New Study Reports “Curative Potential” of a Combination Therapy for Some Leukemia Patients

New Study Reports “Curative Potential” of a Combination Therapy for Some Leukemia Patients

This article was originally published on Dana-Farber Cancer Institute

Chemoimmunotherapy combined with a targeted drug given for two years has achieved undetectable minimal residual disease (MRD) for a high proportion of younger patients with chronic lymphocytic leukemia (CLL), Dana-Farber scientists report.

The phase 2 clinical trial results are so favorable that they represent a step toward “a new standard of care with curative potential for a broad population of younger, fit patients” with CLL, say the investigators, led by Matthew Davids, MD, MMSc, and Jennifer Brown, MD, PhD. “Younger” in this setting is 65 years of age or below.

The report in The Lancet Haematology describes the regimen administered to 85 patients with CLL whose median age was 55 years. The study looked at individuals whose cancer carried a mutated IGHV gene — which confers a higher likelihood of a long-lasting response to chemotherapy — and those with an unmutated IGHV gene — who rarely have durable responsesto chemotherapy.

The study examined the combined effect of two different treatment options for CLL patients. The first, chemoimmunotherapy consisting of fludarabine, cyclophosphamide, and rituximab (FCR), can improve overall survival for younger fit patients with CLL who have an IGHV mutation. The second, the targeted drug ibrutinib, can be effective for CLL patients irrespective of their IGHVstatus, but requires continuous treatment. The clinical trial examined if FCR plus time-limited ibrutinib could achieve long-lasting responses in younger, fit patients with either mutational status.

The patients, who were previously untreated, received oral ibrutinib for seven days, then up to six 28-day cycles of FCR along with continuous ibrutinib. Responders continued on maintenance ibrutinib for up to two years, and those who had undetectable MRD in bone marrow after two years were able to stop treatment.

Thirty-three percent of patients achieved the primary endpoint — a complete response with undetectable minimal residual disease in bone marrow two months after the last cycle of ibrutinib plus FCR. A best response of undetectable minimal residual disease in bone marrow was achieved by 84 percent of patients at any time during the study. “Many of the patients had deepening responses while on the two years of ibrutinib maintenance, so later converted to undetectable MRD,” explains Davids.

The researchers say the results of the trial demonstrated that the combination of chemotherapy and two years of ibrutinib maintenance leads “to the best of our knowledge, to the highest proportion of patients achieving undetectable minimal residual disease in the bone marrow ever published for any regimen used to treat this disease.” The approach, they add, could increase the potential to “functionally cure patients with mutated IGHV and, importantly, might for the first time extend this potential to patients with unmutated IGHV.”


Survival increased in chronic lymphocytic leukemia patients treated with ibrutinib and rituximab

Survival increased in chronic lymphocytic leukemia patients treated with ibrutinib and rituximab

This article was originally published on 2 minute medicine

1. Previously untreated chronic lymphocytic leukemia (CLL) patients experienced increased progression-free and overall survival when treated with ibrutinib-rituximab compared to current standard chemoimmunotherapy.

2. Rates of high grade adverse events were similar between treatment groups.

Evidence Rating Level: 1 (Excellent)  

Study Rundown: CLL is a common hematologic malignancy often treated with a combination of fludarabine, cyclophosphamide, and rituximab. While efficacious, there are notable side effects to the chemoimmunotherapy regimen. The Bruton’s tyrosine kinase (BTK) inhibitor ibrutinib has shown efficacy in patients with relapsed or refractory CLL, though how it performs as a first line treatment is not yet determined. This phase 3 trial evaluated ibrutinib-rituximab in comparison to standard chemoimmunotherapy in untreated CLL patients and found both progression-free and overall survival to be improved relative to standard therapy at almost 3 years after treatment initiation. The incidence of grade 3 or 4 adverse events was similar in the treatment groups, though infections were more common in the ibrutinib-rituximab group.

This randomized trial provides support for use of a new regimen as a first line treatment in CLL patients. Strengths of the study include its randomized design, mutational analysis of immunoglobulin heavy-chain variable region (IGHV) patients, and extensive subgroup analysis. The utility of this study is limited by its discordance with other trials evaluating ibrutinib in CLL.

