Stem Cell Clinic

MGTA-456 Earns Regenerative Medicine Advanced Therapy Designation – Adrenoleukodystrophy News

TheU.S. Food and Drug Administration (FDA) has granted Magenta Therapeuticscell therapy MGTA-456 the Regenerative Medicine Advanced Therapy (RMAT) designation for treating inherited metabolic disorders, including cerebral adrenoleukodystrophy.

The RMAT designation is a program established to help expedite the development and approval of promising therapies. It isgiven to products that have preliminary clinical evidence of being able to treat, modify, or cure a serious or fatal disease, and potentially address unmet medical needs for that disease.

This RMAT designation was based on the encouraging clinical data we have presented thus far, and it is an important milestone that recognizes the transformative, life-saving potential of MGTA-456 for children suffering from inherited metabolic disorders, John Davis, MD, chief medical officer at Magenta Therapeutics, said in press release.

MGTA-456 is a stem cell-based therapy specifically,allogenic hematopoietic stem cell therapy (HSCT) designed to help stop the progression of inherited metabolic disorders. This is possible through the delivery of a high-dose of stem cells to help regenerate the patients immune system. The procedure requires patients to receive a transplant of healthy blood-forming cells, orhematopoietic stem cells (HSCs), from a genetically identical donor (allogenic), to replace their own diseased cells.

MGTA-456 is being developed as a treatment for multiple diseases.

The therapy is currently being evaluated by Magenta in a Phase 2 clinical trial (NCT03406962) in patients with various genetic metabolic disorders, including cerebral adrenoleukodystrophy (cALD), Hurler syndrome, metachromatic leukodystrophy, or globoid cell leukodystrophy. All participants are older than 6 months.

This Phase 2 clinical trial is being conducted at four U.S. medical centers: Cincinnati Childrens Hospital, Duke University, Emory University, and the University of Minnesota.

Previous results from the first five patients two of them with cALD who were followed for six months, showed a rapid and consistent benefit withMGTA-456 treatment that was not seen with other investigational therapies.

Patients with cALD, in particular, were found to have resolution of brain inflammation on magnetic resonance imaging (MRI) scans, as early as 28 days after the treatment.Additionally, patients had stable neurological function scores at six months post-treatment, suggesting that the progression of the disease was halted.The Loes score, which quantifies the severity of brain abnormalities and atrophy, also was stable at six months.

All five patients analyzed achieved the primary goal of neutrophil engraftment, in which levels of neutrophils (a type of immune cell) have reached an absolute count of 500 or more neutrophils per cubic millimeter of blood for three consecutive days. Of note, a robust neutrophil engraftment means that the transplant of the stem cells was succeeded.

Following the success of this trial, Magenta expects to present further data before the end of 2019.

We look forward to collaborating closely with the FDA as we seek to rapidly advance MGTA-456 through the ongoing Phase 2 study, and into potential pivotal studies in 2020, Davis said.

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MGTA-456 Earns Regenerative Medicine Advanced Therapy Designation - Adrenoleukodystrophy News

Equine Tendon Injuries: Reducing the Strain – TheHorse.com

Equine athletes can sustain a variety of orthopedic injuries, but tendonswhich attach muscle to boneare especially prone to strain and damage. Superficial digital flexor tendon injuries remain a frequent and frustrating cause of morbidity (disease) in athletic horses, having been noted as the primary reason for retirement of Thoroughbred racehorses over a 12-year period (Lam 2007). The initial tendon injury leads to not only pain and an inability to work but also re-injury rates as high as 82% (Genovese 1997). Subsequently, tendon healing strategies and optimized rehabilitation have become big-ticket items for the equine sports medicine community.What Do I Do if I Suspect an Injury?

The classic signs of tendon injury in horses include general symptoms of inflammation: heat, pain, and swelling. Depending on where the injury is located, you might not detect obvious swelling or heat (for example, deep digital flexor tendon injuries within the hoof capsule dont cause overt swelling and are best diagnosed with MRI). Tendon injuries of the mid-metacarpal (or cannon bone) region, however, often cause noticeable swelling, heat, and pain upon palpation. Initially, tendon-associated lameness can be severe, depending on the extent of the injury and its severity and chronicity.

If you suspect your horse has a tendon or soft-tissue injury, connect with your veterinarian immediately to get an appropriate diagnosis and begin therapy. Most commonly, a complete musculoskeletal and lameness evaluation will help the veterinarian determine which soft tissue structures are involved. Your vet might recommend an ultrasound evaluation as a first-line imaging tool to assess tendinous tissue, but MRI evaluation might also be indicated in some cases to further characterize the injury. Once your veterinarian has made a diagnosis, you can discuss treatment options to get your horse on the road to recovery.

