Stem cell therapy approved for MS patients in Scotland – HeraldScotland

MULTIPLE sclerosis campaigners have hailed a huge step forward for patients in Scotland after a stem cell therapy was recommended for use on the NHS for the first time.

Haematopoietic stem cell transplantation (HSCT) has been described as a game-changer for MS after an international clinical trial showed that it could reboot patients immune systems and halt the progress of the disease.

Some patients who had been in wheelchairs prior to treatment said their condition improved so dramatically it was like they had never been diagnosed with MS.

READ MORE: Scots MS patients 'missing out' on pioneering stem cell treatment available in England

The Scottish Health Technologies Group (SHTG) said there is now sufficient evidence for it to recommend making HSCT available on the NHS in Scotland to MS patients who have the relapsing-remitting form of the disease, and who were not responding to drug treatments.

Iain Robertson, chairman of the SHTG, said: Our committee members were able to advise that this treatment should be considered for those with this particular type of MS who have not responded to treatment with disease-modifying therapies.

We hope that our advice will be of use in helping decide the best course of treatment for these patients.

The SHTG also stressed that patients must be made aware of the demands, risks and uncertainties of the treatment, which uses chemotherapy to wipe out patients' 'faulty' immune systems before replenishing it with a transplant of stem cells harvested from their own bone marrow.

It puts patients at high risk from infections, which can be fatal, but the theory is that the treatment works by enabling patients to 'reset' their immune system to stop it attacking the central nervous system as is the case in MS.

READ MORE: Anger of Scots MS patients travelling abroad for stem cell therapy available to some on NHS England

HSCT is not considered an effective treatment for patients with the progressive form of MS, however, as stem cells cannot regrow nerves or repair damaged myelin - the protective sheath which coats nerves.

It will also be unavailable to patients with relapsing-remitting MS who no longer show signs of inflammation on an MRI brain scan.

Scotland has one of the highest rates of MS in the world, but until now Scottish patients seeking HSCT have had to travel overseas to Mexico, Russia and Israel and bankroll their own private treatment at a cost of around 40-60,000.

It has also been available privately in London since 2017, but with a 100,000 price tag.

A small number of MS patients in England have been able to access the treatment on the NHS, however, because there are clinical trials into HSCT taking place at NHS hospitals in Sheffield and London.

Morna Simpkins, director of MS Society Scotland, said: The decision from SHTG to approve HSCT for the treatment of MS is good news and could help in the development of a clear pathway, for people who could potentially benefit, to access it.

We will push to ensure that this decision leads to real change for people with MS by continuing to engage with other groups to offer the treatments, including HSCT, which are right for them.

READ MORE: Stem cells help mother with MS make 'remarkable' recovery

The SHTG said eligible patients must have equal access to the procedures regardless of where they live, but it is unlikely all health boards will be able to provide it.

The MS Society wants a centre, or centres, of excellence set up where patients from across Scotland can be referred.

Lucy Clarke from the Scottish HSCT Network said the recommendation was "a huge step forward" for people in Scotland living with MS.

Ms Clarke underwent HSCT in Russia and credits it with substantially reversing her disability.

She added: This important decision supports HSCT as a treatment option where other treatments have failed. We will continue to push so that this treatment is available to people in Scotland who need it.

A Scottish Government spokeswoman said: We are grateful to the Scottish Health Technologies Group for this important work.

"NHS Boards are expected to consider their advice on technologies in the planning and provision of its services and clinicians are expected to follow their professional judgement, working within the management structure of their Board.

We will work closely with MS Society Scotland, other third sector bodies and the clinical community to consider what the Technologies Groups findings means for provision in Scotland, including the information that needs to be available to people about eligibility and risks.

Read the original:
Stem cell therapy approved for MS patients in Scotland - HeraldScotland

Battle brews over how tightly to regulate new cancer treatment – Crain’s Detroit Business

In a letter to the commission, Republican State Sens. Curt Vanderwall, John Bizon and Mike Shirkey argued against regulation, saying it should be made available in as many places as possible, especially in northern Michigan and the Upper Peninsula, where no FACT accredited hospitals are located.

Vanderwall, who testified against the regulation and is chair of the Senate health policy committee, said he doesn't believe restricting access, especially in northern Michigan, is in the best interests of patients.

"I'm just very concerned right now that we're going to limit access to care," Vanderwall said at the Sept. 19 commission meeting. "I appreciate the cost of the drug. I understand that. As you know I work very hard to make sure and we have things that are going to address some of these drug costs. But in this situation I really feel that we need to make sure that we follow the federal standard and allow the free market to play."