Click to read the study in NEJM


Cirmtuzumab-Imbruvica Combo Trial Enrolling CLL, SLL Patients for Phase 2

Cirmtuzumab-Imbruvica Combo Trial Enrolling CLL, SLL Patients for Phase 2

This article was originally published on Lymphoma News Today

Following promising early efficacy results for a cirmtuzumabImbruvica treatment combo, a Phase 2 clinical trial has now been opened to people with chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL).

The opening of this randomized trial was based on the promising results seen in the prior Phase 1 stage, in which 100% of the first nine CLL/SLL patients responded to treatment with Oncternal Therapeutics‘ investigational therapy cirmtuzumab in combination with Imbruvica (ibrutinib).

The Phase 2 clinical trial is now open to an additional 90 patients with CLL/SLL. Participants will be randomly selected to receive the combination of cirmtuzumab plus Imbruvica, or Imbruvica alone. More information on enrollment can be found on the trial’s page here.

Imbruvica, by Janssen and AbbVie, is the standard of care for people with CLL/SLL and mantle cell lymphoma (MCL) who have received at least one prior therapy. The molecule binds permanently to the Bruton’s tyrosine kinase protein, which is key in the growth and proliferation of B-cell cancers.

Cirmtuzumab is an antibody that blocks the ROR1 receptor in tumor cells. ROR1 is rarely seen in healthy cells, but in tumor cells it works as a receptor for growth signals, helping the tumor grow and survive.

Researchers have been testing a combination of these two treatments in patients with CLL/SLL and MCL who have failed prior therapies but have not received a prior BTK inhibitor.

The CIRLL Phase 1b/2 trial (NCT03420183) was first designed in three parts. First, participants received ascending doses of cirmtuzumab alone, followed by a combination of cirmtuzumab and Imbruvica. The goal was to determine the optimal dosing regimen for further testing.

The recommended dosing regimen then was further tested in part 2 (Phase 1b). In total, 66 patients were included in these two Phase 1 parts.

Preliminary data from these parts were presented in a poster, “Phase 1/2 Trial of Cirmtuzumab and Ibrutinib: Planned Analysis of Phase 1 CLL Cohorts,” at the American Society of Clinical Oncology (ASCO) annual meeting, held in the spring in Chicago.

The analysis included the first 12 CLL/SLL patients, for whom the treatment was safe and well-tolerated. The therapy led to an objective response rate of 91.7%. Three of the 11 patients who responded had clinical or confirmed complete responses.

Researchers noted that all nine patients who completed 12 weeks of treatment in part 2 — in which participants received a recommended dose of 600 mg cirmtuzumab plus 420 mg Imbruvica — achieved a response to treatment.

Preliminary data also was presented for the first six MCL patients. One participant, whose disease had returned after a stem cell transplant, had a complete response after three months of treatment, which was maintained at least until month 11. The treatment also led to the resolution of a tumor in the chest.

The findings in CLL/SLL participants led researchers to open a third part (Phase 2), to compare the safety and efficacy of the combination versus Imbruvica alone. The main goal is to compare the percentage of patients achieving a complete response.

“We are very pleased to be opening the randomized Phase 2 portion of the CIRLL study for patients with CLL and continue to be encouraged by the interim results from the study for both patients with CLL and patients with mantle cell lymphoma,” James Breitmeyer, MD, PhD, president and CEO of Oncternal, said in a press release.


Ibrutinib, Rituximab for Patients With CLL Superior to Standard Chemoimmunotherapy

Ibrutinib, Rituximab for Patients With CLL Superior to Standard Chemoimmunotherapy

This article was originally published on The Center For Biosimilars

A combination of ibrutinib and rituximab for patients with chronic lymphocytic leukemia (CLL) is superior to current treatment, according to results of a phase 3 trial published recently.

As compared with standard chemoimmunotherapy with fludarabine, cyclophosphamide, and rituximab, the ibrutinib-rituximab combination led to longer progression-free survival, as well as overall survival. The authors also said the combination treatment is better tolerated and is less toxic that standard chemoimmunotherapy. While the current CLL treatment standard works for many patients, it is also toxic, causing severe myelosuppression and creating a risk of infections due to immunosuppression of T cells.

