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Update Tracy (leukapheresis procedure on June 12, 2023)
Today June 18, 2023
Thank you to our #GBMDay sponsors. Because of your generous support, we are able to shine a light on the urgent, unmet needs of the #glioblastoma (#GBM) community: https://t.co/T2XEwjPSqr pic.twitter.com/CfrNjFodXM
— National Brain Tumor Society (@NBTStweets) July 17, 2023
Thanks for the info, Baxers. Much appreciated.
Professor Linda Liau’s Lectures in Taiwan.
July 18,2023
The Department of Neurological Institute
Taichung Veterans General Hospital,Taiwan.
Professor Linda Liau : “Immunotherapy for malignant brain tumor”.
July 25,2023
College of Medicine
National Cheng Kung University
No. 1 University Rd, Tainan, Taiwan
Special Lecture by Faculty of Medicine, July 25, 2023, Linda M. Liau, MD, PhD, MBA - "Current advances in brain tumor immunotherapy and vaccines"
Recently,
June 17, 2023
Taiwan Top Science Student Project
GAIA STEM Lectures Series 24 – Linda Liau- Personalized Immunotherapy for Brain Cancer.
In February 2019, Shenell Malloy was enjoying a Super Bowl weekend getaway with friends in Cabo San Lucas in Mexico. She’d been feeling brief instances of numbness and tingling on one side of her body, but she didn’t pay much attention at first. After all, she was healthy and only 36, a loving wife and mother with a busy and active life. However, when she arrived in San Diego, she had a full-blown seizure.
After many brain scans, MRIs, and blood work, doctors confirmed an unidentified brain abnormality. She got a second opinion from the UCLA Neurology Department, where her surgeon, the brilliant Dr. Linda Liau, performed a six-hour operation the very next day. That surgery gave us the answer: Shenell had a brain tumor, a grade 4 glioblastoma.
ATLnsider,
the DCVax-L (ATL-DC) along with poly-ICLC + Pembrolizumab (Keytruda) combination trial is a window of opportunity trial.
https://classic.clinicaltrials.gov/ct2/show/NCT04201873
Here is an interesting read:
The Future Glioblastoma Clinical Trials Landscape: Early Phase 0, Window of Opportunity, and Adaptive Phase I-III Studies.
https://link.springer.com/article/10.1007/s11912-023-01433-1
#Glioblastoma is a very aggressive brain tumor that can cause life-threatening effects.
— UCLA Neurology (@UCLANeurology) July 12, 2023
Drs. PhiOanh Leia Nghiemphu and Timothy Cloughesy discuss clinical research trial findings for glioblastoma treatment.https://t.co/HpjeFKXhdg pic.twitter.com/gRegY4cxmR
It’s really a trial that’s a window of opportunity trial without changes in dosing like a traditional Phase 1 trial. It’s not a dose finding trial and also not an efficacy trial. Please realize the “phase” here doesn’t matter. Focus on the purpose of the trial.
— PhiOanh "Leia" NghiemPhu, MD (@Leia_N_MD) July 6, 2023
Excellent thoughts hyperopia!
Here is an interesting read:
https://www.frontiersin.org/articles/10.3389/fimmu.2023.1226841/full
July 11, 2023
Bringing function to the forefront of cell therapy: how do we demonstrate potency?
Mark W. Lowdell and Ben Weil
Help for Hannah
1 day ago
·
My dear #nwbo $NWBO friends.
— Johnny Boy Zoots (@JohnnyBoyZoots) July 8, 2023
We believe. We support if we can.
I can. I did.
I've #justdonated to save Hannah's Life with the DC-Vax which is not available on the NHS Please share/Donate/Send Love x. Donate on @justgiving and help raise £150000 https://t.co/UQ9KMVYTsU
hyperopia,
Thank you as always for your incredible work for this board.
Great summary, thanks Chiugray!
Thanks abeta for sharing Hannah's story!
