Micrograph showing DLBCL

The US Food and Drug Administration (FDA) has granted orphan drug designation for eFT508 to treat diffuse large B-cell lymphoma (DLBCL).

eFT508 is a highly selective inhibitor of MNK1 and MNK2, enzymes that integrate signals from several oncogenic and immune signaling pathways.

The FDA grants orphan designation to drugs or biologics intended to treat a disease or condition affecting fewer than 200,000 patients in the US.

The orphan designation for eFT508 provides several incentives for eFFECTOR Therapeutics, the company developing eFT508.

These incentives include increased access to FDA reviewers to discuss clinical trial designs, the ability to qualify for tax credits for certain clinical research costs, the ability to apply for annual grant funding, a waiver of Prescription Drug User Fee Act filing fees, and the potential for 7 years of US marketing exclusivity if eFT508 is approved.

eFFECTOR has dosed the first subject in a phase 1/2 trial of eFT508 in patients with B-cell hematologic malignancies. The study is designed to evaluate the safety, pharmacokinetics, pharmacodynamics, and antitumor activity of eFT508.

eFFECTOR presented preclinical research of eFT508 in DLBCL at the 2015 ASH Annual Meeting. The poster is available for download from the eFFECTOR website.

The researchers reported that eFT508 demonstrated anti-proliferative activity against multiple DLBCL cell lines, including the TMD8, OCI-Ly3, and HBL1 cell lines.

eFT508 also exhibited “significant anti-tumor activity” in mouse models of TMD8 and HBL-1 ABC-DLBCL.

Finally, the researchers found that eFT508 synergized with everolimus, ibrutinib, and venetoclax both in vitro and in vivo.

Micrograph showing DLBCL

The US Food and Drug Administration (FDA) has granted orphan drug designation for eFT508 to treat diffuse large B-cell lymphoma (DLBCL).

eFT508 is a highly selective inhibitor of MNK1 and MNK2, enzymes that integrate signals from several oncogenic and immune signaling pathways.

The FDA grants orphan designation to drugs or biologics intended to treat a disease or condition affecting fewer than 200,000 patients in the US.

The orphan designation for eFT508 provides several incentives for eFFECTOR Therapeutics, the company developing eFT508.

These incentives include increased access to FDA reviewers to discuss clinical trial designs, the ability to qualify for tax credits for certain clinical research costs, the ability to apply for annual grant funding, a waiver of Prescription Drug User Fee Act filing fees, and the potential for 7 years of US marketing exclusivity if eFT508 is approved.

eFFECTOR has dosed the first subject in a phase 1/2 trial of eFT508 in patients with B-cell hematologic malignancies. The study is designed to evaluate the safety, pharmacokinetics, pharmacodynamics, and antitumor activity of eFT508.

eFFECTOR presented preclinical research of eFT508 in DLBCL at the 2015 ASH Annual Meeting. The poster is available for download from the eFFECTOR website.

The researchers reported that eFT508 demonstrated anti-proliferative activity against multiple DLBCL cell lines, including the TMD8, OCI-Ly3, and HBL1 cell lines.

eFT508 also exhibited “significant anti-tumor activity” in mouse models of TMD8 and HBL-1 ABC-DLBCL.

Finally, the researchers found that eFT508 synergized with everolimus, ibrutinib, and venetoclax both in vitro and in vivo.

Micrograph showing DLBCL

The US Food and Drug Administration (FDA) has granted orphan drug designation for eFT508 to treat diffuse large B-cell lymphoma (DLBCL).

eFT508 is a highly selective inhibitor of MNK1 and MNK2, enzymes that integrate signals from several oncogenic and immune signaling pathways.

The FDA grants orphan designation to drugs or biologics intended to treat a disease or condition affecting fewer than 200,000 patients in the US.

The orphan designation for eFT508 provides several incentives for eFFECTOR Therapeutics, the company developing eFT508.

These incentives include increased access to FDA reviewers to discuss clinical trial designs, the ability to qualify for tax credits for certain clinical research costs, the ability to apply for annual grant funding, a waiver of Prescription Drug User Fee Act filing fees, and the potential for 7 years of US marketing exclusivity if eFT508 is approved.

eFFECTOR has dosed the first subject in a phase 1/2 trial of eFT508 in patients with B-cell hematologic malignancies. The study is designed to evaluate the safety, pharmacokinetics, pharmacodynamics, and antitumor activity of eFT508.

eFFECTOR presented preclinical research of eFT508 in DLBCL at the 2015 ASH Annual Meeting. The poster is available for download from the eFFECTOR website.

The researchers reported that eFT508 demonstrated anti-proliferative activity against multiple DLBCL cell lines, including the TMD8, OCI-Ly3, and HBL1 cell lines.

eFT508 also exhibited “significant anti-tumor activity” in mouse models of TMD8 and HBL-1 ABC-DLBCL.

Finally, the researchers found that eFT508 synergized with everolimus, ibrutinib, and venetoclax both in vitro and in vivo.

1. A 63-year-old man complains of a mildly painful and tender rash on his penis that has been there for almost two years. The patient is uncircumcised; when the foreskin is retracted, a bright red, erythematous, nonscaly, circumferential plaque is visible on the glans penis, spreading to the foreskin. He denies pain on urination, discharge, fever, malaise, arthralgias, and sexual contact outside of his marriage.

Diagnosis: Zoon balanitis is a benign condition that typically affects uncircumcised middle-aged to elderly men.^1,2 Worldwide prevalence among uncircumcised men is approximately 3%.² The etiology is unknown; it’s thought that this condition may be caused by friction, trauma, heat, lack of hygiene, exogenous or infectious agents, an IgE hypersensitivity, or a chronic infection with Mycobacterium smegmatis.^1,2

Typically, the appearance of the lesion precedes diagnosis by about one to 2 years.¹ The patient usually complains of mild pruritus and tenderness. Undergarments may be bloodstained.

The lesion associated with Zoon balanitis is a solitary, glistening, shiny, red-to-orange plaque of the glans penis or prepuce of an uncircumcised male. Pinpoint erythematous spots or “cayenne pepper spots” may also be associated with this condition.

For more information on this case, see “Erythematous penile lesion.” J Fam Pract. 2012;61(12):753-755.

2. A 21-year-old man presents with bumps on his penis. He admits to having unprotected sexual intercourse with more than six women in the past year. He is otherwise healthy.

Diagnosis: Genital warts are caused by human papillomavirus infection. The incubation period after exposure ranges from three weeks to eight months. Anogenital warts represent the most common viral sexually transmitted infection in the United States; there are approximately 1 million new cases of genital warts per year. Most infections are transient and clear up within two years, but some infections persist and recur.

The diagnosis is usually clinical; genital warts are typically asymptomatic and present as flesh-colored, exophytic lesions on the genitalia, including the penis, vulva, vagina, scrotum, perineum, and perianal skin. External warts can appear as small bumps, or they may be flat, verrucous, or pedunculated. Treatment options include cryotherapy and prescription imiquimod cream.