In the trial, 89.4% of those treated with the ibrutinib–rituximab combination had not seen their disease progress after 3 years, compared with 72.9% of those who had the traditional combination.

The study randomized a total of 529 patients, giving 354 patients the ibrutinib-rituximab combination and 175 the chemoimmunotherapy regimen. After 33.6 months, the hazard ratio (HR) for the study drug combination for PFS or death was 0.35 (95% CI, 0.22-0.56; P <.001). For OS, the study drug combo had an HR of 0.17 (95% CI, 0.05-0.54; P <.001). All patients were aged 70 years or younger.

Infections were less common in the patients taking ibrutinib–rituximab (37 patients compared with 32 patients). Both groups had a similar rate of serious adverse events. Patients taking the 2-drug combination also reported less fatigue.

The ibrutinib–rituximab combination was especially beneficial for patients without immunoglobulin heavy-chain variable region mutation, a group that historically did not respond as well to the standard treatment regimen.

Patients will likely see a new standard of care, according to the lead author.

“These results will fully usher the treatment of chronic lymphocytic leukemia into a new era,” Tait Shanafelt, MD, professor of medicine at Stanford, said in a statement. “It seems likely that, in the future, these patients will be able to forego chemotherapy altogether.”


Pharmac's dismal ranking little surprise to cancer patients

Pharmac's dismal ranking little surprise to cancer patients

Republished with permission from Graham Adams. Sourced from NOTED.co.nz

By Graham Adams
Aug 16, 2019

New Zealand has the worst record of funding new treatments among 20 OECD countries.

The news this week that New Zealand has been rated the worst among 20 OECD countries for funding modern medicines and pharmaceutical investment overall will come as a surprise to many people but perhaps not to cancer patients.

Cancer treatment gets so much media attention that patients are often acutely aware of what ground-breaking drugs would be available to them if they had the good fortune to live in other developed countries — including Australia. As a result, there is a regular parade of cancer-stricken supplicants to MPs and Parliament begging for their lives and alleging that the funding system overseen by Pharmac is deficient.

The news of our dismal performance came in a report commissioned by Medicines New Zealand, a lobby group for the pharmaceutical industry. It showed that of 304 modern medicines for a range of diseases funded internationally between 2011-17, only 17 were publicly funded in New Zealand.

Australia funded 83, the UK 174, and Germany 178.

And of 70 new cancer drugs funded across the 20 nations in that period, New Zealand had funded only six. Australia’s tally was 26.

Arthritis, asthma, diabetes and cardiovascular disease, among other conditions, fared poorly too.

For all the claims made about Pharmac’s unique approach to funding drugs much more cheaply than in other countries, it is increasingly apparent its strategy of delaying buying drugs until their prices come down is not fit for purpose — particularly when revolutionary cancer treatments are being developed at an astonishing speed.

While Pharmac’s CEO Sarah Fitt is undoubtedly correct in claiming that a “simple count of [funded] medicines won’t tell you how effective a health system is”, she is glossing over the fact that her organisation’s tardy funding decisions amount to rationing by delay for drugs that have proven to be very effective in a battery of clinical trials and medical practice.

As the report noted, the OECD average for approving new treatment from the time of a drug’s registration is 233 days. In New Zealand, it is 512.

As a cancer patient myself, I am very aware of the life-saving drugs available for free in public health systems overseas. I have chronic lymphocytic leukaemia (CLL) and one thing is starkly apparent to me. If my New Zealand-born brother who lives in Brisbane is diagnosed with the same aggressive form of the disease as me, he will be eligible for free access to two wonder drugs — ibrutinib and venetoclax — through Australia’s hospital system. However, New Zealanders with CLL like me can only obtain these drugs through a clinical trial, a drug company’s compassionate access scheme or personally paying more than $130,000 a year (which often means begging from strangers on Givealittle).