The bodys intrinsic tendon repair process involves three continuous phases. The initial inflammatory phase typically occurs during the first one to three days following injury. Initial triage strategies usually consist of decreasing inflammation using cryotherapy (icing), rest, anti-inflammatories, and supportive care. The subsequent phases of repair (two to 28 days) and remodeling (60 days onward) are characterized by tissue reorganization and scar tissue formation. Once you and your veterinarian have gotten the initial pain and inflammation under control, you can aim rehabilitation strategies toward encouraging organized scar tissue formation that more closely resembles the tendons uninjured architecture.

Several treatment options are available for tendon injuries, yet no singular one-size-fits-all therapy currently exists. Researchers have described significant success using mesenchymal stem cells to treat tendon injuries in racehorses, with the most notable statistic being a significantly reduced re-injury rate. Despite the exciting research around biologic treatments such as these, controlled exercise and physical therapy remain the most important cornerstones of tendon healing.

Eccentric loading exercises have been shown to be particularly effective. These involve lengthening the muscle or tendon fibers while theyre load-bearing. Take, for example, the down phase of a biceps curl or a heel raise in humans. While the exact mechanism behind its therapeutic effect remains unknown, researchers believe this controlled mechanical stimulation helps the bodys scar tissue become more organized and functional.

Horses most commonly perform eccentric loading through assisted physical therapy exercises such as the withers pull (applying gentle lateral pressure to the side of the withers until the horse shifts his weight subtly toward your pull; hold that stretch for 3-5 seconds).

Similarly, controlled forelimb loading can help strengthen the soft tissues of the entire limb, including the supportive musculature of the shoulder, elbow, carpus (knee), and digit. Other eccentric physical therapy aids include surface changes, ground pole work, backing exercises, and modifications of the withers-pull exercise. Your veterinarian can prescribe specific therapy recommendations.

In summary, stem cell and physical therapy represent promising strategies for tendon repair. Recognizing the clinical signs, contacting your veterinarian, and getting appropriate treatment and rehabilitation plans in place will ultimately lead to the fastest recovery.

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Equine Tendon Injuries: Reducing the Strain - TheHorse.com

Bader Philanthropies to redevelop King Drive building into cafe and wellness center – BizTimes – Milwaukee Business News

Bader Philanthropies, Inc. plans to redevelop a vacant two-story building located across from its headquarters on Martin Luther King Drive into a cafe and wellness center.

The renovated building, located 3338 N. Dr. Martin Luther King, Jr. Drive in Milwaukees Harambee neighborhood, will be occupied by Sams Place eatery and cafe and Shalem Healing, Inc., a nonprofit medical clinic.

The new center will combine two properties, including an existing 1910 building, formerly occupied by Churches of the First Born a Rock Foundation Inc., and a recently demolished building at 3356 N. Dr. Martin Luther King Drive.The project will involve renovating the existing building and adding about 3,000 square feet, for a total of about 10,000 square feet.

The total project, including the building purchase and construction, is expected to cost $4.6 million. The new center is scheduled to open in spring 2020.

Bader Philanthropies moved from its former location in the Historic Third Ward to a formerly vacant building at 3300 N. Martin Luther King Jr. Drive in July 2018.

Bader Philanthropies moved to Harambee to deepen our commitment to Milwaukee, said Daniel Bader, president and chief executive officer of Bader Philanthropies. We wanted to be in a place where we could truly live our mission by putting down roots and embedding ourselves in a neighborhood. Harambee residents are giving a voice to ideas that have the potential to enhance the quality of life, the health and wellbeing of our neighbors. Sams Place and Shalem Healing are examples of what our neighbors would like to see present in their neighborhood.

Shalem Healing, currently located at 800 E. Locust St. in the Riverwest neighborhood, offers integrated medical and holistic care on a sliding scale based on patients income. It serves about 3,000 annually .

Shalem Healings commitment to holistic wellness, especially for people living in the underserved areas of the city, is the core of what we do, said Dr. Robert Fox, founder of Shalem Healing. It is simple we want to make holistic care such as herbal medicine, acupuncture, food as medicine, stem cell treatment, nutraceuticals, and proper education on seemingly chronic diseases as accessible to the residents living in Harambee and many others who will travel from around Milwaukee.

Sams Place will be a jazz-themed eatery and cafe, offering coffee through its roasting division, Abyssinia Coffee Roasters. It will be the second location for operator CITY.NET Jazz Caf. Its current location is in downtown at 306 E. Wisconsin Ave.

Sams Place is all about community, said Sam Belton, owner of Sams Place. People from all walks of life can gather here, meet a friend or neighbor, and connect for a meeting in a great eatery while experiencing the sounds of classic jazz. Patrons can also enjoy freshly roasted coffee on site and expect live performances from local and national artists. Our roots are deep in Milwaukee and we are dedicated to rebuilding our community.