Keeping everything crossed that they can raise the money for her treatment!
https://www.justgiving.com/crowdfunding/gail-iredale?utm_term=zxmewna3z&fbclid=IwAR1so6BL1BGU4a5K9LJuaALIOodkp0qAqQ259eCNibkIxE_1H7yIbScNJRI
$NWBO people. Hannah has GBM4 & is now post resection with her tumour stored correctly by Ashkan but the crux of the matter is they need to raise £150k to begin getting the DCVax made. They are at £30k so far...
— Tommy Bax 🇬🇧 🇹🇼 (@TommyBaxendale) July 7, 2023
Dig deep people, her life is on the line!https://t.co/b2EvJbKSdk
Happy Birthday Springbok80.I wish you have not just a great and wonderful day but also a wonderful year throughout.
Happy Birthday beartrap!! I wish your day to be sprinkled with lots of laughter, joy, and fun. May have a great day!
We welcome the #NHSWorkforcePlan & the emphasis it places on; specialty training in cancer, investing in CNS & embracing innovations in tech. But there’s more to do if we’re to truly address the workforce crisis for those with #braintumours: https://t.co/hFqKjPiSNn
— The Brain Tumour Charity (@BrainTumourOrg) July 3, 2023
vator,
Brain cancer charity OurBrainBank is to publish a white paper as part of a ‘Use Your Brains!’ campaign to demand greater recognition of, and support for, what it calls the neglected cancer glioblastoma (GBM).
The paper, to be published on July 5, is an in-depth study into the state of care and therapies available to people diagnosed with GBM in the UK. It concludes that five vital steps should be taken immediately to improve the situation for UK GBM patients and to ensure they do not miss out on the benefits of the personalised cancer treatment revolution built on recent advances in genomics, the science of cancer genetics.
The campaign is being spearheaded by Nicola Nuttall, mother of Laura – who tragically passed away from the disease in May aged just 23 – and journalist Richard Clemmow, the widower of former BBC executive Jana Bennett, who died in January 2022, aged 66. Both are UK trustees of the charity.
The white paper will be launched at the British Neuro-Oncology Society conference in Manchester next week.
From the Whitepaper (27 pages- interesting read IMO):
Laura Nuttall went on to meet a host of stars after writing a bucket list including two meetings with former First Lady Michelle Obama.
https://www.facebook.com/photo/?fbid=10163117565320160&set=pb.645890159.-2207520000.
Glioblastoma multiforme is a brain cancer nicknamed "The Terminator" because its so deadly. The Sunday Times talks to @shitscaredmum about her new fight launching this week for improved NHS care for the cancer that took her daughter's life 1/4https://t.co/TkyNQH85Y0
— Shaun Lintern (@ShaunLintern) July 2, 2023
‘They hope we’ll go away’: mother still battling for bespoke brain tumour care.
Shaun Lintern, Health Editor
Sunday July 02 2023, The Sunday Times
When her daughter Laura was diagnosed with a brain tumour, Nicola Nuttall did what any mother would do and began searching the internet for everything she could find out about what doctors had told her was a glioblastoma multiforme.
She learnt it had its own nickname — the Terminator — because it was so deadly.
“I was sure there was something we could do. Then you start to do the research and you realise there’s nothing. You really don’t want to look up the cancer your daughter has just been diagnosed with and find out that’s what its nickname is.”
Laura died in May, aged 23, five years after her diagnosis, and now her mother is leading a campaign by the charity OurBrainBank to demand better NHS care for glioblastoma (GBM) patients.
This week Nuttall, 53, will travel to the British Neuro-oncology Society conference in Manchester with other families to unveil a report by the charity that reveals the NHS is failing to hit government targets to genetically sequence rare tumours to help deliver personalised care.
In the UK, about 3,200 cases of glioblastoma are diagnosed a year. Although it is classed as a rare disease, it is the most common brain tumour in adults. The median survival time from diagnosis is 14 months.