Ibrutinib and venetoclax are targeted drugs that have transformed the treatment landscape for those with CLL. Just how revolutionary ibrutinib has been is something Tauranga consultant physician Dr Neil Graham knows only too well from personal experience. Speaking to the Health select committee last week, he had to fight back tears as he recounted how ibrutinib — which he has been able to access through a compassionate access scheme — had saved his life after he had transfusion-dependent bone marrow failure as a consequence of CLL.

He told the committee that ibrutinib has put him into remission and he has been able to continue working productively for the past five years — time that otherwise would have been denied to him, and to the patients he serves.

Writer and TV presenter Clive James is undoubtedly the most famous face of the revolution in CLL drugs. In 2015, he said he was “embarrassed” to still be alive after he had predicted a year earlier that his death — from the double-whammy of chronic lymphocytic leukaemia and emphysema — was likely within months.

Such a prognosis was undoubtedly correct at the time he signalled his imminent demise in 2014 but, soon after that, he began treatment with ibrutinib. His health rallied and he is still alive and writing.

In a book of poems published in 2017 — titled for obvious reasons Injury Time — he described ibrutinib as a “little cluster-bomb of goodness”.

My own experience of coming back from death’s door is similar to that of Dr Graham and Clive James. I knew in 2015 that I was in very serious trouble when a doctor told me rather brutally: “I’ve just read your oncologist’s report. It makes for pretty grim reading, doesn’t it?”

I had been aware that my prospects weren’t very good but the oncologist clearly had been far franker writing to his colleague about my dire prospects than he had been to me in person. I understood the aggressive variant of CLL I have — due to a chromosomal aberration termed “17p deletion” — was difficult to treat but I didn’t realise just how slim the chances of conventional treatment working were.

I had naively assumed there would be something in the oncologist’s armoury that would help. In fact, I learned later that conventional chemotherapy treatment has only a five per cent chance of working for 17p deleted patients. As one haematologist told me: “Standard chemotherapy works well for some 17p patients, but the numbers are very, very small.”

By extreme good luck, I was put in a clinical trial for an experimental drug combination. It held back the progression of my disease for only six months but it made me eligible for another clinical trial of a second-generation version of ibrutinib.

Now, nearly four years later, I am still in remission.

When the drug stops working, if I’m very lucky I’ll get access to a clinical trial for venetoclax and the drug will be provided free. But if I don’t manage to be accepted onto a trial, I’ll have to bankrupt myself to stay alive — as many New Zealand cancer sufferers are doing right now.

However, if I were a citizen of Australia, as my brother is, I would be eligible to have the drug funded for a cost of around $33 a month.

Ibrutinib was first registered by Medsafe in New Zealand in 2015 and subsequently prioritised for funding by Pharmac in 2016.

Venetoclax was registered in 2017 and, last September, Pharmac’s Pharmacology and Therapeutics Advisory Committee recommended venetoclax “be funded with a high priority” for CLL patients with aggressive disease, including 17p patients.

In April this year, the recommendation was repeated as a high priority for funding.

Now, nearly a year later after the recommendation was made last September, the drug remains unfunded.

It is worth noting that Pharmac would undoubtedly be able to buy ibrutinib and venetoclax at a much lower price than its “rack rate”, which could make it not much more expensive per patient than hospital-based dialysis over a year.

Dr Graham wants Pharmac to fund ibrutinib and venetoclax for patients with aggressive CLL disease. He told NOTED: “The timeframes are simply unacceptable for treating malignant diseases generally, and CLL specifically. People are dying waiting for a decision on funding.”

There are about 2000 CLL patients in New Zealand. Dr Graham says most will respond very well to standard chemotherapy and may get remissions of up to 10 years, which is counted as the nearest thing to a cure in treating cancer.

But for the small number who don’t respond to such treatment, their disease quickly becomes a life-or-death matter.

The brutal truth, as he puts it, is that while Pharmac vacillates, people die.


Dr Neil Graham's talk - presented to the Health Select Committee

Dr Neil Graham's talk - presented to the Health Select Committee

The following talk was presented by Dr Neil Graham to the Health Select Committee on 7 August 2019 to support the submission calling for the Select Committee to strongly recommend funding of two life-saving treatments.

