Moving forward, a surprise and some very good news!

It's been another busy week in which we have moved forward on a number of fronts....

...On the home front, the new garden shed has been assembled, moved into place, anchored, made weathertight and kitted out with shelves and boxes and is now providing a home for the garden stuff that's been stored under a tarpaulin since the old shed came down in the spring.  

The new paving has been chosen, ordered and paid for, ready to be laid by the contractor at the end of the month, together with the railway sleepers that will form the new raised beds. A decision has been made on the replacement deck boards, so we can now calculate quantities and place an order.  

Still things to do, but the garden makeover is moving forward on schedule.

...On the family front, we visited Steve's mum in Bristol on Saturday afternoon and our nephew Nick, partner Kate and their daughter Esme on Sunday morning (where we were given some lovely pictures drawn by Esme!)

By Sunday afternoon, the car was loaded up and we were on the road from Bristol to London, helping our son take some heavy, bulky items to his new flat share. Then back on the road to Oxford, under the huge super moon on Sunday evening.  A round trip of about 300 miles over the course of two days, but worth it to spend some time with the family!  

....On the health front, Steve has felt good and has been relatively active recently, because of everything that's been happening.  By this time next week, we will have a clearer idea of what's going on inside, and find out whether there are any clinical trials recruiting locally which might be suitable for him.  Following an assessment a fortnight ago, I now have an appointment to see a spinal specialist at the end of the month to find out more about treatment options.  

Regular readers of the blog may have picked up that the dragonfly has become a symbol for many in the meso community on Facebook.  Imagine my surprise and delight when one arrived in the post in the form of a dragonfly brooch from a "mystery" sender.  

But I've guessed...Thank you Jan, such a lovely thought!

As everyone in the mesothelioma community knows, there are many heartbreaking stories out there on a daily basis. We feel for our friends going through treatment, like Lou in Australia; those awaiting treatment like Amanda's Ray; those like Tess, who have decided not to have any more treatment, at least for the time being; those facing the end of life, and those who have to stand by and watch their loved ones decline, feeling helpless...When bad news happens daily, good news is a great boost to all of us....

And, oh boy, there is some good news to share and celebrate! Fellow meso blogger Mavis, who was diagnosed around the same time as Steve, has been taking part in a Phase 1 clinical trial of a new immunotherapy drug MK3475 at the Royal Marsden Hospital.  Yesterday she found out the results of the first scan taken since starting the trial: shrinkage on all tumours!  The meso community on Facebook is buzzing with delight and relief....could this be is the silver bullet that will stop mesothelioma in its tracks?  Far too early to jump to those sort of conclusions, but it does look very promising!

Here is a link to Merk's page listing a range of trials involving MK3475 Pembrolizumab worldwide, in case you want to follow it up.  I believe this is the one that Mavis is taking part in Study of Pembrolizumab (MK-3475 in participants with advanced solid tumours (MK-3475-028/KEYNOTE-28) NB It is only open to patients whose tumours have NOT responded to current therapy.

Sadly for Steve, people with a clinical history of autoimmune disease, such as arthritis, are excluded from the trial so taking this immunotherapy drug is not an option for him, unless it's designated as a Promising Innovative Medicine (PIM) under the Early Access to Medicines Scheme.  Let's hope so.

Mesothelioma - making sense of recent developments

April 2014 is shaping up to be a significant month for the mesothelioma community and other people with rare and life threatening diseases.  

With announcements about new regulations, draft legislation, new schemes and consultations coming thick and fast, it's easy to get confused or overlook something.  I thought this would be a good time - between Steve's end of trial scan and hearing the results next week - to take stock of all these changes, join the dots and look at the bigger picture.  

So here goes....

Clinical Trials Regulation in Europe
For mesothelioma and other diseases for which there is currently no cure, the way forward has to be through research and clinical trials.  The problem at the moment is that there is no legal requirement to register clinical trials or publish results.  