For more information on this case, see “Genital bumps.”

3. A 27-year-old man is worried that he may have contracted chlamydia. About five days ago, he experienced pain and a burning feeling with urination. Three days ago, a painful, growing lump near the head of his penis developed. The patient has a purulent urethral discharge and fluctuant, yellowish white, tender swelling on the left side of the frenulum. There are no ulcers, but there is a single 2-cm lymph node in the right inguinal area that is of normal consistency, mobile, nontender, and nonfluctuant.

Diagnosis: The clinical history was consistent with a diagnosis of gonococcal urethritis complicated by a periurethral gland abscess. The location of the swelling was most consistent with an abscess in the Tyson’s gland (also known as tysonitis). The Tyson’s (or preputial) glands of the penis are sebaceous-type glands on either side of the frenulum at the balanopreputial sulcus.¹ In women, an abscess of the periurethral Skene’s gland is an analogous gonorrheal complication.

The differential diagnosis of acute swelling on the penile shaft includes syphilis, chancroid, lymphogranuloma venereum, herpes simplex virus, Behçet’s syndrome, a drug reaction, erythema multiforme, Crohn’s disease, lichen planus, amebiasis, scabies, trauma, and cancer.

For more information on this case, see “Returning traveler with painful penile mass.” J Fam Pract. 2011 May;60(5):285-287.

4. A 32-year-old man presents with a week-long history of painful vesicles on the shaft of his penis associated with tender groin adenopathy. Two days ago, the vesicles broke and the pain worsened. The patient had similar lesions a year ago but did not seek medical care. He has had three different female sexual partners over the past two years but has no knowledge of them having any sores or diseases.

Diagnosis: Genital herpes presents with multiple transient, painful vesicles that appear on the penis, vulva, buttocks, perineum, vagina, or cervix. The vesicles break down and become ulcers that develop crusts while healing. Recurrences typically occur 2 to 3 times a year. The duration is shorter and less painful than in primary infections. The lesions often heal completely by 8 to 10 days.

Antiviral therapy is recommended for an initial genital herpes outbreak. Although systemic antiviral drugs can partially control the signs and symptoms of herpes episodes, they do not eradicate the latent virus.

For more information on this case, see “Painful vesicles on penis.”

1. A 63-year-old man complains of a mildly painful and tender rash on his penis that has been there for almost two years. The patient is uncircumcised; when the foreskin is retracted, a bright red, erythematous, nonscaly, circumferential plaque is visible on the glans penis, spreading to the foreskin. He denies pain on urination, discharge, fever, malaise, arthralgias, and sexual contact outside of his marriage.

Diagnosis: Zoon balanitis is a benign condition that typically affects uncircumcised middle-aged to elderly men.^1,2 Worldwide prevalence among uncircumcised men is approximately 3%.² The etiology is unknown; it’s thought that this condition may be caused by friction, trauma, heat, lack of hygiene, exogenous or infectious agents, an IgE hypersensitivity, or a chronic infection with Mycobacterium smegmatis.^1,2

Typically, the appearance of the lesion precedes diagnosis by about one to 2 years.¹ The patient usually complains of mild pruritus and tenderness. Undergarments may be bloodstained.

The lesion associated with Zoon balanitis is a solitary, glistening, shiny, red-to-orange plaque of the glans penis or prepuce of an uncircumcised male. Pinpoint erythematous spots or “cayenne pepper spots” may also be associated with this condition.

For more information on this case, see “Erythematous penile lesion.” J Fam Pract. 2012;61(12):753-755.

2. A 21-year-old man presents with bumps on his penis. He admits to having unprotected sexual intercourse with more than six women in the past year. He is otherwise healthy.

Diagnosis: Genital warts are caused by human papillomavirus infection. The incubation period after exposure ranges from three weeks to eight months. Anogenital warts represent the most common viral sexually transmitted infection in the United States; there are approximately 1 million new cases of genital warts per year. Most infections are transient and clear up within two years, but some infections persist and recur.

The diagnosis is usually clinical; genital warts are typically asymptomatic and present as flesh-colored, exophytic lesions on the genitalia, including the penis, vulva, vagina, scrotum, perineum, and perianal skin. External warts can appear as small bumps, or they may be flat, verrucous, or pedunculated. Treatment options include cryotherapy and prescription imiquimod cream.

For more information on this case, see “Genital bumps.”

3. A 27-year-old man is worried that he may have contracted chlamydia. About five days ago, he experienced pain and a burning feeling with urination. Three days ago, a painful, growing lump near the head of his penis developed. The patient has a purulent urethral discharge and fluctuant, yellowish white, tender swelling on the left side of the frenulum. There are no ulcers, but there is a single 2-cm lymph node in the right inguinal area that is of normal consistency, mobile, nontender, and nonfluctuant.

Diagnosis: The clinical history was consistent with a diagnosis of gonococcal urethritis complicated by a periurethral gland abscess. The location of the swelling was most consistent with an abscess in the Tyson’s gland (also known as tysonitis). The Tyson’s (or preputial) glands of the penis are sebaceous-type glands on either side of the frenulum at the balanopreputial sulcus.¹ In women, an abscess of the periurethral Skene’s gland is an analogous gonorrheal complication.

The differential diagnosis of acute swelling on the penile shaft includes syphilis, chancroid, lymphogranuloma venereum, herpes simplex virus, Behçet’s syndrome, a drug reaction, erythema multiforme, Crohn’s disease, lichen planus, amebiasis, scabies, trauma, and cancer.

For more information on this case, see “Returning traveler with painful penile mass.” J Fam Pract. 2011 May;60(5):285-287.

4. A 32-year-old man presents with a week-long history of painful vesicles on the shaft of his penis associated with tender groin adenopathy. Two days ago, the vesicles broke and the pain worsened. The patient had similar lesions a year ago but did not seek medical care. He has had three different female sexual partners over the past two years but has no knowledge of them having any sores or diseases.

Diagnosis: Genital herpes presents with multiple transient, painful vesicles that appear on the penis, vulva, buttocks, perineum, vagina, or cervix. The vesicles break down and become ulcers that develop crusts while healing. Recurrences typically occur 2 to 3 times a year. The duration is shorter and less painful than in primary infections. The lesions often heal completely by 8 to 10 days.

Antiviral therapy is recommended for an initial genital herpes outbreak. Although systemic antiviral drugs can partially control the signs and symptoms of herpes episodes, they do not eradicate the latent virus.

For more information on this case, see “Painful vesicles on penis.”

1. A 63-year-old man complains of a mildly painful and tender rash on his penis that has been there for almost two years. The patient is uncircumcised; when the foreskin is retracted, a bright red, erythematous, nonscaly, circumferential plaque is visible on the glans penis, spreading to the foreskin. He denies pain on urination, discharge, fever, malaise, arthralgias, and sexual contact outside of his marriage.

