Stem cell treatment in the UAE sees ‘favorable’ outcomes for coronavirus patients – CNBC

A man wears a protective mask as he exercises on May 03, 2020 in Dubai, United Arab Emirates.

Francois Nel | Getty Images

A new treatment that helps to relieve coronavirus symptoms could be brought to market in three months' time if further trials go well, according to a researcher involved in the project.

"It's very early to say at this stage," said Dr.Fatima al-Kaabi, head of hematology and oncology at the Sheikh Khalifa Medical City in the United Arab Emirates.

"We've been happy that our initial safety results are promising, that's why we're heading into the next phase, of effectiveness of this treatment," she told CNBC's Hadley Gamble on Monday.

"If all ... went well and it worked well, then I would propose ... three months' time," she said, when asked how quickly the treatment, which was developed by doctors and researchersat the Abu Dhabi Stem Cell Center, could reach the market.

To date, there are no known vaccines or specific antiviral medicines against Covid-19.U.S. health officials say developing a vaccine will take at least 12 to 18 months.

The UAE has 14,163 cases and126 deaths due to the coronavirus, based on data from Johns Hopkins University.

The remedy uses a "minimally invasive" method where a Covid-19 patient's stem cells are extracted, activated and turned into a fine mist to be inhaled. This alleviates symptoms such as shortness of breath and possibly coughing, said Dr. al-Kaabi.

"It is hypothesized to have its therapeutic effect by regenerating lung cells and modulating the immune response to keep it from overreacting to the COVID-19 infection and causing further damage to healthy cells,"the UAE's ministry of health and preventionsaid a statement.

Some 73 patients with moderate to severe symptoms received this treatment, and all were "successfully treated and cured," the statement said, adding that none reported "immediate adverse effects." Around a quarter of these patients were intubated and in the intensive care unit.

The treatment was given along with "conventional medical intervention" and will not replace established protocols, according to the statement.

"We're hopeful," said Dr. al-Kaabi, noting that the results of further trials on the efficacy of the treatment will only be out a couple of weeks' time. "We've seen (a) favorable outcome."

Another treatment for the coronavirus, an antiviral drug from Gilead Sciences, has been in the spotlight following positive preliminary results from trials. America's Food and DrugAdministration granted the medicine emergency use authorization last week. That means doctors can administerremdesivir to patients hospitalized with Covid-19, even though the drug has not undergone the same FDA review as other treatments.

Separately, researchers cut short a study testing anti-malaria drug chloroquine as a potential Covid-19 treatment last month. The drug gained widespread international attention after two small studies published in France found the coronavirus infection cleared a lot faster for patients taking it when compared to a control group.

However, citing a high risk of death, scientists have now scrapped the trials, warning it should prompt some degree of skepticism from the public toward enthusiastic claims of the drug. President Donald Trump had touted chloroquine as a potential "game changer" in the fight against the virus.

CNBC'sBerkeley Lovelace Jr. andWilliam Feuer contributed to this report.

The rest is here:
Stem cell treatment in the UAE sees 'favorable' outcomes for coronavirus patients - CNBC

Coronavirus Update: Baptist Hospital Patient Credits Stem Cell Treatment With Saving Her Life – CBS Miami

MIAMI (CBSMiami) The U.S. Food and Drug Administration gave Baptist Health approval to test a stem cell treatment on COVID-19 patients and so far its come back with positive results.

The treatment has proven successful with three patients.

One of those patients, Ruth Ramirez says it saved her life.

Ramirez was discharged from the hospital on Friday afternoon.

They saved my life. They definitely saved my life, said Ramirez.

Ramirez recently received stem cells from an umbilical cord known as mesenchymal cells.

Mesenchymal stem cells have the ability to reduce cytokine levels, said doctor Guenther Koehne.

Baptist Hospital says patients like Ramirez showed a reduction of their oxygen requirement from 100% to less than 50% within days of the infusion, accompanied by a significant reduction in levels of various key circulating inflammatory markers.

I couldnt breathe. I had a fever a headache I was nauseous, said Ramirez.