On 2 April, the European Parliament voted to adopt a new Clinical Trials Regulation.  This will require, amongst other things,

• All new trials to be registered on a publicly accessible EU clinical trials register before they begin
• A summary of the results to be published within a year of the trial's completion, including a summary understandable to non-medical people
• Clinical Study Reports to be publicly available 

There are often not enough patients in one country alone to develop new or improved treatments for rare diseases. The new regulation will make it easier to conduct cross-border, multinational trials within the EU by cutting red tape and simplifying the current rules by having:

• One straightforward authorisation procedure for all EU Member States, resulting in one single assessment outcome
• Simplified reporting procedures, with one report for all Member States
• Applying the same rules about protection of subjects to trials performed outside the EU where they are to be applied to trials within Member States
• A lighter touch regulation for "low intervention trials", for example trials comparing medicines which are already authorised  

The new regulation will also strengthen transparency and, in the process, avoid the redundancy and duplication which can happen when trials are not conducted and reported on in the public domain.  

As far as I can see, this is all good stuff.  There are only two downsides.  Firstly, although approved, the regulation won't come into effect until mid-2016, when the EU portal and database (currently under development) ought to be ready.  

Secondly, it is only concerned with new trials. It does not address the existing problem that we do not have full reporting on all trials for the medicines currently being used. Evidence is routinely and legally withheld, making it difficult to make an informed decision abut which treatment is best for a particular individual.  This harms not only patients but the pharmaceutical industry's reputation.  Shame on you!  

To find out more about All Trials which has campaigned for this change CLICK HERE . There are other useful links on this page.

The Early Access to Medicines Scheme (EAMS) 

This UK government scheme came into effect on 7 April. 

EAMS will see doctors working with patients to make innovative and promising drugs available as soon the Medicines and Healthcare Products Regulatory Agency - the UK’s regulator - has signalled that the benefits outweigh the risks following an initial scientific assessment.  Severely ill patients with life-threatening and seriously debilitating conditions will be offered the lifeline of trying ground-breaking new medicines years before they would normally reach them.

The scheme will be funded by pharmaceutical companies which develop innovative treatments, meaning patients will benefit from world-class breakthroughs at no cost to the NHS. Once drug manufacturers have received a scientific opinion from the Medicines and Healthcare products Regulatory Agency, doctors will work with patients to prescribe the drugs when appropriate.

In return, the companies will be able to gain experience of their medicines being used in the NHS and work closely with regulators to look at the value of the drugs, gaining guidance and advice much earlier in the regulatory process.

As a result, the process of patient access will be speeded up and new drugs could be made available to patients months or sometimes years before the treatment is licensed.

The scheme involves a 2 stage approach:

• Stage 1: Promising Innovative Medicines (PIM) Designation - Based on early clinical data (for example, a Phase 2 trial) where a drug shows early promise, the maker can apply for a PIM.  This can happen several years before the full clinical trial is complete and the drug licensed
• Stage 2: Early Access to Medicines Scientific Opinion - This will describe the benefits and risks of the medicine and support doctors make a decision as to whether the drug might be suitable to help a patient with a life threatening disease like mesothelioma, for whom there are no other treatment options

Full details of EAMs are now on the website of the Medicines and Healthcare products Regulation Agency, the body responsible for regulating the scheme.  You can read about it in more detail by CLICKING HERE .  The scheme is now up and running, and the deadline for submitting the first applications for a Scientific Opinion is 5 May.  

As far as we are concerned, this is wonderful news.  There have been a number of promising clinical trials involving immunotherapy drugs which Steve is unable to take part in because his medical history of autoimmune disease (arthritis) which might skew the drug trial results.  However, if one of more of these drugs achieved a PIM designation and the Scientific Option indicated that in his case, the benefits would outweigh the risk, that would open up treatment options for him in future, which can only be a good thing!

The Medical Innovation Bill aka Saatchi Bill

One of the things which holds back the medical profession from taking risks associated with new, innovative treatments is fear of litigation if it all goes horribly wrong.  This is something that the Medical Innovation Bill seeks to address, while still protecting the patient's interests.  

