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Insider Insights: Understanding Multiple Myeloma

Multiple Myeloma is a type of cancer that explicitly affects plasma cells essential components of our immune system. These plasma cells go rogue, multiplying uncontrollably and crowding out healthy cells, leading to various health challenges. It might not be a household name, but its impact is significant. In this section, we'll unravel the basics, from what Multiple Myeloma is to why it's essential to comprehend it.

Multiple Myeloma: Incidence and Prevalence

Multiple Myeloma, a form of blood cancer might be relatively rare, accounting for just 1.8% of all new cancer cases in the United States. However, its impact is profound. According to the American Cancer Society, approximately 34,920 new cases were diagnosed in 2021, highlighting the importance of understanding its various aspects.

Multiple Myeloma: Symptoms & Signs

Multiple Myeloma is often tricky to detect in its early stages. It usually manifests through symptoms such as

Bone pain (especially in the spine, chest, or hips)

Recurrent infections

Fatigue

Weight loss

Mental fogginess or confusion

Constipation

It is essential to identify these signs early as it helps take timely action and address the issue. Medicines like Carfilnat 60mg Carfilzomib Injection, are used to treat Multiple Myeloma.

Diagnostic Tests for Multiple Myeloma

Getting to the bottom of Multiple Myeloma involves a series of diagnostic tests.

Blood and Urine Analyses

These tests play a crucial role in detecting abnormalities associated with Multiple Myeloma. Blood tests may reveal elevated levels of specific proteins (M proteins made by myeloma cells) or abnormalities (beta-2-microglobulin) in blood cell counts. Urine tests can detect abnormal proteins (Bence Jones proteins) that may indicate the presence of the disease.

Imaging Tests like X-rays

X-rays provide a visual insight into the bones, helping identify any lesions or fractures caused by Multiple Myeloma. This diagnostic tool aids in assessing the extent of bone involvement and contributes to the staging of the disease.

Bone Marrow Tests

Bone marrow tests such as Bone marrow biopsies & aspirations involve extracting a small sample of bone marrow for examination. This procedure helps determine the presence and activity of myeloma cells. It's a crucial step in diagnosing Multiple Myeloma and provides valuable information for planning an effective treatment strategy.

Each piece of this diagnostic puzzle contributes to a clearer picture of the condition.

Staging System

Understanding the staging system helps map out the extent of the disease.

Stage I

Smoldering or asymptomatic myeloma.

M protein in the blood or urine but no symptoms or evidence of organ damage.

Lower levels of abnormal plasma cells in the bone marrow.

Stage II

Calcium levels may be normal or slightly elevated.

Moderate levels of abnormal plasma cells in the bone marrow.

Stage III

Multiple Myeloma has spread extensively. 

There are signs of kidney problems. 

Calcium levels might be elevated.

Higher levels of abnormal plasma cells in the bone marrow.

This knowledge guides healthcare professionals in tailoring each individual's most effective treatment plan.

Treatment Options:-

Chemotherapy

Chemotherapy, a reliable cancer treatment, is a critical player in battling Multiple Myeloma. This treatment involves using powerful drugs to target and control the abnormal plasma cells responsible for the disease. It targets and controls abnormal plasma cell multiplication and growth to restore balance to the body's blood cell production.

Stem Cell Transplantation

Stem cell transplantation, a sophisticated approach, rejuvenates the bone marrow. In this procedure, the patient's stem cells are harvested and reintroduced into the body after being enhanced in a controlled environment. This process acts like a reset button for the immune system, enhancing its ability to combat Multiple Myeloma. 

Targeted Therapy

Enter the era of targeted therapy with Carfilnat 60mg Carfilzomib Injection, containing the potent carfilzomib. This modern approach disrupts the growth mechanisms of Multiple Myeloma cells with precision, offering a more tailored and effective treatment.

Carfilnat 60mg Carfilzomib Injection

Carfilnat 60mg is a form of targeted therapy, meaning it focuses explicitly on the growth mechanisms of Multiple Myeloma cells with precision. 

This Carfilzomib injection disrupts the intricate processes fueling cancerous cells, halting their uncontrolled growth. It's like sending a highly specialized task force to dismantle the essential components driving the disease. This targeted approach is designed to minimize the damage to healthy cells, making the treatment more tolerable for patients with relapsed multiple Myeloma (when the cancer returns) and refractory multiple Myeloma (when the cancer no longer responds to treatment).

Benefits of Carfilnat 60mg Carfilzomib Injection

Precision Targeting

Carfilnat 60mg injection, containing carfilzomib, offers a precision-targeted approach to treating Multiple Myeloma. It blocks or turns off signals that encourage cancer cells to grow or multiply and Modify proteins within cancer cells that cause those cells to die.

Improved Disease Control

Carfilnat 60mg Carfilzomib injection's ability to disrupt the abnormal growth of myeloma cells plays a crucial role in controlling the progression of Multiple Myeloma by preventing new blood vessels from forming, which cuts off blood supply to the tumor.

Minimized Side Effects

By honing in on cancer cells precisely, Carfilnat 60mg Carfilzomib injection reduces the impact on healthy cells, leading to a lower incidence of side effects. It delivers the toxins that kill cancer cells without harming healthy cells. This targeted approach enhances the treatment's overall tolerability, improving patients' quality of life.

Conclusion:-

Multiple Myeloma is more than statistics or treatments; it's about individuals facing it. Each story is acknowledged as unique, and each journey is seen as a testament to strength and hope. The collective goal is highlighted – striding towards a future where Multiple Myeloma is understood and conquered.

Carfilnat 60mg Carfilzomib Injection brings many benefits, ranging from precision targeting and enhanced treatment efficacy to minimized side effects and improved disease control. As a modern advancement in Multiple Myeloma treatment, it represents hope and progress in the ongoing battle against this complex condition.

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Comprehensive Overview of Multiple Myeloma Treatment Methods: A Doctor's Perspective

Multiple myeloma is a challenging cancer that affects plasma cells in the bone marrow. Early detection and treatment are critical to managing the disease. With various multiple myeloma treatment methods available today, we can significantly improve patient outcomes. This post will explore the most common treatments, including chemotherapy, targeted therapy, immunotherapy, and drugs like lenalidomide.

Standard Treatment Methods for Multiple Myeloma

Chemotherapy

Chemotherapy is a cornerstone of multiple myeloma treatment. It works by killing fast-growing cells, including cancerous plasma cells. Common chemotherapy drugs used include melphalan and cyclophosphamide.

Chemotherapy is often combined with other treatments to increase effectiveness. For example, it can be used before a stem cell transplant or in cases of aggressive disease. Despite its benefits, chemotherapy can cause side effects such as fatigue, nausea, and infections due to a weakened immune system.

Targeted Therapy

Targeted therapy specifically attacks proteins or genes that are key to cancer cell growth. Unlike chemotherapy, which affects both healthy and cancerous cells, targeted therapy is more precise.

Drugs like bortezomib (Velcade), carfilzomib (Kyprolis), and ixazomib (Ninlaro) are common targeted treatments for multiple myeloma. They work by inhibiting proteasomes, causing cancer cells to die.

Targeted therapies are often combined with other drugs, such as lenalidomide, to boost their effectiveness. These drugs generally have fewer side effects than traditional chemotherapy, although they can cause neuropathy or gastrointestinal issues.

Immunotherapy

Immunotherapy is one of the most promising advancements in multiple myeloma treatment. This therapy uses the body's immune system to target and destroy cancer cells.

Lenalidomide (Revlimid) and pomalidomide (Pomalyst) are immunomodulatory drugs widely used in newly diagnosed and relapsed cases of multiple myeloma. Lenalidomide enhances the immune system’s ability to kill cancer cells and prevents the formation of blood vessels that feed tumors.

