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Multiple Sclerosis: Overcoming Pain

In March, Real World Health Care will launch a new series focusing on the behavioral health impacts of chronic illnesses. Until then, we will revisit a few of our recent blog posts that touch briefly on related behavioral health issues. This week, we revisit our interview with Dawn Ehde, PhD, Department of Rehabilitation Medicine, University of Washington School of Medicine. Dr. Ehde serves as a clinical psychologist and professor at UW. She conducts research evaluating the efficacy of various behavioral, exercise, and pharmacological interventions for chronic pain, depression, and/or fatigue in adults with multiple sclerosis (MS) and other acquired neurological conditions.

Dr. Ehde discusses some of her recent clinical and research work on cognitive-behavioral interventions for MS-related pain.

Living with MS and Pain

Real World Health Care: In 2015, you published an article: Utilization and Patients’ Perceptions of the Effectiveness of Pain Treatments in Multiple Sclerosis. Can you summarize the key results of your study and the implications for patients with MS?

Dawn Ehde, PhD, University of Washington School of Medicine

Dawn Ehde: We conducted this survey to learn more about pain management from the perspective of people living with MS and pain. We found that people with MS and pain try a lot of different treatments to manage pain, but few treatments provide adequate pain relief.

Nonprescription medications such as nonsteroidal anti-inflammatories and physical modalities such as massage were some of the most common methods used. Many use more than one treatment to manage pain. Some of the treatments that individuals rated as most helpful, such as hypnosis, were infrequently used. In fact, we found that very few people surveyed had tried or accessed behavioral pain treatments such as training in mindfulness meditation, cognitive behavioral self-management, or self-hypnosis. This was the case even though there is good evidence that these types of treatments are beneficial to many people with chronic pain, including MS, and have few or no negative side effects. This study highlighted for me the need to improve access to these types of non-pharmacological pain management.

Integrated Care Approach

RWHC: Are you currently involved in any other research relating to pain management in MS patients?

DE: We have several studies in various stages that address pain management in MS. We recently published a study that found that an eight-session telephone-delivered self-management intervention was effective in reducing pain (both its severity and its interference with activities) and fatigue. It also was effective in improving mood, quality of life, and resilience. The benefits were maintained at 6- and 12-month follow ups. Patient satisfaction with the treatment was high as well.

The study I am most excited about is the MS Care study, which is a comparative effectiveness trial that evaluated the benefits of an integrated care approach to pain and depression management in the clinic called “collaborative care.” The MS version, called “MS Care,” aims to improve the quality of pain care in the clinic by adding an MS Care manager to coordinate care, deliver brief behavioral treatments, initiate or adjust other medical treatments, and ensure patients do not slip through the cracks. We also offer patients the choice of getting their care management by phone or in person. Seventy-five percent chose the phone. We found that patients with chronic pain and/or depression randomly assigned to MS Care had significantly improved pain and depression symptoms, including less severe pain, less interference, less disability, and less fatigue. Additional details on the results are available at http://www.uwmscare.org/background.

Opportunities in MS Pain Research

RWHC: What are some of the biggest challenges facing researchers who are studying pain management in MS patients? How can those challenges be overcome?

DE: I actually see a lot of opportunities as an MS pain researcher. The MS community is interested in improving pain management and supporting research in this area. For example, the National MS Society has named pain as one of its research priorities. We also often find people with MS are willing participants in our research, both as participants as well as stakeholders who guide us in our research. For example, we used stakeholders to guide our MS Care study. At times, we have had to work hard to convince potential funders that pain is an issue that warrants funding and study, but that has improved in the time that I’ve been doing research. We have come a long way since I first started in this area, when pain was not always recognized as an important problem deserving attention in MS.

Challenges for MS Clinicians

RWHC: What are some of the biggest challenges facing clinicians who are treating MS patients with pain management problems?

DE: MS presents many different symptoms to manage, and thus both patients and clinicians have a lot to discuss and manage in the typical clinic appointment. One challenge is that pain management is often only one of several issues being addressed. As such, it may be difficult to fully manage a complex issue like chronic pain. I think these challenges may be overcome by rethinking how we approach and deliver pain care. We need to look at harnessing technology — including telehealth technologies — to improve care. We also need to empower patients and the MS community to recognize that pain management is something that requires active self-management and multimodal strategies.

Pain Management Therapies

RWHC: What do you see as the most promising pharmaceutical therapies for treating pain in MS patients? What are the caveats that must be understood by clinicians when prescribing such therapies?

DE: As a psychologist, I’m less able to speak to promising pharmaceutical therapies on the horizon. However, I think there are promising practices for how we deliver pain care, including medications and other rehabilitation interventions. We did manage medications in our MS Care study, and our goal within that was to promote the appropriate and effective use of pain medications and other medications that can benefit pain management, such as some of the antidepressants which have analgesic benefits. We know from our research that too often, patients get started on a treatment, perhaps at a “low dose,” and for whatever reason, they don’t have adequate follow up to adjust, intensify, or change treatment plan. In the MS Care study, we closely and quickly followed patients’ pain and adjusted treatments to optimize their benefits or switched treatments if needed. We also know that physical activity — whether it is physical therapy or encouraging physical activity — benefits people with MS and likely helps with pain management.

RWHC: Do you see a role for non-pharmaceutical pain management therapies in treating pain in patients with MS?

DE: Certainly. This is where I’ve spent most of my energy, not only because I am a psychologist, but also because many people with MS want to use non-pharmacological therapies and strategies. The people with MS I know and our stakeholders are eager to advance our understanding and use of non-pharmacological treatments such as mindfulness meditation, relaxation, and cognitive behavioral coping skills.

Partnering with Patients

RWHC: What initially got you interested in this field? What continues to inspire you?

DE: I have had family and friends with MS, and thus was drawn to learning more about MS. I started out conducting chronic pain research in people where chronic pain such as headaches was the primary problem. When I started working with patients with MS clinically, I was struck by how little we knew about MS pain management and how people with MS pain were not accessing care we knew was helpful in other pain populations.

I’ve been inspired and continue to be inspired by the people with MS whom we’ve partnered with to conduct our research. Our best research has resulted from partnering with people living with MS. They’ve also taught me a lot about resiliency. Although MS can present many challenges like pain, many people with MS and pain live full, meaningful and happy lives.

I also have been fortunate to have training grants from the National MS Society, which have allowed me to train postdoctoral fellows in MS and rehabilitation research. They represent the next generation of clinical researchers in pain and symptom management in MS.