The law as it currently stands dictates that patients are only offered standard treatments.  However innovation departs from standard treatment.  The Bill supports doctors who endeavour to act in the best interests of the patient without fear of litigation.  

It deters irresponsible experimentation but encourages a much needed attitude change in favour of innovation in the provision of patent care. 

The Health Minister has said the Government will support the draft Bill but ONLY if it has public support.  You can read more about the Bill and add your support by CLICKING HERE!  The consultation closes on 25 April.  

Please, please, please support this Bill.  It's an important piece of the jigsaw.  

The EAMS scheme described above will only work to patients' benefit if the medical profession is bold enough to take advantage of it and prescribe promising, but unlicensed, drugs to patients who need a lifeline, having exhausted all other treatment options.  Doctors are far more likely to be prepared to make such decisions if the fear of litigation is removed.  

The Bill proposed by Maurice Saatchi (who lost his wife to cancer) would give the medical profession the reassurance it needs that innovation won't lead to litigation if decisions are made with care, the best interests of the patient at heart, and based on the best information available.  

Moreover, it will cover innovative medicine of all types - not just drugs, but also surgery and other medical procedures, and therefore benefit a wide range of patients who might otherwise die before today's innovation becomes tomorrow's gold star treatment or cure.

Mesothelioma Priority Setting Partnership (PSP)
With avenues for research, clinical trials and innovative treatment opening up as a result of the developments described above, it is of the utmost importance that patients, their families and carers have an input into setting priorities for research into the disease.  The Mesothelioma PSP has been consulting widely with this group and with the healthcare professionals who treat them.  

The consultation is due to close 30 April!  If you fall into this group and have yet to give your views on what research would make a difference to you and what researchers should be working on, then please go the the PSP website and complete the questionnaire before it's too late.  This is your chance.  Use it!  

To access the survey, please CLICK HERE!  

So what does it mean to us?
If you managed to get this far, well done you - and thank you!  

As I've been writing this, it's helped me gather my thoughts on our situation....

Steve had his end-of-trial scan on Tuesday.  Next Monday, we will find out the results of the scan and whether taking the combination of vandetanib and selumetanib in the VanSel Early Phase clinical trial has stopped his cancer growing or spreading.  If the side effects of treatment are anything to go by, something must be happening, but whether it's happening to the cancer cells as well as normal healthy cells we will have to wait until Monday to find out.

We are both aware that these drugs are primarily aimed at people with non-small cell lung cancer.  It remains to be seen whether they act the same way on mesothelioma.  However, if you don't test, you will never know.   So if it doesn't work out, at least we will not be plagued by thoughts of "what if...."

When Steve started the drug trial back in early February, we had no idea that the developments summarised above were even a twinkle in someone's eye.  The fact that they have reached these various stages by the end of the drug trial brings me hope that Steve will have other treatment options in the not too distant future, if the Vansel combination doesn't work for him.  Those of you in a similar position will know how good that feels.  

Take away treatment options, and you are likely to take away hope - at least until a cure is found.  And it will be found, one day.  

A huge and heartfelt hank you to all those who are working on it! 

VanSel1 drug trial Cycle 2 Days 16-21

One more dose of selumetanib tonight and Steve will have finished the third week of cycle 2 of the VanSel1 clinical drug trial.  Just one more week to go before we know whether it's all been worthwhile.

When asked how he's doing these days, Steve's stock answer is that he has felt better, but it's bearable.  Which is true up to a point.  None of the horrible metallic taste associated with standard chemo.  Not a lot of nausea.  No tingly fingers or toes (peripheral neuropathy). No need for blood transfusions or problems with low white blood cell counts, compromising his immune system.

But the fatigue is still there - he has little energy, so has been taking it easy most of the time, over the last 10 weeks. Daily diarrhoea takes its toll over a sustained period. He literally feels drained, although taking imodium is usually an effective way to manage this side effect.