Lenalidomide is often combined with dexamethasone, a corticosteroid that enhances its anti-cancer effects. However, side effects such as low blood counts, fatigue, and an increased risk of blood clots need to be managed closely.

Stem Cell Transplantation: A Curative Option

For some patients, stem cell transplantation offers a more curative approach. Two types of transplants are used: autologous (using the patient’s own stem cells) and allogeneic (using donor stem cells).

In many cases, patients undergo autologous stem cell transplantation after high-dose chemotherapy to reduce cancer cells. Although it can lead to long-term remission, there are risks, including infection and relapse.

Lenalidomide: A Pillar in Multiple Myeloma Therapy

Lenalidomide is a central drug in the treatment of multiple myeloma. It is used in both newly diagnosed and relapsed patients, often combined with other treatments like bortezomib and dexamethasone.

One of the biggest challenges with lenalidomide is its cost. The price of lenalidomide can range from $10,000 to $20,000 per month, making it difficult for many patients to afford. Despite its cost, lenalidomide is highly effective at prolonging progression-free survival and improving quality of life.

The Cost of Multiple Myeloma Treatment

The cost of multiple myeloma treatments can be significant. Beyond lenalidomide, other drugs like bortezomib and carfilzomib are also expensive, especially when used in combination therapy.

In addition to drug costs, patients often incur expenses related to stem cell transplants, hospital stays, and supportive care like blood transfusions or bone-strengthening medications. Navigating insurance coverage and patient assistance programs is crucial for many patients to manage these costs effectively.

Conclusion: The Future of Multiple Myeloma Treatment

The treatment landscape for multiple myeloma is evolving rapidly, with exciting new therapies like CAR-T cell therapy and bispecific antibodies showing promise in clinical trials. As a doctor, I am hopeful that these advancements will lead to even better outcomes for my patients.

For now, the key to successful treatment lies in a personalized approach that balances the effectiveness of therapies like chemotherapy, immunotherapy, and targeted therapy with their side effects and costs. Despite the high cost of medications like lenalidomide, their benefits make them invaluable tools in the fight against multiple myeloma.

Frequently Asked Questions (FAQ)

1. What is the most common treatment for multiple myeloma?

The most common treatment for multiple myeloma typically includes chemotherapy, targeted therapy, and immunotherapy. A combination of these treatments is often used depending on the patient’s stage and health. Stem cell transplants are also considered for patients eligible for more aggressive treatments.

2. How does lenalidomide help in treating multiple myeloma?

Lenalidomide (Revlimid) is an immunomodulatory drug that helps enhance the immune system’s ability to attack cancer cells. It also prevents the growth of blood vessels that tumors need to survive. Lenalidomide is often combined with other medications like dexamethasone and is used in both newly diagnosed and relapsed patients.

3. Why is the cost of multiple myeloma treatment so high?

The cost of multiple myeloma treatments can be high due to the expense of newer therapies like lenalidomide, bortezomib, and carfilzomib. Additionally, stem cell transplants, hospital stays, and supportive care add to the overall cost. Many patients rely on insurance and assistance programs to help cover these expenses.

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Carfilzomib Market Statistics, Segment, Trends and Forecast to 2034

TheCarfilzomib Market: Trends, Opportunities, and Future Outlook

Introduction

In recent years, the pharmaceutical industry has seen significant advancements in the treatment of multiple myeloma, a type of cancer that affects plasma cells in the bone marrow. Among these advancements, Carfilzomib has emerged as a critical player. This proteasome inhibitor, marketed under the brand name Kyprolis, has made substantial impacts in the management of multiple myeloma. In this blog, we will explore the current state of theCarfilzomib market, its growth drivers, opportunities, and future outlook.

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What is Carfilzomib?

Carfilzomib is a next-generation proteasome inhibitor used in the treatment of multiple myeloma, especially in patients who have relapsed or are refractory to other therapies. Unlike its predecessor, bortezomib, Carfilzomib is known for its more selective action on the proteasome, potentially leading to fewer side effects and enhanced efficacy. Approved by the FDA in 2012, it has since become an integral part of combination therapies for multiple myeloma.

Carfilzomib Market Landscape

Growth Drivers

Rising Incidence of Multiple Myeloma: The increasing number of multiple myeloma cases globally is a significant driver for the Carfilzomib market. As the global population ages, the prevalence of multiple myeloma is expected to rise, leading to a higher demand for effective treatments.

Advancements in Treatment Protocols: Carfilzomib is often used in combination with other drugs like lenalidomide and dexamethasone, enhancing its effectiveness. This combination therapy approach has shown promising results, making Carfilzomib a preferred choice in advanced treatment regimens.

Increasing Awareness and Diagnosis: Improved diagnostic techniques and greater awareness about multiple myeloma have led to earlier detection and treatment. This trend is likely to boost the demand for Carfilzomib as part of first-line and subsequent lines of treatment.

Ongoing Clinical Trials: Continuous research and clinical trials exploring new indications and combination therapies for Carfilzomib are expanding its potential market. Studies focusing on different stages of multiple myeloma and other cancers could open new avenues for Carfilzomib use.

Carfilzomib Market Challenges

High Cost of Treatment: Carfilzomib is a high-cost drug, and its price can be a barrier to access, particularly in developing countries. The high cost of treatment may limit its market potential and lead to a preference for alternative therapies.

Side Effects and Resistance: Although Carfilzomib is well-tolerated, some patients may experience side effects such as cardiovascular issues or renal complications. Additionally, resistance to the drug can develop, leading to the need for alternative therapies.

Competition from Other Therapies: The multiple myeloma treatment landscape is competitive, with several other proteasome inhibitors and novel therapies in development. This competition can impact Carfilzomib’s market share and pricing strategies.

Market Opportunities

Expanding Indications: Research into expanding the use of Carfilzomib to other types of cancer or earlier stages of multiple myeloma could provide significant market opportunities. Successful clinical trials in these areas could lead to new indications and broaden its market.

Developing Markets: As healthcare infrastructure improves in developing regions, there ispotential for growth in these Carfilzomib Market Strategic partnerships and pricing strategies could enhance Carfilzomib’s reach in these areas.

Combination Therapies: Exploring new combination therapies and optimizing treatment regimens can improve patient outcomes and increase the demand for Carfilzomib. Collaborations with other pharmaceutical companies and research institutions could drive innovation in this space.

Carfilzomib Market Future Outlook

The Carfilzomib market is poised for continued growth driven by advancements in treatment protocols and increasing prevalence of multiple myeloma. However, challenges such as high treatment costs and competition from alternative therapies will require strategic planning and innovation.

The future of Carfilzomib will likely involve ongoing research and development to expand its therapeutic indications and improve patient outcomes. As the market evolves, stakeholders will need to navigate these dynamics to maximize the potential of Carfilzomib in the treatment of multiple myeloma and beyond.

Conclusion

Carfilzomib Market has made asignificant impact in the management of multiple myeloma, and its market prospects remain strong. With ongoing advancements in treatment protocols, increasing awareness, and expanding indications, Carfilzomib is set to continue playing a crucial role in cancer therapy. However, addressing market challenges and seizing emerging opportunities will be essential for stakeholders to fully realize the potential of this innovative drug.