GBM can have multiple mutations but without genome sequencing there is no chance of personalised treatments. Sufferers are obliged to raise hundreds of thousands of pounds to have it carried out abroad.
According to data from NHS England, only 61 patients with GBM had received whole genome sequencing by January this year, just 1.3 per cent of patients.
In 2018, Matt Hancock, then health secretary, promised five million genomes would be mapped within five years, but OurBrainBank says that by December last year only 556,000 tests for cancer had been performed. So far only 10 per cent of the targeted genome sequences has been completed.
Many hospitals lack the facilities to freeze the tumour tissue for genetic testing and the charity is demanding this be made available in all trusts, along with universal genome sequencing. It is also demanding better information on the NHS website and an increase in drug trials to treat GBM.
“We feel we’re making huge progress with cancer and we are at the forefront of research, and making huge leaps forward on things like breast cancer,” Nuttall said. “But nothing has changed in decades in the treatment for this cancer. It’s surgery, radiotherapy and one type of chemotherapy. That’s it. Personalised medicine is the only way to treat cancers as complex as this. You need to understand what drives the cancer to grow. Our children deserve better and this cancer destroys families.”
Last month, Baroness McDonagh, the first female general secretary of the Labour Party, died aged 61 from glioblastoma. In March, her sister, the Labour MP Siobhain McDonagh, said the treatment of GBM patients by the NHS was “a complete and utter national scandal”. Baroness Jowell, another Labour peer, died in 2018 from the same cancer.
Nuttall said only about £10 million of the £40 million pledged by the government had been spent. She said: “It really just feels like we’re just a cancer in the corner that they hope will go away and be quiet, but w It comes as the National Cancer Research Institute announced it is to close. Established in 2001, it aims to improve cancer outcomes by identifying gaps in research and bringing together clinicians, patients, industry and charities to target funding. It also runs the UK cancer research database of clinical trials. Lawrence Young, professor of molecular oncology at the University of Warwick, said: “This is shocking news that will significantly impact cancer research and the development of new treatments in the UK.”
Before her death, Laura completed a bucket list of achievements including meeting Michelle Obama and commanding a Royal Navy warship. Nuttall is now channelling her daughter’s activism, saying: “The best legacy I could ever give to Laura is that no one else has to go through what we’ve been through.”
https://www.ourbrainbank.org/use-your-brains/
It's not a race to the bottom, but if you're diagnosed with breast cancer there's an 88% chance you'll be alive in 5 yrs, with #glioblastoma it's 5%
— shitscaredmum (@shitscaredmum) July 2, 2023
It's our mission to change that. @ourbrainbank
Grieving mother demands improvements in ‘neglected’ cancer https://t.co/0EwxL2nDaj
Grieving mother demands improvements in ‘neglected’ cancer
Nicola Nuttall’s daughter Laura died in May, aged 23.
Ella Pickover
6 hours ago
The cancer which led to the death of campaigner Laura Nuttall is being “neglected” and treated as “the cancer in the corner”, her mother has said.
Nicola Nuttall said her daughter would be “marching on the House of Commons” if she was still alive, demanding improvements for the care of people with glioblastoma multiforme.
Her daughter died in May, aged just 23, after years of campaigning and raising awareness of the rare and aggressive brain cancer.
Mrs Nuttall called for a number of improvements in care, including genome sequencing of brain tumours and more information for those affected by the disease.
She is backing a new report by the charity OurBrainBank which states that glioblastoma is being “neglected” by the healthcare system and that it is not getting the same level of research as other cancers.
She said that families should be able to access more information about the disease to be included on the NHS website – currently just listed as a “malignant tumour” on nhs.uk – and that newly diagnosed people are coming to her when they cannot find information online.
“I don’t have any medical training but they’re coming to me and asking me for advice and support, and I’m happy to give it, but it’s really poor that somebody that runs an indoor play centre is seen as an expert in glioblastoma,” she told the PA news agency.