Besides Vanderwall, seven other organizations objected to the proposed standards, including the Cancer and Hematology Centers of Western Michigan in Grand Rapids; the Alliance for Regenerative Medicine; the Biotechnology Innovation Organization; and Celgene, a biopharmaceutical company that manufactures one of the CAR T-cell medications.

Arguing in favor of regulation, Greg Yanik, M.D., a pediatric hematologist-oncologist at Michigan Medicine in Ann Arbor, estimated about 300 patients annually in Michigan would qualify for IECT therapy.

"We're not talking 3,000, we're not talking 30,000," Yanik said. "Do we want unregulated access where let's say 100 hospitals can each treat three patients? No. I really firmly believe that the SAC recommendations will probably end up with 10 to 20 hospitals and those with fairly large oncology group practices each treating 15 to 20 patients."

Yanik said in his 30 years as an oncologist, he has never seen such a toxic combination of agents involved in the therapy protocol. He said the potential benefits are great, but the risks are even higher.

"I'm Roman Catholic. Every patient I give CAR T or these IECT therapies to, right before I treat, I go like this (making the sign of the cross and saying a prayer)," he said. "That tells you how serious I feel about this therapy."

In March 2018, Karmanos became the first cancer center in Michigan to offer CAR-T therapy for an aggressive type of non-Hodgkin Lymphoma. The FDA approved CAR T-cell for lymphoma in October 2017.

Pam Darling, 65, a homemaker in Spring Lake, was one of the early recipients of CAR T-cell cancer treatment at Karmanos. She told Crain's in 2017 she found a lump on her neck and it went to her primary care physician, who recommended she see an oncologist.

After three outpatient chemotherapy treatments and then a radiation treatment, Darling said, the cancer, diffuse large B-cell lymphoma (DLBCL), the most common type of non-Hodgkin lymphoma, became very aggressive.

"I looked at stem cell transplant therapy, but that didn't go through. My oncologist recommended CAR T-cell and I was told by Blue Care Network that Karmanos was the only hospital doing that at the time," Darling said.

In May 2018, Darling began treatment at Karmanos. She spent three weeks at Karmanos, the last two in the intensive care unit.

"I was totally out of it. I had no idea where I was. I couldn't breathe, and they put me on a ventilator," said Darling, adding that after she was discharged she had to stay at a hotel room at MotorCity Casino for four days until Karmanos doctors cleared her to travel home.

"I took a CT scan in May, and they said there was no cancer there. It could come back, but (Abhinav Deol, M.D) said if I passed the year mark with no cancer, I have a good chance at it not returning," Darling said.

On whether she believes hospitals should be tightly regulated, Darling said that is a tough question to answer.

"I would want a place like Karmanos to go get this treatment. It is very difficult, and I was impressed with the medical team," she said. "It was expensive to travel. I will tell you that. I wished I could have it done in Grand Rapids, if they had it at the time. Before, I probably would have wanted to go to a hospital that is closer. But knowing what I know now, I would want it to be" like Karmanos.

Deol said CAR T-cell therapy is a one-shot treatment that has a good chance at putting the patients' disease in remission. But because it is a new procedure, data is scant on success rates.

"Following CAR T therapy, we're seeing initial positive response rates in the range of 80 percent; and long-term response rates, approximately one year or longer, up to 45 percent," Deol said in a statement. "As more data becomes available, we hope to see an even longer time in remission."

Go here to see the original:
Battle brews over how tightly to regulate new cancer treatment - Crain's Detroit Business

Quadruple Therapy for Newly Diagnosed Patients With MM Shows PFS Benefit Over Triple Therapy – AJMC.com Managed Markets Network

Quadruple therapy for newly diagnosed patients with multiple myeloma (MM) led to significantly longer progression-free survival (PFS), according to an abstract presented at the 17th International Myeloma Workshop 2019 in Boston, Massachusetts, last month.

Compared with triple therapy, quadruple therapy was associated with deep responses before and after autologous stem cell transplantation (ASCT), with a significant PFS gain across all risk groups.

Given MMs significant spatial and temporal clonal heterogeneity, researchers believe that therapeutic agents with different mechanisms of action are required to improve both response and outcomes. These agents may be administered in combination or sequentially.

The UK NCRI Myeloma XI phase 3 trial compareda standard chemotherapy regimen of cyclophosphamide, dexamethasone plus thalidomide with a newer regimen of cyclophosphamide, dexamethasone plus lenalidomide with or without carfilzomib. Patients who did not have the best response might be randomized to receive a combination of cyclophosphamide, dexamethasone plus bortezomib. Patients may have also received more intensive chemotherapy, along with ASCT.

