AIVITA Biomedical Announces Publication Concerning a Predictive Biomarker for Melanoma Patients Treated with the Company’s Platform Immunotherapy -…

IRVINE, Calif., Oct. 2, 2019 /PRNewswire/ --AIVITA Biomedical Inc., a biotech company specializing in innovative stem cell applications, today announced the publication of an article titled "Preliminary observations on soluble programmed death-1 protein as a prognostic and predictive biomarker in patients with metastatic melanoma treated with patient-specific autologous vaccines" in the oncology journal Oncotarget. Robert O. Dillman, M.D., Chief Medical Officer at AIVITA, and other key members of the AIVITA team authored the article.

The publication suggests that because of its role as an immune checkpoint, levels of soluble programmed cell death protein-1 (sPD-1) could be useful as a prognostic biomarker or predictive biomarker in cancer patients treated with vaccines. The authors theorized that very low blood levels of sPD-1 may indicate lack of an existing anti-cancer immune response, while very high levels may indicate an active immune response that is suppressed. In between these extremes, a decrease in PD-1 following cancer vaccine injections may indicate an enhanced immune response that has not been suppressed.

Blood samples were obtained at baseline and four weeks later during a randomized trial in which patients with metastatic melanoma were treated with either AIVITA's immunotherapy, or an active control article. Median survival was more than twice as long in patients treated with AIVITA's immunotherapy. The combination of a very low baseline sPD-1, or absence of a very high PD-1, at baseline followed by a decline in sPD-1 at week-4 of the study was predictive of surviving 3 or more years in patients treated with AIVITA's immunotherapy, but not with the control article. Among patients treated with AIVITA's immunotherapy, these sPD-1 criteria appropriately classified 80% of 3-year survivors, and 86% of patients who did not survive three years.

"These observations suggest that sPD-1 may be a useful biomarker for melanoma patients being treated with our platform immunotherapy, and/or to predict efficacy after only three injections," said Dr. Robert O. Dillman, Chief Medical Officer at AIVITA. "We look forward to confirming these results in larger studies and investigating whether it can predict response in other cancers."

AIVITA is currently conducting three clinical studies investigating its platform immunotherapy in patients with ovarian cancer, glioblastoma and melanoma. AIVITA uses 100% of proceeds from the sale of its ROOT of SKIN skincare line to support the treatment of women with ovarian cancer.

CLINICAL TRIAL DETAIL

OVARIAN CANCER

AIVITA's ovarian Phase 2 double-blind study is active and enrolling approximately 99 patients who are being randomized in a 2:1 ratio to receive either the autologous cancer stem cell-targeting immunotherapy or autologous monocytes as a comparator.

Patients eligible for randomization and treatment will be those (1) who have undergone debulking surgery, (2) for whom a cell line has been established, (3) who have undergone leukapheresis from which sufficient monocytes were obtained, (4) have an ECOG performance grade of 0 or 1 (Karnofsky score of 70-100%), and (5) who have completed primary therapy. The trial is not open to patients with recurrent ovarian cancer.

For additional information about AIVITA's AVOVA-1 trial patients can visit: http://www.clinicaltrials.gov/ct2/show/NCT02033616

GLIOBLASTOMA

AIVITA's glioblastoma Phase 2 single-arm study is active and is enrolling approximately 55 patients to receive the cancer stem cell-targeting immunotherapy.

Patients eligible for treatment will be those (1) who have recovered from surgery such that they are about to begin concurrent chemotherapy and radiation therapy (CT/RT), (2) for whom an autologous tumor cell line has been established, (3) have a Karnofsky Performance Status of > 70 and (4) have undergone successful leukapheresis from which peripheral blood mononuclear cells (PBMC) were obtained that can be used to generate dendritic cells (DC). The trial is not open to patients with recurrent glioblastoma.

For additional information about AIVITA's AV-GBM-1 trial please visit: http://www.clinicaltrials.gov/ct2/show/NCT03400917

MELANOMA

AIVITA's melanoma Phase 1B open-label, single-arm study will establish the safety of administering anti-PD1 monoclonal antibodies in combination with AIVITA's cancer stem cell-targeting immunotherapy in patients with measurable metastatic melanoma. The study will also track efficacy of the treatment for the estimated 14 to 20 patients. This trial is not yet open for enrollment.

Patients eligible for treatment will be those (1) for whom a cell line has been established, (2) who have undergone leukapheresis from which sufficient monocytes were obtained, (3) have an ECOG performance grade of 0 or 1 (Karnofsky score of 70-100%), (4) who have either never received treatment for metastatic melanoma or were previously treated with enzymatic inhibitors of the BRAF/MEK pathway because of BRAF600E/K mutations and (5) are about to initiate anti-PD1 monotherapy.

For additional information about AIVITA's AV-MEL-1 trial please visit: http://www.clinicaltrials.gov/ct2/show/NCT03743298

About AIVITA Biomedical

AIVITA Biomedical is a privately held company engaged in the advancement of commercial and clinical-stage programs utilizing curative and regenerative medicines. Founded in 2016 by pioneers in the stem cell industry, AIVITA Biomedical utilizes its expertise in stem cell growth and directed, high-purity differentiation to enable safe, efficient and economical manufacturing systems which support its therapeutic pipeline and commercial line of skin care products.

View original content to download multimedia:http://www.prnewswire.com/news-releases/aivita-biomedical-announces-publication-concerning-a-predictive-biomarker-for-melanoma-patients-treated-with-the-companys-platform-immunotherapy-300929591.html

SOURCE AIVITA Biomedical, Inc.

Read more:
AIVITA Biomedical Announces Publication Concerning a Predictive Biomarker for Melanoma Patients Treated with the Company's Platform Immunotherapy -...

