The hardest thing Deborah Sims has ever done was kiss young children goodbye last August and move to London – hopeful of getting on a Phase I clinical trial to potentially cure an aggressive form of chronic lymphocytic leukaemia (CLL)/small lymphocytic leukaemia (SLL).
Deborah's husband, Robert, and children Cameron (11), Marlowe (9) and Natasha (6), didn't know if their adored wife and mother would come home again.
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The Phase I trial for a combination of venetoclax (ABT-199) and obinutuzumab offers the chance of a cure to people like Deborah, who was diagnosed with blood cancer in December 2011.
Back then, just days before Christmas when Deborah took her daughter to the doctor, it was happenstance that her GP asked about her health.
She'd seen a different GP about a lump in her neck earlier that year. When she got the "all clear" from an ultrasound and blood test, Deborah got on with her busy life, forgetting about the follow-up due six weeks later.
"When my GP said, 'I see you came in six months ago, is the lump still there?', I said 'yes and there are a few more too'. She examined me, gave me a hug, and asked if I had private health insurance."
The GP told Deborah to go directly to a haematologist. The next day she had a lymph node biopsy and the following week was told she had CLL, which is rare in someone her age. She was 38.
Almost 80 per cent of all new CLL cases are diagnosed in people over the age of 60. It is rare in people under 40 and occurs more frequently in men than in women.
It came totally out of the blue and I had the worst Christmas of my life, said Deborah. "I mourned myself for two weeks and went into my shell."
Deborah went on 'watch and wait' and, expecting to have lots of time to think about treatment options, began her own in-depth research into CLL while continuing to work full-time.
"I'm a journalist and I needed information. I joined forums, subscribed to medical journals, and was referred to a number of specialists for second opinions," she said.
"It was highly likely I'd need a stem cell transplant (SCT) at some stage in the future. My youngest sister is a perfect match, so I knew early that I had that option. But the more you know about stem cell transplants, the less you want one," said Deborah, now aged 42. "I'm always doing a risk assessment to give myself the best chance of being here to care for my children."
This included paying for a genetic test that is not the standard of care in early diagnosis.
"I wanted to know how bad my markers were, and I have the type of CLL you don't want to have," said Deborah. "By October 2012 I was really sick and very tired. I couldn't schedule afternoon meetings at work."
In January 2013 Deborah started chemotherapy.
"From the first day, I wished I'd had it earlier," she said. "I had no side-effects apart from a sudden feeling of wellness."
Three months later she returned to 'watch and wait', with three-monthly bone marrow biopsies, but at six months it was evident her disease was slowly progressing and her specialist talked about when she'd have a transplant.
"I was so well and in mid-2014 was doing more research into clinical trials," said Deborah.
By her next appointment, in December 2014, she was starting to feel sick, the lumps had returned and she was losing weight. A transplant was earmarked for early-2015 and Deborah had her hair cut short in preparation.
Before the transplant was scheduled, Deborah dipped into her superannuation fund to attend a patient conference on CLL clinical trials in the US in April 2015. While there she had a consultation with Professor Thomas Kipps, an international CLL expert.
He said 'you should not have a transplant. We are on the verge of a cure. We just have to work out what the best drug is. You need something to buy yourself some time'.
The next day, it was fortuitous that one of the guest speakers, Dr John Gribben from the UK, sat next to Deborah. He told her about a clinical trial in London that he believed would be the best possible treatment for her at that stage.
It was with venetoclax, which ironically was developed in Melbourne. The Leukaemia Foundation's National Research Program helped to fund early work on the precursor to venetoclax by Dr Kylie Mason and her team at the Walter and Eliza Hall Institute.
The only way Deborah could access venetoclax in Australia was through a Phase III randomised trial that meant a 50% chance of getting the new drug, as the other arm of the trial was chemo.
"I'd already had chemo, so I couldn't take that risk," said Deborah.
She asked one of her Australian specialists – "if you were me, what would you do?" and he answered – "I'd get on a plane to London".
"It knocked me that the best trial was 10,000 miles away," said Deborah, who used her super again to go the UK.
I want to help the Leukaemia Foundation provide information to Australians like Deborah who are being forced to battle through a complex maze to gain access to life-saving medicines.
Deborah had previously lived in London for 10 years, but getting on the trial was not for the faint hearted.
"I had to be sick enough to go on the trial, well enough to tolerate a Phase I trial, I had to go back to work there, get a national health scheme number, and a referral to Barts (St Bartholomew's Hospital). There was a lot of paperwork and no guarantee I'd get on the trial," Deborah explained.
"There were only 40 places in the world for this trial – two at Barts and none in Australia, which was very frustrating. According to my risk assessment, this could buy me a long remission and possibly a cure."
She went back to work at the BBC as a freelance reporter and started writing a blog.
When I heard the great news I was on the trial, I was so excited, it felt like I'd won the lottery, she said.
In November, Robert, Cameron, Marlowe, and Natasha arrived in London for nine weeks to coincide with Deborah starting what they call 'mummy's magic medicine'.
After they returned to Australia in late January, Deborah found it incredibly difficult to cope.
The good news to follow this though was that Deborah had no side-effects from the treatment and a CT scan in February showed she was in partial remission.
"My blood work is completely fantastic. I'm working again and going out. It's given me my life back again," she said.
The length of the trial is three years and to stay on the trial Deborah is committed to her monthly day-long appointment in London. When she gets to no detectable disease she can go off the treatment, although may need to stay on venetoclax on an ongoing basis. Early last month (April 2016), Deborah got the fantastic news that she is in complete remission with 0.0345% of her lymphocytes being CLL, after four months on venetoclax.
"This is fantastic – I'm very happy," she said. "By October, I'm hoping to have no detectable disease (molecular remission) so I can stay on the trial. I'm loathe to come off the drug. It could be the Glivec** of CLL."
Deborah has applied to have venetoclax dispensed in Australia. In April, venetoclax was approved as a second line therapy for 17(del) CLL by the FDA in the US. It is not expected to be TGA approved and PBS listed here for at least two years, when the results of clinical trials are known and it is proven to be both effective and safe.
In the meantime, Deborah will continue to travel to London every three weeks for her treatment as part of a treatment odyssey that has cost her $400,000 in lost income, flights, accommodation and living expenses – so far.
Raising awareness of confusing blood cancer medicine maze
A Leukaemia Foundation delegation met with Federal Parliamentarians in March 2016 to highlight the plight of many Australians like Deborah who are being forced to battle through a complex maze to gain access to life-saving medicines.
"The lengths people go to access non-subsidised medicines – from fundraising to going overseas to join a clinical trial – are well documented," said Anthony Steele, the Leukaemia Foundation's Head of Support Services.
"We know that the Federal Government is doing its utmost to ensure new medicines are made available. There are good reasons why medicines are not listed on the PBS if they are still under investigation. However, the system does need to evolve to match the rapidly evolving world of anti-cancer therapy. Solutions are urgently needed."
Read the full story about the Leukaemia Foundation's parliamentary delegation to launch our 'Accessing non-PBS Funded Blood Cancer Drugs in Australia' booklet here.
Donate here to support our ongoing work in supporting and advocating for Australians with blood cancer like Deborah.
** a daily tablet used to treat people with chronic myeloid leukaemia (CML)
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