MADISON, Wis. Kidney transplant patients like Tessa Adolph, from Rockford, Illinois, face an age-old problem to protect their new kidney and bodies after transplant surgery: how to prevent infections while also safeguarding their new kidney from damage or rejection.

In Adolphs case, the risk came from the BK virus.

At 19, she was diagnosed with Henoch-Schnlein purpura, a rare condition that causes small blood vessels in the body organs, including kidneys, to become inflamed and bleed. This transitioned into a condition called IgA nephropathy, or Bergers disease, that over time can cause kidney scarring and eventual kidney failure, she said.

People with IgA nephropathy produce too much of an antibody that the body uses to protect itself from disease-causing pathogens. In most cases, the condition develops over decades, but Adolphs condition was extremely aggressive and after two years her kidney function was down to about 7%. She had to undergo kidney dialysis for seven months while she waited for a kidney transplant.

We tried many things to treat it, even chemotherapy and I lost all my hair, but in the end, I needed a transplant, she said.

Her mom donated a kidney and through a process called paired kidney exchange, Adolph was able to get a new kidney. Then, a new problem arose, or more precisely, reawakened the BK virus.

The BK virus causes symptoms similar to a common cold, and most people are infected with this virus at a young age. The human immune system typically fights it off, but the virus continues to linger quietly in the body. The immune system usually keeps it in check, but during transplantation, medications are used to suppress the activity of the immune system to keep the body from rejecting the transplanted kidney.

While immunosuppression is necessary to protect the new organ, it comes with a tradeoff: dormant viruses like BK can start to proliferate, according to Dr. Sandesh Parajuli, transplant physician, UW Health, and associate professor of medicine, UW School of Medicine and Public Health.

We walk this balance between preventing infection and causing damage to the kidney or outright rejection, he said. But there just havent been many good options for treating BK reinfection.

Traditionally, no antiviral drugs have been effective for fighting BK reinfection, and some are even harmful to new kidneys, without much benefit, so Parajuli felt there had to be a better option, he said.

One solution was made possible by the Program for Advanced Cell Therapy, or PACT, at UW Health and the UW School of Medicine and Public Health. The program conducts clinical trials examiningcytomegalovirus reinfectionin bone marrow transplant patients and other cell therapy trials. Parajuli thought a similar path could be tried with BK.

Earlier this year, he collaborated with PACT to launch a first-of-its-kind phase 1 trial using T-cells a type of immune system cell donated from a close relative of Adolph to treat the infection. T-cells used for this procedure are collected from donor blood, and those that target BK specifically are purified for transfusion at PACTs manufacturing laboratory at University Hospital.

It takes six hours to prepare the T-cells. Once they are ready, they are infused into the patient to treat the infection.

Adolphs care team had struggled to contain her BK infection despite multiple treatments, and she eventually had to go back on antiviral drugs, she said. So, in March, Adolph decided to become the first person to enroll in the trial, using cells donated by her father.

I was like, yeah, Im down, she said. At that point, I was just tired of dealing with these things.

Like other PACT cell therapies, a significant advantage of these approaches is a very low risk of side effects other than possible injection site irritation because the treatments use the bodys own immune system to fight off the virus, according to Parajuli.

During the five-minute injection, Adolph felt a little dizzy, but experienced no lasting side effects, she said.

The goal of a phase 1 trial is to determine what dose is safe for patients to take. Establishing how well a drug or device works comes in later trial phases, Parajuli said.

In Adolphs case, the drug had an initial impact but did not effectively fight off the BK infection. However, phase 1 data are critical for future studies, and she is still glad she participated, Adolph said.

Im hoping my participation helps someone else down the road, she said.

Parajuli hopes to enroll 20 patients. Given the hundreds of kidney transplant patients UW Health cares for in a year, there will be no short supply of potential participants, he said.

The trial is free for participants, however, the cost of undertaking the trial is significant, he said.

These are incredibly expensive trials, and we are looking for assistance in the form of private donations, biotech industry support and the like, but the potential of this treatment will keep us working to make this a reality, Parajuli said. Kidney transplant patients deserve to have another option to treat BK reinfection.

A recorded interview with Parajuli is available, and he and Adolph are available for a limited number of interviews today.