“My written submission was to request funding for ibrutinib and venetoclax for CLL, two examples of the new era of cancer treatment medications.

In early 2019 the Cancer at the Crossroads conference was held in Wellington, a major national conference to assess and review the status of cancer treatment in NZ.
An editorial was published in April this year in the NZMJ, reviewing the conference.

To quote from the first paragraph of that editorial:

“Cancer is the leading cause of death in Aotearoa/NZ. The number of those affected by cancer is forecast to increase by 50% in the next fifteen years…..

Our survival rates from cancer lag behind those of Australia, Canada, and Scandinavian countries, and are not improving at the same rate as elsewhere “.

This is a red flag for how we manage the commonest cause of death in our country. We are not doing well by international standards.

In the last decade or so there has been a revolution in cancer medication development internationally, of a magnitude greater, perhaps, than any one area of disease ever. It has been likened in effect to the development of antibiotics for infectious diseases almost a century ago. And it is growing exponentially. It has been predicted that cancer will become controllable, even curable, in the next decade or so.

Some NZers have had their lives saved by these new medications – two of those (Ben and I) sit in front of you today. For me, five years ago, I had transfusion-dependent bone marrow failure. I was likely to die. I was given access to ibrutinib, went into remission, and have been well ever since, working, contributing to society, paying taxes, and living a full life. Ibrutinib saved my life.

Other NZers have not been so lucky.

Most new era life-saving cancer drugs are not funded. If you are wealthy, sell your house to pay, beg in its various guises, move to Australia, or get into a drug trial, you live. If not, you die. And die of a treatable disease. And NZers ARE dying, unnecessarily, because they can’t access these life-saving medications.

Pharmac can’t, or won’t fund them. This is in contrast to Australia, UK, Canada etc, where these medications, including ibrutinib and venetoclax, are funded.

This is still not widely known nationally, but there is an increasing awareness because of this May’s march on Parliament, Blair Vining’s case publicity, and others. When this becomes widely known, it is likely that the NZ population will be critical of NZ health services for allowing this to happen. It is unlikely to be tolerated. You may remember the outcry over hepatitis C in the early 1990’s.

Two other points:
Pharmac has said recently that medications have a relatively small role in cancer treatment. Whilst overall this has some truth, it is not the case for leukaemias, where prevention and screening for early detection have no role, and surgery and radiotherapy have a minimal role. Treatment is all chemotherapy/medications.
And it is the mainstay of treatment in the context of attempted cure in the case of serious cancer in the form of metastatic disease.

Secondly, international guidelines, written by world authorities and specialist societies are not adhered to in NZ, as medications when not funded are generally not able to be used. In CLL, venetoclax, and, even more so, ibrutinib are the first choice medications in international guidelines for severe forms of CLL. But they are not used when not funded.

So people in NZ die unnecessarily.

NZ will increasingly stand out as doing badly in cancer medicine because of this lack of funding – what has been described as third world medicine in a first world country.
So, to finish, the current Pharmac model is not working for modern oncology practice.
My submission makes suggestions regarding this (5 – 9).

The Pharmac model needs to change for modern oncology.

Current Pharmac processing of applications for oncology medications takes almost two years. When you’re dying of cancer, this is woefully inadequate. A rapid access programme for cancer treatments must be developed for these patients.

I would ask/beg this committee to recommend review of Pharmac’s model in modern oncology practice, particularly funding. We spend 5% of our health budget on medications. Other similar countries spend 10 – 15%+.

Involved groups (clinicians, policy makers, Pharmac, patients, and the industry) need to get together and develop a new model to stop the disgrace of NZers dying unnecessarily.”


Health Select Committee hears pleas from survivors and sufferers

Health Select Committee hears pleas from survivors and sufferers

This story was originally sourced here from RNZ

Politicians heard emotional pleas today from cancer survivors, people with disabilities and health agencies to boost Pharmac’s funding for life saving drugs.

Petitions were presented to the Health Select Committee at Parliament – calling on medicines to be funded – including for breast, lung, ovarian cancers, along with multiple myeloma.

Emily Beswick and her daughter, Stella, travelled 500 kilometres from Cambridge to today’s select committee.