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Blood Cancer Drugs Market to Generate Exciting Opportunities in the Industry by 2027

The global blood cancer drugs market is anticipated to reach USD 55.6 billion by 2025 according to a new report published by Polaris Market Research. The report ‘Blood Cancer Drugs Market [By Blood Cancer Type (Leukemia (Acute Myeloid Leukemia, Chronic Myeloid Leukemia, Acute Lymphoblastic Leukemia, Chronic Lymphocytic Leukemia), Lymphoma (Hodgkin Lymphoma, Non-Hodgkin Lymphoma (B-Cell Lymphoma, T-Cell Lymphoma)), and Myeloma; By Drugs (Rituaxan/Mabthera (Rituximab), Gleevac/Glivec (Imatinib), Revlimid (Lenalidomide), Velcade (Bortezomib), Tasigna (Nilotinib), Pomalyst (Pomalidomide), Vidaza (Azacitidine), Kyprolis (Carfilzomib), Adcetris (Brentuximab Vedotin), and Others); By Treatment Approaches (Chemotherapeutic, mAbs/Targeted Therapies, and Immunotherapeutic); By Region]: Market Size & Forecast, 2017 – 2025’ provides insights on the current market scenario and the future prospects.

The demand for blood cancer drug is primarily driven by growing death incidences by blood cancer, and continuous innovation for developing novel treatments with the help of several ongoing clinical trials. Moreover, increasing research and development of biological and targeted therapies as treatment will spur the blood cancer drugs market during the upcoming period. However, the high price of drugs and the stringent government policies will limit the growth of blood cancer drugs market during the forecast period.

Get Sample copy of this report: https://www.polarismarketresearch.com/industry-analysis/global-blood-cancer-drugs-market/request-for-sample

Most of the blood cancers start in the bone marrow, where blood is produced. In blood cancer the growth of normal blood cells is dislodged by the uncontrollable growth of abnormal blood cells. These cancerous cells prevent the blood from performing many of its functions. Hence, the existing treatments of blood cancer are being the foundation for developing the new drugs. The steady flow of the blood cancer drugs has created opportunity for research and development in the existing market. For instance, Amgen Inc. received approval for BLINCYTO in July 2017, which is used in treating B-cell precursor Acute Lymphoblastic leukemia. Similarly, European blood cancer drugs market witnessed the approvals of Gazyvaro, by Roche AG that is used in treating advanced follicular lymphoma. Also, novel technologies like CAR-T are likely to be launched this year.

The global blood cancer drugs market is segmented into blood cancer type, drugs and treatment approaches. On the basis of blood cancer type, the global blood cancer drugs market is segmented into leukemia, lymphoma and myeloma. The lymphoma segment is expected to drive the majority market of blood cancer drugs followed by leukemia. The global market of this segment is primarily driven by the increasing prevalence of lymphoma, and presence of effective treatments in the market. On the basis of drugs, the global blood cancer drugs market is further categorized into Rituaxan/Mabthera (Rituximab), Gleevac/Glivec (Imatinib), Revlimid (Lenalidomide), Velcade (Bortezomib), Tasigna (Nilotinib), Pomalyst (Pomalidomide), Vidaza (Azacitidine), Kyprolis (Carfilzomib), Adcetris (Brentuximab Vedotin), and Others. This continuous innovation for treating various sub-types of blood cancers has led to the development of novel types of treatments. For instance, the combination of Revlimid and Velcade has emerged as the preferential drugs in trials for treating multiple myeloma.

The leading companies operating in this industry include Johnson & Johnson Inc., Amgen Inc., Bayer AG., Pfizer, Inc., AbbVie Inc., Roche Holding AG., Celgene Corporation, AstraZeneca, Novartis AG, GlaxoSmithKline PLC, Merck & Co., Inc., and Eli Lily & Co. among others.

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About Polaris Market Research

Polaris Market Research is a global market research and consulting company. The company specializes in providing exceptional market intelligence and in-depth business research services for our clientele spread across different enterprises. We at Polaris are obliged to serve our diverse customer base present across the industries of healthcare, technology, semi-conductors and chemicals among various other industries present around the world

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Phone: 1-646-568-9980

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Best multiple myeloma treatment in India

Multiple myeloma is cancer of the plasma cells (a type of white blood cells) of the bone marrow. Plasma cells are protein-making cells that generally produce the different kinds of antibodies for our immune system. In multiple myeloma, the plasma cells become malicious and cancerous. These myeloma cells stop making different forms of protein in response to the immune system's needs and instead start to produce a single abnormal type of protein sometimes termed a monoclonal or M protein. Multiple myeloma plasma cell populations accumulate in the bone marrow, and these collections of cells called plasmacytomas can erode the hard outer shell or cortex of the bone that normally surrounds the marrow. These weakened bones show thinning of the bone, as seen in nonmalignant osteoporosis or what appear to be punched out or lytic bone lesions. People often refer to multiple myeloma simply as myeloma (also termed Kahler's disease after the physician who first described this cancer). The disease usually occurs in people past middle age.

In India, there are large number of options available for Best multiple myeloma treatment in India.

However, rarely it can occur in a child. One type of myeloma-related plasma cell neoplasm is called a monoclonal gammopathy of undetermined significance (MGUS). In MGUS, medical professionals only find low levels of M protein and people have no symptoms; MGUS infrequently develops into multiple myeloma.

Plasma cell neoplasm is another name for multiple myeloma. Causes of multiple myeloma What triggers plasma cells into malicious multiple myeloma is unknown. The cancerous myeloma plasma cells proliferate and crowd out normal plasma cells and can corrode areas of bones. The proteins produced in large amounts can cause many of the symptoms of the disease by making the blood more viscous and depositing the proteins in organs that can interfere with the functions of the kidneys, nerves, and immune system.

Causes of multiple myeloma are not known exactly. But patients more likely to get affected • older than 65 years • people of African-American origin • overweight or obese people • family member with it

Stages of multiple myeloma

There are four stages of multiple myeloma. While many health care professionals use different staging, these are various stages cited by many clinicians:

• Smoldering: multiple myeloma with no symptoms • Stage I: early disease with little anemia, relatively small amount of M protein and no • bone damage • Stage II: more anemia and M protein as well as bone damage • Stage III: still more M protein, anemia, as well as signs of kidney damage Because staging criteria differ according to different groups, some clinicians simply define the individual's multiple myeloma without assigning a stage and simply estimate a prognosis for their patient.

Symptoms of multiple myeloma

Patients with myeloma may be asymptomatic with an unexplained increase in protein in the blood. With more advanced disease, some myeloma patients may have weakness due to anemia caused by inadequate production of red blood cells, with bone pain due to the bone damage, and as the abnormal M protein can accumulate and damage the kidneys resulting in patient’s unexplained kidney damage and decreased kidney function. Multiple myeloma cancer cells may be in or outside the bone marrow.

The following symptoms and signs of multiple myeloma -

• Anemia • Bleeding • Nerve damage • Bone tenderness or pain, including back pain • Enlarged tongue • Skin lesions (rash) • Infections Weakness, fatigue or tiredness • Kidney failure and/or other end-organ damage• Spinal cord compression • • Loss of appetite and weight loss • Leg swelling • Hypocalcaemia • Diagnosis of multiple myeloma • First sign of multiple myeloma is found when a routine blood test shows an abnormal amount of protein in the bloodstream or an unusual stickiness of red blood cells causing them to stack up almost like coins, an unusual formation for red blood cells. The health care professional will do a history and physical exam, looking for signs and symptoms of multiple myeloma. If multiple myeloma is suspected, several studies help confirm the diagnosis.

They include a bone marrow aspiration and biopsy most commonly from the large bones of the pelvis. Cells obtained from the marrow are studied by a pathologist to determine if there is one (plasmacytoma) or more (multiple myeloma) abnormal types or numbers of cells • Medical professionals also study a sample of the bone marrow aspirate for more detailed • Characteristics such as the presence or absence of abnormal numbers or types of chromosomes (DNA) by what is called cytogenetic testing.