Pain Management for Cancer Survivors

In March, Real World Health Care will launch a new series focusing on the behavioral health impacts of chronic illnesses. Until then, we will revisit a few of our recent blog posts that touch briefly on related behavioral health issues. This week, we revisit our interview with Judith A. Paice, PhD, RN, who is the lead author for the American Society of Clinical Oncology’s guideline, Management of Chronic Pain in Survivors of Adult Cancer. Dr. Paice is Research Professor of Medicine, Hematology/Oncology, at Northwestern University’s Feinberg School of Medicine and a full member of the Robert H. Lurie Comprehensive Cancer Center. Dr. Paice’s clinical work focuses on the management of cancer-related pain, and her research focuses on the study of chemotherapy-induced peripheral neuropathy. We spoke about the ASCO guideline and the need for clinicians to balance pharmaceutical and non-pharmaceutical approaches to pain management.

Real World Health Care: Why did ASCO issue a guideline for the management of pain in survivors of adult cancer?

Judith A. Paice, Feinberg School of Medicine, Northwestern University

Judith Paice: The oncology field has evolved tremendously in recent years. Not only are people living longer with cancer, but they’re being cured of their disease thanks to some fantastic treatments. These treatments provide good clinical responses, but they can also cause significant toxicity, some of which may lead to chronic pain syndromes. The goal of the guideline was to alert oncologists to the presence of these long-term, persistent pain syndromes. A secondary goal was to provide support for chronic pain syndrome treatment.

Clinician Guidance

RWHC: What are the most important take-aways for clinicians?

JP: First are ASCO’s recommendations for screening and assessment. Second are recommended treatment options, both pharmacological and, equally important, non-pharmacological treatments. The guideline also provides insights and risk mitigation strategies for clinicians around the long-term use of opioids.

Today’s oncologists are faced with a very different pain management phenomenon than they were 20 years ago, when opioids were primarily used at end of life. Opioids are now being used for patients with a much longer survival trajectory — 20 to 30 years or more. As clinicians, we need to ask if such long-term use of opioids is appropriate and safe. How do we go about determining that? The guideline helps oncologists with those types of assessments and decision making.

RWHC: What do you feel are the biggest challenges facing oncologists in managing chronic pain in cancer patients, and how is ASCO helping clinicians manage those challenges?

JP: Our society is facing a serious public health problem in the opioid abuse and misuse epidemic. As a result of this problem, we’ve seen regulations at both the state and federal level that are having a chilling effect on the availability of opioids, even for those in desperate need of these medications. ASCO has a position paper on protecting access to treatment for cancer-related pain that I encourage all clinicians to read. ASCO also advocates for better third-party reimbursement for physical therapy, occupational therapy, cognitive behavioral therapy and mental health counseling. These are crucial therapies for patients facing the “new normal” of cancer survivorship, yet most third party payers provide little or no support for these treatments. As clinicians, we need to help our patients maintain function and cope with the fact that their lives are going to be very different. For patients, it’s more than surviving cancer. It’s about finding their own inner strength in survivorship.

New Pain Management Treatments

RWHC: Where do the biggest opportunities lie for new pharmaceutical pain management treatments?

JP: Several new findings in the laboratory may lead to novel agents that do not produce opioid related adverse effects. This is promising, assuming the findings can be translated into a clinical setting. There have also been numerous compounds that proved effective in animal models of pain, but when moved into the clinical setting, they either had adverse effects that weren’t seen in animals, or they didn’t have the efficacy they presented in the lab. It is very difficult to develop a model of cancer pain in animals.

Unfortunately, there haven’t been many completely new drugs. Most of the agents approved recently are slight variations of existing compounds or an update in the delivery method: a spray instead of a tablet, for example. The industry has been more focused recently on abuse-deterrent compounds. This is a somewhat controversial area because while such formulations prevent people from crushing, snorting or injecting the drugs, they don’t keep people from taking more than what is prescribed.

Non-Pharmacologic Therapies

RWHC: What should the role be for non-pharmaceutical pain management therapies in treating cancer patients?

JP: For quite a long time, there was a tendency in medicine to rely only on pharmaceutical therapies. This made sense when patients did not have long-term survival prospects and when managing pain meant helping the patient get from their bed to a chair. Today, cancer patients are living longer. They want to get back to work and function safely without the risk of falls and other complications.

We’ve seen good data around the usefulness of physical therapy, occupational therapy and cognitive behavioral therapy for many chronic pain situations, including cancer-related pain. These non-pharmacologic therapies must go hand-in-hand with pharmaceutical therapies.

Part of the challenge with non-pharmacologic therapies is limited reimbursement. The other big challenge is getting buy-in from patients. Most of us want a quick fix. Redefining expectations can be difficult. Physical and occupational therapy can be demanding, and access to specialists who understand the special needs of those surviving cancer are in short supply. Also, there remains a stigma attached to seeing a mental health counselor. It’s important for cancer patients to know that they aren’t “weak” if they need support to help them cope with the physical and emotional challenges of being a cancer survivor. Our field needs to do a better job educating our patients about the importance of including non-pharmacologic therapies as part of our pain management repertoire.

 

 

Patient Agitation in Alzheimer’s Disease: Implications for Patients and Caregivers

In March, Real World Health Care will launch a new series focusing on the behavioral health impacts of chronic illnesses. Until then, we will revisit a few of our recent blog posts that touch briefly on related behavioral health issues. This week, we feature our interview with Anton Porsteinsson, MD, Department of Psychiatry, University of Rochester School of Medicine. Dr. Porsteinsson is the Director of the University of Rochester Alzheimer’s Disease Care, Research and Education Program and has devoted his career to the care and study of individuals with memory disorders. Following up on his recent randomized clinical trial of citalopram, we discussed why it’s important to focus on treatments for AD-related agitation — both for AD patients and their caregivers.

Real World Health Care: Why is it important to study agitation in patients with Alzheimer’s disease?

Anton Porsteinsson, MD, University of Rochester School of Medicine

Anton Porsteinsson: Agitation is quite common in people with AD. It has a huge impact on their quality of life, as well as their family members’ and caregivers’ quality of life.

RWHC: Prior to your CitAD Randomized Clinical Trial, why had previous pharmacological treatment options been deemed unsatisfactory?

AP: A number of medications have been studied over time for this condition, including atypical and conventional anti-psychotics and mood stabilizers. But the efficacy they showed, if any, was modest at best. At the same time, these medications have serious potential complications such as increases in cerebrovascular events, sedation, falls, Parkinsonism and even increased mortality.

RWHC: Your trial focused on patients receiving psychosocial intervention. Why is psychosocial intervention important for AD patients?