“Every time I get an email that says ‘I’m sorry for contacting you, could you give me some advice’, my heart just sinks because it’s another family that are looking for a miracle where there really aren’t any.”
The report also calls for whole genome sequencing for everybody that has a brain tumour removed in surgery.
“When that tumour is removed the actual tissue that’s removed is like gold dust,” Mrs Nuttall said.
“You can use it for genomic sequencing so you can identify exactly what that tumour was made up of.
“And in the long term gives (scientists) the option of using frozen tissue to make a vaccine which is looking like quite promising treatments for glioblastoma.”
She added: “So it’s really important that something that could be seen as just medical waste is actually treated properly and used as a resource going forward.
“And once we know what the mutations are, then there’s the option to develop novel drug treatments. But at the moment there is one kind of chemotherapy and that’s all that’s given to people with glioblastoma, and it may well work for a period of time for some people, but we really do need personalised medicine as is available in other kinds of cancers.
“It just feels like this one is really neglected.
“I know it’s a hard one, I know the prognosis is really low, but we can change that by putting some effort into it.
“And you know, if you go on the NHS website and put ‘glioblastoma’ in, you’ll just get a page on malignant tumours, you won’t actually find any specific information on it.
“So it really does feel like it’s kind of the cancer in the corner that nobody is putting any effort into.”
She said that she will keep campaigning because “Laura would be expecting us to continue the fight”.
Mrs Nuttall added: “There’s been no improvement in decades for this cancer.
“We’re just going to have to be more noisy about it because unless we do that nothing is going to change.”
Ms Nuttall, from Barrowford, Lancashire, was diagnosed with brain cancer in autumn 2018 following a routine eye test.
She was initially told by medics that she would have 12 months to live and dropped out of university in London and underwent an operation to remove the largest of eight tumours.
But in September 2019 she was able to resume her studies closer to home at the University of Manchester and completed a number of items on her bucket list before she died, including completing her degree, meeting Michelle Obama, piloting a Royal Navy patrol boat and presenting a television weather bulletin.
In August 2021 comedian Peter Kay performed two sell-out gigs in Manchester to raise money for Ms Nuttall and Hollywood A-lister Sandra Bullock sent her a touching video message just days before she died in May.
The OurBrainBank report sets out the state of care and therapies available to people diagnosed with glioblastoma in the UK.
The document, which will be presented to the British Neuro-Oncology Society conference in Manchester later this week, says that although there have been many cancer advances in recent years, little progress has been made on glioblastoma, with the standard of care in the NHS unchanged for almost 20 years.
The authors set out the steps which should be taken to improve the situation for patients including genome sequencing for brain tumours removed during surgery, better information on glioblastomas on NHS and Government websites and more clinical trials.
The campaign is also being backed by journalist Richard Clemmow, the widower of former BBC executive Jana Bennett, who died in January 2022, aged 66.
He said: “Glioblastoma has been consistently overlooked by those in charge of the NHS at all levels. It’s a scandal.
“Whilst huge advances have been made in treatment of more common cancers, the situation for people diagnosed with glioblastoma hasn’t changed in 20 years.
We should hold politicians and those in healthcare to account. We all need to use our brains and focus our minds on the report’s findings immediately.”
A Department of Health and Social Care spokesperson said: “Brain cancer can be a devastating disease – which is why we’ve specifically allocated £40 million for research in this area, on top of £1 billion a year for wider health research.
“We’ve invested in every suitable application made and the funding will continue to be available for further studies to develop new treatments and therapies for brain tumours.
“To encourage further successful applications, we are investing in infrastructure, workshops for researchers and training for clinicians.”
For more information visit: https://www.ourbrainbank.org/use-your-brains/
Iclight,
"Owain will live the life he deserves,"
"What I am about to share is no short of a miracle. Thank you to everyone who has supported us through Owain's stage 4 brain tumour diagnoses."