Improved Gene Therapy in Sickle Cell Disease – Technology Networks

Researchers at the National Institutes of Health (NIH) have developed a new and improved viral vector--a virus-based vehicle that delivers therapeutic genes--for use in gene therapy for sickle cell disease. In advanced lab tests using animal models, the new vector was up to 10 times more efficient at incorporating corrective genes into bone marrow stem cells than the conventional vectors currently used, and it had a carrying capacity of up to six times higher, the researchers report.

The development of the vector could make gene therapy for sickle cell disease much more effective and pave the way for wider use of it as a curative approach for the painful, life-threatening blood disorder. Sickle cell disease affects about 100,000 people in the United States and millions worldwide.

"Our new vector is an important breakthrough in the field of gene therapy for sickle cell disease," said study senior author John Tisdale, M.D., chief of the Cellular and Molecular Therapeutic Branch at the National Heart, Lung, and Blood Institute (NHLBI). "It's the new kid on the block and represents a substantial improvement in our ability to produce high capacity, high efficiency vectors for treating this devastating disorder."

Researchers have used virus-based vehicles for years in gene therapy experiments, where they have been very effective at delivering therapeutic genes to bone marrow stem cells in the lab before returning them to the body. But there's always room for improvement in their design in order to optimize effectiveness, Tisdale noted. He compared the new virus-based vehicle to a new and improved car that is also far easier and cheaper for the factory to produce.

The study was supported by the NHLBI and the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), both part of the NIH. It was published online today in Nature Communications.

Sickle cell disease is an inherited blood disorder caused by a mutation, or misspelling, in the beta-globin gene (or -globin gene). This mutation causes hemoglobin, the main ingredient of blood cells, to produce sickle-shaped cells that can stick to the walls of blood vessels, causing blockage, pain, anemia, organ damage, and early death. With gene therapy, doctors modify the patient's bone marrow hematopoietic (blood-producing) stem cells in the lab by adding a normal copy of the beta-globin gene through the use of a viral vector. They then reinfuse the modified stem cells into the patient to produce normal, disc-shaped red blood cells.

For the past 30 years, researchers have been designing these beta-globin vectors in a reverse structural orientation, meaning the therapeutic genes incorporated into the virus are translated, or "read," from right to left by the viral vector-making machinery--much like reading an English sentence backwards. The reason for the reverse orientation is the sensitive expression of a key molecular component of the vector called intron 2. This segment is required for high-level beta-globin gene expression but gets clipped out during the normal vector preparation process if it is left in the natural, forward direction. Gene therapy trials using reverse-oriented vectors for sickle cell disease and beta-thalassemia have largely been encouraging, the researchers said, but this complicated gene translation process has made vector preparation and gene-transfer efficiency more difficult.

About 10 years ago, Tisdale and Naoya Uchida, M.D., Ph.D., a staff scientist in his lab, searched for an improved delivery vehicle--like designing a better car--and decided to undertake a radical redesign of the beta-globin vector. They came up with a unique work-around design that left intron 2 intact and created the new forward-oriented beta-globin vector. In contrast to the old vector, the gene sequence, or "message," of the new beta-globin vector is read from left to right--like reading a normal sentence--making the gene translation approach less complicated, Tisdale explained.

The researchers tested the new vectors in mice and monkeys and compared the results to reverse-oriented vectors. They found that the new vectors could transfer a much higher viral load--up to six times more therapeutic beta-globin genes than the conventional vectors--and had four to 10 times higher transduction efficiency, a measure of the ability to incorporate corrective genes into repopulating bone marrow cells. The new vectors also showed a capacity for longevity, remaining in place four years after transplantation. Researchers also found that they could be produced in much higher amounts than the conventional vectors, potentially saving time and lowering costs associated with large-scale vector production.

"Our lab has been working on improving beta-globin vectors for almost a decade...and finally decided to try something radically different--and it worked," Tisdale said. "These findings bring us closer to a curative gene therapy approach for hemoglobin disorders."

Reference: Uchida et al. 2019.Development of a forward-oriented therapeutic lentiviral vector for hemoglobin disorders. Nature Communications. DOI: https://doi.org/10.1038/s41467-019-12456-3.

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

Read this article:
Improved Gene Therapy in Sickle Cell Disease - Technology Networks

Biochemistry Graduate is the Researcher’s Researcher at Advanced BioCatalytics – AroundtheO

It was 2015 and Eric Nummedal was ready to celebrate the fruit of his yearlong experiment on neurons and vision. The junior and his lab mates had reviewed every step, double-checked every calculation, eliminated every assumption. All that remained were the resultsnumbers sure to confirm the teams theories.

But the numbers didnt say what Nummedal expected them to say about receptive fields in the brain. In fact, the numbers didnt make much sense.

Was there gnashing of teeth? Issuing of expletives? Launching of lab equipment?

Not Nummedal. He simply chuckled, shrugged his shoulders, and began developing a new line of inquiry based on what the team had just learned.

What we were hoping would be the final piece of a puzzle was ultimately the first piece of a new puzzle, says Nummedal, BS 16 (biochemistry, biology). Thats extremely satisfyingwhen things dont make sense and it makes questions, youre pushing the boundaries of knowledge, and thats what science is all about.

It takes a special kind of scientist to draw inspiration from failure. Nummedal isthat scientist.

As an undergraduate, he cut a wide swath of excellence, earning honors and scholarships and winning awards in the hard sciences and business. That track record set Nummedal up for immediate success upon graduation: his first job was research lead for a life sciences company, and hes moved to new employers twice since to take on ever more responsibility for R & D.

Nummedal has dabbled in business leadership and helped develop cancer immunotherapies and stem-cell treatments to restore vision. His employers have tasked him with exhaustive investigations ranging from data analytics and cellular mechanisms to sensory systems, education outcomes, and business strategy.