Diagnosis: Zoon balanitis is a benign condition that typically affects uncircumcised middle-aged to elderly men.^1,2 Worldwide prevalence among uncircumcised men is approximately 3%.² The etiology is unknown; it’s thought that this condition may be caused by friction, trauma, heat, lack of hygiene, exogenous or infectious agents, an IgE hypersensitivity, or a chronic infection with Mycobacterium smegmatis.^1,2

Typically, the appearance of the lesion precedes diagnosis by about one to 2 years.¹ The patient usually complains of mild pruritus and tenderness. Undergarments may be bloodstained.

The lesion associated with Zoon balanitis is a solitary, glistening, shiny, red-to-orange plaque of the glans penis or prepuce of an uncircumcised male. Pinpoint erythematous spots or “cayenne pepper spots” may also be associated with this condition.

For more information on this case, see “Erythematous penile lesion.” J Fam Pract. 2012;61(12):753-755.

2. A 21-year-old man presents with bumps on his penis. He admits to having unprotected sexual intercourse with more than six women in the past year. He is otherwise healthy.

Diagnosis: Genital warts are caused by human papillomavirus infection. The incubation period after exposure ranges from three weeks to eight months. Anogenital warts represent the most common viral sexually transmitted infection in the United States; there are approximately 1 million new cases of genital warts per year. Most infections are transient and clear up within two years, but some infections persist and recur.

The diagnosis is usually clinical; genital warts are typically asymptomatic and present as flesh-colored, exophytic lesions on the genitalia, including the penis, vulva, vagina, scrotum, perineum, and perianal skin. External warts can appear as small bumps, or they may be flat, verrucous, or pedunculated. Treatment options include cryotherapy and prescription imiquimod cream.

For more information on this case, see “Genital bumps.”

3. A 27-year-old man is worried that he may have contracted chlamydia. About five days ago, he experienced pain and a burning feeling with urination. Three days ago, a painful, growing lump near the head of his penis developed. The patient has a purulent urethral discharge and fluctuant, yellowish white, tender swelling on the left side of the frenulum. There are no ulcers, but there is a single 2-cm lymph node in the right inguinal area that is of normal consistency, mobile, nontender, and nonfluctuant.

Diagnosis: The clinical history was consistent with a diagnosis of gonococcal urethritis complicated by a periurethral gland abscess. The location of the swelling was most consistent with an abscess in the Tyson’s gland (also known as tysonitis). The Tyson’s (or preputial) glands of the penis are sebaceous-type glands on either side of the frenulum at the balanopreputial sulcus.¹ In women, an abscess of the periurethral Skene’s gland is an analogous gonorrheal complication.

The differential diagnosis of acute swelling on the penile shaft includes syphilis, chancroid, lymphogranuloma venereum, herpes simplex virus, Behçet’s syndrome, a drug reaction, erythema multiforme, Crohn’s disease, lichen planus, amebiasis, scabies, trauma, and cancer.

For more information on this case, see “Returning traveler with painful penile mass.” J Fam Pract. 2011 May;60(5):285-287.

4. A 32-year-old man presents with a week-long history of painful vesicles on the shaft of his penis associated with tender groin adenopathy. Two days ago, the vesicles broke and the pain worsened. The patient had similar lesions a year ago but did not seek medical care. He has had three different female sexual partners over the past two years but has no knowledge of them having any sores or diseases.

Diagnosis: Genital herpes presents with multiple transient, painful vesicles that appear on the penis, vulva, buttocks, perineum, vagina, or cervix. The vesicles break down and become ulcers that develop crusts while healing. Recurrences typically occur 2 to 3 times a year. The duration is shorter and less painful than in primary infections. The lesions often heal completely by 8 to 10 days.

Antiviral therapy is recommended for an initial genital herpes outbreak. Although systemic antiviral drugs can partially control the signs and symptoms of herpes episodes, they do not eradicate the latent virus.

For more information on this case, see “Painful vesicles on penis.”

MIAMI BEACH – The Food and Drug Administration has accepted pathological complete response rate (pCR) as a surrogate endpoint for disease-free and overall survival in clinical trials for neoadjuvant therapy of breast cancer.

Yet the specimen collection and histopathologic methods used to measure pCR have differed considerably across major neoadjuvant trials for breast cancer, said Michael F. Press, MD, PhD, of the USC/Norris Comprehensive Cancer Center at the University of California, Los Angeles.

In a video interview conducted at the annual Miami Breast Cancer Conference, held by Physicians’ Education Resource, Dr. Press outlined the problems associated with a lack of standardization of outcomes measures, and described how residual cancer burden may be a more effective, validated measures for comparing outcomes across clinical trials.

MIAMI BEACH – The Food and Drug Administration has accepted pathological complete response rate (pCR) as a surrogate endpoint for disease-free and overall survival in clinical trials for neoadjuvant therapy of breast cancer.

Yet the specimen collection and histopathologic methods used to measure pCR have differed considerably across major neoadjuvant trials for breast cancer, said Michael F. Press, MD, PhD, of the USC/Norris Comprehensive Cancer Center at the University of California, Los Angeles.

In a video interview conducted at the annual Miami Breast Cancer Conference, held by Physicians’ Education Resource, Dr. Press outlined the problems associated with a lack of standardization of outcomes measures, and described how residual cancer burden may be a more effective, validated measures for comparing outcomes across clinical trials.

MIAMI BEACH – The Food and Drug Administration has accepted pathological complete response rate (pCR) as a surrogate endpoint for disease-free and overall survival in clinical trials for neoadjuvant therapy of breast cancer.

Yet the specimen collection and histopathologic methods used to measure pCR have differed considerably across major neoadjuvant trials for breast cancer, said Michael F. Press, MD, PhD, of the USC/Norris Comprehensive Cancer Center at the University of California, Los Angeles.

In a video interview conducted at the annual Miami Breast Cancer Conference, held by Physicians’ Education Resource, Dr. Press outlined the problems associated with a lack of standardization of outcomes measures, and described how residual cancer burden may be a more effective, validated measures for comparing outcomes across clinical trials.

ATLANTA – Obese females who live in urban settings are significantly more likely to develop atopic diseases, compared with their male counterparts, results from a single-center study showed.

“Some research has shown that obese girls are much more likely to be atopic, but many of them only look at one disease alone, such as atopic dermatitis or food allergies,” lead study author Sairaman Nagarajan, MD, MPH, said in an interview at the annual meeting of the American Academy of Allergy, Asthma, and Immunology. “Most of the studies are in asthma.”

Doug Brunk/Frontline Medical News

The mean age of patients was 9 years, 23% were obese, and 55% were male. The researchers observed no differences in laboratory biomarkers nor in the prevalence of individual/or cumulative atopic disease in obese children, compared with controls. When stratified by gender, obese females had a significantly higher mean atopic disease score, compared with controls (4.00 vs. 2.62, respectively; P less than .001), while males had a significantly lower mean atopic disease score, compared with controls (3 vs. 3.42; P less than .001). Regression models yielded similar results; obese females had a significantly higher mean atopic disease score, compared with controls (by a mean elevation of 1.37 points; P less than .005), while males had a significantly lower mean atopic disease score (by a mean decline of -0.42 points; P less than .006). “From this we can say that female gender is a positive risk factor for atopy, and urban obese females may be particularly likely to benefit from lifestyle modification therapy like exercise and diet in controlling weight, and thereby allergies,” Dr. Nagarajan said.