IVs suggest checkpoint inhibitors in use too
Owain James
Everything is going well, just wanted to provided a quick update; Owain has had 3 DC Vaccines, we’re now doing everything to increase his immune response including Intravenous Vit C, Helleborous, Artemisinin and an injection of Mistletoe therapy 3x per week. We are working hard to educate ourselves on how to block all the cancer pathways, with improved diet, exercise, supplements and off label drugs. Attached is a photo of Owains daily night routine of drugs and supplements. Owain has a scan at the end of the month so fingers crossed!!
I agree. Incredible news, so happy for Owain and his family.
https://m.facebook.com/photo.php?fbid=10222648862706471&id=1119976259&set=a.3026995510127&eav=AfbcbQq8ViL6tNcknhSLXCY3gCwyqx816Bzhc9166j3CZDe0A0VukDxu5YGnXNc8Twg&paipv=0&source=48
In September 2022, out of the blue, Owain James was diagnosed with a 14cm-wide (5.5in) grade 4 brain tumour (GBM).
Surgeons quickly operated and managed to remove 50% of it,
He has been told by his doctor that two kinds of treatment could extend his life - one called DCVax-L and one called Optune.
Owain chose the DCVax-L treatment option.
Unfortunately, because Owain didn’t have much fresh frozen tumour saved, only 3 vaccines could be made
On Feb. 8 Owain had his first of 3 vaccines.
Owain has had his first of 3 vaccines with the fantastic Professor Ashkan 👏💪🏽#DCVAX #fundsforowain
— FundsForOwain (@FundsForOwain) February 8, 2023
Please if you can donate anything at all to help Owain get this treatment the link is here… https://t.co/7uVByWubS2 pic.twitter.com/72pNRN82no
"Minus a few cancerous cells the tumour is now a cyst.
This is really rare to happen".
An update from Owain: So, I got my scan results back and it shows there’s no active cancer cells and the cyst has shrunk and is shrinking! The best possible result in such a short space of time???? I would like to thank my incredibly amazing wife who I owe everything to as she pretty much saved my life. The amazing Kate who comes to the house every week for my IV treatments & Emma & Harry at Cryocube who look after me when having hyperbaric oxygen therapy. Onwards and Upwards and love you all.
BRITISH NEURO-ONCOLOGY SOCIETY
ANNUAL MEETING 2023
WEDNESDAY 5TH JULY 2023
LUNCHTIME EXHIBITOR SHOWCASE – NORTHWEST BIOTHERAPEUTICS
13.00 – 13.30
DCVax®-L - Mechanism of action and immunological effects
Dr. Marnix Bosch
Chief Technical Officer
Northwest Biotherapeutics Inc.
Flipper44,
I was wrong.
Yesterday I asked LL if it's possible to manufacture an autologous dendritic cell vaccine (DCVax-L) by using cryopreserved apheresis material.
Here is LL’s reply
June 29, 2023
Hi xxxxx,
It is potentially possible, but the viability and potency of the dendritic cells may not be as good as when they are fresh.
LL
The final product is tested for identity, purity, potency, and stability against pre-established release criteria for the product. And then as I just said, there are a number of additional tests (sterility, endotoxin, mycoplasma) to ensure that the product contains no contaminants. All of this testing takes about a month or more.
Hi xxxxx,
It is potentially possible, but the viability and potency of the dendritic cells may not be as good as when they are fresh.
LL
I agree Doc logic.
Here is a shocking, ignorant and dispiriting reply.
My sister died of of a glioblastoma brain tumour on Saturday
— Siobhain McDonagh (@Siobhain_Mc) June 27, 2023
3,200 diagnosed with the tumour yearly
I asked @RCPhysicians why oncology students get no training in brain tumours... Margaret doesn't matter and neither do the other 3,200
Read the patronising and outrageous reply pic.twitter.com/Zh1ymkGcZ6
Baroness Margaret McDonagh, the first female general secretary of the Labour Party, has died aged 61.