Stella has spinal muscular atrophy and uses a wheelchair.

She sat quietly at the back of the select committee room – listening – smiling from time to time.

While medication to help her condition is available overseas – here in New Zealand – it’s not.

And for Ms Beswick that’s devastating.

“We feel that all hope is gone – we have been waiting 13 years for a treatment to arrive and now that it’s finally here – it is out of reach,” she said.

Spinal muscular atrophy is a childhood version of motor neurone disease but the drugs to halt it are not funded here.

MS Beswick said her daughter’s quality of life could be improved.

Neil Graham has been in remission from chronic lymphocytic leukemia for five years.

He broke down as he told the select committee he was given compassionate access to a drug that probably saved his life.

“Since then, I’ve been working, making a useful contribution to society, paying taxes and having a full and enjoyable existence,” he said.

Mr Graham said the Pharmac model needed to change.

“Current Pharmac processing of applications for approval for cancer medications take almost two years – and if you’re dying of cancer – that’s just not acceptable,” he said.

Joy Wilkie is in remission from the blood cancer myeloma.

No new anti-myeloma medicines have been funded here in the last five years.

“When I relapse – and I know I will – our world class clinicians know what best international treatment I need to give me more years of meaningful life,” she said.

Pharmac wasn’t available for an interview and it did not did not appear at the select committee today.

In statement, it said funding decisions take time and it is aware that not everyone has time on their side.

But late this afternoon, it announced on its website a proposal to fund Kadcyla for some breast cancer patients, Alectinib for lung cancer and Ocrelizumab for multiple sclerosis from 1 December.

Meanwhile, a Cancer Action Plan is expected to be announced by Health Minister David Clark later this month.


Leukaemia advocacy group to tell Health Select Committee NZ’s current cancer drug funding scheme is a ‘national disgrace’

Leukaemia advocacy group to tell Health Select Committee NZ’s current cancer drug funding scheme is a ‘national disgrace’

Dr Neil Graham, founder and executive director of CLL Advocates NZ, a new patient support group for New Zealanders with Chronic Lymphocytic Leukaemia (CLL), will appear before the Health Select Committee today in support of their submission to fund life-saving treatments for suffers of CLL.

The Health Select Committee hearing follows the CLL Advocates NZ’s petition, signed by over 1,000 New Zealanders, fighting for funding of ibrutinib (Imbruvica) and venetoclax (Venclexta™).

Neil Graham is calling on the members of the Select Committee to strongly recommend the funding of these two life-saving treatments, to prevent the unnecessary deaths of New Zealanders with the aggressive form of this cancer.

“These treatments are widely funded in many similar countries to NZ, such as Australia, Canada, UK, and Scandinavia, where many lives have been saved. They are not funded in NZ however, and most patients in NZ are unable to afford their cost. As a result, New Zealanders are dying unnecessarily from lack of access to these medications. Those who can pay live, those who can’t die.” said Dr Graham.

Dr Graham is also asking the Select Committee to urgently review the Pharmac model which is patently unable to accommodate the new wave of drugs.

“It is a national disgrace. The current Pharmac model for cancer medication funding is no longer appropriate for providing modern life-saving cancer treatment. It needs to be changed. Funding applications to Pharmac for cancer medications currently take almost two years to be processed. For people dying of cancer, this is woefully too long” said Dr Graham.

Dr Graham, who is living with CLL, had compassionate access to one of these drugs, ibrutinib, and nearly five years later continues to work and live life to the full. He says Pharmac’s continuing refusal to fund the drug is not only a death sentence for New Zealanders with aggressive CLL, but flies in the face of international clinical opinion and best practice.

“Ibrutinib has been registered in NZ since 2015, and is funded in 44 of 45 countries of similar or lower wealth, NZ being the exception. Evidence for the effectiveness of this treatment is incontrovertible, for example overall survival for patients on ibrutinib at first line is 88%. It’s hard to imagine why the evidence that was so compelling for 44 other countries has not persuaded Pharmac to the same position,” said Dr Graham.

Dr Graham will also be joined by Dr Ben Schrader and Dr Rob Weinkove to present in support of this submission.