He noted that it remains unclear whether the findings would apply to children who live in nonurban settings. “It’s difficult to say because there are some variables which are unique to urban minority populations like the housing that they live in and the kind of stores they buy food from,” he said.

Dr. Nagarajan reported having no financial disclosures.

[email protected]

ATLANTA – Obese females who live in urban settings are significantly more likely to develop atopic diseases, compared with their male counterparts, results from a single-center study showed.

“Some research has shown that obese girls are much more likely to be atopic, but many of them only look at one disease alone, such as atopic dermatitis or food allergies,” lead study author Sairaman Nagarajan, MD, MPH, said in an interview at the annual meeting of the American Academy of Allergy, Asthma, and Immunology. “Most of the studies are in asthma.”

Doug Brunk/Frontline Medical News

The mean age of patients was 9 years, 23% were obese, and 55% were male. The researchers observed no differences in laboratory biomarkers nor in the prevalence of individual/or cumulative atopic disease in obese children, compared with controls. When stratified by gender, obese females had a significantly higher mean atopic disease score, compared with controls (4.00 vs. 2.62, respectively; P less than .001), while males had a significantly lower mean atopic disease score, compared with controls (3 vs. 3.42; P less than .001). Regression models yielded similar results; obese females had a significantly higher mean atopic disease score, compared with controls (by a mean elevation of 1.37 points; P less than .005), while males had a significantly lower mean atopic disease score (by a mean decline of -0.42 points; P less than .006). “From this we can say that female gender is a positive risk factor for atopy, and urban obese females may be particularly likely to benefit from lifestyle modification therapy like exercise and diet in controlling weight, and thereby allergies,” Dr. Nagarajan said.

He noted that it remains unclear whether the findings would apply to children who live in nonurban settings. “It’s difficult to say because there are some variables which are unique to urban minority populations like the housing that they live in and the kind of stores they buy food from,” he said.

Dr. Nagarajan reported having no financial disclosures.

[email protected]

ATLANTA – Obese females who live in urban settings are significantly more likely to develop atopic diseases, compared with their male counterparts, results from a single-center study showed.

“Some research has shown that obese girls are much more likely to be atopic, but many of them only look at one disease alone, such as atopic dermatitis or food allergies,” lead study author Sairaman Nagarajan, MD, MPH, said in an interview at the annual meeting of the American Academy of Allergy, Asthma, and Immunology. “Most of the studies are in asthma.”

Doug Brunk/Frontline Medical News

The mean age of patients was 9 years, 23% were obese, and 55% were male. The researchers observed no differences in laboratory biomarkers nor in the prevalence of individual/or cumulative atopic disease in obese children, compared with controls. When stratified by gender, obese females had a significantly higher mean atopic disease score, compared with controls (4.00 vs. 2.62, respectively; P less than .001), while males had a significantly lower mean atopic disease score, compared with controls (3 vs. 3.42; P less than .001). Regression models yielded similar results; obese females had a significantly higher mean atopic disease score, compared with controls (by a mean elevation of 1.37 points; P less than .005), while males had a significantly lower mean atopic disease score (by a mean decline of -0.42 points; P less than .006). “From this we can say that female gender is a positive risk factor for atopy, and urban obese females may be particularly likely to benefit from lifestyle modification therapy like exercise and diet in controlling weight, and thereby allergies,” Dr. Nagarajan said.

He noted that it remains unclear whether the findings would apply to children who live in nonurban settings. “It’s difficult to say because there are some variables which are unique to urban minority populations like the housing that they live in and the kind of stores they buy food from,” he said.

Dr. Nagarajan reported having no financial disclosures.

[email protected]

ATLANTA – Within a cohort of children diagnosed with peanut allergy who were followed since 2001, peanut IgE levels increased significantly over time in all races, according to a preliminary analysis of data.

In an interview in advance of the annual meeting of the American Academy of Allergy, Asthma, and Immunology, lead study author Yasmin Hamzavi, MD, said that recent publications have implicated race as a factor in food sensitization, but how this may impact the management of food allergy has not been elucidated. “The novel aspect of this study is that we are not just looking at the baseline peanut IgE levels between the races, but at the rate of change in peanut IgE over time between the different races,” said Dr. Hamzavi, a fellow in the division of allergy and immunology at Northwell Health, Great Neck, N.Y. “My question was, does the level of peanut IgE decrease or increase faster in one race versus another?”

Doug Brunk/Frontline Medical News

A significant increase in peanut IgE over time was observed among all races (P less than .0002). In addition, white and Asian children showed an increasing trend in peanut IgE, while black children demonstrated a decreasing trend over time (P less than .099), a finding that Dr. Hamzavi described as “surprising and unusual.” She called for larger studies exploring factors for the noted increase among all races, such as changes in testing methods, food avoidance, and increasing sensitization. “Understanding the changes in peanut sensitization over time is a crucial step in determining the likelihood of clinical reactivity,” she said.

Dr. Hamzavi is reviewing data for a similar analysis of children with milk and egg allergy. She reported having no financial disclosures.

[email protected]

ATLANTA – Within a cohort of children diagnosed with peanut allergy who were followed since 2001, peanut IgE levels increased significantly over time in all races, according to a preliminary analysis of data.

In an interview in advance of the annual meeting of the American Academy of Allergy, Asthma, and Immunology, lead study author Yasmin Hamzavi, MD, said that recent publications have implicated race as a factor in food sensitization, but how this may impact the management of food allergy has not been elucidated. “The novel aspect of this study is that we are not just looking at the baseline peanut IgE levels between the races, but at the rate of change in peanut IgE over time between the different races,” said Dr. Hamzavi, a fellow in the division of allergy and immunology at Northwell Health, Great Neck, N.Y. “My question was, does the level of peanut IgE decrease or increase faster in one race versus another?”

Doug Brunk/Frontline Medical News

A significant increase in peanut IgE over time was observed among all races (P less than .0002). In addition, white and Asian children showed an increasing trend in peanut IgE, while black children demonstrated a decreasing trend over time (P less than .099), a finding that Dr. Hamzavi described as “surprising and unusual.” She called for larger studies exploring factors for the noted increase among all races, such as changes in testing methods, food avoidance, and increasing sensitization. “Understanding the changes in peanut sensitization over time is a crucial step in determining the likelihood of clinical reactivity,” she said.

Dr. Hamzavi is reviewing data for a similar analysis of children with milk and egg allergy. She reported having no financial disclosures.

[email protected]

ATLANTA – Within a cohort of children diagnosed with peanut allergy who were followed since 2001, peanut IgE levels increased significantly over time in all races, according to a preliminary analysis of data.

