Delving into the Latest Updates on Palifermin with Synapse

Background:
X-linked severe combined immunodeficiency (XSCID) is a rare inherited disorder that affects the immune system. It is caused by a change in the IL2RG gene. Researchers are investigating a new type of gene therapy for people with XSCID. This technique, called base-edited stem cell transplants, involves collecting a person s own stem cells, editing the genes to repair IL2RG gene, and returning the edited cells to the person.
Objective:
To test base-edited stem cell transplants in people with XSCID.
Eligibility:
People aged 3 years and older with XSCID.
Design:
Participants will be screened. They will have a physical exam. They may give blood, urine, and stool samples. They may have tests of their heart and lung function. They may have fluid and cells drawn from their bone marrow.
Participants will undergo apheresis. Blood will be taken from the body through a needle inserted into 1 arm. The blood will pass through a machine that separates out the stem cells. The remaining blood will be returned to the body through a different needle. The collected stem cells will undergo gene editing.
Participants will be admitted to the hospital 1 week before treatment. They will receive a central line: A flexible tube will be inserted into a large vein. This tube will be used to administer drugs and draw blood during their stay. They will receive drugs to prepare their bodies for the treatment.
The base-edited stem cells will be infused through the central line. Participants will remain in the hospital for at least 3 weeks while they recover.
Follow-up visits will continue for 15 years.

Start Date07 May 2025

Sponsor / Collaborator

National Institute of Allergy & Infectious Diseases

NCT06325709

/ SuspendedPhase 1/2IIT

Phase 1/2 Trial of Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-linked Chronic Granulomatous Disease

Background:
Chronic granulomatous disease (CGD) is a rare immune disorder caused by a mutation in the CYBB gene. People with CGD have white blood cells that do not work properly. This places them at risk of developing infections that may be life-threatening. Stem cell transplant can cure CGD but transplanting stem cells donated by other people can have serious complications. In addition, not everyone has a matched donor. Another approach is a type of gene therapy that involves base-editing to correct the mutation in a person s own stem cells. Researchers want to know if the base-edited stem cells can improve the white cells' functioning and result in fewer CGD-related infections.
Objective:
To learn if base-edited stem cells will improve white blood cells' ability to fight against infections in people with CGD.
Eligibility:
Males aged 18 years and older with X-linked CGD.
Design:
This is a non-randomized study. Participants with the specific mutation under study will be screened during the initial phase.
During the development phase, participants will undergo apheresis to collect stem cells for base-editing correction of the mutation.
During the treatment phase, participants will receive the base-edited cells after chemotherapy with busulfan. Participants will remain in the hospital until their immunity recovers.
Follow-up visits will continue for 15 years....

Start Date17 Apr 2024

Sponsor / Collaborator

National Institute of Allergy & Infectious Diseases

NCT04177004

/ RecruitingPhase 1IIT

A Randomized Pilot Study of Human Lysozyme Goat Milk in Recipients of Standard Myeloablative Allogeneic Hematopoietic Stem Cell Transplantation

This phase I trial studies the side effects of human lysozyme goat milk in preventing graft versus host disease in patients with blood cancer undergoing a donor stem cell transplant. Sometimes the transplanted cells from a donor can cause an immune response against the body's own normal cells (call graft versus host disease). The goat milk in the study is from goats that have been genetically engineered to produce human lysozyme in the milk. Human lysozyme is a natural enzyme found in human milk and acts as an antimicrobial. Lysozyme is key to the digestive health of breast-fed human infants, since it helps the growth of beneficial gut bacteria and reduces the growth of bacteria that causes diarrhea and intestinal disease. Giving human lysozyme goat milk may reduce the rate of graft versus host disease in blood cancer patients undergoing a donor stem cell transplant.