Sams Place will occupy the first floor and Shalem Healing will occupy the second floor.

Bader has retained JCP Construction, LLC as the general contractor and American Design, Inc. as the architect for the project.

We are excited to have JCP Construction build what will be an extraordinary community resource for the neighborhood in which they grew up, said Franklin Cumberbatch, vice president for engagement of Bader Philanthropies. And, American Design will give this historic building a second life.

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Bader Philanthropies to redevelop King Drive building into cafe and wellness center - BizTimes - Milwaukee Business News

What Are Myelodysplastic Syndromes and How Are They Treated? – Dana-Farber Cancer Institute

Medically reviewed by David P. Steensma, MD

Myelodysplastic syndromes (MDS) are a group of diseases of the blood and marrow that result in the body being unable to produce enough healthy, mature blood cells.

MDS develops when theblood stem cells found in the bone marrow become injured (i.e., they acquire amutation in the DNA that determines their function) and do not mature intohealthy white blood cells, red blood cells, and platelets. Normally, whiteblood cells work to fight infection, red blood cells carry oxygen, andplatelets help blood clot to stop bleeding. In MDS, cells may be reduced innumber or may not work the way they should. As a result, there are fewerhealthy red blood cells, white blood cells, and platelets.

In addition, a type of immature bone marrow cell called a blast cell can increase in MDS.Fewer than 5% of the marrow cells are blast cells in healthy persons.In some patients with MDS, the blasts increase to 5% or greater.If 20% or more blast cells are present in the marrow, the diagnosis is acute leukemia rather than MDS.

Myelodysplastic syndromes occur mostly in older patients; 75% of patients are more than 65 years old. The disease is very rare in children. Risk factors for MDS include:

There are two major categories of myelodysplastic syndromes.The first is primary or de novo MDS, where an external factor triggering thedisease cannot be identified. In contrast, secondary or therapy-related MDSis developed after use of chemotherapy or radiation for cancer. The term secondary MDS is also used todescribe MDS resulting in children with inborn genetic syndromes.

In about one-third of patients, MDS progresses to a condition known as acute myelogenous leukemia (AML). Secondary or therapy-related MDS, from chemotherapy or radiation, has a higher risk for evolving to AML than primary MDS.

The most common presenting symptom of MDS is fatigue and decreased ability to exercise or to carry on the usual daily activities.Anemia (low red cell count) is present in most patients with MDS; anemia can cause symptoms such as shortness of breath, swelling of the ankles, paleness or other signs and symptoms.

Less common presenting symptoms include bleeding (e.g. nose bleeds or gum bleeding) or easy bruising, due to low platelet counts or platelets that do not work well. Occasionally, patients with low white cells will first be diagnosed after they develop a serious infection MDS is often discovered accidentally during routine blood tests.

At Dana-Farber/Brigham and Womens Cancer Center, adults with MDS are treated in the Hematologic Oncology Treatment Center. For these patients, there are several treatment options.

The only potentially curative approach for MDS is a bone marrow or stem cell transplant, also known as an allogeneic hematopoietic cell transplant, to destroy abnormal cells in bone marrow with chemotherapy or radiotherapy (conditioning) and replace them with healthy cells from a donor.

This approach has many potential serious side effects, including infections or a form of rejection called graft-versus-host disease. Older patients, such as those in their 60s or 70s, are often given a lower dose of radiation or chemotherapy prior to transplant, also known as reduced-intensity conditioning. This therapy may not destroy all bone marrow cells but is enough to allow donor cells to grow in the bone marrow.

Chemotherapy is useful for treating patients with MDS because it treats the entire body, killing abnormal cells and allowing normal ones to grow. A class of low-intensity chemotherapy drugs called hypomethylating agents kill cells that divide rapidly and activate some genes that help cells mature. Using one of these drugs can improve blood counts (sometimes eliminating the need for blood transfusions), lower the number of abnormal cells such as blasts, and lower the chances of getting leukemia.

Side effects include an initial a decrease in blood cell counts, which often later improves as the drug begins to take effect, as well as fever, skin rash, nausea/vomiting, and fatigue. Examples of hypomethylating agents include azacitidine and decitabine.

For patients who have an abnormality of chromosome 5 intheir bone marrow cells and have anemia and do not have increased blasts, apill called lenalidomide may be helpful for improving blood counts. Lenalidomide can cause blood clots or worsenlow white blood count and platelets, and it doesnt work as well for patientswithout a chromosome 5 abnormality.

In rare cases, intensive chemotherapy similar to that givento patients with acute leukemia may be necessary. In certain cases, immune suppressing therapywith anti-thymocyte globulin, cyclosporin, tacrolimus or other drugs may beused.