In an interview in advance of the annual meeting of the American Academy of Allergy, Asthma, and Immunology, lead study author Yasmin Hamzavi, MD, said that recent publications have implicated race as a factor in food sensitization, but how this may impact the management of food allergy has not been elucidated. “The novel aspect of this study is that we are not just looking at the baseline peanut IgE levels between the races, but at the rate of change in peanut IgE over time between the different races,” said Dr. Hamzavi, a fellow in the division of allergy and immunology at Northwell Health, Great Neck, N.Y. “My question was, does the level of peanut IgE decrease or increase faster in one race versus another?”

Doug Brunk/Frontline Medical News

A significant increase in peanut IgE over time was observed among all races (P less than .0002). In addition, white and Asian children showed an increasing trend in peanut IgE, while black children demonstrated a decreasing trend over time (P less than .099), a finding that Dr. Hamzavi described as “surprising and unusual.” She called for larger studies exploring factors for the noted increase among all races, such as changes in testing methods, food avoidance, and increasing sensitization. “Understanding the changes in peanut sensitization over time is a crucial step in determining the likelihood of clinical reactivity,” she said.

Dr. Hamzavi is reviewing data for a similar analysis of children with milk and egg allergy. She reported having no financial disclosures.

[email protected]

MIAMI BEACH – Growing evidence continues to point to a widening separation between female and male breast cancers, particularly with discoveries suggesting different pathways to disease and important genetic distinctions.

Therefore, the traditional practice of extrapolating findings from female breast cancer research to men with breast cancer no longer makes sense, Patrick I. Borgen, MD, said at the annual Miami Breast Cancer Conference, held by Physicians’ Education Resource.

The incidence of male breast cancer is increasing. At the same time, fewer men with breast cancer get referred for and undergo genetic testing for their disease, said Dr. Borgen, chair of the department of surgery at Maimonides Medical Center in Brooklyn, N.Y.

Dr. Borgen explained in a video interview that both maternal and paternal inheritance of breast cancer are important, and they tie the lineage into a hypothesis for why BRCA mutations – which can predispose people to worse survival – have persisted through generations.

MIAMI BEACH – Growing evidence continues to point to a widening separation between female and male breast cancers, particularly with discoveries suggesting different pathways to disease and important genetic distinctions.

Therefore, the traditional practice of extrapolating findings from female breast cancer research to men with breast cancer no longer makes sense, Patrick I. Borgen, MD, said at the annual Miami Breast Cancer Conference, held by Physicians’ Education Resource.

The incidence of male breast cancer is increasing. At the same time, fewer men with breast cancer get referred for and undergo genetic testing for their disease, said Dr. Borgen, chair of the department of surgery at Maimonides Medical Center in Brooklyn, N.Y.

Dr. Borgen explained in a video interview that both maternal and paternal inheritance of breast cancer are important, and they tie the lineage into a hypothesis for why BRCA mutations – which can predispose people to worse survival – have persisted through generations.

MIAMI BEACH – Growing evidence continues to point to a widening separation between female and male breast cancers, particularly with discoveries suggesting different pathways to disease and important genetic distinctions.

Therefore, the traditional practice of extrapolating findings from female breast cancer research to men with breast cancer no longer makes sense, Patrick I. Borgen, MD, said at the annual Miami Breast Cancer Conference, held by Physicians’ Education Resource.

The incidence of male breast cancer is increasing. At the same time, fewer men with breast cancer get referred for and undergo genetic testing for their disease, said Dr. Borgen, chair of the department of surgery at Maimonides Medical Center in Brooklyn, N.Y.

Dr. Borgen explained in a video interview that both maternal and paternal inheritance of breast cancer are important, and they tie the lineage into a hypothesis for why BRCA mutations – which can predispose people to worse survival – have persisted through generations.

MIAMI BEACH – The remarkable progress seen with immune checkpoint inhibitors in metastatic melanoma, non–small-cell lung cancer, and other tumors has yet to be replicated in breast cancer, but it’s early days yet, and breast cancer researchers need more time before the ultimate clinical benefits of immunotherapy in breast cancer can be ascertained, said Adam M. Brufsky, MD, PhD, of the University of Pittsburgh.

Early studies with inhibitors of programmed death-1 (PD-1) and its ligand PD-L1 in patients with advanced triple-negative breast cancer have yielded only minimal response rates to date, but it it’s far too early to give up on the concept, Dr. Brufsky cautioned at the annual Miami Breast Cancer Conference, held by Physicians’ Education Resource.

In a video interview, he discussed the challenges of treating breast cancers, which may be less immunogenic and have a lower tumor mutational burden than other malignancies that respond more readily to PD-1 inhibition. Several large, phase III clinical trials of checkpoint inhibitors combined with cytotoxic chemotherapy are underway, he said, and those eventual findings may shed light on the optimal approach to using immunotherapy to treat patients with refractory metastatic breast cancers.

MIAMI BEACH – The remarkable progress seen with immune checkpoint inhibitors in metastatic melanoma, non–small-cell lung cancer, and other tumors has yet to be replicated in breast cancer, but it’s early days yet, and breast cancer researchers need more time before the ultimate clinical benefits of immunotherapy in breast cancer can be ascertained, said Adam M. Brufsky, MD, PhD, of the University of Pittsburgh.

Early studies with inhibitors of programmed death-1 (PD-1) and its ligand PD-L1 in patients with advanced triple-negative breast cancer have yielded only minimal response rates to date, but it it’s far too early to give up on the concept, Dr. Brufsky cautioned at the annual Miami Breast Cancer Conference, held by Physicians’ Education Resource.

In a video interview, he discussed the challenges of treating breast cancers, which may be less immunogenic and have a lower tumor mutational burden than other malignancies that respond more readily to PD-1 inhibition. Several large, phase III clinical trials of checkpoint inhibitors combined with cytotoxic chemotherapy are underway, he said, and those eventual findings may shed light on the optimal approach to using immunotherapy to treat patients with refractory metastatic breast cancers.

MIAMI BEACH – The remarkable progress seen with immune checkpoint inhibitors in metastatic melanoma, non–small-cell lung cancer, and other tumors has yet to be replicated in breast cancer, but it’s early days yet, and breast cancer researchers need more time before the ultimate clinical benefits of immunotherapy in breast cancer can be ascertained, said Adam M. Brufsky, MD, PhD, of the University of Pittsburgh.

Early studies with inhibitors of programmed death-1 (PD-1) and its ligand PD-L1 in patients with advanced triple-negative breast cancer have yielded only minimal response rates to date, but it it’s far too early to give up on the concept, Dr. Brufsky cautioned at the annual Miami Breast Cancer Conference, held by Physicians’ Education Resource.

In a video interview, he discussed the challenges of treating breast cancers, which may be less immunogenic and have a lower tumor mutational burden than other malignancies that respond more readily to PD-1 inhibition. Several large, phase III clinical trials of checkpoint inhibitors combined with cytotoxic chemotherapy are underway, he said, and those eventual findings may shed light on the optimal approach to using immunotherapy to treat patients with refractory metastatic breast cancers.