After maximal response was achieved, patients were treated with either long-term lenalidomide, lenalidomide with vorinostat, or receive no further treatment.

The intensive induction phasecarfilzomib, lenalidomide, cyclophosphamide, and dexamethasone (KCRD)was compared with a response-adapted approach of sequential triplet therapies in newly diagnosed transplant-eligible patients.

Carfilzomib, a selective proteasome inhibitor (PI), is approved for the treatment of patients with relapsed or refractory MM; combination regimens incorporating a PI and immunomodulatory drug (IMiD) have been associated with deep responses and extended survival in patients with newly diagnosed MM.

In the study, 1056 patients were randomized between KCRD (28 day cycles of carfilzomib 36 mg/m2 given intravenously days 1-2, 8-9, and 15-16; cyclophosphamide 500 mg PO day 1 and 8; lenalidomide 25 mg days 1-21; dexamethasone 40 mg days 1-4, 8-9, and 15-16), and IMiD triplet cyclophosphamide, thalidomide, and dexamethasone (CTD)/cyclophosphamide, lenalidomide, and dexamethasone (CRD) prior to ASCT.

Patients with a suboptimal response to CTD/CRD underwent response-adapted intensification randomization to a different PI (bortezomib, plus cyclophosphamide and dexamethasone, a combination known as CVD) containing triplet or no CVD. A maintenance randomization at 3 months post-ASCT compared lenalidomide with observation.

Molecular high-risk (HiR) was classified by t(4;14), t(14;16), t(14;20), del(17p) or gain(1q) with ultrahigh risk (UHiR) the presence of >1 lesions.

KCRD treatment was linked with a significantly longer PFS than IMiD triplet therapy (hazard ratio [HR] 0.63; 95% CI 0.51-0.76; 3 year PFS KCRD 64.5% vs CTD/CRD 50.3%; P <.0001). There was no significant toxicity due to the quadruplet regimen.

In addition, a higher proportion of patients receiving KCRD induction were able to undergo ASCT than those who received response-adapted induction. In an analysis restricted to those who had completed ASCT, KCRD induction was still associated with a significantly longer PFS.

There was no significant difference in PFS outcome between molecular risk groups, with a benefit for quadruple therapy over triplet therapy.

In patients receiving KCRD there was no difference in response rate at the end of initial induction by risk group, but UHiR disease was associated with significantly shorter PFS than both patients with standard risk (SR) and HiR. There was no difference in outcome between patients with HiR (1 adverse lesion only) and SR.

An exploratory analysis compared the patients receiving KCRD to patients in the CTD/CRD arm who had received the optimum response-adapted approach (ie excluding those with a suboptimal response randomized to no CVD).

KCRD was associated with significantly longer PFS than using a response adapted sequential triplet approach (HR 0.64; 95% CI 0.52-0.78;P .0001).

Reference

Pawlyn C, Davies F, Cairns D, et al. Quadruplet KCRD (carfilzomib, cyclophosphamide, lenalidomide and dexamethasone) induction for newly diagnosed myeloma patients. Presented at: 17th International Myeloma Workshop; Boston, Massachusetts September 12-15, 2019. Abstract OAB-002.

Link:
Quadruple Therapy for Newly Diagnosed Patients With MM Shows PFS Benefit Over Triple Therapy - AJMC.com Managed Markets Network

Some animals pause their own pregnancies, but how they do it is still a mystery – The Conversation AU

Putting your pregnancy on pause until the time is right to give birth sounds like something out of a sci-fi novel, but for many mammals whats known as embryonic diapause is an essential part of raising their young.

Although scientists have known since the 1850s that some animals have this ability, it is only now becoming clear how it could teach us valuable lessons about human pregnancy, stem cells, and cancer.

More than 130 species of mammal can pause their pregnancies. The pause can last anywhere between a couple of days and 11 months. In most species (except some bats, who do it a little later) this happens when the embryo is a tiny ball of about 80 cells, before it attaches to the uterus.

Its not just a single group of mammals, either. Various species seem to have developed the ability as needed to reproduce more successfully. Most carnivores can pause their pregnancies, including all bears and most seals, but so can many rodents, deer, armadillos, and anteaters.

Read more: What marsupials taught us about embryo implantation could help women using IVF

More than a third of the species that take a breather during gestation are from Australia, including some possums and all but three species of kangaroo and wallaby.

The record-holder for pregnancy pause time is the tammar wallaby, which has been studied extensively for its ability to put embryos on hold for up to 11 months.

The main advantage to pausing pregnancy is that it separates mating and birth. There are two main ways in which animals do this.