Start Date30 Apr 2021

Sponsor / Collaborator

City of Hope National Medical Center

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100 Clinical Results associated with Palifermin

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100 Translational Medicine associated with Palifermin

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100 Patents (Medical) associated with Palifermin

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752

Literatures (Medical) associated with Palifermin

01 Mar 2025·Cellular Signalling

KGF impedes TRIM21-enhanced stabilization of keratin 10 mediating differentiation in hypopharyngeal cancer

Article

Author: Wang, Guangyi ; Chai, Fangyu ; Huang, Yichuan ; Niu, Yanfang ; Yang, Tao ; Jiang, Yan ; Shen, Yibang ; Fu, Tao ; Zhang, Jisheng

KGF, also known as FGF7, is a member of the fibroblast growth factor (FGF) family that binds with high affinity to the FGF receptor 2b (FGFR2b) and regulates various cellular processes, including cell proliferation and differentiation in a variety of tumors. However, its potential role in hypopharyngeal cancer (HPC) remains largely unknown. In our study, we observed increased expression of FGFR2b in HPC. KGF treatment inhibited the expression of the differentiation marker keratin 10 (K10) protein at the post-transcriptional level in FaDu cells. Furthermore, treatment with the proteasome inhibitor MG132 was found to attenuate KGF-induced K10 reduction, suggesting the involvement of the ubiquitin-proteasome system. Using mass spectrometry and immunoprecipitation analysis, we identified the E3 ubiquitin ligase TRIM21 as a K10-interacting protein. Unexpectedly, instead of causing degradation, TRIM21 enhanced K10 protein stability through K6-linked ubiquitination of K10 at lysine 163 (K163) in the context of KGF exposure. Meanwhile, KGF treatment decreased TRIM21 protein levels, which were regulated by the p38 MAPK pathway, leading to K48-linked ubiquitination-mediated degradation of TRIM21. Notably, TRIM21 knockdown significantly promoted proliferation, inhibited differentiation and migration of FaDu cells, whereas TRIM21 overexpression had opposite effects in vitro and suppressed xenograft tumor growth in vivo. Our study demonstrates that TRIM21 may act as a tumor suppressor in HPC. However, TRIM21 overexpression decreased the sensitivity of FaDu cells to 5-fluorouracil, whereas TRIM21 knockdown or KGF administration significantly increased 5-fluorouracil sensitivity. Taken together, these findings highlight the intricate balance between protein stabilization and degradation orchestrated by KGF. This ubiquitination-mediated non-degradation mechanism of TRIM21 may provide novel therapeutic strategies for HPC and other cancers.

01 Feb 2025·Cancer Treatment Reviews

Managing strategies of chemotherapy and radiotherapy-induced oral mucositis

Review

Author: Wu, Yuqi ; Qiu, Yan ; Shi, Wenjin ; Luo, Xiaobo ; Chen, Qianming ; Li, Chunyu ; Jiang, Yuchen ; Liu, Xiangfei

Radiotherapy and chemotherapy are widely employed as primary non-surgical cancer treatments; however, their non-selective cytotoxicity often leads to adverse events such as oral mucositis (OM), particularly in head and neck cancer therapies. International guidelines provide recommendations for managing chemoradiotherapy-induced OM in various clinical contexts. Subsequently, emerging researches have introduced evidence supporting novel approaches or existing regimens for OM prevention and treatment. The repurposing of established drugs has garnered significant interest due to its shorter development timeline, improved safety profiles, and lower costs compared to new drug development. For example, clinical trials assessing established drugs such as melatonin, clonidine, and pentoxifylline indicate promising potential for managing OM. Additionally, several emerging pharmacological interventions have demonstrated considerable efficacy; SAMITAL and rhIL-11 are supported by phase II clinical trials and prospective studies, while probiotics like Streptococcus salivarius K12 and curcumin have shown effectiveness in randomized clinical trials. Furthermore, recent high-level studies have reinforced the efficacy of non-pharmacological interventions, such as photobiomodulation (PBM) and cryotherapy, over the past two years. In all, given the evidence supporting different strategies, PBM and oral cryotherapy are highly recommended for managing OM when feasible. Topical clonidine, melatonin, oral pentoxifylline, topical SAMITAL or rhIL-11, oral SsK12, and curcumin may also be utilized but would benefit from validation in larger trials. Besides, Verbascoside, Palifermin, Amifostine, and Avasopasem manganese can be suggested for OM management, while the side effects should be monitored. The accessibility and cost/effectiveness of specific managing strategies of OM should be considered when selecting appropriate options.