— BBC Woman's Hour (@BBCWomansHour) June 29, 2023
Her sister Siobhain McDonagh, the Labour MP for Mitcham and Morden, joins @HayleyHassall to share her tribute to the woman she loved.
Listen now ⬇️
Thank you for having me on @BBCWomansHour
— Siobhain McDonagh (@Siobhain_Mc) June 29, 2023
If you are able, I would be so grateful if you could donate in Margaret's memory to Glioblastoma research and drug trials run by the phenomenal Dr Paul Mulholland @UCLHCharity #UCLH
Let's make a change happen👇https://t.co/nvqssiGbaU
Siobhain McDonagh
In memory of Baroness Margaret McDonagh
We are fundraising in Margaret's memory for University College London Hospitals Charity UK because we want to fund research into Glioblastoma.
Story
Margaret was diagnosed with a Glioblastoma brain tumour on November 29th 2021 after collapsing at a party.
An extraordinary woman, she was the youngest and the first ever female General Secretary of the Labour Party. Her ambition was to run two General Elections in 1997 and 2001 which resulted in two full terms of a Labour Government, the only time that this has ever happened n Labours history.
A highly successful business woman she went on the set up the Pipeline with her business partner Lorna Fitzsimmons, an organisation dedicated to advancing women's careers both in business and the public sector.
She tragically passed away on Saturday June 24th two days before he sixty second birthday. The treatment of Glioblastoma brain tumour has not changed in twenty years and its outcomes have not improved in 30 years, there are very few drug trials and no mandatory training for medical oncologists in brain tumours.
We are fundraising in order to allow Dr Paul Mulholland, Consultant in Medical Oncology and Honorary Associate Professor at the University College Hospital to continue his research and drug trials, so that there may be better outcomes for patients in the future. 3200 people will be diagnosed this year with a Glioblastoma, and without this work nothing will change.
Paul, was Margaret's doctor and is Europe's leading medical oncologist in Glioblastoma, he has the skills, the scientific support and the laboratory to have a real change in making progress in the treatment of this terrible condition.
Every penny raised in Margaret's memory will be spent in his department on the research and drug trials that will help other patients in the future.
Glioblastoma Research Group | UCL Cancer Institute - UCL – University College London
Thank you in advance for your generosity.
Siobhain
hyperopia,
I don’t think the current manufacturing process still uses fresh monocytes.
Note the dates of the collection of the monocytes (June 12) and availability of the vaccine(mid/end July).
Manufacturing process DCVax-L= 8 days.
Quality control = 2 days
Tracy:
Tracy Verblis did her leukapheresis 2 weeks ago. She still needs help funding her treatment for DCVax-L.
— rj (@sharpie510) June 27, 2023
"So close. So very close."
🫶https://t.co/yJFkOKBUKt$nwbo #dcvax #glioblastoma #ForAllSolidTumors #cancermoonshothttps://t.co/nwoNKq3YOd pic.twitter.com/zPEb5Ljkln
Back home in Ireland on Tuesday and that will be it until I get the confirmation that the Vaccine is manufactured. This will most likely be mid/end of July.
Nicola Nuttall
“The impact her story has had on people around the world has staggered me. I’ve had messages from Australia, the US, Singapore, Costa Rica and Brazil . When I made it public that Laura had died, 10 million people saw the post. That astonishes me. It’s incredible to think that, at 23, her life touched so many people.”