Bone marrow biopsy can assess the concentrations of cells in the marrow and the presence of abnormal invasive growth of cellular elements. • Blood testing and urine testing by several methods can determine levels and types of National Comprehensive Cancer Network (NCCN) recommended that health care professionals use a serum free light chain assay and fluorescence in situ hybridization (FISH) test to further • Monoclonal protein produced and if there is kidney damage.

Identify multiple myeloma in patients

X-ray studies to identify skeletal lesions and MRI for spinal cord lesions in multiple myeloma.

Medical treatment for multiple myeloma

The therapy is decided based upon the patient's condition and the cancer management team, made with the patient's input. The choices for treatment(s) often include combinations of drugs, some of which medical professionals give as pills and others by intravenous injection.

These include drugs that affect or modulate the immune system, steroids, and some oral or injectable chemotherapy drugs. These are usually used in combinations. There may be a role for high-dose chemotherapy followed by the administration of bone marrow called a stem cell transplant. Numerous factors come into play in determining whether to do such a transplant. Other medical treatments may include steroids, bisphosphonate therapy, blood or platelet transfusions, plasmapheresis, and other combination therapy depending on the individual patient's disease stage.

Radiation therapy may treat painful areas of bone damage. Surgeons can surgically repair broken bones in many cases.

There are many drugs used to treat multiple myeloma. Medical professionals often use the following drugs in combination with dexamethasone,

• Bortezomib Velcade -- protease inhibitor • Lenalidomide (Revlimid) -- immune cell modulation • Melphalan (Alkeran) -- alkylating agent that is toxic to myeloma cells • Carfilzomib (Kyprolis) -- protease inhibitor that is FDA approved usually for patients • who have failed a previous treatment • Daratumumab (Darzalex) -- monoclonal antibody that may damage or kill multiple • Myeloma cells (and others) that have CD38 protein on their surface • Elotuzumab (Empliciti) -- a compound that activates the body's natural killer cells to • Destroy multiple myeloma cells, usually in combination with Revlimid and Decadron • Ninlaro (Ixazomib) -- This proteasome inhibitor, in combination with Revlimid and • Dexamethasone, improves the survival rates of some patients with multiple myeloma.

Hospitals offer best multiple myeloma treatment in India, the charges for autologous stem cell transplant ranges between USD 15000 to USD 21000 depending on the status of the disease and individual's response to the treatment provided at the hospitals.

Related Articles -

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About GHN Healthcare -

GHN Healthcare Services is a leading Medical treatment Assistance Company based in New Delhi (National Capital region), India. The Organization actively engaged in providing Medical assistance to foreign Patients who choose to travel to India for quality medical care at a reasonable cost. GHN Healthcare Services is associated with 25+ Top-Notch Hospitals and has a network of 500+ Super Specialists to offer world-class medical care at a reasonable cost.

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Multiple Myeloma Market Size Forecast, Epidemiology Analysis, Emerging Drug Uptake and Key Companies Assessment by DelveInsight

(LAS VEGAS, US) The Multiple Myeloma Market size across the 8MM  i.e. the United States, Eu5(the UK, Germany, France, Italy, Spain), China and Japan, was $16.27bn in 2019 and is increasing with a modest CAGR during the study period (2017-2030), according to DelveInsight, a leading company in healthcare analytics and consulting.  

The market size for Multiple Myeloma therapies will increase dramatically by 2030 due to the arrival of upcoming therapies that will carve out niche roles in the drug landscape. DelveInsight's latest report on Multiple Myeloma Epidemiology and Market indicates that the impressive growth in market size is due to the rise in incident cases of Multiple Myeloma in 8MM, incorporation of immune-therapies in treatment landscape increased patient adherence and adoption of newer therapies. Also, rich emerging pipeline, better diagnosis, rising awareness and expected increase in investment in the R&D activities are some additional factors that are going to fuel the market.

Some key highlights from the report:

The     total incident cases of Multiple Myeloma are expected to reach 91,520 in     2020 in the 8MM

Higher     usage of bortezomib based regimen observed across the US, EU5 and JP;     Bortezomib + Lenalidomide + dex regimen is the preferred treatment choice     in the US and Japan in the first-line compared to Bortezomib +     Melphalan ± Prednisone and Bortezomib + dex regimen in EU5. In the case     of China, higher usage of thalidomide based regimen found in the     first-line setting

Among     emerging therapies, Bristol Myers Squibb and Bluebird Bio's anti-BCMA CAR     T Cell Therapy Idecabtagene Vicleucel (Ide-cel, bb2121) is expected to     result in significant revenues owing to promising results, one-time dosing     and premium pricing in heavily pretreated patients

Key     Companies fuelling the Multiple Myeloma market size growth are     GlaxoSmithKline, Bristol-Myers Squibb, AbbVie, Roche, Janssen Research     & Development, Merck Sharp & Dohme Corp., Pfizer, Takeda, Amgen,     AstraZeneca etc.

Patent     expiry of multiple blockbuster drugs like Revlimid, Pomalyst, Darzalex and     Kyprolis is on the lines to expire from 2026 onwards, and this will erode     the sales value significantly and the market is expected to decline from     2028 onwards due to cumulative impacts of patent expiry.

Download Multiple Myeloma Sample: https://www.delveinsight.com/sample-request/multiple-myeloma-market

Though Multiple Myeloma is not common cancer; still, it is the second most diagnosed blood cancer in the United States. It is worth highlighting that it is a heterogeneous haematological malignancy in which epidemiology plays an increasingly important role. Over the past years, intensive clinical and molecular epidemiological research has extended the information about its pathogenesis, risk factors, and prognostic components which aided the approval of new drugs. Despite arduous research endeavors, the etiology remains enigmatic. The untimely or prolonged diagnosis has a severe impact on the clinical course of Multiple myeloma and a negative impact on disease-free survival. It's an unfortunate fact that approximately 95% of all the Multiple Myeloma cases are diagnosed at distant stages. The report reviews the changing epidemiology and provides the forecasts upto 2030, highlights treatment patterns, and the health disparity observed in important subgroups of Multiple Myeloma.

Multiple myeloma report contains epidemiological analysis segmented as the following: 

Total     Multiple Myeloma Incident Cases 

Total     Multiple Myeloma Incident cases by Age Distribution

Total     Symptomatic Multiple Myeloma Cases 

Cases     of Multiple Myeloma by Treatment Line 

Gender-specific     cases of Multiple Myeloma

Multiple Myeloma Market Scenario

The current standard of care of Multiple myeloma treatment includes stem cell transplant, conventional chemotherapy, targeted therapy, surgery, and radiation therapy. Chemotherapies can be given alone or in conjunction with other drugs including corticosteroids, proteasome inhibitors, immune-modulators, and monoclonal antibodies, anti-resorptive agents such as bisphosphonates and NSAIDs, or narcotics.  Currently, Lenalidomide is the market leader in multiple myeloma treatment landscape, and usage as monotherapy and also in combination with other therapies can be found across all the settings. Darzalex is also being used by the healthcare experts in combination as doublet, triplet, and quadruplet with existing therapies for the good treatment strategies and better result outcomes and has impacted the MM treatment landscape significantly. Label expansion along with higher usage of Darzalex has translated into higher revenues and expected to generate maximum revenue by 2025 before the competition and expected patent expiry would erode the sales value. At present, among the IMid's agents, Revlimid dominates the Multiple Myeloma market in the 8MM, where it is included in all lines of Multiple Myeloma therapy either as monotherapy or in combination with other drugs. In 2017, Revlimid generated a revenue of USD 7,140 million in the 8MM. Despite the loss of patent exclusivity among the major markets and approval of other potential pipeline candidates, it will continue to maintain a strong presence during the forecast period as a molecule. The novel emerging therapies are expected to bring a paradigm shift in the Multiple Myeloma treatment landscape by catering to larger unmet needs.