Tying them all together, Nummedal says, is the approach ingrained in him as an undergraduate in the lab to get at the truth of something: the scientific method, those unbending rules of hypothesis, experiment, and deduction established centuries ago to investigate phenomena, obtain knowledge, andtest theories.

The scientific method is one of our greatest achievements, says Nummedal. You wouldnt think there would be any relationship between cancer immunotherapies or brain research and what Im doing now, but its all about applying the scientific method and showing whether something is true or not.

Nummedal recently joined Advanced BioCatalytics, an industrial biotech company in California, where he guides research and development. The companys trademarked productbased on protein synergistsimproves the performance of cleaners, industrial and agricultural wetting agents, and wastewater treatment applications, among other uses.

They are challenging me in ways I havent been challenged before, says Nummedal, who manages the research that guides decision-making about new products to develop. But to understand a new field, you just tie that into research. There is a specific way that ideas are presented and answers are found, and with the background I have I know how to get them.

Nummedal has championed the value of scientific inquiry to students in multiple returns to campus. As a graduate of the McNair Scholars Program, a federally funded initiative that supports undergraduate research, he delights in helping young scientists network into promising jobs and urges them to connect with him on LinkedIn.

The process of research is very intimately connected not only with expanding knowledge but expanding yourself, Nummedal says. When youre struggling with something, when you feel challenged, when you think youre the stupidest person in the roomthat should excite you. You have the opportunity to grow. Taking advantage of that opportunity is up to you.

--By Matt Cooper

Cooper is managing editor forOregon Quarterly.

See the article here:
Biochemistry Graduate is the Researcher's Researcher at Advanced BioCatalytics - AroundtheO

Pike families to enter part of mine day before widow’s cancer treatment begins – Stuff.co.nz

Pike River widowAnna Osborne is hoping stem-cell treatment will help herlive long enoughto seeher granddaughter start school.

Osborne, whose husband Milton died in the 2010 Pike River mine disaster, will be one of 30relatives to travel 170 metres into the West Coast mine's access tunnel, or drift, on Thursday.On Friday, she will begin intensive chemotherapy in preparation for a stem-cell transplant.

"IThe thought of standing that close to Milt and of how far we have come will give me strength. I wouldn't miss that for the world," she said.

Osbornewasdiagnosed with Hodgkin's lymphoma in 2002 when she was 36.

READ MORE:* Pike River re-entry team breaks through into mine drift* New date for Pike River mine drift reopening after failed attempt* Pike River widow 'full of nerves' for mine drift re-entry * Pike River re-entry: Police won't be among first inside mine after risk assessment raised safety concerns* The road to getting back into Pike River

She had radiation for six weeks and went into remission, but the cancercame back just before the Pike River tragedy in November 2010, when 29 men where killed in a series of explosions at the coal mine. Osborne helped campaign for the legalisation of medicinal cannabis while undergoing chemotherapy in 2015.

"It's now got to the stage where my only option is to get stem-cell treatment to get a few more years. The future doesn't look too bright if I don't [go ahead it]," she said.

Pike River mine widow Anna Osborne witnesses the tunnel reentry on May 21, 2019.

Her stem-cells wereharvested and frozenin August. She would go straight to Christchurch for tests after entering the mine drift,then begin six days of intensive chemotherapy on Saturday.

"Within 24 hours of the chemotherapy being finished they will put my stem cells back in and then it will be a wait and see game."

After the stem cell transplant, she would be in isolation in Christchurch for potentially several months.

Osborne said the treatment had its own risks.

"The chemotherapy kills all your good cells and makes you really susceptible to infection. The oncologist has said there's a chance I could die because of it. But with only a month to a year to live without it, I have nothing to lose," she said.

If the treatment is a success, she could live for maybe another five years, she said.

PIKE RIVER RECOVERY AGENCY

Pike River mine families at the mine portal.

She wanted to see her 2-year-old granddaughter'sfirst day at school, and see the Pike River re-entry completed with the hope of new evidence being found to bring those responsible for the disaster to justice.

"I'm going to really fight. I have confidence and trust in my oncology team. I haven't got time to die I have too much unfinished business," she said.

It had been a "long, long journey" of protesting against plans to permanently seal the mine.

The National Government had refusedto enter the mine, citing safety concerns.

"We have fought hard for truth and justice and it is almost here. That's something we need to thank all of New Zealand for without that support, our men and the evidence that could get them justice would have been locked away forever."

Osborne said she had previously been up to the 30m seal inside the mine's drift.

"It was a small distance but it was very emotional. Behind the seals entombs our men ... Going right up to the 170m seal is another milestone in getting closer to where we want to go."

Pike River Recovery Agency

The Pike River mine reopened for the first time on May 21, 2019, after an explosion in November 2010 killed 29 men.

The journey to the 170m sealthe furthest point anyone has been into the drift since 2011 will be the last chance for families to enter the area before full recovery of the 2.3-kilometre tunnel begins.

The Pike River Recovery Agency has been given a $36 million budget to recover the drift.

Pike River mum Sonya Rockhouse, whose son Ben died in the mine and other son Daniel survived the blast,said entering the drift wouldbe emotional.

"This will be the closest most of us will have been to our loved ones since that awful day in 2010.

"I don't know how I'm going to feel when I'm there. Right now I'm nervous about it but also proud that Anna and I have been able to help the families get this far."

Continued here:
Pike families to enter part of mine day before widow's cancer treatment begins - Stuff.co.nz

Adcetris May Be Effective in Heavily Pre-Treated Hodgkin’s Cases, Study Shows – Lymphoma News Today

Treatment with Adcetris (brentuximab vedotin) is most effective in people with relapsed or refractory Hodgkins lymphoma who have chemotherapy-sensitive disease, and when used as consolidation therapy immediately after stem cell transplant, a study found.

