What’s new in lupus research + treatments?

Posted 2 weeks ago by

Keeping tabs on the latest info about lupus research is no easy task. So we’ve boiled down some highlights and trends in the world of lupus research and drug development — take a peek.

Rising areas of research

  • Stem cells – Initial studies have shown that mesenchymal stem cells (MSC) — a type of stem cell that lives in bone marrow, umbilical cord blood, and many other tissues — may suppress the inflammation associated with autoimmune disease, including systemic lupus erythematosus (SLE). An upcoming phase II clinical trial will take a closer look at the effectiveness and safety of MSC obtained from umbilical cords for the treatment of adults with SLE (check out this PatientsLikeMe guide to clinical trials to learn more about the phases and process for developing new treatments).
  • Gut bacteria – Research has also shown something called gut microbiota dysbiosis or intestinal dysbiosis (an imbalance in the amounts and types of bacteria in the gut) in people with autoimmune diseases like lupus. A small new study published in February 2018, which observed both mice and humans, found that lupus may be linked to an altered and less diverse microbiota (a biological community of bacteria or other organisms) in the gut. The researchers called for more research on lupus and gut health.
  • Biomarkers – SLE disease activity within the body has been difficult to monitor through blood tests because the condition is complex and affects people’s bodies differently. But the researchers behind a new study published in Nature in February 2018 believe they’ve identified two biomarkers (short for “biological markers,” or signs of biological/disease activity) strongly associated with active SLE. The biomarkers are called CIC and IL-6.
  • Cell biology – In 2014, the National Institutes of Health (NIH) launched a new program called the Accelerating Medicines Partnership for Rheumatoid Arthritis and Systemic Lupus Erythematosus (AMP RA/SLE) — a public/private partnership aimed at learning more about these conditions and speeding up the development of treatments. Earlier this year, the NIH rolled out the first data set and said it “holds clues for potential research targets that may lead to future treatment options.”

Clinical trial trends

Since the landmark FDA approval of Benlysta (belimumab) in 2011 — the first approved targeted biologic treatment for lupus and the first drug approved for lupus in more than 50 years — researchers have been working on a growing number of potential new treatments. (The FDA approved the first self-injectable form of Benlysta in 2017 for people with SLE; it’s called Benlysta SC, and the “SC” stands for “subcutaneous”.)

Treatments currently “in the pipeline” for possible development aim to offer either improvements on similar existing treatments or new types of treatments altogether. Several of the potential treatments have completed phase II trials and some are now somewhere in the phase III. (Again, check out our clinical trial guide to understand the phases and process.)

Here are some of the treatments in various stages of development…

For SLE:

  • Atacicept – Biopharmaceutical company EMD Serono recently announced positive results of a phase II clinical trial of atacicept — a protein that targets the immune system and appears to reduce flares in people with SLE.
  • Stelara (ustekinumab) – This treatment, marketed by Janssen, already has FDA approval for the treatment of plaque psoriasis, psoriatic arthritis and Crohn’s disease. In late 2017, a phase II clinical trial showed good potential for Stelara as a possible treatment for SLE. Janssen is continuing to study Stelara’s effects on SLE in phase III of the ongoing trial this year.
  • Lupozor (rigerimod) – This potential treatment by ImmuPharma showed mixed results in a phase III trial, so the company is weighing its next steps. The treatment failed to show a significant response rate over the “standard of care” treatment for lupus, but it performed somewhat better than a placebo and had no reports of adverse events. Lupuzor appeared to benefit European patients but not U.S. patients, Lupus News Today reports. The treatment aims to suppress the activation of auto-reactive T-cells — immune cells that attack the body’s own tissues — without affecting healthy immune cells.