The first way is to mate soon after giving birth, to have a backup pregnancy in case something happens to the newborn young. The stress of lactating triggers a pause that lasts during suckling, and the pregnancy restarts once the young leave.

The second way is to pause every pregnancy until the time is right (usually depending on the season). For example, minks mate around the start of March but put the embryos on pause until after the spring equinox (March 21), when the days are growing longer in their northern hemisphere homes. This ensures that the young are born in spring when conditions improve, and not in winter.

The tammar wallaby combines these two methods (suckling in the first half of the year, short days in the second) to pause for almost a year and give birth in January. This ensures the young leave the pouch the following spring instead of in the middle of a hot Australian summer.

Diapause was first identified in 1854 after hunters in Europe noticed that pregnancy in roe deer seemed to last a lot longer than normal. Since then scientists have been fascinated by this process and it has helped us understand more about basic reproductive processes in all mammals.

But it took until 1950 before our knowledge of pregnancy had increased enough so that we could confirm what the hunters had observed 100 years earlier.

But how the process worked at the molecular level is still a mystery. Until recently, there seemed to be no connection between which species used it and which didnt and there didnt seem to be a unifying mechanism for how pregnancy was paused. Even the hormones controlling diapause are different between mammal groups.

However, research now suggests that regardless of what hormones affect the uterus, the molecular signalling between the uterus and the embryo is conserved, at least between the mouse, mink and tammar wallaby.

Furthermore, researchers in Poland paused embryos from sheep (a non-diapause species) by transferring them into a mouse uterus and then back into the sheep with no ill effects.

This indicates the potential for diapause could lie in all mammals, including humans.

Its unlikely that pausing pregnancy will become the norm in humans. For starters, youd have to know you were pregnant within five days of conceiving to match the time when most species start diapause.

Understanding how mammals pause their pregnancies does have significant implications for our understanding of how to make healthy embryos. The time when the embryo enters into diapause is the same time in IVF when an embryo is transferred into the uterus. Diapause could help us improve how we grow embryos in culture or how to recognise which is the best embryo to transfer.

Read more: Explainer: what are stem cells?

Diapause could also help create better stem cells and find new cancer treatments. The first stem cells ever isolated by scientists came from a mouse embryo in diapause, when the cell cycle of the embryo is arrested. Stem cells are also remarkably similar to a diapaused embryo.

So understanding how diapause works at the molecular level could lead to new therapies to halt cell division or to identify markers for tumour stem cells, which are thought to be responsible for metastasis in cancer.

Follow this link:
Some animals pause their own pregnancies, but how they do it is still a mystery - The Conversation AU

Hormones Control your Health, Mood and Behavior A balanced hormone means happier, healthier life and success in career and relationship. – Magazine of…

Non-surgical regenerative cell-based treatment uses the bodys natural healing ability to repair damaged bones, muscles, cartilage, tendons and ligaments.Knee injuries are painful and often patients are unable to walk. Our treatment protocol always uses products following FDA guidelines.Injections done with ultrasound guided needle recognition capability to ensure safety as well target the area needing treatment. Plasma; Alpha-2-Macroglobulim (A2M) is the new biologic treatment for your arthritic knee (osteoarthritis)When your hips hurt, or your knee is stiff, or your back is throbbing, that means your joint is bone on bone and there is no lubrication to ease movement.Regenerative medicine giving new hope to patients suffering from painful joint injuries such as knee, shoulder and hip with a chance to live a pain free life.Regenerative cell-based ultrasound guided injection now available to treat pain associated with joint injury. There are indications that it reduces the pain and swelling of the joints and helps lubricating and improve movements.Commonly Treated Conditions: Osteoarthritis of the Hips, Knee, and Shoulders Rotator Cuff tears of the Shoulder Meniscus, ACL and PCL tears of the kneeOur stem cell treatment using your own stem cells and with using imaging guidance ensures precise injection of stem cell, it is a highly-specialized practice.Besides treating above injuries we have advance stem cell micro-needling treatment for the following: Cell-based PRP Hair Restoration combining micro-needling with growth factors and hair follicles voluma vitamins plus BLotinyl T1, Biotin, Anti-aging and Kopexil. Non-toxin facial renewal Anti-Aging APGF Advanced Peptide Micro-needling PRP, Dual Anti-Aging Ampoules for deep hydration, more collagen to reduce wrinkles and firm skin.Dr. Ibrahim is the staff physician at Valencia Medical Center specializing in regenerative medicine, pain management, and rejuvenation. Call for a consultation at 661-222-9117.

Visit link:
Hormones Control your Health, Mood and Behavior A balanced hormone means happier, healthier life and success in career and relationship. - Magazine of...