AP: Medications should rarely be the first line of treatment for AD patients. Not everyone needs to be treated with medications. That’s why it’s important to evaluate the root cause of the agitation problem. Is the patient agitated because of something going on in his or her environment? I remember a situation from a few years ago when I was seeing a summertime spike in agitation-related consultations from patients in a particular nursing home. It turns out the nursing home didn’t have air conditioning. So it’s not surprising patients were bothered and agitated. In AD cases, agitation may have as much to do with your roommate as your receptors.

Psychosocial intervention helps to channel nervous energy and restlessness by involving patients in something purposeful, or even giving them some sort of outlet, like an area to pace around where they are safe and not in anyone’s way.

RWHC: The trial also focused on the effects of citalopram on caregiver distress. Why is this an important area of study?

AP: Caregivers, who are often family members with little or no medical training, may not understand what is going on with their loved one. They may take the patient’s behavior personally, which can cause a great deal of stress. Even if you’re a saint, it can build up and take a lot out of you.

Providing care for someone with AD is very hard under the best of circumstances. It’s even more difficult when the patient is verbally or physically aggressive, uncooperative, or agitated. Caregivers need advice, support and tools to help them handle the situation. They need to learn to give themselves breaks, that it’s OK not to be perfect, and that help is available for them. I find that a lot of caregiver stress is alleviated when, as health care providers, we listen to them and take their concerns seriously.

Caregivers need to know that agitation in AD patients is common and that there are ways to deal with it. Providers must connect them with resources like the Alzheimer’s Association and community agencies. We need to help alleviate their concerns about finances. And we need to help them set up a working plan on how to deal with their situation — to bring order to the chaos.

RWHC: How will the results of the CitAD Randomized Clinical Trial inform your future AD research?

AP: This trial was extremely educational for the research community. It was one of the first studies to show that a medication was effective in multiple ways — both on a clinical scale in reducing agitation among patients and in reducing caregiver distress. We also found efficacy for other AD patient behaviors like anxiety, irritability and delusions or hallucinations.

On the flip side, we discovered some complications. Citalopram has been used widely for decades with vulnerable populations. But in the last five or six years, it’s been found to not be as safe as once thought. It has the traditional SSRI side-effects of mild gastrointestinal distress and mild sleep pattern disturbances. But it also has been found to have an impact on cardiac conduction, especially in higher doses. In fact, when we were about three-quarters of the way through the study, the FDA suggested that, for people older than 60, there should be a dose limit of 20mg per day.

We confirmed this finding in our study. We also saw a drug placebo difference on a cognitive measure, the MMSE (Mini Mental State Examination). It isn’t clear if this was due to baseline differences between the two groups and drift toward the mean, as the placebo group improved on the MMSE and the drug group saw a modest decline, or if it was a true modest cognitive toxicity. Until proven otherwise, we have opted to assume this is a potential side effect and we warn against it.

For our next study, we considered testing a lower dose of citalopram (20mg daily), but then we found that the active isomer of citalopram (S-citalopram) seemed to be better correlated to benefits seen in the study, while the inactive isomer (R-citalopram) more correlated with the adverse effects. S-citalopram is available as a generic drug, approved for depression and anxiety. We intend to study that drug further.

RWHC: What are some other areas of AD research you’re currently involved in within the URMC Memory Care Program? What do you see as your most promising area of research?

AP: We have a broad portfolio of research programs at URMC. We’re one of the more active academic-based clinical research programs in the country. Currently, we’re conducting two behavior-focused studies. One is ongoing and is based on positive findings on dextromethorphan hydrobromide and quinidine sulfate, with a new formulation that uses less quinidine. We’re also looking at methylphenidate for treatment of apathy in patients with AD.

We’re also investigating new imaging techniques and various biomarkers to improve our ability to identify those at risk. And, we’re working to find better ways of monitoring the progression of the disease and response to treatment through the ADNI study, which just received a fourth wave of funding.

Other areas we’re investigating include prevention studies with people who are cognitively normal, but who have elevated beta-amyloid or genetic biosignatures that indicate future pre-disposition. We’re looking at a passive and active vaccine against amyloid production. And we have a number of different studies on the prodromal stage of AD, working with beta secretase inhibitors that block the production of beta-amyloid.

It’s actually a very exciting time in Alzheimer’s disease research. We’re seeing improved funding from federal sources and a rejuvenation of interest from the pharmaceutical industry. I’m quite optimistic that in the next five to ten years, we will make substantive progress in terms of our ability to limit AD. I think it’s overly optimistic to expect a cure in that timeframe, but we can certainly make a dent, particularly from an early intervention standpoint. Treating this disease early is the critical factor.

 

Skin Cancer Awareness and Prevention Efforts in Focus at American Academy of Dermatology

In March, Real World Health Care will launch a new series focusing on the behavioral health impacts of chronic illnesses. Until then, we will revisit a few of our recent blog posts that touch briefly on related behavioral health issues. This week, we revisit our profile of the American Academy of Dermatology. We spoke with the AAD’s new President, Henry W. Lim, MD, about the organization’s mission and some of the challenges and opportunities associated with preventing and treating melanoma and other skin diseases.

Real World Health Care: Please tell our readers about the overall mission of the American Academy of Dermatology.

Henry W. Lim, MD, American Academy of Dermatology

Henry Lim: The American Academy of Dermatology promotes leadership in dermatology and excellence in patient care through education, research and advocacy.

As the largest, most influential and representative dermatology group in the United States, and the largest such organization in the world, the AAD works to make sure its values reflect this mission. The AAD’s values include putting patients first, encouraging its members to adhere to an uncompromising code of clinical and ethical standards, fostering an interest in our members to pursue lifelong learning, encouraging collaboration and working within our communities and embracing diversity.

Public Education: Sun Safety

RWHC: How does the AAD’s mission address melanoma?

HL: It is estimated that 161,790 new cases of melanoma will be diagnosed in the U.S. in 2017.  That is a staggering number that could be reduced if people incorporated skin cancer detection and prevention behaviors into their lives.

The AAD works to increase public awareness of skin cancer and its risks through its SPOT Skin Cancer campaign, which is designed to create a world without skin cancer through public awareness, community outreach programs and services, and advocacy that promote the prevention, detection and care of skin cancer.

The first step toward a world without skin cancer is educating the public about prevention. The Academy has long communicated sun-safety messages to the public about the importance of skin cancer prevention and detection.

In addition, dermatologists have led the medical community in finding and treating skin cancer. For more than 30 years, dermatologists across the country have hosted 2.5 million free SPOTme® skin cancer screenings that have detected 28,822 suspected melanomas and 256,329 suspected skin cancer lesions.