Here is the original post:
UW Health, UW-Madison School of Medicine and Public Health: Innovative clinical trial targets recurrent BK infection in kidney transplant recipients -...

Abhinav Achreja, PhD, Research Fellow at the University of Michigan Biomedical Engineering and Deepak Nagrath, Ph.D. Associate Professor of Biomedical Engineering work on ovarian cancer cell research in the bio-engineering lab at the North Campus Research Center (NCRC). Image credit: Marcin Szczepanski, Michigan Engineering

The way that tumor cells enable their uncontrolled growth is also a weakness that can be harnessed to treat cancer, researchers at the University of Michigan and Indiana University have shown.

Their machine-learning algorithm can identify backup genes that only tumor cells are using so that drugs can target cancer precisely.

Most cancer drugs affect normal tissues and cells. However, our strategy allows specific targeting of cancer cells.

The team demonstrated this new precision medicine approach treating ovarian cancer in mice. Moreover, the cellular behavior that exposes these vulnerabilities is common across most forms of cancer, meaning the algorithms could provide better treatment plans for a host of malignancies.

This could revolutionize the precision medicine field because the drug targeting will only affect and kill cancer cells and spare the normal cells, said Deepak Nagrath, a U-M associate professor of biomedical engineering and senior author of the study in Nature Metabolism. Most cancer drugs affect normal tissues and cells. However, our strategy allows specific targeting of cancer cells.

This approach is known as collateral lethalityusing information gleaned from genes that cancer cells discard to find weaknesses. The human body comes with many mechanisms designed to protect against cancer. Cancer cells themselves used to contain suppressor genes that prevent their spread. Those cells however, have a clever strategy for dealing with that; they simply delete a portion of their DNAthe part that includes those suppressor genes.

In doing so, the cells typically lose other genes that are necessary for survival. To avoid death, the cells find a paraloga gene that can serve a similar function. Usually there are one or, possibly, two genes that can step in and perform the same function to keep the cell alive.

What if you could identify the right paralog and target it in a way that shuts down its vital function for the cell?

When a direct replacement for the deleted metabolic gene is not available, our algorithms use a mathematical model of the cancer cells metabolism to predict the paralogous metabolic pathway they might use, said Abhinav Achreja, a U-M research fellow in biomedical engineering and lead author on the research paper. These metabolic pathways are important to the cancer cells and can be targeted selectively.

Study abstract: Metabolic collateral lethal target identification reveals MTHFD2 paralog dependency in ovarian cancer (DOI: 10.1038/s42255-022-00636-3)

Attacking metabolic pathways essentially shuts down the cells energy source. In examining ovarian cancer cells, U-Ms team zeroed in on one gene, UQCR11, that was often deleted along with a suppressor gene. UQCR11 plays a vital role in cell respirationhow cells break down glucose for energy in order to survive.

Disturbances in this process can lead to a major imbalance of an important metabolite, NAD+, in the mitochondria, where respiration takes place. Despite all odds, ovarian cancer cells continue to thrive by relying on their backup plan.

U-Ms algorithm correctly sorted through multiple options and successfully predicted a cell missing UQCR11 would turn to the gene MTHFD2 as its backup supplier of NAD+.

Researchers at the Indiana University School of Medicine helped validate the findings in the lab. This team, led by professor of medicine Xiongbin Lu, developed genetically modified cell and animal models of ovarian cancers with the deletions. Six out of six mice tested showed complete cancer remission.

This research was supported by funding from the National Cancer Institute, Office of the Director for the National Institutes of Health, University of Michigan Precision Health Scholars Award, and Forbes Scholar Award from Forbes Institute of Cancer Discovery.

Continued here:
Shutting down backup genes leads to cancer remission in mice - University of Michigan News

Read the Full Video Transcript

Pedro Barata: Hello and welcome. My name is Pedro Barata. I'm a GU medical oncologist and an associate professor of medicine at Tulane Medical School in New Orleans, Louisiana. It's my true pleasure to be joined today by Dr. Laurence Albiges. She's really well-known and a star in the kidney clinical arena. She's an MD/PhD medical oncologist, and currently the head of the Medical Oncology Department at Gustave Roussy in Villejuif, France. So welcome, Dr. Albiges. Thank you so much for taking the time to be with us today.