There have been stomach cramps and times when his appetite has all but disappeared - one day, he couldn't even finish the banana he thought he might be able to eat for one meal....Then there are times when he suddenly feels the need to eat or become nauseous.  All completely unpredictable, so no point in spending a lot of time cooking elaborate meals.  We need things which can be prepared very quickly when needed, and can be eaten in small batches. Tapas to the rescue!

However, the once awful skin rash is not very noticeable now; the cough appears to be easing off a little and yesterday, for the first time in ages, Steve ate three meals - most unusual, but encouraging.  In addition, this weekend he has seemed brighter in himself.  

So... the side effects are bearable, but not the sort of thing you would wish to live with on a daily basis, given a choice. Likewise, the knock-on impact of these side effects which make it difficult to go out, visit family and friends and travel any distance would not make for the most enjoyable or fulfilling life on a long term basis.  

But if it means that the cancer stayed stable, would you be prepared to live with those sort of restrictions, and manage those side effects, if it gave you extra time? Tough choice....but not one we have to face quite yet, and maybe not at all...We'll just have to wait and see. 

Without doubt, Steve's skin condition has improved slowly since taking a break in treatment then resuming with a lower dose. Who knows, perhaps the worst of the remaining side effects may ease off over the next week in a similar manner, albeit taking a little longer....  

On the bright side, we cheered on Oxford to a convincing win in the Boat Race today!  

And being tied to the house has meant that more progress has been made with spring cleaning, redecorating and reclaiming the garden from the wilderness that has taken over over the last five years since diagnosis, when home maintenance and gardening were the last thing on our minds.  

Busy week ahead.  More of that in a future post. I'm ending today with good news.  The European Parliament has voted in favour of new legislation which will require all clinical trials to be registered and results published on a publicly accessible data base.  What a pity we have to wait until 2016 before it comes into effect.  

Still, it's another piece in the jigsaw puzzle of beneficial changes that are on the cards, including the (Saatchi) Medical Innovation Bill (if you haven't given your support yet please follow THIS LINK and sign the petition!) and the Early Access to Medicines Scheme (EAMS) , which starts this month! 

Early Access to Medicines Scheme and Medical Innovations (Saatchi) Bill

Two exciting developments of particular interest to people with mesothelioma and other life threatening conditions for which there is no cure at the moment, are the Early Access to Medicines Scheme and the Medical Innovation Bill, aka The Saatchi Bill. These were both discussed in the House of Commons last Tuesday (25 March).  

Earl Howe is the Minister responsible for quality at the Department of Health who spoke at the meeting.  The full text of his peech is set out below.  Worth reading!  

I am very pleased to be here today to talk about this important subject. I do, of course, recognise the concerns raised by the other speakers before me regarding access to new and promising medicines for these terrible conditions.

I would like to assure those present that our priority is to ensure that patients, including those with rare and life threatening or limiting conditions, have access to new and effective treatments on terms that represent value to the NHS and the taxpayer.

Early Access to Medicines Scheme

I would like to spend a few minutes setting out the aims of the scheme, which I hope you will agree is an important development.

Through the scheme, the Medicines and Healthcare products Regulatory Agency will provide a scientific opinion on promising new, unlicensed or off-label medicines to treat, diagnose or prevent life threatening or seriously debilitating conditions which do not have adequate treatment options.

Our hope is that the scheme, which will operate within the current regulatory structure, could give patients with these conditions access much sooner to medicines that represent a significant advance in their area of unmet need.

MHRA is responsible for managing the scientific aspects of the scheme, which will follow a two-step process.

Step one includes giving a new medicine a promising innovative medicines designation.

This will provide an early indication that a product may be a possible candidate for early access, based on the available clinical data. The designation will be issued following an MHRA scientific meeting and could be given several years before the product is licensed.

Companies who wish to move to step two must hold a promising innovative medicines designation and provide further relevant data on their product’s quality, safety and efficacy.

At step two, the MHRA will produce a scientific opinion describing the benefits and risks of the medicine, based on information submitted by the applicant after sufficient data have been gathered from the patients who will benefit from the medicine.