To assist the public with learning more about skin cancer prevention and detection, the AAD offers a variety of free, online videos, downloadable handouts and skin self-exam resources, including a body mole map, as well directories to find a dermatologist and skin cancer screenings.

Melanoma & Skin Cancer Awareness

RWHC: What is the AAD doing in 2017 to recognize Skin Cancer Awareness Month?

HL: The AAD’s 2017 SPOT Skin Cancer campaign, Check Your Partner. Check Yourself, encourages the public to be aware of changes on their skin that could be signs of skin cancer. Research has shown that women are more likely to detect suspicious spots on others.  Men over the age of 50 have a higher risk of developing melanoma, than the general population, so the campaign encourages women – often the health care decision makers of a household – to check their partner’s skin regularly, check their own skin, and to visit the AAD’s SpotSkinCancer website to find a free SPOTme® screening in their area.

RWHC: Do you have additional initiatives you’d like to highlight?

HL: In addition to the activities for Skin Cancer Awareness Month in May and the SpotSkinCancer™ website, the AAD works with state dermatology societies and state legislatures to introduce and support laws and regulations that protect consumers and promote awareness about skin cancer prevention and the dangers of indoor tanning. As a result, 42 states have enacted tanning bed restrictions to potentially reduce the risk of melanoma and other forms of skin cancer.

The AAD’s Shade Structure Program awards shade structure grants to schools and non-profit organizations across the country in order to protect children and adolescents from the sun’s harmful rays.  Since its launch in 2000, the AAD’s Shade Structure Program has awarded 350 shade structure grants, which provide shade for more than 600,000 individuals each day.

The AAD also has a strategic social media presence on Facebook, Twitter, YouTube and Pinterest, designed to raise awareness about skin cancer detection and prevention.  Social media, including paid, promoted posts, reach our targeted audiences – the public, our members and the media – with links to AAD resources.  We encourage our followers to like, share and re-tweet our skin cancer awareness videos and tips.

Melanoma Research

RWHC: Does the AAD underwrite or otherwise support research into melanoma detection and/or treatment?

HL: While AAD is not a research funding organization, the AAD does provide annual awards for Young Investigators in Dermatology.  These awards recognize outstanding basic and clinical/translational research by young dermatology investigators and some of the projects are related to melanoma.

The purpose of the award is to acknowledge research contributions by individuals at the start of promising research careers that further the improvement of diagnosis and therapeutics in the practice and science of dermatology.

RWHC: What do you see as the biggest challenges facing researchers studying melanoma treatments and clinicians treating melanoma?

HL: The rapidly changing health care environment presents major challenges to researchers and clinicians in all aspects of dermatologic care, not just those studying and treating melanoma. 

A significant challenge is the inadequate funding for research, together with the pressure to increase clinical revenue generated by clinician researchers.  For many years, the American Academy of Dermatology Association (AADA) has been active in advocating increased research funding by NIH to dermatology research, including through our support of the 21st Century Cures Act.

The current health care system also presents barriers that impede patient access to the best possible care from a qualified physician.  To combat this, the AADA is working with all dermatology care providers and other physicians to confront these challenges.

In particular, the AAD recently launched a new specialty positioning campaign, SkinSerious, to raise awareness of the serious impact of skin disease. Our goal is also to improve access to dermatologists’ expertise and increase collaboration with our physician peers to ensure high-quality patient care. We know that when dermatologists work with other physicians as part of the health care team, everyone can benefit from improved patient outcomes and lowered health care costs.

Other concerns within the health care environment that the AADA is closely monitoring include the rise of big data and the growth of teledermatology.  We closely follow developments at the federal and state levels and, when appropriate, the AADA will take action on issues that can be influenced positively for dermatology and pursue opportunities to impact health care policy. 

Promising Melanoma Treatments

RWHC: What do you see as the most promising or breakthrough melanoma treatments on the horizon?

HL: This is an exciting era in melanoma research.  In-depth understanding of the molecular pathways of melanoma development has led to the availability of immune checkpoint inhibitors; combinations of these medications are being looked at in clinical trials.  Metabolic manipulation of the peri-tumoral environment to inhibit the growth of melanoma is being actively investigated.  Understanding of the genes responsible for melanoma resulted in the availability of gene expression profile (GEP) test that can be used to determine biologic behavior of melanoma.

Melanoma Prevention

RWHC: What are the biggest challenges facing the medical community in terms of increasing awareness of and adherence to melanoma prevention efforts among the general public?

HL: The challenges facing the medical community around melanoma prevention are two-fold.

One is the misconception that a tan is a sign of health.  Tanning is a protective physiologic response of our skin to damage caused by ultraviolet radiation.  There is no such thing as a healthy tan, yet people continue to seek the sun or use indoor tanning, thereby increasing their risk of skin cancer.  This is a particularly challenging message to get across to young women and men, who feel peer and societal pressure to be tan.

The AADA was instrumental, along with several other organizations, in having the FDA re-classify tanning lamps from the Class I to Class II medical device category, which requires more supervision and restriction in their purchase and use.  For the past several years, the AAD has released a new public service advertisement that focuses on the dangers of tanning, particularly targeting young women.  We know that melanoma is the second most common cancer in young women, and this may be due in part to their tanning habits.

The 2016/2017 public service advertisement is called “Arms,” and features two young women comparing their tans at various stages in their lives. The emotional ad concludes with the two friends clasping hands in the hospital as one of them reveals she has advanced stage melanoma.  This PSA, and our previous ones, have resonated strongly with young women, especially on social media, where they have liked and shared the video with their friends.

The second challenging misconception is that many people believe that sun exposure is the best source of vitamin D.

While our bodies need vitamin D to build and maintain strong, healthy bodies, the AAD does not recommend getting vitamin D from sun exposure or indoor tanning because of the increased risk of skin cancer.  In fact, it has been demonstrated that sun exposure that results in increased vitamin D levels is directly correlated with DNA damage.

Vitamin D from food and dietary supplements offers the same benefits — without the danger of skin cancer — as vitamin D obtained from UV light.  Vitamin D cannot be used by the body until it is processed by the liver and the kidneys. The usable form of vitamin D created by this process is the same, regardless of how it enters the body.

The AAD recommends dietary sources (foods naturally rich in vitamin D, fortified foods and beverages) and vitamin supplements as sources of vitamin D that are available year-round and can easily be incorporated into a healthy lifestyle. Good sources include fortified milk, cheeses and yogurt, fortified cereal, and oily fish like salmon and tuna. Research shows that vitamin D supplements are well tolerated, safe, and effective when taken as directed by a physician.