MIAMI BEACH – Driven by efficacy demonstrated in ovarian cancer, a number of PARP inhibitors are in development and hold promise for treatment of breast cancer as well, including patients positive for the BRCA mutation.

Interestingly, early evidence suggests these agents could also treat BRCA-negative women, potentially expanding their future clinical utility, Kimberly L. Blackwell, MD, said in a video interview at the annual Miami Breast Cancer Conference, held by Physicians’ Education Resource.

MIAMI BEACH – Driven by efficacy demonstrated in ovarian cancer, a number of PARP inhibitors are in development and hold promise for treatment of breast cancer as well, including patients positive for the BRCA mutation.

Interestingly, early evidence suggests these agents could also treat BRCA-negative women, potentially expanding their future clinical utility, Kimberly L. Blackwell, MD, said in a video interview at the annual Miami Breast Cancer Conference, held by Physicians’ Education Resource.

MIAMI BEACH – Driven by efficacy demonstrated in ovarian cancer, a number of PARP inhibitors are in development and hold promise for treatment of breast cancer as well, including patients positive for the BRCA mutation.

Interestingly, early evidence suggests these agents could also treat BRCA-negative women, potentially expanding their future clinical utility, Kimberly L. Blackwell, MD, said in a video interview at the annual Miami Breast Cancer Conference, held by Physicians’ Education Resource.

NATIONAL HARBOR, MD. – Patients with early cervical cancer with negative sentinel lymph node biopsy who did not receive pelvic lymphadenectomy experienced less postsurgical morbidity than patients who underwent lymphadenectomy, with no drop in overall or recurrence-free survival, investigators report.

In SENTICOL2, a 30-center prospective randomized study that compared sentinel lymph node (SLN) biopsy alone with SLN biopsy plus pelvic lymph node dissection for patients with early cervical cancer, recurrence-free survival at 3 years was 92% for patients receiving SLN only, compared with 95% in patients who had pelvic node dissection.

Overall survival at 3 years was 98% for the SLN-only patients, compared with 99% for those who had pelvic node dissection. None of these differences were statistically significant, and there were no significant differences between the study arms’ non-censored progression-free and overall survival rates at 6 years, Patrice Mathevet, MD, reported at the annual meeting of the Society of Gynecologic Oncology.

The study’s primary objective, said Dr. Mathevet, professor of obstetrics and gynecology at the University of Lausanne, Switzerland, was to compare the surgical morbidity experienced in each study arm, including quality of life and lymphedema–related signs and symptoms. A secondary objective was to assess and compare both overall survival and the 3-year recurrence-free rate for each study arm.

Patients were included if they had cervical cancer at International Federation of Gynecology and Obstetrics stage IA, IB1, or IIA1, with no sign of regional or distant metastases. Pregnant patients were excluded.

SLN scintigraphy was performed using the combined technetium and patent blue dye method. Patients with no identified or unilateral SLNs were excluded from the study and underwent pelvic lymph node dissection, as were patients in whom SLN scintigraphy was not performed.

Once SLNs were identified for the remaining patients, any patients with SLN metastases found on frozen section were excluded as well, while patients with grossly non-suspicious nodes and those with negative nodes on frozen section were randomized to receive just SLN biopsy with or without hysterectomy, or to receive pelvic lymph node dissection with or without hysterectomy.

SLNs in both study arms were examined by 200 micrometer serial sectioning with immunohistochemistry. For the pelvic node dissection arm, the nodes were submitted for pathology examination according to the study institution’s standard of care.

Over a 40-month period from March 2009 to July 2012, 267 patients with early cervical cancer were recruited. In the end, 206 patients were included in the study and 61 patients with positive or absent SLN results became ineligible according to the protocol criteria.

Of the eligible patients, 105 were randomized to the SLN biopsy–only arm, while 101 were allocated to the SLN biopsy plus pelvic lymph node dissection arm. Taking both study arms together, the median number of SLNs taken was three per patient, with a median one node per side.

When Dr. Mathevet and his colleagues looked at pathology from the pelvic lymph node dissection arm, they found that there were no false negatives, meaning that all patients with negative SLNs also had negative pelvic nodes.

Patient quality of life as assessed by the Short Form Health Survey (SF-36) was significantly lower for patients who received pelvic lymph node dissection. Lower extremity lymphedema signs and symptoms were also more common in patients who had pelvic lymph node dissection, indicated by the larger mean mid-thigh circumference and more common patient-reported leg heaviness and leg fatigue in the pelvic node dissection group.

The lymphedema findings were encapsulated in a global lymphatic morbidity score of 33 for the SLN-only group, compared with 52 for the pelvic node dissection group (P = .0046).

“Sentinel lymph node biopsy may improve the management of early cervical cancer, as sentinel lymph node biopsy alone induced less surgical morbidity than full pelvic lymph node dissection,” said Dr. Mathevet.

Patients were followed for a mean of 51 months post-procedure, with a total of 16 patients lost to follow-up. Patients in the SLN-only arm had a total of 11 recurrences, of which 5 were metastatic, 4 were local, and 2 were pelvic nodal. One of the pelvic nodal recurrences led to the patient’s death.

In the patients receiving pelvic node dissection, there were four metastatic and two local recurrences, but the difference in recurrence rates between the two groups was not statistically significant.

“This study leads to the morbidity-sparing approach in cervical cancer treatment while omitting the full pelvic lymph node dissection if the sentinel lymph nodes are negative,” said Dr. Mathevet.

Dr. Mathevet reported no relevant conflicts of interest.

[email protected]

On Twitter @karioakes

NATIONAL HARBOR, MD. – Patients with early cervical cancer with negative sentinel lymph node biopsy who did not receive pelvic lymphadenectomy experienced less postsurgical morbidity than patients who underwent lymphadenectomy, with no drop in overall or recurrence-free survival, investigators report.

In SENTICOL2, a 30-center prospective randomized study that compared sentinel lymph node (SLN) biopsy alone with SLN biopsy plus pelvic lymph node dissection for patients with early cervical cancer, recurrence-free survival at 3 years was 92% for patients receiving SLN only, compared with 95% in patients who had pelvic node dissection.

Overall survival at 3 years was 98% for the SLN-only patients, compared with 99% for those who had pelvic node dissection. None of these differences were statistically significant, and there were no significant differences between the study arms’ non-censored progression-free and overall survival rates at 6 years, Patrice Mathevet, MD, reported at the annual meeting of the Society of Gynecologic Oncology.

The study’s primary objective, said Dr. Mathevet, professor of obstetrics and gynecology at the University of Lausanne, Switzerland, was to compare the surgical morbidity experienced in each study arm, including quality of life and lymphedema–related signs and symptoms. A secondary objective was to assess and compare both overall survival and the 3-year recurrence-free rate for each study arm.

Patients were included if they had cervical cancer at International Federation of Gynecology and Obstetrics stage IA, IB1, or IIA1, with no sign of regional or distant metastases. Pregnant patients were excluded.