War in the Blood review – Love, hope and the search for a cancer cure

War in the Blood review – Love, hope and the search for a cancer cure

The feature-length film about a revolutionary treatment for leukaemia did justice to the extraordinary science and the moving stories of the patients involved Martin Pule spent his childhood dismantling radios, computers and any other devices he came across, reassembling them in alternative ways and pressing them into new services. Even allowing for childish curiosity, it speaks to a different way of conceptualising the world – as a miscellany of parts, only temporarily constituted in their current forms. Most of us are just pleased to come across a device already disposed to work.

War in the Blood (BBC Two) was about what happens when such a child grows up, becomes a doctor and turns that mindset towards the human body. “I realised that cells are machines you might take apart, and genetic code is not really that dissimilar to computer programmes.” Now at the University College London’s Cancer Institute, he became a pioneer of CAR T-cell therapy. As he explains it to the viewer, with the unhurried simplicity of someone with knowledge to share and nothing to prove, this is the process of taking a leukaemia patient’s or a donor’s T-cells (the mainstay of our immune systems; they hunt and kill cells infected with viruses) and inserting into them a piece of DNA that teaches them to recognise cancer cells, which they would otherwise ignore, and treat them with the same lethal force they do viruses. Then they return the T-cells to the patient and … well, you hope for the best.

The therapy has recently started phase-one trials – the first tests on humans. The subjects must be terminally ill patients who have run out of treatment options and are willing to undergo the intervention knowing that, of course, it may not work. So far, however, the research suggests that the approach may eventually become a cure for all cancers.

The programme follows two such patients: middle-aged former military man Graham Threader, who was diagnosed with leukaemia in August 2015, and 18-year-old Mahmoud Kayiizi, who has relapsed with the same disease after several rounds of treatment and a bone marrow transplant. Mahmoud looks both younger and much older than his years. “I just want to live,” he says from his hospital bed with a literally bone-deep weariness. “It would be heartbreaking for my mum …” Graham will be treated with his brother’s donated cells, Mahmoud with his own, both weaponised in Dr Pule’s laboratory and administered by a team led by Dr Claire Roddie.

It is a film about love, hope and the tempering of both with realism. Doctors must do it with patients, being careful neither to fan the flames of optimism with their knowledge of the studies coming out of the US (which has more data on the therapy than we do) or douse them with their appreciation of how long any path to any cure must be.

At one point, Mahmoud’s consultant, Dr Ben Carpenter, mentions the risk of something and his mother, Fatuma – her face bright with hope and faith – says: “It’s not a risk. You are saving him.” The doctor pauses, balancing duty with care, kindness with honesty, professional ethics with human impulse. “There are definite risks involved,” he replies gently. “But we know the risks of not doing this are much higher.”

Spouses must guide spouses, as Melly does for Graham, reminding him to weigh his quality of life – and his family’s life – against prolonged suffering in the name of treatment, however pioneering. Sometimes the normal dynamic reverses: patients must guide loved ones and children must do so for parents. After good initial results, Mahmoud cautioned his mother about being “too hasty”. He doesn’t want her to celebrate too early. Much, much older than his years, then.

At an hour and 40 minutes, it was a film that was able to do justice to all its parts. It twined them round each other immaculately, keeping the march of the days and the clinical results clear to the uninitiated viewer without losing sight of the intimacy between everyone involved, or distancing the viewers from Graham or Mahmoud’s experiences, which lay at the heart of everything. It did a particularly fine job of honouring the dual perspective that exists in any clinical trial – the immediate, patient-based focus on individual treatment and outcomes by the administering doctors, and the detached, long-term view the scientists developing the treatment must take – without letting the more naturally sympathetic former outweigh or diminish the latter.
There was agony and ecstasy by turns, and endless courage from Graham and Mahmoud and the people that love them. Lives and cells were taken apart and put back together in different forms. Godspeed, everyone.


March for the Right to Live

March for the Right to Live

On Thursday 7 May hundreds of cancer sufferers marched to the steps of Parliament to ask that the drugs that extend or improve the quality of lives are funded by Pharmac.

Watch the video of the march and Ben Schrader’s speech below.