The fact is these myths are harmful because the consequences of this misinformation could be potentially fatal.

RWHC: What personally inspires you to build awareness of the importance of preventing melanoma?

HL: Having been in dermatology practice for 40 years, I see on a regular basis the devastating effects that melanoma has on patients and their family.  The risk of developing melanoma can be significantly decreased by sensible photoprotection, and avoidance of tanning beds.  The exciting new developments in the treatment and genetic profiling of melanoma reflect the value of investment in scientists and research projects, and I look forward to additional treatments in the future that will benefit patients.

 

 

 

 

Categories: General, Melanoma

Helping Others Get the Help They Need

Editor’s Note: We conclude our series on Multiple Myeloma with a profile of a patient who was struggling to afford her treatments until she received a grant from our sponsor, the HealthWell Foundation. 

The searing back pain was the first indication to Marilyn Gould that something wasn’t quite right. The 67-year-old retired architect from Baltimore started to feel the pain at age 62 and began visiting a variety of doctors and specialists to find out what was wrong.

Marilyn Gould

Blood work indicated high protein levels — a potential marker for multiple myeloma.  Imaging did not reveal any tumors.  Eventually, her L5 vertebra cracked and she had to have surgery.  It was during this surgery that a tumor was discovered inside her vertebra.  Two surgeries were needed to remove the tumor and replaced it with a titanium cage. 

“At that point, there was no question: I had multiple myeloma,” said Marilyn.

Relief, Followed by Concern

“Getting the formal diagnosis was almost like a relief, because I knew I was a candidate for the disease and tried to educate myself about what was going to happen,” she said. “But nobody told me exactly what to do.”

Marilyn’s hematologist suggested that she consult with a multiple myeloma specialist, who started her on a course of treatment that involves an oral chemotherapy medication, steroids, an antibiotic and four 30-minute bone infusions a year — a regimen she has followed for the last 5 years.

As can happen with many chronic and life-altering diseases, multiple myeloma and its related treatments have had an impact on Marilyn’s lifestyle, though she says she still remains fairly active.

“I don’t run anymore and can no longer play golf,” she said. “But I do a lot of walking, and some bike riding. I also practice yoga, which has helped tremendously with my stability and balance, which have been compromised after two back surgeries.”

The disease and its treatments have also had an impact on Marilyn’s financial security.

“My Medicare Part D coverage still leaves me with a significant copay for the oral chemotherapy drug that I need to take for three weeks every month,” she said. “It’s a $10,000 copay every year — a difficult amount to afford on a fixed income, or just about any income. I originally received financial assistance from another copay assistance foundation, but when they couldn’t renew my grant, I turned to the HealthWell Foundation.”

Reaching Out

Marilyn said that financial assistance for treatment copays is a common topic when she speaks with other multiple myeloma patients as part of her volunteer outreach work with the Leukemia & Lymphoma Society.

“Just about everyone I speak with has financial concerns,” she said. “I let them know about the existence of copay assistance foundations like HealthWell, as well as other help that is available, like the Maryland-based MobilityLink program, which provides transportation to and from treatments for people who can’t drive or don’t have access to other transportation.”

While Marilyn is quick to point out that she is not a medical professional and can’t give medical advice, she does have words of wisdom to share with other multiple myeloma patients.

“Don’t be afraid to seek a second opinion, especially if a bone marrow transplant has been suggested,” she said. “And because multiple myeloma tends to hit people over 50 more than younger people, I tell everyone of that age to stay current with their blood work.”

Supporting Multiple Myeloma Patients

The HealthWell Foundation, sponsor of Real World Health Care, is proud to have supported the multiple myeloma patient community in recent years with copayment and premium assistance. We have helped more than 9,000 multiple myeloma patients afford their treatments since 2015 — thanks to the generous support of our donors. Due to high patient volume, our multiple myeloma fund is temporarily closed until we receive additional funding. We invite corporations and individuals to help us meet this demand by contributing to our Multiple Myeloma Medicare Access Fund, so no one goes without essential medications because they cannot afford them.

 

Multiple Myeloma Treatment: Too Many Choices?

Real World Health Care continues our multiple myeloma series with an interview with Edward A. Faber Jr., DO, MS, associate director of the Blood and Marrow Transplant Program managed by Oncology Hematology Care, Inc. in conjunction with the Mercy Health and The Jewish Hospital in Cincinnati. He also serves as principal investigator for OHC’s multiple myeloma clinical trials. Dr. Faber is a member of OHC’s board of directors and the Dayton Clinical Oncology Program and serves on the board of trustees for the Leukemia and Lymphoma Society. We discussed his clinical trial work and his views on promising new treatments for multiple myeloma.

Early Work in Multiple Myeloma

Real World Health Care: What attracted you to the field of multiple myeloma and what keeps you inspired?

Dr. Edward A. Faber, Oncology Hematology Care, Inc.

Edward Faber: I initially sought a career in bone marrow transplantation. During my training and fellowship, I decided to pursue a career in academic medicine, at a time when multiple myeloma was almost in its infancy. There have been so many advances over the past decade, not only with new individual medications but also with new combination therapies.

The inspiration comes from the patients, as quality of life and survival have improved over the past 10-15 years. This is a testament to the collaborative effort between academia, cooperative groups and pharmaceutical companies.

Clinical Trials

RWHC: What is the significance of your recent clinical trials?

EF: Over the past five to six years, we have been involved with clinical trials involving carfilzomib, pomalidomide, panobinostat, and daratumumab. We’re also comparing combination therapies between carfilzomib (KRd) versus bortezomib (RVd). These trials have demonstrated the efficacy of the combination of carfilzomib and panobinostat. For example, daratumumab, a potent choice for our patients with multiple myeloma, may offer benefits towards patients with smoldering myeloma.

Bone marrow transplant is an accepted treatment option, and the large Eastern Cooperative Oncology Group (ECOG) trial comparing KRd and RVd with lenalidomide maintenance may prove to offer a viable strategy outside of transplant. Quite simply, the research that we have been participating in has led to improvements in combination therapies with the newer medications for patients in the current community.

Research Collaboration

RWHC: What are some of the biggest challenges facing multiple myeloma researchers?

EF: The challenges facing multiple myeloma researchers are not unlike those facing all researchers, from the standpoint of availability of clinical trials, and making clinical trials available to large portions of the population.

Funding is always an issue for basic science researchers, bench top researchers, and clinical scientists.