The trigger for an Early Access to Medicines scientific opinion does not necessarily have to be the submission of a dossier for marketing authorisation application, but the availability of a sufficiently compelling case based on the total data and evidence collected to date as assessed by the MHRA.

This is, of course, conditional on data from the development process of the product which indicates that the benefit:risk profile of the medicine is positive.

The scientific opinion will be made available on the MHRA’s website to assist clinicians and patients in making treatment decisions, and to support informed consent by patients to the risks and benefits of the product.

The scheme will be launched and ready to receive applications in April 2014. I understand that full details, together with guidance, will be published on the MHRA website in due course.

Conclusion

This is an exciting initiative and just one way in which the Government is supportive of improving access to new medicines.

We are also consulting on a draft Medical Innovation Bill which aims to encourage responsible innovation in certain circumstances, and to discourage irresponsible innovation. The consultation paper was published on 27 February and the consultation period runs until 25 April. I would encourage anyone with views on the proposals to respond to the consultation.

We continue to support the existing incentives offered at EU level to encourage the development of medicines for small numbers of patients (‘Orphan’ medicines), and the development of the Commission’s plans for adaptive licensing, including their launch of the EMA adaptive licensing pilot on 19 March.

In giving patients access to the next generation of medicines before they are licensed, I hope you will agree that this scheme could help seriously ill patients in areas of unmet clinical need to have earlier access to new and effective medicines.

The Early Access to Medicines Scheme (EAMS)

Announcement from the UK's Medicines and Healthcare Products Regulatory Agency: 

"The Early Access to Medicines Scheme (EAMS) aims to give patients with life threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorisation when there is a defined unmet medical need.

Under the scheme, Medicines and Healthcare products Regulatory Agency (MHRA), will give a scientific opinion on a new medicine or indication that has demonstrated a positive risk/benefit balance.

The scheme will begin taking applications from companies from early April 2014. MHRA will provide advice on the scheme when it is launched and we look forward to receiving applications from then. 

The scheme is voluntary and the opinion from MHRA does not replace the normal licensing procedures for medicines. 

MHRA is responsible for the scientific aspects of the scheme and the scientific opinion will be provided after a two-step evaluation process:

• step 1, the promising innovative medicines (PIM) designation
• step 2, the early access to medicines scientific opinion.

Promising innovative medicines (PIM) designation

The PIM designation will give an indication that a product may be eligible for the Early Access to Medicines Scheme (based on early clinical data). The PIM designation will be issued after an MHRA scientific meeting and could be given several years before the product is licensed.

Companies who want to move to step 2 must hold a PIM designation and provide further relevant data on quality, safety and efficacy.

Early access to medicines scientific opinion

The scientific opinion will describe the benefits and risks of the medicine, based on the information submitted to MHRA by an applicant after sufficient data have been gathered from the patients who will benefit from the medicine. The opinion will support the prescriber and patient to make a decision on whether to use the medicine before its licence is approved".

Announcing the scheme on 14 March, Dr Ian Hudson, Chief Executive of the Medicines and Healthcare Products Regulatory Agency said:

“We are delighted that the Early Access to Medicines Scheme will be launched in April 2014. The scheme is intended to enable patient access to medicines for treatment of life threatening or seriously debilitating conditions where there is an unmet need. This is a major new development in medicines policy in the UK.

“The scheme offers a way by which unlicensed medicines can be available to patients before approval of a licence to benefit public health. It will also enable companies to gain additional knowledge and experience of these medicines in clinical use.”

That's got to be good news for people with mesothelioma, especially those who have had the standard treatment of Alimta and cisplatin (or carboplatin) and whose cancer starts to grow again.  When you each the stage of second, third, fourth line treatment (or more), few people have the time or the option to take part in a clinical trial, especially where there is a 50-50 chance of getting a placebo, rather than the trial drug.

I do hope that the drug companies take up this option if the initial results in a clinical trial look promising.