 Report includes exhaustive analysis of Multiple Myeloma Pipeline Therapies

Idecabtagene     vicleucel (ide-cel): Bristol Myers Squibb and bluebird bio

Venetoclax     (Venclexta, Venclyxto): AbbVie

JNJ-68284528     LCAR-B38M/JNJ-4528): Janssen Research and Development

Keytruda     (pembrolizumab): Novartis

Melflufen     (melphalan flufenamide): Oncopeptides

Cetrelimab     (JNJ-63723283): Janssen Research and Development

REGN5458:     Regeneron Pharmaceuticals

Iberdomide:     Celgene

NY-ESO-1     C259 T Cells: GlaxoSmithKline

Braftovi     (encorafenib): Pfizer

JCARH125:     Celgene Corporation

Felzartamab     (MOR202): I-Mab Biopharma

Chidamide     (Epidaza): Shenzhen Chipscreen Bioscience

The past couple of decades have shown vast changes in the treatment landscape of Multiple Myeloma, starting with the use of stem cells trailed by the availability of novel treatments such as immunomodulators and proteasome inhibitors that have transformed the natural history of the indication, leading to increased survival times. Ongoing advancements in  emerging Novel therapies such as CAR-T cell and monoclonal antibodies are showing promising results in treating multiple myeloma patients and are also expected to drive the growth of the market. Recent years have witnessed an influx of several pharma companies exploring the Multiple Myeloma market through novel targets and therapies. Although the emerging pipeline candidates will reduce the gaps however, the opportunities will still remain. As a ray of hope, the launch of new drugs have extended the median overall survival of Multiple Myeloma patients to 4-6 years; however the stark reality is that the ten year survival rate is 3%. The long term burden of the therapies makes life pretty difficult for the patients. There is a dire need for a permanent cure. Another hurdle is the existing expensive treatment options that create a barrier for the aged uninsured population to get hold of costly therapies. The factors like drug-induced toxicities, differences in clinical care, access to the therapies in real-world settings are the observed gaps between trial-based and real-world outcomes. DelveInsight believes that the Multiple Myeloma market is an attractive prospect for the companies due to the high unmet needs and low regulatory hurdles. 

Key Trends of Multiple Myeloma Market Report: https://www.delveinsight.com/report-store/multiple-myeloma-market

Scope of the Report

Geography     Covered: 8MM - The United States, EU5 (Germany, France, Italy, Spain,     and the United Kingdom), Japan, and Chine.

Study     Period: 3-year historical and 11-year forecasted analysis (2017-2030).

Markets     Segmentation: By Geographies, By Therapies (Forecasted + Historical).

Companies     Covered: GlaxoSmithKline, Bristol-Myers Squibb, AbbVie, Roche, Janssen     Research & Development, Merck Sharp & Dohme Corp., Pfizer, Takeda,     Amgen, AstraZeneca, and several others.

Analysis:     Comparative and conjoint analysis of emerging therapies, Attribute     Analysis,

Case     Studies

KOL's     Views

Analyst's     View

The Multiple Myeloma Marketed drugs covered report are:

Sarclisa     (Isatuximab): Sanofi

Darzalex     (Daratumumab): Janssen Research and Development

Empliciti     (Elotuzumab): Bristol-Myers Squibb and AbbVie

Velcade     (bortezomib): Takeda

Pomalyst     (Pomalidomide): Celgene Corporation

Revlimid     (Lenalidomide): Celgene Corporation

Farydak     (panobinostat): Novartis Pharmaceuticals

Kyprolis     (Carfilzomib): Amgen

Ninlaro     (ixazomib): Takeda Pharmaceuticals

Blenrep     (Belantamab Mafodotin): GlaxoSmithKline

Table of Contents

1

Key Insights

2

Executive Summary of Multiple Myeloma

3

KOL Views

4

SWOT Analysis of Multiple Myeloma 

5

Multiple Myeloma Market Overview at a Glance

6

Disease Background and Overview: Multiple Myeloma 

7

Diagnosis of Multiple Myeloma

8

Multiple Myeloma Epidemiology and Patient Population

9

The United States Multiple Myeloma Epidemiology

10

EU-5 Multiple Myeloma Epidemiology

11

Japan Multiple Myeloma Epidemiology

12

China Multiple Myeloma Epidemiology

13

Multiple Myeloma Treatment

14

Unmet Needs in the Multiple Myeloma Market

15

Patient Journey of Multiple Myeloma

16

Key Endpoints of Multiple Myeloma Clinical Trials

17

Multiple Myeloma Marketed Therapies

18

Multiple Myeloma Emerging Therapies

19

Multiple Myeloma 8 Major Market Analysis

20

8MM Multiple Myeloma Market Size

21

The United States Multiple Myeloma Market Size

22

EU-5 Multiple Myeloma Market Size

23

Japan Multiple Myeloma Market Size

24

China Multiple Myeloma Market Size

25

Market Access and Reimbursement of Multiple Myeloma (MM)  Therapies

26

Multiple Myeloma Market Drivers

27

Multiple Myeloma Market Barriers

28

Appendix

29

DelveInsight Capabilities

30

Disclaimer

31

About DelveInsight

Request a WebEx Walkthrough of the Report: https://www.delveinsight.com/report-store/multiple-myeloma-market

Related Report:

CAR T-Cell Therapy for Multiple Myeloma-Market Insights and Market Forecast-2030 : This report delivers an in-depth understanding of the CAR T-Cell Therapy use for Multiple Myeloma as well as the CAR T-Cell Therapy market trends for Multiple Myeloma. It provides current treatment practices, emerging drugs, CAR T-Cell Therapy market share of the various CAR T-Cell Therapies for Multiple Myeloma, the individual therapies, current and forecasted Multiple Myeloma CAR T-Cell Therapy market Size from 2017 to 2030.

About DelveInsight

DelveInsight is a leading Business Consultant, and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing end to end comprehensive solutions to improve their performance.  Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve.

Contact Us: Shruti Thakur  info@delveinsight.com  +1(919)321-6187  www.delveinsight.com 

SOURCE DelveInsight Business Research, LLP

[pharmaphorumuk]

Scotland backs Merck/Pfizer’s Bavencio combo for kidney cancer

The Scottish Medicines Consortium (SMC) backed NHS use of nine new medicines at its meeting this week, including Merck KGaA and Pfizer’s Bavencio as a first-line treatment for advanced kidney cancer.

The decision means that PD-1 inhibitor Bavencio (avelumab) can be given to previously-untreated adults diagnosed with advanced renal cell carcinoma – the most common form of kidney cancer – as a combination regimen with Pfizer’s targeted cancer drug Inlyta (axitinib).

Kidney cancer is the seventh most common cancer in Scotland, with approximately 1,200 new cases diagnosed every year.

Pfizer said that Bavencio/Inlyta had previously been available to patients in the UK as part of the Early Access to Medicines Scheme (EAMS), and is the first immunotherapy combination therapy for RCC to be cleared for use in both Scotland and England.

NICE cleared first-line use of the dual regimen in first-line RCC patients in July, but opted to make it available with interim funding via the Cancer Drugs Fund (CDF), which kicks in when upcoming trial evidence is expected to confirm whether the drug will be cost-effective.

The SMC decision came after a PACE (Patient and Clinician Engagement) meeting which heard that RCC is a devastating and incurable disease that is generally diagnosed at an advanced stage with a five-year survival rate of approximately 12%.

It is based on the results of the JAVELIN Renal 101 study, which showed that Bavencio/Inlyta extended progression-free survival for an average of five months compared with Pfizer’s older targeted drug Sutent (sunitinib).

