grants to pregnant women to encourage them to eat more healthily.

The “health in pregnancy” grant will be formally announced by the health secretary Alan Johnson on Wednesday in the government’s first major speech on health policy.

Women are expected to be given a £200 grant and advice on eating healthily from 2009 with the aim being to reduce the inequality between rich and poor families across England and Wales.

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