The findings were presented at the 2019 European Society for Medical Oncology (ESMO) Congress, held Sep. 27 to Oct. 1 in Barcelona, Spain, in a poster titled Prognostic Factors Influencing Outcome After Therapy With Brentuximab Vedotin in Patients with Relapsed or Refractory Hodgkins Lymphoma.

Adcetris is an antibody-drug conjugate that works by binding to CD30, a protein found at the surface of malignant cancer cells. Once bound, it releases a toxic compound called monomethyl auristatin E that kills cancer cells.

It is approved for multiple lymphoma types, including advanced classical Hodgkins lymphoma (cHL). The therapy has been used to treat people newly diagnosed with cHL, patients at risk of their disease returning or worsening after a stem cell transplant, and those with cHL who failed a stem cell transplant.

Clinical studies have shown that Adcetris improves the prognosis and survival of Hodgkins lymphoma patients with refractory disease. But prognostic factors influencing responses to treatment and outcomes have not been explored.

To address that, a team led by Veselina Goranova-Marinova, MD, PhD, from the Medical University of Plovdiv, reviewed the clinical records of 64 people with Hodgkins lymphoma who were treated with Adcetris in six hematology clinics in Bulgaria.

Patients had received a median of four prior therapies (range 2 to 12), and 30 (46.9%) had received at least three prior lines of therapy. Autologous stem cell transplant using the patients own stem cells was performed in 45 individuals (70.3%).

The analysis showed that, despite being heavily treated, more than half of the patients (60.9%) responded to Adcetris. There were 39.1% complete responses. An additional 10.9% achieved stable disease after treatment.

At the time of the analysis, more than half of the patients were still alive, and the median time to disease worsening or death was 14 months.

Researchers noted that partial or complete responses were higher among patients who had responded to their chemotherapy regimen deemed chemo-sensitive before the stem cell transplant, those who had received a stem cell transplant, and patients who started Adcetris as a consolidation therapy within three months of their transplant.

However, the only factor predicting longer event-free survival the time patients live without disease progression or complications was the response to Adcetris. While individuals achieving stable disease or experiencing disease progression lived without an event for a median of seven months, more than half of those with partial or complete response to Adcetris remained event-free.

Adcetris improves the therapeutic response and prolongs progression-free survival in the patients with Hodgkins lymphoma who previously had really bad prognosis, the researchers said.

It was most effective in patients with chemosensitive disease, and when used as consolidation therapy early, by [the third] month after [autologous stem cell transplant], they concluded.

Total Posts: 366

Ins Martins holds a BSc in Cell and Molecular Biology from Universidade Nova de Lisboa and is currently finishing her PhD in Biomedical Sciences at Universidade de Lisboa. Her work has been focused on blood vessels and their role in both hematopoiesis and cancer development.

Visit link:
Adcetris May Be Effective in Heavily Pre-Treated Hodgkin's Cases, Study Shows - Lymphoma News Today

Bone marrow donor registry pleas for more diversity to help save people with cancer – ABC News

Updated October 02, 2019 17:25:10

The hardest thing Daniel Roberts has ever had to do was watch his young niece Remy battle leukaemia.

On a warm summer day five years ago, the Warrnambool-based real estate agent was playing horseback rides with the two-year-old, carrying her around his brother's kitchen as she giggled with delight.

"The next day she had bruises all over her, and people thought I'd been a bit rough with her on my back," he said.

"She went to the doctor for a check-up and blood test. That's how we found out her bone marrow was failing."

Remy was diagnosed with aplastic anaemia, a rare condition in which the body stops producing enough new blood cells.

The once-energetic and vivacious girl became lethargic, and covered in deep bruises.

Her only hope was a bone marrow transfusion, but finding a match was easier said than done.

"I wanted to be able to save my brother's little girl, but no-one in the family was a match, which was quite unbelievable," Mr Roberts said.

Remy's condition deteriorated she required blood transfusions every seven to 10 days but thankfully an unrelated donor was found.

Now six, she is back at school and getting stronger every day.

"Remy, she was right on the cusp of not being here with us, so to see her with us today is absolutely amazing," he said

Despite not being able to help his niece, Mr Roberts stayed on the bone marrow donor list only one in 1,500 donors are asked to donate in any given year.

But just two years after a random donor saved his niece's life, Mr Roberts got the call.

"I'm six-foot-three and pretty bulletproof. I'm tough as nails and never get a tear in my eye or anything like that, but I couldn't stop actually crying," he said.

"I just couldn't believe I got to pay it back so quickly."

His donation helped save a five-year-old boy who was also fighting aplastic anaemia.

"The amount of tests and all that you go through is nothing compared to what that little fella was probably going through," Mr Roberts said.

"But that said, I'd do it again in a heartbeat.

"My niece was dying. She got a random match from somebody, and now she's fantastic. I just can't believe you can pay someone back so quickly."

Every 40 minutes, someone in Australia is diagnosed with a blood cancer.

And for most, a blood stem cell or bone marrow transplant from a stranger is their only hope.

But if you are Indigenous or ethnically diverse, the chances of finding a match are much harder, because donors need to have the same genetic background as the recipient.

Less than 1 per cent of people on the bone marrow donor registry are from Aboriginal or Middle Eastern background, less than 3 per cent are Asian, and less than 5 per cent are Pacific islander.

The head of the registry, Lisa Smith, said despite Australia's multicultural make-up, eight of every 10 people on the registry were of north Caucasian background.

"The registry itself is largely unknown and really lacking diversity that reflects Australia's multiculturalism," she said.

"It genuinely is one-in-a-million odds to get a match, so when we ring a donor, they could very well be the only person on the planet that's able to help that patient."

The Australian Bone Marrow Donor Registry is trying to find 5,000 new donors this year.

"We need donors that are young and ethnically diverse, because our current donor pool, if characterised as a person, is a middle-aged white female," Ms Smith said.