For lupus nephritis:

  • Orelvo (voclosporin) – Aurinia Pharmaceuticals is working on developing this potential treatment, which aims to reduce inflammation and stop further damage to the kidneys. After announcing positive phase II trials in 2017, Aurinia launched a phase III trial of voclosporin, which they plan to give the brand name Orelvo.
  • Orencia (abatacept) – This treatment from Bristol-Myers Squibb already has approval for the treatment of adult rheumatoid arthritis (RA) or juvenile idiopathic arthritis. In 2013, a phase II trial of abatacept found that it is well-tolerated by people with lupus nephritis, although it did not meet the specific treatment goals of the study. The drug is currently being studied further in a phase III trial.

For SLE or lupus nephritis

  • Anifrolumab – AstraZeneca is winding down two phase III studies of this potential SLE treatment (both trials are expected to be completed in 2018). Anifrolumab, which is given intravenously, “works in a different way to Benlysta by targeting interferon, a protein involved in inflammation,” Reuters reported, along with promising phase II clinical trial results in 2015. AstraZeneca is also studying anifrolumab as a possible lupus nephritis treatment, with a phase II clinical trial expected to wrap up around 2020.

Full disclosure: PatientsLikeMe has partnered with some of these pharmaceutical companies on research projects. The information shared here is not sponsored content. Questions about our partnerships? At PatientsLikeMe, we’re all about transparency so check out who we’ve worked with here.

Which areas of lupus research are you following most closely? Chime in below or join PatientsLikeMe to talk with others about this topic in our forums.

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Epidiolex: First FDA-approved drug made from cannabis component (CBD)

Posted 3 weeks ago by

In a historic move, the U.S. Food and Drug Administration (FDA) recently approved Epidiolex — an epilepsy medication made from purified cannabidiol (CBD), found in cannabis. Read all about it and what it could mean for the future of cannabis-based treatments. As background, see these other recent write-ups we’ve done on medical cannabis/marijuana and CBD.

What is Epidiolex?

Epidiolex “is a liquid formulation of pure plant-derived cannabidiol as a treatment for various orphan pediatric epilepsy syndromes,” according to GW Pharmaceuticals, the U.K.-based company that markets the medication (an oral solution).

Some other info to keep in mind:

  • Limited scope of approval — The FDA approved the treatment specifically for two rare and severe forms of child-onset epilepsy — Lennox-Gastaut syndrome and Dravet syndrome — in patients 2 years of age and older.
  • No THC — Epidiolex is made from purified CBD and does not contain tetrahydrocannabinol (THC), the component of marijuana that causes a euphoric “high.”
  • Thorough research — Researchers studied the treatment’s effectiveness in three randomized, double-blind, placebo-controlled clinical trials involving 516 patients. Epidiolex (taken along with other medications) helped reduce the frequency of seizures when compared with a placebo. Research also found the medication has a very low potential for abuse.
  • Side effects — The FDA says the most common side effects that occurred in Epidiolex-treated patients in the clinical trials were: sleepiness, sedation and lethargy; elevated liver enzymes; decreased appetite; diarrhea; rash; fatigue, malaise and weakness; insomnia, sleep disorder and poor quality sleep; and infections.
  • Cannabis and CBD still controlled substances — Despite the approval of Epidiolex, other CBD and cannabis products are still currently (July 2018) “Schedule I” controlled substances under federal law, the FDA says (again, check out our CBD report for more info on laws and other FAQs).

Some reports say that Epidiolex could be prescribed for off-label uses (for patients with other forms of epilepsy), and its approval could open the door for other cannabis-based treatments

“This approval serves as a reminder that advancing sound development programs that properly evaluate active ingredients contained in marijuana can lead to important medical therapies,” says FDA Commissioner Scott Gottlieb, M.D. “The FDA is committed to this kind of careful scientific research and drug development.”

GW Pharmaceuticals lists other possible treatments they’re studying or developing. GW also markets Sativex (nabiximols) in several other countries to treat cancer pain and multiple sclerosis spasticity, Forbes reports, and a U.S. phase 3 trial is planned to test Sativex for MS spasticity.

What’s your reaction to the approval of Epidiolex? Join PatientsLikeMe or log in to discuss Epidiolex and CBD with others in the forum, and explore treatments members have tried.

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