This story was first published in CLL News May 2016

The hardest thing Deborah Sims has ever done was kiss her three young children goodbye last August [2015] and move to London – hopeful of getting on a clinical trial.

“I didn’t know if I’d come back,” said Deborah from Melbourne last month [March 2016], during her first trip home to Australia since starting the trial in November [2015].

The Phase I trial for a combination of venetoclax (Venclexta®, formerly known as ABT-199)] and obinutuzumab was considered a chance of a cure for the aggressive form of CLL/SLL she was diagnosed with in December 2011.

Back then, just days before Christmas, when Deborah took her two-year old 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” for 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.”

Then she told Deborah to go directly to an appointment with 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 at the time.

“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.

“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. I’m doing everything I can to avoid having one,” said Deborah, then 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.”

One of her three consulting specialists talked about clinical trials and some of the drugs in the pipeline, in particular ibrutinib and ABT-199 (venetoclax).

“I did everything I could to avoid chemo and even looked at importing bendamustine from Germany.

“By October 2012 I was really sick and very tired. I couldn’t schedule afternoon meetings at work,” said Deborah.

In January 2013 she started the FCR chemo regimen.

“From the first day, I wished I’d had it (FCR) earlier. I had no side-effects apart from a sudden feeling of wellness and I responded really well.”

Three months later, even though Deborah still had a trace of residual CLL in her bone marrow it was suggested that she stop FCR after three (of six) cycles, to avoid the extra toxicity from continuing the treatment.

She returned to watch and wait, with three-monthly bone marrow biopsies, but at six months it was evident she was relapsing 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, whose relapse was very slow.

Her appointments were extended to six months, but 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, but she needed more treatment to reduce the bulky disease prior to the SCT.

Motivated by wealthy businessman, Ron Walker’s success with Keytruda® in treating his melanoma, Deborah dipped into her superannuation fund to attend a patient conference on CLL clinical trials in the U.S. in April last year [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, at the conference, it was fortuitous that one of the guest speakers, Dr John Gribben from the UK, sat next to Deborah. They chatted and he told her about a clinical trial in London that would be the best possible treatment for her at that stage.

It was with ABT-199 [venetoclax], which ironically was developed in Melbourne. The only way Deborah could access ABT-199 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. She had previously lived in London for 10 years, and had friends there, 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,” said Deborah.

She went back to work at the BBC as a freelance reporter and started writing a blog: www.abtandme.com.

“When I heard the great news I was on the trial, I was so excited, it felt like I’d won the lottery.”

The first six weeks of the trial were “full on” with lots of monitoring and blood tests as the dose of ABT-199 was gradually increased until she reached the full daily dose (four tablets) in January. The obinutuzumab was given in monthly infusions that finished after six months (in April).

She has had no side-effects from the combination 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.”

Deborah’s children, Cameron, 11, Marlowe, nine, and Natasha, six, and her husband, Robert, arrived in London for nine weeks in November to coincide with her starting what they call ‘mummy’s magic medicine’.

“They left at the end of January and by mid-February I wasn’t coping.”

Since January, Deborah has only needed to go to Barts one day a month. The length of the trial is three years and to stay on the trial Deborah is committed to her monthly appointment in London.

“When I get to no detectable disease I can go off the treatment although I may need to stay on ABT-199 on an ongoing basis.”

In February, she came home to Australia for three weeks. She returned to London in mid-March and is still working for the BBC. She has another flight home booked in April and while she’s here, she’ll have a bone marrow test to see if she has achieved complete remission after six months on the trial.

“I’m hoping it will show there’s very little disease left and that I’ll stay on ABT-199. I’m loathed to come off the drug. It could be the Glivec** of CLL.”

While on the trial, ABT-199 is free, but Deborah’s treatment odyssey has cost her $400,000 in lost income, flights, accommodation and living expenses. She has applied to have the drug dispensed in Australia.

* stage IV CLL, unmutated with del 6q, which is associated with more rapid disease progression.

** a daily tablet used to treat people with CML.