Bavencio/Inlyta isn’t however the first immunotherapy-based regimen to be recommended by the SMC for first-line RCC use.

Last month the panel gave a green light in that setting to Merck & Co/MSD’s PD-1 inhibitor Keytruda (pembrolizumab) plus Inlyta, and last year also backed Bristol-Myers Squibb’s PD-1 drug Opdivo (nivolumab) with CTLA4 inhibitor Yervoy (ipilimumab).

The SMC also backed the following drugs for NHS Scotland use:

Bristol Myers Squibb’s Revlimid (lenalidomide) as monotherapy for the maintenance treatment of adult patients with newly diagnosed multiple myeloma who have undergone autologous stem cell transplantation (ASCT). And in combination with rituximab for adults with previously treated follicular lymphoma;

BioMarin’s Brineura (cerliponase alfa) for neuronal ceroid lipofuscinosis type 2 (CLN2), a life limiting, inherited condition in children that leads to progressive brain damage;

Janssen’s Imbruvica (ibrutinib) for the treatment of rare blood cancer Waldenstrom’s macroglobulinemia (WM) in combination with rituximab;

Amgen’s Kyprolis (carfilzomib) for blood cancer multiple myeloma in patients who have received at least one prior therapy;

Novartis’ Mayzent (siponimod) as a treatment for people with active secondary progressive multiple sclerosis (SPMS);

Menarini Pharma’s Vaborem (meropenem/vaborbactam), an antibiotic for the treatment of a number of multidrug-resistant serious infections; and

Dr Falk Pharma’s orodispersible Jorveza (budesonide) for eosinophilic esophagitis (EoE) a chronic, allergic inflammatory disease of the oesophagus.

The post Scotland backs Merck/Pfizer’s Bavencio combo for kidney cancer appeared first on .

from https://pharmaphorum.com/news/scotland-backs-merck-pfizers-bavencio-combo-for-kidney-cancer/

[newsmatters]

Natco Pharma's marketing partner gets USFDA nod for anti-cancer drug

Natco Pharma’s marketing partner gets USFDA nod for anti-cancer drug

on Monday said its marketing partner Breckenridge Pharmaceutical Inc has received approval from the US health regulator for anti-cancer drug Carfilzomib Vials. “Breckenridge Pharmaceutical Inc. (BPI), has received approval for its abbreviated new drug application (ANDA) for Carfilzomib Vials ANDA (generic for Kyprolis), from the US Food and Drug Administration (USFDA),” Natco Pharma said in a…

[floratraci]

Global  Multiple Myeloma Drugs Market

The expanding predominance of multiple myeloma is one of the main considerations that is driving the development of the multiple myeloma drugs market. Multiple Myeloma is the second most regular hematological cancer. It frames in the plasma cells. Plasma cells are a significant piece of the immune system and are in charge of making antibodies that help battle against infections and different diseases. The global burden of multiple myeloma has been progressively consistently throughout the previous 3 decades. In the year 2016, there were around 130,000 instances of multiple myeloma globally, and roughly, 98,000 passings happened around the same time. Indeed, even after the endorsement of viable treatment alternatives, the disease stays incurable in an immense patient pool, bringing about a considerable disease burden. Aside from this, the dispatch of new therapeutic drugs and the active clinical pipeline for multiple myeloma treatment will likewise drive the market during the estimate time frame. For example, in February 2019, Sanofi reported the aftereffects of Phase III clinical trials of isatuximab, its enemy of CD38 therapy for the treatment of multiple myeloma. Receive free sample copy of the report: http://99-report.com/report/433--global--multiple-myeloma-drugs-market The global multiple myeloma drugs market report covers an inside and out examination of the market including statistical and subjective data points, alongside the key market drivers and opportunities and restraints that have positive or negative consequences for the general global market. An inside and out investigation of the territorial and nation level market at the different section and sub-portion levels has been given. The report involves an aggressive investigation of the key players working in the market and covers inside and out data identified with the focused scene of the market and the ongoing systems and item dispatches that will help or influence the market sooner rather than later. In view of the drug class, the multiple myeloma drugs market has been sectioned into chemotherapy drugs, corticosteroids, immunomodulating agents, proteasome inhibitors, monoclonal antibodies, and others.Despite the fact that the manner by which immunomodulating agents influence the immune system isn't clear, these drugs have been utilized for the powerful treatment of multiple myeloma throughout the previous two decades. The key drugs in this section incorporate thalidomide, lenalidomide, and pomalidomide. Monoclonal antibodies are required to develop with a noteworthy CAGR during the estimate time frame. The monoclonal antibodies endorsed for the treatment of multiple myeloma are daratumumab and elotuzumab. The global net offers of DARZALEX (daratumumab) were around US$ 699 million in the primary quarter of 2019. At present, a few clinical trials are being directed for various monoclonal antibodies for the treatment of multiple myeloma.Another class of drugs utilized for the treatment of multiple myeloma are proteasome inhibitors. Proteasome inhibitors act by controlling cell division. They work by halting proteasomes (enzyme complexes) in cells from breaking down proteins that are basic for controlling cell division. Proteasome inhibitors, for example, Bortezomib, Carfilzomib, and Ixazomib have been affirmed for the treatment of multiple myeloma.North America is to represent the biggest market share all through the gauge time frame The high frequency of multiple myeloma in the United States is one of the central point behind the predominance of North America in the multiple myeloma drugs market. The American Cancer Society gauges that roughly 32,110 new instances of multiple myeloma will be diagnosed in the United States in the year 2019, while 12,960 passings are required to happen around the same time. Aside from this, the high selection pace of fresher therapeutic treatments, combined with the good repayment situation, will likewise drive the development of this market. Additionally, the vast majority of the key drug makers are available in this district. In any case, the Asia-Pacific district is required to demonstrate noteworthy development during the estimate time frame, inferable from the rising patient base. Aside from this, the expanding reception pace of novel treatments, combined with the help from government just as non-government associations, is required to drive the development of the multiple myeloma drugs market in this district. Organization Profiles and Competitive Intelligence: The real players working in the multiple myeloma drugs market are Celgene Corporation (US), Janssen Biotech, Inc. (US), Bristol-Myers Squibb Company (US), Millennium Pharmaceuticals (US), Genzyme Corporation (US), Novartis AG (Switzerland), and Amgen, Inc. (US), among others. Celgene Corporation is one of the real players in the multiple myeloma drugs market. The organization's key immunomodulating agents, Pomalyst (Pomalidomide) and Revlimid (Lenalidomide) have demonstrated high treatment proficiency and are required to drive the multiple myeloma drugs market. Revlimid® deals for the year 2018 were US$ 9.68 billion and a 18% year-on-year increment over the earlier year. The organization is further growing its multiple myeloma treatment portfolio by organic and inorganic techniques. In January 2018, the organization obtained Juno Therapeutics, Inc. The organization Juno Therapeutics is a pioneer in the improvement of CAR-T cell treatments for the treatment of various sorts of cancers. Juno's JCAR017 is an incredible fit with Celgene's lymphoma program. It is the top tier CD-19 CAR-T for the treatment of backslid or hard-headed diffuse enormous B-cell lymphoma (DLBCL). The majority of these organizations are focussing on the advancement of combination treatments for the treatment of multiple myeloma. To procure the report/gain in-depth insights, visit: http://99-report.com/sample/433--global--multiple-myeloma-drugs-market

[lovekedars-blog]

Growing Demand for Refractory Multiple Myeloma Treatment to Significantly Increase Revenues Through 2029

Multiple myeloma is a disorder in malignant plasma cell which mostly affects secondary organs such as bone, renal, bone marrow, immune as well as neurologic dysfunction. Multiple myeloma is a malignancy in clonal plasma cell that results from complex interactions between bone marrow stromal cells, malignant progenitor cells and the bone marrow microenvironment. Refractory multiple myeloma can be defined as a disease that is progressive or non-responsive on therapy or within sixty days of last treatment in patients with less response. According to Janseen report 2016, an estimate of 38,000 new multiple myeloma cases were recorded each year in Europe. In addition to that, 28% multiple myeloma patients die within a year of diagnosis.