"From a transplant clinician's perspective, they're looking for donors that are young between 18 and 30, are male and are reflecting the ethnicity of their patient."

Pamela Bou Sejean knows the importance of a strong donor list better than most.

She was diagnosed with Hodgkin lymphoma in 2010, and after chemotherapy and radiation failed, she was told her "last chance" was a stem cell transplant.

Stem cells from the umbilical cord blood of a Spanish donor gave Ms Bou Sejean a second chance at life.

"To know someone's out there that's actually saved my life, 'thank you' doesn't seem enough," she said.

She went into remission in 2012 and founded the charity Ur The Cure, which aims to demystify stem-cell donations and promote greater diversity on the donor list.

"Often it's people's last chance for a cure, and you need a stem cell match with someone who shares an ethnical or cultural heritage."

There is a 30 per cent chance that a match can generally be found within a person's immediate family. After that, it gets harder.

"It's kind of like winning TattsLotto, but it's not winning money it's winning the chance to live."

For Warrnambool mother Julia Thompson, storing her umbilical cord for stem cells was a no-brainer.

Following her own cancer battle, Ms Thompson gave birth to her son Hugh in 2017.

She is now in remission, having used her stem cells as part of her treatment.

It was that treatment that inspired her to store Hugh's umbilical cord and associated stem cells for future use.

"It's difficult that it's only available in selected hospitals in Australia, but it's really an amazing insurance policy for him," she said.

"We knew that because of my history with cancer, the likelihood of Hugh having siblings was very slim.

"Given my history and the fact that stem cell blood saved my life, I knew how important that was. It's really something to consider for the future of your child and family."

Topics:leukaemia,bones-and-muscles,people,medical-research,health,science-and-technology,human-interest,warrnambool-3280,geelong-3220,melbourne-3000

First posted October 02, 2019 06:54:09

Read the original:
Bone marrow donor registry pleas for more diversity to help save people with cancer - ABC News

Warwick mum’s fundraising ball to help fund 50000 treatment to fight the progression of MS – Warwick Courier

A Warwick mum is holding a fundraising ball to help raise 50,000 to help her fight the progression of her Multiple Sclerosis.

Catherine Cook, who has three young children, was diagnosed with MS in 2000 when she was in her early 30s.

She has been trying to raise 50,000 so that she can have a course of haematopoietic stem cell transplantation (HSCT), which could reverse the effects of her condition.

Read more: Warwick mum's 50,000 fundraising drive to help her fight the progression of MSI am trying to change mine and my familys lives",said Catherine. "MS is causing me lots of problems, including pain and exhaustion on a daily basis meaning that I cant join in with a lot of family activities.

I have to plan everything in advance to ensure I get enough rest. I want to get my life back so I can be the mum my children need me to be."

Catherine has been holding various fundraising event throughout the year to help raise the money for the treatment. The fundraising ball, which is taking place next weekend in Warwick, is also raising money for her treatment.

Explaining the treatment Catherine said: HSCT is the only known medical treatment that has halted the progression of MS for the majority of patients who have gone through it. Its a chemotherapy-based treatment which removes your immune system and reboots it using your own harvested stem cells.

Its not available as a standard treatment on the NHS; either you have to be part of a medical study or be one of the most extreme people suffering from MS.

Its offered at in Pueblo, Mexico and Im asking for help to fund my treatment and after care. It isnt risk-free and its a harsh treatment to endure. But almost 700 people have been treated in Mexico including BBC News correspondent Caroline Wyatt.

"I have been accepted for treatment on May 4 2020, so the clock it ticking for me to raise the 50,000 needed. I am currently almost half way there after starting fundraising in February this year, but I know I need to keep going as so much is at stake, not least our three young children who need me to be more active with them and participate in activities with them, instead of in bed."

The ball takes place in the Court House in Warwick on October 12 from 7pm to 11.30pm and will feature food, a raffle, auction and music. Tickets cost 38.

The raffle is open to anyone and features prizes such as meals, wine and vouchers. Raffle tickets cost 5 a strip or three strips for 10.

To donate, buy raffle tickets or tickets for the fundraising ball click here or go to: https://www.rebootcathwithhsct.com/

Read this article:
Warwick mum's fundraising ball to help fund 50000 treatment to fight the progression of MS - Warwick Courier

I hope treatment will stop Multiple Sclerosis in its tracks – Echo Live

AN ordinary man with ordinary interests, Patrick Keane loves life, even though it has become more of a challenge than ever before.

He cannot play a round of golf or go for a walk by the beach any more. But he doesnt want pity and exudes positivity. So he enjoys soaking in the sea air around Clonea or Garryvoe without stretching his legs.

But reality has a way of crashing through the faade at times, no matter how strong you are.

For Patrick, it can be when he wakes up and cannot feel his legs, or loses his train of thought, or misses his mouth when trying to eat because he cannot feel the spoon in his hand. Or if he gets out of bed and falls and screams at himself, Just get up will ya, ya fool!

But the man known as Pa to his friends, or Patch to his mother Margaret, brother Paul and sisters Yvonne and Elaine, dusts himself down, drags himself back to an upright position and goes about taking on the day in as fearless a fashion as possible, given what he must encounter now and the knowledge of what lies ahead.

Multiple sclerosis affects the central nervous system, the brain and the spine, and while the rate of degeneration differs for all, there is no escaping the degeneration. Ireland has 8,000 sufferers with apparently no cure.

Patrick, who was diagnosed in January, 2009, is not without hope however. A doctor, Denis Federenko, has been providing stem cell transplants to people from all over the world at the AA Maximov Hospital in Moscow, with some success, by wiping out the faulty immune system with drugs used to treat cancer and replacing it with stem cells taken from the patient.

Irish comedian, Stephen Garland is one who underwent treatment in November, 2016, having been told he was around six months away from being confined to a wheelchair.