Laurence Albiges: Thank you, Pedro. Thank you for the invitation.

Pedro Barata: Absolutely. By the way, I start by congratulating you for your fantastic talk and presentation of a very important study, your phase 2 Keynote-B61 that basically explore the combination of lenvatinib with pembrolizumab as first-line treatment for patients with non-clear cell renal cell carcinoma. So again, very elegant presentation, and I was hoping we could talk a little bit about that today if that's okay with you.

Laurence Albiges: Sure. That will be my pleasure. So actually, this is a phase 2 in an area that is clearly an unmet need. Namely, what we call non-clear cell renal cell carcinoma that should not be named as such because these are different entities that are usually having a worse prognosis than clear cell, and that are really challenging to treat. And so what we've been reporting this time, and it's the first time we're presenting the data, is a phase 2, a non-randomized phase 2, that enroll patient with non-clear cell histology that never been previously treated and that were treated with a combination of pembrolizumab administered every six weeks with lenvatinib at 20 milligrams per day. So it's a combination of VEGF TKI, lenvatinib, plus immune checkpoint pembrolizumab. The primary endpoint of this study is response rate.

Pedro Barata: Fantastic. Thank you so much. And so remind us, as you were saying, which you raise a very, very good point, right? Non-clear cell behaves differently from clear cell. And we can actually go further and argue that actually within non-clear cell group, there's many different diseases that are part of these broad classification of non-clear cell. Can you remind us what type of patients were enrolled in these phase two, as far as histology goes, and then go ahead also and give us kind of the highlights, if you will, on the efficacy portion of this study.

Laurence Albiges: Sure. So that study is about a 150-patient study. What has been presented at this ESMO are the very first results with an efficacy population of 82 patient. These were patient for which we had sufficient follow-up. And with regard to your question of histology, the vast majority of those patients are papillary nccRCC carcinoma. They account for 51 patient in the efficacy population. So that's about 62% of patient. The second most frequent entity are what we call chromophobia RCC, and we know those patients are hard to treat because they tend to be less sensitive or less responding to an immune checkpoint when used as single agent. And then we had other histologies, such as translocation RCC or unclassified tumors, or even more rare tumors, such as medullary carcinoma. So these are, as you can see, a very [inaudible] patient population.

Laurence Albiges: What are the key results? Well, first, the response rate, that is achieving 47.6%. So almost one patient out of two that has objective response. The disease control rate, that is 79%. So basically, almost 80% of patient that has either partial response or stable disease or even complete response. And if you look at the response by histology [inaudible], it's very consistent across papillary and classified translocation and other tumor type. The histology where we saw the less responsive were it chromophobe tumor, as anticipated. And I think, at this point, it's important to stress that we already had data with pembrolizumab single agents that were previously reported by David McDermot. We had seen other regimen with combination with VEGF TKI, and this is the first report of lenvatinib, plus pembrolizumab in this histology of patient with non-clear cell. There was no significant signal in terms of safety. This is the regimen that we are used to handle.

Laurence Albiges: And so, therefore, there was no new safety signal. I think it's important that out of the 82 patient that were accessible for response, we could see some degree of tumor shrinkage in the vast majority of them. It was 86% of patient that had some degree of tumor shrinkage. So clearly, a combination that is active, for now, the follow-up is short. We only had 8.2 months follow-up. Nevertheless, many patients were still under treatment at the time of analysis. Therefore, we will be waiting for further follow-up and the entire patient population to be followed, to provide final analysis.

Pedro Barata: No, it is so important as you mentioned and summarized these results. It's very good news, in my opinion, promising results for patients with non-clear cell histologies. And so thank you for highlighting it so elegantly for us. And as you mentioned, so now we have data with lenvatinib, pembrolizumab. There are other combos being explored out there with proof of concept and promising results. So can you tell us what the next steps for these efforts seem to be? I mean, sounds like you're going to update with longer, with more mature follow-up, you're going to update those results and hopefully we'll see the data being consistent over time. Is any other perspectives you can share with the audience as far as next steps for the non-clear cell RCC patients?