Supportive therapy involves treating or preventing the symptoms of MDS, rather than treating the MDS directly by trying to eliminate abnormal cells. Supportive therapy can be used alone or with other treatments.

One supportive therapy option is hematopoietic growth factors. Hematopoietic growth factors are hormone-like substances that occur naturally in the body but can be produced synthetically and given in large doses than the body can make. These growth factors are typically injected subcutaneously (under the skin) to help bone marrow make new blood cells. Erythropoietin (epoetin or darbepoetin) is a growth factor that increases red cells. Less commonly, growth factors filgrastim or pegfilgrastim are used to increase white cells, or growth factors romiplostim or eltrombopag are used to increase platelets. Luspatercept is a new drug that improves red cell growth in certain MDS subtypes.

For patients who have had many red cell transfusions andhave an excess of body iron, iron chelation medications such as deferasirox ordeferoxamine may be used to lower iron levels and prevent injury to bodyorgans.

Transfusions with red cells or platelets as needed andantibiotics to prevent or treat infections are other examples of supportivecare used for MDS.

At Dana-Farber/Boston Childrens Cancer and Blood Disorders Center, children with MDS are treated through the Bone Marrow Failure and Myelodysplastic Syndrome Program. Treatment is similar to adults, including supportive care, except that hematopoietic cell transplant is used more commonly in children.

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What Are Myelodysplastic Syndromes and How Are They Treated? - Dana-Farber Cancer Institute

Selma Blair Says She’s Growing Peach Fuzz On Her Face Amidst MS Treatment Recovery – Women’s Health

Selma Blair has been super open when it comes to sharing about her life with multiple sclerosis. Since her diagnosis in August 2018, she's been raising awareness about MS and how it has affected life for her and her 8-year-old-son son, Arthur. Her Instagram is full of pics and posts documenting her days and treatments (and some fun fashion pics, minus pants), with captions revealing how MS has impacted her body and appearance.

Selma's latest post features a close-up photo of her face, showing she's starting to grow some peach fuzz.

"Face it. I have rather thick and substantial peach fuzz. This is a new development. I also have very small ears. Earlier development. School pick up musings. Carry on. #selfie," she captioned her post.

She's getting lots of love and support from her friends and fans.

"You are gorgeous and that is that ," said one fan.

And many lamenting they have peach fuzz too.

"You should see my peach fuzz!!!," wrote another friend.

While Selma didn't specify the suspected cause of her peach fuzz, she's currently in the midst of recovering from a stem cell treatment for her MS that required her to undergo chemotherapy. She's been open about how these treatments have impacted her vision, and caused insomnia and memory problems, as well as hair loss.

The bright side: Apparently she's already back to rocking school pickup like a boss. Here's hoping Selma's feeling better while she recuperates in LA!

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Selma Blair Says She's Growing Peach Fuzz On Her Face Amidst MS Treatment Recovery - Women's Health

Reassessing What’s Really Important After Relapse – Myasthenia Gravis News

I have started this column many times, but I have had trouble putting my emotions into words. I am trying to digest and cope with how my body is slowly failing me, and I dont know how I feel about it.

If you have been following me, you know that I had a stem cell transplant in January. It was a trial treatment and seemed to put me into complete remission. I was functioning like a normal adult for the first time in more than 15 years. I was able to go to the gym, work a full day, and still have the energy to cook a delicious dinner for my husband. I felt like I had been given a second chance at life, and boy, was I going to grab it with both hands!

Over time, I have noticed that my body is not as responsive as it was immediately following my transplant. I need a nap to make it through the day, and I have not set foot in the gym for more than two months. I began to slur my words again, and I have to be careful not to choke on my food during meals I have already had a few close calls.

I have had to reassess what is important to me. Do I really want that six pack, or do I just want to be able to make it through a day without collapsing in a heap? Do I want to push myself to go to every event and pay for it for up to a week?

I went to see my doctor when things had been going downhill for about a month. We made the decision to begin chemotherapy once again. I am only authorized for three rounds and I have already had two. The side effects of chemo are terrible and I am incapacitated for at least three days after a treatment. But I need that joy of life back.

This relapse has been far more difficult emotionally as I now know what it is like to live a full and healthy life. I know what it is like to do everything possible, and still have energy to entertain friends and family. I am back on antidepressants, and I am coping. I will never give up. Something has to work. Something.

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Myasthenia Gravis News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional med

I am a Myasthenia Gravis Warrior, wife, friend, and fur-mum. I love to read, crochet, sleep & find the good in every day. I always try to find the silver lining!

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Reassessing What's Really Important After Relapse - Myasthenia Gravis News