He returned home just before Christmas that year and has thrived since, even writing a show about his journey to Russia and back.

Garland brought his creation, Post-Disposed, back to the world-renowned Edinburgh Fringe Festival in August, confirming his continuing improved health.

The treatment costs between 50,000 and 60,000 including aftercare and other expenses. It can be hard to ask for help but so many people have extended a hand to Patrick, without ever being asked. People Patrick doesnt know from Adam or Eve have even contributed to his cause and it moves him to tears.

Time is against the Corkman however, because if his condition exceeds 6.5 on a scale of 0-8, he will not be taken on. At present, he is between 5.5 and 6, and has been accepted, but clearly the treatment must take place sooner rather than later.

To that end, a Breakfast With The Stars event is taking place at The Park Hotel, Dungarvan on Thursday, October 10. Tipperarys All-Ireland-winning manager, Liam Sheedy and multiple champion jockey Davy Russell will regale patrons with stories from their careers, prompted by MC Marty Morrissey.

All proceeds will go towards funding Patricks treatment and ancillary expenses. Ticket purchasing details are below.

Here is more about Patricks story...

************

LAYLA walks into the family home in Ballinroad, just outside Dungarvan. It was Patrick who named her, after the Eric Clapton song that he happened to be listening to when the family were discussing what to call the now 12-year-old dog.

Music is a boon, a real infusion of energy, pumping the blood, making him feel like he could jump out of his skin and dance like the old days. He cant but that burst of adrenaline is a godsend.

I have the car adapted with hand controls, Patrick explains. I dont use my feet for driving. Push and pull the lever in the car.

On a Sunday morning I love nothing better, especially when I am down at home. Head down the coast road to Tramore, listen to a bit of music.

I would listen to absolutely anything. I am influenced by dads taste in music. All the older stuff, 70s, 80s rock music kind of stuff. You could find me listening to dance music two minutes later. Once it has a beat I couldnt care less.

Patricks dad, Richie Keane, was a hard worker, who was brilliant with his hands and especially with cars. And all his life he had a smaller shadow. The son looked up to the father like he was Superman.

When he was sick and on his last legs, talking through the window from the house he taught me how to take the sump off my car because it was cracked and leaking oil. He was able to guide me through it without looking at it, word for word.

I restored a Jeep that he left when he passed away. It was a 1983, same age as myself, Mercedes jeep. Very rare, like hens teeth. Restored it to about 25,000 to 30,000 worth.

I had to sell it. I couldnt drive it anymore because the leg was so bad. That was hard. That was the last connection. It was something I had to remember him by. The day I saw that go out the drive was tough.

Richie died in November, 2008, around the time of Patricks 25th birthday. Richies mother passed away two weeks later. Patrick was diagnosed with MS in January, 2009. Already a celiac and diabetic, he was accustomed to restrictions and putting up with things. This was a different stratosphere though.

It began with losing his balance and the development of numbness down his right side.

I went to the doctor, and I think, by the look on his face, he knew there was something not right. He sent me to Ardkeen (Hospital) and I got checked out. They had me in isolation for about a week. Then I was told it was MS.

I didnt know what it was but it was something I heard from a conversation when I was younger, That poor fella has MS. I didnt know I would end up the way I am now.

I have friends since that have been diagnosed after me that I have gotten to know from reaching out. There are a couple of them in a wheelchair. As it progresses, that is where you are heading. But there is a gentleman up the road and he must have it for 20-something years. Unless someone told you, you wouldnt know he has MS.

Part of the difficulty of dealing with MS is that no two cases are alike. Keeping active, having physiotherapy regularly and working are advantages and Patrick has not declined as quickly as others because of that.

But he has hit the secondary stage, where there are cognitive problems and, in particular, his short-term memory is affected.

People say to me, do you get pain with it? I dont know what new pain or old pain is. I just get on with. I have my days where I whinge and moan and cry. God knows I have them. But there is people out there worse off. I have what I have.

It could get worse. Now that it is gone to progressive MS, it probably will. When or how long? Who knows?

It is a horrible disease, there is no two ways. But you get up, get on with it, and do what you can.

He went to Australia not long after the diagnosis. It was a real gesture of defiance, one that probably scared his mother but he knew too that it might well be a case of now or never.

A blocklayer by trade, Patrick eventually had to give that up as his coordination worsened and he nearly fell off a roof. He is on crutches now for three years and would not be able to catch a football if you threw it at him. He wears a leg cast too, to reduce the instances of tripping himself up, without eradicating them totally. He has a mobility scooter.

A 35-year-old man doesnt dream about a mobility scooter, he wants a flash sports car. But it allowed me to go down to the Greenway for the first time. That was nice, to be on the Greenway, to be out, and see the whole lot of it, it was lovely. But watching other people cycle off down killed me.

My two nephews were out the front playing soccer. I used to do the exact same thing. They were saying Patrick, are you coming out to play? I said I would love to but that gets you. Its the simple little things.

Katie, my partner, I dont know where I would be without her. My mam, the girls and my brother, they are fantastic. They have been with me since the start of it.

I decided I want to be independent. If I didnt have the car I dont know what I would be doing. That is my freedom. I can get into the car, go to the shop for coffee. I will get there. I will get the same place as anyone else will, but it will take me longer.

He lives in Cork with Katie and her son Aaron, and works for Voxpro, who have been tremendously supportive. If he were housebound, he would wither.

I am an outgoing guy. I would chat away, waffle on about anything for hours on end. But you take that away I dont know have I changed since the diagnosis.

I have tried to remain as positive as possible but sometimes its hard. Simple little things you take for granted. Just run out there to get the clothes off the line. Now I have had to get handrails put into the house so that I can get up when I trip and fall.