Laurence Albiges: Yeah. Thank you. So what I'm not yet able to provide you with is the progression-free survival, given that for now, median follow-up is at 8.2 months. What we have at six months is 72.3% of patient that are progression-free at that step. So we haven't yet reached the median PFS. And of course, we don't have yet the overall survival for this patient population. I think these items are very important. As we said, these patients tend to have a poor prognostic. And so, therefore, being able to generate prospective data of combination regimen in this space is very important. So we are eagerly waiting for those longer-term follow-up to define the potential role of this combination in patient with non-clear cell.

Pedro Barata: That's fantastic. Well, Dr. Albiges, I feel like we could stay and talk about this much longer. I really appreciate your taking the time to talk to us. Again, congratulations. Very elegant presentation and I'm looking forward to actually follow-up data and also the publication, right? So again, thank you and congratulations.

Laurence Albiges: Thank you very much.

Link:
Pembrolizumab in Combination with Lenvatinib as First-Line Treatment for Non Clear Cell Renal Cell Carcinoma (nccRCC), KEYNOTE-B61 - Laurence Albiges...

The Oncologic Drug Advisory Committee voted 9 to 4 that the current benefits of poziotinib did not outweigh its risks for the treatment of patients with nonsmall cell lung cancer (NSCLC) with HER2 exon 20 insertion mutations.1

The decision was made on day 1 of the September 22/23 ODAC Meeting by 14 voting members. The vote followed an extensive discussion about the overall risk/benefit of poziotinib 16 mg once daily with consideration of the limited response the drug has shown, short durability of response, high toxicity, inadequate dosage optimization, and the fact the confirmatory clinical trial was delayed.

A new drug application was submitted to the FDA for poziotinib as a potential treatment option for previously treated locally advanced or metastatic non-small cell lung cancer (NSCLC) harboring HER2 exon 20 insertion mutations. The proposed indication was supported by results from the phase 2 ZENITH20 clinical trial. The NDA was accepted by the FDA on February 11, 2022, but then raised to the ODAC.

ZENITH20 is a multicenter, multicohort, open-label phase 2 study (NCT03318939) primarily evaluating objective response rate by independent review committee (IRC) in patients with advanced or metastatic NSCLC. The secondary outcomes of the study include disease control rate (DCR), duration of response (DOR), progression-free survival (PFS), safety/tolerability, and quality of life.2

Results reported in 2021 were from 90 patients who had received a median of 2 prior lines of therapy. Poziotinib achieved an ORR of 27.8% (95% CI, 18.9%-38.2%) with partial response observed in 25 patients. The DCR achieved with poziotinib was 70.0% (95% CI, 59.4%-79.2%). Seventy-four percent of patients treated with poziotinib in the study had tumor reduction at a median shrinkage of 22%.

The agent showed a 5.1-month (95% CI, 4.2 to 5.5) median duration of response, and clinical benefit with poziotinib in the study was irrespective of lines and types of prior therapy, presence of central nervous system metastasis, and types of HER2 mutations.

The median PFS observed with the agent was 5.5 months (95% CI, 3.9-5.8) with a 5.1-month median DOR.

Safety findings from ZENITH20 showed that the grade 3 or higher treatment-related adverse events (TRAEs) observed were rash (48.9%), diarrhea (25.6%), and stomatitis (24.4%). The majority of patients in the study (76.7%) required dose reduction. Due to TRAEs, 13.3% of patients Permanently discontinued poziotinib.

I think the bottom line from this study is that it clearly does have substantial activity in the population was studied in when you consider the alternatives that are available and the lack of oral medications that are approved for these patients. I think it's unquestionably from my clinical perspective, it's a clinically meaningful activity. We have some patients that have been on the drug for years and are doing quite well on it and are very grateful that they had this oral drug available. I think this could potentially provide an important option for patients and for healthcare providers for this important unmet need. And it's worth pointing out that if this is approved, well her two mutations occur not just in lung cancer but in more than 20 Different malignancies. One would love to see then that if this is approved If we could build on those studies and look at the activity in those other tumor types as well, said John Heymach, MD, PhD, a medical oncologist, and chair of the Department of Thoracic-Head & Neck Med Onc, Division of Cancer Medicine at the University of Texas MD Anderson Cancer Center.

Despite the results from ZENITH20, the FDA holds that better therapies are now available for patients with HER2 exon 20 insertion mutation-positive NSCLC.