01 Jan 2025·IUBMB Life

KGF secreted from HSCs activates PAK4/BMI1, promotes HCC stemness through PI3K/AKT pathway

Article

Author: Li, Zhen ; Chen, Qiuyang ; Li, Qinghua ; Xie, Rongrong ; Chen, Dawei ; Wang, Wenchao

AbstractIn our present study, we investigated the interaction between HSCs and HCC, also explored the molecular mechanism. Clinical samples were collected from HCC and adjacent tissue with different degree of liver fibrosis. HCC cells were co‐cultured with LX‐2 cell by Transwell system or cultured with conditioned medium (CM), which was collected from LX‐2. The tumor spheroid growth and colony formation analyses were performed to evaluate the cell stemness. Flow cytometry analysis was conducted on cell apoptosis after 5‐Fu treatment. Co‐immunoprecipitation assay confirmed the interaction between BMI1 and PAK4. Our results showed that BMI1 was highly expressed in HCC and was correlated with HCC liver fibrosis. Both co‐cultured with LX‐2 and cultured with CM promoted HCC stemness, also increased KGF level and BMI1 expression. KGF treatment had a similar effect with co‐culture with LX‐2 on HCC. BMI1 overexpression promoted HCC stemness and activated PI3K/AKT pathway, which was reversed by PI3K inhibition. PAK4 was activated by KGF, then phosphorylated S315 site and promoted protein stability of BMI1, therefore enhanced HCC stemness. BMI1 also had a promote effect on liver fibrosis. In summary, we found that KGF secreted by HSCs activated PAK4, which phosphorylated S315 and promoted protein stability of BMI1, and further promoted liver fibrosis and HCC stemness through the PI3K/AKT signaling pathway. Our present study deeply studied the interaction and mechanism between HSCs and HCC, which might provide a new insight for HCC therapy.

100 Deals associated with Palifermin

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External Link

KEGG	Wiki	ATC	Drug Bank
D05338	Palifermin	V03AF08	DB00039

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Stomatitis	United States	Swedish Orphan Biovitrum AB	15 Dec 2004

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Non-Hodgkin Lymphoma	Phase 2	-	Amgen, Inc.	15 Jul 2002
Stomatitis	Preclinical	European Union	Swedish Orphan Biovitrum AB	25 Oct 2005
Stomatitis	Preclinical	Iceland	Swedish Orphan Biovitrum AB	25 Oct 2005
Stomatitis	Preclinical	Norway	Swedish Orphan Biovitrum AB	25 Oct 2005
Stomatitis	Preclinical	Liechtenstein	Swedish Orphan Biovitrum AB	25 Oct 2005
Hematologic Neoplasms	Preclinical	-	Amgen, Inc.	15 Jul 2002