SLN scintigraphy was performed using the combined technetium and patent blue dye method. Patients with no identified or unilateral SLNs were excluded from the study and underwent pelvic lymph node dissection, as were patients in whom SLN scintigraphy was not performed.

Once SLNs were identified for the remaining patients, any patients with SLN metastases found on frozen section were excluded as well, while patients with grossly non-suspicious nodes and those with negative nodes on frozen section were randomized to receive just SLN biopsy with or without hysterectomy, or to receive pelvic lymph node dissection with or without hysterectomy.

SLNs in both study arms were examined by 200 micrometer serial sectioning with immunohistochemistry. For the pelvic node dissection arm, the nodes were submitted for pathology examination according to the study institution’s standard of care.

Over a 40-month period from March 2009 to July 2012, 267 patients with early cervical cancer were recruited. In the end, 206 patients were included in the study and 61 patients with positive or absent SLN results became ineligible according to the protocol criteria.

Of the eligible patients, 105 were randomized to the SLN biopsy–only arm, while 101 were allocated to the SLN biopsy plus pelvic lymph node dissection arm. Taking both study arms together, the median number of SLNs taken was three per patient, with a median one node per side.

When Dr. Mathevet and his colleagues looked at pathology from the pelvic lymph node dissection arm, they found that there were no false negatives, meaning that all patients with negative SLNs also had negative pelvic nodes.

Patient quality of life as assessed by the Short Form Health Survey (SF-36) was significantly lower for patients who received pelvic lymph node dissection. Lower extremity lymphedema signs and symptoms were also more common in patients who had pelvic lymph node dissection, indicated by the larger mean mid-thigh circumference and more common patient-reported leg heaviness and leg fatigue in the pelvic node dissection group.

The lymphedema findings were encapsulated in a global lymphatic morbidity score of 33 for the SLN-only group, compared with 52 for the pelvic node dissection group (P = .0046).

“Sentinel lymph node biopsy may improve the management of early cervical cancer, as sentinel lymph node biopsy alone induced less surgical morbidity than full pelvic lymph node dissection,” said Dr. Mathevet.

Patients were followed for a mean of 51 months post-procedure, with a total of 16 patients lost to follow-up. Patients in the SLN-only arm had a total of 11 recurrences, of which 5 were metastatic, 4 were local, and 2 were pelvic nodal. One of the pelvic nodal recurrences led to the patient’s death.

In the patients receiving pelvic node dissection, there were four metastatic and two local recurrences, but the difference in recurrence rates between the two groups was not statistically significant.

“This study leads to the morbidity-sparing approach in cervical cancer treatment while omitting the full pelvic lymph node dissection if the sentinel lymph nodes are negative,” said Dr. Mathevet.

Dr. Mathevet reported no relevant conflicts of interest.

[email protected]

On Twitter @karioakes

NATIONAL HARBOR, MD. – Patients with early cervical cancer with negative sentinel lymph node biopsy who did not receive pelvic lymphadenectomy experienced less postsurgical morbidity than patients who underwent lymphadenectomy, with no drop in overall or recurrence-free survival, investigators report.

In SENTICOL2, a 30-center prospective randomized study that compared sentinel lymph node (SLN) biopsy alone with SLN biopsy plus pelvic lymph node dissection for patients with early cervical cancer, recurrence-free survival at 3 years was 92% for patients receiving SLN only, compared with 95% in patients who had pelvic node dissection.

Overall survival at 3 years was 98% for the SLN-only patients, compared with 99% for those who had pelvic node dissection. None of these differences were statistically significant, and there were no significant differences between the study arms’ non-censored progression-free and overall survival rates at 6 years, Patrice Mathevet, MD, reported at the annual meeting of the Society of Gynecologic Oncology.

The study’s primary objective, said Dr. Mathevet, professor of obstetrics and gynecology at the University of Lausanne, Switzerland, was to compare the surgical morbidity experienced in each study arm, including quality of life and lymphedema–related signs and symptoms. A secondary objective was to assess and compare both overall survival and the 3-year recurrence-free rate for each study arm.

Patients were included if they had cervical cancer at International Federation of Gynecology and Obstetrics stage IA, IB1, or IIA1, with no sign of regional or distant metastases. Pregnant patients were excluded.

SLN scintigraphy was performed using the combined technetium and patent blue dye method. Patients with no identified or unilateral SLNs were excluded from the study and underwent pelvic lymph node dissection, as were patients in whom SLN scintigraphy was not performed.

Once SLNs were identified for the remaining patients, any patients with SLN metastases found on frozen section were excluded as well, while patients with grossly non-suspicious nodes and those with negative nodes on frozen section were randomized to receive just SLN biopsy with or without hysterectomy, or to receive pelvic lymph node dissection with or without hysterectomy.

SLNs in both study arms were examined by 200 micrometer serial sectioning with immunohistochemistry. For the pelvic node dissection arm, the nodes were submitted for pathology examination according to the study institution’s standard of care.

Over a 40-month period from March 2009 to July 2012, 267 patients with early cervical cancer were recruited. In the end, 206 patients were included in the study and 61 patients with positive or absent SLN results became ineligible according to the protocol criteria.

Of the eligible patients, 105 were randomized to the SLN biopsy–only arm, while 101 were allocated to the SLN biopsy plus pelvic lymph node dissection arm. Taking both study arms together, the median number of SLNs taken was three per patient, with a median one node per side.

When Dr. Mathevet and his colleagues looked at pathology from the pelvic lymph node dissection arm, they found that there were no false negatives, meaning that all patients with negative SLNs also had negative pelvic nodes.

Patient quality of life as assessed by the Short Form Health Survey (SF-36) was significantly lower for patients who received pelvic lymph node dissection. Lower extremity lymphedema signs and symptoms were also more common in patients who had pelvic lymph node dissection, indicated by the larger mean mid-thigh circumference and more common patient-reported leg heaviness and leg fatigue in the pelvic node dissection group.

The lymphedema findings were encapsulated in a global lymphatic morbidity score of 33 for the SLN-only group, compared with 52 for the pelvic node dissection group (P = .0046).

“Sentinel lymph node biopsy may improve the management of early cervical cancer, as sentinel lymph node biopsy alone induced less surgical morbidity than full pelvic lymph node dissection,” said Dr. Mathevet.

Patients were followed for a mean of 51 months post-procedure, with a total of 16 patients lost to follow-up. Patients in the SLN-only arm had a total of 11 recurrences, of which 5 were metastatic, 4 were local, and 2 were pelvic nodal. One of the pelvic nodal recurrences led to the patient’s death.

In the patients receiving pelvic node dissection, there were four metastatic and two local recurrences, but the difference in recurrence rates between the two groups was not statistically significant.

“This study leads to the morbidity-sparing approach in cervical cancer treatment while omitting the full pelvic lymph node dissection if the sentinel lymph nodes are negative,” said Dr. Mathevet.

Dr. Mathevet reported no relevant conflicts of interest.