Tomorrow we'll celebrate Laura's life
— shitscaredmum (@shitscaredmum) June 26, 2023
There'll be tears, but hopefully some laughs too & you're more than welcome to join us on the livestream from 17:30
You can also leave a message on this page & we're so touched by those left already -thank you so much https://t.co/qgE6lLa1Ve
We are deeply saddened by the death of @liamdbergin, three years after his glioblastoma (GBM) diagnosis 💔 Liam was a tireless campaigner, fundraiser and friend to us. His family, including his wife and three children, have paid tribute ➡️ https://t.co/mDvAO1cIYl pic.twitter.com/DZXnZmnyS8
— Brain Tumour Research (@braintumourrsch) June 26, 2023
Good question hankmanhub.
I had a conversation with Joel's mother about this. Her reply:
Joels frozen tumour was only big enough to make 4 vaccines. Some of his tumour had already been sent to Heidelberg for analysis as part of the ipilimumab clinical trial he was part of. So they could only make 4 although 5 were given in the clinical trial for DCVax. Prof Ashkan said that 3 is enough to reprogram the immune system and the other doses are maintenance doses. This treatment is really expensive and we have had to crowdfund for it as you know. It’s probably cost around £190000. We did explore the German option with IOZK but decided against it for various reasons.
Joel Danziger received the devastating diagnosis of glioblastoma in the summer of 2019.
Within a few days he was having emergency brain surgery to remove a golf ball sized brain tumour.
On the 25th March 2021 Joel had his very first dose of Dendritic Cell Vaccine, (DCVax-L). The treatment, at Kings College Hospital in London, was administered by Keyoumars Ashkan, Professor of Neurosurgery and his team. Joel was then his one and only patient having this treatment. (due to Covid precautions.)
On the 5th of May 2021 Joel had his fourth and final dose of DCVax-L (Not enough tumour tissue to made more than 4 vaccine doses)
UPDATE
June 24, 2023
Ian Runacres
"Whenever Joel comes home we go to his fave Chinese restaurant. Drinking Lucky Buddha beer and he was as Joels’s scan results are stable. Huge relief."
No DCVax-L treatment IMO.
Margaret McDonagh has been on a course of treatment which involves a monthly four-day trip to Dusseldorf, Germany.
March 9, 2023
MP: NHS has abandoned my sister over brain cancer treatment
Labour’s Siobhain McDonagh explained how her sister has made monthly trips to Germany for treatment.
https://www.standard.co.uk/news/politics/siobhain-mcdonagh-nhs-germany-labour-margaret-b1066130.html
Very sorry to hear that Margaret McDonagh, Labour's first female General Secretary, has passed away . She was diagnosed with glioblastoma.
.
I am devastated to hear that Margaret McDonagh, Labour’s first female General Secretary, has passed away.
— Keir Starmer (@Keir_Starmer) June 24, 2023
I know I speak for many when I say I will miss her friendship and wise counsel in the years ahead. pic.twitter.com/m1cP54nzPW
At 9.38am this morning my extraordinary sister peacefully passed away at home surrounded by friends who loved her. She was the first female,the youngest & most successful @UKLabour General Secretary in history. She was kind, generous and brave. I loved with my whole heart pic.twitter.com/o1aQWPZsWB
— Siobhain McDonagh (@Siobhain_Mc) June 24, 2023
Interesting toughts on effective collection and utilization of apheresis or leukapheresis starting materials by Mark Sawicki, President and CEO of Cryoport Systems and Chief Scientific Officer of Cryoport, cell therapies.
Standardize for Success
High growth predictions for regenerative medicine are exciting, but what if current capacity can’t keep pace?
Mark Sawicki | 04/25/2023 | 4 min read | Opinion
The emergence of regenerative medicine as a viable therapy class has amplified the focus on current clinical product distribution standards and emphasized the need for enhanced requirements that parallel current manufacturing standards in the industry. The regenerative medicines market generated $13.22 billion in revenue in 2019 – but it is expected to reach a staggering $172.17 billion by 2030(1). According to the US Department of Health and Human Services Report, 2020: A New Vision – A Future for Regenerative Medicine, “Regenerative medicine will be the standard of care for replacing tissue/organ systems in the human body.” For instance, a definitive cure for heart-valve disease in the US alone could provide annual cost savings of $23.4 billion.