I would wake in the mornings and the legs are like jelly. They shake, you cant control it, you let them shake out and that could be for 20 or 30 seconds. Real spasticity and stiffness in the legs. With the heat during the summer, I sat in the car with the air con on. I would be good for doing weather forecasta. I know what its like when I wake up in the morning, ever before the curtains are pulled. I feel it in my body.

He has been trawling for potential treatments, along with his medical team. Dr Federenkos work stands up to inspection. Stopping the MS in its tracks without the need for further medication would be a tantalising prospect.

Reversing the effects is something he dare not even contemplate, though the treatment has had that effect in Garland and many others.

Being given the green light, after Dr Federenko reviewed his case and medical records, was like an infusion in itself. Patrick details every step of the treatment and though it sounds daunting, it isnt compared to the alternative.

He set up the GoFundMe page (https://www.gofundme.com/f/stop-ms-progression-with-stem-cells-transplant) and was staggered by the reaction. Meanwhile, the local community has rallied, as have his work colleagues and friends, organising fund-raiser after fund-raiser. People he didnt know had events. He finds it hard to process.

To ask for someones help, it is a sign that you are not able. I have talked about this treatment for so long and I got so bad in my legs and balance and everything, I finally gave in and set up the GoFundMe page. It killed me to have to do it.

There was a donation yesterday on GoFundMe from Jamaica. I dont know the person but they found it in their heart to say, There you go. People said to me, Sorry I cant give you too much, I want to give you more. If it is a euro or 20 cent, it could be that 20 cent or euro that gets me over the line.

Whatever happens, he will not give up because it is what he learned when he was Richies shadow: It is instilled in me from dad, I would always have looked up to him. Even now, since he has passed away, I would always say to myself, What would he do? Would he have approved of that? He would always say to stick at something until you get it. If you are going to start something, do it. Just dont walk away from it.

Breakfast With The Stars, featuring Tipperary manager Liam Sheedy, one of the all-time greats of National Hunt racing Davy Russell and RTs Marty Morrissey, takes place at The Park Hotel on Thursday, October 10. To book a table of 10 for 1000 contact Michael Ryan (087 2585299) or The Local Bar (058 41854).

Donations can also be made to Patrick Keanes GoFundMe pagehttps://www.gofundme.com/f/stop-ms-progression-with-stem-cells-transplant

Read more:
I hope treatment will stop Multiple Sclerosis in its tracks - Echo Live

Stem Cell Therapy Market Synthesis and Opportunity Assessment 2019-2024 – Markets Gazette

Global Stem Cell Therapy Market 2019 by Manufacturers, Regions, Type and Application, Forecast to 2024:This report tracks the major market events including product launches, development trends, mergers & acquisitions and the innovative business strategies opted by key market players. Along with strategically analysing the key markets, the report also focuses on industry-specific drivers, restraints, opportunities and challenges in the Stem Cell Therapy market.

In the last several years, global stem cell therapy market developed fast at a average growth rate of 46.81%. In 2017, the global stem cell therapy market size was 235 million USD, and the market is expected to be 277 million USD.

On the basis of therapeutic application, the global stem cell therapy market is segmented into Musculoskeletal Disorder, Wounds & Injuries, Cornea, Cardiovascular Diseases, and other applications. The musculoskeletal disorders and Wounds & Injuries are expected to dominated the market.

According to this study, over the next five years the Stem Cell Therapy market will register a 20.7% CAGR in terms of revenue, the global market size will reach US$ 710 million by 2024, from US$ 280 million in 2019.

Top Companies in the Global Stem Cell Therapy Market: INTERSCIENCE, IUL,S.A., UVP, AID, BioMerieux, Schuett, Synbiosis, BioLogics, WTW, Bibby Scientific, SK-Electronics, SP Scienceware, KROWNUS, Instem, Rocker, Shineso, ORIENTOP, Wseen, Yalien, YLN others.

Click the link to Get a Sample Copy of the Report:

https://www.marketinsightsreports.com/reports/03051125289/global-stem-cell-therapy-market-growth-status-and-outlook-2019-2024/inquiry?source=nevadagreentimes&Mode=54

This report segments the global Stem Cell Therapy market on the basis of Type are:

On the basis of Application, the Global Stem Cell Therapy market is segmented into:

For comprehensive understanding of market dynamics, the global Stem Cell Therapy market is analyzed across key geographies namely:

North America (United States, Canada, Mexico)

Asia-Pacific (China, India, Japan, South Korea, Australia, Indonesia, Malaysia, Philippines, Thailand, Vietnam)

Europe (Germany, France, UK, Italy, Russia, Rest of Europe)

Central & South America (Brazil, Rest of South America)

Middle East & Africa (GCC Countries, Turkey, Egypt, South Africa, Other)

Avail Discount at (Special Offer: This report is available up to 10% discount for a limited time only):

https://www.marketinsightsreports.com/reports/03051125289/global-stem-cell-therapy-market-growth-status-and-outlook-2019-2024/discount?source=nevadagreentimes&mode=54

-Comprehensive assessment of all opportunities and risk in the Stem Cell Therapy market.

-Stem Cell Therapy market recent innovations and major events.

-Detailed study of business strategies for growth of the Stem Cell Therapy market-leading players.

-Conclusive study about the growth plot of Stem Cell Therapy market for forthcoming years.

-In-depth understanding of Stem Cell Therapy market-particular drivers, constraints and major micro markets.

-Favourable impression inside vital technological and market latest trends striking the Stem Cell Therapy market.

Buy Full Report

https://www.marketinsightsreports.com/report/purchase/03051125289?mode=su?source=nevadagreentimes&Mode=54

-Key Strategic Developments: The study also includes the key strategic developments of the market, comprising R&D, new product launch, M&A, agreements, collaborations, partnerships, joint ventures, and regional growth of the leading competitors operating in the market on a global and regional scale.