Ruth, an employee of the Miami Cancer Institute, tested positive for COVID-19 back on April 7th. She was admitted to the ICU and ended up on a ventilator fighting for her life.

Knowing she may lose consciousness she gave her sister power of attorney. That is when doctors Koehne and Javier Perez Fernandez approached the family about this FDA approved experimental therapy.

Im a person who jumps. I jump with hope with the best outcome there is on the other side. I think she took that into consideration with my characteristic and said Ruth would probably do this.

According to friends, Ruth was in ICU for three weeks, on a ventilator, and unable to breathe on her own.

All that time, she was away from her two small kids. That was several days ago.

On Friday, she was discharged.

To be here in this room, alone, and not being able to hold them. It was hard you know its hard.

As soon as they told me that I was going home I was like what?? And that I tested negative again I was like wait!! That changed my mood completely

Still fuzzy on the timeline, Ruth says shes unsure where along the way she received the treatment but is thankful for the doctors and wants others to know there is hope.

You know I hear the bells here all the time the eye of the tiger thats the song that you walk out of when you come out with coronavirus. Its such a pleasure hearing it all the time now. More than I heard it yesterday.

Not only did the doctors step up, but so did her co-workers who set up this Ruthie-strong go fund me page to help with bills and expenses.

They also helped take care of her family.

They would send food to them. Groceries. My kids were taken care of my sister was taken care of. She didnt have to leave from the house.

Theyre just amazing.

The University of Miami is also working on a clinical trial using mesenchymal cells which we reported on in April.

Click here if you would like to donate to her GoFundMe page.

RELATED:Drive-Through Testing LocationsTrack The Spread Of The Coronavirus In Real TimeTips To Protect Your Vehicle While It Sits Unused During Coronavirus Stay-At-Home Ordercoronavirus

Follow this link:
Coronavirus Update: Baptist Hospital Patient Credits Stem Cell Treatment With Saving Her Life - CBS Miami

Investigational new drug may be beneficial in treating severe COVID-19 – BioNews

4 May 2020

A novel stem cell therapy may play a part in improving the survival rate of ventilator-dependent COVID-19 patients.

Twelve COVID-19 patients at Mount Sinai Hospital in New York were treated with a new type of stem cell therapy, resulting ina survival rate of 83 percent, significantly higher than the 12 percent survival rate for similar untreated patients.

Dr Keren Osman who led the team Mount Sinai Hospital, New Yorktold CBS News: 'What we saw in the very first patient was that within four hours of getting the cells, a lot of her parameters started to get better'.

Ryoncil (remestemcel-L) comprises culture-expanded mesenchymal stem cells (MSCs):rare cells that secrete factors that promote tissue repair and modulate an immune response.

The initial trial of Ryoncil with COVID-19 patients included two intravenous infusions of the drug over five days. Ten of the total 12 patients were able to come off ventilators after treatment. All patients had received other experimental therapies before receiving Ryoncil.

Dr Osman said 'we don't know if the ten people taken off ventilators would not have recovered if they had not been given the stem cell treatment and we would never dare to claim that it was related to the cells A randomised controlled trial would be the only way to make a true comparison.'

A300-person randomised, controlled clinical trial is now planned to determine the safety and efficacy of Ryoncil in the treatment of COVID-19 patients suffering from acute respiratory distress syndrome (ARDS), which results when the patient is unable to get enough oxygen and usually requires the use of a ventilator.

The overreaction of the immune system responsible for these effects is often termed a 'cytokine storm', where the systemic response to destroy the virus ends up damaging the infected lung tissue. Cytokines are small proteins released from certain cells in the immune system and play a role in cell signalling. Therapeutic use of Ryoncil is thought to down-regulate pro-inflammatory cytokine production and stimulate increased production of anti-inflammatory cytokines.

Ryoncil is currently under review by the US Food and Drug Administration (FDA) for the treatment of acute graft versus host diseaseand other rare diseases. The drug has been evaluated through several clinical trials, totalling over 1100 patients, including evidence of improved lung function in patients with similar biomarkers to COVID-19 patients with ARDS.