One key challenge in the regenerative medicine space is the effective collection and utilization of apheresis or leukapheresis starting materials. The stability of fresh leukapheresis starting material is usually limited to a 24- to 48-hour window because of the decrease in cell viability in nonfrozen conditions (2), so any unforeseen shipment delays can reduce manufacturing success rates. Managing manufacturing slots can also be expensive, and if the manufacturing window is missed, it can cause backlogs for future planned patients. In an effort to overcome these challenges, many companies have decided to cryopreserve the fresh leukapheresis starting material (3). Cryopreservation provides scheduling flexibility while de-risking the logistical process.
In the US, leukapheresis collection activities are governed by CFR Title 21, Part 1271. Current regulations provide significant flexibility in collection-based activities, including accreditation and characterization, typically leaving qualification to the manufacturer. Although 21 CFR Part 1271 outlines cGTP requirements associated with the manufacture of CAR-T therapies, the CFR doesn’t address standardization of leukapheresis collection and processing activities. It also does not define whether leukapheresis material should be defined as a cGMP starting material with the associated cGMP regulations. And the demarcation of where cGMP requirements need to begin to be implemented in the process is unclear. On the other side of the pond, within the EU, the EMA has explicitly defined the collection and preservation of starting material as activities that occur before manufacturing. Regulation no. 1394/2007 on ATMPs, established by the EC, provides the framework for ATMPs.
Additional regulation around collection and processing activities associated with cell and gene therapy collection are anticipated as more clinical and market data on commercial therapies becomes available. In my view, regardless of regulatory status for the collection and processing of ATMP starting materials, it is critical that the industry initiates efforts to standardize the activities associated with processes.
Any standardized solution must address product consistency (regardless of geographic location), and also accommodate and provide efficiencies and scalability to the industry as a whole. I believe major areas to focus on include (but are not limited to): expanded patient and donor access into the community care setting; streamlined audit and quality system management of collection and processing activities; consistent standards and SOPs against cGTP requirements for processing across all sites; integrated data management competencies across all sites; integrated logistics management minimizing costly fresh leukapheresis material movement; truncated processing timelines to ensure product quality; scalable processes supporting significant patient volumes.
The ability to effectively optimize these key inadequacies will be a basic prerequisite for the industry to support projected volumes in the near future, while standardizing processes and optimizing cost of goods.
Based on internal Cryoport data, we conservatively anticipate clinical and commercial patients’ treatment cycle demand to exceed a minimum of 90,000 patients a quarter by the end of 2027. This dwarfs the current demand cycle of approximately 9,000 patients per quarter (as of Q1 2022). Moreover, based on discussion with physicians in the cell and gene therapy space, these numbers are further suppressed by the lack of accessibility to the collection and processing of blood products in community care centers – a significant latent opportunity for the market. Estimates suggest that the unaddressed opportunity in community care centers is around 80 percent of the overall patient opportunity. Consider that leukapheresis capacity is growing at a CAGR of around eight percent; this is expected to support only 13,000 patients a quarter by 2027(4).
The current high cost of therapeutic leukapheresis and cellular therapies, stringent donor recruitment criteria, and long procedural time for leukapheresis are all restraining growth. Manual coordination of operations that span across patient scheduling, apheresis procedure, cell processing and treatment and multiple touchpoints across clinical, hospitals, apheresis centers, manufacturers and delivery companies is a logistical nightmare (5).
Cellular therapies offer potential new therapeutic approaches to address a variety of unmet needs for individuals affected by serious and life-threatening conditions. Standardized collection, processing, storage, and distribution is crucial to both the availability of these therapies and continued innovation in the industry. The implementation of the considerations mentioned above – alongside new standardized regulations and requirements – will help secure and even expedite the regenerative medicine industry’s growth in the safest way possible.