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


Tandem Meetings ASTCT and CIBMTR2023	Not Applicable	Graft vs Host Disease	-	Palifermin 540 µg/kg	ehssisjrpf(xpdqohbvuo) = pwlvxnqedd fmqxbnhmmj (outriwbzxs ) View more	-	01 Feb 2023
				TMS (tacrolimus, methotrexate, sirolimus)	damjkaqsit(jpnscyloly) = ucjxmblztd svfyixvdie (atpzpfiuzw ) View more
EBMT2020	Not Applicable	Lymphoma	73	Palifermin + BEAM conditioning	(gkrzjpfcfh) = ipioonttub fxpkjezbac (jshkojeomx ) View more	-	29 Aug 2020
				BEAM conditioning	(gkrzjpfcfh) = dktrouujmi fxpkjezbac (jshkojeomx ) View more
Tandem Meetings ASTCT and CIBMTR2020	Not Applicable	Hematopoietic stem cell transplantation	44	Palifermin	kktxhcqgxm(phddyrsolo) = zbnesshrnp cnbkpvbsdo (rfhtfojqja, 7 - 16) View more	Negative	01 Mar 2020
NCT02037347 (CTgov)	Phase 1/2	Stevens-Johnson Syndrome	1	Palifermin	awhucyzzov(mcpdvqxraz) = ebflrjnxbk vfoiwtcbwm (fwukamchih, bcgzmgjbmf - lzgcomixjf) View more	-	30 Nov 2016
NCT00109031 (CTgov)	Phase 3	Leukemia \| Stomatitis \| Lymphoma	47	Placebo+Etoposide+cyclophosphamide+palifermin (Palifermin 60 µg/kg for 3 Days)	gqnendfipu(zzbxprjrje) = oaplvxyoub tgciyaljap (uxgrbjukro, xgeljtrpuf - avxzirbqsy) View more	-	15 Sep 2014
				Placebo+Etoposide+cyclophosphamide+palifermin (Palifermin 180 μg/kg on Day -1)	gqnendfipu(zzbxprjrje) = vrbjnkmvoq tgciyaljap (uxgrbjukro, cysfqnfyuo - ieyvffdlin) View more
NCT00101582 (CTgov)	Phase 3	Mucositis \| Stomatitis \| Head and Neck Neoplasms \| Squamous Cell Carcinoma	188	Radiotherapy+cisplatin chemotherapy (Placebo)	tpkpddbsko(wlyquyferd) = bqrcrzfefh yxhlbdowki (jarlohmsmf, lkeaaqqclc - snlyxaweti) View more	-	15 Sep 2014
				Radiotherapy+palifermin+cisplatin chemotherapy (Palifermin)	tpkpddbsko(wlyquyferd) = lzpleqnhzl yxhlbdowki (jarlohmsmf, gctujysjzb - zersoxpzai) View more
ASCO2014	Not Applicable	Acute Lymphoblastic Leukemia \| Solid tumor	816	Palifermin	(sahojjarvs) = uanzjjbcfe ltpwsejmpu (creoteolip ) View more	-	20 May 2014
				No Palifermin	(sahojjarvs) = xukicivess ltpwsejmpu (creoteolip ) View more
NCT00360971 (CTgov)	Phase 3	Radiation Injuries \| Pain \| Locally Advanced Malignant Neoplasm \| Mucositis \| Stomatitis \| Head and Neck Neoplasms	21	placebo+cisplatin (Placebo)	qgtrktuddr(ecqeiscqyz) = wzwsiiymjs hockusfwsp (gnnupieoco, hajeetnbde - pxmyuilcpr) View more	-	15 May 2014
				neck dissection+palifermin+cisplatin (Palifermin)	qgtrktuddr(ecqeiscqyz) = gsdunahcoi hockusfwsp (gnnupieoco, vtgsmvotqz - fhmfsxtele) View more
NCT01233921 (CTgov)	Not Applicable	Marginal zone lymphoma recurrent \| Burkitt Lymphoma \| BCR-ABL Negative Atypical Chronic Myeloid Leukemia \| Aggressive-Phase Chronic Myelocytic Leukemia \| Recurrent Hodgkin Lymphoma \| Secondary Myelodysplastic Syndrome \| Hairy Cell Leukemia \| Sezary Syndrome \| Chronic lymphocytic leukaemia refractory \| Chronic Eosinophilic Leukemia \| Small Lymphocytic Lymphoma \| Primary Myelofibrosis \| Adult Lymphoblastic Lymphoma \| Chronic phase chronic myeloid leukemia \| Recurrent Adult Acute Lymphoblastic Leukemia \| Adult acute myeloid leukemia with del(5q) \| Chronic Myelomonocytic Leukemia \| Adult Acute Lymphocytic Leukemia \| Diffuse Large B-Cell Lymphoma \| Blast Phase Chronic Granulocytic Leukemia \| Mantle cell lymphoma recurrent \| Graft vs Host Disease \| Acute Promyelocytic Leukemia \| Leukemia, Neutrophilic, Chronic \| Immunoblastic Large-Cell Lymphoma \| Recurrent Grade 3a Follicular Lymphoma \| Refractory Multiple Myeloma \| Stage II Non-Contiguous Adult Diffuse Small Cleaved Cell Lymphoma	6	flow cytometry+palifermin (Arm I (Palifermin))	lkqctikkbn(vbvxiwogxg) = jdtsoeefaf pizolryjzo (xoqkrzpkdd, hvrxekunly - pvjtsmgqlx) View more	-	02 Apr 2014
				flow cytometry (Arm II (no Palifermin))	lkqctikkbn(vbvxiwogxg) = xndcvpiyxt pizolryjzo (xoqkrzpkdd, zgcmpvegxm - vavhvqvlcy) View more
NCT00626639 (CTgov)	Phase 1/2	Locally Advanced Malignant Neoplasm \| Mucositis \| Stomatitis \| Locally Advanced Head and Neck Squamous Cell Carcinoma	5	Radiotherapy+Cisplatin (Placebo)	(aopaeuhrgk) = bcmoqnfioi odbfbppmgr (whugfpxzif, awxthhagdd - hxgsudqcyu) View more	-	06 Mar 2014
				Radiotherapy+palifermin+Cisplatin (Palifermin)	(aopaeuhrgk) = bpvqevubrg odbfbppmgr (whugfpxzif, nrgedzrswz - czbeeecqrv) View more

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Palifermin

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