Request For Report Sample@https://www.persistencemarketresearch.com/samples/29806

Refractory multiple myeloma treatment is expected to register significant growth in the market owing to increasing prevalence of multiple myeloma, advancement in research and development for innovative therapeutics for the refractory multiple myeloma treatment, introduction of monoclonal antibody for refractory multiple myeloma treatment are some of the factors driving the growth of the refractory multiple myeloma treatment market. However, currently there is no standard of care for refractory multiple myeloma treatment. Moreover, cost associated with refractory multiple myeloma treatment and huge expenses in research and development in bringing out novel therapeutics may hinder the growth of the refractory multiple myeloma treatment market.

The global refractory multiple myeloma treatment market is segmented on the basis of treatment type and distributional channel

Refractory Multiple Myeloma Treatment Market Segmentation by Treatment

Refractory Multiple Myeloma Treatment Market Segmentation by Distributional Channel

Immunomodulatory drugs (IMiDs)

Cytotoxic agents

High-Dose Therapy And Autologous Stem Cell Transplantation (HDT-SCT)

Allogeneic Stem Cell Transplantation

Thalidomide (THAL)

Lenalidomide

Pomalidomide

Bortezomib

Carfilzomib

Hospitals

Cancer Institutes

Ambulatory Surgical Centers

Others

Geographically, refractory multiple myeloma treatment market is categorized into five key regions: North America, Latin America, Europe, Asia Pacific, and Middle East & Africa. North America register significant growth in refractory multiple myeloma treatment market due to advanced healthcare facilities, improved medications and launch of new therapeutics will fuel the growth of the refractory multiple myeloma treatment market. Europe also share considerable growth in the refractory multiple myeloma treatment market due to rising demand and adoption of new treatment option in the region will foster growth in the refractory multiple myeloma treatment market. Asia Pacific has considerable revenue generation in the refractory multiple myeloma treatment market owing to increasing prevalence of refractory multiple myeloma treatment and increasing awareness among people for early diagnosis and treatment will propel the growth of the refractory multiple myeloma treatment market. Middle East and Africa has less contribution in driving the growth of the refractory multiple myeloma treatment market owing to poor healthcare facilities, lack of awareness among people for proper treatment, less availability of medications in the region may impact the growth of the refractory multiple myeloma treatment market.

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Examples of the market participants in the global refractory multiple myeloma treatment market identified across the value chain include: Karyopharm Therapeutics, Cellectar Biosciences, Inc., Sanofi, Janssen Pharmaceutical, Takeda Pharmaceuticals, Bluebird Bio, Inc, Novartis, GlaxoSmithKline plc, Amgen Inc., Actinium Pharmaceuticals, MedImmune LLC, Millennium Pharmaceuticals, Inc., Incyte Corporation, Sorrento Therapeutics, Inc., Onyx Therapeutics, Inc., SymBio Pharmaceuticals.

Know More About Report@https://www.persistencemarketresearch.com/market-research/refractory-multiple-myeloma-treatment-market.asp

[azveille]

Protein-slaying drugs could be the next blockbuster therapies

When Craig Crews first managed to make proteins disappear on command with a bizarre new compound, the biochemist says that he considered it a “parlour trick”, a “cute chemical curiosity”.

Today, that cute trick is driving billions of US dollars in investment from pharmaceutical companies such as Roche, Pfizer, Merck, Novartis and GlaxoSmithKline. “I think you can infer that pretty much every company has programmes in this area,” says Raymond Deshaies, senior vice-president of global research at Amgen in Thousand Oaks, California, and one of Crews’s early collaborators.

The drug strategy, called targeted protein degradation, capitalizes on the cell’s natural system for clearing unwanted or damaged proteins. These protein degraders take many forms, but the type that is heading for clinical trials this year is one that Crews, based at Yale University in New Haven, Connecticut, has spent more than 20 years developing: proteolysis-targeting chimaeras, or PROTACs.

Large and unwieldy, PROTACs defy conventional wisdom on what a drug should be. But they also raise the possibility of tackling some of the most indomitable diseases around. Because they destroy rather than inhibit proteins, and can bind to them where other drugs can’t, protein degraders could conceivably be used to go after targets that drug developers have long considered ‘undruggable’: cancer-fuelling villains such as the protein MYC, or the tau protein that tangles up in Alzheimer’s disease.

“This is new territory,” says Alessio Ciulli, a biochemist at the University of Dundee, UK. “We’re breaking the rules of what we thought would be druggable.”

The field has reason to be optimistic. In 2014, scientists discovered that the myeloma treatment lenalidomide (Revlimid), one of the world’s best-selling drugs, works in a similar way to protein degraders to chew up two formerly untouchable proteins1,2.

Yet the field lacks published data confirming that PROTACs and other emerging compounds can make undruggable proteins disappear. And there are questions about where and how these odd-looking molecules will work in the body.

For now, all eyes are on Arvinas, a biotech company in New Haven, Connecticut, founded by Crews. It’s scheduled to begin testing a PROTAC for prostate cancer, albeit attacking a protein that’s been targeted successfully by other drugs. “We’re on the cusp of proving these PROTACs can be drugs,” says Ian Taylor, senior vice-president of biology at Arvinas. “Right behind that will be: can we do this with an undruggable?”

An academic exercise

In diagrams, PROTACs often look like dumb-bells. They are molecules made up of two binding ends connected by a thin tether.

The action happens on the ends. One grabs on to the target protein, while the other latches on to a ubiquitin ligase — part of the cell’s natural rubbish-disposal system that labels defective or damaged proteins by slapping a small protein called ubiquitin onto them (see ‘Marked for destruction’). Ubiquitin tags act as sort of ‘Please collect’ stickers that instruct the cell’s protein shredder, called the proteasome, to do its thing.

Proximity accounts for a lot in biology, so by simply bringing together the ligase and the target protein, a PROTAC ensures that the target will get marked for destruction. Ligases are efficient and ubiquitin, as the name suggests, is plentiful, so a single PROTAC should be able to perform its catch-and-release function repeatedly throughout the cell, suggesting that only a small amount of such a drug is needed for potent activity.

The earliest-known published description of a PROTAC-like molecule is in a patent filed in 1999 by two scientists at Proteinix, a biotechnology company in Gaithersburg, Maryland. In the patent (see go.nature.com/2vyjf9l), John Kenten and Steven Roberts proposed co-opting the cell’s protein-degradation system. Colleagues dismissed the idea, saying that Kenten and Roberts were complicating drug discovery by trying to bind to two proteins — the unwanted protein and the ligase — at once. “There was not a lot of enthusiasm internally for it,” recalls Kenten, now research director at Meso Scale Diagnostics in Rockville, Maryland. Proteinix did not pursue the approach.

But on the other side of the United States, another pair of minds was mulling the same idea. During a research retreat in 1998 at a scenic resort on Semiahmoo Bay in northwest Washington, Deshaies paused in front of a poster by Crews to listen to him talk about using small molecules to link two proteins together. Deshaies, then a biochemist at the California Institute of Technology in Pasadena, was knee-deep in the study of ubiquitin ligases. The human genome encodes roughly 600 of them, which need to form a complex with other proteins to do the tagging. About a year earlier, Deshaies had co-discovered3 a protein family now known to contain 250 ubiquitin ligases.