Staying up-to-date with the current large cooperative groups, as well as the national scientific groups such as the American Society of Hematology and American Society of Clinical Oncology, is more important than ever, in order to learn and understand which available therapies offer more promise and move those therapies forward so that they eventually can lead to FDA indications.

Sequencing Therapies

RWHC: How are clinicians overcoming treatment challenges in multiple myeloma?

EF: The availability of a large number of drugs to treat multiple myeloma patients is both positive and a challenge for clinicians because there can be just too many choices.

The most important step begins at initial diagnosis and knowing how to sequence available therapies — especially combination therapies — over time to maximize benefit. These combinations must be interpreted in the context of the patient’s other medical issues. Certain side effects, such as cardiac toxicities, need to be avoided in certain patients. Patients should be encouraged to develop a good relationship with a physician who has focused interest in multiple myeloma. If that physician is aligned with a large academic center, he or she will be in a better position to help translate recent research and trial successes to hospital exam rooms, where the majority of myeloma patients are treated.

 

Multiple Myeloma: A Rare and Complex Cancer

We continue our series on multiple myeloma with an interview with Gareth J. Morgan, MD, FRCP, FRCPath, PhD, professor of hematology and director of the Myeloma Institute at the University of Arkansas for Medical Sciences (UAMS). Dr. Morgan also serves as deputy director of the Winthrop P. Rockefeller Cancer Institute at UAMS.

Dr. Morgan is responsible for all clinical, research, and administrative operations at the Myeloma Institute, which is one of the largest programs in the world focused on the research and treatment of multiple myeloma and related diseases. Clinically, he directly oversees 8 physician specialists, 14 mid-level providers, and 7 hospitalists, all experts in the management of myeloma and related plasma cell diseases. He also manages 8 research teams, which represent an integrated program of the genetics and biology of myeloma.

Mechanisms of Malignant Transformation

Real World Health Care: What got you interested in the field of multiple myeloma and what keeps you inspired?

Dr. Gareth Morgan, University of Arkansas for Medical Sciences

Gareth Morgan: Scientifically, I saw the opportunity to exploit the transition of MGUS (monoclonal gammopathy of undetermined significance) to smoldering myeloma to multiple myeloma as a model to understand the mechanism that drives malignant transformation. I reasoned that if we understood these, we should be able to manipulate them therapeutically.

I continue to be inspired by the ability to translate the biology and genetics of myeloma into clinical practice, where better and less toxic treatments can and are being developed to help patients achieve better outcomes and increased survival. Patients are the true source of inspiration.

Myeloma Genome Project

RWHC: Tell us about your recent research work and its significance to the multiple myeloma patient community.

GM: This is an exciting time for the field of myeloma, and our research investigations have led to some exciting discoveries in the biology of myeloma based on the genetic variations within the human genome. With our colleagues in Europe, have identified eight new genetic variations that could be linked to an increased risk of developing myeloma.

We are also focused on developing a molecular classification of myeloma based on patient subgroups with distinct pathogenesis and clinical behavior. We have partnered with Celgene and the Dana–Farber Cancer Institute in establishing a global collaboration called the Myeloma Genome Project. The goal of this project is to compile and analyze the largest set of genomic and clinical data to design a molecular classification system to improve the diagnosis, prognosis and treatment of myeloma. Up to this point, we have collated the data that we already have, and now we are bringing other national and international investigators in to really form a global consortium. This data will help us identify patients who have distinct clinical outcomes, and allow us to take a stratified-risk approach to designing treatment strategies. This initiative could really lead the way in developing specific clinical trials for targeted treatments for patients in the future.

Personalized Medicine: The Future of Cancer Care

RWHC: What are the most promising new treatments for multiple myeloma? Are there any on the horizon that hold the possibility for a cure?

GM: I believe that stem cell transplantation remains the backbone of treatment for many patients, and we don’t want to move away from a strategy we know to be successful for many. So, we are now incorporating new treatments to increase cure rates and to give more patients higher and better responses overall. This involves moving away from the one-size-fits-all approach to a more directed, personalized one which includes the use of novel agents, immunotherapy, and targeted-based treatments.

Immunotherapy is one of the more exciting developments for patients with relapsed/refractory disease, and we are now looking at incorporating these agents into the newly diagnosed setting. Using different combinations of antibodies that address the different components of the immune system is going to be a really important way forward. In addition, the increased understanding of cancer genomics has given us a wealth of information about the biological processes involved in the initiation and development of myeloma cells, which has really powered the concept of developing targeted therapies directed at specific mutations at the molecular level. This approach, also known as personalized or precision medicine, clearly represents the future of cancer care.

Underlying Causes of Myeloma

RWHC: What are some of the biggest challenges facing multiple myeloma researchers?

GM: Some challenges myeloma researchers face include gaining a better understanding of the underlying causes of myeloma and designing prevention and early intervention strategies, as well as developing strategies to prevent precursor states of myeloma—MGUS (monoclonal gammopathy of undetermined significance) and smoldering myeloma—from developing into active multiple myeloma. Of course, funding remains one of the biggest challenges faced by researchers, and it probably always will be.

Increasing collaborations with university and industry partners provides an opportunity to gain access to much larger datasets and to develop clinical trials to help answer some of these important questions.

Overcoming Resistance to Multiple Myeloma Treatment

RWHC: What are some of the biggest challenges facing clinicians treating multiple myeloma patients?

GM: Over the last decade, we’ve seen an unprecedented improvement in multiple myeloma as novel agents and treatment combinations expand. Despite the vast improvement, there remains a proportion of patients who relapse and are more likely to have aggressive disease that is refractory to therapy. I think one of the challenges in myeloma is how do we treat and design strategies that can overcome treatment resistance for these high-risk patients to improve their long-term outcomes.

This challenge requires a change that focuses on the use of genetic analyses to segment myeloma at the molecular level to enhance risk segmentation to develop biologically-stratified treatment approaches. We are also exploring the use of imaging studies to recognize high-risk and DNA-based features. Collecting the sequencing and imaging data and analyzing it consistently provides a resource for the myeloma community that can continue to grow into the future.

Another important challenge facing clinicians is patient access to a myeloma specialist. Myeloma is a rare and complex cancer, so oncologists can’t use concepts developed for more common cancers, such as breast or colon, in myeloma. The key is having a focused strategy that is directed by a myeloma expert. Unfortunately, some patients don’t have access to a specialist because of location, insurance, or other financial restrictions. At the Myeloma Institute we incorporate a team approach, whereby our team of myeloma experts direct the treatment strategy and for practical purposes, patients can receive much of their treatment locally. So, if myeloma experts can partner with local oncologists who can then deliver some of the treatment, then it’s an ideal setting for patients, because they receive the benefit of a myeloma-specific strategy and risk stratification upfront, and in the long-term, they have the comfort and security of being close to home.