Poziotinib, as demonstrated by the limited response rate with poor durability observed in the primary efficacy population is not improved over available therapies, explained Nicole Drezner, MD, clinical team lead in the Division of Oncology 2 at the FDA, during the FDA presentation.

For patients with nonsmall cell lung cancer, who have received both prior platinum-based chemotherapy and an immune checkpoint inhibitor, available therapy includes docetaxel in combination with ramucirumab [Cyramza] with a benchmark or are of 23%. Anti-PD-L1 therapies are considered available therapy that is not previously received and are associated with lower ORRs with more substantial durability than what is observed with chemotherapy. Trastuzumab dereuxtecan [Enhertu], a HER2- targeting antibody drug conjugate received accelerated approval last month for the treatment of patients with HER2-mutated non-small cell lung cancer as an indication which would include the patients who comprise the primary efficacy population in this application. The drug demonstrated a response rate of 58%, with a duration of response is 8.7 months, both considerably greater than what was observed with poziotinib, Drezner added.

Comparing the data from DESTINY-Lung02 (NCT04644237) which supported the accelerated approval of trastuzumab dereuxtecan to the ZENITH20 data3, Dezner noted that the ORR observed with poziotinib was low with poor durability. Further, the safety profile of poziotinib was brought into question.

The applicant states that the safety profile of poziotinib is similar to other drugs in class. However, in our assessment, poziotinib is more toxic than other tyrosine kinase inhibitors [TKIs] for lung cancer, especially at the 16-milligram dose. Eight of 10 patients experienced grades 3 to 4 adverse events. Similarly, over 80% of patients require a drug interruption and over 50% of patients needed a dose reduction, said Justin N. Malinou, MD, clinical reviewer, Division of Oncology 2 Thoracic and Head and Neck Oncology Office of Oncologic Diseases at the FDA, during the FDA presentation.

Regarding dose optimization, the FDA highlighted that dose reductions occurred within the first months of treatment. Beyond the 24-week mark, most patients were receiving a 12 mg once daily dose of poziotinib.

In FDA review of the applicants clinical pharmacology package, we identified significant areas of concern regarding the lack of dosage optimization. Given that the applicant has provided insufficient data over the clinically relevant dose range, we cannot determine if alternative dosages may provide acceptable efficacy and an improved toxicity profile. Therefore, we continue to assert that the applicant failed to adequately justify their proposed dosage of 16 milligrams once daily, said Jeanne Fourie Zirkelbach, PhD, team lead, Clinical Pharmacology Division of Cancer Pharmacology 2 Office of Clinical Pharmacology at the FDA, during the FDA presentation.

Finally, the FDA originally recommended that a confirmatory trial for poziotinib start recruitment in 2020. To date, no patients have been enrolled in the confirmatory trial. The FDA noted that it would be 4 years before results from the confirmatory trial would be available.

With Spectrum Pharmaceuticals alluding to their success and progress with poziotinib and the FDA asserting that they are unhappy with the development of poziotinib, ODAC members carried out an in-depth discussion.

A point was raised by Spectrum Pharmaceuticals was that poziotinib showed similar dose reduction and discontinuations as other approve TKIs like mobocertinib (Exkivity) and neratinib (Nerlynx). Moreover, poziotinib had the same ORR as agents like mobocertinib. However, the FDA responded that the duration of response with mobocertinib greatly exceeded poziotinib.

The applicant also mentioned that multiple sites for the confirmatory trials have been opened and will started enrolled patients in the next few weeks. But the FDA pointed out that only 3 of the sites were in the Unites States (US) and that failure to accrue patients at US sites would results in findings that are not reflective a US population.

During the open public hearing,7 out of 8 presentations were from patients who testified that poziotinib has either helped them or a family member. The patients did mentioned toxicities but noted that they were manageable.

Joshua K. Sabari,MD, a medical oncologist and assistant professor,Department of Medicine at NYU Grossman School of Medicine, as medical director, Kimmel Pavilion 12, at NYU Langone Health stated that although trastuzumab deruxtecan is now standard for HER2 exon 20 insertion mutation-positive NSCLC, some patients are not eligible to receive it. Therefore, poziotinib may be another option, according to Sabari.

ODAC member opinions varied during the discussion. Some oncologists noted that the risk-benefit profile of poziotinib was acceptable considering that toxicities related to TKIs can be managed in the clinic. One member noted that the short duration of response observed with poziotinib in ZENITH20 could be due to toxicity.