Go here to read the rest:
Investigational new drug may be beneficial in treating severe COVID-19 - BioNews

Subcutaneous Formulation of Daratumumab Approved for Multiple Myeloma – Cancer Therapy Advisor

The Food and Drug Administration (FDA) has approved Darzalex Faspro (daratumumab and hyaluronidase-fihj; Janssen) for the treatment of newly diagnosed or relapsed/refractory multiple myeloma in adult patients.

Darzalex Faspro is a new subcutaneous (SC) formulation that contains daratumumab, a CD38-directed cytolytic antibody, with hyaluronidase, an endoglycosidase. The new SC formulation is administered over approximately 3 to 5 minutes compared with the intravenous (IV) formulation of daratumumab (Darzalex) that is administered over hours. Darzalex Faspro is indicated for the treatment of adult patients with multiple myeloma:

The approval was based on data from the phase 3 COLUMBA and the phase 2 PLEIADES studies. The open-label, noninferiority COLUMBA study evaluated the efficacy and safety of Darzalex Faspro monotherapy in 522 patients with refractory or relapsed multiple myeloma. Patients were randomized to receive either Darzalex Faspro (1800mg/30,000 units) SC or daratumumab 16mg/kg IV once weekly until unacceptable toxicity or disease progression. Results showed that Darzalex Faspro achieved noninferiority with an ORR of 41.1% compared with 37.1% for daratumumab (risk ratio [RR] 1.11; 95% CI, 0.89-1.37). The geometric mean ratio comparing Darzalex Faspro to daratumumab IV for maximum Ctrough was 108% (90% CI, 96-122).

Additionally, the multicenter, single-arm, phase 2 PLEIADES study assessed the efficacy and safety of Darzalex Faspro in combination with bortezomib, melphalan and prednisone in 67 patients with newly diagnosed multiple myeloma who were ineligible for transplant. Findings from this group of patients showed an ORR of 88.1% (95% CI, 77.8-94.7). Moreover, in PLEIADES, the combination of Darzalex Faspro with lenalidomide and dexamethasone was evaluated in 65 patients who had received at least 1 prior line of therapy. Results from this group showed an ORR of 90.8% (95% CI, 81.0-96.5).

With regard to safety, Darzalex Faspro demonstrated a similar profile to that observed with intravenous daratumumab. Moreover, treatment with Darzalex Faspro was associated with a significant reduction in systemic administration-related reactions compared with intravenous daratumumab (13% vs 34%, respectively).

Since the approval of daratumumab, a robust body of evidence has established its use as a treatment for multiple myeloma in both the frontline and relapsed and refractory settings, said Saad Z. Usmani, MD, Division Chief of Plasma Cell Disorders, Levine Cancer Institute. With Darzalex Faspro there may be fewer administration-related reactions compared to intravenous Darzalex, providing an additional treatment option that may help patients, oncologists and nursing staff.

Darzalex Faspro is expected to be available the week of May 11, 2020 in single-dose vials containing 1800mg daratumumab and 30,000 units hyaluronidase per 15mL. To prevent medication errors, the vial labels should be checked to ensure that the drug being prepared and administered is for subcutaneous use; Darzalex Faspro should not be administered intravenously.

The Company will also offer a copay savings card to assist patients.

For more information visit darzalex.com.

This article originally appeared on MPR

More:
Subcutaneous Formulation of Daratumumab Approved for Multiple Myeloma - Cancer Therapy Advisor

Sam back home in Limehouse after treatment donating stem cells to help a patient beat blood cancer – East London Advertiser

PUBLISHED: 13:06 04 May 2020 | UPDATED: 13:18 04 May 2020

Mike Brooke

Sam in the London Clinic... after being matched to save a patient's life in worldwide appeal for stem cell donors. Picture: DKMS charity,

DKMS

A life-saving transplant procedure for Sam Schmidt to donate some of his blood stem cells to help save a patients life has gone well.

Email this article to a friend

To send a link to this page you must be logged in.

The 24-year-old accounts manager from Limehouse was finally found to be a compatible match for a patient in a worldwide search after registering with a stem cell charity a year ago.