 

Multiple Myeloma & Cardiac Risk

Real World Health Care continues our series on multiple myeloma with a discussion about the cardiac effects of cancer treatment with Dr. Robert Frank Cornell.  Dr. Cornell is the clinical director of the plasma cell disorder program and the director of the plasma cell/lymphoma clinical trial research program at Vanderbilt University Medical Center.

Dr. Cornell’s research focuses on translational and early phase clinical trials for the treatment of multiple myeloma. He also conducts research and clinical trials for relapsed/refractory multiple myeloma and other plasma cell disorders. He also researches cardio-oncology to better understand the effects of chemotherapy on the heart and vascular system.

Effective Treatments Carry Risks

Real World Health Care: What initially intrigued you about the cardiac effects of cancer treatments?

Robert Frank Cornell, MD, Vanderbilt University Medical Center

Robert Frank Cornell: I was initially drawn to study cardio-oncology due to some cardiac toxicities observed with a drug used to treat myeloma called carfilzomib. While this drug was found to be very effective in treating myeloma, it had a small but definitive increased risk of cardiac toxicities including heart failure, arrhythmia, hypertension, coronary syndrome and rarely sudden cardiac death.

I continue to be inspired by researching cardiac toxicities because it is through our research efforts we have determined means to both predict patients at risk for cardiac toxicity and mitigate the severity of the effects in order for patients to continue to receive this effective therapy. As more drugs are developed, such as CAR T-cell therapies, there will be ongoing need to understand any potential cardiac risk associated with treatments with the goal of improved patient outcomes and quality of life.

Many Myeloma Patients Already at Risk

RWHC: Can you describe some of the common cardiac complications involved in multiple myeloma treatment?

RFC: Since the average age of diagnosis for myeloma is around 68 years, many patients already have risk factors for cardiac toxicities such as hypertension, hyperlipidemia, diabetes mellitus, obesity and kidney dysfunction. The addition of chemotherapy in this patient population increases the risk of possible cardiac complications.

One of the most common drugs used to treat myeloma is dexamethasone. This drug is a corticosteroid and can result in fluid retention and high blood pressure. Another treatment commonly used for myeloma is high dose melphalan followed by autologous hematopoietic cell transplantation (stem cell transplant). During the course of the transplantation process, patients are monitored for development of cardiac toxicities. It is not uncommon for patients to develop atrial fibrillation (a fib) or have an exacerbation of preexisting a fib during the course of transplant due to the added stress on the heart.

Carfilzomib, as mentioned above, also increases the risk as cardiac complications. The drug class of immunomodulatory/cereblon-binding agents including lenalidomide and pomalidomide increases the risk for thromboembolic events including deep vein thrombosis (clot in the deep veins of the leg) or pulmonary embolus (clot in the artery of the lung). While this is not a traditional cardiac complication, many cardiologists monitor for this since it is thought that the blood vascular system may play a role in the development of these.

The majority of other FDA approved treatments for myeloma have very low risk for cardiac toxicity such as the drug class of reversible proteasome inhibitors including bortezomib and Ixazomib (though there are case reports of cardiac problems with bortezomib) and monoclonal antibodies including elotuzumab and daratumumab.

Collaborative Care

RWHC: What sort of challenges do clinicians face when treating multiple myeloma patients in terms of minimizing cardiac complications?

RFC: The biggest challenge I see is how best to manage a patient considered to be at very high risk for cardiac complications during the course of treatment. This patient is identified according the medical history as already having a known history of cardiac difficulties. The best strategy to overcome this is with a collaborative cardiac and oncology effort to optimize the patient’s cardiac status prior to and during the course of treatment. This includes optimization of a patient’s blood pressure, cholesterol, diabetes, heart failure, rhythm control and volume status. Should cardiac problems arise during the course of treatment, then prompt evaluation by a cardiologist should be considered, particularly in high-risk patients.

Underlying Causes of Cardiac Complications

RWHC: How can researchers overcome the challenges they face when studying cardiac complications of cancer therapies?

RFC: One of the biggest challenging for the research of cardiac complications is the determination of the underlying cause for the cardiac complication. Since patients with myeloma already often have risk factors for cardiac events, it can be challenging determining if the cardiac event occurred from the treatment itself, from supportive care as part of the treatment such as intravenous fluids, or simply occurred by chance since the patient was at higher risk for cardiac complications regardless of treatment. The cardiac event may have also occurred due to a combination of these factors. To overcome this challenge, ongoing research and collaboration between hematology and cardiology are needed to better understand the cardiac complications, develop strategies to prevent or mitigate the events and optimize patients care and outcomes to both improve survival and quality of life.

Multiple Myeloma: Promising New Therapies, But Challenges Remain

This week, our series on multiple myeloma continues as we talk about immunotherapies and other promising new treatments with Shaji Kumar, MD. Dr. Kumar is a professor of medicine in the division of hematology at Mayo Clinic, where he chairs the myeloma group across all three Mayo sites. Dr. Kumar conducts National Institutes of Health-funded research on resistance mechanisms to common myeloma drugs and epidemiology of disease progression. He also receives funding from the Multiple Myeloma Research Foundation to study the relationship between molecular profiles, treatment regimens for patients with multiple myeloma and outcomes.

Inspirations

Real World Health Care: How did you become interested in the field of multiple myeloma?

Shaji Kumar, MD, Mayo Clinic

Shaji Kumar: I did my fellowship training at Mayo Clinic. Mayo is one of the most renowned institutions in the field of plasma cell disorders. Training at Mayo, and having the fortune of working with people like Professor Robert Kyle, was very inspiring and led to my interest in this group of disorders. The patients I see keep me inspired. While we have made some progress in this area, much work needs to be done and we need to develop a cure for this disease. The progress so far convinces me that we can reach this goal if we keep at it.

High-Risk Multiple Myeloma Patients

RWHC: What is the significance of your research work to the multiple myeloma patient community?

SK: I am involved in several aspects of myeloma research. In the laboratory, we try to understand how the myeloma cell survives, especially how the other cells in the body help them grow, and try to develop new drug combinations that can lead to better treatments. I try to translate these findings to the clinic in the form of early clinical trials, which if successful, can lead to larger phase 3 trials. I am the principal investigator of several phase 1, 2 and 3 clinical trials. Another area of great interest to me is risk stratification and identification of high risk myeloma. What we have seen is that patients with high risk myeloma continue to do badly despite all the new therapies. Moreover, there are still significant issues with the current systems for identifying high risk patients. We are trying to develop new approaches for risk stratification in myeloma and develop new treatment approaches for these patients.