[email protected]

On Twitter @karioakes

Photo by Linda Bartlett

The US Food and Drug Administration (FDA) has granted orphan drug designation for BI 836858, an anti-CD33 monoclonal antibody (mAb), in the treatment of myelodysplastic syndromes (MDS).

BI 836858 previously received orphan designation for the treatment of acute myeloid leukemia (AML).

The FDA grants orphan designation to drugs and biologics intended to treat, diagnose, or prevent rare diseases/disorders affecting fewer than 200,000 people in the US.

Orphan designation provides companies with certain incentives to develop products for rare diseases.

This includes a 50% tax break on research and development, a fee waiver, access to federal grants, and 7 years of market exclusivity if the product is approved.

About BI 836858

BI 836858 is a fully human, immunoglobulin G1 anti-CD33 mAb. It has been engineered for improved binding to FcgRIIIa to mediate natural killer cell antibody-dependent cellular cytotoxicity against CD33-expressing tumor cells.

BI 836858 is being developed by Boehringer Ingelheim.

A phase 1/2 trial (NCT02240706) of BI 836858 in patients with MDS is ongoing. The phase 1 portion was designed to evaluate various doses of the mAb in patients with low or intermediate-1 risk MDS with symptomatic anemia.

The phase 2 portion was designed to compare BI 836858 plus best supportive care to best supportive care alone in patients with low- or intermediate-1-risk MDS who have symptomatic anemia but do not have a 5q deletion.

BI 836858 is also being tested in combination with decitabine in a phase 1/2 study (NCT02632721) of patients with AML.

The goals of the phase 1 portion and the phase 1 extension are to determine the maximum-tolerated dose/recommended dose, safety, pharmacokinetics, and efficacy of BI 836858 in combination with decitabine.

The goals of the phase 2 portion of the study are to investigate the efficacy, safety, and pharmacokinetics of BI 836858 in combination with decitabine compared to decitabine monotherapy.

BI 836858 was previously evaluated in combination with decitabine in a preclinical study. The combination exhibited activity against AML in vitro. The research was published in Blood last year.

BI 836858 is also being evaluated as part of the Leukemia & Lymphoma Society’s Beat AML Master Trial program to advance treatment for patients with AML.

In this trial, investigators are using genomic technology to identify AML mutations in newly diagnosed patients over the age of 60 and match the patients with an investigational drug or drugs best suited to attack the mutations found.

Photo by Linda Bartlett

The US Food and Drug Administration (FDA) has granted orphan drug designation for BI 836858, an anti-CD33 monoclonal antibody (mAb), in the treatment of myelodysplastic syndromes (MDS).

BI 836858 previously received orphan designation for the treatment of acute myeloid leukemia (AML).

The FDA grants orphan designation to drugs and biologics intended to treat, diagnose, or prevent rare diseases/disorders affecting fewer than 200,000 people in the US.

Orphan designation provides companies with certain incentives to develop products for rare diseases.

This includes a 50% tax break on research and development, a fee waiver, access to federal grants, and 7 years of market exclusivity if the product is approved.

About BI 836858

BI 836858 is a fully human, immunoglobulin G1 anti-CD33 mAb. It has been engineered for improved binding to FcgRIIIa to mediate natural killer cell antibody-dependent cellular cytotoxicity against CD33-expressing tumor cells.

BI 836858 is being developed by Boehringer Ingelheim.

A phase 1/2 trial (NCT02240706) of BI 836858 in patients with MDS is ongoing. The phase 1 portion was designed to evaluate various doses of the mAb in patients with low or intermediate-1 risk MDS with symptomatic anemia.

The phase 2 portion was designed to compare BI 836858 plus best supportive care to best supportive care alone in patients with low- or intermediate-1-risk MDS who have symptomatic anemia but do not have a 5q deletion.

BI 836858 is also being tested in combination with decitabine in a phase 1/2 study (NCT02632721) of patients with AML.

The goals of the phase 1 portion and the phase 1 extension are to determine the maximum-tolerated dose/recommended dose, safety, pharmacokinetics, and efficacy of BI 836858 in combination with decitabine.

The goals of the phase 2 portion of the study are to investigate the efficacy, safety, and pharmacokinetics of BI 836858 in combination with decitabine compared to decitabine monotherapy.

BI 836858 was previously evaluated in combination with decitabine in a preclinical study. The combination exhibited activity against AML in vitro. The research was published in Blood last year.

BI 836858 is also being evaluated as part of the Leukemia & Lymphoma Society’s Beat AML Master Trial program to advance treatment for patients with AML.

In this trial, investigators are using genomic technology to identify AML mutations in newly diagnosed patients over the age of 60 and match the patients with an investigational drug or drugs best suited to attack the mutations found.

Photo by Linda Bartlett

The US Food and Drug Administration (FDA) has granted orphan drug designation for BI 836858, an anti-CD33 monoclonal antibody (mAb), in the treatment of myelodysplastic syndromes (MDS).

BI 836858 previously received orphan designation for the treatment of acute myeloid leukemia (AML).

The FDA grants orphan designation to drugs and biologics intended to treat, diagnose, or prevent rare diseases/disorders affecting fewer than 200,000 people in the US.

Orphan designation provides companies with certain incentives to develop products for rare diseases.

This includes a 50% tax break on research and development, a fee waiver, access to federal grants, and 7 years of market exclusivity if the product is approved.

About BI 836858

BI 836858 is a fully human, immunoglobulin G1 anti-CD33 mAb. It has been engineered for improved binding to FcgRIIIa to mediate natural killer cell antibody-dependent cellular cytotoxicity against CD33-expressing tumor cells.

BI 836858 is being developed by Boehringer Ingelheim.

A phase 1/2 trial (NCT02240706) of BI 836858 in patients with MDS is ongoing. The phase 1 portion was designed to evaluate various doses of the mAb in patients with low or intermediate-1 risk MDS with symptomatic anemia.

The phase 2 portion was designed to compare BI 836858 plus best supportive care to best supportive care alone in patients with low- or intermediate-1-risk MDS who have symptomatic anemia but do not have a 5q deletion.

BI 836858 is also being tested in combination with decitabine in a phase 1/2 study (NCT02632721) of patients with AML.

The goals of the phase 1 portion and the phase 1 extension are to determine the maximum-tolerated dose/recommended dose, safety, pharmacokinetics, and efficacy of BI 836858 in combination with decitabine.

The goals of the phase 2 portion of the study are to investigate the efficacy, safety, and pharmacokinetics of BI 836858 in combination with decitabine compared to decitabine monotherapy.

BI 836858 was previously evaluated in combination with decitabine in a preclinical study. The combination exhibited activity against AML in vitro. The research was published in Blood last year.

BI 836858 is also being evaluated as part of the Leukemia & Lymphoma Society’s Beat AML Master Trial program to advance treatment for patients with AML.

In this trial, investigators are using genomic technology to identify AML mutations in newly diagnosed patients over the age of 60 and match the patients with an investigational drug or drugs best suited to attack the mutations found.