Importantly, poziotinib is an oral agent, which means patients can take their medication in an outpatient setting. One ODAC member raised the point that if toxicities associated with poziotinib required patients to come back to the clinic, it impacts. the favorability of the risk-benefit profile.

REFERENCES:

1. Oncologic Drugs Advisory Committee (ODAC) Meeting. FDA website. September 22, 2022. Accessed September 22, 2022. https://www.fda.gov/advisory-committees/advisory-committee-calendar/september-22-23-2022-meeting-oncologic-drugs-advisory-committee-meeting-announcement-09222022

2. Le X, Cornelissen R, Garassino M, et al. Poziotinib in non-small-cell lung cancer HarboringHER2exon 20 insertion mutations after prior therapies: ZENITH20-2 Trial. J Clin Oncol. 2022;40(7): 710-718. doi: 10.1200/JCO.21.01323

3. FDA grants accelerated approval to fam-trastuzumab deruxtecan-nxki for HER2-mutant non-small cell lung cancer. FDA website. August 11, 2022. Accessed September 22, 2022. https://bit.ly/3LyWDMK

See more here:
FDA's ODAC Votes That Benefits Do Not Outweigh Risks for Poziotinib in HER Exon 20 Ins+ NSCLC - Targeted Oncology

An inspirational East Kilbride teenager studying to become a nurse is living proof that trials of new treatments for children with cancer can save lives.

Katie Currie was just three years old when diagnosed with acute lymphoblastic leukaemia.

Her parents Neil and Siobhan said they felt like their world came crashing down, as months of treatment began.

Then two years later in 2008, aged five, Katie relapsed.

The Lanarkshire Live app is available to download now.

Get all the news from your area as well as features, entertainment, sport and the latest on Lanarkshires recovery from the coronavirus pandemic straight to your fingertips, 24/7.

The free download features the latest breaking news and exclusive stories, and allows you to customise your page to the sections that matter most to you.

Head to the App Store and never miss a beat in Lanarkshire - iOS - Android

After a nationwide search for a donor whose stem cells were a match - a campaign supported by the East Kilbride News at the time - she had a stem cell transplant on September 11, 2008.

Katie began a clinical trial that Cancer Research UK helped fund for a new chemotherapy treatment called mitoxantrone, and the trial results were striking.

Mitoxantrone proved so effective that all children taking part in the trial were put on the new chemotherapy drug as soon as possible.

Now Katie, who is in remission, is sharing her remarkable story to support Childhood Cancer Awareness Month this September.

She is passionate about giving back after the treatment shes received, and that starts with studying for a degree in child health nursing at Edinburgh Napier University.

Katie, 19, said: I am living proof that clinical trials work and feel proud I took part in a trial that helped change in a positive way how some childhood cancers are treated.

I was so young when I was diagnosed that I dont remember much from then, but I do remember being in hospital and I remember the nurses.

It is surreal to think about what I went through then and how far I have come. Now Im keen to do everything I can to put something back and help the NHS.

Cancer Research UK funded scientists helped show that the drug mitoxantrone can increase survival by more than 50 per cent in children whose acute lymphoblastic leukaemia has come back after treatment.

Katie is one of around 330 young people up to the age of 25 who are diagnosed with cancer in Scotland every year.

Acute lymphoblastic leukaemia (ALL), a cancer of the white blood cells has a particularly high survival rate. Thanks to major advances in treatment, more than nine in 10 children in the UK with ALL now survive for at least five years, compared with around seven in 10 in the 1980s.

Professor Vaska Saha, based at the University of Manchester, was researching ways to improve treatment options for children like Katie Currie who were facing leukaemia for a second time and led research on mitoxantrone.

The drug works by jamming a molecule in cancer cells responsible for untwisting DNA. Blocking this process literally ties the cells DNA up in knots so it cant grow and multiply. It also works on cancer cells that have resisted previous treatment- even if theyre hidden among healthy cells.

Professor Saha was part of an international team working on the clinical trial, known as ALLR3, comparing the drug idarubicin, a chemotherapy treatment normally used for young people whose leukaemia had relapsed to mitoxantrone, which had proved successful in early trials in the lab.