He underwent the procedure at The London Clinic before the weekend to donate stem cells after being successfully matched with a blood cancer patient.

He felt strange being tested due to Covid-19 with everyone having to wear masks and gloves, but felt lucky to have the chance potentially to save someones life and help a family.

Sam registered with the DKMS charity, known as We Delete Blood Cancer, when he moved to Narrow Street in Limehouse last year from west London.

He was inspired after learning about 42-year-old Peter McCleave, a father of two in Cheshire diagnosed with blood cell cancer and given a few years to live.

Peter has launched his own search for donors with his 10,000 people campaign while still waiting for a match. He has attracted potential donors to register with the DKMS Foundation, but so far has found no match to save his own life.

If you value what this story gives you, please consider supporting the East London Advertiser. Click the link in the yellow box below for details.

Read this article:
Sam back home in Limehouse after treatment donating stem cells to help a patient beat blood cancer - East London Advertiser

Outcomes of Stem Cell Transplantation in Patients With Newly Diagnosed Transformed Fanconi Anemia – Hematology Advisor

Patientswith newly diagnosed transformed Fanconi anemia (FA) have poor outcomes andshould achieve complete remission (CR) prior to allogeneic hematopoietic stemcell transplantation (alloHSCT), according to results from a study published inthe American Journal of Hematology.

Stefano Giardino, MD, of the hematopoietic stem cell transplantation unit at the Istituto Giannina Gaslini in Italy, and colleagues retrospectively analyzed outcomes of 74 patients with transformed FA (36 male; median age, 14 years); 35 patients had myelodysplastic syndrome, 35 had acute leukemia, and 4 patients had high-risk cytogenetic abnormality. All patients underwent alloHSCT from 1999 to 2016.

The primary end points were overall survival (OS) and event-free survival (EFS). Secondary endpoints included the incidence of grade 2 to 4 acute graft-vs-host disease (AGVHD) and chronic graft-vs-host disease (CGVHD), non-relapse mortality (NRM), and incidence of relapse. To identify potential factors that may influence outcomes, the researchers assessed the type of diagnosis, preHSCT cytoreductive therapies and related toxicities, disease status prior to HSCT, donor type, and conditioning regimen.

Ata median follow-up of 7 years, 5-year OS and EFS were 42% and 39%,respectively; 5-year cumulative incidence relapse and NRM rates were 21% and40%, respectively. Patients in CR during transplant had better OS than those whostill had active disease (OS, 71% vs 37%, respectively; P =.04). No other factors had a significant effecton patient outcomes.

Of22 patients who received cytoreductive therapy prior to HSCT, 40.9% experienceda grade 3 to grade 4 toxicity event; this did not appear to effect survivalafter HSCT (3 year OS, toxicity preHSCT 48% vs no toxicity 51%; P =.98).At 100 days, the cumulative incidence of grade 2 to 4 AGVHD was 38%, and the cumulativeincidence of 5-year CGVHD was 40%.

At5 years, NRM was 40%, while incidence of relapse was 21%. Transplant-relatedevents were the cause of mortality in 81% of patients (34 of 42 deaths).

Limitationsof the study included the retrospective design and incomplete data for somevariables. The authors highlighted the large number of patients for this raredisorder as the primary strength.

Inorder to optimize the chances of the only curative option for FA in malignanttransformation, a sequential cytoreductive therapy followed by HSCT appears areasonable approach in FA patients with AL, if a previously identified donor israpidly available, wrote the authors. However, since the risk oftreatment-related complications is high, these patients should be managed inhighly specialized centers and transplant approaches aimed at reducing theoccurrence of [GVHD] and transplant-related complications should be prioritized.

Reference

Giardino S, Latour RP, Aljurf M, et al. Outcome of patients with Fanconi anemia developing myelodysplasia and acute leukemia who received allogeneic hematopoietic stem cell transplantation: a retrospective analysis on behalf of EBMT group [published online April 8, 2020]. Am J Hematol. doi: 10.1002/ajh.25810

Read the original post:
Outcomes of Stem Cell Transplantation in Patients With Newly Diagnosed Transformed Fanconi Anemia - Hematology Advisor