“It wasn’t that big of a leap to come to the idea of, well, gee, if you could link things to ubiquitin ligases then you could potentially drive the ubiquitination of a protein — and its degradation,” recalls Deshaies. He and Crews continued to chat all weekend and parted ways with a plan to find funding to explore the idea.

At the time, Crews was developing a drug that worked in the opposite way to PROTACs. It blocked the ubiquitin system in cells, causing proteins to build up to dangerous levels and eventually trigger cell death. The result of that work, carfilzomib (Kyprolis), is now used to treat the blood cancer multiple myeloma. “I thought the flip side would be equally as interesting,” says Crews. “That certainly has turned out to be the case.”

Crews and Deshaies soon published a study demonstrating that their first PROTAC, Protac-1, successfully grabbed and led to the degradation of a cancer-associated protein called METAP2 in extracts from Xenopusfrog eggs4.

Still, Protac-1 was far from being a drug, says Crews, who called the paper an “academic exercise”. First-generation PROTACs had low activity in human cells, probably because the compounds struggled to get inside. They relied on big, unwieldly peptides to bind to the ligases. The scientists had to find a way to make the ligase-binding ends more drug-like — “Something that had potential to be a pharmaceutical,” says Crews. Or they needed to move on.

With funding and research support from GlaxoSmithKline in London, Crews pushed ahead, mainly targeting one particular ligase, the von Hippel–Lindau disease tumour suppressor (VHL). In 2012, Crews, together with his graduate student Dennis Buckley and Ciulli, a former visiting fellow in Crews’ lab, reported on a small-molecule binder for VHL5. Crews finally began to believe that PROTACs really could become drugs.

Fishing for small molecules

Crews wasn’t the only one chasing protein degraders. In 2010, while at the Dana-Farber Cancer Institute in Boston, Massachusetts, chemical biologist James Bradner read a paper by a team of researchers in Japan, led by Hiroshi Handa, then at the Tokyo Institute of Technology in Yokohama6. Handa had been trying to understand why the infamous drug thalidomide, approved in some countries in the late 1950s and early 1960s to help with nausea in pregnancy, caused problems with limb development. (It is now approved to treat multiple myeloma and a skin condition.) Using thalidomide as the bait to fish for proteins in cells, Handa discovered that the drug hooks on to and blocks the activity of a ubiquitin ligase called cereblon. That inhibition, his team found, affects limb growth and development in zebrafish and chicks6.

Bradner realized that if thalidomide binds to a ubiquitin ligase — no easy feat, because such enzymes are notoriously difficult to grab — then perhaps he could find a way to bind to the same ligase but target it to proteins implicated in disease. In 2013, Buckley joined Bradner’s team as a postdoctoral researcher, and they began the search for small molecules that bind to cereblon.

In May and June 2015, three teams — led by Bradner, Ciulli and Crews — published five separate papers describing small-molecule PROTACs with potent, drug-like activity7–11. With Ian Churcher at GlaxoSmithKline, Crews bound a PROTAC to VHL and used it to degrade the levels of several proteins to less than 10% of those present in untreated cells7. Bradner and his colleagues bound cereblon to their PROTAC to reduce levels of a cancer-causing protein8, and Ciulli, by then at the University of Dundee, and his team degraded the same protein, using VHL as the ligase9. The protein degraders worked both in cells in a dish and in human tumours in mice.

As well as designing drug-like protein degraders, Crews and Bradner’s teams have both built systems — HaloPROTACs10 and dTAG12, respectively — that enable researchers to put targeted protein degradation to work as a tool in the laboratory, using genetic tags to mark proteins for destruction in cultured cells and in mice. With dTAG, “you can deplete a protein in minutes or hours and monitor what happens”, says Behnam Nabet, a chemical biologist who led development of the system with Nathanael Gray at the Dana-Farber Cancer Institute. “This gives you a lot of power to study oncogenes and kinases and proteins that have very rapid activity.” The dTAG materials are currently freely available: more than 150 academic labs use the probe to investigate the effects of depleting specific proteins in cells, says Nabet.

Bradner, who left Dana-Farber in 2016 to become president of the Novartis Institutes for Biomedical Research, estimates that around 30 separate tools already incorporate the technology. “The path to chemical probes is now well established,” he says. “But the challenge to make real-world medicines from these ligands is significant.”

Gold rush

Following the 2015 flurry of small-molecule PROTACs, Deshaies, who had left the field, penned an opinion piece declaring that PROTACs had the potential to become a major new class of drug, possibly surpassing two of the hottest drug-development areas of all time — protein kinase inhibitors and monoclonal antibodies13. “The gold rush is on!” Deshaies wrote at the time.

Since then, he says, it has only intensified. He joined Amgen in 2017 and now oversees the company’s work in the area.

The Arvinas trial, expected to begin by mid-2019, will include 28–36 men with metastatic prostate cancer and will last around 9 months, says Taylor. It is usual for any new class of drug to go after a well-known target, where the biology and toxicology are well-understood, and Arvinas’s first candidate is no exception. It degrades the androgen receptor, a protein that is already targeted by a handful of approved drugs. The company hopes that by degrading rather than inhibiting the receptor, its PROTAC will be able to treat people who have become resistant to or see no benefit from existing drugs. And if the candidate succeeds, the field will finally have the clinical data that everyone is looking for. Arvinas will have shown that a PROTAC can be a drug.

That’s crucial because there has been considerable doubt about whether protein degraders can work in humans. Fully assembled PROTACs break well-known rules of thumb for drugs. Chief among them is size. A good small-molecule drug typically has a mass of less than 500 daltons. Current PROTACs range upwards of 1,000 daltons. Yet the molecules can still enter cells7,10,11. Crews suspects that this is because they are probably recognized by the cell membrane as two smaller molecules that happen to be tethered together, rather than a single large one.

“We’re throwing out preconceived notions we’ve had about larger-than-average small molecules,” says Taylor.

Also out of the window are preconceived ideas about undruggables. The problem with many of these tough protein targets is that most small-molecule drugs or monoclonal antibodies need to bind to an active site on an enzyme or a receptor to work. But an estimated 80% of proteins in human cells lack such a site. PROTACs, however, can grab a protein by any nook, cranny or crevice — they don’t need to be sitting in an active pocket to work. So they could make those proteins accessible.

There’s already some evidence to support this approach. Last year, a team at the Institute of Cancer Research in London produced a small molecule that can bind to a transcription-factor regulator that doesn’t have an active site14. They were able to create a potent PROTAC by attaching a binder for the ubiquitin ligase cereblon.

The field still lacks published evidence of a PROTAC that can target and degrade a valuable undruggable protein. Deshaies says that Amgen has a PROTAC effective in both cultured cells and animals against an unnamed high-value cancer target that has been historically tough to bind. Arvinas claims to have in vivo evidence of PROTACs degrading tau in the brains of mice. On its website, the company says that injecting its tau-protein degrader directly into the mouse hippocampus reduced levels of tau by 50%.

By developing PROTACs for an array of diseases, including those that affect the brain, Taylor says that many researchers hope to show that the technology is “therapeutic-area agnostic”. Various teams are also working to expand the pool of ligases that protein degraders can recruit. There are only four main ones used at present, including VHL and cereblon, and a wider variety of available ligases could enable drug developers to match the most potent ligase–PROTAC combination with their cell type or protein of interest. “Potentially, any ligase can be hijacked through this approach,” says Ciulli, who is collaborating with German pharmaceutical company Boehringer Ingelheim on the development of PROTACs.

Buoyed by fresh targets, improved potency, and a clinical trial about to begin, researchers are ready to prove that protein degraders can be more than a parlour trick. “The sky is the limit,” says Ciulli. “It is just a question of when.