A total of 216 children and young people including Katie took part in the trial. They were split in to two groups. One group was given the drug idarubicin and the other group mitoxantrone.

The study evaluated the number of children whose leukaemia hadnt got worse over the three years since treatment.

Results showed that just over six out of 10 children (64.6 per cent) who had mitoxantrone, compared to less than four out of 10 children (35.9 per cent) who had idarubicin, had cancers that hadnt grown at all in the timeframe.

The study also found that the number of children alive three years after treatment, regardless of whether their cancer had grown or not, was far higher in the group on mitoxantrone compared to idarubicin. This is known as overall survival.

The trial had been planned to continue for several years. But it was halted early and all the children who had originally been given idarubicin were transferred to mitoxantrone.

Professor Saha said: A difference in outcomes like that had never been reported before. It took all of us by surprise.

Now Katie is packing her bags this September to head off to her second year at university studying nursing. Her parents are hugely proud of everything shes achieved but they recall vividly the early years when life seemed less positive.

Dad Neil said: When Katie was first diagnosed and started treatment in Glasgow, we put our trust in the doctors at the hospital.

We took it day by day, especially after she relapsed. When the cancer came back, you fear everything, but we spoke to the team about the trial and put complete faith in them.

Mum Siobhan said: We believed that, with the clinical trial Katie had the best chance of recovery. Without these trials, amazing new treatments may never be found. Mitoxantrone probably saved Katies life.

Katie, who has some side-effects from her experience including endocrine issues and the loss of vision in one eye after she developed CMV retinitis, has annual check-ups now.

Katie said: I have just had the first set of these check-ups in the adult services department, as they had been in the childrens unit before.

I felt anxious going but the doctor that treated me was still there for the checks, so that definitely helped. It is still scary though, as you dont know what they are going to say and it was a huge relief that everything was ok.

She said she always knew she wanted to be involved in healthcare, adding: "I knew I wanted to do something in medicine after what had happened to me, and I was interested in being a paramedic too but it changed over the years. By doing this, I really want to say thank you for the help I had and I am passionate about nursing.

I started uni in Sept 2021 and I still had to be shielded a bit, but I really enjoyed the course and I am just starting the second year now. I have been on two placements already and I think that my own experience has really helped me with my understanding with patients.

The course is three years long and then I can choose where I focus on. I am just so pleased to be able to do this after my experience and to give back for the help I had.

Dad Neil said the whole family including Katies sister Libby, 17, are proud of Katie.

He said: Katie has been through so much but she is resilient and so upbeat in all that she does. She just gets on with things and is so kind too.

At an early age, she talked about being involved in medicine and we did ask if she was sure she wanted to go into that after all she had experienced, but she was adamant.

Thinking back to those days of Katies treatment and relapse when she was so young, it is just amazing to be here now and for her to be off to Edinburgh following her dreams.

"Back then, we put our faith in the research and everything that goes on behind the scenes. That has been incredible for us and for Katie, but we know there is more that still needs to be done to make treatments more effective and kinder too, so that there are less side-effects.

"We know how important this work is for children in the future and thats why we support Cancer Research UK and all the research that needs to be funded.

Cancer Research UK celebrates its 20th anniversary in 2022. However, its history dates back to the founding of the Imperial Cancer Research Fund in 1902.

Thanks in part to the charitys work, childrens cancer survival in the UK has more than doubled since the 1970s when just over a third of children diagnosed survived beyond ten years. Today, its around 8 in 10 but theres still much further to go.

Cancer Research UK spokeswoman in Scotland, Lisa Adams, said: Were grateful to Katie and her family for their support.

Cancer in children and young people is different to cancer in adults- from the types of cancer to the impact of treatments and the long-term side effects survivors often experience.

Supporters can help by wearing a gold ribbon badge the awareness symbol of Childhood Cancer Awareness Month available from Cancer Research UK shops and selected TK Maxx stores during September.

Find out more here.

Don't miss the latest headlines from around Lanarkshire. Sign up to our newsletters here.

And did yo u know Lanarkshire Live is on Facebook? Head over to our page to give us a like and share.

Read more from the original source:
Courageous Lanarkshire teen who survived leukaemia thanks to a clinical trial pursues nursing dream - Daily Record