Monoclonal Antibodies and Immunotherapy

RWHC: What promise do monoclonal antibodies and chimeric antigen receptor (CAR T-cell) therapies hold for the treatment of multiple myeloma? Could a cure be around the corner?

SK: Immunotherapy is the next frontier for myeloma. The results so far have been spectacular. The monoclonal antibodies have opened up a new class of drugs, and daratumumab especially has led to high response rates and deep responses in patients with myeloma. The next step is to combine the current classes of drugs to develop the most effective regimens. Other immunotherapy approaches, especially CAR T-cells, while early in the testing phase, has shown significant benefit among patients with very few options left. Other approaches to enhancing the T-cell immunity such as BiTE platforms as well as vaccination approaches are in clinical trials. All in all, this is an exciting time for myeloma and I am convinced that a cure is around the corner. What needs to be determined is if this will come from treating myeloma with these approaches or whether we need to intervene at the smoldering phase.

What Triggers Multiple Myeloma?

RWHC: What are some of the biggest challenges facing researchers studying multiple myeloma?

SJ: There are many challenges facing researchers. These include the lack of understanding the triggers for development of the disease, and the ability to identify patients who progress to myeloma in a specific fashion. Lack of good models for testing the myeloma cells outside of the patient, both for its behavior and responses to treatment, remains a problem. In the clinical trial arena, the ability to do clinical trials fast and also have faster readouts using surrogate endpoints remain a challenge.

The Multiple Myeloma Landscape

Editor’s Note: This article originally appeared in Biotech Primer Weekly. For more of the science behind the headlines, please subscribe.

Emily Burke, BiotechPrimer.com

Multiple myeloma is a cancer formed by a type of white blood cell called a plasma cell. These cells are the antibody-producing cells of our immune system and play a critical role in our defense against infections. If they begin to grow and divide in an uncontrolled manner, however, they form a plasmacytoma – a mass of cells within the bone marrow that no longer function in our defense but instead simply take up space and interfere with the functions of healthy cells. Instead of producing normal disease-fighting antibodies, plasmacytoma cells produce abnormal antibodies called M proteins, which don’t provide any benefit to the body and crowd out normally functioning antibodies.

Easily Confused: Plasma Cells vs Blood Plasma

Plasma cells are specialized white blood cells that produce infection-fighting antibody proteins. Most plasma cells are found in the bone marrow. Blood plasma is the straw-colored liquid component of blood that holds blood cells in suspension, made up of water (95%), proteins, glucose, clotting factors, electrolytes, hormones, carbon dioxide, and oxygen.

Picking Apart Plasmacytoma

Plasmacytoma formation can lead to a host of problems with recognizable clinical symptoms. Because all blood cells are formed in the bone marrow, over-production of plasma cells can essentially “crowd out” normal blood-forming cells. This can lead to anemia, caused by a shortage of oxygen-carrying red blood cells; increased bruising and bleeding due to a reduction in clot-promoting platelets; and an increased risk of infections due to lower levels of healthy infection-fighting white blood cells.

Although multiple myeloma is classified as a blood cancer, it has a significant impact on bone health. As the plasmacytoma grows, bone-forming cells called osteoblasts are suppressed. At the same time, production of a substance that activates bone-reabsorbing cells, osteoclasts, is increased. The resultant damage to the bone structure results in soft spots or lesions which may extend from the inner bone marrow to the outside surface of the bone. Bone lesions result in significant pain and increase the risk of fracture. Bone destruction also releases excessive calcium into the bloodstream, which leads to a range of symptoms including changes in urination, restlessness, confusion, increased thirst, nausea, and loss of appetite. Excess blood calcium, combined with high levels of M protein, also contributes to the impaired kidney function seen in multiple myeloma patients.

Unmasking Multiple Myeloma

There is no one diagnostic test for multiple myeloma. Blood and urine tests to detect some of the symptoms listed above such as low blood cell counts, elevated blood calcium levels, and impaired kidney function may suggest multiple myeloma. These tests can be followed by a bone marrow biopsy for confirmation.

Most cases of multiple myeloma have no known cause, although some research suggests that regular exposure to herbicides, insecticides, petroleum products, heavy metals, and asbestos increases the risk of developing the disease. And although there is not a specific gene yet associated with multiple myeloma, abnormalities in chromosome structure or number are associated with the disease.

Multiple Myeloma Treatments

Once considered incurable, there are now a number of effective treatments for multiple myeloma, and several more are in the pipeline.

Currently, there are two FDA-approved monoclonal antibody therapeutics approved to treat multiple myeloma.  They work by recognizing and binding to proteins on the surface of multiple myeloma cells, activating the patient’s immune system to destroy those cells.

Another type of approved therapy for multiple myeloma is a small molecule proteasome inhibitor therapy. A proteasome is a specialized compartment within the cell that gets rid of damaged proteins by digesting them. If the proteasome is inhibited, damaged proteins build up within the cell. This triggers a process called apoptosis – essentially, cell suicide. In other words, the cancer cell kills itself.

Small molecule histone deacetylase (HDAC) inhibitors have also been shown to be safe and effective in treating multiple myeloma. HDACs are enzymes that modify chromosomes (strands of DNA that contain our genes) and influence how often specific genes are activated. Some cases of multiple myeloma are associated with changes in gene activation. By inhibiting HDACs, this faulty gene expression can be corrected.

In the Pipeline

Two novel drugs in the multiple myeloma pipeline are Mivebresib and Selinexor.

Mivebresib influences the activation of specific genes by inhibiting a group of proteins called Bromodomain and Extra Terminal motif (BET) proteins. In some types of cancer, genes are activated or deactivated inappropriately due to BET activity. By inhibiting BET, normal gene activity may be restored to these cells.

Selinexor helps to increase the number of tumor suppressor proteins present in the nucleus of cancer cells. These proteins help to protect against cancer by detecting DNA damage and promoting apoptosis in those cells that have high levels of DNA damage. In many types of cancer cells, tumor suppressor proteins are transported out of the nucleus, where they can no longer do their job of detecting DNA damage. Selinexor blocks this transport and enables tumor suppressor proteins to do their job of triggering apoptosis in cancer cells.

A number of CAR-T therapies are also in development for multiple myeloma, with several early stage clinical trials ongoing. 

Multiple myeloma is a complex type of cancer. In recent years, a better understanding of the disease has led to the approval of several new therapeutics. In the coming years, we can look forward to additional approvals as novel therapeutics move through the pipeline.