This is my Pick of the Week’s News, as published by Multiple Sclerosis News Today.
A news story about a new company raising nearly $50 million to further develop stem cell transplants that might extend them as a therapy to more patients is, potentially, very exciting indeed.
Magenta Therapeutics has completed its first round of financing, raising $48.5 million to develop ways of bringing bone marrow stem cell transplants to more patients with autoimmune diseases, such as multiple sclerosis (MS), among other illnesses.
The new company aims to develop the first complete platform that can overcome the challenges in stem cell transplants, taking a patient-focused approach, it announced in a press release.
By improving the preparation of patients using antibodies, as well as making the gathering and expansion of stem cells more efficient, the company hopes to change the entire approach to transplant use, so that the therapy is more widely applicable. In addition to autoimmune diseases, the company is also focusing on treating people with genetic blood disorders and cancer.
“Technical and scientific hurdles have relegated stem cell transplantation to a last resort for deadly diseases today, but new science is ready to be advanced to the clinic that could fundamentally open up this powerful medicine to patients suffering from earlier stage cancers, blood disorders and a large set of aggressive autoimmune diseases, including multiple sclerosis and scleroderma,” Jason Gardner, chief executive officer, president, and co-founder of Magenta Therapeutics, said in the release.
The company is also talking about hematopoietic stem cells and stem cells being “curative,” but I did not find any mention of autologous cells, i.e., those collected from the patient, nor of the use of chemotherapy, so it’s yet another case of “wait and see.”
Now here is something close to my own heart. Since moving to live in the sun in the south of Spain last year, I have been using sun cream to protect my skin from the sun because of the risk of skin cancer. Then I find that I am vitamin D deficient.
People are going out of their way to avoid direct sunlight and, in the process, becoming ever more vulnerable to serious ills that appear to be countered by moderate sun and ultraviolet light exposure, from cancer and heart disease to multiple sclerosis, according to dermatologists and other health experts in an extensive review of literature on the benefits and risks of being outdoors on sunny days.
“Public health authorities in the United States are recommending that men, women and children reduce their exposure to sunlight, based on concerns that this exposure will promote skin cancer,” the researchers wrote. “On the other hand, data show that increasing numbers of Americans suffer from … serious health problems caused by insufficient sun exposure.”
Beginning with benefits historically linked to sun exposure — the discovery that adequate sunlight could treat rickets in 1919, was linked to vitamin D absorption in 1931, and to lower cancer mortality rates across North America (possibly via vitamin D) in the 1960s — the authors noted the sudden turn in the following years toward an emphasis on sun risks and melanoma.
“Avoidance of intentional sun exposure and use of chemical sunscreens persisted as the standard advice of physicians and public health authorities for reducing the risk of melanoma and other forms of skin cancer,” they wrote. “The risks of inadequate sun exposure have been largely ignored.”
OK, so now I am taking vitamin D supplement and will only use sun cream if in the sun for an extended period of time.
Congratulations to this young Queensland-based researcher for winning a prestigious award to her important work.
Dr. Rebecca Coll, a young researcher at the University of Queensland Institute of Molecular Bioscience, won the 2016 Research Australia Discovery Award for her work in identifying promising anti-inflammatory compounds that are able to block the NLRP3 inflammasome — a key driver of inflammation. The discovery could greatly benefit patients with inflammatory diseases, such as multiple sclerosis (MS).
Coll, who received the award at the Research Australia Health and Medical Research Awards ceremony, recently held in Sydney, led research into the biological characterization of a small molecule drug — called MCC950 — that inhibits the NLRP3 inflammasome and, possibly, its disease-causing inflammation.
“Uncontrolled inflammation contributes to the progression of many diseases including diabetes, arthritis, cancer, multiple sclerosis and neurodegenerative diseases such as Alzheimer’s and Parkinson’s,” Coll said in a news release. “We believe the NLRP3 inflammasome could be a fundamental factor driving the damaging inflammation associated with these diseases.”
Now we can look forward to seeing where Dr. Coll’s discovery takes us.
I believe that all research that leads toward the development of promising new treatments, whether immune-based or not, should be regarded as steps forward at this early stage.
A recent study published in Nature Communications showed, for the first time, that a protein complex called LUBAC is responsible for controlling the late-stage development of immune T-cells before they are released into the bloodstream.
Several types of cells compose the immune system, working together to fight infections or cancer. Among these are T-cells, which need to pass through the thymus, where bad cells are eliminated and others are taught to fight disease. Only a small number of T-cells pass this developmental process and are allowed out of the thymus.
The study, “Linear ubiquitin chain assembly complex coordinates late thymic T-cell differentiation and regulatory T-cell homeostasis,” led by Dr. Charis Teh, Dr. Daniel Gray and colleagues, found that faulty LUBAC activity caused defective T-cell development, with major impacts on autoimmune response.
“By showing that LUBAC is essential for T-cell development, we also revealed a new stage of ‘T-cell education’ that had not previously been appreciated. We’re excited by the prospect that this new checkpoint may be important for ensuring autoimmune T-cells are not allowed to complete their development,” Teh said in a press release.
I do not profess to know enough to comment on the science involved in this research, but will look forward to further developments.
This is what I like to see, the launch of a new website and a newly designed magazine – both to give users an easier and better experience. Well done, MS Foundation.
The Multiple Sclerosis Foundation, which is celebrating its 30th anniversary this year, has launched a new website to make access to information and services easier, and a newly designed MS Focus magazine.
MS Focus is the foundation’s publication dedicated to common challenges, solutions, and stories of the multiple sclerosis (MS) community. The new MS Foundation website includes some new programs that are available online for the first time, such as the Emergency Assistance Program to help MS patients keep their homes if rent or utilities fall behind; or the Health Care Assistance Grant, to help those who are uninsured afford specialized MS care.
In addition, the new website makes it easier to navigate and find information about grants and programs as well as education and support services.
The foundation used the launch of MS Focus to release new videos highlighting people whom the organization has helped. One of these is tennis player Eric Pierce, who was diagnosed with MS at age 32. Like many others, Pierce took the diagnosis as devastating news, and was forced to learn how to cope with his new condition under a completely new set of circumstances.
Then Pierce learned about MS Focus’ wheelchair tennis program.
“I felt like my world opened up, that the MS diagnosis was not the end of my life, it actually was almost the beginning of a new chapter,” Pierce said in a news release provided by MS Focus to Multiple Sclerosis News Today.
All in all, I think it looks good for the MS Foundation as it celebrates its 30th anniversary by looking purposely into the future.