-Key Market Features: The report evaluated key market features, including revenue, price, capacity, capacity utilization rate, gross, production, production rate, consumption, import/export, supply/demand, cost, market share, CAGR, and gross margin. In addition, the study offers a comprehensive study of the key market dynamics and their latest trends, along with pertinent market segments and sub-segments.

-Analytical Tools: The Global Stem Cell Therapy Market report includes the accurately studied and assessed data of the key industry players and their scope in the market by means of a number of analytical tools. The analytical tools such as Porters five forces analysis, SWOT analysis, feasibility study, and investment return analysis have been used to analyze the growth of the key players operating in the market.

Finally, Stem Cell Therapy Market report is the believable source for gaining the Market research that will exponentially accelerate your business. The report gives the principle locale, economic situations with the item value, benefit, limit, generation, supply, request and Market development rate and figure and so on. This report additionally Present new task SWOT examination, speculation attainability investigation, and venture return investigation.

Customization of the Report: This report can be customized as per your needs for additional data up to 3 companies or countries or 40 analyst hours.

MarketInsightsReports provides syndicated market research on industry verticals including Healthcare, Information and Communication Technology (ICT), Technology and Media, Chemicals, Materials, Energy, Heavy Industry, etc. MarketInsightsReports provides global and regional market intelligence coverage, a 360-degree market view which includes statistical forecasts, competitive landscape, detailed segmentation, key trends, and strategic recommendations.

Irfan Tamboli (Head of Sales) Market Insights Reports

Phone: + 1704 266 3234 | +91-750-707-8687

sales@marketinsightsreports.com | irfan@marketinsightsreports.com

The rest is here:
Stem Cell Therapy Market Synthesis and Opportunity Assessment 2019-2024 - Markets Gazette

Tenaya Therapeutics bags $92M to develop triple threat for heart disease – FierceBiotech

Tenaya CEO Faraz Ali(Tenaya Therapeutics)

Although heart disease remains the leading cause of death worldwide, its an area that hasnt seen as much interest or investment as other areas, and its treatments still focus on dealing with symptoms. With a multipronged approach and a fresh infusion of $92 million, Tenaya Therapeutics is trying to change that.

The heart is a complicated organ, and it can go wrong in different ways. Part of what weve learned the hard way is that prior approaches are not working, Tenaya CEO Faraz Ali told FierceBiotech.

Founded in 2016 by scientists from the Gladstone Institute in California and the University of Texas Southwestern Medical Center, Tenaya is going after the underlying causes of heart disease to head off heart failure. It raised $50 million in its series A round from The Column Group, which also pitched into its $92 million B round alongside the likes of Casdin Capital and GV.

Like this story? Subscribe to FierceBiotech!

Biopharma is a fast-growing world where big ideas come along every day. Our subscribers rely on FierceBiotech as their must-read source for the latest news, analysis and data in the world of biotech and pharma R&D. Sign up today to get biotech news and updates delivered to your inbox and read on the go.

RELATED: GV leads $58.5M round for Verve, a startup looking to pit gene editing against heart attacks

In an age where companies are forming themselves around one platform thats disease-agnostic and can be applied to many different areas, South San Francisco-based Tenaya is doing the opposite. It's working on three different platforms for heart disease:regenerative treatments, gene therapies and precision medicines.

The company was founded with a big, bold mission to follow the science and use the right tool for the job, Ali said. If the problem is loss of cardiomyocytes (the muscle cells that make the heart beat), such as after a heart attack, we can look for a way to generate new myocytes, create new tissue and improve the ability of the heart to contract that way.

Thats where regenerative treatments come in. Tenaya's approach delivers transcription factors that can nudge heart fibroblasts, cells that play a role in scar formation after a heart attack, to become heart muscle.

If the problem stems from geneticsif a faulty gene doesnt cause heart muscle to die, but leads to an arrhythmia or scarring, stopping it from working properlythe solution is not to create new muscle, but to get the muscle thats there to work, Ali said. And thats where gene therapy, adding a healthy copy of a defective gene, comes in.

Finally, Tenayas taking a leaf out of the book of cancer drug developers: Its become quite the norm to look for therapies that work in a particular genetic background, he said. The companys precision medicine platform uses stem cell-derived heart muscle cells as disease models to identify new targets for heart failure and screen new drugs. Its first focus is on small molecules for the treatment of dilated cardiomyopathiesa group of conditions in which an enlarged heart chamber makes it less efficient at pumpingin genetically defined patient groups.

What the funding allows us to do is advance multiple promising projects from each of the platforms out of the pure research stage and into the clinic, Ali said. Tenaya hopes to move to human studies over the next few years.

RELATED: Renovacor bags $11M to push precision medicine for rare heart disease

Though the company isnt divulging just yet which targets its going after, Ali did say some of its programs are chasing more prevalent conditions such as heart attacks while others are looking at smaller, more defined populations.

Once we get a signal of efficacy and safety and advance into later-stage clinical development, we could potentially expand into larger, more prevalent indications, he said.

Tenaya has about 45 staffers who are mostly focused on research, early development and manufacturing. If all goes to plan, the company plans to double its size by the end of 2021, adding more employees in development and manufacturing.

Two-thirds of our platforms that were working on [gene therapy and regenerative treatments] are heavily dependent on viral vectors, adeno-associated viruses Everyone learned in the last decade or so that manufacturing is the Achilles heel of the gene therapy spaceit's difficult to do and highly technical, its nothing like small molecule manufacturing, Ali said. We made the decision to invest early, and well ahead of being in the clinic, to invest in manufacturing.

Tenaya isnt just doing it because other gene therapy players have proved it necessary. If it wants to go after larger heart disease populations rather than the smaller groups affected by rare disease, its going to need a lot of viral vector to deliver its treatments.

See more here:
Tenaya Therapeutics bags $92M to develop triple threat for heart disease - FierceBiotech